Read the Report - Colorado BioScience Association

Transcription

The Last 60 Days in Precision Medicine:
5/26/16 -7/26/16
Copyright ©2016 by NewsRx.
Published by NewsRx, Atlanta, Georgia.
Limit of Liability/Disclaimer of Warranty: While the publisher and authors have used their best efforts in
preparing this report, they make no representations or warranties with respect to the accuracy or completeness
of the contents of this report and specifically disclaim any implied warranties of merchantability or fitness
for a particular purpose. No warranty may be created or extended by sales representatives or written sales
materials. The advice and strategies contained herein may not be suitable for your situation. You should
consult with a professional where appropriate. Neither the publisher nor the authors shall be liable for any
loss of profit or any other commercial damages, including but not limited to special, incidental, consequential,
or other damages.
This publication is designed to provide accurate and authoritative information in regard to the subject
matter covered. It is sold with the understanding that the publisher is not engaged in rendering professional
services. If legal, accounting, medical, psychological or any other expert assistance is required, the services of
a competent professional person should be sought.
For general information on other NewsRx products and services, please contact our Customer Services
department at NewsRx, 100 City View, Suite 125, 3330 Cumberland Blvd., Atlanta, GA 30339, USA, or call
1-800-726-4550 or 770-507-7777, or visit our website: http://www.newsrx.com/Butter/.
ii
Table of Contents
1
Biotechnology
. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
1
2
Cancer
. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
8
3
Cardiology . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 36
4
Clinical Trials and Studies . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43
5
Drugs and Therapies . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 49
6
Genetics
7
Government Agencies Offices and Entities . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 94
8
Hospital
9
Oncology . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 121
10
Additional Research
. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74
. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 104
. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 146
Index . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 206
iii
Chapter 1
Biotechnology
Forentis Partners, LLC
Forentis Fund Receives Commitment from Single Oak Ventures
By a News Reporter-Staff News Editor at Biotech Week – Forentis Partners, LLC has announced that Forentis
Fund, a $50 million special purpose venture fund focused on biotechnology and precision medicine, has received
its first institutional investment commitment. The fund has received a commitment letter from Single Oak
Ventures for $5 million.
“This is a great start for our fund,” stated Jay Goth, managing member. “I have worked with the principals
of Single Oak in the past and their confidence in us is highly appreciated.”
“The approach that Forentis is taking is very appealing,” said Ralph Williams, founding partner at Single
Oak. “They have taken a true hands-on approach to working with the companies they plan to invest in, and in
discussions with Forentis and their target companies we come away with a high degree of appreciation for all
of the work that they have done so far and see that they have great opportunities ahead.”
Forentis Fund is dedicated to bridging the gap between biotechnology innovations and suffering patients.
By funding the companies that are building an ecosystem of biological discovery, advanced predictive analytics
and rapid commercialization processes that can effectively reduce the time to bring solutions to market and the
price associated with them, the fund becomes the financial catalyst for critical medical innovation.
The commitment from Single Oak is subject to the completion of its current funding round and traditional
due diligence which has begun. The two firms anticipate closing the transaction this summer. About Single Oak
Ventures Single Oak Ventures is a fund focused on bridging the gap between higher risk, network dependent
angel investing and the high minimum, large funds typically associated with traditional venture capital firms.
Single Oak is managed by an experienced team of industry leaders with extensive backgrounds in venture
capital, finance, securities law, sales, marketing and executive management. Single Oak Ventures is one of
a new generation of funds which provides access to top companies and industry veterans while employing a
uniquely diversified investment strategy. Their Three Strategy-One Fund approach to investing allows for a
diversified asset allocation within the fund and provides the opportunity to take an opportunistic approach
to investing across a wide array of technologies. About Forentis Fund Forentis Fund is in the business of
saving lives and returning reason to the pharmaceutical and diagnostic industries. The fund is strategically
located at the Murrieta Innovation Center in Murrieta, California, providing it with access to the leading
biotechnology research centers of Los Angeles, San Diego and Orange County. The fund is within easy driving
distance of UCSD, UCR, UCI, UCLA and USC. Forentis Fund is available only to accredited investors. More
information on the fund and its investment approach can be found at www.forentis.com. View source version
on businesswire.com: http://www.businesswire.com/news/home/20160609006425/en/
Our reports deliver fact-based news of research and discoveries from around the world. Copyright 2016,
NewsRx LLC
(2016-06-22), Forentis Fund Receives Commitment from Single Oak Ventures, Biotech Week, 116, ISSN:
1537-4699, BUTTER® ID: 011930546
1
Biotechnology
Kazakhstan weighs life sciences and precision medicine as a new economic sector
By a News Reporter-Staff News Editor at Biotech Business Week – The economic value of integrating healthy
life-extension technologies into national healthcare systems is undeniable, resulting in increased population
productivity, healthier citizens and growth of novel industries. Billions of dollars can be saved through a decreased and delayed burden on the pension system and on healthcare spending for the treatment of age-related
diseases.
The Biogerontology Research Foundation (BGRF) is working with the UK’s Centre for the Advancement
of Sustainable Medical Innovation (CASMI), alongside a team drawn from the Kazakhstan government,
Nazarbayev University and other academic leaders to establish the potential for a Precision & Personalised
Medicine Initiative in Kazakhstan.
“Our ambition is to establish a global translational platform for biotech companies to accelerate R&D and
commercialisation processes of innovative technologies to treat biological ageing and age-related diseases. We
have already started extensive work to streamline the regulatory environment, create advanced clinical trial
capabilities and advanced therapy manufacturing facilities and form a pool of potential biotech partners” said
Avi Roy, President of BGRF.
Kazakhstan can be considered a role model of government ambition and willpower by adopting innovations
and cutting-edge biomedical technologies a key driver for economic growth.
The country has already invested significantly in the creation of an academic and industrial base by establishing one of the most advanced universities in Central Asia, Nazarbayev University; the National Medical
Holding which consists of 6 specialised medical centers with state of the art equipment and highly qualified
staff, the National Institute for Biotechnology and the Astana Business Campus, which promises to become a
Silicon Valley for progressive biomedical technologies.
“The program we are developing is first of its kind, but may serve as a model for other countries that are
committed to innovation and technological progress. The idea is simple: identify the most promising technologies required to extend healthy productive longevity for the entire population to keep people healthier and more
productive for as long as it is possible. To do that we partnered with the best minds from all over the world and
conducted thorough analytical studies”, said Zhaxybay Zhumadilov, General Director of the National Laboratory Astana, Nazarbayev University.
So far two joint workshops have been completed, one in Oxford and another in Astana at the Nazarbayev
University, coinciding with the 4th International Conference on Regenerative Medicine and Healthy Aging. At
this second workshop the first results of the scoping study were presented and reviewed by prominent scientists, industry players and policy makers including the head of CASMI Prof. Richard Barker, former director
of the Human Genome Project, Prof. Charles Cantor, president of Nazarbaev University, Shigeo Katsu, general director of Nazarbayev University, Prof. Zhaxybay Zhumadilov, dean of School of Medicine of Nazarbayev
University and dean of School of Medicine of Nazarbayev University, Prof. Massimo Pignatelli.
NewsRx LLC
(2016-06-13), Kazakhstan weighs life sciences and precision medicine as a new economic sector, Biotech
Business Week, 75, ISSN: 1543-6861, BUTTER® ID: 011822387
Biotechnology Innovation Organization
Missouri Governor Nixon Signs Bill Ensuring Patient Access to Interchangeable Biologic
Medicines
By a News Reporter-Staff News Editor at Biotech Week – The Biotechnology Innovation Organization (BIO)
and Missouri Biotechnology Association (MOBIO) commend Governor Jay Nixon for signing critical legislation
to create a pathway for the substitution of interchangeable biologic medicines.
Governor Nixon signed Senate Bill 875 this week, following recent passage in both the Missouri Senate and
House of Representatives. The policies outlined in the bill align with BIO’s principles on biologic substitution, therefore BIO and MOBIO support this important legislation and are grateful to Governor Nixon for his
leadership on this issue.
“Senate Bill 875 enjoys the support of physicians across Missouri and the country, patient groups, and both
innovator biologic and biosimilar manufacturing companies. This bill includes communication on all biologic
medicines dispensed in order to maintain a consistent and complete medical record,” said Jim Greenwood, BIO’s
2
President and Chief Executive Officer. “By signing this bill into law, Governor Nixon has added Missouri to the
list of states that allow retail pharmacies to substitute interchangeable biologic medicines.”
While the U.S. Food and Drug Administration (FDA) oversees approval of biologic medicines and designation of interchangeability, policies governing whether one product may be substituted in place of a doctor’s
prescription and whether a pharmacist must inform patients and doctors are covered by state law. Senate
Bill 875 seeks to properly preserve patient access to accurate prescription information, maintain incentives
for innovation and promote a competitive market for biologic therapies. BIO will continue to advocate for full
communication in the substitution process, as patients and their physicians should have the right to know what
biologic medicine the patient receives from the pharmacy.
“Large molecule biologics are the future of precision medicine. In America alone, more than 900 biologics
are in development, for more than 100 diseases. By signing this legislation, Governor Nixon will be opening
the door to long-term savings for Missouri’s citizens, and almost immediate savings to the state budget,” said
Kelly Gillespie, President & CEO of MOBIO.
While the FDA recently approved the biosimilar product for sale in the U.S., the agency continues to develop
a pathway for the development and approval of safe and effective interchangeable biologic products. However,
there is still a major role for states to play in ensuring communication of substitution occurs and physicians
remain engaged in the process.
“Interchangeable biologics are not generics. Even slight changes to a biologic drug can change its properties entirely,” said Greenwood. “Unlike conventional generic medicines, interchangeable biologics are not the
same as the drugs they seek to substitute. In fact, two biologics made using different cell lines and differing
manufacturing processes will rarely, if ever, be exactly the same. Those suggesting interchangeable biologics
and generics are the same are wrong.”
Patients and physicians managing chronic conditions are generally aware of which biologic treatments work
best in their unique circumstances. Communicating with patients and physicians allows everyone involved
the opportunity to discuss past treatment experiences so that any possible unexpected issues can be better
understood and avoided.
Gillespie concluded, “Thankfully, for what was likely a terminal prognosis even thirty years ago, many of
those are now manageable diseases thanks to advances in medical research. And, with the promises of better understanding genetics, advancements in coordinating wellness, and marrying big data analytics to the
biological sciences and personalized medicine - science is yielding a territory where our next generation will
have it better than us.” About BIO BIO is the world’s largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States
and in more than 30 other nations. BIO members are involved in the research and development of innovative
healthcare, agricultural, industrial and environmental biotechnology products. BIO also produces the BIO
International Convention, the world’s largest gathering of the biotechnology industry, along with industryleading investor and partnering meetings held around the world. BIO produces BIOtechNOW, an online portal
and monthly newsletter chronicling “innovations transforming our world.” Subscribe to BIOtechNOW.
Upcoming BIO Events BIO International Convention June 6-9, 2016
San Francisco, CA
BIO Investor Forum October 18-19, 2016
San Francisco, CA
View source version on businesswire.com:
http://www.businesswire.com/news/home/
20160608006541/en/
NewsRx LLC
(2016-06-22), Missouri Governor Nixon Signs Bill Ensuring Patient Access to Interchangeable Biologic
Medicines, Biotech Week, 165, ISSN: 1537-4699, BUTTER® ID: 011930573
3
Biotechnology
Research Conducted at Columbia University Has Provided New Information about
Biomedicine and Biomedical Engineering (A dual wedge microneedle for sampling of
perilymph solution via round window membrane)
By a News Reporter-Staff News Editor at Biotech Week – Research findings on Biotechnology are discussed in
a new report. According to news reporting out of New York City, New York, by NewsRx editors, research stated,
“Precision medicine for inner-ear disease is hampered by the absence of a methodology to sample inner-ear fluid
atraumatically. The round window membrane (RWM) is an attractive portal for accessing cochlear fluids as it
heals spontaneously.”
Our news journalists obtained a quote from the research from Columbia University, “In this study, we report on the development of a microneedle for perilymph sampling that minimizes the size of RWM perforation,
facilitates quick aspiration, and provides precise volume control. Here, considering the mechanical anisotropy
of the RWM and hydrodynamics through a microneedle, a 31G stainless steel pipe was machined into wedgeshaped design via electrical discharge machining. The sharpness of the needle was evaluated via a surface
profilometer. Guinea pig RWM was penetrated in vitro, and 1 mu L of perilymph was sampled and analyzed
via UV-vis spectroscopy. The prototype wedge shaped needle was successfully fabricated with the tip curvature
of 4.5 mu m and the surface roughness of 3.66 mu m in root mean square. The needle created oval perforation
with minor and major diameter of 143 and 344 mu m (n=6). The sampling duration and standard deviation
of aspirated volume were 3 s and 6.8 % respectively. The protein concentration was 1.74 mg/mL. The prototype needle facilitated precise perforation of RWMs and rapid aspiration of cochlear fluid with precise volume
control.”
According to the news editors, the research concluded: “The needle design is promising and requires testing
in human cadaveric temporal bone and further optimization to become clinically viable.”
For more information on this research see: A dual wedge microneedle for sampling of perilymph solution via
round window membrane. Biomedical Microdevices , 2016;18(2):15-22. Biomedical Microdevices can be contacted at: Springer, Van Godewijckstraat 30, 3311 Gz Dordrecht, Netherlands. (Springer - www.springer.com;
Biomedical Microdevices - http://www.springerlink.com/content/1387-2176/ )
Our news journalists report that additional information may be obtained by contacting H. Watanabe,
Columbia University, Dept. of Otolaryngol Head & Neck Surg, 630 W 168th St, New York, NY 10032, United
States. Additional authors for this research include L. Cardoso, A.K. Lalwani and J.W. Kysar.
NewsRx LLC
(2016-06-22), Research Conducted at Columbia University Has Provided New Information about
Biomedicine and Biomedical Engineering (A dual wedge microneedle for sampling of perilymph solution via
round window membrane), Biotech Week, 535, ISSN: 1537-4699, BUTTER® ID: 011930794
Biotechnology
Research Conducted at Columbia University Has Provided New Information about
Biomedicine and Biomedical Engineering (A dual wedge microneedle for sampling of
perilymph solution via round window membrane) (A dual wedge microneedle for sampling
...)
By a News Reporter-Staff News Editor at Ivy League Week – Research findings on Biotechnology are discussed
in a new report. According to news reporting out of New York City, New York, by NewsRx editors, research
stated, “Precision medicine for inner-ear disease is hampered by the absence of a methodology to sample innerear fluid atraumatically. The round window membrane (RWM) is an attractive portal for accessing cochlear
fluids as it heals spontaneously.”
Our news journalists obtained a quote from the research from Columbia University, “In this study, we report on the development of a microneedle for perilymph sampling that minimizes the size of RWM perforation,
facilitates quick aspiration, and provides precise volume control. Here, considering the mechanical anisotropy
of the RWM and hydrodynamics through a microneedle, a 31G stainless steel pipe was machined into wedgeshaped design via electrical discharge machining. The sharpness of the needle was evaluated via a surface
profilometer. Guinea pig RWM was penetrated in vitro, and 1 mu L of perilymph was sampled and analyzed
via UV-vis spectroscopy. The prototype wedge shaped needle was successfully fabricated with the tip curvature
4
of 4.5 mu m and the surface roughness of 3.66 mu m in root mean square. The needle created oval perforation
with minor and major diameter of 143 and 344 mu m (n=6). The sampling duration and standard deviation
of aspirated volume were 3 s and 6.8 % respectively. The protein concentration was 1.74 mg/mL. The prototype needle facilitated precise perforation of RWMs and rapid aspiration of cochlear fluid with precise volume
control.”
According to the news editors, the research concluded: “The needle design is promising and requires testing
in human cadaveric temporal bone and further optimization to become clinically viable.”
For more information on this research see: A dual wedge microneedle for sampling of perilymph solution via
round window membrane. Biomedical Microdevices , 2016;18(2):15-22. Biomedical Microdevices can be contacted at: Springer, Van Godewijckstraat 30, 3311 Gz Dordrecht, Netherlands. (Springer - www.springer.com;
Biomedical Microdevices - http://www.springerlink.com/content/1387-2176/ )
Our news journalists report that additional information may be obtained by contacting H. Watanabe,
Columbia University, Dept. of Otolaryngol Head & Neck Surg, 630 W 168th St, New York, NY 10032, United
States. Additional authors for this research include L. Cardoso, A.K. Lalwani and J.W. Kysar.
NewsRx LLC
(2016-06-21), Research Conducted at Columbia University Has Provided New Information about
Biomedicine and Biomedical Engineering (A dual wedge microneedle for sampling of perilymph solution via
round window membrane) (A dual wedge microneedle for sampling ...), Ivy League Week, 309, ISSN: 0000-0000,
BUTTER® ID: 011927320
Biotechnology
Studies from University of Toronto Provide New Data on Pharmacogenomics (PharmacoGx:
an R package for analysis of large pharmacogenomic datasets)
By a News Reporter-Staff News Editor at Information Technology Newsweekly – Current study results on
Biotechnology have been published. According to news reporting originating in Toronto, Canada, by VerticalNews journalists, research stated, “Pharmacogenomics holds great promise for the development of biomarkers
of drug response and the design of new therapeutic options, which are key challenges in precision medicine.
However, such data are scattered and lack standards for efficient access and analysis, consequently preventing
the realization of the full potential of pharmacogenonnics.”
The news reporters obtained a quote from the research from the University of Toronto, “To address these
issues, we implemented PharmacoGx, an easy-to-use, open source package for integrative analysis of multiple
pharnnacogenomic datasets. We demonstrate the utility of our package in comparing large drug sensitivity
datasets, such as the Genomics of Drug Sensitivity in Cancer and the Cancer Cell Line Encyclopedia. Moreover,
we show how to use our package to easily perform Connectivity Map analysis.”
According to the news reporters, the research concluded: “With increasing availability of drug -related data,
our package will open new avenues of research for meta-analysis of pharmacogenomic data.”
For more information on this research see: PharmacoGx: an R package for analysis of large pharmacogenomic datasets. Bioinformatics , 2016;32(8):1244-1246. Bioinformatics can be contacted at: Oxford Univ Press,
Great Clarendon St, Oxford OX2 6DP, England. (Oxford University Press - http://www.oup.com/ ; Bioinformatics - bioinformatics.oxfordjournals.org)
Our news correspondents report that additional information may be obtained by contacting P. Smirnov,
University of Toronto, Dept. of Comp Sci, Toronto, ON, Canada. Additional authors for this research include
Z. Safikhani, N. El-Hachem, D. Wang, A. She, C. Olsen, M. Freeman, H. Selby, D.M.A. Gendoo, P. Grossmann,
A.H. Beck, H. Aerts, M. Lupien, A. Goldenberg and B. Haibe-Kains.
NewsRx LLC
(2016-06-07), Studies from University of Toronto Provide New Data on Pharmacogenomics (PharmacoGx:
an R package for analysis of large pharmacogenomic datasets), Information Technology Newsweekly, 320, ISSN:
1944-1800, BUTTER® ID: 011883432
5
Biotechnology
Studies from Yonsei University Update Current Data on Xenografts (Isolation and
characterization of tumorspheres from a recurrent pineoblastoma patient: Feasibility of a
patient-derived xenograft)
By a News Reporter-Staff News Editor at Clinical Oncology Week – Research findings on Biotechnology are
discussed in a new report. According to news reporting originating from Seoul, South Korea, by NewsRx correspondents, research stated, “The existence of tumorspheres (TSs) might confer treatment resistance to pineoblastoma (PB). The existence of PB TSs with cellular immortalization potential has not yet been reported.”
Our news editors obtained a quote from the research from Yonsei University, “We developed a procedure
for isolating TSs from recurrent PB (rPB) and tested whether their properties made them suitable for use
as a patient-derived xenograft (PDX). Immunocytochemical staining, RT-PCR and quantitative real-time PCR
showed that, among stemness proteins, CD133, musashi and podoplanin were expressed at elevated levels in
rPB TSs, but nestin was not. rPB TSs cultured under neuro-glial differentiation conditions expressed TUBB3,
but not GFAP, MBP or NeuN. Unlike glioblastoma TSs, rPB TSs showed no clear evidence of invasion in 3D
invasion assay or increased expression of genes associated with epithelial-mesenchymal transition. An orthotopic xenograft showed that tumor xenografts replicated the histopathological features of the patient tumor and
expressed similar genome profiles, as determined by short tandem repeat genotyping. These data demonstrate
the isolation and the characterization of rPB TSs for the first time.”
According to the news editors, the research concluded: “Using an orthotopic xenograft, we showed that rPB
TSs could replicate the patient tumor, demonstrating their potential as a PDX for precision medicine.”
For more information on this research see: Isolation and characterization of tumorspheres from a recurrent pineoblastoma patient: Feasibility of a patient-derived xenograft. International Journal of Oncology ,
2016;49(2):569-578. International Journal of Oncology can be contacted at: Spandidos Publ Ltd, Pob 18179,
Athens, 116 10, Greece.
The news editors report that additional information may be obtained by contacting J. Kwak, Yonsei University, Coll Med, Severance Hosp, Dept. of Radiol, 50-1 Yonsei Ro, Seoul 120752, South Korea. Additional
authors for this research include J.K. Shim, D.S. Kim, J.H. Lee, J. Choi, J. Park, K.J. Shin, S.H. Kim, P. Kim,
Y.M. Huh, E.H. Kim, J.H. Chang, S.H. Kim and S.G. Kang.
NewsRx LLC
(2016-07-25), Studies from Yonsei University Update Current Data on Xenografts (Isolation and characterization of tumorspheres from a recurrent pineoblastoma patient: Feasibility of a patient-derived xenograft),
Clinical Oncology Week, 154, ISSN: 1543-6780, BUTTER® ID: 012089955
Southwest Research Institute
SwRI, UTSA Invest in Two New Joint Biomaterial Research Projects
By a News Reporter-Staff News Editor at Pharma Business Week – Southwest Research Institute (SwRI) and
The University of Texas at San Antonio (UTSA) Office of the Vice President for Research announced two new
research projects through the Connecting through Research Partnerships (Connect) Program. The projects are
slated to begin Sept. 1, 2016, with each receiving $125,000 in funding. The two projects will investigate biofilm
corrosion in pipelines and an ultrasound drug delivery methodology.
“These joint UTSA and SwRI programs leverage talent at both organizations, build strong teams for future
contract opportunities, and accelerate the transition of fundamental research to the public,” said Dr. Michael
MacNaughton, vice president of the SwRI Chemistry and Chemical Engineering Division.
Biofilms often cause microbiologically influenced corrosion (MIC) and are a serious problem in pipelines
and other infrastructure. SwRI and UTSA will collaborate to gain a better understanding of MIC by collecting
genomic and metabolic data from biofilms. These data will be used to develop models that can predict corrosion
and identify potential novel inhibitors of biofilm formation. This research is geared toward the petroleum
industry where problematic biofilms occur in many of the production and distribution processes. However, it
has broad implications in other pipeline industries as well as medical applications where dental and other types
of implants are used.
Manager Dr. Tony Reeves, Principal Scientist Dr. Kennedy Gauger, and Scientist Kenneth Lange, all of
SwRI’s Pharmaceuticals and Bioengineering Department, will collaborate with UTSA College of Engineering
researcher Dr. Heather Shipley, chair of the civil and environmental engineering department, and Dr. Gisella
6
Lamas-Samanamud, a postdoctoral fellow, on the project “Molecular Characterization and Quorum Sensing of
Microbiologically Influenced Corrosion (MIC) in Pipeline Populations.”
Precision medicine, also called personalized medicine, takes the approach that no one treatment fits all
patients, tailoring medical decisions by considering the predicted response of an individual. Together UTSA
and SwRI will explore new ways to monitor a drug once it has been given in vivo. The team will develop a
new approach using acoustic-sensitive liposomes for ultrasound-mediated drug release and then monitor the
real-time drug concentrations in deep tissue.
Staff Engineer Dr. Jian Ling of SwRI’s Pharmaceuticals and Bioengineering Department and UTSA’s Dr.
Jing Yong Ye will collaborate on “Ultrasound Mediated Drug Delivery in 3D Tissue Model Quantified by Photoacoustic Tomography.” Ye is the interim department chair of biomedical engineering in the UTSA College of
Engineering.
“The ability to control the release of therapeutics in targeted tissues with a desired spatial distribution and
at an adjustable rate according to the drug response of each individual is important for personalized medicine,”
Ling said.
Over the past six years, 11 projects have been funded under the joint SwRI-UTSA Connect Program.
“Fostering collaborative opportunities for researchers from our two institutions has led to scientific discoveries and advanced technologies. The Connect program continues to surpass our expectations. These two projects
focus on priority funding areas - biomaterials and biomedicine. Together we are finding new ways to address
these difficult technical challenges,” said SwRI Executive Vice President Walt Downing.
“When we leverage the research expertise of both institutions and cross-pollinate efforts through the Connect
program, we can spark innovation and progress,” said Dr. Bernard Arulanandam, UTSA interim vice president
for research. “Our funding selection committee is looking to fund research that finds solutions for specific
challenges. This year, with the two chosen projects - precision medicine and pipe corrosion in the petroleum
industry -we can have a systemic impact on the people, and the industries, of Texas.”
The Connect program was founded to enhance scientific collaboration between SwRI and UTSA
and increase their research funding base.
http://www.swri.org/9what/releases/2016/
swri-utsa-joint-biomaterial-research.htm View source version on businesswire.com: http:
//www.businesswire.com/news/home/20160706006268/en/
NewsRx LLC
(2016-07-18), SwRI, UTSA Invest in Two New Joint Biomaterial Research Projects, Pharma Business Week,
74, ISSN: 1543-6667, BUTTER® ID: 012060897
7
Chapter 2
Cancer
Sarah Cannon Research Institute
Cancer Experts from Sarah Cannon Research Institute Present Insights at American Society
of Clinical Oncology’s 2016 Annual Meeting
By a News Reporter-Staff News Editor at Clinical Trials Week – Sarah Cannon announced that it will present
cancer research insights through more than 100 presentations selected by the American Society of Clinical
Oncology (ASCO®) at the 2016 Annual Meeting. Hosted in Chicago, June 3-7, the meeting brings together
more than 30,000 cancer experts from around the world to review the latest research to improve the diagnosis
and treatment of cancer.
“For more than two decades, Sarah Cannon investigators have been at the forefront of conducting clinical
trials in the community to ensure patients can receive novel therapies closer to home,” said Howard A. “Skip”
Burris III, MD, Chief Medical Officer at Sarah Cannon. “We look forward to discussing the latest advancements
in targeted cancer treatments and immunotherapies, and sharing our drug development expertise through
presentations that feature more than 60 abstracts related to phase 1 trials.”
Highlights of Sarah Cannon’s research at the ASCO® Annual Meeting include a clinical symposium presentation by John D. Hainsworth, MD, co-founder of Sarah Cannon Research Institute, who will present early
results of My Pathway, a phase IIa umbrella basket study, which investigates a targeted therapy for advanced
solid tumors based on molecular profiles. Dr. Hainsworth’s presentation will be included in the press program
on Saturday, June 4 at 8-9am CDT.
“As precision medicine advances, we are finding new ways to personalize treatment plans based on a patient’s unique cancer,” said Dr. Hainsworth. “We look forward to sharing results from this study, which matches
patients with molecular abnormalities to corresponding targeted treatments.”
Additionally, Jeffrey R. Infante, MD, Director, Drug Development Program, Sarah Cannon Research Institute, will present in a clinical symposium where he will discuss a phase 1b study of an OX40 agonist and a
PD-L1 inhibitor in patients with advanced solid tumors. Dr. Infante will also present in an educational session
on brain metastasis and barriers for this patient population when enrolling in a clinical trial.
Several other Sarah Cannon investigators are presenting noteworthy studies in educational sessions and
oral sessions, which include: Melissa Johnson, MD, Associate Director, Lung Cancer Research, Sarah Cannon Research Institute, and Professor Charles Swanton Sarah Cannon UK’s personalized medicine lead and
Professor of Personalized Medicine at UCL Cancer Institute, will present in an educational session on “Developmental Therapeutics and Translational Research, Clinical Trials, Tumor Biology.” Dr. Johnson will discuss
“Blood-based Molecular Testing: Ready for Prime Time?” which explores using a more rapid and less invasive
method for tumor surveillance and early cancer detection. Professor Swanton will present on tumor heterogeneity and the design of targeted therapies. Johanna C. Bendell, MD, Director, GI Cancer Research Program,
Sarah Cannon Research Institute, will discuss the clinical activity and safety of cobimetnib and atezolizumab in
patients with colorectal cancer in an oral presentation. Dr. Bendell will also present in an educational session
on the treatment of RAS wild-type patients with anti-EGFR first line therapy. Gerald S. Falchook, MD, MS,
Director, Drug Development, Sarah Cannon Research Institute at HealthONE, will be discussing the safety,
pharmacokinetics, pharmacodynamics and antitumor activity in a phase 1b study evaluating anti-ErbB3 antibody KTN3379 alone and with targeted therapies in adults with advanced tumors. David Spigel, MD, Chief
Scientific Officer and Director, Lung Cancer Research Program, Sarah Cannon Research Institute, will be featured in a Meet the Professor session entitled “Prescribing Immunotherapy: Choosing Who and When” at the
8
meeting. Howard A. “Skip” Burris III, MD, Chief Medical Officer at Sarah Cannon, will lead a poster discussion
session and share his insights on “Unique Targets for Antibody Drug Conjugates: Making Chemotherapy Great
Again?”
For a full listing of all presentations authored by Sarah Cannon investigators, visit: http://
sarahcannon.com/asco16 .
Additional investigators presenting for Sarah Cannon at the conference include: Hendrik-Tobias Arkenau,
MD, PhD, FRCP, Todd M. Bauer, MD, Jesus G. Berdeja, MD, William Bruce Donnellan, MD, Ian W. Flinn, MD,
PhD, F. Anthony Greco, MD, Erika P. Hamilton, MD, Lowell Hart, MD, Suzanne F. Jones, PharmD, Kathleen
Moore, MD, Manish Patel, MD, Saeed Rafii, MD, PhD, MRCP, Kent C. Shih, MD, David Waterhouse, MD,
Henry Q. Xiong, MD, PhD and Denise A. Yardley, MD.
The researchers represent Sarah Cannon’s global network of strategic sites: Sarah Cannon Research Institute at Tennessee Oncology, Sarah Cannon Research Institute at HealthONE, Sarah Cannon Research Institute - United Kingdom, Colorado Blood Cancer Institute, The Center for Cancer and Blood Disorders - Ft.
Worth, Sarah Cannon Research Institute at Florida Cancer Specialists, Sarah Cannon Research Institute at
HCA Midwest Health, The Stephenson Cancer Center at the University of Oklahoma and Oncology Hematology Care, Inc. About Sarah Cannon Research Institute Sarah Cannon Research Institute is the research
arm of HCA’s global cancer institute, Sarah Cannon. Focused on advancing therapies for patients, it is one
of the world’s leading clinical research organizations conducting community-based clinical trials throughout
the United States and United Kingdom. Sarah Cannon’s network of strategic sites includes more than 275
physicians who engage in research. The organization has led more than 220 first-in-man clinical trials since
its inception in 1993, and has been a clinical trial leader in more than two-thirds of approved cancer therapies over the last 10 years. Additionally, Sarah Cannon offers management, regulatory, and other research
support services for drug development and industry sponsors as well as strategic investigator sites through
its contract research organization (CRO), Sarah Cannon Development Innovations (formerly known as SCRI
Development Innovations). For more information, visit sarahcannon.com. View source version on businesswire.com: http://www.businesswire.com/news/home/20160531005228/en/
NewsRx LLC
(2016-06-13), Cancer Experts from Sarah Cannon Research Institute Present Insights at American Society of Clinical Oncology’s 2016 Annual Meeting, Clinical Trials Week, 56, ISSN: 1543-6764, BUTTER® ID:
011823180
ArcherDX, Inc.
Cancer Genetics, Inc. Partners with ArcherDX to Offer Archer® NGS Assays as a
Sequencing Service for Biotech and Pharma
By a News Reporter-Staff News Editor at Clinical Trials Week – ArcherDX, Inc., a leader in NGS-based gene
fusion detection, has announced that Cancer Genetics, Inc., (CGI) has become a Certified Service Provider
of Archer® FusionPlex® NGS assays. Additionally, CGI plans to use Archer VariantPlexTM assays to identify
copy number variants (CNVs) and point mutations in tumor samples and will be testing these assays in cell-free
variant detection.
With headquarters in New Jersey and locations in North Carolina, California, India and China, CGI actively
supports biopharma with genomics-based, comprehensive biomarker assessment technologies and services,
including markers for immuno-oncology.
Dr. Jason Myers, CEO of ArcherDX, highlighted the importance of having an international network of
testing labs to support clinical trials services. “Our biopharma partners look at the market from a global
perspective. Given the nature of cancer as a genetic disease and the varying incidence rates in different ethnic
groups, it is important to be able to use genomic approaches to identify and enroll patients from around the
world and integrate our information with other clinical and genomic features.”
Dr. Rita Shaknovich, Group Medical Director of CGI, elaborated, “There is a global demand to support
clinical trials, especially in the areas of tyrosine kinase inhibitor (TKI) development, as the relevance of new
gene fusions is linked to potential positive clinical outcomes. ArcherDX is the established leader in modern gene
fusion detection, such as NTRK fusions, which are found in a broad range of cancers including adenocarcinomas,
high-grade gliomas, pilocytic astrocytomas and leukemias. We are thus pleased to provide their assays to
support clinical trials and other services to our global biopharma partners.”
9
CGI offers a comprehensive range of laboratory services to support biomarker and companion diagnostics strategies and has a strong network of key opinion leaders around the world. By combining strengths
in biomarker and diagnostic testing with established collaborations with leading hospitals and medical centers, CGI offers biopharma partners innovative technology and patient insight. About ArcherDX ArcherDX
addresses the bottlenecks associated with using NGS in translational research by offering a robust platform
for targeted sequencing applications. By combining proprietary Anchored Multiplexed PCR (AMP™) chemistry and easy-to-use, lyophilized reagents, Archer NGS assays generate highly enriched sequencing libraries
to detect gene fusions, point mutations, CNVs and RNA abundance. Complemented by the Archer suite of
bioinformatics software, ArcherDX technology dramatically enhances complex mutation identification and discovery.
ArcherDX is headquartered in Boulder, Colorado. Archer Certified Service Provider Program The Archer
Certified Service Provider Program is a partnership forged between ArcherDX and participating laboratories.
These collaborative efforts are the beginning of ongoing relationships that continue far past the certification
process. Benefits of the program to providers are increased referrals and endorsements, early access to new
ArcherDX technologies and co-branding with ArcherDX, including listing on ArcherDX’s list of providers. Note:
Archer kits and analysis software are for research use only and not for use in diagnostic procedures. http:
//archerdx.com Archer®, FusionPlex®, VariantPlex™ and AMP™ are trademarks of ArcherDX, Inc. About
Cancer Genetics Incorporated Cancer Genetics, Inc. is a leader in enabling precision medicine in oncology from
bench to bedside through the use of oncology biomarkers and molecular testing. CGI is developing a global
footprint with locations in the US, India and China. The company has established strong clinical research
collaborations with major cancer centers including Memorial Sloan Kettering Cancer Center, Cleveland Clinic,
Mayo Clinic, Keck School of Medicine of USC, and the US National Cancer Institute.
CGI offers laboratory services that provide critical genomic and biomarker information. Its state-of-theart reference labs are CLIA-certified and CAP-accredited in the US and have licensure from several states,
including New York. http://cancergenetics.com View source version on businesswire.com: http://www.
businesswire.com/news/home/20160602005475/en/
NewsRx LLC
(2016-06-13), Cancer Genetics, Inc. Partners with ArcherDX to Offer Archer® NGS Assays as a Sequencing
Service for Biotech and Pharma, Clinical Trials Week, 212, ISSN: 1543-6764, BUTTER® ID: 011823322
Biotechnology
Epigenetics: New tool for precision medicine
By a News Reporter-Staff News Editor at Biotech Week – Four new papers, co-published by an international
consortium of biomedical researchers, mark the feasibility of epigenetic analysis for clinical diagnostics and
precision medicine. Epigenetic analysis addresses key limitations of genetic testing, helping to ensure that
patients are accurately diagnosed and treated with the right drug at the right time.
Epigenetic changes occur in all cancers, and in various other diseases. Measuring these changes provides
unprecedented insights into the disease mechanisms at work in individual patients, which is important for
better diagnosis and patient-specific treatment decisions.
In a series of four papers led by Christoph Bock (CeMM Research Center for Molecular Medicine of the
Austrian Academy of Sciences, Vienna) and Stephan Beck (University College London, UCL), an international
group of scientists have validated the feasibility of epigenetic analysis for clinical applications.
Building upon years of technology development in laboratories around the world, this series of papers shows
the accuracy and robustness of epigenetic tests. Going forward, clinical researchers will optimize and apply
these methods for specific diseases, and it is expected that epigenetic tests will become widely used for selecting
personalized treatments in cancer and other diseases.
Epigenetics refers to chemical modifications of the DNA and its associated proteins that control gene activity
independent of the genetic code. These epigenetic modifications define how two meters of DNA in each human
cell are folded into tiny cell nuclei.
Epigenetic modifications can be inherited during cell division, which helps maintain the ˜200 cell types of
the human body carrying the same genes. Moreover, epigenetic mechanisms provide an interface by which the
environment influences gene activity.
In many diseases, including all cancers, the epigenetic control of the genome is heavily distorted. Measuring
these alterations provides a detailed picture of the disease-specific changes, which is often informative for
10
distinguishing disease subtypes or identifying suitable treatments. Therefore, epigenetics has much to offer
for improving disease diagnosis and treatment choice.
The now published studies, which have been performed in the context of the European BLUEPRINT project
and the International Human Epigenome Consortium, constitute a milestone for utilizing epigenetic information in clinical diagnostics and precision medicine.
1. In a study published in Nature Biotechnology and coordinated by CeMM, 18 research groups from three
continents compared all promising methods for analyzing DNA methylation in the clinic. This multicenter
benchmarking study identifies the most accurate methods and shows that epigenetic tests based on DNA methylation are a mature technology ready for broad clinical use. http://doi.org/10.1038/nbt.3605
2. Also in Nature Biotechnology, the UCL team presents a computational validation of genome-wide DNA
methylation sequencing technology, confirming its practical use for identifying DNA methylation differences
between samples and disease subtypes. http://doi.org/10.1038/nbt.3524
3. The UCL team further extend their analysis in a paper published in Nature Communications, where
they present new bioinformatic methods for discovering disease-specific DNA methylation patterns from costeffective low-coverage DNA methylation sequencing data. http://doi.org/10.1038/ncomms11306
4. Finally, a Nature Communications paper by CeMM researchers - in collaboration with the clinicians at
the University of Southampton and the Royal Bournemouth Hospital - demonstrates the utility of chromatin
mapping for identifying disease subtypes and predicting prognosis in chronic lymphocytic leukemia. This study
highlights the clinical utility of epigenetic biomarkers especially for diseases with widespread heterogeneity
between individual patients. http://doi.org/10.1038/ncomms11938
Quotes
Christoph Bock (Principal Investigator at CeMM): “Epigenetic tests have a key role to play for making precision medicine a clinical reality. Epigenetics captures part of each cell’s individual history, and it can predict
how cancer cells will react to drug treatment. This can be very useful for personalized therapy.”
Stephan Beck (Professor at UCL): “This exciting new technology will advance our ability to understand
phenotypic plasticity in health and disease, an important aspect of cancer research.”
Giulio Superti-Furga (Scientific Director of CeMM): “It is part of CeMM’s mission to provide innovative
platforms for precision medicine and to make them ready for biomedically important applications. We are
proud of the work that Christoph Bock has done together with Stephan Beck and members of the BLUEPRINT
consortium. It shows that epigenetics bridges genomics and disease.”
NewsRx LLC
(2016-07-13), Epigenetics: New tool for precision medicine, Biotech Week, 77, ISSN: 1537-4699, BUTTER®
ID: 012040381
Gynecology
Findings from Northwestern University Feinberg School of Medicine Provides New Data
about Gynecologic Oncology (Toward precision medicine for preserving fertility in cancer
patients: existing and emerging fertility preservation options for women)
By a News Reporter-Staff News Editor at Health & Medicine Week – Data detailed on Gynecology have been
presented. According to news reporting out of Chicago, Illinois, by NewsRx editors, research stated, “As the
number of young cancer survivors increases, quality of life after cancer treatment is becoming an ever more
important consideration. According to a report from the American Cancer Society, approximately 810,170
women were diagnosed with cancer in 2015 in the United States.”
Our news journalists obtained a quote from the research from the Northwestern University Feinberg School
of Medicine, “Among female cancer survivors, 1 in 250 are of reproductive age. Anticancer therapies can result
in infertility or sterility and can have long-term negative effects on bone health, cardiovascular health as a
result of reproductive endocrine function. Fertility preservation has been identified by many young patients diagnosed with cancer as second only to survival in terms of importance. The development of fertility preservation
technologies aims to help patients diagnosed with cancer to preserve or protect their fertility prior to exposure
to chemo-or radiation therapy, thus improving their chances of having a family and enhancing their quality
of life as a cancer survivor. Currently, sperm, egg, and embryo banking are standard of care for preserving
fertility for reproductive-age cancer patients; ovarian tissue cryopreservation is still considered experimental.
11
Adoption and surrogate may also need to be considered. All patients should receive information about the fertility risks associated with their cancer treatment and the fertility preservation options available in a timely
manner, whether or not they decide to ultimately pursue fertility preservation.”
According to the news editors, the research concluded: “Because of the ever expanding number of options for
treating cancer and preserving fertility, there is now an opportunity to take a precision medicine approach to
informing patients about the fertility risks associated with their cancer treatment and the fertility preservation
options that are available to them.”
For more information on this research see: Toward precision medicine for preserving fertility in cancer
patients: existing and emerging fertility preservation options for women. Journal of Gynecologic Oncology ,
2016;27(2):e22.
Our news journalists report that additional information may be obtained by contacting S.Y. Kim, Dept. of
Obstetrics and Gynecology, Northwestern University Feinberg School of Medicine, Chicago, IL, United States.
Additional authors for this research include S.K. Kim, J.R. Lee and T.K Woodruff.
NewsRx LLC
(2016-06-17), Findings from Northwestern University Feinberg School of Medicine Provides New Data about
Gynecologic Oncology (Toward precision medicine for preserving fertility in cancer patients: existing and
emerging fertility preservation options for women), Health & Medicine Week, 1690, ISSN: 1532-4605, BUTTER® ID: 011848891
Foundation Medicine, Inc.
Foundation Medicine Presents New Data at ASCO 2016 Demonstrating that
FoundationOne® May Help Predict Response to Cancer Immunotherapy Across a Variety
of Advanced Cancers
By a News Reporter-Staff News Editor at Biotech Business Week – Foundation Medicine, Inc. (NASDAQ:FMI)
and it collaborators presented data showing that higher tumor mutational burden, as estimated by comprehensive genomic profiling with FoundationOne®, successfully predicted a greater likelihood of response and
longer response duration to cancer immunotherapies in patients with advanced bladder cancer and metastatic
melanoma as well as several other tumor types. The results were presented in two oral sessions, and several
poster discussions at the 2016 Annual Meeting of the American Society of Clinical Oncology (ASCO) taking
place in Chicago.
“Successful application of cancer immunotherapy in the clinic is one of the most important advances in
cancer treatment in decades,” said Vincent Miller, M.D., chief medical officer at Foundation Medicine. “These
data suggest that tumor mutational burden could serve as an independent predictive biomarker to aid clinicians
in identifying patients who are most likely to benefit from cancer immunotherapies that target either the PD-1
or PD-L1 proteins.”
Cancer immunotherapy works by helping the immune system mount an effective anti-cancer response, a process that depends in part on the recognition of cancer-specific proteins called neoantigens. Tumor mutational
burden has been shown to correlate well with the number of neoantigens, and therefore it may help identify
patients most likely to respond to cancer immunotherapies. By combining comprehensive genomic profiling of
315 genes utilizing the FoundationOne assay, with Foundation Medicine’s advanced and proprietary algorithm
that filters out normal individual genomic variants, FoundationOne can reliably and accurately measure tumor
mutational burden without the need for whole exome sequencing. Foundation Medicine expects to provide a
CLIA-certified version of tumor mutational burden on all FoundationOne and FoundationOne® Heme reports
to physicians in the third quarter 2016. Overview of Data Presentations The IMvigor 210 study of TecentriqTM
(atezolizumab; anti-PD-L1; Genentech/Roche) in locally advanced or metastatic urothelial carcinoma evaluated
three separate biomarkers: PD-L1 protein expression as measured by immunohistochemistry, molecular subtype as measured by gene expression and The Cancer Genome Atlas, and mutational load (often referred to as
tumor mutational burden) as measured by FoundationOne. All three biomarkers were shown to be independent
predictors of response to Tecentriq.
“PD-L1 Expression, Cancer Genome Atlas (TCGA) Subtype and Mutational Load are Independent Predictors of Response to Atezolizumab (atezo) in Metastatic Urothelial Carcinoma (mUC; Imvigor210)”, by Jonathan
E. Rosenberg, M.D., Memorial Sloan Kettering Cancer Center [Abstract #104, Clinical Science Symposium,
Sunday June 5, 9:57-10:09 AM].
12
“These results are particularly exciting given the amount of variability inherent to using immunohistochemistry (IHC) to measure biomarkers. There are many different PD-L1 IHC tests, for example, and pathologists
often do not see agreement between them,” stated Vamsidhar Velcheti, M.D., assistant professor, Solid Tumor
Oncology, Taussig Cancer Institute, Cleveland Clinic. “We need a truly quantitative and reproducible approach
to predicting response to immunotherapies, and measuring tumor mutational burden using FoundationOne
may provide us with that solution.”
In a separate melanoma study, higher mutational burden as measured by FoundationOne was associated with a greater likelihood of response and a more durable response to pembrolizumab; anti-PD-1; Merck,
nivolumab; anti-PD-1; Bristol Myers Squibb, and Tecentriq, thereby providing oncologists with greater confidence in the potential for clinical benefit from a host of newly approved immunotherapies.
“Hybrid Capture-Based Next Generation Sequencing (HC NGS) in Melanoma Identifies Markers of Response to Anti-PD1/PD-L1”, by Douglas Buckner Johnson, M.D., M.S.C.I., Vanderbilt-Ingram Cancer Center
[Abstract #105, Clinical Science Symposium, Sunday June 5, 10:21-10:33 AM]
Dr. Miller continued, “The clinical reality is that some patients respond very well to cancer immunotherapies
and others do not. As a result, the ability to leverage our molecular information platform to identify the right
candidates for these immunotherapies is an important advance for the field of precision medicine. Matching
the right therapy with the right patient has the potential to both improve outcomes and increase efficiency in
the current care model.”
NewsRx LLC
(2016-06-20), Foundation Medicine Presents New Data at ASCO 2016 Demonstrating that FoundationOne®
May Help Predict Response to Cancer Immunotherapy Across a Variety of Advanced Cancers, Biotech Business
Week, 82, ISSN: 1543-6861, BUTTER® ID: 011908362
DNA Research
Genetic testing can help deliver precision medicine to men with advanced prostate cancer
By a News Reporter-Staff News Editor at Biotech Business Week – Genetic testing in men with advanced
prostate cancer could pick up a significant proportion whose disease may be caused by inherited mutations in
genes involved in repairing DNA damage, a major new study reveals.
Testing prostate cancer patients for mutations in key DNA repair genes could identify those who may benefit
from precision treatments that specifically target DNA repair weaknesses in cancer cells.
Some 12 per cent of men with advanced prostate cancer in the study population were found to have inherited
mutations in one of several genes involved in DNA repair - such as the breast cancer gene BRCA2.
The researchers, whose study is published in the prestigious journal the New England Journal of Medicine
today (Wednesday), believe that genetic testing could form a valuable part of the treatment pathway for advanced prostate cancer.
A team at The Institute of Cancer Research, London, and The Royal Marsden NHS Foundation Trust - with
colleagues at seven other world-leading cancer centres in the US - used a simple saliva test to analyse the DNA
of patients.
They examined the DNA code of 20 genes known to have a role in DNA repair in 692 men with advanced
prostate cancer.
Some 12 per cent of men had at least one ‘germline’ mutation in a DNA repair gene - meaning an error
that is either inherited or developed before birth. The most commonly defective gene was BRCA2, which was
mutated in 5 per cent of men.
Men who have inherited mutations in certain DNA repair genes may benefit from treatment with new drugs
called PARP inhibitors, which exploit weaknesses in the processes for DNA repair within cancer cells.
Where BRCA2 mutations are detected in men with advanced prostate cancer, the study opens up the possibility that relatives could also be tested for BRCA2 mutations. More research is needed to clarify the role of
the other DNA repair genes in causing prostate cancer and the implications for testing.
The study is the largest of its kind to date, and the first to comprehensively analyse germline mutations in
men with advanced, or metastatic, prostate cancer - where the disease has spread around the body.
The study was funded by the Prostate Cancer Foundation and Stand Up to Cancer, through a Movember and
Prostate Cancer UK grant to The London Movember Centre of Excellence, through the Experimental Cancer
Medicine Centre at The Institute of Cancer Research (ICR) and The Royal Marsden, and by Cancer Research
UK, the Medical Research Council and the NIHR Biomedical Research Centre at The Royal Marsden and the
ICR.
13
Professor Johann de Bono, Professor of Experimental Cancer Medicine at The Institute of Cancer Research,
London, and Consultant Oncologist at The Royal Marsden NHS Foundation Trust, who led the study in the
UK, said:
“Our study has shown that a significant proportion of men with advanced prostate cancer are born with
DNA repair mutations - and this could have important implications for patients.
“Genetic testing for these mutations could identify men with advanced prostate cancer who may benefit from
precision treatment. We could offer these men drugs such as PARP inhibitors, which are effective in patients
with certain DNA repair mutations and are showing important anti-tumour activity in ongoing clinical trials.
“Where we find BRCA2 mutations, we could also offer genetic testing and counselling to relatives of the
patient to consider how we can reduce their cancer risk. We also need to establish the impact of having DNA
repair defects on survival in men with prostate cancer, and whether we can predict who will develop severe
disease, so we can design new treatment strategies to cure this disease.”
Professor Paul Workman, Chief Executive of The Institute of Cancer Research, London, said:
“This valuable study has given us new insights into the causes of aggressive forms of prostate cancer that
have spread round the body. It suggests testing for inherited DNA repair mutations could become an important
stage in the treatment of men with advanced prostate cancer, by helping direct use of targeted cancer therapies.
“There is huge diversity in prostate cancers from patient to patient - some men live for decades with localised
tumours, while in others cancers develop rapidly and spread round the body. Genetic markers that can detect
the patients at high risk are desperately needed to improve men’s survival chances.”
NewsRx LLC
(2016-07-18), Genetic testing can help deliver precision medicine to men with advanced prostate cancer,
Biotech Business Week, 56, ISSN: 1543-6861, BUTTER® ID: 012056463
Clinical Trials and Studies
Genomic Data Commons at University of Chicago launches new era of cancer data sharing
By a News Reporter-Staff News Editor at Computer Weekly News – The Genomic Data Commons (GDC), a
next-generation platform that enables unprecedented data access, analysis and sharing for cancer research,
publicly launched at the University of Chicago on June 6, opening the door to discoveries for this complex set
of diseases.
The GDC went live with approximately 4.1 petabytes of data from National Cancer Institute-supported
research programs, including some of the largest and most comprehensive cancer genomics datasets in the
world – such as The Cancer Genome Atlas and Therapeutically Applicable Research to Generate Effective
Treatments – and more than 14,000 anonymized patient cases. One petabyte equals 1 million gigabytes.
Vice President Joe Biden toured the GDC operations center at the University of Chicago in advance of his
appearance to announce the project at the annual meeting of the American Society for Clinical Oncology on
June 6.
The Data Commons centralizes, standardizes and harmonizes genomic and clinical data on a unified and
interoperable platform. Cancer researchers can access these data for analyses and submit their own datasets to
share with the research community. By making high-quality data broadly accessible, the GDC provides muchneeded tools to accelerate studies of the biological mechanisms of cancer and the development of personalized
treatments for individual patients.
UChicago developed and operates the Data Commons with NCI funding under a subcontract from Leidos
Biomedical Research at the Frederick National Laboratory for Cancer Research, in collaboration with the Ontario Institute for Cancer Research.
Development of the GDC began in 2014 at UChicago’s Center for Data Intensive Science (CDIS). Over the
past two years, the team has created an innovative suite of tools, software and infrastructure – based on CDIS
open-source projects such as the Bionimbus Protected Data Cloud – to curate the massive amounts of data held
by the GDC.
“Today, making discoveries from cancer genomic data is challenging because diverse research groups analyze
different cancer datasets using various methods that are not easily comparable,” said GDC principal investigator Robert Grossman, professor of medicine and director of CDIS at UChicago. “The Genomic Data Commons
brings together genomic datasets and analyzes the data using a common set of methods so that researchers may
more easily make discoveries, and, in this sense, democratizes the analysis of large cancer genomic datasets.”
“Big data” is recognized as essential to efforts in understanding and treating cancer. Cancer is as complex
as is it is devastating. It involves a host of genetic, lifestyle and environmental factors, and is now known to
14
comprise hundreds of diseases – each with unique features, driving forces and vulnerabilities to treatments.
Large sample sizes are required to provide the statistical power to understand which combinations of drugs are
effective against which combinations of mutations that drive cancer.
Breaking Barriers
While enormous amounts of genomic and clinical data have been gathered by NCI-funded research, several barriers have prevented researchers from making full use of them. Genomic data from different projects,
clinical trials and cancer types are siloed in different locations with local management systems, making data
sharing difficult. These large datasets can take months to download, and not all researchers have access to the
sophisticated tools needed to study them. In addition, disparate collection and analysis approaches by separate
research groups inhibit collaborative work.
The GDC breaks down these barriers by bringing cancer genomics datasets and associated clinical data into
one location that any researcher may access. It harmonizes the data with a common set of analytic pipelines
to make it easier to study the information, which in the past has typically been available as separate datasets
analyzed with separate pipelines. By making these data available using modern computing and network technology, the GDC makes it possible for any researcher to ask new and fundamental questions about cancer.
Built and managed by Grossman’s team at the University of Chicago, the GDC will:
A Foundation for the Future
As the first step in a next generation knowledge system for cancer, the GDC enables and accelerates efforts
to identify both high- and low-frequency cancer driver mutations, assists in revealing the genetic determinants
of response to therapy, and informs the composition of clinical trial cohorts.
The GDC will help bridge siloes by providing researchers with access to high-quality data, the tools needed
to share and study them, and support to submit their own data. It will house data from a new era of programs
that will sequence the DNA of patients enrolled in NCI clinical trials. These datasets will lead to a much deeper
understanding of which therapies are most effective for different cancers. The GDC will support clinical trials
that focus on single patients, known as “n of 1” clinical trials, and will become an important component in how
precision medicine is used to treat individual patients.
The GDC also creates a foundation for future cloud-based technologies that could allow researchers to analyze large-scale datasets and perform experiments remotely, such as through the NCI’s Cancer Cloud Pilots
Program. In addition, the open-source software being developed by the CDIS has the potential to become a
model for data-intensive research efforts for other diseases, such as Alzheimer’s and diabetes, which would
greatly benefit from similar large-scale, data-driven approaches to develop cures.
“We are at a crossroads today in whether we will have the critical mass of cancer-related data needed to
power new discoveries and improve cancer care,” Grossman said. “Over time, I expect the GDC will play a more
and more important role in providing the data required at the scale required so that precision medicine fulfills
its promise.”
NewsRx LLC
(2016-06-22), Genomic Data Commons at University of Chicago launches new era of cancer data sharing,
Computer Weekly News, 266, ISSN: 1944-1606, BUTTER® ID: 011931137
Genomic Health, Inc.
Genomic Health Launches Oncotype SEQ Liquid Biopsy Mutation Panel
By a News Reporter-Staff News Editor at Clinical Trials Week – Genomic Health, Inc. (Nasdaq: GHDX) announced the commercial launch of Oncotype SEQ™ Liquid Select, the first of several non-invasive liquid biopsy
tests that the company plans to deliver through its Oncotype IQ™ Genomic Intelligence Platform. Oncotype
SEQ is a blood-based test that uses next-generation sequencing to identify and assess actionable genomic alterations in a panel of 17 select genes to inform the treatment of stage IV solid tumors, including lung, breast,
colon, melanoma, ovarian, and gastrointestinal stromal tumors.
The test is designed to provide clinically actionable information focused on genomic markers that have
either been included in National Comprehensive Cancer Network (NCCN(®)) guidelines or associated with
sensitivity or resistance to relevant FDA-approved therapies. The test can also match eligible patients with
actively enrolling Phase II-IV clinical trials specific to their tumor type.
“As the world’s leading provider of genomic-based cancer diagnostic tests, we are uniquely positioned to
make a significant impact in the field of liquid biopsy with Oncotype SEQ, the newest clinically actionable test
in our Oncotype IQ portfolio,” said Phil Febbo, M.D., chief medical officer, Genomic Health. “This blood-based
test will provide oncologists with important genomic information reported in a manner that will allow efficient
15
interpretation and identification of potential treatment options. With our unique and extensive experience in
the commercialization and reimbursement of advanced diagnostics, Genomic Health is leveraging its worldclass channel to expand the delivery of precision medicine to physicians and patients beyond the research
setting.”
By analyzing cell-free DNA (cfDNA) isolated from a patient’s blood through a simple blood draw, Oncotype
SEQ examines tumor-derived genomic alterations that are associated with malignant transformation and response or resistance to therapy. Using unique bioinformatics methodology, the test detects the four major types
of variants with very high sample-level sensitivity and specificity. The comprehensive Oncotype SEQ report
provides physicians with important molecular alterations information in a streamlined format that facilitates
interpretation and connection to potential treatment options.
“Liquid biopsy assesses cancer changes through a simple blood draw, a desirable choice for many patients,
as it is a minimally invasive way to capture tumor genomic alterations and heterogeneity with the potential to
be more cost- and time-efficient,” said Lee Schwartzberg, M.D., chief, Division of Hematology Oncology, University of Tennessee. “Oncotype SEQ has robust analytical performance representative of the targeted patient
population tested. Armed with this clinically meaningful information, Oncotype SEQ will allow physicians to
be more effective and efficient in exploring treatment and clinical trial options for late-stage cancer patients.”
The initial phase of the targeted launch will be focused on select clinics for the treatment of stage IV lung
cancer patients. Liquid biopsy is a particularly viable option for lung cancer patients due to the difficulty and
risk associated with lung tissue biopsy and the number of clinically actionable alterations. With successful analytical validation results being submitted for presentation, Genomic Health has launched a global, multi-center
clinical concordance study as part of the company’s goal to establish further evidence to support reimbursement.
By delivering insights into targeted therapy options, Oncotype SEQ Liquid Select is designed to address the
needs of more than 350,000 patients who recur or present with late-stage disease each year in the United States
alone. Oncotype SEQ will be performed at Genomic Health’s CLIA-certified laboratory using the company’s
proprietary cfDNA sequencing platform. Keywords for this news article include: Cancer, Genetics, Oncology,
Genomic Health Inc..
NewsRx LLC
(2016-06-27), Genomic Health Launches Oncotype SEQ Liquid Biopsy Mutation Panel, Clinical Trials Week,
110, ISSN: 1543-6764, BUTTER® ID: 011946552
Station X, Inc.
Georgetown University’s Innovation Center for Biomedical Informatics Adopts GenePool
from Station X to Power Cancer Research
By a News Reporter-Staff News Editor at Journal of Engineering – Station X, Inc., a pioneer in solving big data
analytics and platform integration challenges for customers engaging in genomics-based clinical research and
medical applications, announced that the Innovation Center for Biomedical Informatics (ICBI) at Georgetown
University has adopted the company’s GenePool platform for use in genomic research related to pancreatic
cancer.
The Innovation Center for Biomedical Informatics (ICBI) was launched in 2012 as an academic hub for innovative research in the field of biomedical informatics with the goal of enabling more individualized approaches
to health care based on patient genetics. ICBI’s mission is to enhance translational research at Georgetown
University Medical Center and, in turn, attract and educate the next generation of scientists and physicians for
whom biomedical informatics will be an integral part of both research and clinical practice. ICBI’s major activities are focused on areas that include precision medicine; biocuration and data coordinating centers; omics
and bioinformatics; big data methods and technology; and medical informatics. For more information please
visit icbi.georgetown.edu.
“Genomics and bioinformatics are central to realizing the promise of precision medicine and there is a pressing need for technologies that facilitate collaboration and bridge from foundational research to translational
applications in the clinic,” said Subha Madhavan, PhD, director of the ICBI. “GenePool will allow us to replace
or augment a number of disparate tools with an integrated solution that can support basic research applications like molecular profiling and then, help us place those findings within the context of individual patient
datasets to support translational research and clinical decision making.”
GenePool is an enterprise-level customizable and extensible multi-omics platform for biomarker discovery,
translational research and clinical genomics. Built upon a modern cloud-based architecture with big data
16
technologies, analytical and visualization tools, the platform is designed for storing, analyzing and managing
patient data in a HIPAA compliant environment. GenePool is pre-loaded with reporting capabilities for incidental findings and tumor profiling, but its flexibility means organizations like ICBI can customize GenePool
for use with their own reporting requirements. GenePool also provides a convenient and dynamic way to securely store variants of interest and their associated metadata for updated reporting and for investigating the
aggregated information.
“GenePool integrates a wide range of data types with a modern and scalable analysis pipeline to accelerate
the identification and validation of biomarkers that play a role in human disease,” said Tod Klingler, Chief Scientific Officer of Station X. “ICBI’s adoption of GenePool, combined with our growing user base across research,
diagnostic and biopharma organizations, is an exciting validation of the platform and its ability to support large
enterprises pursuing a variety of precision medicine applications.”
Through GenePool, ICBI can aggregate genomic, biochemical, and clinical information from public and
proprietary databases to further define molecular subtypes that they could use in the development of custom
reports supported by the comprehensive analysis of rich and ever-growing content libraries such as The Cancer
Genome Atlas (TCGA), ClinVar, COSMIC, dbSNP, FDA Pharmacogenomics Biomarkers, PharmaADME, and
many others.
GenePool integrates with existing LIMS systems and leading third-party data storage and management
platforms including Amazon Web Services, DNAnexus, Google Cloud Platform, Illumina BaseSpace, Microsoft
Azure and Seven Bridges Genomics, among others.
For more information about GenePool, please visit www.stationxinc.com. About Station X Station X is
a leading provider of cloud-based enterprise software solutions that are being used to solve big data analytics and platform integration challenges for customers utilizing genomic data in clinical research and medical
applications. GenePool, our flagship product, provides a secure modern architecture that enables secure collaboration and streamlines the delivery of medically relevant insights via innovative solutions for managing and
interpreting complex data. Leading academic and commercial organizations around the world are relying on
Station X to help them deliver next-generation precision medicine solutions. For more information please visit
www.stationxinc.com. View source version on businesswire.com: http://www.businesswire.com/news/
home/20160525005452/en/
NewsRx LLC
(2016-06-06), Georgetown University’s Innovation Center for Biomedical Informatics Adopts GenePool from
Station X to Power Cancer Research, Journal of Engineering, 886, ISSN: 1945-872X, BUTTER® ID: 011867923
Ignyta, Inc.
Ignyta to Present at 2016 Cantor Fitzgerald Healthcare Conference
By a News Reporter-Staff News Editor at Clinical Trials Week – Ignyta, Inc. (Nasdaq: RXDX), a biotechnology
company focused on precision medicine in oncology, announced that Jacob Chacko, M.D., its Chief Financial
Officer, will make a presentation at the 2016 Cantor Fitzgerald Healthcare Conference on July 13, 2016, at 1:00
p.m. Eastern time (10:00 a.m. Pacific time) in New York, NY, and will discuss Ignyta’s novel Rx/Dx approach
and review recent updates from ongoing clinical trials of the company’s robust pipeline of molecularly targeted
therapies.
A webcast of the presentation will be available during the presentation in the Investors section of the company’s website at http://investor.ignyta.com , and will be archived and available at that site for 14 days.
About Ignyta, Inc. At Ignyta, we fight cancer - a formidable opponent that manifests as thousands of different
molecularly defined diseases and takes away millions of lives globally, every year. In this fight, our vision is
not just to shrink tumors but to eradicate residual disease - the source of cancer relapse and recurrence - in
precisely defined patient populations by 2030. We will work tirelessly to achieve this vision by pursuing an
integrated therapeutic (Rx) and companion diagnostic (Dx) strategy for treating cancer patients. Our Rx efforts are focused on discovering, in-licensing or acquiring, then developing and commercializing, molecularly
targeted therapies that, sequentially or in combination, are foundational for eradicating residual disease. Our
Dx efforts aim to pair these product candidates with biomarker-based companion diagnostics that are designed
to precisely identify, at the molecular level, the patients who are most likely to benefit from the therapies we
develop. We believe that only through this integrated Rx/Dx approach can we succeed in this fight.
For more information, please visit: www.ignyta.com. View source version on businesswire.com: http:
17
NewsRx LLC
(2016-07-18), Ignyta to Present at 2016 Cantor Fitzgerald Healthcare Conference, Clinical Trials Week, 80,
ISSN: 1543-6764, BUTTER® ID: 012057165
Carcinogenesis
Investigators at Nagoya University Detail Findings in Carcinogenesis (Oxidative stress as an
iceberg in carcinogenesis and cancer biology)
By a News Reporter-Staff News Editor at Biotech Week – A new study on Carcinogenesis is now available.
According to news reporting from Nagoya, Japan, by NewsRx editors, the research stated, “After the conquest
of numerous infectious diseases, the average life span for humans has been enormously prolonged, reaching
more than 80 years in many developed countries. However, cancer is one of the top causes of death, and its
incidence continues to increase in many countries, including Japan.”
The news correspondents obtained a quote from the research from Nagoya University, “I was deeply influenced during my career as a cancer researcher by the concept of oxidative stress, which was established by
Helmut Sies in 1985. I have no doubt that oxidative stress is a major cause of carcinogenesis in humans but
that other factors and chemicals modify it Notably, established cancer cells are more oxidatively stressed than
their non-tumorous counterparts are, and this stress may be associated with selection under oxidative stress
and, thus, faster proliferation compared with non-tumorous cells. For cancer prevention, both avoidance of
specific risks that are associated with genetic susceptibility and decreasing oxidative stress in general should
delay carcinogenesis. For cancer therapy, individualization and precision medicine require further research in
the future.”
According to the news reporters, the research concluded: “In addition to the currently burgeoning array of
humanized antibodies and protein kinase inhibitors, novel methods to increase oxidative stress only in cancer
cells would be helpful.”
For more information on this research see: Oxidative stress as an iceberg in carcinogenesis and cancer biology. Archives of Biochemistry and Biophysics , 2016;595():46-49. Archives of Biochemistry and Biophysics can be contacted at: Elsevier Science Inc, 360 Park Ave South, New York, NY 10010-1710, USA. (Elsevier - www.elsevier.com; Archives of Biochemistry and Biophysics - http://www.journals.elsevier.com/
archives-of-biochemistry-and-biophysics/ )
Our news journalists report that additional information may be obtained by contacting S. Toyokuni, Nagoya
University, Grad Sch Med, Dept. of Pathol & Biol Responses, Showa Ku, 65 Tsurumai Cho, Nagoya, Aichi
4668550, Japan.
NewsRx LLC
(2016-06-08), Investigators at Nagoya University Detail Findings in Carcinogenesis (Oxidative stress as an
iceberg in carcinogenesis and cancer biology), Biotech Week, 568, ISSN: 1537-4699, BUTTER® ID: 011887765
Liquid Biotech USA, Inc.
Liquid Biotech USA, Inc. Announces Collaboration with the University of Pennsylvania and
Funding of Sponsored Research Agreement
By a News Reporter-Staff News Editor at Biotech Business Week – Liquid Biotech USA, Inc. (“Liquid”) announced the funding of a Sponsored Research Agreement with The University of Pennsylvania (“PENN”) to
study circulating tumor cells (CTCs) from cancer patients. The funding will be used to assess how changes in
CTC levels correlate with clinical outcomes in cancer patients undergoing a variety of anti-cancer therapies.
These data will then be employed to support the design of a therapeutic, decision-making study using CTC
enumeration and genetic profiling as personalized real-time guidance. The ultimate goal of these clinical studies is to utilize CTC-derived information to guide anti-cancer therapy and surveillance via Liquid’s precision
medicine platform.
“The clinical study we are supporting will allow us to understand the actionable biological implications of
CTC determinants in real time during anti-cancer treatment” said Dr. Philip M. Sass, Chairman, President,
and CEO of Liquid Biotech USA, Inc. “then we should be able to correlate CTC levels, genetics, disease stage
and clinical outcome. The goal would be that our platform empowers the oncologist to make clinical decisions
18
quickly, with precision, and in real-time to prevent or manage the spread of cancer based on factors such as
CTC enumeration and genetic markers. “
NewsRx LLC
(2016-07-11), Liquid Biotech USA, Inc. Announces Collaboration with the University of Pennsylvania and
Funding of Sponsored Research Agreement, Biotech Business Week, 26, ISSN: 1543-6861, BUTTER® ID:
012019855
Heart Disease
Little cost difference between tests to diagnose coronary heart disease
By a News Reporter-Staff News Editor at Economics Week – 1. Little cost difference between CT angiography
and stress test for diagnosing heart disease
Abstract: http://www.annals.org/article.aspx?doi=10.7326/M15-2639
Editorial: http://www.annals.org/article.aspx?doi=10.7326/M16-1048
URL goes live when the embargo lifts
For patients with suspected coronary artery disease (CAD), computed tomographic angiography (CTA) and
functional diagnostic testing strategies have similar costs through 3 years of follow up. Results of this prospective economic study are published in Annals of Internal Medicine.
Chest pain is a common reason for patients to seek medical care and a challenge for doctors who must
diagnose the cause. Typically, clinicians rely on health history and noninvasive tests to assess for CAD, such as
CTA and functional stress tests. The recent PROMISE (PROspective Multicenter Imaging Study for Evaluation
of Chest Pain) trial examined the effect of these different diagnostic testing strategies for CAD on patient
outcomes and found little difference between them. A planned secondary aim of PROMISE was to conduct an
economic analysis to assess cost differences between the approaches.
Researchers analyzed economic data for 9,649 patients enrolled in PROMISE between July 2010 and
September 2013. They looked at cost of the initial outpatient testing strategy, hospital-based costs, and physician fees for the first 90 days and then estimated out to 3 years. The data showed that an initial CTA strategy
had costs similar to those of a functional stress testing strategy, but patterns of care differed. Patients in the
CTA group had less follow-up noninvasive testing and more invasive catheterization and revascularization.
After 90 days, the choice of test had little effect on differential costs.
According to the author of an accompanying editorial, the PROMISE economic analysis is disappointing because it lacks data on treatment costs associated with heart disease. Stable ischemic heart disease therapeutic
management includes a focus on symptom control, lifestyle modification, and targeted risk factor modifying
treatments, which may impart a heavy economic burden on many patients. The author suggests further research to determine the value of symptom-guided treatment without diagnostic testing as a way to eliminate
the commonplace finding of “testing begetting more testing.”
Note: For an embargoed PDF, please contact Cara Graeff. For an interview with the lead author, Dr. Daniel
Mark, please contact Sarah Avery at sarah.avery@duke.edu or 919-660-1306.
2. Novel trial approaches may help to make cancer treatments more precise
Abstract: http://www.annals.org/article.aspx?doi=10.7326/M15-2413
URL goes live when the embargo lifts
Newer trial designs may help to personalize cancer treatments based on DNA alterations in tumors, rather
than on primary tumor site or stage of disease. The result could be more effective treatments. A summary of
three of these novel trial designs is published in Annals of Internal Medicine.
Traditional trial designs in oncology have proceeded along three phases of research. Phase 1 oncology trials seek to find the maximum dose tolerated by the patient’s normal tissues among patients with metastatic
disease of various primary tumor sites that were resistant to known treatments; phase 2 trials are used to evaluate antitumor drug activity in patients with advanced disease of a given primary tumor site in which other
treatments have failed; and phase 3 trials are randomized controlled trials (RCTs) comparing a new treatment
to a standard-of-care control in patients who are representative of the general population of patients with a
specified primary site and stage of cancer. However, developments in tumor biology and genomics have indicated that tumors of a primary site represent a heterogeneous collection of diseases that differ with regard
to DNA drivers. This means that conventional approaches to clinical trial design and analysis may no longer
be appropriate as clinicians look to base treatments on molecular phenotypes. This movement is known as
precision medicine.
19
The author describes new clinical trial designs in oncology that may help to advance precision medicine.
In phase 2 basket trials, which can be randomized or nonrandomized and include a single drug or multiple
individual drugs, patient eligibility is based on a defined genomic alteration rather than on primary tumor
site. For example, eligibility may require that the tumor contains a V600E mutation in the BRAF gene in a
patient with any type of solid tumor. Phase 3 enrichment designs conducted for regulatory approval have eligibility limited to patients with a single primary site of disease and genomic alteration. Approval of a drug is
accompanied by an approval of a companion diagnostic for identifying the patients who have tumors for which
the drug is effective. For example, trastuzumab was approved for breast cancer with an assay for amplification
of the HER2 gene overexpression of the HER2 protein as the companion diagnostic test. And finally, phase
3 umbrella designs involve several molecularly targeted test drugs and a population of patients with a single
primary site of disease. Phase 3 umbrella trials consist of a combination of several enrichment designs conducted with a common genomic alteration testing infrastructure. For example, in the Lung-MAP (Lung Master
Protocol-phase II/III Biomarker Driven Master Protocol for Second Line Therapy of Squamous Cell Lung Cancer) study, patients with advanced squamous cell lung cancer who have undergone one previous treatment are
screened for genomic alterations in more than 200 genes using a sequencing platform that has been analytically
validated for accuracy. As a result of this tumor characterization, patients are recommended for 1 of 5 subtrials
with the umbrella framework.
These new trial designs are promising in oncology precision medicine and have may indications in other
disease areas, as well, according to the author.
Note: For an embargoed PDF, please contact Cara Graeff. For an interview with the lead author, Dr. Richard
Simon, please contact the National Cancer Institute Press Officers at ncipressofficers@mail.nih.gov or 301-4966641.
NewsRx LLC
(2016-06-10), Little cost difference between tests to diagnose coronary heart disease, Economics Week, 153,
ISSN: 1945-6913, BUTTER® ID: 011902414
Neuroblastomas
Loss of a microRNA family, let-7, found key in neuroblastoma
By a News Reporter-Staff News Editor at Cancer Weekly – Great strides have been made in treating neuroblastoma, the most common cancer in infants and toddlers. However, advanced cases are often fatal, and children
who survive often face life-long physical and intellectual challenges related to their treatment. A study led
by researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, finds that a microRNA
called let-7 plays a central role in curbing neuroblastoma and could focus efforts to find a targeted, nontoxic
alternative to chemotherapy.
Published by Nature (Advance Online) on July 6, the study unifies several theories about neuroblastoma
and also has implications for other solid tumors in which let-7 is lost, such as Wilms tumor, lung, breast, ovarian
and cervical cancers, says first author John Powers, PhD, of the Division of Pediatric Hematology/Oncology at
Boston Children’s Hospital.
The let-7 family of microRNAs (bits of genetic code that regulate genes) is known to be involved in both stemcell differentiation and tumor suppression. Recent research had implicated LIN28B, a protein that inhibits let-7
maturation, in neuroblastoma. But the new study, through work on neuroblastoma cells and analysis of patient
data, found that LIN28B is only one of several cancer mechanisms that involve let-7 suppression.
“We’re showing that let-7 inhibition is central to the development of this disease,” Powers says. “So critical
in fact that neuroblastoma uses at least three distinct ways of eliminating it.”
Powers and colleagues first demonstrated that genetic amplification of the oncogene MYCN – producing
many thousands of copies of its mRNA – allows neuroblastomas to sequester let-7, taking it out of circulation.
“Children with a MYCN-amplification event, which occurs in about 25 percent of cases, have the poorest
prognosis,” says Powers, a senior scientist in the laboratory of George Q. Daley, MD, PhD, director of the Stem
Cell Transplantation Program at Dana-Farber/Boston Children’s. “A big question in neuroblastoma research
has been, ‘why do you need so much MYCN mRNA?’ We found that MYCN is expressed at such high levels that
it is able to sponge up otherwise functional let-7.”
Second, the researchers showed that chromosome deletions associated with neuroblastoma (in the 11q and
3p regions) are home to multiple let-7 family members and that genetic loss of let-7 also tracks with poor
outcomes.
20
“Genetic loss of let-7 was not widely appreciated in neuroblastoma,” says Powers. “People knew about chromosomal losses and were looking for a tumor suppressor, but they were mainly looking at the protein-coding
genes, not microRNAs.”
Interestingly, data from 202 patients with neuroblastoma showed that chromosomal loss of let-7 rarely
occurred in patients with MYCN amplification. The two cancer-promoting events, both suppressing let-7, were
nearly mutually exclusive, meaning most neuroblastomas involved either one or the other.
“From the cancer’s point of view, you have to deal with let-7,” says Powers. “If MYCN is not amplified, the
tumor instead just loses it genetically. In either case, let-7 is mitigated.”
Likewise, if let-7 is eliminated through chromosomal loss, MYCN amplification becomes unnecessary for
the cancer to spread. “Once a tumor has disrupted let-7 genetically, it doesn’t need to amplify MYCN because
it doesn’t need to sponge let-7,” says Powers.
Powers believes that strategies focused on restoring let-7 could provide a fresh, low-toxicity approach to
neuroblastoma and other cancers in which let-7 is lost. Let-7 itself could potentially be a drug, if the challenges
of delivery (common to all RNA therapeutics) can be overcome.
The team’s findings, if replicated in a larger number of patient samples, could also help establish genetic
typing of neuroblastomas, predicting disease severity based on LIN28B levels, genetic loss of let-7 and MYCN
amplification and perhaps guiding a precision medicine approach.
Powers also hopes to apply the findings to create better mouse models of neuroblastoma. The current models
do not account for let-7, he says.
NewsRx LLC
(2016-07-26), Loss of a microRNA family, let-7, found key in neuroblastoma, Cancer Weekly, 301, ISSN:
1532-4567, BUTTER® ID: 012100273
ECOG-ACRIN Cancer Research Group
May 31 set for NCI-MATCH precision medicine trial to resume gene testing of patients
By a News Reporter-Staff News Editor at Clinical Trials Week – Noon Eastern Time, the ECOG-ACRIN Cancer Research Group (ECOG-ACRIN) announced. The trial will be available in nearly 900 medical facilities
nationwide.
The trial opened to patient enrollment in August 2015 with 10 treatment arms and a goal to genetically
screen 3000 patients for possible treatment in the trial. Enrollment of new patients was paused in November
2015 for a planned scientific review.
The NCI-MATCH trial will determine whether treating patients with certain drugs or drug combinations
that target changes in the tumor genes will shrink the cancer, regardless of its location in the body, such as the
breast, lungs, etc. Changes in tumor genes are believed to drive cancer growth.
Treatments that show promise in the NCI-MATCH trial can then advance to larger, more definitive clinical
trials.
During the pause in enrollment of new patients, investigators analyzed many facets of this innovative study
that ignores the specific type of cancer in favor of looking for common genetic changes across tumor types.
Based upon this analysis, several changes to the trial design are now in effect:
The launch of the trial last August was met with strong support by physicians, advocates, and patients, and
as a result, patient registration for genetic screening was rapid from the start. Nearly 800 patients enrolled in
the first three months of the trial, far surpassing the original estimate of 50 patients per month.
To address the expected return to a high pace of enrollment when the trial resumes, laboratory capacity is
now in place to handle the processing of 100 patients per week.
Part of the increase in the overall screening goal to 5000 patients is the importance of selecting the right
individuals for the trial. Trial leaders will strengthen communication with enrolling physicians about the
importance of referring into the trial only those patients who have adequate function of major organs and are
able to carry out light daily physical activities.
Patients must be able to withstand being off treatment for up to six weeks because the process of genetic
testing can take up to three or four weeks to complete, starting from the time of the patient’s tumor biopsy.
Then, for patients who have a gene abnormality matching one of the 24 treatment arms, it can take up to two
additional weeks to evaluate whether they meet the eligibility requirements for entering the specific treatment
arm.
NewsRx LLC
21
(2016-06-13), May 31 set for NCI-MATCH precision medicine trial to resume gene testing of patients, Clinical Trials Week, 367, ISSN: 1543-6764, BUTTER® ID: 011823462
Multiple Myeloma Research Foundation
Multiple Myeloma Research Foundation MMRF Founder Kathy Giusti to Co-Chair Harvard
Business School/Kraft Precision Medicine Accelerator
By a News Reporter-Staff News Editor at Cancer Weekly – The Multiple Myeloma Research Foundation
(MMRF), a leader in precision medicine, announced that its founder, Kathy Giusti, whom Fortune recently
named as one of three business leaders who are disrupting medicine, has been appointed faculty co-chair of the
Kraft Precision Medicine Accelerator at Harvard Business School (HBS).
The HBS Kraft Precision Medicine Accelerator is made possible by a $20M endowment from the Kraft
Family Foundation, under the leadership of foundation President, Robert K. Kraft. The Kraft family, through
its family foundation, is committed to giving back to the community. The foundation’s primary mission includes
supporting education, healthcare, science, and the needs of underserved individuals.
“Precision medicine has the potential to revolutionize the way we prevent, diagnose, treat, and, ultimately,
cure cancer and other devastating diseases. I look forward just as much to sharing the MMRF model as I do
to learning best practices from other world-class organizations focused on this promising approach,” said Ms.
Giusti, who is also a multiple myeloma patient.
“The promise of precision medicine will only be realized if we abandon a siloed approach to research and
work collaboratively toward a greater good - two approaches Kathy Giusti has embraced and advocated in her
nearly two decades of service to the cancer community. I am certain her vision and leadership of the HBS/Kraft
Precision Medicine Accelerator will greatly improve the lives of people with cancer and other diseases,” said
Robert Kraft.
The mission of the HBS/Kraft Precision Medicine Accelerator is to speed innovation and medical breakthroughs in precision medicine, the process by which genomic information and other unique characteristics of
a person’s disease are used to predict which treatments will be most effective. The Accelerator will convene
best-in-class leaders from the business, medical, scientific, and technological communities to identify and solve
challenges slowing the advancement of precision medicine, disseminate best practices and models to overcome
these challenges, and, ultimately, enable the faster commercialization of high-impact innovations.
“Many of the biggest challenges in advancing breakthroughs promised by precision medicine are business
challenges-how to best develop and commercialize medical solutions for public benefit, and how to optimally
share data, for example. HBS is uniquely positioned to address these challenges by drawing on the ingenuity
and expertise of our faculty, alumni, and students, and by convening the leaders who can develop innovative
new models,” said HBS Dean Nitin Nohria. About the Multiple Myeloma Research FoundationThe Multiple
Myeloma Research Foundation (MMRF) was established in 1998 as a 501© (3) non-profit organization by twin
sisters Karen Andrews and Kathy Giusti, soon after Kathy’s diagnosis with multiple myeloma. The mission
of the MMRF is to relentlessly pursue innovative means that accelerate the development of next-generation
multiple myeloma treatments to extend the lives of patients and lead to a cure. As the world’s number-one
private funder of multiple myeloma research, the MMRF has raised more than $300 million since its inception
and directs nearly 90% of its total budget to research and related programming. As a result, the MMRF has
been awarded Charity Navigator’s coveted four-star rating for 11 consecutive years, the highest designation for
outstanding fiscal responsibility and exceptional efficiency. About the Harvard Business School Kraft Precision Medicine AcceleratorThe Harvard Business School Kraft Precision Medicine Accelerator is a partnership
between Harvard Business School, The Robert and Myra Kraft Family Foundation, and the Broad Institute. It
was established with a $20 million endowment from the Kraft Foundation with a focus on increasing the rate
of innovation in, and adoption of, Precision Medicine models and best practices across disease states.
The Harvard Business School Kraft Precision Medicine Accelerator will convene leaders in science, technology, and business to assess the Precision Medicine landscape, prioritize the challenges, and drive to solutions that speed innovation and cures. All knowledge will be widely disseminated throughout the Precision Medicine ecosystem through communications, case studies, and stakeholder education. For more
information, visit: HBS Kraft Precision Medicine Accelerator. View source version on businesswire.com:
http://www.businesswire.com/news/home/20160613005694/en/
NewsRx LLC
22
(2016-06-28), Multiple Myeloma Research Foundation MMRF Founder Kathy Giusti to Co-Chair Harvard
Business School/Kraft Precision Medicine Accelerator, Cancer Weekly, 301, ISSN: 1532-4567, BUTTER® ID:
011958688
Digestive System Diseases and Conditions
New Data from Chinese Academy of Sciences Illuminate Findings in Hepatocellular
Carcinoma and Genetics (Hepatocellular carcinoma cell lines retain the genomic and
transcriptomic landscapes of primary human cancers)
By a News Reporter-Staff News Editor at Gastroenterology Week – New research on Digestive System Diseases
and Conditions is the subject of a report. According to news originating from Beijing, People’s Republic of
China, by NewsRx correspondents, research stated, “Hepatocellular carcinoma (HCC) cell lines are useful in
vitro models for the study of primary HCCs. Because cell lines acquire additional mutations in culture, it is
important to understand to what extent HCC cell lines retain the genetic landscapes of primary HCCs.”
Our news journalists obtained a quote from the research from the Chinese Academy of Sciences, “Most
HCC cell lines were established during the last century, precluding comparison between cell lines and primary
cancers. In this study, 9 Chinese HCC cell lines with matched patient-derived cells at low passages (PDCs) were
established in the defined culture condition. Whole genome analyses of 4 HCC cell lines showed that genomic
mutation landscapes, including mutations, copy number alterations (CNAs) and HBV integrations, were highly
stable during cell line establishment. Importantly, genetic alterations in cancer drivers and druggable genes
were reserved in cell lines. HCC cell lines also retained gene expression patterns of primary HCCs during
in vitro culture. Finally, sequential analysis of HCC cell lines and PDCs at different passages revealed their
comparable and stable genomic and transcriptomic levels if maintained within proper passages.”
According to the news editors, the research concluded: “These results show that HCC cell lines largely
retain the genomic and transcriptomic landscapes of primary HCCs, thus laying the rationale for testing HCC
cell lines as preclinical models in precision medicine.”
For more information on this research see: Hepatocellular carcinoma cell lines retain the genomic and transcriptomic landscapes of primary human cancers. Scientific Reports , 2016;6():1-13. Scientific Reports can be
contacted at: Nature Publishing Group, Macmillan Building, 4 Crinan St, London N1 9XW, England. (Nature
Publishing Group - http://www.nature.com/ ; Scientific Reports - http://www.nature.com/srep/ )
The news correspondents report that additional information may be obtained from Z.X. Qiu, Univ Chinese
Academy Sci, Beijing 100049, People’s Republic of China. Additional authors for this research include K.K.
Zou, L.P. Zhuang, J.J. Qin, H. Li, C. Li, Z.T. Zhang, X.T. Chen, J. Cen, Z.Q. Meng, H.B. Zhang, Y.X. Li and L.J.
Hui.
NewsRx LLC
(2016-06-27), New Data from Chinese Academy of Sciences Illuminate Findings in Hepatocellular Carcinoma and Genetics (Hepatocellular carcinoma cell lines retain the genomic and transcriptomic landscapes of
primary human cancers), Gastroenterology Week, 207, ISSN: 1543-6748, BUTTER® ID: 011947884
Biochemistry
New Findings from University of Michigan Update Understanding of Biochemistry
(Multiparametric Biomechanical and Biochemical Phenotypic Profiling of Single Cancer
Cells Using an Elasticity Microcytometer)
By a News Reporter-Staff News Editor at Cancer Weekly – Current study results on Biochemistry have been
published. According to news reporting originating in Ann Arbor, Michigan, by NewsRx journalists, research
stated, “Deep phenotyping of single cancer cells is of critical importance in the era of precision medicine to
advance understanding of relationships between gene mutation and cell phenotype and to elucidate the biological nature of tumor heterogeneity. Existing microfluidic single-cell phenotyping tools, however, are limited to
phenotypic measurements of 1-2 selected morphological and physiological features of single cells.”
The news reporters obtained a quote from the research from the University of Michigan, “Herein a microfluidic elasticity microcytometer is reported for multiparametric biomechanical and biochemical phenotypic
profiling of free-floating, live single cancer cells for quantitative, simultaneous characterizations of cell size,
23
cell deformability/stiffness, and surface receptors. The elasticity microcytometer is implemented for measurements and comparisons of four human cell lines with distinct metastatic potentials and derived from different
human tissues. An analytical model is developed from first principles for the first time to convert cell deformation and adhesion information of single cancer cells encapsulated inside the elasticity microcytometer to cell
deformability/stiffness and surface protein expression.”
According to the news reporters, the research concluded: “Together, the elasticity microcytometer holds
great promise for comprehensive molecular, cellular, and biomechanical phenotypic profiling of live cancer
cells at the single cell level, critical for studying intratumor cellular and molecular heterogeneity using lowabundance, clinically relevant human cancer cells.”
For more information on this research see: Multiparametric Biomechanical and Biochemical Phenotypic
Profiling of Single Cancer Cells Using an Elasticity Microcytometer. Small , 2016;12(17):2300-2311. Small
can be contacted at: Wiley-V C H Verlag Gmbh, Postfach 101161, 69451 Weinheim, Germany. (Wiley-Blackwell
- http://www.wiley.com/ ; Small - http://onlinelibrary.wiley.com/journal/10.1002/(ISSN)
1613-6829 )
Our news correspondents report that additional information may be obtained by contacting S.H. Hu, University of Michigan, Michigan Center Integrat Res Crit Care, Ann Arbor, MI 48109, United States. Additional
authors for this research include G.Y. Liu, W.Q. Chen, X. Li, W. Lu, R.H.W. Lam and J.P. Fu.
NewsRx LLC
(2016-05-31), New Findings from University of Michigan Update Understanding of Biochemistry (Multiparametric Biomechanical and Biochemical Phenotypic Profiling of Single Cancer Cells Using an Elasticity
Microcytometer), Cancer Weekly, 260, ISSN: 1532-4567, BUTTER® ID: 011766352
Clinical Oncology
New ‘game plan’ for oncologists reflects rapid advances and need for immediate information
By a News Reporter-Staff News Editor at Health & Medicine Week – (SEATTLE) - The field of oncology is
rapidly changing, thanks to new discoveries and treatments, and patients with cancer are living longer, Journal
of Clinical Oncology, lays out a “game plan” for the American Society of Clinical Oncology that helps set the
stage for incorporating new therapies and approaches into clinical guidelines as quickly – and as accurately –
as possible.
The report also lays the groundwork for ASCO guidelines to become more of a digital resource for clinicians and introduces plans to fold the organization’s new web-based rapid-learning system, CancerLinQ, into
practice. These moves are especially important as oncology becomes increasingly focused on providing a personalized, precision medicine approach to treatment, said senior author Dr. Gary Lyman, Fred Hutchinson
Cancer Research Center researcher, breast cancer oncologist and co-director of the Hutchinson Institute for
Cancer Outcomes Research (HICOR).
“We’re in an era of such rapid changes in cancer care and the understanding of the disease – with new
therapies and new diagnostics – that we cannot afford not to have the most up-to-date evidence-based guidance
for shared decision-making in cancer treatment,” Lyman said. “CancerLinQ, will allow point-of-care clinical
decision support utilizing the latest evidence-based recommendations to provide patients with the best care.
Clinicians can access CancerLinQ from wherever they have access to the internet.”
ASCO began creating clinical practice guidelines more than 20 years ago, updating them as new treatments
and therapies became available. They are used by oncologists to provide patients with the best, evidence-based
care. Lyman, a leader in the field of precision oncology and a member of the ASCO board of directors, has
played a key role in establishing and updating the guidelines.
New guideline innovations covered in this report include:
“There are new drugs coming out every week, if not every day. There’s an overwhelming amount of information for oncologists to keep track of. It’s one thing for those of us in academic settings who see one type
of cancer, but in a practice where you’re treating all kinds of cancer, it’s a daunting, overwhelming process.
Clinical oncologists must keep up with the latest advances diagnostically and therapeutically in every cancer
they might possibly encounter, and every stage of those cancers from early to advanced, and also deal with
things like end-of-life care, survivorship and comorbidities or other medical problems,” Lyman said.
NewsRx LLC
(2016-07-22), New ‘game plan’ for oncologists reflects rapid advances and need for immediate information,
Health & Medicine Week, 2483, ISSN: 1532-4605, BUTTER® ID: 012081284
24
Personalis, Inc.
Personalis Announces Partnership with Mt. Sinai Services to Offer its ACE Platform for
Next Generation Sequencing and Interpretation to Support Tumor Molecular Profiling and
Immuno-Oncology Clinical Trials in Canada
By a News Reporter-Staff News Editor at Biotech Business Week – Personalis, Inc., a leading precision medicine
company focused on advanced NGS-based clinical diagnostic and research services announced that the company
has entered into a letter of intent (LOI) with Mount Sinai Services, Inc. of Toronto, Ontario, Canada, a global
provider of customized clinical laboratory and research services. The LOI contains broad terms for the development of a partnership framework for Personalis and Mount Sinai Services to offer the Personalis patented, and
CLIA validated ACE (Accuracy and Content Enhanced) sequencing and analysis technology for clinical diagnostic and research tumor molecular profile sequencing and analysis and immuno-oncology sequencing clinical
trials for customers throughout Canada. The ACE Platform® incorporates proprietary sample prep and sequencing processes which produce the highest levels of gene finishing and coverage of difficult-to-sequence
regions.
Personalis currently provides comprehensive genomic testing for tumor-profiling through its ACE CancerPlus™ test for solid tumors. As the largest and most comprehensive cancer panel available today, the ACE
CancerPlus test combines both DNA and RNA sequencing of over 1,400 genes, using the Personalis proprietary
ACE technology. The ACE CancerPlus Test result reports out on 181 genes of clinical relevance, producing
comprehensive tumor genomic profile data to guide therapy and ongoing research.
The ACE ImmunoID™ service offers multiple solutions for the comprehensive analysis of tumor mutation
burden and neoantigen identification including the leading ACE Cancer Exome and Transcriptome covering
over 20,000 genes and the ACE Cancer DNA/RNA Panel available with over 1,400 genes. The Personalis ACE
assays and informatics platform achieves industry leading coverage and sensitivity enabling variants to be
identified more accurately and reliably in regions that may be completely missed by typical exomes, transcriptomes, and panels. The ACE assays together with advanced informatics are CLIA validated to enable clinical
diagnostic and clinical trials use.
“We are pleased to work with Mount Sinai Services to offer the Personalis ACE technology to support
immuno-oncology clinical trials and clinical laboratory and research tumor profiling in Canada,” said John
West, CEO of Personalis. “Mount Sinai Services has been a leader in advancing personalized medicine in
Canada and understands the complexities and value of comprehensive and accurate analysis and interpretation in cancer research and clinical care. Their longstanding reputation for innovation and high quality
customer service provides Personalis with a technically robust partner in Canada.”
“As a leading, independent clinical diagnostic and research laboratory with a strong focus on precision
medicine and diagnostics, Mount Sinai Services is delighted to work with Personalis to develop this strategic partnership to advance cancer therapeutic development and clinical care,” said Dr. Azar Azad, Managing
Director of Mount Sinai Services. “We are confident that our expertise in providing customized laboratory services, combined with the capabilities of the Personalis ACE Platform will contribute to advancing translational
medicine by providing a comprehensive solution for pharma discovery and development of cancer therapies,
and personalized clinical diagnostic cancer care.” About Personalis, Inc. Personalis, Inc. (www.personalis.com)
a leading precision medicine company focused on advanced NGS-based clinical diagnostic and research services for cancer and inherited genetic disease. The company’s ACE Exome technology is designed to obtain
the most comprehensive and accurate genomic data tumor burden for tumor molecular profile and immunooncology applications. The company’s clinical laboratory is GCP compliant, CLIA licensed and CAP accredited. About Mount Sinai Services, Inc. Mount Sinai Services is a CAP/CLIA and ISO 15189 certified global
provider of customized clinical diagnostic laboratory and research services. Mount Sinai Services works closely
with world-renowned clinicians and researchers at Sinai Health System, Lunenfeld-Tanenbaum Research Institute, University of Toronto and other Canadian academic and healthcare providers to offer comprehensive
services supporting discovery and innovation in the life sciences, including medical device and diagnostic assay development and validation, clinical trial management and providing customized services to biotech and
pharma industry. Mount Sinai Services provides specialized clinical laboratory testing throughout Canada
and supports processes to achieve new assay reimbursements.
Mount Sinai Services is committed to providing its clients with superior customer service and high-quality
results based on a truly collaborative approach. For information, visit www.mountsinaiservices.com.
For more information please visit www.personalis.com. View source version on businesswire.com: http:
25
NewsRx LLC
(2016-06-20), Personalis Announces Partnership with Mt. Sinai Services to Offer its ACE Platform for
Next Generation Sequencing and Interpretation to Support Tumor Molecular Profiling and Immuno-Oncology
Clinical Trials in Canada, Biotech Business Week, 97, ISSN: 1543-6861, BUTTER® ID: 011908376
Personalized medicine leads to better outcomes for patients with cancer
By a News Reporter-Staff News Editor at Clinical Trials Week – In a meta-analysis of hundreds of clinical trials
involving thousands of patients, researchers at University of California San Diego School of Medicine report
that therapeutic approaches using precision medicine, which emphasizes the use of individual genetics to refine
cancer treatment, showed improved response and longer periods of disease remission, even in phase I trials.
The findings are published in the June 6, 2016 issue of JAMA Oncology.
After reviewing 346 phase I clinical trials involving 13,203 patients, the authors found that in precision
medicine treatment arms more than 30 percent of patients responded to treatment compared to 4.9 percent
of patients enrolled in non-personalized arms. Patients treated with precision medicine also benefited from
longer progression-free survival with a median of 5.7 months before their disease worsened compared to 2.95
months for the others.
“Our analysis shows that in the era of precision medicine, phase I clinical trials using personalized therapy
with a biomarker-based approach can do more than assessing the toxicity and side effects,” said Maria Schwaederle, PharmD, lead author and UC San Diego Moores Cancer Center researcher at the Center for Personalized
Cancer Therapy. “These early trials can result in improved outcomes for patients, even among people whose
disease is resistant to standard treatments, by selecting patients who will respond best using a personalized
approach from the start.”
The study involved 58 precision medicine treatment arms and 293 that did not. Personalized arms led to improved outcomes across tumor types. The use of a personalized approach was associated with higher response
rate of 24.5 percent in patients with solid tumors compared to 4.5 percent in non-personalized strategies. Similarly, blood cancers had a 24.5 percent response rate compared to 13.5 percent. In both tumor types, using
precision medicine gave patients a longer progression-free survival (4.1 versus 2.8 months in solid tumors and
13.6 versus 4 months in hematologic tumors).
In a sub-analysis of 234 arms testing targeted drugs, the authors found that using biomarkers to assign
patients to treatments led to response rates of 31.1 percent compared to 5.1 percent for those that did not. This
shows the importance of pairing targeted therapy with a biomarker.
Another sub-analysis of the precision medicine trials showed that while the use of both genomic and protein
biomarkers improved outcomes, genomic biomarkers performed better. Targeting genomic alterations resulted
in a 42 percent response rate compared to a 22.4 percent response if the biomarker was directed at a protein
overexpression.
“What we observed is that phase I trials can serve both to inform us on the effectiveness of new therapies as
well as identify patients likely to benefit most if a personalized approach is employed,” said Razelle Kurzrock,
MD, director of the Center for Personalized Therapy and Clinical Trials Office at Moores Cancer Center and
senior author of the paper. “Another important point is that targeted drugs in and of themselves are often quite
useless if not combined with a patient’s individual tumor biomarkers to determine whether they are likely to
benefit from a particular therapy.”
NewsRx LLC
(2016-06-20), Personalized medicine leads to better outcomes for patients with cancer, Clinical Trials Week,
127, ISSN: 1543-6764, BUTTER® ID: 011909362
26
Proteomics
Proteins key to unlocking cancer for National Cancer Moonshot
By a News Reporter-Staff News Editor at Cancer Vaccine Week – The National Cancer Moonshot initiative
needs to move beyond genomics to target the proteins that are driving cancer, according to an Inova Health
System and George Mason University collaborative paper published in the American Association for Cancer
Research.
President Barack Obama announced the National Cancer Moonshot during his 2016 State of the Union
Address. About $1 billion is expected next year to fund the initiative’s goal of achieving a decade’s worth of
research in five years by focusing on immunotherapy, genomics and combination therapies.
Thomas P. Conrads, associate director of Scientific Technologies for the Inova Schar Cancer Institute
and chief scientific officer of the Department of Defense Gynecologic Cancer Center of Excellence, partnered
with Emanuel “Chip” Petricoin, co-director of George Mason’s Center for Applied Proteomics and Molecular
Medicine, on a thought piece to draw attention to the importance of proteins in personalized medicine, especially cancer.
While the pair lauded the Moonshot initiative, they agreed it needs to center on more than genomics –
proteomics is essential.
“After all, while the genome is the information archive, it is the proteins that actually do the work of the cell
and represent the structural cellular machinery,” Petricoin and Conrads wrote. “It is the proteins that comprise
most of the biomarkers that are measured to detect cancers, constitute the antigens that drive immune response
and inter- and intracellular communications, and it is the proteins that are the drug targets for nearly every
targeted therapy that is being evaluated in cancer trials today.”
Northern Virginia’s largest health system, Inova, and Virginia’s largest public research university, George
Mason, announced a strategic partnership in December to conduct translational research and share resources
for the benefit of patients, the community and the region.
“Establishment of the Inova-George Mason University Center for Clinical Proteomics affirms the strong
commitment of these two prominent Northern Virginia institutions to the precision medicine renaissance and
the recognition that major strides will only be possible through dedicated support for and inclusion of proteomics
in this initiative,” Conrads said.
Mason researchers have used proteomics to find personalized treatments for metastatic breast cancer patients, giving new treatment options to patients who previously didn’t have any.
“The involvement of our existing clinical proteomics efforts in a variety of cutting-edge precision medicine
trials today, and the results we are seeing firsthand by including proteomics in a ‘multi-omic’ engine for precision
medicine, serve to validate our investment in the area as well as reaffirm the need to be a world leader in the
arena,” Petricoin said.
NewsRx LLC
(2016-06-06), Proteins key to unlocking cancer for National Cancer Moonshot, Cancer Vaccine Week, 38,
ISSN: 1543-6802, BUTTER® ID: 011861945
27
Oncology
Reports from National Cancer Institute Describe Recent Advances in Oncology Research
(Genome-wide association studies and epigenome-wide association studies go together in
cancer control)
By a News Reporter-Staff News Editor at Cancer Weekly – New research on Oncology is the subject of a report.
According to news originating from Rockville, Maryland, by NewsRx editors, the research stated, “Completion of
the human genome a decade ago laid the foundation for: using genetic information in assessing risk to identify
individuals and populations that are likely to develop cancer, and designing treatments based on a person’s
genetic profiling ( precision medicine). Genome-wide association studies (GWAS) completed during the past
few years have identified risk-associated single nucleotide polymorphisms that can be used as screening tools
in epidemiologic studies of a variety of tumor types.”
Our news journalists obtained a quote from the research from National Cancer Institute, “This led to the
conduct of epigenome-wide association studies (EWAS). This article discusses the current status, challenges
and research opportunities in GWAS and EWAS.”
According to the news editors, the research concluded: “Information gained from GWAS and EWAS has
potential applications in cancer control and treatment.”
For more information on this research see: Genome-wide association studies and epigenome-wide association studies go together in cancer control. Future Oncology , 2016;12(13):1645-1664. Future Oncology can be
contacted at: Future Medicine Ltd, Unitec House, 3RD Floor, 2 Albert Place, Finchley Central, London, N3
1QB, England.
The news correspondents report that additional information may be obtained from M. Verma, National
Cancer Institute, Methods & Technol Branch, Epidemiol & Genom Res Program, Div Canc Control & Populat
SciNIH, 9609 Med Center DrSuite 4E102, Rockville, MD 20850, United States.
NewsRx LLC
(2016-07-19), Reports from National Cancer Institute Describe Recent Advances in Oncology Research
(Genome-wide association studies and epigenome-wide association studies go together in cancer control), Cancer Weekly, 117, ISSN: 1532-4567, BUTTER® ID: 012066783
Oncology
Researchers at Erasmus University Medical Center Report New Data on Adrenocortical
Carcinoma (5th International ACC Symposium: Classification of Adrenocortical Cancers
from Pathology to Integrated Genomics: Real Advances or Lost in Translation?)
By a News Reporter-Staff News Editor at Cancer Weekly – Researchers detail new data in Oncology. According
to news reporting out of Rotterdam, Netherlands, by NewsRx editors, research stated, “For the clinician, despite
its rarity, adrenocortical cancer is a heterogeneous tumor both in term of steroid excess and tumor evolution.
For patient management, it is crucial to have an accurate vision of this heterogeneity, in order to use a correct
tumor classification.”
Our news journalists obtained a quote from the research from Erasmus University Medical Center, “Pathology is the best way to classify operated adrenocortical tumors: to recognize their adrenocortical nature and to
differentiate benign from malignant tumors. Among malignant tumors pathology also aims at prognosis assessment. Although progress has being made for prognosis assessment, there is still a need for improvement.
Recent studies have established the value of Ki67 for adrenocortical cancer (ACC) prognostication, aiming also
at standardization to reduce variability. The use of genomics to study adrenocortical tumors gives a very new
insight in their pathogenesis and molecular classification. Genomics studies of ACC give now a clear description of the mRNA (transcriptome) and miRNA expression profile, as well as chromosomal and methylation
alterations. Exome sequencing also established firmly the list of the main ACC driver genes. Interestingly,
genomics study of ACC also revealed subtypes of malignant tumors with different pattern of molecular alterations, associated with different outcome. This leads to a new vision of adrenocortical tumors classification
based on molecular analysis. Interestingly, these molecular classifications meet also the results of pathological
analysis.”
According to the news editors, the research concluded: “This opens new perspectives on the development
and use of various molecular tools to classify, along with pathological analysis, ACC, and guides patient management at the area of precision medicine.”
28
For more information on this research see: 5th International ACC Symposium: Classification of Adrenocortical Cancers from Pathology to Integrated Genomics: Real Advances or Lost in Translation? Hormones &
Cancer , 2016;7(1):3-8.
Our news journalists report that additional information may be obtained by contacting R.E. de Krijger, Dept.
of Pathology, Erasmus MC University Medical Center, Rotterdam, Netherlands.
NewsRx LLC
(2016-06-28), Researchers at Erasmus University Medical Center Report New Data on Adrenocortical Carcinoma (5th International ACC Symposium: Classification of Adrenocortical Cancers from Pathology to Integrated Genomics: Real Advances or Lost in Translation?), Cancer Weekly, 161, ISSN: 1532-4567, BUTTER®
ID: 011958570
Genetic Engineering
Researchers at M.D. Anderson Cancer Center Target Genetic Engineering (RAS signaling
and anti-RAS therapy: lessons learned from genetically engineered mouse models, human
cancer cells, and patient-related studies)
By a News Reporter-Staff News Editor at Health & Medicine Week – Investigators publish new report on Genetic Engineering. According to news reporting originating in Houston, Texas, by NewsRx editors, the research
stated, “Activating mutations of oncogenic RAS genes are frequently detected in human cancers. The studies
in genetically engineered mouse models (GEMMs) reveal that Kras-activating mutations predispose mice to
early onset tumors in the lung, pancreas, and gastrointestinal tract.”
The news reporters obtained a quote from the research from M.D. Anderson Cancer Center, “Nevertheless, most of these tumors do not have metastatic phenotypes. Metastasis occurs when tumors acquire additional genetic changes in other cancer driver genes. Studies on clinical specimens also demonstrated that
KRAS mutations are present in premalignant tissues and that most of KRAS mutant human cancers have comutations in other cancer driver genes, including TP53, STK11, CDKN2A, and KMT2C in lung cancer; APC,
TP53, and PIK3CA in colon cancer; and TP53, CDKN2A, SMAD4, and MED12 in pancreatic cancer. Extensive
efforts have been devoted to develop therapeutic agents that target enzymes involved in RAS posttranslational
modifications, that inhibit downstream effectors of RAS signaling pathways, and that kill RAS mutant cancer
cells through synthetic lethality. Recent clinical studies have revealed that sorafenib, a pan-RAF and VEGFR
inhibitor, has impressive benefits for KRAS mutant lung cancer patients. Combination therapy of MEK inhibitors with either docetaxel, AKT inhibitors, or PI3K inhibitors also led to improved clinical responses in
some KRAS mutant cancer patients. This review discusses knowledge gained from GEMMs, human cancer
cells, and patient-related studies on RAS-mediated tumorigenesis and anti-RAS therapy. Emerging evidence
demonstrates that RAS mutant cancers are heterogeneous because of the presence of different mutant alleles
and/or co-mutations in other cancer driver genes.”
According to the news reporters, the research concluded: “Effective subclassifications of RAS mutant cancers
may be necessary to improve patients’ outcomes through personalized precision medicine.”
For more information on this research see: RAS signaling and anti-RAS therapy: lessons learned from
genetically engineered mouse models, human cancer cells, and patient-related studies. Acta Biochimica Et
Biophysica Sinica , 2016;48(1):27-38. (Wiley-Blackwell - http://www.wiley.com/ ; Acta Biochimica Et Biophysica Sinica - http://onlinelibrary.wiley.com/journal/10.1111/(ISSN)1745-7270 )
Our news correspondents report that additional information may be obtained by contacting B. Fang, Dept.
of Thoracic and Cardiovascular Surgery, The University of Texas MD Anderson Cancer Center, Houston, TX
77030, United States.
NewsRx LLC
(2016-05-27), Researchers at M.D. Anderson Cancer Center Target Genetic Engineering (RAS signaling
and anti-RAS therapy: lessons learned from genetically engineered mouse models, human cancer cells, and
patient-related studies), Health & Medicine Week, 923, ISSN: 1532-4605, BUTTER® ID: 011735907
29
Personalized Medicine
Researchers at PLA General Hospital Discuss Findings in Personalized Medicine
(Epigenome-based personalized medicine in human cancer)
By a News Reporter-Staff News Editor at Cancer Weekly – New research on Personalized Medicine is the subject
of a report. According to news reporting from Beijing, People’s Republic of China, by NewsRx journalists,
research stated, “Cancer genome sequencing has created an opportunity for precision medicine. Thus far,
genetic alterations can only be used to guide treatment for small subsets of certain cancer types with these key
alterations.”
The news correspondents obtained a quote from the research from PLA General Hospital, “Similar to mutations, epigenetic events are equally suitable for personalized medicine. DNA methylation alterations have been
used to identify tumor-specific drug responsive markers. Methylation of MGMT sensitizes gliomas to alkylating agents is an example of epigenetic personalized medicine. Recent studies have revealed that 5-azacytidine
and decitabine show activity in myelodysplasia, lung and other cancers. There are currently at least 20 kinds
of histone deacetylase inhibitors in clinical testing.”
According to the news reporters, the research concluded: “Inhibitors targeting other epigenetic regulators
are being clinically tested, such as EZH2 inhibitor EPZ-6438.”
For more information on this research see: Epigenome-based personalized medicine in human cancer.
Epigenomics , 2016;8(1):119-33.
Our news journalists report that additional information may be obtained by contacting W. Yan, Dept. of
Gastroenterology & Hepatology, Chinese PLA General Hospital, #28 Fuxing Road, Beijing 100853, People’s
Republic of China. Additional authors for this research include J.G. Herman and M. Guo.
NewsRx LLC
(2016-06-14), Researchers at PLA General Hospital Discuss Findings in Personalized Medicine (Epigenomebased personalized medicine in human cancer), Cancer Weekly, 151, ISSN: 1532-4567, BUTTER® ID:
011833767
University of California - Los Angeles
Scientists discover biomarkers that could give cancer patients better survival estimatesl
By a News Reporter-Staff News Editor at Cancer Weekly – People with cancer are often told by their doctors
approximately how long they have to live, and how well they will respond to treatments, but what if there were
a way to improve the accuracy of doctors’ predictions?
A new method developed by UCLA scientists could eventually lead to a way to do just that, using data about
patients’ genetic sequences to produce more reliable projections for survival time and how they might respond
to possible treatments. The technique is an innovative way of using biomedical big data – which gleans patterns
and trends from massive amounts of patient information – to achieve precision medicine – giving doctors the
ability to better tailor their care for each individual patient.
The approach is likely to enable doctors to give more accurate predictions for people with many types of
cancers. In this research, the UCLA scientists studied cancers of the breast, brain (glioblastoma multiforme,
a highly malignant and aggressive form; and lower grade glioma, a less aggressive version), lung, ovary and
kidney.
In addition, it may allow scientists to analyze people’s genetic sequences and determine which are lethal
and which are harmless.
The new method analyzes various gene isoforms – combinations of genetic sequences that can produce an
enormous variety of RNAs and proteins from a single gene – using data from RNA molecules in cancer specimens. That process, called RNA sequencing, or RNA-seq, reveals the presence and quantity of RNA molecules
in a biological sample. In the method developed at UCLA, scientists analyzed the ratios of slightly different
genetic sequences within the isoforms, enabling them to detect important but subtle differences in the genetic
sequences. In contrast, the conventional analysis aggregates all of the isoforms together, meaning that the
technique misses important differences within the isoforms.
SURVIV (for “survival analysis of mRNA isoform variation”) is the first statistical method for conducting
survival analysis on isoforms using RNA-seq data, said senior author Yi Xing, a UCLA associate professor of
microbiology, immunology and molecular genetics. The research is published in the journal Nature Communications.
30
The researchers report having identified some 200 isoforms that are associated with survival time for people
with breast cancer; some predict longer survival times, others are linked to shorter times. Armed with that
knowledge, scientists might eventually be able to target the isoforms associated with shorter survival times in
order to suppress them and fight disease, Xing said.
The researchers evaluated the performance of survival predictors using a metric called C-index and found
that across the six different types of cancer they analyzed, their isoform-based predictions performed consistently better than the conventional gene-based predictions.
The result was surprising because it suggests, contrary to conventional wisdom, that isoform ratios provide a
more robust molecular signature of cancer patients than overall gene abundance, said Xing, director of UCLA’s
bioinformatics doctoral program and a member of the UCLA Institute for Quantitative and Computational
Biosciences.
“Our finding suggests that isoform ratios provide a more robust molecular signature of cancer patients in
large-scale RNA-seq datasets,” he said.
The researchers studied tissues from 2,684 people with cancer whose samples were part of the National
Institutes of Health’s Cancer Genome Atlas, and they spent more than two years developing the algorithm for
SURVIV.
According to Xing, a human gene typically produces seven to 10 isoforms.
“In cancer, sometimes a single gene produces two isoforms, one of which promotes metastasis and one of
which represses metastasis,” he said, adding that understanding the differences between the two is extremely
important in combatting cancer.
“We have just scratched the surface,” Xing said. “We will apply the method to much larger data sets, and
we expect to learn a lot more.”
NewsRx LLC
(2016-06-21), Scientists discover biomarkers that could give cancer patients better survival estimatesl, Cancer Weekly, 551, ISSN: 1532-4567, BUTTER® ID: 011921268
Seven Bridges
Seven Bridges Is Named to MIT Technology Review’s Annual 50 Smartest Companies List
By a News Reporter-Staff News Editor at Biotech Business Week – Seven Bridges, the biomedical data analysis
company, has been named to MIT Technology Review’s 2016 list of 50 Smartest Companies. To make the list, a
company must have innovative technology and a business model that is both practical and ambitious, with the
result that it has set the agenda in its field over the past 12 months.
This Smart News Release features multimedia. View the full release here: http://www.businesswire.
com/news/home/20160621006057/en/
Jason Pontin, publisher and editor in chief, states, “Each year we identify 50 companies that are ‘smart’ in
the way they create new opportunities. Some of this year’s stars are large companies that are using digital technologies to redefine industries, while others wrestle with technological changes. Also on the list are ambitious
startups looking to make their mark in an existing marketplace or create a new market entirely.”
“We are honored to be included and recognized for our role in driving precision medicine initiatives forward,” said James Sietstra, President, Seven Bridges. “Our technology helps enable scalable and collaborative
biomedical data analysis. We are excited to help researchers around the world collaborate on their work and
learn from vast quantities of genomic data.”
The list is included in MIT Technology Review’s annual business issue, which is available online now at
www.technologyreview.com and on newsstands worldwide on July 5, 2016. About MIT Technology Review
Founded at MIT in 1899, MIT Technology Review is an independent media company whose mission is to explain the commercial, social, and political impact of new technologies. Readers are a global audience of business and thought leaders, innovators and early adopters, entrepreneurs and investors. We’re first to report on
a broad range of new technologies, informing our audiences about how important breakthroughs will impact
their careers and lives. Subscribe. Follow: Twitter, Facebook, LinkedIn, Google+. About Seven Bridges Seven
Bridges is the biomedical data analysis company accelerating breakthroughs in genomics research for cancer,
drug development and precision medicine. The scalable, cloud-based Seven Bridges Platform empowers rapid,
collaborative analysis of millions of genomes in concert with other forms of biomedical data. Thousands of
researchers in government, biotech, pharmaceutical and academic labs use Seven Bridges, including three of
the largest genomics projects in the world: U.S. National Cancer Institute’s Cancer Genomics Cloud pilot, the
Million Veteran Program and Genomics England’s 100,000 Genomes Project. As the NIH’s only commercial
31
Trusted Partner, Seven Bridges authenticates and authorizes access to one of the world’s largest cancer genomics dataset. The company has offices in Cambridge, Mass.; Belgrade; London and San Francisco. View
source version on businesswire.com: http://www.businesswire.com/news/home/20160621006057/en/
NewsRx LLC
(2016-07-04), Seven Bridges Is Named to MIT Technology Review’s Annual 50 Smartest Companies List,
Seven Bridges
Seven Bridges to Lead Precision Medicine Plenary Session and Cancer Genomics Workshop
at Festival of Genomics Boston
By a News Reporter-Staff News Editor at Cancer Weekly – Seven Bridges, the biomedical data analysis company,
announced that its senior scientists and collaborators will lead several sessions at Boston’s Festival of Genomics,
to be held June 27-29 at the Boston Convention Center.
The program includes a Seven Bridges plenary session on precision medicine and a workshop providing a
hands-on experience with the Cancer Genomics Cloud (CGC), a pilot system funded by the National Cancer
Institute and powered by Seven Bridges. A third session will feature Dr. Brian O’Connor, Technical Director of
the UCSC Genomics Institute, discussing the cloud-based infrastructure for the PanCancer Analysis of Whole
Genomes (PCAWG) project.
“Cloud technology is opening the door to unprecedented levels of collaboration and scale when it comes to
genomics research,” said James Sietstra, President of Seven Bridges. “We look forward to giving Festival of
Genomics participants the opportunity to learn more about population-scale genomics projects, dive into the
rich data on the CGC, and explore how these advance our understanding of cancer.”
NewsRx LLC
(2016-07-05), Seven Bridges to Lead Precision Medicine Plenary Session and Cancer Genomics Workshop
at Festival of Genomics Boston, Cancer Weekly, 448, ISSN: 1532-4567, BUTTER® ID: 011995947
Strata Oncology
Strata Oncology Completes Series A Financing and Signs Strategic Partnership with
Thermo Fisher Scientific
By a News Reporter-Staff News Editor at Clinical Trials Week – Strata Oncology, a precision medicine company,
announced the completion of a Series A financing of $12M. The financing will fuel Strata’s state-of-the-art
tumor sequencing operation and implementation of the Strata Trial, a nation-wide effort aimed at substantially
increasing the number of enrollments in precision medicine clinical trials. Arboretum Ventures and Baird
Capital co-led the financing, with additional funding provided by Strata’s existing investor, Michigan eLab.
“For most cancer patients in the U.S., tumor sequencing is not standard of care, so patients remain unaware
of their eligibility for promising precision medicine clinical trials,” said Dan Rhodes, Ph.D., Strata Oncology
co-founder and CEO. “By providing no-cost tumor sequencing for 100,000 cancer patients, Strata intends to be
the catalyst, helping patients find the right trials and helping pharma find the right patients.”
Additionally, the company has entered into a strategic partnership with Thermo Fisher Scientific, which will
serve as the technology provider and contribute Ion S5™ XL next-generation sequencing systems, AmpliSeq™
technology, and Oncomine™ assays.
“Thermo Fisher is committed to enabling its growing list of strategic partners to drive the era of precision medicine through its targeted, next-generation sequencing technology, which will help laboratories and
clinicians provide the best oncology treatment possible in the future,” said Joe Bernardo, president of Clinical
Sequencing at Thermo Fisher.
Rhodes, who is the former co-founder and CEO of Compendia Bioscience, a cancer genomics firm that was
acquired in 2012 by Life Technologies, co-founded Strata Oncology in 2015 with oncologist Keith Flaherty, M.D.,
from Harvard Medical School and Massachusetts General, and pathologist Scott Tomlins, M.D., Ph.D., from
the University of Michigan Medical School. Flaherty is a world leader in precision oncology clinical trials and
Tomlins is an expert in tumor sequencing and molecular pathology.
Rhodes and Flaherty are joined on Strata’s Board of Directors by Jan Garfinkle (Arboretum Ventures),
Nicole Walker (Baird Capital) and Jeff Williams, founder and CEO of NeuMoDx Molecular.
32
NewsRx LLC
(2016-06-13), Strata Oncology Completes Series A Financing and Signs Strategic Partnership with Thermo
Fisher Scientific, Clinical Trials Week, 165, ISSN: 1543-6764, BUTTER® ID: 011823279
Genetics
Studies from King’s College Add New Findings in the Area of Public Health Genomics
(Shooting for the Moon or Flying Too Near the Sun? Crossing the Value Rubicon in
Precision Cancer Care)
By a News Reporter-Staff News Editor at Cancer Weekly – Investigators discuss new findings in Genetics. According to news originating from London, United Kingdom, by NewsRx correspondents, research stated, “In
his last two State of the Union addresses, President Barack Obama has focused on the need to deliver innovative solutions to improve human health, through the Precision Medicine Initiative in 2015 and the recently
announced Cancer Moonshot in 2016. Precision cancer care has delivered clear patient benefit, but even for
high-impact medicines such as imatinib mesylate (Glivec) in chronic myeloid leukaemia, the excitement at the
success of this practice-changing clinical intervention has been somewhat tempered by the escalating price of
this ‘poster child’ for precision cancer medicine (PCM).”
Our news journalists obtained a quote from the research from King’s College, “Recent studies on the costs of
cancer drugs have revealed significant price differentials, which are a major causative factor behind disparities
in the access to new generations of immunological and molecularly targeted agents. In this perspective, we will
discuss the benefits of PCM to modern cancer control, but also emphasise how increasing costs are rendering the
current approaches to integrating the paradigm of PCM unsustainable. Despite the ever increasing pressure
on cancer and health care budgets, innovation will and must continue.”
According to the news editors, the research concluded: “Value-based frameworks offer one of the most rational approaches for policymakers committed to improving cancer outcomes through a public health approach.”
For more information on this research see: Shooting for the Moon or Flying Too Near the Sun? Crossing
the Value Rubicon in Precision Cancer Care. Public Health Genomics , 2016;19(3):132-136. Public Health
Genomics can be contacted at: Karger, Allschwilerstrasse 10, Ch-4009 Basel, Switzerland. (Karger - http:
//www.karger.com/ ; Public Health Genomics - http://content.karger.com/ProdukteDB/produkte.
asp?Aktion=JournalHome&ProduktNr=224224 )
The news correspondents report that additional information may be obtained from M. Lawler, Kings Coll
London, Kings Hlth Partners, Integrated Canc Center, Inst Canc Policy, London WC2R 2LS, United Kingdom.
Additional authors for this research include D. French, R. Henderson, A. Aggarwal and R. Sullivan.
NewsRx LLC
(2016-07-26), Studies from King’s College Add New Findings in the Area of Public Health Genomics (Shooting for the Moon or Flying Too Near the Sun? Crossing the Value Rubicon in Precision Cancer Care), Cancer
Weekly, 482, ISSN: 1532-4567, BUTTER® ID: 012100433
Transgenomic, Inc.
Transgenomic Releases New Data at ASCO 2016 Confirming Concordance of ICE
COLD-PCR ICP Liquid and Tissue Biopsies
By a News Reporter-Staff News Editor at Cancer Weekly – Transgenomic, Inc. (TBIO), (NASDAQ: TBIO),
announced that it is unveiling new data at the American Society for Clinical Oncology (ASCO) 2016 Annual
Meeting further confirming the utility, speed and efficiency of its ICE COLD-PCR™ (ICP) technology for liquid
biopsy detection of tumor mutations. The company is distributing a new educational handout at the meeting
highlighting expanded concordance data showing that ICP plasma-based liquid biopsies detect all of the mutations identified using conventional tissue samples and also detect additional tumor alterations missed by conventional methods. Another educational handout presents data showing how use of ICP with Thermo Fisher’s
Veriti® thermal cycler expedites the testing of liquid biopsy samples, producing accurate results rapidly and
efficiently.
TBIO President and CEO Paul Kinnon commented, “As the highest profile cancer meeting of the year, ASCO
is an excellent venue for presenting our expanded concordance study data further confirming the accuracy
33
and superior performance of ICE COLD-PCR liquid biopsies for tumor detection and monitoring. We also are
unveiling new study data highlighting the speed and efficiency of our ICP enrichment technology when used
with a leading thermal cycler. We believe ICP’s accuracy, versatility and ease of use have the potential to
enable wide adoption of routine genomic testing in cancer research and patient care, and the new data we are
discussing at ASCO further supports the technology’s near-term clinical and commercial potential.”
At the ASCO meeting, Transgenomic is distributing an educational report, “CRC Concordance Update Stage IV CRC Sample Analysis with ICE COLD-PCRTM,” which describes the results of an expanded concordance study assessing the ability of its multiplexed ICE COLD-PCR technology to detect a key tumor mutation
in matched plasma samples compared to detection in the same patients using tissue and plasma samples analyzed using conventional Sanger sequencing methods. The expanded study included 32 patients with late stage
colorectal cancer. Use of ICP-enriched testing resulted in an overall 96.9% concordance rate, with a 94.7% concordance rate for the mutation positive samples. In addition, actionable mutations were detected in two patient
samples analyzed using ICP-enriched methods that were missed using conventional PCR with Sanger sequencing. Notably, the missed mutations were detected by ICP in both the plasma and tissue samples from these
patients.
The researchers conclude that the high concordance rates achieved in the study and the ability of the ICPenriched approach to detect relevant mutations missed by conventional methods support the clinical validity
and utility of ICP-based liquid and tissue biopsies for mutation detection and monitoring in cancer patients.
Anil Vachani, MD, MS, Associate Professor of Medicine at the Hospital of the University of Pennsylvania
and the Veteran’s Administration Medical Center and an investigator working with TBIO, commented, “The
ability to accurately and efficiently detect tumor mutations from non-invasive blood-based patient samples is
essential for realizing the potential of precision medicine approaches to transform cancer treatment. I welcome
studies that increase our confidence in the validity of new technologies such as ICE COLD-PCR to help enable
use of targeted and other precision treatment approaches.”
A second TBIO educational handout, “Simple and Effective Clinical Testing Protocol Using ICE COLDPCRTM across Targeted Cancer Gene Panels using the Veriti® Thermal Cycler and Sanger Sequencing,” details
a new study that demonstrates how combining multiplexed ICE COLD-PCR with the Thermo Fisher Veriti
thermal cycler expedites mutation detection and monitoring using liquid biopsy samples. It shows that with
the Veriti thermal cycler, ICE COLD-PCR amplifications can be grouped based on temperature and primer
annealing parameters to enable multiple amplicon enrichments in a single thermal cycler run. By combining
the flexibility of the Veriti thermal cycler, the superior enrichment power of ICP and the rapid turnaround time
of Sanger sequencing, liquid biopsy test results were generated in about four days, compared to total turnaround
times of about four weeks for conventional tissue biopsies. Rapid results are an important advantage in cancer
clinical testing and patient monitoring, where critical patient treatment decisions increasingly rely on up-todate genomic data.
The study researchers note too that these results provide an opportunity for molecular diagnostic laboratories to reevaluate the use of Sanger sequencing for confirmation of mutation detection results from nextgeneration sequencing (NGS) platforms, and point out that by using this protocol, Sanger platforms can also
be re-considered for front line mutation detection, with the potential to produce accurate results more rapidly
and cost effectively than NGS platforms.
ICE COLD-PCR achieves its ultra-high sensitivity through selective amplification of mutant DNA. The
result is up to a 500-fold increase in sensitivity in identifying mutations with the most precise sequence alteration detection rates available. ICP was originally developed by the laboratory of Dr. Mike Makrigiorgos at the
Dana-Farber Cancer Institute, which has exclusively licensed rights to the technology to Transgenomic.
NewsRx LLC
(2016-06-21), Transgenomic Releases New Data at ASCO 2016 Confirming Concordance of ICE COLD-PCR
ICP Liquid and Tissue Biopsies, Cancer Weekly, 614, ISSN: 1532-4567, BUTTER® ID: 011921314
34
Transgenomic, Inc.
Transgenomic Study Data Shows 100% Concordance between ICE COLD-PCR™ ICP Liquid
Biopsies and Conventional Tissue Biopsy Results; ICP Also Identifies More Tumor
Mutations Than Conventional Methods
By a News Reporter-Staff News Editor at Clinical Trials Week – Transgenomic, Inc. (NASDAQ:TBIO), announced new concordance study data confirming the superior performance of its ICE COLD-PCR™ (ICP) enrichment technology over standard biopsied tissue PCR results for detection of tumor mutations in cancer
patients. The data show that plasma-based liquid biopsy testing using ICP is 100% concordant with an established approach that uses standard PCR. The matched patient data further indicate that the ICP-enabled
liquid biopsies detected more tumor mutations than tissue biopsy testing using standard PCR. Transgenomic
is in the process of submitting the study to a peer-reviewed scientific publication.
The concordance study was reviewed by Anil Vachani, MD, MS, Associate Professor of Medicine at the
Hospital of the University of Pennsylvania and the Veteran’s Administration Medical Center, and a Director
of Clinical Research at the University of Pennsylvania Perelman School of Medicine. Dr. Vachani’s research
includes the study of methods to optimize the use of targeted cancer therapies. Dr. Vachani noted, “High
sensitivity detection of genetic biomarkers from cell free DNA has the potential to provide guidance to clinical
oncologists for personalized, precision cancer treatment. Liquid biopsies are a source of genetic information
when there is no available tumor tissue and they make it possible to monitor treatment effectiveness and
the emergence of drug resistance on an ongoing basis, thereby enabling more effective real-time treatment
decisions. This can be critical for patients with metastatic disease. Based on these initial encouraging results,
ICP may be a useful technology for helping to facilitate the broad use of cell free DNA liquid biopsies in cancer.
I look forward to seeing the results of further confirmatory studies now underway.”
In this study, Transgenomic analyzed 22 matched samples from chemo-naive late-stage colorectal cancer
(CRC) patients. The purpose was to compare the concordance between tissue biopsy results using traditional
PCR and Sanger sequencing with plasma liquid biopsy results obtained using ICE COLD-PCR and Sanger
sequencing. The study also compared mutation detection results from the matched tissue/FFPE samples analyzed with Sanger sequencing and either standard PCR or ICE COLD-PCR.
The concordance between the conventional tumor biopsy results and the matched ICP-enriched plasma sample liquid biopsy results was 100%. In addition, the ICP-enriched biopsies identified seven mutations missed by
the conventional PCR approach–four additional mutations were identified in the ICP-enriched plasma samples
and three additional mutations were identified in tissue samples that were analyzed with ICE COLD-PCR.
Transgenomic President and CEO Paul Kinnon commented, “We are delighted with the latest results from
our studies, which show 100% concordance between ICE COLD-PCR-based liquid biopsy results and tissuebased results from the same patients, as well as ICP’s superior performance in identifying tumor mutations
that were missed by conventional PCR methods. This new data further validates the clinical utility of ICP as a
robust method to detect and monitor actionable mutations from a liquid biopsy sample, an essential component
needed for adoption of precision medicine. We expect to submit additional data to peer reviewed scientific
settings in the coming months.”
Transgenomic’s ICP cancer assays exponentially amplify targeted regions of exons that have been determined to be predictive and/or prognostic for many types of cancer, including colorectal cancer, melanoma, breast
cancer and non-small cell lung cancer. The 17-target mutation panel matches the set of mutation data currently
used routinely in clinical settings for identifying and selecting therapeutic or clinical trial options for patients
being treated for multiple cancer types. Additional mutations are being added as the science advances.
ICE COLD-PCR achieves its ultra-high sensitivity through selective amplification of mutant DNA. The
result is up to a 500-fold increase in sensitivity in identifying mutations with the most precise sequence alteration detection rates available. ICP was originally developed by the laboratory of Dr. Mike Makrigiorgos at the
Dana-Farber Cancer Institute, which has exclusively licensed rights to the technology to Transgenomic.
NewsRx LLC
(2016-05-30), Transgenomic Study Data Shows 100% Concordance between ICE COLD-PCR™ ICP Liquid
Biopsies and Conventional Tissue Biopsy Results; ICP Also Identifies More Tumor Mutations Than Conventional Methods, Clinical Trials Week, 1945, ISSN: 1543-6764, BUTTER® ID: 011749414
35
Chapter 3
Cardiology
Cardiovascular Research
AHA announces strategic collaboration with Amazon Web Services to advance precision
cardiovascular medicine with AWS cloud
By a News Reporter-Staff News Editor at Journal of Engineering – DALLAS - The American Heart Association
(AHA) announces a new collaboration with Amazon Web Services, Inc. (AWS), a leading cloud computing infrastructure, to enable and advance discoveries in cardiovascular science and medicine using cloud technology.
The collaboration with AWS marks a major new initiative for the AHA’s Institute for Precision Cardiovascular
Medicine. The Institute aims to advance the science and practice of precision medicine to improve cardiovascular health in both individuals and populations by fostering research that takes into account the systems biology
arising from a person’s genes, environment and lifestyle.
Driven by advanced methods of aggregating, integrating and analyzing patient data, precision cardiovascular medicine has the potential to improve, preserve and prolong health, as well as ultimately reduce overall
healthcare costs. It represents a clear improvement from the current approach, which–despite major advances–
still focuses largely on managing symptoms and serious events when they occur.
“The promise of precision cardiovascular medicine and care can be realized when research and technology
come together to deliver new insights,” said AHA CEO Nancy Brown. “The AHA and AWS collaboration will
unite the global research community to accelerate discovery in cardiovascular health and usher in a new era
of tailored prevention and treatment that will help patients and lessen the global burden of cardiovascular
disease.”
The AHA–with its commitment to patients, basic, clinical and population science, and to evidence-based clinical practice–has allocated $30 million over five years to launch The Institute and is spearheading a fundraising
campaign designed to generate $100-$200 million for further development. As an ambitious next step, AHA
has announced a new Data Grant Portfolio, awarding 14 grants over the course of 12 months, focused in four areas: Data Mining, Methods Validation, Innovative Development and Fellowships. The competitive grants and
fellowships are aimed at fostering cross-disciplinary learning, research and collaboration among a wide variety
of professionals across biomedical research, including computational biology and computer science. AWS is
providing credits that can be used by grant recipients for free access to AWS services that can be used toward
computational storage and analysis on AWS, as part of the AHA Data Grant Portfolio. The AHA will supply
the funding for research expenses including salaries. Cash incentives will also be awarded to the grantees in
each area with the best ideas for enhancing and enabling data discovery. These data grants complement a full
portfolio of precision medicine research awards that the AHA has funded since 2014.
“AWS is uniquely positioned to provide scalable, cost-efficient solutions for the scientific community, while
delivering the industry-shaping technology and high-performance computing necessary to facilitate the most
demanding research projects,” said Teresa Carlson, Vice President Worldwide Public Sector, AWS, Inc. “We’re
honored to be the AHA’s collaborator on this incredibly important endeavor to improve cardiovascular health
worldwide.”
Through these grants and fellowships, the AHA hopes to enable the scientific, mathematics and technology
community to discover solutions to overcome the current obstacles in accessing and utilizing data. Through
these solutions, the strategic collaboration between AHA and AWS strives to improve cardiovascular health
and wellbeing around the world.
36
Data Mining Grants are aimed at uncovering patterns and knowledge within existing data sets that will
inform standards and protocols for organizing and categorizing current and future cardiovascular diseasebased
data. These grants will also focus on data harmonization steps necessary for data mining.
Methods Validation Grants will focus on validating pre-existing methods including algorithms and analytic
tools used to maximize the use of data in predicting outcomes.
Innovative Development Grants will focus on developing tools that enrich our ability to identify novel approaches and/or tools to analyze data.
Fellowship Awards are intended to cross-train scientists interested in learning computational biology or
more information about specific cohorts. The data fellowships aim to establish a future generation of researchers with enhanced skills and capabilities.
Applicants can learn more and apply on the AHA website: http://institute.heart.org . More information on deadlines for submission for each grant and fellowship within the Data Grant Portfolio are listed on
the AHA’s Institute website at http://institute.heart.org .
NewsRx LLC
(2016-07-25), AHA announces strategic collaboration with Amazon Web Services to advance precision
cardiovascular medicine with AWS cloud, Journal of Engineering, 277, ISSN: 1945-872X, BUTTER® ID:
012094290
Cardiovascular Diseases and Conditions
Findings on Thrombosis Reported by Investigators at University of Pretoria (Novel
Diagnostic and Monitoring Tools in Stroke: an Individualized Patient-Centered Precision
Medicine Approach)
By a News Reporter-Staff News Editor at Hematology Week – Fresh data on Cardiovascular Diseases and
Conditions are presented in a new report. According to news reporting originating from Arcadia, South Africa,
by NewsRx correspondents, research stated, “Central to the pathogenesis of ischaemic stroke are the normally
protective processes of platelet adhesion and activation. Experimental evidence has shown that the ligandreceptor interactions in ischaemic stroke represent a thrombo-inflammatory cascade, which presents research
opportunities into new treatment.”
Our news editors obtained a quote from the research from the University of Pretoria, “However, as antiplatelet drugs have the potential to cause severe side effects in ischaemic stroke patients (as well as other
vascular disease patients), it is important to carefully monitor the risk of bleeding and risk of thrombus in
patients receiving treatment. Because thrombo-embolic ischaemic stroke is a major health issue, we suggest
that the answer to adequate treatment is based on an individualized patient-centered approach, inline with the
latest NIH precision medicine approach. A combination of viscoelastic methodologies may be used in a personalized patient-centered regime, including thromboelastography (TEG®) and the lesser used scanning electron
microscopy approach (SEM). Thromboelastography provides a dynamic measure of clot formation, strength,
and lysis, whereas SEM is a visual structural tool to study patient fibrin structure in great detail. Therefore,
we consider the evidence for TEG® and SEM as unique means to confirm stroke diagnosis, screen at-risk patients, and monitor treatment efficacy. Here we argue that the current approach to stroke treatment needs to
be restructured and new innovative thought patterns need to be applied, as even approved therapies require
close patient monitoring to determine efficacy, match treatment regimens to each patient’s individual needs,
and assess the risk of dangerous adverse effects. TEG® and SEM have the potential to be a useful tool and
could potentially alter the clinical approach to managing ischaemic stroke. As envisaged in the NIH precision
medicine approach, this will involve a number of role players and innovative new research ideas, with benefits
that will ultimately only be realized in a few years.”
According to the news editors, the research concluded: “Therefore, with this ultimate goal in mind, we
suggest that an individualized patient-orientated approach is now available and therefore already within our
ability to use.”
For more information on this research see: Novel Diagnostic and Monitoring Tools in Stroke: an Individualized Patient-Centered Precision Medicine Approach. Journal of Atherosclerosis and Thrombosis ,
2016;23(5):493-504. Journal of Atherosclerosis and Thrombosis can be contacted at: Japan Atherosclerosis
Soc, Nichinai-Kaikan B1, 3-28-8 Hongo Bunkyo-Ku, Tokyo, 113-0033, Japan.
37
The news editors report that additional information may be obtained by contacting S. de Villiers, University
of Pretoria, Fac Hlth Sci, Dept. of Physiol, Private Bag x323, ZA-0007 Arcadia, South Africa. Additional authors
for this research include A. Swanepoel, J. Bester and E. Pretorius.
NewsRx LLC
(2016-06-27), Findings on Thrombosis Reported by Investigators at University of Pretoria (Novel Diagnostic
and Monitoring Tools in Stroke: an Individualized Patient-Centered Precision Medicine Approach), Hematology
Cardiovascular Diseases
Investigators from Mayo Clinic Have Reported New Data on Cardiovascular Diseases
(Precision Medicine, Cardiovascular Disease and Hunting Elephants)
By a News Reporter-Staff News Editor at Cardiovascular Week – Current study results on Cardiovascular
Diseases have been published. According to news reporting out of Rochester, Minnesota, by NewsRx editors,
the research stated, “Precision medicine postulates improved prediction, prevention, diagnosis and treatment of
disease based on patient specific factors especially DNA sequence (i.e., gene) variants. Ideas related to precision
medicine stem from the much anticipated ‘genetic revolution in medicine’ arising seamlessly from the human
genome project (HGP).”
Our news journalists obtained a quote from the research from Mayo Clinic, “In this essay I deconstruct the
concept of precision medicine and raise questions about the validity of the paradigm in general and its application to cardiovascular disease. Thus far precision medicine has underperformed based on the vision promulgated by enthusiasts. While niche successes for precision medicine are likely, the promises of broad based
transformation should be viewed with skepticism. Open discussion and debate related to precision medicine
are urgently needed to avoid misapplication of resources, hype, iatrogenic interventions, and distraction from
established approaches with ongoing utility.”
According to the news editors, the research concluded: “Failure to engage in such debate will lead to negative
unintended consequences from a revolution that might never come.”
For more information on this research see: Precision Medicine, Cardiovascular Disease and Hunting Elephants. Progress in Cardiovascular Diseases , 2016;58(6):651-660. Progress in Cardiovascular Diseases can
be contacted at: W B Saunders Co-Elsevier Inc, 1600 John F Kennedy Boulevard, Ste 1800, Philadelphia,
PA 19103-2899, USA. (Elsevier - www.elsevier.com; Progress in Cardiovascular Diseases - http://www.
journals.elsevier.com/progress-in-cardiovascular-diseases/ )
Our news journalists report that additional information may be obtained by contacting M.J. Joyner, Mayo
Clinic, Dept. of Anesthesiol, Rochester, MN 55905, United States.
NewsRx LLC
(2016-06-20), Investigators from Mayo Clinic Have Reported New Data on Cardiovascular Diseases (Precision Medicine, Cardiovascular Disease and Hunting Elephants), Cardiovascular Week, 49, ISSN: 1543-6845,
BUTTER® ID: 011908656
38
Khush F. Mehta Joins CardioDx as Chief Executive Officer
By a News Reporter-Staff News Editor at Cardiovascular Week – CardioDx, Inc., a molecular diagnostics company specializing in cardiovascular genomics, announced the appointment of Khush Mehta as President, Chief
Executive Officer, and a member of the Board of Directors of CardioDx. With a proven track record for commercial success and execution in the global healthcare industry, Mehta will help drive the organization to the
next level of commercial growth, value creation for its stakeholders, and the expansion of the company’s product portfolio through innovation and partnerships. He will focus on overseeing the day-to-day operations and
accelerating profitable growth in the near and long-term. As CEO, Mehta succeeds David Levison, who has
been named the Chief Strategy Officer of the company.
With more than 20 years in the healthcare industry, Mehta brings a wealth of experience in strategic business development and operations to CardioDx. Prior to joining CardioDx, he served as the Global Head of
the Healthcare Enterprise Solutions at Siemens. Mehta has also held additional executive leadership roles at
Siemens Healthcare, including Chief Strategy Officer and Head of Siemens Healthcare Asia Pacific. He earned
a Bachelor and a Master of Commerce and Economics at the Sydenham College of Commerce and Economics
in Mumbai, India along with a Master of Business Administration in competitive strategies and international
finance from Yale University.
“This is an exciting time of change within healthcare. Khush is a proven leader with a track record for execution, clear business vision, and a demonstrated record for driving growth and profitability for companies,”
said Ajit Singh, Chairman of the Board. “With his extensive years of leadership experience, Khush has a deep
understanding of the global healthcare marketplace and its supply chain, and possesses strong operational acumen to drive commercial success in the U.S. and international markets. Having worked with Khush previously,
I know that his skills will accelerate the next phase of expanded growth and innovation.”
“I am honored and excited to lead CardioDx in advancing the commercialization of the Corus CAD test
during this transformational time within the healthcare industry,” said Khush Mehta, Chief Executive Officer
of CardioDx. “The PROMISE trial substudy recently presented at the 65th American College of Cardiology
Annual Scientific Meeting further underscored the science behind our test that gives physicians an effective
solution for clinical decision-making for obstructive coronary artery disease.1 I look forward to working with
the entire CardioDx team as we embark on this journey for improving the quality of cardiovascular care.”
As the Chief Strategy Officer, Levison, the founder of CardioDx, will lead the company in business development, health payer coverage, and next generation technology. “Since the company was founded in 2004,
CardioDx has had a strong commitment to improving healthcare quality and efficiency through genomic technologies and expanding patient access to new diagnostic tests that provide precision medicine in cardiovascular
medicine,” said David Levison, Chief Strategy Officer of CardioDx. “With that continued promise, I am excited
to focus my attention in the clinical and genomic diagnostics space and look forward to supporting further
expanded patient access and the delivery of next generation products to patients worldwide.”
“I am very pleased that David will continue to add his industry knowledge and expertise as we embark
on the next phase of growth. As the Founder and CEO, his leadership for more than a decade has taken the
company from concept to having served more than 175,000 patients and thousands of clinicians in a dynamic
healthcare environment. We are grateful to David’s dedication to the organization, and I look forward to his
future contributions,” said Ajit Singh.
NewsRx LLC
(2016-06-13), Khush F. Mehta Joins CardioDx as Chief Executive Officer, Cardiovascular Week, 170, ISSN:
1543-6845, BUTTER® ID: 011822804
39
Stem Cell Research
Researchers from Stanford University Report Details of New Studies and Findings in the
Area of Stem Cell Research (Induced pluripotent stem cells: at the heart of cardiovascular
precision medicine)
By a News Reporter-Staff News Editor at Cardiovascular Week – Data detailed on Stem Cell Research have been
presented. According to news reporting originating in Stanford, California, by NewsRx journalists, research
stated, “The advent of human induced pluripotent stem cell (hiPSC) technology has revitalized the efforts in
the past decade to realize more fully the potential of human embryonic stem cells for scientific research. Adding
to the possibility of generating an unlimited amount of any cell type of interest, hiPSC technology now enables
the derivation of cells with patient-specific phenotypes.”
The news reporters obtained a quote from the research from Stanford University, “Given the introduction
and implementation of the large-scale Precision Medicine Initiative, hiPSC technology will undoubtedly have
a vital role in the advancement of cardiovascular research and medicine. In this Review, we summarize the
progress that has been made in the field of hiPSC technology, with particular emphasis on cardiovascular
disease modelling and drug development.”
According to the news reporters, the research concluded: “The growing roles of hiPSC technology in the
practice of precision medicine will also be discussed.”
For more information on this research see: Induced pluripotent stem cells: at the heart of cardiovascular
precision medicine. Nature Reviews Cardiology , 2016;13(6):333-349. Nature Reviews Cardiology can be contacted at: Nature Publishing Group, 75 Varick St, 9TH Flr, New York, NY 10013-1917, USA. (Nature Publishing
Group - http://www.nature.com/ ; Nature Reviews Cardiology - http://www.nature.com/nrcardio/ )
Our news correspondents report that additional information may be obtained by contacting I.Y. Chen, Stanford University, Dept. of Radiol, Sch Med, Stanford, CA 94305, United States. Additional authors for this
research include E. Matsa and J.C. Wu.
NewsRx LLC
(2016-06-13), Researchers from Stanford University Report Details of New Studies and Findings in the
Area of Stem Cell Research (Induced pluripotent stem cells: at the heart of cardiovascular precision medicine),
Cardiovascular Week, 294, ISSN: 1543-6845, BUTTER® ID: 011822935
Heart Disorders and Diseases
Researchers from Uppsala University Describe Findings in Atrial Fibrillation and Genetics
(The changing circumstance of atrial fibrillation - progress towards precision medicine)
By a News Reporter-Staff News Editor at Cardiovascular Week – New research on Heart Disorders and Diseases is the subject of a report. According to news reporting from Uppsala, Sweden, by NewsRx journalists,
research stated, “The prevalence of atrial fibrillation (AF) in the general population is between 1% and 2% in
the developed world and is higher in men than in women. The arrhythmia occurs much more commonly in the
elderly, and the estimated lifetime risk of developing AF is one in four for men and women aged 40 years and
above.”
The news correspondents obtained a quote from the research from Uppsala University, “Projected data from
multiple population-based studies in the USA and Europe predict a two- to threefold increase in the number
of AF patients by 2060. The high lifetime risk of AF and increased longevity underscore the important public
health burden posed by this arrhythmia worldwide. AF has multiple aetiologies and a broad variety of presentations. The primary pathologies underlying or promoting the occurrence of AF vary more than for any
other cardiac arrhythmia, ranging from autonomic imbalance to organic heart disease and metabolic disorders, such as diabetes mellitus, metabolic syndrome, hyperthyroidism and kidney disease, and lifestyle factors
such as smoking, alcohol consumption and participation in endurance sports. Biomarkers are increasingly
being investigated and, together with clinical and genetic factors, will eventually lead to a clinically valuable
detailed classification of AF which will also incorporate pathophysiological determinants and mechanisms of
the arrhythmia.”
According to the news reporters, the research concluded: “In turn, this will allow the development and
application of precision medicine to this troublesome arrhythmia.”
For more information on this research see: The changing circumstance of atrial fibrillation - progress towards precision medicine. Journal of Internal Medicine , 2016;279(5):412-427. Journal of Internal Medicine
40
can be contacted at: Wiley-Blackwell, 111 River St, Hoboken 07030-5774, NJ, USA. (Wiley-Blackwell - http:
//www.wiley.com/ ; Journal of Internal Medicine - http://onlinelibrary.wiley.com/journal/10.
1111/(ISSN)1365-2796 )
Our news journalists report that additional information may be obtained by contacting A.J. Camm, Uppsala
University, Inst Med Sci, Dept. of Cardiol, Uppsala, Sweden. Additional authors for this research include I.
Savelieva, T. Potpara, G. Hindriks, L. Pison and C. Blomstrom-Lundqvist.
NewsRx LLC
(2016-05-30), Researchers from Uppsala University Describe Findings in Atrial Fibrillation and Genetics
(The changing circumstance of atrial fibrillation - progress towards precision medicine), Cardiovascular Week,
628, ISSN: 1543-6845, BUTTER® ID: 011747054
Researchers’ Work from Harvard University Focuses on Cardiovascular Research (Big data
analytics to improve cardiovascular care: promise and challenges)
By a News Reporter-Staff News Editor at Information Technology Newsweekly – Investigators publish new report on Cardiovascular Research. According to news originating from Boston, Massachusetts, by VerticalNews
correspondents, research stated, “The potential for big data analytics to improve cardiovascular quality of care
and patient outcomes is tremendous. However, the application of big data in health care is at a nascent stage,
and the evidence to date demonstrating that big data analytics will improve care and outcomes is scant.”
Our news journalists obtained a quote from the research from Harvard University, “This Review provides an
overview of the data sources and methods that comprise big data analytics, and describes eight areas of application of big data analytics to improve cardiovascular care, including predictive modelling for risk and resource
use, population management, drug and medical device safety surveillance, disease and treatment heterogeneity, precision medicine and clinical decision support, quality of care and performance measurement, and public
health and research applications. We also delineate the important challenges for big data applications in cardiovascular care, including the need for evidence of effectiveness and safety, the methodological issues such as
data quality and validation, and the critical importance of clinical integration and proof of clinical utility.”
According to the news editors, the research concluded: “If big data analytics are shown to improve quality of
care and patient outcomes, and can be successfully implemented in cardiovascular practice, big data will fulfil
its potential as an important component of a learning health-care system.”
For more information on this research see: Big data analytics to improve cardiovascular care: promise and
challenges. Nature Reviews Cardiology , 2016;13(6):350-359. Nature Reviews Cardiology can be contacted at:
Nature Publishing Group, 75 Varick St, 9TH Flr, New York, NY 10013-1917, USA. (Nature Publishing Group http://www.nature.com/ ; Nature Reviews Cardiology - http://www.nature.com/nrcardio/ )
The news correspondents report that additional information may be obtained from J.S. Rumsfeld, Harvard
University, TH Chan Sch Public Hlth, 677 Huntington Ave, Boston, MA 02115, United States. Additional
authors for this research include K.E. Joynt and T.M. Maddox.
NewsRx LLC
(2016-06-14), Researchers’ Work from Harvard University Focuses on Cardiovascular Research (Big data
analytics to improve cardiovascular care: promise and challenges), Information Technology Newsweekly, 227,
ISSN: 1944-1800, BUTTER® ID: 011839773
41
Sudden Cardiac Death
Study Results from McGill University Broaden Understanding of Sudden Cardiac Death
(Predicting the risk of sudden cardiac death)
By a News Reporter-Staff News Editor at Cardiovascular Week – Investigators publish new report on Sudden
Cardiac Death. According to news reporting from Montreal, Canada, by NewsRx journalists, research stated,
“Sudden cardiac death (SCD) is the result of a change of cardiac activity from normal (typically sinus) rhythm
to a rhythm that does not pump adequate blood to the brain. The most common rhythms leading to SCD are
ventricular tachycardia (VT) or ventricular fibrillation (VF).”
The news correspondents obtained a quote from the research from McGill University, “These result from
an accelerated ventricular pacemaker or ventricular reentrant waves. Despite significant efforts to develop
accurate predictors for the risk of SCD, current methods for risk stratification still need to be improved. In
this article we briefly review current approaches to risk stratification. Then we discuss the mathematical basis
for dynamical transitions (called bifurcations) that may lead to VT and VF. One mechanism for transition to
VT or VF involves a perturbation by a premature ventricular complex (PVC) during sinus rhythm. We describe
the main mechanisms of PVCs (reentry, independent pacemakers and abnormal depolarizations). An emerging
approach to risk stratification for SCD involves the development of individualized dynamical models of a patient
based on measured anatomy and physiology.”
According to the news reporters, the research concluded: “Careful analysis and modelling of dynamics of
ventricular arrhythmia on an individual basis will be essential in order to improve risk stratification for SCD
and to lay a foundation for personalized (precision) medicine in cardiology.”
For more information on this research see: Predicting the risk of sudden cardiac death. Journal of
Physiology-London , 2016;594(9):2445-2458. Journal of Physiology-London can be contacted at: WileyBlackwell, 111 River St, Hoboken 07030-5774, NJ, USA.
Our news journalists report that additional information may be obtained by contacting C. Lerma, McGill
University, Dept. of Physiol, 3655 Prom Sir William OslerOff 1118, Montreal, PQ H3G 1Y6, Canada.
NewsRx LLC
(2016-06-06), Study Results from McGill University Broaden Understanding of Sudden Cardiac Death
(Predicting the risk of sudden cardiac death), Cardiovascular Week, 493, ISSN: 1543-6845, BUTTER® ID:
011860539
42
Chapter 4
Precision for Medicine
ACT Oncology Wins the PACT 2016 Enterprise Award for Emerging Healthcare Companies
By a News Reporter-Staff News Editor at Biotech Business Week – ACT Oncology announced it has been
awarded the Philadelphia Alliance for Capital and Technologies (PACT) 2016 Enterprise Award for Emerging Healthcare companies. The Enterprise Awards, held annually, are the region’s most prestigious business
honors for innovative life sciences and technology companies, leaders, and entrepreneurs.
The Enterprise Healthcare Emerging Award recognizes companies that are providing an innovative technology solution that has the potential to make a significant and positive impact on a clinical problem in healthcare.
The 20(th) Annual Enterprise Awards comprised 400 companies vying for awards in 10 categories.
“We’re thrilled to have our work recognized by the Philadelphia Alliance for Capital and Technologies,”
said Patricia Devitt, president, ACT Oncology. “By focusing on oncology clinical trial execution for biotech,
pharmaceutical, and nonprofit oncology organizations, our solutions help clients succeed in this new era of
precision medicine.”
ACT Oncology provides drug development services focused on the design and execution of clinical trials
on behalf of innovative life sciences companies and not-for-profit patient organizations. They are an industry
leader in oncology clinical trials with unparalleled experience conducting studies across all tumor types and
hematology indications.
Recently acquired by Precision for Medicine, Precision and ACT Oncology are continuing to innovate by creating the first comprehensive, fully integrated precision oncology clinical development solution, combining the
highest standards in clinical trial management with the most advanced biomarker capabilities and processes.
ACT Oncology surpassed the criteria for eligibility and is particularly known for their dedication to advancements in oncology research, their highly seasoned and well-retained study teams, and their consistent
performance in clinical trial execution, resulting in a high rate of repeat business within the small to mid-sized
biotech industry.
PACT is a leading resource for fast-growing companies and a driver of entrepreneurship and innovation in
the Philadelphia region. The Enterprise Awards are just one facet of PACT, which aims to create a strategic
hub for the region’s emerging growth companies in the information technology, clean technology, and healthcare
industries, manifesting in a competitive edge for the Greater Philadelphia region.
NewsRx LLC
(2016-05-30), ACT Oncology Wins the PACT 2016 Enterprise Award for Emerging Healthcare Companies,
43
Amplion
Biomarkers Triple Clinical Trial Success Rate According To Largest Study Of Its Kind
By a News Reporter-Staff News Editor at Clinical Trials Week – Amplion (inventors of BiomarkerBase™),
Biomedtracker, and BIO announces the publication of the largest study of clinical drug development success
rates ever. The report examines ˜10,000 clinical programs by therapeutic area as well as three unique categories: rare diseases, chronic high prevalence diseases, and programs utilizing selection biomarkers. The results show dramatic differences between disease groups and modalities to be presented during the “The State
of the Innovation Industry” session at BIO International in San Francisco on June 6.
“The dramatic increase in Likelihood of Approval (LOA) when utilizing biomarkers from 1 in 10 to 1 in
4 is really the big takeaway in this report,” said Dave Thomas, Senior Director of Industry Research for BIO.
“Combining Amplion’s BiomarkerBase with BioMedTracker’s clinical transition records enabled us, for the first
time, to quantify the benefit of using selection biomarkers in drug development. In combination with the rare
disease section of the report, these results appear to be revealing the overall strength in targeting well-defined,
homogenous patient populations.”
This study was possible due to the clinical program monitoring and data entry by Informa’s Biomedtracker
service. “BIO has long partnered with BioMedTracker to analyze clinical development successes and failures,”
said Michael Hay, Head of Intelligence Products at Informa. “Now by partnering with Amplion we have been
able to analyze the effects of biomarkers in clinical trial success and show in detail the substantial positive
effect a good biomarker strategy can have on successful development outcomes.”
Having real data to tie LOA to use of biomarkers will influence the advancement of precision medicine.
“Tripling your LOA through the use of selection biomarkers is not only impressive, but it makes a clear statement about where the future of drug development is headed,” said John Audette, Founder and President of
Amplion. “As the use of patient selection biomarkers increases, and drug developers realize higher clinical success, this will also expedite the identification and validation of companion diagnostics, another transformative
component of the precision medicine landscape.”
Amplion will talk about the biomarker data that contributed to the report findings during their upcoming
webinar, “Triple your clinical trial success rate with biomarkers,” on June 21st.
NewsRx LLC
(2016-06-06), Biomarkers Triple Clinical Trial Success Rate According To Largest Study Of Its Kind, Clinical Trials Week, 277, ISSN: 1543-6764, BUTTER® ID: 011861063
Clinical Research
Findings from University of Paris Provide New Insights into Clinical Trials and Studies
(Treatment Algorithms Based on Tumor Molecular Profiling: The Essence of Precision
Medicine Trials)
By a News Reporter-Staff News Editor at Clinical Trials Week – Investigators publish new report on Clinical
Research. According to news reporting out of Paris, France, by NewsRx editors, research stated, “With the advent of high-throughput molecular technologies, several precision medicine (PM) studies are currently ongoing
that include molecular screening programs and PM clinical trials. Molecular profiling programs establish the
molecular profile of patients’ tumors with the aim to guide therapy based on identified molecular alterations.”
Our news journalists obtained a quote from the research from the University of Paris, “The aim of prospective
PM clinical trials is to assess the clinical utility of tumor molecular profiling and to determine whether treatment selection based on molecular alterations produces superior outcomes compared with unselected treatment. These trials use treatment algorithms to assign patients to specific targeted therapies based on tumor
molecular alterations. These algorithms should be governed by fixed rules to ensure standardization and reproducibility.”
According to the news editors, the research concluded: “Here, we summarize key molecular, biological, and
technical criteria that, in our view, should be addressed when establishing treatment algorithms based on
tumor molecular profiling for PM trials.”
For more information on this research see: Treatment Algorithms Based on Tumor Molecular Profiling:
The Essence of Precision Medicine Trials. Jnci-Journal of the National Cancer Institute , 2016;108(4):66-75.
Jnci-Journal of the National Cancer Institute can be contacted at: Oxford Univ Press Inc, Journals Dept, 2001
Evans Rd, Cary, NC 27513, USA.
44
Our news journalists report that additional information may be obtained by contacting C. Le Tourneau,
University of Paris, EA7331, Paris, France. Additional authors for this research include M. Kamal, A.M. Tsimberidou, P. Bedard, G. Pierron, C. Callens, E. Rouleau, A. Vincent-Salomon, N. Servant, M. Alt, R. Rouzier, X.
Paoletti, O. Delattre and I. Bieche.
NewsRx LLC
(2016-05-30), Findings from University of Paris Provide New Insights into Clinical Trials and Studies (Treatment Algorithms Based on Tumor Molecular Profiling: The Essence of Precision Medicine Trials), Clinical
Trials Week, 765, ISSN: 1543-6764, BUTTER® ID: 011748306
Biotechnology
Findings in the Area of Cancer Gene Therapy Reported from Jackson Laboratory (The
clinical trial landscape in oncology and connectivity of somatic mutational profiles to
targeted therapies)
By a News Reporter-Staff News Editor at Cancer Gene Therapy Week – New research on Biotechnology is the
subject of a report. According to news reporting originating in Farmington, Connecticut, by NewsRx journalists,
research stated, “Precision medicine in oncology relies on rapid associations between patient-specific variations
and targeted therapeutic efficacy. Due to the advancement of genomic analysis, a vast literature characterizing
cancer-associated molecular aberrations and relative therapeutic relevance has been published.”
The news reporters obtained a quote from the research from Jackson Laboratory, “However, data are not
uniformly reported or readily available, and accessing relevant information in a clinically acceptable time-frame
is a daunting proposition, hampering connections between patients and appropriate therapeutic options. One
important therapeutic avenue for oncology patients is through clinical trials. Accordingly, a global view into
the availability of targeted clinical trials would provide insight into strengths and weaknesses and potentially
enable research focus. However, data regarding the landscape of clinical trials in oncology is not readily available, and as a result, a comprehensive understanding of clinical trial availability is difficult. To support clinical
decision-making, we have developed a data loader and mapper that connects sequence information from oncology patients to data stored in an in-house database, the JAX Clinical Knowledgebase (JAX-CKB), which can be
queried readily to access comprehensive data for clinical reporting via customized reporting queries. JAX-CKB
functions as a repository to house expertly curated clinically relevant data surrounding our 358-gene panel, the
JAX Cancer Treatment Profile (JAX CTP), and supports annotation of functional significance of molecular variants. Through queries of data housed in JAX-CKB, we have analyzed the landscape of clinical trials relevant to
our 358-gene targeted sequencing panel to evaluate strengths and weaknesses in current molecular targeting
in oncology. Through this analysis, we have identified patient indications, molecular aberrations, and targeted
therapy classes that have strong or weak representation in clinical trials. Here, we describe the development
and disseminate system methods for associating patient genomic sequence data with clinically relevant information, facilitating interpretation and providing a mechanism for informing therapeutic decision-making.”
According to the news reporters, the research concluded: “Additionally, through customized queries, we
have the capability to rapidly analyze the landscape of targeted therapies in clinical trials, enabling a unique
view into current therapeutic availability in oncology.”
For more information on this research see: The clinical trial landscape in oncology and connectivity of
somatic mutational profiles to targeted therapies. Human Genomics , 2016;10():4. (Sage Publications - http:
//www.sagepub.com/ ; Human Genomics - hgp.sagepub.com)
Our news correspondents report that additional information may be obtained by contacting S.E. Patterson,
The Jackson Laboratory for Genomic Medicine, 10 Discovery Dr, Farmington, CT, 06032, United States. Additional authors for this research include R. Liu, C.M. Statz, D. Durkin, A. Lakshminarayana and S.M Mockus.
NewsRx LLC
(2016-06-06), Findings in the Area of Cancer Gene Therapy Reported from Jackson Laboratory (The clinical
trial landscape in oncology and connectivity of somatic mutational profiles to targeted therapies), Cancer Gene
Therapy Week, 5, ISSN: 1543-6837, BUTTER® ID: 011860570
45
Ignyta, Inc.
Ignyta Announces Interim Data from Phase 1 Clinical Trial of Investigational Precision
Medicine RXDX-105 at ASCO 2016
By a News Reporter-Staff News Editor at Clinical Trials Week – Ignyta, Inc. (Nasdaq: RXDX), a biotechnology
company focused on precision medicine in oncology, announced that interim results from the Phase 1 clinical
trial of RXDX-105, the company’s orally available, small molecule multikinase inhibitor with potent activity
against such targets as RET and BRAF, were presented at the 2016 Annual Meeting of the American Society
of Clinical Oncology (ASCO) in Chicago (Abstract 2574). RXDX-105 is Ignyta’s second clinical stage compound
after entrectinib, the furthest advanced inhibitor for solid tumors with NTRK (neurotrophin receptor kinase),
ROS1, or ALK gene rearrangements.
“We continue to be encouraged by the safety and preliminary antitumor activity data from our Phase 1
clinical trial of RXDX-105,” said Pratik Multani, M.D., Chief Medical Officer of Ignyta. “Given the performance
of RXDX-105 thus far, we look forward to the results from the Phase 1b portion of the study, in which we
are further evaluating this investigational agent and its activity in targeted patient populations with relevant
molecular alterations, as well as in patients with unselected lung cancer.”
The Phase 1 dose escalation portion of the clinical trial was designed to determine the maximum tolerated
dose (MTD) and/or recommended Phase 2 dose (RP2D), as well as preliminary anti-cancer activity, of single
agent RXDX-105 in patients with advanced or metastatic solid tumors.
NewsRx LLC
(2016-06-20), Ignyta Announces Interim Data from Phase 1 Clinical Trial of Investigational Precision
Medicine RXDX-105 at ASCO 2016, Clinical Trials Week, 90, ISSN: 1543-6764, BUTTER® ID: 011909329
Bacterial Infections and Mycoses
Reports Summarize Sepsis Findings from Emory University (Pathophysiology of septic
shock: From bench to bedside)
By a News Reporter-Staff News Editor at Clinical Trials Week – Fresh data on Bacterial Infections and Mycoses are presented in a new report. According to news reporting originating in Atlanta, Georgia, by NewsRx
journalists, research stated, “Our understanding of sepsis and its resultant outcomes remains a paradox. On
the one hand, we know more about the pathophysiology of sepsis than ever before.”
The news reporters obtained a quote from the research from Emory University, “However, this knowledge
has not been successfully translated to the bedside, as the vast majority of clinical trials for sepsis have been
negative. Yet even in the general absence of positive clinical trials, mortality from sepsis has fallen to its lowest
point in history, in large part due to educational campaigns that stress timely antibiotics and hemodynamic
support. While additional improvements in outcome will assuredly result from further compliance with evidence based practices, a deeper understanding of the science that underlies the host response in sepsis is
critical to the development of novel therapeutics. In this review, we outline immunopathologic abnormalities
in sepsis, and then look at potential approaches to therapeutically modulate them.”
According to the news reporters, the research concluded: “Ultimately, an understanding of the science underlying sepsis should allow the critical care community to utilize precision medicine to combat this devastating
disease on an individual basis leading to improved outcomes.”
For more information on this research see: Pathophysiology of septic shock: From bench to bedside. Presse
Medicale , 2016;45(4):E93-E98. Presse Medicale can be contacted at: Masson Editeur, 21 Street Camille
Desmoulins, Issy, 92789 Moulineaux Cedex 9, France.
Our news correspondents report that additional information may be obtained by contacting K.W. McConnell,
Emory University, Sch Med, Dept. of Surg, Emory Crit Care Center, Atlanta, GA 30322, United States.
NewsRx LLC
(2016-06-20), Reports Summarize Sepsis Findings from Emory University (Pathophysiology of septic shock:
From bench to bedside), Clinical Trials Week, 654, ISSN: 1543-6764, BUTTER® ID: 011909836
46
Clinical Research
Studies from Stanford University Describe New Findings in Clinical Trials and Studies
(Translation of Human-Induced Pluripotent Stem Cells From Clinical Trial in a Dish to
Precision Medicine)
By a News Reporter-Staff News Editor at Clinical Trials Week – Investigators publish new report on Clinical
Research. According to news reporting originating from Stanford, California, by NewsRx correspondents, research stated, “The prospect of changing the plasticity of terminally differentiated cells toward pluripotency
has completely altered the outlook for biomedical research.”
Our news editors obtained a quote from the research from Stanford University, “Human-induced pluripotent
stem cells (iPSCs) provide a new source of therapeutic cells free from the ethical issues or immune barriers of
human embryonic stem cells. iPSCs also confer considerable advantages over conventional methods of studying
human diseases. Since its advent, iPSC technology has expanded with 3 major applications: disease modeling,
regenerative therapy, and drug discovery.”
According to the news editors, the research concluded: “Here we discuss, in a comprehensive manner, the
recent advances in iPSC technology in relation to basic, clinical, and population health.”
For more information on this research see: Translation of Human-Induced Pluripotent Stem Cells From
Clinical Trial in a Dish to Precision Medicine. Journal of the American College of Cardiology , 2016;67(18):21612176. Journal of the American College of Cardiology can be contacted at: Elsevier Science Inc, 360 Park Ave
South, New York, NY 10010-1710, USA.
The news editors report that additional information may be obtained by contacting N. Sayed, Stanford
University, Sch Med, Div Cardiol, Dept. of Med, 265 Campus Dr G1120B, Stanford, CA 94305, United States.
Additional authors for this research include C. Liu and J.C. Wu.
NewsRx LLC
(2016-05-30), Studies from Stanford University Describe New Findings in Clinical Trials and Studies (Translation of Human-Induced Pluripotent Stem Cells From Clinical Trial in a Dish to Precision Medicine), Clinical
Hypertension
Study finds wide geographic differences in treatment of diabetes, hypertension, depression
By a News Reporter-Staff News Editor at Diabetes Week – New York, NY () – An international observational
study led by Columbia University researchers has uncovered widespread differences in the treatment of patients with common chronic diseases, including type 2 diabetes, hypertension, and depression. Using data from
250 million patient records in four countries, the study demonstrates the feasibility of performing large-scale
observational research to obtain information about clinical practice among diverse groups of patients.
Findings from the study, performed in collaboration with the Observational Health Data Sciences and Informatics (OHDSI) program, were published online today in Proceedings of the National Academy of Sciences
(PNAS).
The study revealed that the vast majority of patients with diabetes worldwide are initially treated with the
medication metformin, although there is wide variation in what second-line treatments are given. In contrast,
the study found significant variation in first-line treatment of hypertension and even greater differences in the
initial treatment of depression. One surprising finding was that 10 percent of diabetes patients, 11 percent of
depression patients, and 24 percent of hypertension patients followed a treatment pathway that was shared
with no one else in the study.
“We found that while the world is moving towards more consistent therapy over time for the three diseases,
there remain significant differences in how they are treated,” said first author George Hripcsak, MD, MS,
the Vivian Beaumont Allen Professor and chair of Biomedical Informatics at Columbia University Medical
Center (CUMC), principal investigator of the OHDSI coordinating center and director of Medical Informatics
Services at NewYork-Presbyterian/CUMC. “This suggests that randomized clinical trials – the gold standard
in evaluating new therapies – may not capture enough of the information needed to make their results more
broadly generalizable to different populations.”
Observational research, in which patterns of care are gleaned from large data sets – such as electronic health
records, insurance claims, and pharmacy records–have the potential to offer insight into real-world treatment
47
scenarios that may inform clinical trial design and, ultimately, clinical practice. But analyzing data from a
variety of sources is often hindered by disparate models for collecting and storing patient records.
To surmount these hurdles, an international group of scientists formed the OHDSI (pronounced ‘odyssey’)
program, which allows researchers to combine and analyze patient data from widely different sources in the US
and abroad. Columbia University serves as OHDSI’s coordinating center. Currently, the research collaborative
involves more than 600 million patient records from 14 countries.
“Modern randomized trials are currently carried out without a clear view of how current treatments are
used,” said study leader David Madigan, PhD, executive vice president and dean of the Faculty of Arts and
Sciences, professor of statistics at Columbia University, and co-principal investigator of the OHDSI coordinating
center. “In the future, before a randomized trial is started, an observational study like ours could be mandatory
to determine the appropriate sample size and composition of control groups, among other factors.”
The study relied on the OHDSI distributed data network, in which researchers from around the world convert patient-level data to a standardized model that can run a common analysis protocol. Investigators from
the 11 research sites participating in the study shared the final, aggregate results, although individual data
were excluded to protect patient privacy. Seven of the research sites had completed their analyses within just
three weeks of beginning the study.
“While the findings are quite interesting, the important point is that we’ve shown that large-scale observational research across widely different databases is feasible,” said Jon Duke, MD, director of the Drug Safety
Informatics Lab and research scientist at the Regenstrief Institute. “And it can be done in a very short amount
of time.”
Future OHDSI studies will focus on medical product safety surveillance, comparative effectiveness research
(making direct comparisons between therapies), patient-level predictive modeling, and other topics. A worldwide request for proposals is planned, in which researchers, citizen scientists, and high school students may
propose research questions to be run on the OHDSI network.
“The creation of such a network is a great opportunity, not only to characterize what treatments are actually
being used, but also to attempt to identify what treatments are potentially better,” said Nigam Shah, MBBS,
PhD, associate professor of medicine at Stanford University. “For example, from the wide variation in secondline treatments for diabetes, we can attempt to identify those that are more effective. OHDSI puts us on a path
to creating personalized evidence, which is a form of precision medicine.”
NewsRx LLC
(2016-06-20), Study finds wide geographic differences in treatment of diabetes, hypertension, depression,
Diabetes Week, 265, ISSN: 1537-1433, BUTTER® ID: 011910343
48
Chapter 5
Drugs and Therapies
NantHealth
Cancer MoonShot 2020 and NantHealth Form Collaborative Melanoma and Sarcoma
Working Group with Columbia University and UPMC to Accelerate Personalized
Immunotherapy
By a News Reporter-Staff News Editor at Cancer Vaccine Week – NantHealth and Cancer MoonShot 2020,
the nation’s most comprehensive cancer collaborative initiative, announced the formation of the Melanoma
and Sarcoma Working Group to accelerate molecular-informed immunotherapy trials. The team consists of
physicians, researchers and oncology professors from across the nation who have come together to focus their
collective wisdom and expertise on identifying and developing the most effective, cancer-directed immunotherapy treatments for patients with melanoma and sarcoma.
Experts within the group will collaborate to develop clinical trial protocols, carefully studying elements such
as standard of care, molecular informed patient selection and novel combination-immunotherapy approaches.
“The establishment of the Melanoma and Sarcoma Working Group reinforces Cancer MoonShot 2020’s commitment to accelerate the war on cancer,” stated Patrick Soon-Shiong, MD, founder and CEO of NantWorks
and leader of the Cancer MoonShot 2020 program. “Through the collaborative effort of these academic and industry thought leaders, we will be able to provide patients with Melanoma and Sarcoma with next-generation
immunotherapy treatment. The momentum to establish national collaborative groups addressing all cancer
types continues, and I am so pleased by the excitement Cancer MoonShot 2020 has generated in the academic
and scientific community.”
The Melanoma and Sarcoma Working Group will be chaired by Richard Carvajal, MD, Columbia University Medical Center, Director of Experimental Therapeutics and Director of the Melanoma Service, who will
spearhead the team’s efforts with ongoing meetings, the development of protocols and continued expansion of
the Working Group.
Other esteemed members of the Working Group include: Gary Schwartz, MD, Columbia University Medical
Center, Division Chief, Hematology/Oncology, and Associate Director, Herbert Irving Comprehensive Cancer
Center John Kirkwood, MD, UPMC, Director, Melanoma and Skin Cancer Program
“As members of the Melanoma and Sarcoma Working Group, we want to share - and gather - new knowledge
and understanding of how to better treat our patients,” stated John Kirkwood, MD, Director of the Melanoma
and Skin Cancer Program at UPMC. “Collectively, we have the passion and the expertise to help accelerate the
goal of winning the war on cancer.” CANCER MOONSHOT 2020 Working Group Established to Date:Pediatric
Cancer Working GroupBreast Cancer Working GroupHead And Neck Cancer Working GroupRadiation And
Immuno-Oncology Working Group About Cancer MoonShot 2020The Cancer MoonShot 2020 program is one of
the most comprehensive cancer collaborative initiative launched to date, seeking to accelerate the potential of
combination immunotherapy as the next generation standard of care in cancer patients. This initiative aims
to explore a new paradigm in cancer care by initiating randomized Phase II trials in patients at all stages of
disease in 20 tumor types in 20,000 patients within the next 36 months. These findings will inform Phase
III trials and the aspirational moonshot to develop an effective vaccine-based immunotherapy to combat cancer by 2020. For more information, please visit www.cancermoonshot2020.org and follow Cancer MoonShot
2020 on Twitter. About NantHealthNantHealth, a member of the NantWorks ecosystem of companies, is a
next-generation, evidence-based, personalized healthcare company enabling improved patient outcomes and
49
more effective treatment decisions for critical illnesses. NantHealth’s unique systems-based approach to personalized healthcare applies novel diagnostics tailored to the specific molecular profiles of patient tissues and
integrates this molecular data in a clinical setting with large-scale, real-time biometric signal and phenotypic
data to track patient outcomes and deliver precision medicine. For nearly a decade, NantHealth has developed
an adaptive learning system, CLINICS, which includes its unique software, middleware and hardware systems infrastructure that collects, indexes, analyzes and interprets billions of molecular, clinical, operational
and financial data points derived from novel and traditional sources, continuously improves decision-making
and further optimizes our clinical pathways and decision algorithms over time. For more information please
visit www.nanthealth.com and follow Dr. Soon-Shiong on Twitter @DrPatSoonShiong. View source version on
businesswire.com: http://www.businesswire.com/news/home/20160617005201/en/
NewsRx LLC
(2016-07-04), Cancer MoonShot 2020 and NantHealth Form Collaborative Melanoma and Sarcoma Working
Group with Columbia University and UPMC to Accelerate Personalized Immunotherapy, Cancer Vaccine Week,
5, ISSN: 1543-6802, BUTTER® ID: 011985913
Canon BioMedical
Canon BioMedical expands genotyping assays supporting drug response research
By a News Reporter-Staff News Editor at Drug Week – The days of giving the same pill to every patient are
fading into the past. Important advances in pharmacogenetics have determined that your genetic makeup
directly influences your reaction to life-saving medications. Directly involved in drug metabolism, the extremely
varied cytochrome P450 (CYP) enzyme family can increase or decrease your drug sensitivity. As an example,
someone with the *3 allele of CYP2C9 will metabolize warfarin, an important anticoagulant drug, at one-tenth
the normal rate (1). Not knowing a patient’s sensitivity to a particular drug can result in a trip to the emergency
room or tragic death. Pharmacogenetic research is working to understand treatment regiments based on genetic
analysis, and Canon BioMedical is excited to introduce eleven new genotyping assays to complement the 32
current assays targeting these important CYP genes.
http://photos.prnewswire.com/prnvar/20160119/323578LOGO
As precision medicine advances, researchers are determining the potential clinical impacts of prescribed
medication based on genetic analysis. Significant pharmacogenetic research is currently performed relating
to cardiology, but research is rapidly growing in the fields of chemotherapy and neuroscience. Seventy-five
Novallele genotyping assays able to detect variants relevant to pharmacogenetics research are currently offered
by Canon BioMedical. These products are not only for the CYP family of enzymes but also identify variations
in the VKORC1, SLC6A4, and DPYD genes.
The Novallele genotyping assays detect genetic variations using polymerase chain reaction (PCR) followed by
high-resolution melting (HRM) analysis on any thermocycler capable of HRM. All Novallele genotyping assays
are functionally verified and focus on relevant genotypes to genetic researchers working to improve health and
advance science.
“Pharmacogenetics is a fundamental discovery endeavor for Canon BioMedical,” states Dennis Snyder, Senior Director of Global Commercial Operations of Canon BioMedical. “Our scientists are engaging with researchers making important discoveries. The expectation is that these discoveries will translate into precise
treatment regiments, what is now defined as precision medicine. By supporting and collaborating with researchers, we will continue to support the future of precision medicine.”
Our new products are now available at www.canon-biomedical.com. In addition, Canon BioMedical will
present the expanded Novallele genotyping library at the Genetics and Genomics Virtual Conference presented
by LabRoots on May 11-12. We will also attend the European Society of Human Genetics Conference taking
place in Barcelona, Spain on May 21 and the American Association of Clinical Chemistry’s Annual Scientific
Meeting and Clinical Lab Expo taking place in Philadelphia, USA on July 31 to feature our new Novallele
genotyping assays.
NewsRx LLC
(2016-05-27), Canon BioMedical expands genotyping assays supporting drug response research, Drug Week,
23, ISSN: 1532-4575, BUTTER® ID: 011736300
50
Corvidia Therapeutics
Corvidia Therapeutics Appoints Sylvie Gregoire as Chair of the Board of Directors
By a News Reporter-Staff News Editor at Biotech Business Week – Corvidia Therapeutics, a precision cardiovascular company, announced the appointment of Sylvie Gregoire as Chair of its Board of Directors, effective
immediately. Dr. Gregoire brings over 25 years of experience in leadership roles at both public and private
companies.
“We are thrilled that Dr. Gregoire has agreed to chair our Board,” said Michael Davidson, MD, Chief Executive Officer of Corvidia Therapeutics. “She possesses exceptional leadership skills and a broad base of
experience, including commercial, regulatory, clinical development and manufacturing oversight, all of which
are critical to a small, start-up company focused on initiating our first pivotal trial involving precision medicine
later this year.”
“I am delighted to chair Corvidia’s Board of Directors. The company has strong potential for success, with
a solid management team backed by top-tier venture funds and a technology platform with the potential to
address serious unmet medical needs with novel compounds,” noted Dr. Gregoire. “I look forward to working
together with the Board and Corvidia’s management team in order to bring their compounds forward into
development.”
In addition to her current Board positions at Novo Nordisk and Galenica, from 2007 to 2013, Dr. Gregoire
served as President of the Human Genetic Therapies division of Shire plc, a public biopharmaceutical company.
From 2005 to 2008, Dr. Gregoire was a director of IDM Pharma, Inc., a public biotechnology company that now
operates as a subsidiary of Takeda Pharmaceuticals, and served as its Executive Chair from August 2006 to
October 2007. Earlier in her career, Dr. Gregoire was a member of the executive management team of Biogen,
Inc., leading Regulatory Affairs and Technical Operations. Dr. Gregoire holds a Bachelor of Pharmacy degree
from Laval University in Quebec, and a Pharmacy Doctoral degree from the State University of New York at
Buffalo.
NewsRx LLC
(2016-06-27), Corvidia Therapeutics Appoints Sylvie Gregoire as Chair of the Board of Directors, Biotech
Oncology
Data on Ovarian Cancer Reported by Researchers at University of Groningen (Studying
platinum sensitivity and resistance in high-grade serous ovarian cancer: Different models for
different questions)
to news reporting originating in Groningen, Netherlands, by NewsRx journalists, research stated, “High-grade
serous ovarian cancer (HGSOC) has the highest mortality rate among all gynecological cancers. Patients are
generally diagnosed in an advanced stage with the majority of cases displaying platinum resistant relapses.”
The news reporters obtained a quote from the research from the University of Groningen, “Recent genomic
interrogation of large numbers of HGSOC patient samples indicated high complexity in terms of genetic aberrations, intra-and intertumor heterogeneity and underscored their lack of targetable oncogenic mutations. Subclassifications of HGSOC based on expression profiles, termed ‘differentiated’, ‘immunoreactive’, ‘mesenchymal’
and ‘proliferative’, were shown to have prognostic value. In addition, in almost half of all HGSOC patients, a
deficiency in homologous recombination (HR) was found that potentially can be targeted using PARP inhibitors.
Developing precision medicine requires advanced experimental models. In the current review, we discuss experimental HGSOC models in which resistance to platinum therapy and the use of novel therapeutics can be
carefully studied. Panels of better-defined primary cell lines need to be established to capture the full spectrum
of HGSOC subtypes. Further refinement of cell lines is obtained with a 3-dimensional culture model mimicking
the tumor microenvironment. Alternatively, ex vivo ovarian tumor tissue slices are used. For in vivo studies,
larger panels of ovarian cancer patient-derived xenografts (PDXs) are being established, encompassing all expression subtypes. Ovarian cancer PDXs grossly retain tumor heterogeneity and clinical response to platinum
therapy is preserved. PDXs are currently used in drug screens and as avatars for patient response. The role of
the immune system in tumor responses can be assessed using humanized PDXs and immunocompetent genetically engineered mouse models. Dynamic tracking of genetic alterations in PDXs as well as patients during
51
treatment and after relapse is feasible by sequencing circulating cell-free tumor DNA and analyzing circulating
tumor cells. We discuss how various models and methods can be combined to delineate the molecular mechanisms underlying platinum resistance and to select HGSOC patients other than BRCA1/2-mutation carriers
that could potentially benefit from the synthetic lethality of PARP inhibitors.”
According to the news reporters, the research concluded: “This integrated approach is a first step to improve
therapy outcomes in specific subgroups of HGSOC patients.”
For more information on this research see: Studying platinum sensitivity and resistance in high-grade
serous ovarian cancer: Different models for different questions. Drug Resistance Updates , 2016;24():5569. (Elsevier - www.elsevier.com; Drug Resistance Updates - http://www.journals.elsevier.com/
drug-resistance-updates/ )
Our news correspondents report that additional information may be obtained by contacting N.G. Alkema,
Dept. of Gynecologic Oncology, Cancer Research Centre Groningen, University of Groningen, University Medical Center Groningen, Groningen, Netherlands. Additional authors for this research include G.B. Wisman,
A.G. van der Zee, M.A. van Vugt and S. de Jong.
NewsRx LLC
(2016-06-28), Data on Ovarian Cancer Reported by Researchers at University of Groningen (Studying platinum sensitivity and resistance in high-grade serous ovarian cancer: Different models for different questions),
Cancer Weekly, 48, ISSN: 1532-4567, BUTTER® ID: 011958489
Certara
Dr. Adam Darwich Is Appointed Certara Lecturer in Precision Dosing at The University of
Manchester, England
By a News Reporter-Staff News Editor at Drug Week – Certara®, the global biosimulation technology-enabled
drug development company, announced that Adam Darwich, PhD, has been appointed Certara Lecturer in
Precision Dosing at the Manchester Pharmacy School at The University of Manchester in England. Certara
has endowed this lectureship, which is part of the University’s precision medicine initiative. It is intended to
advance teaching and research of modeling and simulation for individual dose optimization, ultimately at the
point of care.
“While biosimulation has been widely accepted and adopted by biopharmaceutical companies and global
regulatory agencies, its implementation in clinical care has been modest to date,” said Certara Chief Executive
Officer Edmundo Muniz, MD, PhD. “We are delighted to have a researcher of Adam’s caliber helping to advance this field and teaching these important modeling techniques to the next generation of scientists in drug
development and patient care. This lectureship is just one way in which Certara is working to make precision
dosing a reality for everyone.”
Later this month, The University of Manchester will host the Health Care Summit on Model-based Precision
Dosing, for which Certara is the sole sponsor. During the Summit, world key opinion leaders will share their
ideas and experiences, and discuss the path forward for implementing biosimulation in individual patient care.
Areas of focus include work done across specific population cohorts, including oncology patients, HIV patients,
obese subjects, pregnant women, and pediatric patients.
Professor Amin Rostami, who is the chair of systems pharmacology at The University of Manchester and
chief scientific officer at Certara, said, “This event provides a unique opportunity for many renowned thought
leaders from across the world, whether they are clinicians or scientists from academia or healthcare sectors,
to debate various activities which will facilitate the use of modeling and simulation for precision dosing. This
event would not have happened if it was not for the forward thinking of Certara which sponsored it.”
Dr. Darwich, who will co-chair the Summit together with Professor Rostami, has extensive PK modeling
experience, which includes PBPK modeling of drug-drug interactions (DDIs), interspecies extrapolation, and
extrapolation to special populations, biopharmaceutics and clinical study design. His previous research focused
on PBPK modeling and simulation of oral drug bioavailability in bariatric surgery patients and mechanismbased inhibition of gut wall metabolism.
As part of his new role, Dr. Darwich is already partnering with healthcare professionals and other collaborators on a number of projects, including modeling biologics, DDIs and special populations.
Underscoring its prominence in this area, Manchester is one of six initial regional centers of excellence for
The Precision Medicine Catapult, the UK’s innovation center for precision medicine. About Certara Certara is
a global biosimulation and regulatory writing company, committed to optimizing drug development decisions.
52
Its clients include hundreds of international biopharmaceutical companies, leading academic institutions, and
key regulatory agencies. Certara’s solutions, which span drug discovery through patient care, increase the
probability of regulatory and commercial success by using the most scientifically-advanced modeling and simulation technologies and regulatory strategies. For more information, visit www.certara.com. View source
version on businesswire.com: http://www.businesswire.com/news/home/20160512005148/en/
NewsRx LLC
(2016-05-27), Dr. Adam Darwich Is Appointed Certara Lecturer in Precision Dosing at The University of
Manchester, England, Drug Week, 26, ISSN: 1532-4575, BUTTER® ID: 011736302
Oncology
Findings from Duke University Provides New Data on Carcinomas (Application of single-cell
RNA sequencing in optimizing a combinatorial therapeutic strategy in metastatic renal cell
carcinoma)
By a News Reporter-Staff News Editor at Health & Medicine Week – Investigators discuss new findings in
Oncology. According to news reporting out of Durham, North Carolina, by NewsRx editors, research stated,
“Intratumoral heterogeneity hampers the success of marker-based anticancer treatment because the targeted
therapy may eliminate a specific subpopulation of tumor cells while leaving others unharmed. Accordingly,
a rational strategy minimizing survival of the drug-resistant subpopulation is essential to achieve long-term
therapeutic efficacy.”
Our news journalists obtained a quote from the research from Duke University, “Using single-cell RNA sequencing (RNA-seq), we examine the intratumoral heterogeneity of a pair of primary renal cell carcinoma and
its lung metastasis. Activation of drug target pathways demonstrates considerable variability between the primary and metastatic sites, as well as among individual cancer cells within each site. Based on the prediction
of multiple drug target pathway activation, we derive a combinatorial regimen co-targeting two mutually exclusive pathways for the metastatic cancer cells. This combinatorial strategy shows significant increase in the
treatment efficacy over monotherapy in the experimental validation using patient-derived xenograft platforms
in vitro and in vivo. Our findings demonstrate the investigational application of single-cell RNA-seq in the
design of an anticancer regimen.”
According to the news editors, the research concluded: “The approach may overcome intratumoral heterogeneity which hampers the success of precision medicine.”
For more information on this research see: Application of single-cell RNA sequencing in optimizing a combinatorial therapeutic strategy in metastatic renal cell carcinoma. Genome Biology , 2016;17():1-17. Genome
Biology can be contacted at: Biomed Central Ltd, 236 Grays Inn Rd, Floor 6, London WC1X 8HL, England.
(BioMed Central - http://www.biomedcentral.com/ ; Genome Biology - genomebiology.com)
Our news journalists report that additional information may be obtained by contacting K.T. Kim, Duke
University, Medical Center, Dept. of Radiat Oncol, Durham, NC, United States. Additional authors for this
research include H.W. Lee, H.O. Lee, H.J. Song, D.E. Jeong, S. Shin, H. Kim, Y. Shin, D.H. Nam, B.C. Jeong,
D.G. Kirsch, K.M. Joo and W.Y. Park.
NewsRx LLC
(2016-07-01), Findings from Duke University Provides New Data on Carcinomas (Application of single-cell
RNA sequencing in optimizing a combinatorial therapeutic strategy in metastatic renal cell carcinoma), Health
& Medicine Week, 2161, ISSN: 1532-4605, BUTTER® ID: 011974606
53
Oncology
Findings on Solid Cancer Reported by Investigators at Zhejiang Cancer Hospital
(Pharmacokinetically guided algorithm of 5-fluorouracil dosing, a reliable strategy of
precision chemotherapy for solid tumors: a meta-analysis)
By a News Reporter-Staff News Editor at Clinical Trials Week – Fresh data on Oncology are presented in a
new report. According to news reporting from Hangzhou, People’s Republic of China, by NewsRx journalists,
research stated, “Precision medicine characterizes a new era of cancer care and provides each patient with the
right drug at the right dose and time. However, the practice of precision dosing is hampered by a lack of smart
dosing algorithms.”
The news correspondents obtained a quote from the research from Zhejiang Cancer Hospital, “A pharmacokinetically guided (PKG) dosing algorithm is considered to be the leading strategy for precision chemotherapy, although the effects of PKG dosing are not completely confirmed. Hence, we conducted a meta-analysis
to evaluate the effects of the PKG algorithm of 5-fluorouracil (5-FU) dosing on patients with solid tumors.
A comprehensive retrieval was performed to identify all of the prospective controlled studies that compared
the body surface area (BSA)-based algorithm with the PKG algorithm of 5-FU in patients with solid tumors.
Overall, four studies with 504 patients were included. The PKG algorithm significantly improved the objective
response rate of 5-FU-based chemotherapy compared with the BSA-based algorithm. Furthermore, PKG dosing
markedly decreased the risk of total grade 3/4 adverse drug reactions, especially those related to hematological
toxicity.”
According to the news reporters, the research concluded: “Overall, the PKG algorithm may serve as a reliable
strategy for individualized dosing of 5-FU.”
For more information on this research see: Pharmacokinetically guided algorithm of 5-fluorouracil dosing, a
reliable strategy of precision chemotherapy for solid tumors: a meta-analysis. Scientific Reports , 2016;6():1-7.
Scientific Reports can be contacted at: Nature Publishing Group, Macmillan Building, 4 Crinan St, London N1
9XW, England. (Nature Publishing Group - http://www.nature.com/ ; Scientific Reports - http://www.
nature.com/srep/ )
Our news journalists report that additional information may be obtained by contacting L. Fang, Zhejiang
Canc Hosp, Dept. of Chemotherapy, Hangzhou, Zhejiang, People’s Republic of China. Additional authors for
this research include W.X. Xin, H.Y. Ding, Y.W. Zhang, L.K. Zhong, H. Luo, J.J. Li, Y.S. Yang and P. Huang.
NewsRx LLC
(2016-06-20), Findings on Solid Cancer Reported by Investigators at Zhejiang Cancer Hospital (Pharmacokinetically guided algorithm of 5-fluorouracil dosing, a reliable strategy of precision chemotherapy for solid
tumors: a meta-analysis), Clinical Trials Week, 353, ISSN: 1543-6764, BUTTER® ID: 011909564
Genomic Health Announces Publication in Journal of Clinical Oncology Demonstrating
Oncotype DX® Predicts Late Distant Recurrence in Breast Cancer
By a News Reporter-Staff News Editor at Clinical Trials Week – Genomic Health, Inc. (Nasdaq: GHDX) announced that the Journal of Clinical Oncology, the official journal of the American Society of Clinical Oncology
(ASCO), published results from a large study confirming the ability of the Oncotype DX(®) Breast Recurrence
Score(™), in combination with quantitative estrogen-receptor (ER) expression, to accurately predict after five
years of tamoxifen therapy the risk of late distant recurrence up to 15 years in patients with early-stage, hormone receptor-positive breast cancer. These findings suggest that Oncotype DX may help identify which patients are most likely to benefit from extended hormonal treatment with tamoxifen.
“Extending tamoxifen treatment for 10 years has been shown to be associated with better outcomes, but not
all patients have the same risk of late distant recurrence - meaning cancer coming back after five years - and
it is important to know a patient’s risk in order to better understand who will benefit the most from extended
hormonal treatment,” said Norman Wolmark, M.D., chairman of the National Surgical Adjuvant Breast and
Bowel Project (NSABP) Foundation, the organization that carried out the study in conjunction with Genomic
Health. “The results of this large study confirm that Oncotype DX helps better define who is at greatest risk of
a late distant recurrence and who would likely derive the greatest benefit from extended tamoxifen therapy.”
The study analyzed the results of two clinical trials: NSABP B-14 (668 patients) and B-28 (1,065 patients).
Patients were followed up on for a median of 13.9 years (B-14) and 11.2 years (B-28). The results reconfirm
54
earlier findings from validation studies of Oncotype DX which demonstrated that Breast Recurrence Score
results were strongly associated with distant recurrence up to 15 years for patients with node-positive and
node-negative disease. The new study also showed that in patients with greater ER expression, the association
of the continuous Breast Recurrence Score with distant recurrence was significant after adjustment for age,
grade and tumor size.
The results suggest that extending tamoxifen beyond five years may provide greater benefit in patients
with high and intermediate Breast Recurrence Score results who also show high ER expression. Conversely,
patients with low Breast Recurrence Score results have a much lower risk of late distant recurrence and would
be expected to have less benefit from extended tamoxifen beyond five years.
Findings from a separate study of Oncotype DX were also recently published in the Journal of Clinical
Oncology. That study evaluated the ability of the Breast Recurrence Score to provide information on breast
cancer progression and survival in newly diagnosed stage IV breast cancer patients. About five percent of
patients are diagnosed with stage IV disease, and there is limited molecular information on the impact of
tumor biology on patient outcomes. Results showed that the Breast Recurrence Score independently predicted
both time to progression and two-year overall survival in patients with ER-positive, HER2-negative metastatic
breast cancer, indicating that biology is the major determinant of outcome, not only for patients diagnosed with
node-negative and node-positive breast cancer, but also for patients diagnosed with stage IV disease.
“The publication of these two studies in the Journal of Clinical Oncology further demonstrates our leadership in the area of breast cancer precision medicine and our ongoing commitment to evaluate our genomic
tests in a wide range of cancer patients,” said Frederick Baehner, M.D., vice president, Pathology, Genomic
Health. “The impressive amount of evidence that we have generated over the last decade has helped us better
understand the biology of breast and other types of cancer throughout the disease continuum - from earlystage to advanced metastatic disease - and continues to inform our liquid biopsy development efforts focused
on late-stage solid tumor cancers.”
NewsRx LLC
(2016-06-06), Genomic Health Announces Publication in Journal of Clinical Oncology Demonstrating Oncotype DX® Predicts Late Distant Recurrence in Breast Cancer, Clinical Trials Week, 114, ISSN: 1543-6764,
BUTTER® ID: 011860918
Lung Cancer
IASLC pleased by FDA approval of blood test to detect mutations in lung cancer
By a News Reporter-Staff News Editor at Pharma Business Week – DENVER - The International Association
for the Study of Lung Cancer (IASLC) praises the U.S. Food and Drug Administration’s (FDA) decision to
approve the first blood test to detect an oncogenic driver mutation in non-small cell lung cancer (NSCLC). This
marks a significant step forward in lung cancer treatment. The cobas EGFR Mutation Test v2 detects epidermal
growth factor receptor (EGFR) gene mutations and the FDA approved a blood-based companion diagnostic for
the EGFR tyrosine kinase inhibitor erlotinib (Tarceva).
Lung cancer is the leading cause of cancer-related deaths among men and women in the U.S. and around
the world. According to the National Cancer Institute, U.S. doctors will diagnose an estimated 221,200 with
lung cancer this year, and 158,040 will succumb to the disease. Lung cancer tumors can shed DNA into a
patient’s blood which means that testing a blood sample, sometimes called a “liquid biopsy,” may detect specific
mutations within the shed DNA.
Health providers perform most genetic tests for NSCLC through lung tissue samples. These procedures
can be invasive and difficult for patients. A reliable blood test could replace the need for direct tissue samples.
This new test may provide additional benefit to patients who may be too ill or are otherwise unable to provide
a tumor specimen for EGFR testing. Current screening methods find EGFR mutations in approximately 1020 percent of Caucasian NSCLC patients and more than 50 percent of Asian NSCLC patients, making it a
promising target for precision therapy treatments.
“This is another example of how lung cancer serves as a role model for precision medicine. The rapid pace
of scientific advances in the disease’s treatment allows for new and novel therapies,” said Fred R. Hirsch, MD,
PhD, Professor of Medicine and Pathology at the University of Colorado Cancer Center and School of Medicine,
and CEO of the IASLC. “This new and less invasive test gives patients and their doctors more options to more
effectively diagnose and treat lung cancer. It is our hope that we can perform many more genetic tests through
blood samples in the future.”
55
Doctors have used erlotinib to help lung cancer patients for more than 10 years. The FDA first approved
it in 2004 to treat patients with locally advanced or metastatic NSCLC after the failure of at least one prior
chemotherapy regimen. In 2013, the FDA approved erlotinib for first-line treatment of patients with metastatic
NSCLC whose tumors harbor EGFR exon 19 deletions or L858R substitution mutations as detected by an FDAapproved test.
NewsRx LLC
(2016-06-20), IASLC pleased by FDA approval of blood test to detect mutations in lung cancer, Pharma
Glomerular Disease
Investigators at University of Michigan Zero in on Glomerular Disease (Pro: ‘The usefulness
of biomarkers in glomerular diseases’. The problem: moving from syndrome to
mechanism–individual patient variability in disease presentation, course ...)
By a News Reporter-Staff News Editor at Clinical Trials Week – Research findings on Glomerular Disease are
discussed in a new report. According to news reporting from Ann Arbor, Michigan, by NewsRx journalists,
research stated, “The diagnosis and treatment decisions in glomerular disease are principally based on renal
pathology and nonspecific clinical laboratory measurements such as serum creatinine and urine protein. Using
these classification approaches, patients have marked variability in rate of progression and response to therapy,
exposing a significant number of patients to toxicity without benefit.”
The news correspondents obtained a quote from the research from the University of Michigan, “Additionally,
clinical trials are at risk of not being able to detect an efficacious therapy in relevant subgroups as patients with
shared clinical-pathologic diagnoses have heterogeneous underlying pathobiology. To change this treatment
paradigm, biomarkers that reflect the molecular mechanisms underlying the clinical-pathologic diagnoses are
needed. Recent progress to identify such biomarkers has been aided by advances in molecular profiling, largescale data generation and multi-scalar data integration, including prospectively collected clinical data.”
According to the news reporters, the research concluded: “This article reviews the evolving success stories
in glomerular disease biomarkers across the genotype-phenotype continuum and highlights opportunities to
transition to precision medicine in glomerular disease.”
For more information on this research see: Pro: ‘The usefulness of biomarkers in glomerular diseases’. The
problem: moving from syndrome to mechanism–individual patient variability in disease presentation, course
and response to therapy. Nephrology Dialysis Transplantation , 2015;30(6):892-8. (Oxford University Press http://www.oup.com/ ; Nephrology Dialysis Transplantation - ndt.oxfordjournals.org)
Our news journalists report that additional information may be obtained by contacting L.H. Mariani, Dept.
of Internal Medicine, Nephrology, University of Michigan, Ann Arbor, MI USA. Arbor Research Collaborative
for Health, Ann Arbor, MI, United States.
NewsRx LLC
(2016-07-04), Investigators at University of Michigan Zero in on Glomerular Disease (Pro: ‘The usefulness
of biomarkers in glomerular diseases’. The problem: moving from syndrome to mechanism–individual patient
variability in disease presentation, course ...), Clinical Trials Week, 344, ISSN: 1543-6764, BUTTER® ID:
011985542
56
Therapeutics
Investigators from Vanderbilt University Have Reported New Data on Therapeutics
(Biomarker Tests for Molecularly Targeted Therapies: Laying the Foundation and Fulfilling
the Dream)
By a News Reporter-Staff News Editor at Clinical Oncology Week – Data detailed on Therapeutics have been
presented. According to news reporting out of Nashville, Tennessee, by NewsRx editors, research stated, “Precision medicine focuses on the management of individual patients on the basis of biomarkers and other distinguishing characteristics, with the overarching objective of improving clinical outcomes. The rapid proliferation
of biomarker tests and targeted therapies has revolutionized patient care in a variety of serious disorders.”
Our news journalists obtained a quote from the research from Vanderbilt University, “Targeted cancer therapies interrupt oncogenic molecular pathways driven by mutations, overexpression, or translocation of specific
genes. However, there is concern that the emergence of large-scale genomic data is exceeding our capacity to
appropriately analyze and interpret the results. In 2014, the Institute of Medicine convened the Committee
on Policy Issues in the Clinical Development and Use of Biomarkers for Molecularly Targeted Therapies. This
committee conducted a study to develop recommendations to address diverse and interconnected development,
regulatory, clinical practice, and reimbursement issues. The committee conducted an extensive search of the
relevant literature and invited testimony from a wide range of experts in the field. The final report of the committee’s study and deliberations was released on March 4, 2016, focusing on ways to achieve 10 goals to further
advance the development and appropriate clinical use of biomarker tests for molecularly targeted therapies.
This article presents an overview of the committee’s study and resulting recommendations, which cover establishment of clinical utility, regulatory oversight, coverage and reimbursement, health system data integration,
as well as education and access.”
According to the news editors, the research concluded: “The committee’s recommendations presented and
discussed here are fundamentally grounded in the understanding that, when properly validated and appropriately implemented, these assays and corresponding therapies hold considerable promise to enhance the quality
of patient care and improve meaningful clinical outcomes.”
For more information on this research see: Biomarker Tests for Molecularly Targeted Therapies: Laying the
Foundation and Fulfilling the Dream. Journal of Clinical Oncology , 2016;34(17):2061-2066,144-145. Journal
of Clinical Oncology can be contacted at: Amer Soc Clinical Oncology, 2318 Mill Road, Ste 800, Alexandria, VA
22314, USA.
Our news journalists report that additional information may be obtained by contacting G.H. Lyman, Vanderbilt University, 221 Kirkland Hall, Nashville, TN 37235, United States.
NewsRx LLC
(2016-06-27), Investigators from Vanderbilt University Have Reported New Data on Therapeutics
(Biomarker Tests for Molecularly Targeted Therapies: Laying the Foundation and Fulfilling the Dream), Clinical Oncology Week, 126, ISSN: 1543-6780, BUTTER® ID: 011946430
Psychiatry
Mayo Clinic introduces precision medicine in psychiatry
By a News Reporter-Staff News Editor at Psychology & Psychiatry Journal – ROCHESTER, Minn. – Mayo
Clinic is highlighting the potential merits of using precision medicine in prescribing antidepressants. Details
appear in the current issue of Mayo Clinic Proceedings. Eleven percent of Americans 12 years and older have
been prescribed antidepressant medication, according to Centers for Disease Control and Prevention data from
2005-2008. These medications are regularly prescribed in psychiatric, pediatric, adolescent, family and general
medicine clinics nationwide.
MULTIMEDIA ALERT: Video and audio are available for download on the Mayo Clinic News Network.
Mark A. Frye, M.D., department chair of Psychiatry and Psychology at Mayo Clinic, recognizes there is
increasing interest in individualizing treatment selection for more than 20 treatments approved by the U.S.
Food and Drug Administration (FDA) for major depressive disorder. By doing so, physicians may be able to
provide greater precision to pharmacotherapy recommendations for individual patients beyond the large-scale,
clinical trials evidence base.
“The medical community continues to recognize that genetic variation may contribute to disparate patient
reactions to drugs,” Dr. Frye says. “For example, some may experience adverse side effects, while others respond
57
positively to the same drug.” He says the different responses to pharmacotherapy provide a unique opportunity
to develop pharmacogenetic guidelines for psychiatry.
These comments are reflected in an evidence review of other studies published in the July issue of Mayo
Clinic Proceedings. This review focuses on two major genetic tests that screen for pharmacokinetic metabolizing
genes CYP2D6 and CYP2C19 – enzymes that metabolize selective serotonin reuptake inhibitors (SSRI).
Dr. Frye explains that using the electronic health record along with genetic testing results has the potential
to further enable prescribers the ability to individualize treatment for their patients taking antidepressants.
Other authors of this study, all of Mayo Clinic, include:
The researchers’ potential competing interests are outlined in the article.
NewsRx LLC
(2016-07-09), Mayo Clinic introduces precision medicine in psychiatry, Psychology & Psychiatry Journal,
107, ISSN: 1944-2726, BUTTER® ID: 012018640
NantHealth, Inc.
NantHealth Announces Commercial Availability of GPS Cancer™ and Launch of GPS
Cancer Browser on a Secure Mobile Platform at the 2016 American Society of Clinical
Oncology Annual Meeting
By a News Reporter-Staff News Editor at Life Science Weekly – NantHealth, Inc. (Nasdaq: NH), a leading
next-generation, evidence-based, personalized healthcare company, announced the commercial availability of
Genomic Proteomic Spectrometry Cancer, or GPS CancerTM, a unique, comprehensive molecular test and decision support solution that measures the proteins present in the patient’s tumor tissue, combined with whole
genomic and transcriptomic sequencing of tumor & normal samples.
GPS CancerTM integrates targeted quantitative proteomics with whole genome (DNA) and whole transcriptome (RNA) sequencing, and a knowledge database containing hundreds of oncogenes and approximately 1,500
cellular pathways to identify genomic and proteomic alterations-from DNA to RNA to protein- targeting proteins with high clinical relevance to each person’s tumor and providing oncologists with a detailed molecular
profile of a patient’s cancer to inform personalized treatment strategies.
With the capability to measure at a quantitative level, proteins which have known clinical significance
relating to activity or resistance to chemotherapy, monoclonal antibody therapy (mAb), hormonal therapy, small
molecule targeted therapy and checkpoint inhibitors, GPS CancerTM is the most comprehensive molecular
profiling solution of a patient’s tumor tissue. Combined with whole genome and transcriptomic analysis, this
test provides informed clinical decision support, arming the physician with insight into the patient’s response
and resistance to particular therapeutics before treatment begins. This valuable information is available within
21 days of receipt of the tissue, thus enabling clinical utility.
NantHealth will be exhibiting GPS CancerTM and its other solutions for cancer care at ASCO booth #12135
from June 3-June 7, 2016.
GPS CancerTM results are available to doctors in an easy-to-read report or accessible through the GPS
CancerTM Genome Browser, a mobile application available on smartphones including the BlackBerry Priv.
The GPS Cancer Genome Browser is the first app enabling a physician and molecular scientists to browse the
patient’s whole genome down to a single base pair and provide visual insight into genomic alterations coupled
to relevant data about that alteration.
GPS CancerTM testing is conducted in the CLIA-certified and CAP-accredited laboratory of NantOmics,
and is an enabler for the Cancer MoonShot 2020, the world’s most comprehensive cancer care collaborative
seeking to accelerate the potential of combination immunotherapy as the next-generation standard of care in
cancer patients.
“2016 has already been a banner year for NantHealth. On the heels of going public, today we’re announcing
the commercial availability of GPS CancerTM as well as the launch of GPS Cancer Genome Browser at one of
the most significant oncology events worldwide at ASCO. The ability to bring next-generation cancer treatments
to patients marks a significant milestone in the war against cancer,” said Dr. Patrick Soon-Shiong, Founder and
CEO of NantHealth. “While genomics has undoubtedly advanced our ability to treat cancer, gene panels have
only given us a partial picture and only look at a fraction of the genome. We have leapfrogged from genomics
to the era of clinically relevant proteomics with this comprehensive integration of DNA, RNA, and quantitative
protein analysis in a single molecular test-GPS CancerTM. Coupled with robust predictive analytics, this 21st
58
century molecular profile offers clinicians and patients a powerful tool in fighting cancer at point of care and
before treatment begins.”
“Moreover, GPS CancerTM is an enabler for facilitating the goals of the Cancer MoonShot 2020, the nation’s
most comprehensive cancer care initiative with the ambitious goal of creating a cancer vaccine by 2020, Dr
Soon-Shiong added, “GPS CancerTM may accelerate efforts to bring novel combinations of therapeutic agents
to cancer patients by providing the molecular fingerprinting foundation necessary to help identify patients
eligible for QUILT, Quantitative Integrative Lifelong Trials. These clinical trials, which are at the heart of
Cancer MoonShot 2020, are aimed to accelerate the potential of immunotherapy as the new standard of care
for cancer patients by harnessing the power of the immune system to fight this disease.”
Today, many tests employ limited gene panels of 10-400 genes and a reference genome against which a patient’s tumor DNA is compared to identify alteration. GPS CancerTM sequences the whole genome of 20,000+
genes and 3 billion base pairs and matches against the patient’s normal DNA, providing oncologists with an
expansive view of alterations to inform personalized treatment strategies specific for that patient. GPS CancerTM extends from genomics to proteomics not only through analysis of RNA but also provides quantitative
proteomics through mass spectrometry to measure the amounts of clinically relevant proteins that are the
targets of or essential for various therapeutics. This clinically relevant information helps oncologists to better
understand how patients may potentially respond to chemotherapies, targeted therapies, and immunotherapies.
“GPS represents the new standard by which all complex molecular testing on tumors will be compared,
there is no other CAP/CLIA approved genomic offering that come close to this level of sophistication and clinical
utility.” -Leonard Sender, MD, Medical Director of the Hyundai Cancer Institute at Children’s Hospital Orange
County (CHOC Children’s).
“As a community oncologist, I couldn’t be more excited to have GPS Cancer available for my patients, particularly those with more difficult cases. I treat my patients like my family, and as such, I want them to have
the best,” says Sibel Blau, MD, Northwest Medical Specialties and Rainier Hematology Oncology. “The unparalleled comprehensiveness of this test helps assure that we leave no stone unturned for our patients.”
“Using the NantOmics clinical quantitative proteomics test provides me a deeper understanding of the biology of my patient’s tumors which has helped me make better treatment decisions,” says Steven W. Mamus,
MD, Medical Director, Oncology/Hematology, Cancer Center of Sarasota-Manatee.
“At Indiana University Health, we aim to identify innovative therapeutic options for metastatic cancer patients through the use of cutting-edge precision medicine technologies. GPS Cancer with its combination of
genomic and proteomic profiling has revealed numerous opportunities for treatment, providing actionable results for the majority of our patients,” says Milan Radovich, PhD, Co-director of the Indiana University Health
Precision Genomics Program.
The payer community has acknowledged the potential of GPS to improve outcomes by offering coverage for
the test. In January 2016, Independence Blue Cross became the first major insurer to offer reimbursement for
GPS Cancer for its members. In May 2016, NantHealth expanded coverage of GPS Cancer nationwide. These
organizations include Bank of America, Sanford Health, and Phoenix Children’s Hospital, which became the
nation’s first self-insured employers to cover next-generation whole genome sequencing for various cancers,
helping their employees-and their families-to gain better insight and treatment strategies in optimizing cancer
therapy. In addition, Sanford Health Plan, an NCQA-accredited regional health plan in the Dakotas, become the
second commercial insurer to offer coverage for GPS Cancer. http://bit.ly/1qb7qkU “At Sanford Health,
we are dedicated to offering our employees affordable and accessible high-quality care. Not only is GPS Cancer
testing a novel approach to cancer care, it offers our employees - and their families - the best possible coverage
for receiving improved cancer treatment options,” said Rick Adcock, Executive Vice President, Sanford Health.
“As one of the founding members of the National Immunotherapy Coalition and Pediatrics Consortium, we are
committed to the Cancer MoonShot 2020 program and its initiatives to win the war on cancer.”
NewsRx LLC
(2016-06-21), NantHealth Announces Commercial Availability of GPS Cancer™ and Launch of GPS Cancer
Browser on a Secure Mobile Platform at the 2016 American Society of Clinical Oncology Annual Meeting, Life
Science Weekly, 789, ISSN: 1552-2474, BUTTER® ID: 011921794
59
Medical, Healthcare
National Brain Tumor Society & Memorial Sloan Kettering Agreement Bolsters Research
Efforts to Combat Deadliest Brain Cancer
By a News Reporter-Staff News Editor at Clinical Trials Week – National Brain Tumor Society (NBTS), the
largest nonprofit organization in the U.S. dedicated to the brain tumor community, announced that it has entered into a Collaborative Agreement with Memorial Sloan Kettering Cancer Center (MSK) for the famed New
York hospital and research institute to officially join its flagship research program, the Defeat GBM Research
Collaborative (Defeat GBM).
“Despite being an area of urgent high-unmet medical need, glioblastoma, or GBM, the most common and
deadliest of all brain cancers, has unfortunately seen little progress in developing more effective treatments
against it during the past few decades,” said David F. Arons, JD, Chief Executive Officer, National Brain Tumor
Society. “With a structured scientific and funding consortium, the Defeat GBM Research Collaborative, NBTS
seeks to leverage team science and collaboration in order to accelerate drug discovery and development activities to advance potentially new, more effective therapies to the clinic as quickly as possible. As such, we are
thrilled that Memorial Sloan Kettering Cancer Center will now be formally participating in this effort, adding
even more expertise to the Collaborative.”
Defeat GBM Research Collaborative is a multi-institutional, multi-disciplinary model for precision medicine
in brain cancer. Four “cores” – Discovery, Drug Development, Predictive Markers (Biomarkers), and
SMART/Adaptive Clinical Trials – composed of research teams led by leaders in the neuro-oncology field work
together to move scientific findings through the drug discovery and development process to the clinic. The goal
of Defeat GBM is to double the percentage of GBM patients surviving five years or more.
NBTS announced the launch of the Defeat GBM Research Collaborative in early 2013, bringing together
a team of expert cancer researchers to develop its unique collaborative scientific infrastructure and funding
model. Throughout 2013, research teams for each core were selected and assembled; project plans were finalized; and agreements were put in place with institutions representing these world-class scientists, including
MD Anderson Cancer Center and Ludwig Cancer Research. In 2014, funding initiated and research got underway.
The new agreement with MSK brings another world-class institution into the Defeat GBM Research Collaborative as an official “Scientific Collaborator” working toward the program’s goals.
“Memorial Sloan Kettering is deeply honored to join National Brain Tumor Society in this important endeavor,” said Lisa DeAngelis, MD, Chair, Department of Neurology. “We see great promise in Dr. Mellinghoff’s
project, and anticipate that it will considerably advance our understanding of the biology of drug response in
glioma.”
Ingo Mellinghoff, MD has been leading a supplemental project to Defeat GBM’s Drug Development Core
focusing on the systems biology of drug response in GBM tumors. These efforts will now be expanded, so that
information from this project can be fed to other cores to help discovery science and biomarker development
efforts understand what happens in GBM tumors, at a systems level, when they are hit with different drugs.
Eventually, targets and compounds of interest identified in the Defeat GBM’s Discovery and Drug Development
cores will be evaluated for systems biology response by Dr. Mellinghoff as well, as means to validate their
potential activity and build their preclinical profiles.
“Dr. Mellinghoff is a true visionary in targeted therapy for glioblastoma and the application of complex,
adaptive cellular systems to understand the mechanism of tumor resistance and heterogeneity,” said Dr. W.K.
Alfred Yung of MD Anderson Cancer Center and Scientific Director of the Defeat GBM Research Collaborative.
“Understanding how tumor cells react and change as part of a system in the face of treatment, will impart a more
predictive approach to new therapy development to the other Cores in the Defeat GBM Research Collaborative,
thus enhancing the preclinical and translational research process. As Scientific Director of the Defeat GBM
Research Collaborative, I’m excited that we’ll be able to engage more deeply with Dr. Mellinghoff and his lab.”
NewsRx LLC
(2016-07-18), National Brain Tumor Society & Memorial Sloan Kettering Agreement Bolsters Research Efforts to Combat Deadliest Brain Cancer, Clinical Trials Week, 363, ISSN: 1543-6764, BUTTER® ID: 012057420
60
Mental Health
New Depression Study Findings Have Been Reported from Washington University (Use of
the Temperament and Character Inventory to Predict Response to Repetitive Transcranial
Magnetic Stimulation for Major Depression)
By a News Reporter-Staff News Editor at Psychology & Psychiatry Journal – Research findings on Mental
Health are discussed in a new report. According to news reporting from St. Louis, Missouri, by VerticalNews
journalists, research stated, “The goal of this study was to investigate the utility of the Temperament and
Character Inventory (TCI) in predicting antidepressant response to repetitive transcranial magnetic stimulation (rTMS). Although rTMS of the dorsolateral prefrontal cortex is an established antidepressant treatment,
little is known about predictors of response.”
The news correspondents obtained a quote from the research from Washington University, “The TCI measures multiple personality dimensions (harm avoidance, novelty seeking, reward dependence, persistence,
self-directedness, self-transcendence, and cooperativeness), some of which have predicted response to pharmacotherapy and cognitive-behavioral therapy. A previous study suggested a possible association between selfdirectedness and response to rTMS in melancholic depression, although this was limited by the fact that melancholic depression is associated with a limited range of TCI profiles. Nineteen patients with a major depressive
episode completed the TCI before a clinical course of rTMS over the dorsolateral prefrontal cortex. Treatment
response was defined as >= 50% decrease in scores on the Hamilton Rating Scale for Depression (Ham-D).
Baseline scores on each TCI dimension were compared between responders and nonresponders through analysis of variance. Pearson correlations were also calculated for temperament/character scores in comparison
with percentage improvement in Ham-D scores. Eleven of the 19 patients responded to rTMS. T-scores for persistence were significantly higher in responders than in nonresponders (P=0.022). Linear regression revealed
a correlation between persistence scores and percentage improvement in Ham-D scores. Higher persistence
scores predicted antidepressant response to rTMS. This may be explained by rTMS-induced enhancement of
cortical excitability, which has been found to be decreased in patients with high persistence.”
According to the news reporters, the research concluded: “Personality assessment that includes measurement of TCI persistence may be a useful component of precision medicine initiatives in rTMS for depression.”
For more information on this research see: Use of the Temperament and Character Inventory to Predict Response to Repetitive Transcranial Magnetic Stimulation for Major Depression. Journal of Psychiatric
Practice , 2016;22(3):193-202. Journal of Psychiatric Practice can be contacted at: Lippincott Williams &
Wilkins, Two Commerce Sq, 2001 Market St, Philadelphia, PA 19103, USA. (Lippincott Williams and Wilkins
- www.lww.com; Journal of Psychiatric Practice - http://journals.lww.com/practicalpsychiatry/
pages/default.aspx )
Our news journalists report that additional information may be obtained by contacting S.H. Siddiqi, Washington University, Sch Med, Dept. of Psychiat, POB 8134660 S Euclid Ave, St Louis, MO 63110, United States.
Additional authors for this research include R. Chockalingam, C.R. Cloninger, E.J. Lenze and P. Cristancho.
NewsRx LLC
(2016-06-18), New Depression Study Findings Have Been Reported from Washington University (Use of the
Temperament and Character Inventory to Predict Response to Repetitive Transcranial Magnetic Stimulation
for Major Depression), Psychology & Psychiatry Journal, 180, ISSN: 1944-2726, BUTTER® ID: 011858005
Drugs and Therapies
New Findings from J. Vaught and Co-Authors Describe Advances in Pharmacology and
Toxicology (Biobanking Comes of Age: The Transition to Biospecimen Science)
By a News Reporter-Staff News Editor at Drug Week – A new study on Drugs and Therapies is now available.
According to news reporting originating from Vancouver, Canada, by NewsRx editors, the research stated,
“Biobanking involves the collection, processing, storage, and distribution of biological specimens and the policies and procedures necessary to accomplish those aims successfully. Although biobanking may also involve
collections for environmental studies or museum archives, most efforts to standardize biobanking practices
have been directed toward human biomedical research.”
Our news editors obtained a quote from the research, “Initially focused primarily on collecting samples
for diagnostic purposes in pathology settings, biobanks have evolved into complex organizations engaged in
advancing personalized (or precision) medicine and translational research. This evolution has involved the
61
development of biobanking best practices and the transformation of a field driven by empirical approaches
into the emerging area of biospecimen science. It has become increasingly important to develop evidencebased practices for collecting biospecimens and data that can be shared with confidence with international
collaborators.”
According to the news editors, the research concluded: “Aside from these technical approaches, other factors
play crucial roles, such as ethical and regulatory issues, business planning and sustainability, and approaches
to data collection and sharing.”
For more information on this research see: Biobanking Comes of Age: The Transition to Biospecimen Science. Annual Review of Pharmacology and Toxicology , 2016;56():211-28.
The news editors report that additional information may be obtained by contacting J. Vaught, International
Society for Biological and Environmental Repositories, Vancouver, British Columbia V5Z 1B3, Canada.
NewsRx LLC
(2016-06-10), New Findings from J. Vaught and Co-Authors Describe Advances in Pharmacology and Toxicology (Biobanking Comes of Age: The Transition to Biospecimen Science), Drug Week, 579, ISSN: 1532-4575,
BUTTER® ID: 011897311
OmniSeq, Llc
OmniSeq Receives New York CLEP Approval For 144 Gene Comprehensive Panel And
Partners With Cure Forward To Advance Precision Medicine For Cancer Patients
By a News Reporter-Staff News Editor at Clinical Trials Week – OmniSeq, a subsidiary of Roswell Park Cancer
Institute, received New York State Clinical Laboratory Evaluation Program (CLEP) approval for its OmniSeq
Comprehensive(SM) panel, a 144 gene, pan-cancer, next-generation sequencing (NGS) tumor profiling diagnostic panel to guide oncology treatment decision-making. OmniSeq Comprehensive provides access to more than
650 therapeutic associations and 850 clinical trial associations, and identifies contraindications for drugs not
appropriate for a particular patient.
“OmniSeq is proud to receive New York State CLEP approval for OmniSeq Comprehensive. CLEP is the
highest standard of Clinical Laboratory Improvement Amendments (CLIA) laboratory validation,” said Carl
Morrison, MD, DVM, President and Chief Scientific Officer of OmniSeq and Executive Director of the Roswell
Park Center for Personalized Medicine. “OmniSeq Comprehensive’s small sample requirements - as little as
one tenth of the typical sample requirement, amongst the least in the industry - will enable us to generate
complete results from a high proportion of precious samples. This panel enables physicians to zero in on the
most important results for patient outcomes.”
According to a November, 2015 article in the Journal of Clinical Oncology (Schwaederle et. al), patients
who received targeted therapy in a personalized way based on harboring a molecular biomarker demonstrated
significantly better response and survival rates. OmniSeq Comprehensive was developed using Thermo Fisher
Scientific’s Oncomine reagents and the Ion Torrent sequencing system, which are also being used by the National Cancer Institute’s (NCI) MATCH trial (NCT02465060) - the largest precision medicine clinical trial of
its kind. Thus, OmniSeq Comprehensive improves patient access to targeted therapeutic options available in
clinical trials.
As part of OmniSeq’s mission to advance precision medicine, OmniSeq is also proud to announce it is partnering with Cure Forward to help patients control their care and be recruited for clinical trials through its
Clinical Trial Exchange. Patients can obtain their OmniSeq Comprehensive test results online and directly
apply that information to understand their disease and treatment options. On Cure Forward’s platform, trial
sponsors can identify patients who match their selection criteria and invite them to apply to their studies.
“Patients and their families are becoming increasingly active participants in devising strategies for their
care. Precision medicine is a framework for matching patients to the particular therapies that match their
condition,” said Martin Naley, Founder, Chief Strategy Officer of Cure Forward.
“Cure Forward offers a unique platform that proactively connects patients to clinical trials, which fits perfectly into our mission as we offer patients more than just therapeutically actionable molecular diagnostic
testing, but a complete service experience,” said Mark Gardner, Chief Executive Officer of OmniSeq. “Our vision is find the right drug or the right clinical trial for every patient, and our partnership with Cure Forward
helps OmniSeq fulfill our promise to patients and providers to deliver the best therapeutic support possible.”
NewsRx LLC
62
(2016-07-11), OmniSeq Receives New York CLEP Approval For 144 Gene Comprehensive Panel And Partners With Cure Forward To Advance Precision Medicine For Cancer Patients, Clinical Trials Week, 565, ISSN:
1543-6764, BUTTER® ID: 012021081
Fight Against Cancer Innovation Trust
Propellon Therapeutics to Accelerate WDR5 Inhibitor Development for Cancers
By a News Reporter-Staff News Editor at Clinical Trials Week – FACIT has announced the creation of a new
Ontario biotechnology company, Propellon Therapeutics Inc. (“Propellon” or “the Company”), to manage the
development and commercialization of a portfolio of first-in-class WDR5 inhibitors for the treatment of various
cancers. Epigenetic targets such as WDR5 represent an exciting class of therapies, possessing the potential
to contribute significantly in precision medicine for cancer patients. Substantial advances have been achieved
with the series of proprietary small molecule WDR5 inhibitors discovered and developed by the Drug Discovery
team at Ontario Institute for Cancer Research (“OICR”). The formation of Propellon reflects a natural translation of early-stage breakthrough cancer innovations, and enables important development activities including
attracting private sector investment and industry partnerships.
The WDR5 protein is critical for the formation and epigenetic activities of MLL1-associated methylation
complexes. Deregulation of the MLL1 complexes has been implicated in acute lymphoblastic leukemia (ALL)
and acute myeloid leukemia (AML). Propellon’s series of novel WDR5 epigenetic modifiers target protein-protein
interactions within the WDR5/MLL1 complex and thereby disrupt pro-cancer methylation activities. This
unique approach may also improve clinical outcomes in patients with solid tumours and is currently under
investigation by the Company and its partners.
Propellon will continue to collaborate with the OICR Drug Discovery team and build upon their significant
progress in preclinical development. During an initial transitional period, FACIT will provide interim corporate
management. Should candidate drugs achieve the milestone of testing in human patients, Propellon has the
potential to access the robust infrastructure for high content clinical trials in Ontario.
“OICR and FACIT share a mission to translate advancements in cancer care for the benefit of both patients
and the Ontario economy,” said Jeff Courtney, Chief Commercial Officer of FACIT. “With a focus on accelerating the development of these promising WDR5 inhibitor therapies, Propellon is well-positioned to attract
investment and global pharma partners to Ontario, and increase the likelihood of successful translation and
improved patient care.”
NewsRx LLC
(2016-07-04), Propellon Therapeutics to Accelerate WDR5 Inhibitor Development for Cancers, Clinical Trials Week, 486, ISSN: 1543-6764, BUTTER® ID: 011985676
RaNA Therapeutics
RaNA Therapeutics to Present at Upcoming Conferences
By a News Reporter-Staff News Editor at Biotech Business Week – RaNA Therapeutics, a leading biotech
developing a new class of revolutionary RNA-targeted medicines, announced RaNA’s CEO Ronald Renaud will
present a corporate overview at the JMP Securities Life Science Conference at 4:30 p.m. EST on Tuesday, June
21, 2016, at the St. Regis New York.
On June 16, the company will also present a poster titled “A Novel Epigenetic Approach to Treat SMA”
at Cure SMA’s Annual SMA Research Conference in Anaheim, California. Sponsored by Cure SMA annually
since 1988, the conference aims to bring together leading spinal muscular atrophy researchers, clinicians and
families living with the disease. About RaNA Therapeutics RaNA Therapeutics is a leading biotechnology
company committed to the development of next generation RNA-targeted medicines that selectively upregulate
gene expression to increase endogenous protein levels for therapeutic benefit. The company was founded by
preeminent global leaders who pioneered the relationship between lncRNA and chromatic modifiers, as well
as breakthrough oligonucleotide technologies. RaNA’s mission is to improve the lives of people suffering from
serious life-altering diseases by creating precision medicines that can change the course of their condition.
RaNA’s technology has broad therapeutic potential to treat a wide range of diseases, including rare genetic
disorders. The company has current lead programs in spinal muscular atrophy and Friedreich’s ataxia. For
more information about the company and its platforms, please visit www.ranarx.com. View source version on
63
NewsRx LLC
(2016-06-27), RaNA Therapeutics to Present at Upcoming Conferences, Biotech Business Week, 62, ISSN:
1543-6861, BUTTER® ID: 011945605
Gastroenterology
Reports Outline Crohn’s Disease Study Findings from University Medical Center (A
Prospective Pharmacogenomic Study of Crohn’s Disease Patients during Routine Therapy
with Anti-TNF- Drug Adalimumab: Contribution of ATG5, NFKB1, and CRP Genes to ...)
By a News Reporter-Staff News Editor at Biotech Week – A new study on Gastroenterology is now available.
According to news reporting out of Ljubljana, Slovenia, by NewsRx editors, research stated, “Crohn’s disease is
often treated with the anti-tumor necrosis factor- drug adalimumab. However, about 20%-40% of patients do
not display adequate therapeutic response.”
Our news journalists obtained a quote from the research from University Medical Center, “We prospectively evaluated, during a routine therapy of Crohn’s disease patients, the candidate autophagy-related genes
ATG12 and ATG5 and the inflammation-related genes NFKB1, NFKBIA, and CRP as potential predictors
of adalimumab treatment response (pharmacodynamics). The associations of haplotypes and SNPs in these
genes with response to drug therapy, biochemical parameters, and body mass were determined at baseline
and after 4, 12, 20, and 30 weeks of therapy. Association analysis showed that haplotypes defined with the
SNPs rs9373839 and rs510432 in ATG5 gene were significantly associated with positive response to therapy (p
<0.002). In addition, allele C and genotypes CC and CT of the rs1130864 in the CRP gene were positively associated with therapeutic response (p <0.002). To the best of our knowledge, this is the first report that supports
the association of SNPs in ATG5 and CRP genes with response to adalimumab therapy in Crohn’s disease.”
According to the news editors, the research concluded: “Further study of these biological pathways in larger
and independent clinical samples is warranted as novel streams of research on precision medicine and diagnostics for Crohn’s disease.”
For more information on this research see: A Prospective Pharmacogenomic Study of Crohn’s Disease Patients during Routine Therapy with Anti-TNF- Drug Adalimumab: Contribution of ATG5, NFKB1, and CRP
Genes to Pharmacodynamic Variability. Omics-A Journal of Integrative Biology , 2016;20(5):296-309. Omics-A
Journal of Integrative Biology can be contacted at: Mary Ann Liebert, Inc, 140 Huguenot Street, 3RD Fl, New
Rochelle, NY 10801, USA.
Our news journalists report that additional information may be obtained by contacting M. Dezelak, Univ
Med Center Ljubljana, Ljubljana, Slovenia. Additional authors for this research include K. Repnik, S. Koder,
I. Ferkolj and U. Potocnik.
NewsRx LLC
(2016-06-22), Reports Outline Crohn’s Disease Study Findings from University Medical Center (A Prospective Pharmacogenomic Study of Crohn’s Disease Patients during Routine Therapy with Anti-TNF- Drug Adalimumab: Contribution of ATG5, NFKB1, and CRP Genes to ...), Biotech Week, 247, ISSN: 1537-4699, BUTTER®
ID: 011930623
64
Pharmacology
Researchers at Duke University Discuss Findings in Pharmacology (Metabolomic Signatures
for Drug Response Phenotypes: Pharmacometabolomics Enables Precision Medicine)
By a News Reporter-Staff News Editor at Drug Week – Current study results on Pharmacology have been published. According to news reporting originating in Durham, North Carolina, by NewsRx journalists, research
stated, “The scaling up of data in clinical pharmacology and the merger of systems biology and pharmacology
has led to the emergence of a new discipline of Quantitative and Systems Pharmacology (QSP). This new research direction might significantly advance the discovery, development, and clinical use of therapeutic drugs.”
The news reporters obtained a quote from the research from Duke University, “Research communities from
computational biology, systems biology, and biological engineering–working collaboratively with pharmacologists, geneticists, biochemists, and analytical chemists–are creating and modeling large data on drug effects
that is transforming our understanding of how these drugs work at a network level. In this review, we highlight developments in a new and rapidly growing field–pharmacometabolomics–in which large biochemical
data-capturing effects of genome, gut microbiome, and environment exposures is revealing information about
metabotypes and treatment outcomes, and creating metabolic signatures as new potential biomarkers.”
According to the news reporters, the research concluded: “Pharmacometabolomics informs and complements
pharmacogenomics and together they provide building blocks for QSP.”
For more information on this research see: Metabolomic Signatures for Drug Response Phenotypes: Pharmacometabolomics Enables Precision Medicine. Clinical Pharmacology and Therapeutics , 2015;98(1):715. (Nature Publishing Group - http://www.nature.com/ ; Clinical Pharmacology and Therapeutics http://www.nature.com/clpt/ )
Our news correspondents report that additional information may be obtained by contacting R. KaddurahDaouk, Dept. of Psychiatry and Behavioral Sciences, Duke University, Durham, North Carolina, United States.
NewsRx LLC
(2016-07-22), Researchers at Duke University Discuss Findings in Pharmacology (Metabolomic Signatures
for Drug Response Phenotypes: Pharmacometabolomics Enables Precision Medicine), Drug Week, 633, ISSN:
1532-4575, BUTTER® ID: 012082505
Nanoparticles
Researchers at Nankai University Release New Data on Nanoparticles (Intraparticle
Molecular Orbital Engineering of Semiconducting Polymer Nanoparticles as Amplified
Theranostics for in Vivo Photoacoustic Imaging and Photothermal Therapy)
By a News Reporter-Staff News Editor at Journal of Engineering – Current study results on Nanoparticles
have been published. According to news reporting originating in Tianjin, People’s Republic of China, by VerticalNews journalists, research stated, “Optical theranostic nanoagents that seamlessly and synergistically
integrate light-generated signals with photothermal or photodynamic therapy can provide opportunities for
cost-effective precision medicine, while the potential for clinical translation requires them to have good biocompatibility and high imaging/therapy performance. We herein report an intraparticle molecular orbital engineering approach to simultaneously enhance photoacoustic brightness and photothermal therapy efficacy of
semiconducting polymer nanoparticles (SPNs) for in vivo imaging and treatment of cancer.”
The news reporters obtained a quote from the research from Nankai University, “The theranostic SPNs
have a binary optical component nanostructure, wherein a near-infrared absorbing semiconducting polymer
and an ultrasmall carbon dot (fullerene) interact with each other to induce photoinduced electron transfer
upon light irradiation. Such an intraparticle optoelectronic interaction augments heat generation and consequently enhances the photoacoustic signal and maximum photothermal temperature of SPNs by 2.6- and
1.3-fold, respectively. With the use of the amplified SPN as the theranostic nanoagent, it permits enhanced
photoacoustic imaging and photothermal ablation of tumor in living mice.”
According to the news reporters, the research concluded: “Our study thus not only introduces a category
of purely organic optical theranostics but also highlights a molecular guideline to amplify the effectiveness of
light intensive imaging and therapeutic nanosystems.”
For more information on this research see: Intraparticle Molecular Orbital Engineering of Semiconducting
Polymer Nanoparticles as Amplified Theranostics for in Vivo Photoacoustic Imaging and Photothermal Therapy.
ACS Nano , 2016;10(4):4472-4481. ACS Nano can be contacted at: Amer Chemical Soc, 1155 16TH St, NW,
65
Washington, DC 20036, USA. (American Chemical Society - www.acs.org; ACS Nano - http://www.pubs.
acs.org/journal/ancac3 )
Our news correspondents report that additional information may be obtained by contacting Y. Lyu, Nankai
Univ, Coll Life Sci, Minist Educ, State Key Lab Med Chem BiolKey Lab Bioact Mat, Tianjin 300071, People’s
Republic of China. Additional authors for this research include Y. Fang, Q.Q. Miao, X. Zhen, D. Ding and K.Y.
Fu.
NewsRx LLC
(2016-05-30), Researchers at Nankai University Release New Data on Nanoparticles (Intraparticle Molecular Orbital Engineering of Semiconducting Polymer Nanoparticles as Amplified Theranostics for in Vivo Photoacoustic Imaging and Photothermal Therapy), Journal of Engineering, 1833, ISSN: 1945-872X, BUTTER®
ID: 011759626
Drug Development
Researchers at University of California Release New Data on Drug Development (Click
chemistry, 3D-printing, and omics: the future of drug development)
By a News Reporter-Staff News Editor at Drug Week – New research on Drug Development is the subject of
a report. According to news reporting originating in San Diego, California, by NewsRx journalists, research
stated, “Genomics is a disruptive technology, having revealed that cancers are tremendously complex and differ
from patient to patient. Therefore, conventional treatment approaches fit poorly with genomic reality.”
The news reporters obtained a quote from the research from the University of California, “Furthermore, it is
likely that this type of complexity will also be observed in other illnesses. Precision medicine has been posited as
a way to better target disease-related aberrations, but developing drugs and tailoring therapy to each patient’s
complicated problem is a major challenge. One solution would be to match patients to existing compounds based
on in silico modeling. However, optimization of complex therapy will eventually require designing compounds
for patients using computer modeling and just-in-time production, perhaps achievable in the future by threedimensional (3D) printing. Indeed, 3D printing is potentially transformative by virtue of its ability to rapidly
generate almost limitless numbers of objects that previously required manufacturing facilities. Companies are
already endeavoring to develop affordable 3D printers for home use. An attractive, but as yet scantily explored,
application is to place chemical design and production under digital control. This could be accomplished by
utilizing a 3D printer to initiate chemical reactions, and print the reagents and/or the final compounds directly.
Of interest, the Food and Drug Administration (FDA) has recently approved a 3D printed drug-levetiracetamindicated for seizures.”
According to the news reporters, the research concluded: “Further, it is now increasingly clear that biologic
materials-tissues, and eventually organs-can also be ‘printed.’ In the near future, it is plausible that highthroughput computing may be deployed to design customized drugs, which will reshape medicine.”
For more information on this research see: Click chemistry, 3D-printing, and omics: the future of drug
development. Oncotarget , 2016;7(3):2155-8.
Our news correspondents report that additional information may be obtained by contacting R. Kurzrock,
Center for Personalized Cancer Therapy and Division of Hematology and Oncology, University of California
San Diego Moores Cancer Center, San Diego, CA, United States.
NewsRx LLC
(2016-07-22), Researchers at University of California Release New Data on Drug Development (Click chemistry, 3D-printing, and omics: the future of drug development), Drug Week, 97, ISSN: 1532-4575, BUTTER®
ID: 012082404
66
Drugs and Therapies
Researchers at Vanderbilt University Have Reported New Data on Clinical Pharmacology
and Therapeutics (Physician Response to Implementation of Genotype-Tailored Antiplatelet
Therapy)
By a News Reporter-Staff News Editor at Drug Week – Current study results on Drugs and Therapies have been
published. According to news reporting originating from Nashville, Tennessee, by NewsRx correspondents, research stated, “Physician responses to genomic information are vital to the success of precision medicine initiatives. We prospectively studied a pharmacogenomics implementation program for the propensity of clinicians
to select antiplatelet therapy based on CYP2C19 loss-of-function variants in stented patients.”
Our news editors obtained a quote from the research from Vanderbilt University, “Among 2,676 patients, 514
(19.2%) were found to have a CYP2C19 variant affecting clopidogrel metabolism. For the majority (93.6%) of the
cohort, cardiologists received active and direct notification of CYP2C19 status. Over 12 months, 57.6% of poor
metabolizers and 33.2% of intermediate metabolizers received alternatives to clopidogrel. CYP2C19 variant
status was the most influential factor impacting the prescribing decision (hazard ratio [HR] in poor metabolizers
8.1, 95% confidence interval [CI] [5.4, 12.2] and HR 5.0, 95% CI [4.0, 6.3] in intermediate metabolizers), followed
by patient age and type of stent implanted.”
According to the news editors, the research concluded: “We conclude that cardiologists tailored antiplatelet
therapy for a minority of patients with a CYP2C19 variant and considered both genomic and nongenomic risks
in their clinical decision-making.”
For more information on this research see: Physician Response to Implementation of Genotype-Tailored
Antiplatelet Therapy. Clinical Pharmacology & Therapeutics , 2016;100(1):67-74. Clinical Pharmacology &
Therapeutics can be contacted at: Wiley-Blackwell, 111 River St, Hoboken 07030-5774, NJ, USA. (Nature Publishing Group - http://www.nature.com/ ; Clinical Pharmacology & Therapeutics - http://www.nature.
com/clpt/ )
The news editors report that additional information may be obtained by contacting J.F. Peterson, Vanderbilt
University, Sch Med, Dept. of Pathol Microbiol & Immunol, Nashville, TN 37212, United States. Additional
authors for this research include J.R. Field, K.M. Unertl, J.S. Schildcrout, D.C. Johnson, Y. Shi, I. Danciu, J.H.
Cleator, J.M. Pulley, J.A. McPherson, J.C. Denny, M. Laposata, D.M. Roden and K.B. Johnson.
NewsRx LLC
(2016-07-15), Researchers at Vanderbilt University Have Reported New Data on Clinical Pharmacology and
Therapeutics (Physician Response to Implementation of Genotype-Tailored Antiplatelet Therapy), Drug Week,
699, ISSN: 1532-4575, BUTTER® ID: 012048127
Diabetes
Researchers from University of Liege Report Recent Findings in Diabetes (Y Precision
medicine: The future in diabetes care?)
By a News Reporter-Staff News Editor at Diabetes Week – Investigators publish new report on Diabetes. According to news reporting out of Liege, Belgium, by NewsRx editors, the research stated, “Personalized medicine
aims at better targeting therapeutic intervention to the individual to maximize benefit and minimize harm.
Type 2 diabetes (T2D) is a heterogeneous disease from a genetic, pathophysiological and clinical point of view.”
Our news journalists obtained a quote from the research from the University of Liege, “Thus, the response
to any antidiabetic medication may considerably vary between individuals. Numerous glucose-lowering agents,
with different mechanisms of action, have been developed, a diversified armamentarium that offers the possibility of a patient-centred therapeutic approach. In the current clinical practice, a personalized approach is
only based upon phenotype, taking into account patient and disease individual characteristics. If this approach
may help increase both efficacy and safety outcomes, there remains considerable room for improvement. In recent years, many efforts were taken to identify genetic and genotype SNP’s (Single Nucleotide Polymorphism’s)
variants that influence the pharmacokinetics, pharmacodynamics, and ultimately the therapeutic response
of oral glucose-lowering drugs. This approach mainly concerns metformin, sulphonylureas, meglitinides and
thiazolidinediones, with only scarce data concerning gliptins and gliflozins yet. However, the contribution of
pharmacogenetics and pharmacogenomics to personalized therapy still needs to mature greatly before routine
clinical implementation is possible.”
67
According to the news editors, the research concluded: “This review discusses both opportunities and challenges of precision medicine and how this new paradigm may lead to a better individualized treatment of T2D.”
For more information on this research see: Y Precision medicine: The future in diabetes care? Diabetes
Research and Clinical Practice , 2016;117():12-21. Diabetes Research and Clinical Practice can be contacted
at: Elsevier Ireland Ltd, Elsevier House, Brookvale Plaza, East Park Shannon, Co, Clare, 00000, Ireland.
(Elsevier - www.elsevier.com; Diabetes Research and Clinical Practice - http://www.journals.elsevier.
com/diabetes-research-and-clinical-practice/ )
Our news journalists report that additional information may be obtained by contacting A.J. Scheen, University of Liege, CHU Liege, CIRM, Clin Pharmacol Unit, Liege, Belgium.
NewsRx LLC
(2016-07-18), Researchers from University of Liege Report Recent Findings in Diabetes (Y Precision
medicine: The future in diabetes care?), Diabetes Week, 81, ISSN: 1537-1433, BUTTER® ID: 012057872
Oncology
Researchers from Wake Forest University School of Medicine Detail New Studies and
Findings in the Area of Lung Cancer (Treatment of brain metastases of lung cancer in the
era of precision medicine)
By a News Reporter-Staff News Editor at Cancer Vaccine Week – Investigators discuss new findings in Oncology. According to news reporting originating from Winston Salem, North Carolina, by NewsRx correspondents,
research stated, “Common and deadly complications of non-small cell lung cancer (NSCLC) are brain metastases (BM). BM portends a poorer prognosis with limited effective treatment options and current management
strategies present several challenges from iatrogenic complications of supportive medications, optimal delivery
of drug across the blood-brain barrier, and preservation of neurocognitive function.”
Our news editors obtained a quote from the research from the Wake Forest University School of Medicine,
“Long term side effects and survivorship issues have become more evident in the era of targeted therapy where
a systemic disease is much better controlled. Targeted therapies and immunotherapy are beginning to provide
improvements in responses and survival rates. With further advancements and experience, our knowledge in
this era of precision medicine will likely lead to strides in improving the quality of life and overall survival of
patients with BM from NSCLC.”
According to the news editors, the research concluded: “In this review, we present the most recent updates
in treatment of BM in NSCLC in regards to targeted and immunotherapy.”
For more information on this research see: Treatment of brain metastases of lung cancer in the era of
precision medicine. Frontiers In Bioscience , 2016;8():219-32.
The news editors report that additional information may be obtained by contacting M.E. Haughton, Dept.
of Medicine, Hematology and Oncology, Wake Forest School of Medicine, Winston-Salem, NC, United States.
Additional authors for this research include M.D. Chan, K. Watabe, M. Bonomi, W. Debinski, G.J. Lesser and
J. Ruiz.
NewsRx LLC
(2016-05-30), Researchers from Wake Forest University School of Medicine Detail New Studies and Findings
in the Area of Lung Cancer (Treatment of brain metastases of lung cancer in the era of precision medicine),
Cancer Vaccine Week, 41, ISSN: 1543-6802, BUTTER® ID: 011749459
68
Rxight
Rxight Advances President’s Precision Medicine Initiative
By a News Reporter-Staff News Editor at Drug Week – In an effort to expand upon President Obama’s Precision
Medicine Initiative and explore innovative ways to individualize patient care, representatives from MD Labs
met with Senior Leadership at the White House Office of Science and Technology Policy (OSTP).
https://photos.prnewswire.com/prnvar/20160516/367962
During their visit, the team discussed how Rxight(™), the most comprehensive pharmacogenetic program
available, helps people receive the right medication and dosing, and avoid medications that could cause unintended side effects and complications. Rxight(™) uses state-of-the-art pharmacogenetic technology that focuses
on the patient’s DNA, rather than relying on a process of trial and error to predict medication response.
President Obama alluded to this technology on January 30, 2015, in his remarks on Precision Medicine
when he said, “What if figuring out the right dose of medicine was as simple as taking our temperature? And
that’s the promise of precision medicine–delivering the right treatments, at the right time, every time to the
right person.”
“We’re proud to support this promising initiative that will help change how medicine is practiced today and
in the future,” said Denis Grizelj, Co-Founder/CEO of MD Labs, creators of the Rxight(™) program. “We want
to help healthcare providers maximize their clinical care, and through Rxight they can be further assured that
the medications they prescribe are tailored for their patients and can reduce the risk of side effects.”
The Rxight(™) pharmacogenetic program is truly unique because it covers over 210 prescription and overthe-counter (OTC) medications, and includes a Personalized Medication Review(®) with a Rxight(™) Certified
Pharmacist. Through a simple cheek swab, the test can identify key genetic markers that highlight how a
patient’s body metabolizes and processes different medications. People who choose to get the test receive a
lifetime of benefits as medication needs are expected to change as one ages.
“With Rxight, we can identify how genetic variation can lead to unintended medication consequences that
may contribute to patients not taking their medication as prescribed and also help reduce long-term health care
costs of medications not suited for that individual.” said Dr. Amina Abubakar, a Rxight™ Certified Pharmacist
running her own pharmacy in Charlotte, North Carolina.
NewsRx LLC
(2016-06-03), Rxight Advances President’s Precision Medicine Initiative, Drug Week, 1644, ISSN: 15324575, BUTTER® ID: 011795480
Oncology
Studies from Harvard University Add New Findings in the Area of Olfactory Neuroblastoma
(Recurrent Olfactory Neuroblastoma Treated With Cetuximab and Sunitinib A Case Report)
By a News Reporter-Staff News Editor at Biotech Week – Current study results on Oncology have been published. According to news reporting from Boston, Massachusetts, by NewsRx journalists, research stated,
“Olfactory neuroblastoma (ONB) is a rare cancer originating in the olfactory epithelium of the nasal vault.
The recurrence rate of ONB is high, as the standard treatment of surgery followed by radiotherapy and/or
chemotherapy is usually unsuccessful.”
The news correspondents obtained a quote from the research from Harvard University, “The use of targeted
therapy based on individual genomic variations after cancer relapse has not been reported. Here, we present the
case of a 44-year-old man who was diagnosed with recurrent ONB and treated with a regimen developed using
whole exome sequencing. Potential targets were first identified and then matched to appropriate drugs. Gene
mutations in the genes encoding EGFR, FGFR2, KDR, and RET were discovered in the patient’s tumor tissue
by whole exome sequencing and the patient was treated with a combination of the targeted drugs cetuximab
and sunitinib. Five days after treatment, enhancement magnetic resonance imaging showed a 65% reduction
in tumor size, and the Visual analog scale headache scores went down to 2/10 from 10/10. Repeat imaging at 1
month showed a complete response.”
According to the news reporters, the research concluded: “This study represents the first demonstration of
an effective personalized treatment of ONB by targeted drugs, and sheds light on how precision medicine can be
used to treat recurrent ONB that fails to respond to routine tumor resection, radiotherapy, and/or chemotherapy.”
69
For more information on this research see: Recurrent Olfactory Neuroblastoma Treated With Cetuximab and Sunitinib A Case Report. Medicine , 2016;95(18):141-145. Medicine can be contacted at: Lippincott Williams & Wilkins, Two Commerce Sq, 2001 Market St, Philadelphia, PA 19103, USA. (Elsevier www.elsevier.com; Medicine - http://www.journals.elsevier.com/medicine/ )
Our news journalists report that additional information may be obtained by contacting L.Z. Wang, Harvard
University, Beth Israel Deaconess Med Center, Sch Med, Boston, MA 02215, United States. Additional authors
for this research include Y. Ding, L. Wei, D.W. Zhao, R.Y. Wang, Y.W. Zhang, X.S. Gu and Z.Q. Wang.
NewsRx LLC
(2016-06-29), Studies from Harvard University Add New Findings in the Area of Olfactory Neuroblastoma
(Recurrent Olfactory Neuroblastoma Treated With Cetuximab and Sunitinib A Case Report), Biotech Week,
306, ISSN: 1537-4699, BUTTER® ID: 011968228
Oncology
Studies from Harvard University Add New Findings in the Area of Olfactory Neuroblastoma
(Recurrent Olfactory Neuroblastoma Treated With Cetuximab and Sunitinib A Case
Report) (Recurrent Olfactory Neuroblastoma Treated With Cetuximab and ...)
By a News Reporter-Staff News Editor at Ivy League Week – Current study results on Oncology have been
published. According to news reporting from Boston, Massachusetts, by NewsRx journalists, research stated,
“Olfactory neuroblastoma (ONB) is a rare cancer originating in the olfactory epithelium of the nasal vault.
The recurrence rate of ONB is high, as the standard treatment of surgery followed by radiotherapy and/or
chemotherapy is usually unsuccessful.”
The news correspondents obtained a quote from the research from Harvard University, “The use of targeted
therapy based on individual genomic variations after cancer relapse has not been reported. Here, we present the
case of a 44-year-old man who was diagnosed with recurrent ONB and treated with a regimen developed using
whole exome sequencing. Potential targets were first identified and then matched to appropriate drugs. Gene
mutations in the genes encoding EGFR, FGFR2, KDR, and RET were discovered in the patient’s tumor tissue
by whole exome sequencing and the patient was treated with a combination of the targeted drugs cetuximab
and sunitinib. Five days after treatment, enhancement magnetic resonance imaging showed a 65% reduction
in tumor size, and the Visual analog scale headache scores went down to 2/10 from 10/10. Repeat imaging at 1
month showed a complete response.”
According to the news reporters, the research concluded: “This study represents the first demonstration of
an effective personalized treatment of ONB by targeted drugs, and sheds light on how precision medicine can be
used to treat recurrent ONB that fails to respond to routine tumor resection, radiotherapy, and/or chemotherapy.”
For more information on this research see: Recurrent Olfactory Neuroblastoma Treated With Cetuximab and Sunitinib A Case Report. Medicine , 2016;95(18):141-145. Medicine can be contacted at: Lippincott Williams & Wilkins, Two Commerce Sq, 2001 Market St, Philadelphia, PA 19103, USA. (Elsevier www.elsevier.com; Medicine - http://www.journals.elsevier.com/medicine/ )
Our news journalists report that additional information may be obtained by contacting L.Z. Wang, Harvard
University, Beth Israel Deaconess Med Center, Sch Med, Boston, MA 02215, United States. Additional authors
for this research include Y. Ding, L. Wei, D.W. Zhao, R.Y. Wang, Y.W. Zhang, X.S. Gu and Z.Q. Wang.
NewsRx LLC
(2016-06-28), Studies from Harvard University Add New Findings in the Area of Olfactory Neuroblastoma
(Recurrent Olfactory Neuroblastoma Treated With Cetuximab and Sunitinib A Case Report) (Recurrent Olfactory Neuroblastoma Treated With Cetuximab and ...), Ivy League Week, 329, ISSN: 0000-0000, BUTTER® ID:
011965000
70
Oncology
Studies from Royal Marsden Hospital Add New Findings in the Area of Solid Cancer
(Immunotherapy Combined or Sequenced With Targeted Therapy in the Treatment of Solid
Tumors: Current Perspectives)
By a News Reporter-Staff News Editor at Cancer Vaccine Week – Fresh data on Oncology are presented in a
new report. According to news reporting out of London, United Kingdom, by NewsRx editors, research stated,
“The advent of newer immunotherapeutic and molecularly targeted agents has provided a number of effective
options for cancer treatment but has also added much complexity in selecting the best initial treatment or treatment plan for each patient. Molecularly targeted agents offer selectivity and are the cornerstone for ‘precision
medicine.’ While targeted agents are associated with high tumor response rates, patients inevitably develop
resistance to these drugs.”
Our news journalists obtained a quote from the research from Royal Marsden Hospital, “Immunotherapies
exploit the endogenous immune system to eradicate cancer and can produce durable disease control that results in longterm, treatment-free survival in some patients. Guidelines for treatment selection in patients
with specific tumor types and clinical features are routinely being reconsidered in order to accommodate the
increasingly complex treatment landscapes. Here, we review current perspectives on the use of immunotherapeutic agents, particularly immune checkpoint inhibitors (nivolumab, pembrolizumab, and ipilimumab), in
combination or in sequence with molecularly targeted agents in patients with advanced melanoma as well as
other tumor types.”
According to the news editors, the research concluded: “We further discuss remaining unmet needs for
patient selection and treatment with approved therapies.”
For more information on this research see: Immunotherapy Combined or Sequenced With Targeted Therapy in the Treatment of Solid Tumors: Current Perspectives. Jnci-Journal of the National Cancer Institute ,
2016;108(6):69-77. Jnci-Journal of the National Cancer Institute can be contacted at: Oxford Univ Press Inc,
Journals Dept, 2001 Evans Rd, Cary, NC 27513, USA.
Our news journalists report that additional information may be obtained by contacting M.B. Atkins, Royal
Marsden Hospital, London SW3 6JJ, United Kingdom.
NewsRx LLC
(2016-07-11), Studies from Royal Marsden Hospital Add New Findings in the Area of Solid Cancer (Immunotherapy Combined or Sequenced With Targeted Therapy in the Treatment of Solid Tumors: Current
Perspectives), Cancer Vaccine Week, 25, ISSN: 1543-6802, BUTTER® ID: 012021401
Type 2 Diabetes
U-M, international research team explore genetic architecture of type 2 diabetes
By a News Reporter-Staff News Editor at Asia Business Newsweekly – ANN ARBOR–New research from a
large international team of scientists offers a more complete picture of the genes responsible for type 2 diabetes,
demonstrating that previously identified common alleles shared by many in the world are the biggest culprits–
not the less common variants some scientists had hypothesized might play a large role in who gets the disease.
The researchers also identified a novel variant specific to East Asians through their study that analyzed
the genes of individuals from five ethnic groups, making this the largest multi-ethnic genetic sequencing study
published to date.
Their findings are reported in the July 11 issue of Nature.
Led by researchers at the University of Michigan School of Public Health, the Wellcome Trust Centre for
Human Genetics at the University of Oxford, the Broad Institute of MIT and Harvard, and the Massachusetts
General Hospital, more than 300 scientists from 22 countries used DNA from 120,000 individuals to pinpoint
genes and their variants, which influence the disease that impacts 10 percent of the world’s population.
“Our study has taken us to the most complete understanding yet of the genetic architecture of type 2 diabetes,” said Michael Boehnke, the Richard G. Cornell Distinguished University Professor of Biostatistics, director of the Center for Statistical Genetics at the U-M School of Public Health and one of three senior authors
of the study. “With this in-depth analysis we have obtained a more complete picture of the number and characteristics of the genetic variants that influence type 2 diabetes risk.”
Through the collaboration that combined two research projects–GoT2D and T2D-GENES–researchers identified more than a dozen genetic regions that harbor variants that influence risk to type 2 diabetes. The majority
71
of these were common variants, found in all human populations, and most had previously been detected by other
genome-wide association studies.
The team identified a novel association between type 2 diabetes and a variant in the gene PAX4, present only
in individuals from East Asia, including Korea, China and Singapore. They also demonstrated that variants
in the gene TM6SF2, previously linked to hepatic steatosis (commonly known as “fatty liver”), influences risk
of type 2 diabetes.
The researchers completed whole genome sequencing of more than 2,600 people and exome sequencing of
13,000, complemented with genome- or exome-wide array genotyping of 111,000 people. Exomes are the portion
of the genome that code for proteins.
Those studied included individuals with ancestral origins in Europe, South and East Asia, the Americas
and Africa. Most previous studies had involved only people of European ancestry.
“This study highlights both the challenges we face and the opportunities that exist in resolving the complex
processes underlying a disease such as type 2 diabetes,” said Mark McCarthy, the Robert Turner Professor of
Diabetic Medicine and group head of the Wellcome Trust Centre for Human Genetics. “In this study, we have
been able to highlight, with unprecedented precision, a number of genes directly involved in the development
of type 2 diabetes. These represent promising avenues for efforts to design new ways to treat or prevent the
disease.”
This in-depth look at the genetics of type 2 diabetes addresses the century-old debate of whether genetic
differences that influence predisposition to common diseases, like diabetes, are ones that are widely shared
within populations, or whether they are more likely to be rare or unique events, specific to an individual and
his or her family.
“While rare variants certainly influence type 2 diabetes risk, our results demonstrate that common variants
shared across populations explain most of the genetic risk to type 2 diabetes,” said U-M’s Boehnke.
Type 2 diabetes is a major threat to global public health as deaths from the disease continue to rise rapidly,
along with its complications including blindness, kidney failure, heart attack, stroke and amputation. Type 2
is the most common form of the diabetes; it impacts the body’s ability to regulate glucose (or sugar). In addition
to its genetic components, environment and behavior play major roles in who gets the disease, with obesity,
excessive stress and an inactive lifestyle among the contributors. Combined with other risk factors such as
smoking, high cholesterol and high blood pressure, people with type 2 diabetes have a much higher risk of
cardiovascular disease.
“To improve treatment and prevention for many diseases it is crucial to take into account the genetic makeup
of each person. Precision medicine relies on the deep understanding of the genetic architecture of a disease. Our
work provides this key information for type 2 diabetes,” said lead author Christian Fuchsberger, who worked on
the research as a postdoctoral fellow at U-M and now is at the Center for Biomedicine at the European Academy
in Bolzano, Italy.
“Our study tells us that most people are at risk for type 2 diabetes due to hundreds or even thousands of
genetic variants, typically shared across populations. While this large range of genetic risk may challenge our
efforts at precision medicine, our consortium also offers a publicly accessible dataset, unprecedented in scope,
for researchers around the world to advance our molecular understanding of type 2 diabetes,” said co-lead
author Jason Flannick, senior group leader at the Broad Institute of Harvard and MIT and research associate
at the Massachusetts General Hospital.
NewsRx LLC
(2016-07-26), U-M, international research team explore genetic architecture of type 2 diabetes, Asia Business Newsweekly, 241, ISSN: 1938-1808, BUTTER® ID: 012104037
72
Infectious Diseases
Vanderbilt, human vaccines project launch studies to decode human immune system
By a News Reporter-Staff News Editor at Clinical Trials Week – Researchers at Vanderbilt University Medical
Center this month began recruiting volunteers to participate in a clinical trial aimed at decoding the human
“immunome,” the genetic underpinnings of the immune system.
The study is the first phase of an international effort led by the Human Vaccines Project, a public-private
partnership of academic research centers, industry, non-profits and government agencies designed to accelerate
the development of next-generation vaccines and immunotherapies.
“I am tremendously excited to launch the Project’s Human Immunome Program, and look forward to generating important new data that should facilitate vaccine design for both infectious diseases and cancers,” said
James Crowe Jr., M.D., director of the Vanderbilt Vaccine Center, in a news release.
“The Project remains committed to rapid, open source communication of these data, to enable the community of global scientists to advance new and fundamental insights on how the human immune system can be
mobilized more effectively to fight disease,” Crowe said.
Progress against infectious diseases and cancer has been hampered by inadequate understanding of the
principal components of the immune system, particularly the massive collection of receptors on B and T cells.
These cells and their receptors enable the immune system to recognize, adapt to and attack an extraordinarily
large number of disease threats.
Thanks to recent advances in genomics, systems biology and bioinformatics, researchers have been able to
sequence and characterize many of these receptors. Such information is critical to engineering highly targeted
vaccines and therapies to confront major infectious diseases and emerging pandemics, as well as autoimmune
disorders and cancer.
“For the first time we have the technological tools to undertake such an ambitious project to decode the
human immune system,” said Wayne C. Koff, Ph.D., President and CEO of the Human Vaccines Project.
“As the Human Genome Project has ushered in a new era in precision medicine, the Human Vaccines Project
has the potential to enable a new era of vaccine and immunotherapeutic development against some of the world’s
most pressing diseases,” Koff said.
As a first step of the pilot study at VUMC, two healthy individuals will undergo leukapheresis, a blood
donation process in which large numbers of circulating white blood cells are removed by filtration, while red
blood cells are returned to the donor.
After the genetic sequences of all of the receptors on the white blood cells from each individual have been
determined, the study will be expanded to include about 100 subjects, representing different ages, genders,
ethnicities and geographies.
This will then form the baseline set of data to expand the study to more than 1,000 subjects, a subset of
whom will be vaccinated with licensed and experimental vaccines. Insights about the fundamental principles
of human immunology gained from this study will help guide next generation vaccine development.
The number of sequences acquired from them could be in the billions, and will constitute the first detailed
account of the immunome, said Crowe, who is the Ann Scott Carell Professor and professor of Pediatrics and
Pathology, Microbiology and Immunology.
Crowe and his colleagues will collaborate with one of the Project’s scientific hubs in La Jolla, California,
which is made up of the J. Craig Venter Institute (JCVI), La Jolla Institute for Allergy and Immunology, University of California, San Diego and The Scripps Research Institute.
JCVI and the San Diego Supercomputer Center at UC San Diego will serve as the Project’s global Bioinformatics Core.
The Human Vaccines Project will fund the project. Because it can cost more than $100,000 to acquire the
genetic repertoire from each individual’s white blood cells, due to the large amount of sequencing required,
eventually more than $100 million will be needed to fully decipher the human immunome, officials said.
But if the complete sequence of the human immunome could be achieved in a decade, it would be a “major
achievement,” said Nobel Laureate Peter Doherty, Ph.D., a member of the Human Vaccines Project Scientific
Steering Committee and Professor at the University of Melbourne.
It would “greatly advance our understanding of the human immune system, enabling rational and targeted
design of vaccines and immunotherapies for major global diseases,” Doherty said.
NewsRx LLC
(2016-07-04), Vanderbilt, human vaccines project launch studies to decode human immune system, Clinical
73
Chapter 6
Genetics
Wellness
ASHG opposes revised EEOC regulations weakening genetic privacy
By a News Reporter-Staff News Editor at Law & Health Weekly – BETHESDA, MD - The American Society
of Human Genetics (ASHG) opposes the U.S. Equal Employment Opportunity Commission’s (EEOC) newly revised Regulations under the Americans with Disabilities Act (ADA) and under the Genetic Information Nondiscrimination Act (GINA).
“These revisions will significantly weaken the patient privacy protections in the ADA and GINA,” said Derek
T. Scholes, PhD, ASHG Director of Science Policy. ASHG played a key role in the inception of GINA and
supported the law’s passage in 2008.
A key component of ADA and GINA is that they prevent workers and their families from being coerced into
sharing sensitive medical or genetic information with their employer. For GINA, genetic information encompasses not only employees’ genetic test results but also their family medical histories. According to a 2007
Senate report on GINA, Congress explicitly crafted the law to include spouses’ medical histories to prevent
employers from discriminating against employees because of the potential costs of their spouses’ medical care.
ADA and GINA do allow employer-sponsored wellness programs to request medical or genetic information,
but require that employee participation in such programs be entirely voluntary. Per the Senate report on GINA,
Congress incorporated this wellness program provision so employees could take advantage of opportunities to
improve their health without fear of workplace discrimination.
Under the final rules issued today, however, employers can require that employees who choose to keep their
and their spouses’ health information private pay significantly higher health insurance premiums. For plans
that cover both the employee and the spouse, the employee could be required to pay a per-person penalty of up
to 30 percent of the cost of self-only coverage. Given that the average cost of a self-only plan is $6,251 per year,
the new rules would allow an employer offering such a plan to impose an annual $3,750 penalty for an employee
and his or her spouse.
“The new EEOC rules mean that Americans could be forced to choose between access to affordable healthcare and keeping their health information private,” said Dr. Scholes. “Employers now have the green light to
coerce employees into providing their health information and that of their spouse, which in turn reveals genetic
information about their children.”
“One of the main reasons for enacting GINA was to reassure people that they could participate in medical
research without employers gaining access to their genetic information,” Dr. Scholes added. “So it is especially
unfortunate that these regulatory changes coincide with the launch of the largest-ever genetics experiment
in the United States, the Precision Medicine Initiative, which will provide participants access to their study
results. People may choose not to participate out of fear that they will be coerced into reporting these results
to their employers’ wellness programs.”
NewsRx LLC
(2016-06-04), ASHG opposes revised EEOC regulations weakening genetic privacy, Law & Health Weekly,
2, ISSN: 1551-5362, BUTTER® ID: 011804698
74
Brain Diseases
Data on Epilepsy and Genetics Reported by Researchers at G. Gaslini Institute
(Management of genetic epilepsies: From empirical treatment to precision medicine)
By a News Reporter-Staff News Editor at Health & Medicine Week – Current study results on Brain Diseases
have been published. According to news reporting from Genoa, Italy, by NewsRx journalists, research stated,
“Despite the over 20 antiepileptic drugs (AEDs) now licensed for epilepsy treatment, seizures can be effectively
controlled in about similar to 70% of patients. Thus, epilepsy treatment is still challenging in about one third
of patients and this may lead to a severe medically, physically, and socially disabling condition.”
The news correspondents obtained a quote from the research from G. Gaslini Institute, “However, there is
clear evidence of heterogeneity of response to existing AEDs and a significant unmet need for effective intervention. A number of studies have shown that polymorphisms may influence the poor or inadequate therapeutic
response as well as the occurrence of adverse effects. In addition, the new frontier of genomic technologies, including chromosome microarrays and next-generation sequencing, improved our understanding of the genetic
architecture of epilepsies. Recent findings in some genetic epilepsy syndromes provide insights into mechanisms of epileptogenesis, unrevealing the role of a number of genes with different functions, such as ion channels, proteins associated to the vesical synaptic cycle or involved in energy metabolism. The rapid progress of
high-throughput genomic sequencing and corresponding analysis tools in molecular diagnosis are revolutionizing the practice and it is a fact that for some monogenic epilepsies the molecular confirmation may influence
the choice of the treatment.”
According to the news reporters, the research concluded: “Moreover, the novel genetic methods, that are able
to analyze all known genes at a reasonable price, are of paramount importance to discover novel therapeutic
avenues and individualized (or precision) medicine.”
For more information on this research see: Management of genetic epilepsies: From empirical treatment to precision medicine. Pharmacological Research , 2016;107():426-429. Pharmacological Research
can be contacted at: Academic Press Ltd- Elsevier Science Ltd, 24-28 Oval Rd, London NW1 7DX, England. (Elsevier - www.elsevier.com; Pharmacological Research - http://www.journals.elsevier.com/
pharmacological-research/ )
Our news journalists report that additional information may be obtained by contacting P. Striano, G Gaslini
Inst Children, Dept. of Head & Neck Dis, Lab Neurosci & Neurogenet, Genoa, Italy. Additional authors for this
research include M.S. Vari, C. Mazzocchetti, A. Verrotti and F. Zara.
NewsRx LLC
(2016-07-01), Data on Epilepsy and Genetics Reported by Researchers at G. Gaslini Institute (Management
of genetic epilepsies: From empirical treatment to precision medicine), Health & Medicine Week, 1982, ISSN:
1532-4605, BUTTER® ID: 011974528
Rheumatoid Arthritis
Disjointed: Cell differences may explain why rheumatoid arthritis varies by location
By a News Reporter-Staff News Editor at Clinical Trials Week – Researchers at the University of California
San Diego School of Medicine, with colleagues in Pennsylvania and China, report that not only are there distinct differences in key cellular processes and molecular signatures between rheumatoid arthritis (RA) and
osteoarthritis (OA) but, more surprisingly, there are joint-specific differences in RA. The findings help explain,
in part, why drugs treating RA vary in effect – why, for example, a treatment that might work in arthritic knees
isn’t effective in an arthritic hip - and provide a potential new template for precisely targeting treatment for
each and every ailing joint.
The results are published in the June 10 online issue of Nature Communications and represent a collaborative project that combined the power of computational science with modern biology and a deep understanding
of the causes of arthritis.
At least 50 million adults and 300,000 children in the United States have some type of arthritis, which
includes more than 100 different diseases, according to the Centers for Disease Control. Osteoarthritis is the
most common type and involves damage to and ultimately the loss of cartilage – the cushion inside joints that
permits them to move smoothly and painlessly. Rheumatoid arthritis is the most common chronic inflammatory
arthritis and also affects joints. It can rapidly damage joints and, in the days before effective therapy, routinely
put patients in a wheelchair after a few years.
75
While OA tends to localize in weight-bearing joints where cartilage is specifically worn away, RA is distributed more symmetrically – both hands may be affected equally, for example – and often evolves from the
small joints of the hands and wrists to the larger weight-bearing joints. Why some joints are affected early,
some late and some not at all has remained unknown.
In their new study, co-corresponding authors Wei Wang, PhD, professor in the departments of Chemistry and
Biochemistry and Cellular and Molecular Medicine, and Gary S. Firestein, MD, professor in the Department
of Medicine, investigated epigenetic patterns in fibroblast-like synoviocytes (FLS) – a specialized type of cell
that lines the inside of joints.
“We hypothesized that changes in epigenetic modifications and gene expression between FLS in different
joints might potentially contribute to differences in synovial inflammation and responses to clinical treatment,”
said Wang.
The researchers discovered that DNA methylation – a fundamental, life-long process in which a methyl group
is added or removed from the cytosine molecule in DNA to promote or suppress gene activity and expression –
does in fact vary between FLS from the knees and hips of RA patients.
“We showed that the epigenetic marks vary from joint to joint in diseases like rheumatoid arthritis,” said
Firestein. “Even more importantly, the differences involved key genes and pathways that are designed to be
blocked by new RA treatments. This might provide an explanation as to why some joints improve while others
do not, even though they are exposed to the same drug.”
Firestein, who is also director of the Clinical and Translational Research Institute at UC San Diego, said
the work “opens up the potential for precision medicine approaches that allow us to target all of the joints, not
just a subset. It has broad implications for how we evaluate new drugs in clinical trials as well.”
NewsRx LLC
(2016-06-27), Disjointed: Cell differences may explain why rheumatoid arthritis varies by location, Clinical
U.S. Food and Drug Administration
FDA advances Precision Medicine Initiative by issuing draft guidances on next generation
sequencing-based tests
By a News Reporter-Staff News Editor at Pharma Business Week – In support of the President’s Precision
Medicine Initiative, the U.S. Food and Drug Administration issued two draft guidances that, when finalized,
will provide a flexible and streamlined approach to the oversight of tests that detect medically important differences in a person’s genomic makeup.
https://photos.prnewswire.com/prnvar/20151222/317925LOGO
The powerful new technology, known as next generation sequencing (NGS), can scan a person’s DNA to
detect genomic variations that may determine whether a person has or is at risk of disease or may help to
inform treatment decisions. While current regulatory approaches are appropriate for conventional diagnostics
that measure a limited number of substances associated with a disease or condition, such as blood glucose or
cholesterol levels, the new sequencing technologies can examine millions of DNA variants at a time, and thus
require a flexible approach to oversight that is adapted to the novel nature of these tests.
“Targeting the right treatments to the right patients at the right time is the goal of the President’s Precision Medicine Initiative,” said FDA Commissioner Robert Califf, M.D. “Soon, patients will have a much more
complete picture of their health than in the past, informed by their genetic and genomic makeup. The FDA is
preparing for this exciting approach at multiple levels.”
The field of genetic and genomic testing is dynamic, and the agency understands that there is a need to
encourage innovation while assuring that NGS-based tests provide accurate and useful results. When the
guidances are finalized, adherence to them will offer appropriate flexible and adaptive regulatory oversight of
these tests, while allowing for variations in development and validation and accommodating the rapid evolution
of NGS technologies.
“The FDA values the input we received from genomics experts, industry, health care providers and patients
from four public workshops and other outreach opportunities. Based on this input, we crafted draft recommendations that we believe will encourage innovation and advance the goal of precision medicine: to speed
the right individualized treatments to patients sooner,” said Jeffrey Shuren, M.D., J.D., director of the FDA’s
Center for Devices and Radiological Health. “Precision care is only as good as the tests that guide diagnosis
76
and treatment. The FDA’s job is to ensure that doctors and patients can depend upon the accuracy, reliability
and clinical validity of these tests. It’s our hope that this approach will achieve just that.”
The first draft guidance, titled “Use of Standards in FDA’s Regulatory Oversight of Next Generation Sequencing (NGS)-Based In Vitro Diagnostics (IVDs) Used for Diagnosing Germline Diseases” provides recommendations for designing, developing and validating NGS-based tests for rare hereditary diseases, and addresses the potential for using FDA-recognized standards to demonstrate analytical validity, which is how well
a test predicts the presence or absence of a particular genomic change.
The second draft guidance, titled “Use of Public Human Genetic Variant Databases to Support Clinical
Validity for Next Generation Sequencing (NGS)-Based In Vitro Diagnostics” describes an approach wherein
test developers may rely on clinical evidence from FDA-recognized public genome databases to support clinical
claims for their tests and provide assurance of accurate clinical interpretation of genomic test results - an easier
path for marketing clearance or approval.
“The draft guidances are an important step in the development of NGS-based tests,” said Francis Collins,
M.D., Ph.D., director of the National Institutes of Health (NIH). “NIH sees great value in these guidances and
encourages test developers to adopt the best practices outlined in the guidances so that high quality tests can
become available to the patients who need them.”
This adaptive approach to regulating NGS-based tests is part of the FDA’s engagement in the Precision
Medicine Initiative (PMI). The PMI, launched by the White House in early 2015, is an innovative approach to
developing a new kind of health care that takes into account individual differences in people’s genes, environments and lifestyles. The FDA’s role in the PMI is foundational: to create regulatory processes that encourage
advances in genomic testing while ensuring that NGS-based tests are safe and effective. The FDA has been
working with experts in the genomics community to conceptualize this flexible approach that strikes the important balance between safeguarding public health and promoting innovation.
The FDA encourages public comments on the draft guidances during the 90-day comment period.
NewsRx LLC
(2016-07-18), FDA advances Precision Medicine Initiative by issuing draft guidances on next generation
sequencing-based tests, Pharma Business Week, 52, ISSN: 1543-6667, BUTTER® ID: 012060878
Dementia and Mental Illness
Findings from University of California Provides New Data on Alzheimer Disease (Age,
APOE and sex: Triad of risk of Alzheimer’s disease)
By a News Reporter-Staff News Editor at Health & Medicine Week – New research on Dementia and Mental
Illness is the subject of a report. According to news reporting out of Los Angeles, California, by NewsRx editors,
research stated, “Age, apolipoprotein E epsilon 4 (APOE) and chromosomal sex are well-established risk factors
for late-onset Alzheimer’s disease (LOAD; AD). Over 60% of persons with AD harbor at least one APOE-epsilon
4 allele.”
Our news journalists obtained a quote from the research from the University of California, “The sex based
prevalence of AD is well documented with over 60% of persons with AD being female. Evidence indicates that
the APOE-epsilon 4 risk for AD is greater in women than men, which is particularly evident in heterozygous
women carrying one APOE-epsilon 4 allele. Paradoxically, men homozygous for APOE-e4 are reported to be
at greater risk for mild cognitive impairment and AD. Herein, we discuss the complex interplay between the
three greatest risk factors for Alzheimer’s disease, age, APOE-epsilon 4 genotype and chromosomal sex. We
propose that the convergence of these three risk factors, and specifically the bioenergetic aging perimenopause
to menopause transition unique to the female, creates a risk profile for AD unique to the female. Further, we
discuss the specific risk of the APOE-epsilon 4 positive male which appears to emerge early in the aging process.
Evidence for impact of the triad of AD risk factors is most evident in the temporal trajectory of AD progression
and burden of pathology in relation to APOE genotype, age and sex.”
According to the news editors, the research concluded: “Collectively, the data indicate complex interactions
between age, APOE genotype and gender that belies a one size fits all approach and argues for a precision
medicine approach that integrates across the three main risk factors for Alzheimer’s disease.”
For more information on this research see: Age, APOE and sex: Triad of risk of Alzheimer’s
disease.
Journal of Steroid Biochemistry and Molecular Biology , 2016;160():134-147.
Journal of
Steroid Biochemistry and Molecular Biology can be contacted at: Pergamon-Elsevier Science Ltd, The
Boulevard, Langford Lane, Kidlington, Oxford OX5 1GB, England.
(Elsevier - www.elsevier.com;
77
Journal of Steroid Biochemistry and Molecular Biology - http://www.journals.elsevier.com/
journal-of-steroid-biochemistry-and-molecular-biology/ )
Our news journalists report that additional information may be obtained by contacting B.C. Riedel, University of California, Keck Sch Med, Dept. of Neurol, Dept. of Pharmacol & Pharmaceut SciSch Pharm, Los
Angeles, CA 90089, United States. Additional authors for this research include P.M. Thompson and R.D. Brinton.
NewsRx LLC
(2016-07-01), Findings from University of California Provides New Data on Alzheimer Disease (Age, APOE
and sex: Triad of risk of Alzheimer’s disease), Health & Medicine Week, 594, ISSN: 1532-4605, BUTTER® ID:
011974041
Eye Diseases and Conditions
Findings on Open-Angle Glaucoma and Genetics Reported by Investigators at University of
Gaziantep [Toward Novel Diagnostics for Primary Open-Angle Glaucoma? An Association
Study of Polymorphic Variation in Ras Homolog Family Member (A, B, C, D) ...]
By a News Reporter-Staff News Editor at Health & Medicine Week – Current study results on Eye Diseases
and Conditions have been published. According to news reporting originating from Gaziantep, Turkey, by
NewsRx correspondents, research stated, “The annual economic burden of visual disorders in the United States
was estimated as $139 billion. The World Health Organization has listed glaucoma in the top 10 priority eye
diseases.”
Our news editors obtained a quote from the research from the University of Gaziantep, “Primary open-angle
glaucoma (POAG) is a common subtype, with a lack of clinical tools for early diagnosis. The Rho GTPases belong
to the Ras superfamily of proteins; the RhoA immunostaining in the optic nerve head in human glaucoma is
reportedly increased. We investigated the association of polymorphisms in the Ras Homolog Family Member A,
B, C, and D genes (RHOA, RHOB, RHOC, and RHOD, respectively). In a total sample of 361 unrelated subjects
(179 patients with POAG and 182 age- and sex-matched healthy controls), RHOA (rs6784820, rs974495), RHOB
(rs62121967), RHOC (rs11102522), and RHOD (rs61891303, rs2282502) polymorphisms were characterized by
the BioMark HD dynamic array system with real-time polymerarse chain reaction. Among these candidate
genetic markers and considering the Bonferroni correction, RHOA rs974495 polymorphism was significantly
associated with POAG (p=0.0011), with the TT genotype increasing the disease risk 4.9 times (95% CI 1.63015.023). The allele and haplotype distributions of the above RHO candidate polymorphisms did not diplay a
significant association. This is the first study, to the best of our knowledge, to identify a significant genotypic
association between POAG and RHOA gene rs974495 polymorphism.”
According to the news editors, the research concluded: “These observations warrant replication in independent samples in the pursuit of precision medicine for rapid and early glaucoma diagnosis, and molecular targets
for innovation in therapeutics of this common eye disease.”
For more information on this research see: Toward Novel Diagnostics for Primary Open-Angle Glaucoma?
An Association Study of Polymorphic Variation in Ras Homolog Family Member (A, B, C, D) Genes RHOA,
RHOB, RHOC, and RHOD. Omics-A Journal of Integrative Biology , 2016;20(5):290-295. Omics-A Journal of
Integrative Biology can be contacted at: Mary Ann Liebert, Inc, 140 Huguenot Street, 3RD Fl, New Rochelle,
NY 10801, USA.
The news editors report that additional information may be obtained by contacting A. Saracaloglu,
Gaziantep Univ, Dept. of Med Biol, Fac Med, Gaziantep, Turkey. Additional authors for this research include
S. Demiryurek, S. Okumus, S. Oztuzcu, I. Bozgeyik, E. Coskun, U. Aksoy, E. Kaydu, I. Erbagci, B. Gurler, B.
Alasehirli and A.T. Demiryurek.
NewsRx LLC
(2016-06-24), Findings on Open-Angle Glaucoma and Genetics Reported by Investigators at University of
Gaziantep [Toward Novel Diagnostics for Primary Open-Angle Glaucoma? An Association Study of Polymorphic
Variation in Ras Homolog Family Member (A, B, C, D) ...], Health & Medicine Week, 2562, ISSN: 1532-4605,
BUTTER® ID: 011936588
78
Stem Cell Research
Findings on Stem Cell Research Detailed by Investigators at University of Manchester (Dual
targeting of p53 and c-MYC selectively eliminates leukaemic stem cells)
By a News Reporter-Staff News Editor at Genomics & Genetics Weekly – Investigators publish new report on
Stem Cell Research. According to news originating from Manchester, United Kingdom, by NewsRx correspondents, research stated, “Chronic myeloid leukaemia (CML) arises after transformation of a haemopoietic stem
cell (HSC) by the protein-tyrosine kinase BCR-ABL. Direct inhibition of BCR-ABL kinase has revolutionized
disease management, but fails to eradicate leukaemic stem cells (LSCs), which maintain CML.”
Our news journalists obtained a quote from the research from the University of Manchester, “LSCs are
independent of BCR-ABL for survival, providing a rationale for identifying and targeting kinase-independent
pathways. Here we show-using proteomics, transcriptomics and network analyses-that in human LSCs, aberrantly expressed proteins, in both imatinib-responder and non-responder patients, are modulated in concert
with p53 (also known as TP53) and c-MYC regulation. Perturbation of both p53 and c-MYC, and not BCR-ABL
itself, leads to synergistic cell kill, differentiation, and near elimination of transplantable human LSCs in mice,
while sparing normal HSCs.”
According to the news editors, the research concluded: “This unbiased systems approach targeting connected
nodes exemplifies a novel precision medicine strategy providing evidence that LSCs can be eradicated.”
For more information on this research see: Dual targeting of p53 and c-MYC selectively eliminates
leukaemic stem cells. Nature , 2016;534(7607):341-346,79-90. Nature can be contacted at: Nature Publishing Group, Macmillan Building, 4 Crinan St, London N1 9XW, England. (Nature Publishing Group http://www.nature.com/ ; Nature - http://www.nature.com/nature/ )
The news correspondents report that additional information may be obtained from S.A. Abraham, University
of Manchester, Stoller Biomarker Discovery Center, Manchester M20 3LJ, Lancs, United Kingdom. Additional
authors for this research include L.E.M. Hopcroft, E. Carrick, M.E. Drotar, K. Dunn, A.J.K. Williamson, K.
Korfi, P. Baquero, L.E. Park, M.T. Scott, F. Pellicano, A. Pierce, M. Copland, C. Nourse, S.M. Grimmond, D.
Vetrie, A.D. Whetton and T.L. Holyoake.
NewsRx LLC
(2016-07-15), Findings on Stem Cell Research Detailed by Investigators at University of Manchester (Dual
targeting of p53 and c-MYC selectively eliminates leukaemic stem cells), Genomics & Genetics Weekly, 72, ISSN:
1532-4591, BUTTER® ID: 012048178
DNA Research
How will genomics enter day-to-day medicine?
By a News Reporter-Staff News Editor at Life Science Weekly – A quiet transformation has been brewing in
medicine, as large-scale DNA results become increasingly available to patients and healthcare providers. Amid
a cascade of data, physicians, counselors and families are sorting out how to better understand and use this
information in making health care decisions.
National experts who have gathered in Clinical Genetics Think Tank meetings at two large pediatric hospitals recently issued their first recommendations for integrating genomics into clinical practice.
The recommendations appeared online May 12, 2016 in Genetics in Medicine, co-led by Ian D. Krantz, M.D.,
director of the Individualized Medical Genetics Center at The Children’s Hospital of Philadelphia (CHOP), and
Ronald D. Cohn, M.D., co-director of the Centre for Genetic Medicine at the Hospital for Sick Kids in Toronto.
“As genetic testing has become more complex, it’s being applied across many more medical specialties and
into primary care,” said Krantz, a clinical geneticist. “These tests will move toward broad use in screening
healthy populations, and our recommendations aim to help people better integrate testing results into clinical
practice.”
Krantz and co-author Sarah Bowdin, M.D., of the Centre for Genetic Medicine at the Hospital for Sick Kids
in Toronto, spearheaded the two Clinical Genetics Think Tanks, hosted at their respective hospitals in 2014
and 2015.
Co-authors of the recommendations are other Think Tank participants: clinical geneticists, genetic counselors, and laboratory professionals and bioinformatics experts. “Our co-authors represent the main stakeholders in this field,” said Krantz. “We also included patients and parents in the Think Tanks, to incorporate their
experiences in dealing with these concerns on an everyday basis.”
79
Krantz added that he and Bowdin launched the Think Tanks after hearing from colleagues struggling with
many similar issues as other institutions established clinical genomic and exome sequencing programs. Among
those challenges were how to best interpret DNA findings, how to report to patients and clinicians about gene
variants of uncertain significance, how to report secondary findings unrelated to the primary reason for the
testing, and how to share findings with other centers. “As each institution independently developed its own
procedures, we thought that exchanging experiences across our field could improve overall practice.”
The recommendations address the pretesting process (including selecting patients and obtaining insurance
coverage), patient and clinician education, interpreting sequence data, and posttest patient care (including
how to return test findings and offer reevaluation of data). Another broad area, added Krantz, is phenotyping–
establishing consistent terminology for patients’ clinical characteristics, so that clinicians can better interpret
the significance of DNA results, share data across centers, and ultimately standardize care for patients.
Krantz compared these new challenges to a more straightforward clinical situation–obtaining a targeted
genetic test for fragile X syndrome–in which a test reveals whether a patient has a specific DNA change that
causes fragile X symptoms. In contrast, current clinical genome and exome sequencing produces many unknowns: for each individual, test results yield many variants of uncertain significance, as well as secondary
findings, which are genetic variants unrelated to the primary condition for which a patient is tested.
Facing a flood of DNA data, families told other Think Tank participants that they often preferred two posttest
sessions to discuss test findings–one to learn the principal diagnostic results, and a second session to discuss
secondary findings that are medically actionable.
Crucially, Krantz added, the data from genomic testing are dynamic–as new scientific knowledge accumulates, the significance of data changes: some findings of uncertain significance will become clearer, and will
become medically actionable in the future, so that healthcare providers will need to devise ways to systematically offer future reevaluation of a patient’s genome. “We need to make these data longitudinal, not static,” he
said.
One emerging issue raised in the Think Tanks is how to best integrate genomic results into each patient’s
electronic health record. This becomes all the more important, said Krantz, as clinical sequencing moves toward general screening of healthy patient populations, including newborns, as part of the progression toward
precision medicine.
One conversation with a family, added Krantz, helped to drive home that issue. He was explaining results of
genomic testing in a child with multiple medical issues. After learning the unexpected secondary finding that
their child carried a cancer predisposition gene, the parents asked about performing the test for their healthy
child too.
“We have framed this document not as a set of overt guidelines, but as recommendations, which we expect
to change as our field evolves,” said Krantz. He added that future Think Tanks may meet to address new
challenges.
NewsRx LLC
(2016-07-19), How will genomics enter day-to-day medicine?, Life Science Weekly, 321, ISSN: 1552-2474,
BUTTER® ID: 012067125
Eye Diseases and Conditions
Investigators from Radboud University Release New Data on Age-Related Macular
Degeneration (Omics in Ophthalmology: Advances in Genomics and Precision Medicine for
Leber Congenital Amaurosis and Age-Related Macular Degeneration)
By a News Reporter-Staff News Editor at Clinical Trials Week – Investigators publish new report on Eye Diseases and Conditions. According to news originating from Nijmegen, Netherlands, by NewsRx editors, the
research stated, “The genomic revolution has had a huge impact on our understanding of the genetic defects
and disease mechanisms underlying ophthalmic diseases. Two examples are discussed here.”
Our news journalists obtained a quote from the research from Radboud University, “The first is Leber congenital amaurosis (LCA), a severe inherited retinal dystrophy leading to severe vision loss in children, and
the second is age-related macular degeneration (AMD), the most common cause of vision loss in the elderly.
Twenty years ago, the genetic causes of these diseases were unknown. Currently, more than 20 LCA genes
have been identified, and genetic testing can now successfully identify the genetic defects in at least 75% of all
LCA cases. Gene-specific treatments have entered the clinical trial phase for three LCA genes, and for seven
LCA genes gene-specific therapies have been tested in model systems. Age-related macular degeneration is a
80
multifactorial disease caused by a combination of genetic and environmental factors. Currently, more than 40
loci have been identified for AMD, accounting for 15%-65% of the total genetic contribution to AMD. Despite
the progress that has been made so far, genetic testing is not yet recommended for AMD, but this may change
if we move to clinical trials or treatments that are dependent on an individual’s genotype. The identification
of serum or plasma biomarkers using other ‘-omics” technologies may further improve predictive tests and our
understanding of the disease mechanisms of AMD.”
According to the news editors, the research concluded: “Ultimately, it is anticipated that predictive tests will
help to stratify patients for the most suitable therapy, which will enable the development of precision medicine,
tailored to individual needs.”
For more information on this research see: Omics in Ophthalmology: Advances in Genomics and Precision
Medicine for Leber Congenital Amaurosis and Age-Related Macular Degeneration. Investigative Ophthalmology & Visual Science , 2016;57(3):1378-1387. Investigative Ophthalmology & Visual Science can be contacted
at: Assoc Research Vision Ophthalmology Inc, 12300 Twinbrook Parkway, Rockville, MD 20852-1606, USA.
(The Association for Research in Vision and Ophthalmology - www.arvo.org; Investigative Ophthalmology &
Visual Science - http://www.iovs.org/ )
The news correspondents report that additional information may be obtained from A.I. den Hollander, Radboud University, Medical Center, Dept. of Human Genet, Nijmegen, Netherlands.
NewsRx LLC
(2016-06-13), Investigators from Radboud University Release New Data on Age-Related Macular Degeneration (Omics in Ophthalmology: Advances in Genomics and Precision Medicine for Leber Congenital Amaurosis and Age-Related Macular Degeneration), Clinical Trials Week, 350, ISSN: 1543-6764, BUTTER® ID:
011823446
Macrogen
Macrogen Launches famplan™ Genetic Carrier Testing, and ABOOBA™ Genetic Test for
Newborns
By a News Reporter-Staff News Editor at Pediatrics Week – Macrogen (CEO Hyon-yong Chong,
www.macrogen.com) (KOSDAQ:038290), a biotechnology company in the area of precision medicine, announced
its official worldwide launch of famplan™, a genetic carrier test, and ABOOBA™, a genetic test for newborns,
in celebration of May 15, the International Day of Families.
famplan™ is an abbreviation for “family plan,” and it serves to identify potential hereditary diseases in
newborns by analyzing their parents’ genetic makeup and identifying genetic disease carriers. As the world’s
most extensive carrier test, famplan™ looks for more than 2,000 hereditary disease-related risks and aids in the
prevention of congenital hereditary problems, helping mothers to give birth to healthy children. In particular,
it is appropriate for future parents who might be worried about the cost, as it is in the most affordable price
range among the established tests.
ABOOBA™ is an abbreviation for “about the baby.” It is a service that analyzes a newborn’s genetic makeup
in order to check for congenital hereditary diseases. Because the ABOOBA™ test uses the newest available
genetic analysis technology, it can offer a risk analysis of all the newborn-related hereditary diseases known
today. Fifty percent of hereditary diseases are found within a year of a child’s birth, but if found early with
ABOOBA™, diseases can be prevented with appropriate intensive care, the time of onset can be delayed, and
even after occurrence, symptoms can be alleviated. Also, the customer can continually receive updated information if a new hereditary disease is found even after running just a single test.
Customers all over the world can request famplan™ and ABOOBA™ tests and their results through hospitals, and the results are out within two to three weeks from the day of reception. In particular, famplan™ offers
a convenient online service at its official websites, famplan.macrogen.com and clinic.macrogen.com. Macrogen
can now offer a total care solution in managing hereditary diseases and their risks from before conception to
right after birth with its launch last November of faest™-an advanced test to analyze genetic makeup before
childbirth-and current launch of famplan™ and ABOOBA™. Future parents who are expecting a child can
start family planning by first identifying the child’s hereditary disease risks with famplan™, and at an early
stage of pregnancy the child’s chromosomes can be checked for abnormality through faest™. Furthermore,
ABOOBA™ can be used after birth to identify the newborn’s risk of getting a hereditary disease, thus offering
81
customers a comprehensive and systematic testing service. In particular, customers can use Macrogen’s package service for testing, thus minimizing the financial burden while still taking advantage of a more systematic
and planned testing service.
CEO Hyon-yong Chong said, “Macrogen was able to offer a total care solution before childbirth through
continuous research and development in this area after the launch of faest™ as well as the accumulated knowhow after operating a genetic makeup analysis service for the past 20 years.” He added, “We will continue to
do our best to give a trustworthy service to parents who want a healthy child so that they can plan for a happy
and healthy family, and manage their family’s health systematically.”
Since last year, Macrogen has been expanding its clinical and general customers with the successive
launches of the advanced genetic makeup testing service faest™, the genetic test for pets MyPETGENE™ and
the genetic identification service iD4U™ in the global market. Macrogen plans to continue to offer diversified
services to the general public so that they can use information on genetic makeup in everyday life. View source
NewsRx LLC
(2016-06-04), Macrogen Launches famplan™ Genetic Carrier Testing, and ABOOBA™ Genetic Test for
Newborns, Pediatrics Week, 90, ISSN: 1944-2645, BUTTER® ID: 011807321
Duke University
Moving beyond race-based drugs
By a News Reporter-Staff News Editor at Physician Law Weekly – DURHAM, N.C. – Prescribing certain medications on the basis of a patient’s race has long come under fire from those uneasy with using race as a surrogate
for biology when treating disease.
But there are multiple challenges to overcome before we can move beyond race-based treatment decisions,
writes Duke University geneticist and bioethicist Charmaine Royal in a perspective piece published May 25 in
the New England Journal of Medicine.
In “Will Precision Medicine Move Us beyond Race?” Royal and colleagues Vence Bonham of the National
Institutes of Health and Shawneequa Callier of The George Washington University describe some of the thorny
issues raised by race-based drugs.
When the first race-based drug, BiDil, was approved by the FDA a decade ago to treat African Americans
with heart failure, advocates heralded it as a way to narrow health disparities between whites and blacks by
targeting the group that suffered the most from the disease.
Yet the drug had not actually been shown to be more effective in African Americans than other patients,
critics noted. What’s more, it was only marketed to African Americans after efforts to obtain FDA and patent
approval for use in the general population failed, leading many to question the commercial motivations for
classifying it as an “ethnic” drug.
Race is also a common factor in prescribing ACE inhibitors, a class of drugs used to treat high blood pressure
and heart disease. ACE inhibitors have been shown to be less effective in blacks than whites, “but one result
of using race to dictate therapy is that individual black patients whose hypertension would respond to ACE
inhibitors may not be offered one,” the authors say.
“What we even mean by race has always been murky and is becoming even more so given changing demographics,” said Royal, an associate professor of African and African American Studies and director of the
Center on Genomics, Race, Identity, Difference at Duke’s Social Science Research Institute. Someone who
self-identifies as black, such as President Obama, likely has ancestors from multiple so-called races.
Given the genetic diversity that exists within racial groups, and the similarities between different groups,
it is likely that a drug labeled for use in African Americans will not work for all African Americans, and that
some non-African Americans would also benefit from the drug.
But getting drug manufacturers and clinicians to go beyond a race-based approach won’t be easy, the authors
say.
The makers of the clot-inhibiting drug Plavix, for example, were sued by the Attorney General of Hawaii in
2014 for failing to disclose that the drug was less effective in patients with certain inherited forms of a liver
enzyme called CYP2C19.
Sharing such information with the public likely would have decreased sales in the state, as the variants
are more common in Asians, Native Hawaiians and other Pacific Islanders – who make up more than half of
Hawaii’s population – than in whites.
82
Technological advances in DNA sequencing and analyzing large datasets will continue to generate insights
about the genetics underlying differences in drug response. The data deluge will only further highlight the
pitfalls of using imprecise race categories to prescribe drugs.
“Prescribing medications on the basis of race oversimplifies the complexities and interplay of ancestry,
health, disease, and drug response,” the authors write.
Eventually, optimizing drug treatments to a patient’s unique genetic makeup, lifestyle, environment and
other factors, rather than race, could help ensure that patients receive the right drug at the right dose – an
approach called precision medicine.
“There are many hurdles to overcome if a precision medicine approach to health care is to replace the use
of race in treatment decisions,” the authors say.
One barrier to understanding the complex interplay between genes, environment and lifestyle is the lack of
participant diversity in biomedical research and clinical trials. Addressing the problem will require recruiting
more participants from minority groups to better reflect the diversity of the U.S. population.
We will also need cost controls for drugs found to be effective only in a few, the authors say.
Lastly, moving beyond race-based drug prescriptions will depend on the ability to equip health care providers
with the resources and training they will need to collect and make sense of more types of data.
“Precision medicine is premised on the idea of improving health outcomes by generating and using many
sources of personal data to more accurately group and treat patients,” the authors say. “If the major challenges
can be overcome, precision medicine could lead the way in reducing and ultimately eliminating the use of crude
racial and ethnic census categories in drug prescribing.”
NewsRx LLC
(2016-06-15), Moving beyond race-based drugs, Physician Law Weekly, 2, ISSN: 1551-5303, BUTTER® ID:
011842918
Addiction Research
New Alcoholism Findings from Ministry of Public Health Reported (From genetic studies to
precision medicine in alcohol dependence)
By a News Reporter-Staff News Editor at Psychology & Psychiatry Journal – New research on Addiction Research is the subject of a report. According to news originating from Beijing, People’s Republic of China, by
VerticalNews correspondents, research stated, “Genetic factors contribute to more than 50% of the variation in
the vulnerability to alcohol dependence (AD). Although significant advances have been made in medications
for AD, these medications do not work for all people.”
Our news journalists obtained a quote from the research from the Ministry of Public Health, “Precise tailoring of medicinal strategies for individual alcoholic patients is needed to achieve optimal outcomes. This
review updates the most promising information on genetic variants in AD, which may be useful for improving
diagnostic, therapeutic, and monitoring strategies. We describe genetic candidates of various neurotransmitter
and enzyme systems. In addition to biological and allelic associations with AD, genetic effects on AD-related
phenotypes and treatment responses have also been described. Gene-gene and gene-environment interactions
have been considered. Potential applications of genomewide and epigenetic approaches for identifying genetic
biomarkers of AD have been discussed. Overall, the application of genetic findings in precision medicine for AD
will likely involve an integrated approach that distinguishes effect sizes of specific genetic predictors with regard
to sex, pharmacotherapy, ethnicity, and AD-related aspects and considers gene-gene and gene-environment interactions.”
According to the news editors, the research concluded: “Our work may pave the way toward more precise
treatment for AD that could ultimately improve clinical management and interventions.”
For more information on this research see: From genetic studies to precision medicine in alcohol dependence. Behavioural Pharmacology , 2016;27(2-3):87-99. Behavioural Pharmacology can be contacted at:
Lippincott Williams & Wilkins, Two Commerce Sq, 2001 Market St, Philadelphia, PA 19103, USA. (Lippincott Williams and Wilkins - www.lww.com; Behavioural Pharmacology - http://journals.lww.com/
behaviouralpharm/pages/default.aspx )
The news correspondents report that additional information may be obtained from Y. Sun, Minist Public
Hlth, Beijing, People’s Republic of China. Additional authors for this research include Y. Zhang, F. Wang, Y.K.
Sun, J. Shi and L. Lu.
83
NewsRx LLC
(2016-06-04), New Alcoholism Findings from Ministry of Public Health Reported (From genetic studies to
precision medicine in alcohol dependence), Psychology & Psychiatry Journal, 365, ISSN: 1944-2726, BUTTER®
ID: 011808057
DNA Research
New DNA Research Findings from Beijing Institute of Genomics Reported (Epigenetic
Pattern on the Human Y Chromosome Is Evolutionarily Conserved)
By a News Reporter-Staff News Editor at Life Science Weekly – Investigators publish new report on DNA
Research. According to news reporting out of Beijing, People’s Republic of China, by NewsRx editors, research
stated, “DNA methylation plays an important role for mammalian development. However, it is unclear whether
the DNA methylation pattern is evolutionarily conserved.”
Our news journalists obtained a quote from the research from the Beijing Institute of Genomics, “The Y chromosome serves as a powerful tool for the study of human evolution because it is transferred between males. In
this study, based on deep-rooted pedigrees and the latest Y chromosome phylogenetic tree, we performed epigenetic pattern analysis of the Y chromosome from 72 donors. By comparing their respective DNA methylation
level, we found that the DNA methylation pattern on the Y chromosome was stable among family members and
haplogroups. Interestingly, two haplogroup-specific methylation sites were found, which were both genotypedependent. Moreover, the African and Asian samples also had similar DNA methylation pattern with a remote
divergence time.”
According to the news editors, the research concluded: “Our findings indicated that the DNA methylation
pattern on the Y chromosome was conservative during human male history.”
For more information on this research see: Epigenetic Pattern on the Human Y Chromosome Is Evolutionarily Conserved. Plos One , 2016;11(1):e0146402. (Public Library of Science - www.plos.org; Plos One www.plosone.org)
Our news journalists report that additional information may be obtained by contacting M. Zhang, Key Laboratory of Genomic and Precision Medicine, China Gastrointestinal Cancer Research Center, Beijing Institute
of Genomics, Chinese Academy of Sciences, Beijing 100101, People’s Republic of China. Additional authors for
this research include C.C. Wang, C. Yang, H. Meng, I.O. Agbagwa, L.X. Wang, Y. Wang, S. Yan, S. Ren, Y. Sun,
G. Pei, X. Liu, J. Liu, L. Jin, H. Li and Y. Sun.
NewsRx LLC
(2016-06-07), New DNA Research Findings from Beijing Institute of Genomics Reported (Epigenetic Pattern on the Human Y Chromosome Is Evolutionarily Conserved), Life Science Weekly, 522, ISSN: 1552-2474,
BUTTER® ID: 011875432
Life Science Research
New Findings on Life Science Research from Duke University School of Medicine
Summarized (Individual Differences in Scotopic Visual Acuity and Contrast Sensitivity:
Genetic and Non-Genetic Influences)
By a News Reporter-Staff News Editor at Life Science Weekly – Investigators discuss new findings in Life
Science Research. According to news reporting out of Durham, North Carolina, by NewsRx editors, research
stated, “Despite the large amount of variation found in the night (scotopic) vision capabilities of healthy volunteers, little effort has been made to characterize this variation and factors, genetic and non-genetic, that
influence it. In the largest population of healthy observers measured for scotopic visual acuity (VA) and contrast sensitivity (CS) to date, we quantified the effect of a range of variables on visual performance.”
Our news journalists obtained a quote from the research from the Duke University School of Medicine,
“We found that young volunteers with excellent photopic vision exhibit great variation in their scotopic VA
and CS, and this variation is reliable from one testing session to the next. We additionally identified that
factors such as Circadian preference, iris color, astigmatism, depression, sex and education have no significant
impact on scotopic visual function. We confirmed previous work showing that the amount of time spent on the
vision test influences performance and that laser eye surgery results in worse scotopic vision. We also showed
84
a significant effect of intelligence and photopic visual performance on scotopic VA and CS, but all of these
variables collectively explain <30% of the variation in scotopic vision. The wide variation seen in young healthy
volunteers with excellent photopic vision, the high test-retest agreement, and the vast majority of the variation
in scotopic vision remaining unexplained by obvious non-genetic factors suggests a strong genetic component.”
According to the news editors, the research concluded: “Our preliminary genome-wide association study
(GWAS) of 106 participants ruled out any common genetic variants of very large effect and paves the way for
future, larger genetic studies of scotopic vision.”
For more information on this research see: Individual Differences in Scotopic Visual Acuity and Contrast
Sensitivity: Genetic and Non-Genetic Influences. Plos One , 2016;11(2):e0148192. (Public Library of Science www.plos.org; Plos One - www.plosone.org)
Our news journalists report that additional information may be obtained by contacting A.J. Bartholomew,
Center for Applied Genomics and Precision Medicine, Duke University School of Medicine, Durham, North
Carolina 27708, United States. Additional authors for this research include E.M. Lad, D. Cao, M. Bach and
E.T Cirulli.
NewsRx LLC
(2016-07-19), New Findings on Life Science Research from Duke University School of Medicine Summarized (Individual Differences in Scotopic Visual Acuity and Contrast Sensitivity: Genetic and Non-Genetic
Influences), Life Science Weekly, 2104, ISSN: 1552-2474, BUTTER® ID: 012068686
Genetics
New Genetic Research Study Findings Have Been Reported by Researchers at Southeast
University (Enhancer, epigenetics, and human disease)
By a News Reporter-Staff News Editor at Genomics & Genetics Weekly – Research findings on Genetics are
discussed in a new report. According to news reporting originating in Jiangsu, People’s Republic of China, by
NewsRx journalists, research stated, “Enhancers encode a huge body of information to determine the precise
tissue specific gene expression pattern during normal development.”
The news reporters obtained a quote from the research from Southeast University, “Nowadays, enhancers
are also considered as key players in directing disease transcriptional program during pathogenesis. New
genomic technologies allow the identification, functional characterization and manipulation of enhancers.”
According to the news reporters, the research concluded: “The advances in the transcriptional enhancer
field hold great promise in linking developmental or disease phenotypes to genetic variants and promoting
For more information on this research see: Enhancer, epigenetics, and human disease. Current
Opinion in Genetics & Development , 2016;36():27-33. Current Opinion in Genetics & Development can
be contacted at: Current Biology Ltd, 84 Theobalds Rd, London WC1X 8RR, England. (Elsevier www.elsevier.com; Current Opinion in Genetics & Development - http://www.journals.elsevier.com/
current-opinion-in-genetics-and-development/ )
Our news correspondents report that additional information may be obtained by contacting Z.J. Luo, Southeast Univ, Inst Life Sci, Key Lab Dev Genes & Human Dis, Nanjing 210096, Jiangsu, People’s Republic of
China.
NewsRx LLC
(2016-06-17), New Genetic Research Study Findings Have Been Reported by Researchers at Southeast University (Enhancer, epigenetics, and human disease), Genomics & Genetics Weekly, 145, ISSN: 1532-4591, BUTTER® ID: 011850330
85
Arthritis
New Psoriatic Arthritis and Genetics Study Findings Have Been Reported by Investigators
at Santa Lucia Foundation (Pharmacogenomics of multifactorial diseases: a focus on
psoriatic arthritis)
By a News Reporter-Staff News Editor at Health & Medicine Week – New research on Arthritis is the subject
of a report. According to news originating from Rome, Italy, by NewsRx correspondents, research stated, “This
review will outline the current pharmacogenomics knowledge about psoriatic arthritis with a special attention
to the perspectives and the challenges for its implementation in the clinical practice. To date, different drugs
have been developed to contrast the symptoms and the progression of psoriatic arthritis.”
Our news journalists obtained a quote from the research from Santa Lucia Foundation, “However, patients
have shown high variability of drug response in relation to their genetic makeup. In this context, the advances
made in the knowledge and the potentialities of genome-drugs associations paved the path for the development
of a precision medicine.”
According to the news editors, the research concluded: “In fact, these associations may be successfully
combined with the environment information to provide new strategies able to prevent and improve the disease
management as well as to enhance the patients quality of life.”
For more information on this research see: Pharmacogenomics of multifactorial diseases: a focus on psoriatic
arthritis. Pharmacogenomics , 2016;17(8):943-951. Pharmacogenomics can be contacted at: Future Medicine
Ltd, Unitec House, 3RD Floor, 2 Albert Place, Finchley Central, London, N3 1QB, England. (Nature Publishing
Group - http://www.nature.com/ ; Pharmacogenomics - http://www.nature.com/tpj/ )
The news correspondents report that additional information may be obtained from R. Cascella, Santa Lucia
Fdn, Mol Genet Lab UILDM, Via Ardeatina 306, I-00146 Rome, Italy. Additional authors for this research
include C. Strafella, G. Longo, M. Maccarone, P. Borgiani, F. Sangiuolo, G. Novelli and E. Giardina.
NewsRx LLC
(2016-07-22), New Psoriatic Arthritis and Genetics Study Findings Have Been Reported by Investigators at
Santa Lucia Foundation (Pharmacogenomics of multifactorial diseases: a focus on psoriatic arthritis), Health
Lung Diseases and Conditions
New Pulmonary Fibrosis and Genetics Findings from School of Medicine Described
(Precision Medicine: The New Frontier in Idiopathic Pulmonary Fibrosis)
By a News Reporter-Staff News Editor at Respiratory Therapeutics Week – Data detailed on Lung Diseases
and Conditions have been presented. According to news reporting out of New York City, New York, by NewsRx
editors, research stated, “Precision medicine is defined by the National Institute of Health’s Precision Medicine
Initiative Working Group as an approach to disease treatment that takes into account individual variability in
genes, environment, and lifestyle.”
Our news journalists obtained a quote from the research from the School of Medicine, “There has been
increased interest in applying the concept of precision medicine to idiopathic pulmonary fibrosis, in particular
to search for genetic and molecular biomarker-based profiles (so called endotypes) that identify mechanistically
distinct disease subgroups. The relevance of precision medicine to idiopathic pulmonary fibrosis is yet to be
established, but we believe that it holds great promise to provide targeted and highly effective therapies to
patients.”
According to the news editors, the research concluded: “In this manuscript, we describe the field’s nascent
efforts in genetic/molecular endotype identification and how environmental and behavioral subgroups may also
be relevant to disease management.”
For more information on this research see: Precision Medicine: The New Frontier in Idiopathic Pulmonary
Fibrosis. American Journal of Respiratory and Critical Care Medicine , 2016;193(11):1213-1218. American
Journal of Respiratory and Critical Care Medicine can be contacted at: Amer Thoracic Soc, 25 Broadway, 18
Fl, New York, NY 10004, USA.
Our news journalists report that additional information may be obtained by contacting R. Brownell, Weill
Cornell Univ, Sch Med, Dept. of Med, New York, NY, United States. Additional authors for this research include
N. Kaminski, P.G. Woodruff, W.Z. Bradford, L. Richeldi, F.J. Martinez and H.R. Collard.
86
NewsRx LLC
(2016-06-27), New Pulmonary Fibrosis and Genetics Findings from School of Medicine Described (Precision
Medicine: The New Frontier in Idiopathic Pulmonary Fibrosis), Respiratory Therapeutics Week, 16, ISSN:
1543-6664, BUTTER® ID: 011952205
LifeNome Inc.
Personal Genomics Revolution comes to Southeast Asia
By a News Reporter-Staff News Editor at Journal of Engineering – Singapore-based Imagene Labs Pte. Ltd.,
a subsidiary of Asia Genomics Pte. Ltd., and New York City-based LifeNome Inc. announced a major international partnership to bring genomic personalization of wellness and lifestyle choices to Southeast Asia.
The partnership will leverage on the state of the art genetics testing facilities of Imagene Labs together
with the proprietary bioinformatics platform of LifeNome to provide health-conscious consumers in the region
with personalized genomics guidance on nutrition, fitness, skin care and other wellness topics. Starting July
2016, Imagene will offer genetic testing along with trait reports to individual consumers as well as physicians,
nutritionists, and fitness trainers in Singapore, Vietnam, the Philippines, Malaysia, Thailand and Indonesia.
“There is no doubt we are entering the age of genetically informed personalization: from precision medicine
to personalized well-being advice,” says Raya Khanin, Ph.D., Chief Scientific Officer of LifeNome. “Our platform leverages information on thousands of genetic variations using rigorous analysis and delivers actionable
insights to the individual. What sets our technology apart is a careful curation of scientific studies and a computational methodology that assesses the cumulative predisposition likelihood when multiple genetic variations
influence a particular well-being trait predisposition such as vitamin D deficiency.”
The nutrition reports will assess potential predispositions and provide guidance on traits such as vitamin
deficiency, predisposition to weight gain, protein processing, lactose intolerance, and gluten sensitivity. Also
offered by Imagene will be information on fitness traits such as endurance, suitability for power exercise and
muscle injury risk, as well as skin care characteristics such as aging, youthfulness, and sunburn potential.
Imagene Labs Managing Director Dr Mun-Yew Wong shares, “We look forward to bringing the computational
technologies of LifeNome and our state-of-the-art genetic testing, interactive consumer engagement apps and
expert services to Southeast Asia. Genetics-based personalization will be the cornerstone of wellness for the
future and we are proud to be a pioneer in the region.”
Brian Young, CEO of LifeNome, shares, “We are thrilled to partner with Imagene Labs as we begin the
global roll out of the industry’s most comprehensive consumer genomic traits platform. Together our state of
the art technology and scientific expertise will provide consumers with the best insights that genomics science
can currently offer.”
NewsRx LLC
(2016-06-13), Personal Genomics Revolution comes to Southeast Asia, Journal of Engineering, 884, ISSN:
1945-872X, BUTTER® ID: 011828962
Nervous System Diseases and Conditions
Reports from Dana-Farber Cancer Institute Add New Data to Findings in Meningioma and
Genetics (Oncogenic PI3K mutations are as common as AKT1 and SMO mutations in
meningioma)
By a News Reporter-Staff News Editor at Health & Medicine Week – New research on Nervous System Diseases
and Conditions is the subject of a report. According to news reporting originating in Boston, Massachusetts, by
NewsRx journalists, research stated, “Meningiomas are the most common primary intracranial tumor in adults.
Identification of SMO and AKT1 mutations in meningiomas has raised the possibility of targeted therapies for
some patients.”
The news reporters obtained a quote from the research from Dana-Farber Cancer Institute, “The frequency
of such mutations in clinical cohorts and the presence of other actionable mutations in meningiomas are important to define. We used high-resolution array-comparative genomic hybridization to prospectively characterize
copy-number changes in 150 meningiomas and then characterized these samples for mutations in AKT1, KLF4,
NF2, PIK3CA, SMO, and TRAF7. Similar to prior reports, we identified AKT1 and SMO mutations in a subset of non-NF2-mutant meningiomas (ie, similar to 9% and similar to 6%, respectively). Notably, we detected
87
oncogenic mutations in PIK3CA in similar to 7% of non-NF2-mutant meningiomas. AKT1, SMO, and PIK3CA
mutations were mutually exclusive. AKT1, KLF4, and PIK3CA mutations often co-occurred with mutations in
TRAF7. PIK3CA-mutant meningiomas showed limited chromosomal instability and were enriched in the skull
base.”
According to the news reporters, the research concluded: “This work identifies PI3K signaling as an important target for precision medicine trials in meningioma patients.”
For more information on this research see: Oncogenic PI3K mutations are as common as AKT1 and SMO
mutations in meningioma. Neuro-Oncology , 2016;18(5):649-655. Neuro-Oncology can be contacted at: Oxford
Univ Press Inc, Journals Dept, 2001 Evans Rd, Cary, NC 27513, USA. (Oxford University Press - http://
www.oup.com/ ; Neuro-Oncology - neuro-oncology.oxfordjournals.org)
Our news correspondents report that additional information may be obtained by contacting M. Abedalthagafi, Dana Farber Canc Inst, Dept. of Canc Biol, Boston, MA 02115, United States. Additional authors for this
research include W.L. Bi, A.A. Aizer, P.H. Merrill, R. Brewster, P.K. Agarwalla, M.L. Listewnik, D. DiasSantagata, A.R. Thorner, P. Van Hummelen, P.K. Brastianos, D.A. Reardon, P.Y. Wen, O. Al-Mefty, S.H.
Ramkissoon, R.D. Folkerth and Ligo.
NewsRx LLC
(2016-06-17), Reports from Dana-Farber Cancer Institute Add New Data to Findings in Meningioma and
Genetics (Oncogenic PI3K mutations are as common as AKT1 and SMO mutations in meningioma), Health &
Medicine Week, 3117, ISSN: 1532-4605, BUTTER® ID: 011849543
Reports from University of Colorado Describe Recent Advances in Cystic Fibrosis and
Genetics (Cystic fibrosis: a model system for precision medicine)
By a News Reporter-Staff News Editor at Biotech Week – Investigators discuss new findings in Lung Diseases
and Conditions. According to news reporting from Aurora, Colorado, by NewsRx journalists, research stated,
“Development of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, small molecule therapies that target the basic defect in cystic fibrosis (CF), represents a new era in CF treatment. This review highlights recent progress in CF therapeutics as an example of precision medicine and personalized approaches to
test CFTR modulators using preclinical model systems.”
The news correspondents obtained a quote from the research from the University of Colorado, “CFTR modulators are now clinically available for approximately 50% of the United States CF population. The CFTR potentiator, ivacaftor, is approved for people with CF ages 2 years and older with at least one gating mutation (G551D,
G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R) or the R117H conductance mutation. The
recent Food and Drug Administration approval of the corrector/potentiator combination, lumacaftor/ivacaftor,
expands modulator therapy to people with CF homozygous for the F508del mutation, ages 12 years and older.
Ivacaftor and lumacaftor, however, do not fully restore CFTR activity. Thus, next-generation correctors and
potentiators are in development. Read-through agents targeting nonsense mutations and genotype agnostic
treatments (gene-editing and gene therapy) are also in various phases of clinical development. CFTR modulators promise to transform the therapeutic landscape in CF in a precision based fashion.”
According to the news reporters, the research concluded: “Areas of ongoing research include developing
drugs for all mutation classes so that all persons with CF can benefit from these therapies, and refining preclinical assays that allow the selection of the most effective treatments on an individual basis.”
For more information on this research see: Cystic fibrosis: a model system for precision medicine. Current Opinion in Pediatrics , 2016;28(3):312-317. Current Opinion in Pediatrics can be contacted at: Lippincott
Williams & Wilkins, Two Commerce Sq, 2001 Market St, Philadelphia, PA 19103, USA. (Lippincott Williams
and Wilkins - www.lww.com; Current Opinion in Pediatrics - http://journals.lww.com/co-pediatrics/
pages/default.aspx )
Our news journalists report that additional information may be obtained by contacting S.L. Martiniano,
University of Colorado, Sch Med, Childrens Hosp Colorado, Dept. of Pediat, 13123 E 16th AveB-395, Aurora,
CO 80045, United States. Additional authors for this research include S.D. Sagel and E.T. Zemanick.
NewsRx LLC
(2016-06-22), Reports from University of Colorado Describe Recent Advances in Cystic Fibrosis and Genetics
(Cystic fibrosis: a model system for precision medicine), Biotech Week, 528, ISSN: 1537-4699, BUTTER® ID:
011930791
88
Diabetes
Research Data from Jackson Laboratory Update Understanding of Diabetes (Transcriptional
Regulation of the Pancreatic Islet: Implications for Islet Function)
By a News Reporter-Staff News Editor at Diabetes Week – Current study results on Diabetes have been published. According to news reporting originating from Farmington, Connecticut, by NewsRx correspondents,
research stated, “Islets of Langerhans contain multiple hormone-producing endocrine cells controlling glucose
homeostasis. Transcription establishes and maintains islet cellular fates and identities.”
Our news editors obtained a quote from the research from Jackson Laboratory, “Genetic and environmental disruption of islet transcription triggers cellular dysfunction and disease. Early transcriptional regulation
studies of specific islet genes, including insulin (INS) and the transcription factor PDX1, identified the first
cis-regulatory DNA sequences and trans-acting factors governing islet function. Here, we review how human
islet ‘omics’ studies are reshaping our understanding of transcriptional regulation in islet (dys) function and
diabetes. First, we highlight the expansion of islet transcript number, form, and function and of DNA transcriptional regulatory elements controlling their production. Next, we cover islet transcriptional effects of genetic
and environmental perturbation.”
According to the news editors, the research concluded: “Finally, we discuss how these studies’ emerging
insights should empower our diabetes research community to build mechanistic understanding of diabetes
pathophysiology and to equip clinicians with tailored, precision medicine options to prevent and treat islet
dysfunction and diabetes.”
For more information on this research see: Transcriptional Regulation of the Pancreatic Islet: Implications for Islet Function. Current Diabetes Reports , 2015;15(9):86-98. Current Diabetes Reports can be contacted at: Current Medicine Group, 400 Market Street, Ste 700, Philadelphia, PA 19106, USA. (Springer www.springer.com; Current Diabetes Reports - http://www.springerlink.com/content/1534-4827/ )
The news editors report that additional information may be obtained by contacting M.L. Stitzel, Jackson
Lab Genom Med JAX GM, Farmington, CT 06032, United States. Additional authors for this research include
I. Kycia, R. Kursawe and D. Ucar.
NewsRx LLC
(2016-07-18), Research Data from Jackson Laboratory Update Understanding of Diabetes (Transcriptional
Regulation of the Pancreatic Islet: Implications for Islet Function), Diabetes Week, 70, ISSN: 1537-1433, BUTTER® ID: 012057863
Researchers at University of Patras Target Integrative Biology (Meta-Analysis of Genes in
Commercially Available Nutrigenomic Tests Denotes Lack of Association with Dietary
Intake and Nutrient-Related Pathologies)
By a News Reporter-Staff News Editor at Life Science Weekly – Current study results on Life Science Research
have been published. According to news reporting from Patras, Greece, by NewsRx journalists, research stated,
“Nutrigenomics is an emerging discipline that aims to investigate how individual genetic composition correlates
with dietary intake, as well as how nutrition influences gene expression. Herein, the fundamental question
relates to the value of nutrigenomics testing on the basis of the currently available scientific evidence.”
The news correspondents obtained a quote from the research from the University of Patras, “A thorough literature search has been conducted in PubMed scientific literature database for nutrigenomics research studies
on 38 genes included in nutrigenomics tests provided by various private genetic testing laboratories. Data
were subsequently meta-analyzed to identify possible associations between the genes of interest and dietary
intake and/or nutrient-related pathologies. Data analysis occurred according to four different models due to
data sparsity and inconsistency. Data from 524,592 individuals (361,153 cases and 163,439 controls) in a total
of 1,170 entries were obtained. Conflicting findings indicated that there was a great incompatibility regarding
the associations (or their absence) identified. No specific–and statistically significant-association was identified
for any of the 38 genes of interest. In those cases, where a weak association was demonstrated, evidence was
based on a limited number of studies. As solid scientific evidence is currently lacking, commercially available
nutrigenomics tests cannot be presently recommended.”
According to the news reporters, the research concluded: “Notwithstanding, the need for a thorough and
continuous nutrigenomics research is evident as it is a highly promising tool towards precision medicine.”
89
For more information on this research see: Meta-Analysis of Genes in Commercially Available Nutrigenomic Tests Denotes Lack of Association with Dietary Intake and Nutrient-Related Pathologies. Omics ,
2015;19(9):512-20. (Mary Ann Liebert, Inc. - www.liebertpub.com; Omics - http://www.liebertpub.com/
overview/omics-a-journal-of-integrative-biology/43/ )
Our news journalists report that additional information may be obtained by contacting C. Pavlidis, 1 Dept.
of Pharmacy, School of Health Sciences, University of Patras , Patras, Greece. Additional authors for this
research include Z. Lanara, A. Balasopoulou, J.C. Nebel, T. Katsila and G.P Patrinos.
NewsRx LLC
(2016-07-12), Researchers at University of Patras Target Integrative Biology (Meta-Analysis of Genes in
Commercially Available Nutrigenomic Tests Denotes Lack of Association with Dietary Intake and NutrientRelated Pathologies), Life Science Weekly, 2750, ISSN: 1552-2474, BUTTER® ID: 012034064
Health and Medicine
Studies from Cochin Hospital Describe New Findings in Endocrinology (The genomics of
adrenocortical tumors)
By a News Reporter-Staff News Editor at Health & Medicine Week – Fresh data on Health and Medicine are
presented in a new report. According to news reporting out of Paris, France, by NewsRx editors, research stated,
“The last decade witnessed the emergence of genomics, a set of high-throughput molecular measurements in
biological samples. These pan-genomic and agnostic approaches have revolutionized the molecular biology and
genetics of malignant and benign tumors.”
Our news journalists obtained a quote from the research from Cochin Hospital, “These techniques have
been applied successfully to adrenocortical tumors. Exome sequencing identified new major drivers in all
tumor types, including KCNJ5, ATP1A1, ATP2B3 and CACNA1D mutations in aldosterone-producing adenomas (APA), PRKACA mutations in cortisol-producing adenomas (CPA), ARMC5 mutations in primary bilateral
macronodular adrenocortical hyperplasia (PBMAH) and ZNRF3 mutations in adrenocortical carcinomas (ACC).
Moreover, the various genomic approaches -including exome sequencing, transcriptome, miRNome, genome and
methylome - converge into a single molecular classification of adrenocortical tumors. Especially for ACC, two
main molecular groups have emerged, showing major differences in outcomes. These ACC groups differ by their
gene expression profiles, but also by recurrent mutations and specific DNA hypermethylation patterns in the
subgroup of poor outcome. The clinical impact of these findings is just starting. The main altered signaling
pathways now become therapeutic targets. The molecular groups of diseases individualize robust subtypes
within diseases such as APA, CPA, PBMAH and ACC. A revised nosology of adrenocortical tumors should impact the clinical research. Obvious consequences also include genetic counseling for the new genetic diseases
such as ARMC5 mutations in PBMAH, and a better prognostication of ACC based on targeted measurements
of a few discriminant molecular alterations.”
According to the news editors, the research concluded: “Identifying the main molecular groups of adrenocortical tumors by extensively gathering the molecular variations is a significant step forward towards precision
medicine.”
For more information on this research see: The genomics of adrenocortical tumors. European Journal of
Endocrinology , 2016;174(6):R249-R265. European Journal of Endocrinology can be contacted at: Bioscientifica
Ltd, Euro House, 22 Apex Court Woodlands, Bradley Stoke, Bristol BS32 4JT, England.
Our news journalists report that additional information may be obtained by contacting S. Faillot, Hopital
Cochin, AP HP, Reference Center Rare Adrenal Dis, Dept. of Endocrinol, 27 Rue Faubourg St Jacques, F-75014
Paris, France.
NewsRx LLC
(2016-07-01), Studies from Cochin Hospital Describe New Findings in Endocrinology (The genomics of
adrenocortical tumors), Health & Medicine Week, 1496, ISSN: 1532-4605, BUTTER® ID: 011974373
90
Vibrent Health
Vibrent Health Funded by the National Institutes of Health to Support Historic U.S.
Precision Medicine Initiative Research Program
By a News Reporter-Staff News Editor at Journal of Engineering – Vibrent Health announced that it has been
selected to provide the technology platform for the groundbreaking Precision Medicine Initiative (PMI) research
program led by the White House. This landmark longitudinal initiative aims to engage one million or more U.S.
participants as partners to improve the ability to prevent and treat diseases based on individual differences in
lifestyle, environment and genetics.
The PMI Program is one of the most ambitious projects in history and will set the foundation for new
ways to enable “patient-powered medicine.” PMI volunteers will be asked to contribute a wide range of health,
environment and lifestyle information. They will also be invited to answer questions about their health history
and status, share their genomic and other biological information through simple blood and fluid tests and grant
access to their clinical data from electronic health records. In addition, mobile health devices and apps will
provide lifestyle data and environmental exposures in real time. All of this will be protected using highest
standards in federal data security such as FedRAMP and implementation guidelines for privacy, trust and
consent. The participants will have access to a wide range of their individual data and insights as well as to
aggregated results.
Volunteers will enroll, consent and donate their data through Vibrent Health’s technology platform using
mobile apps, web interfaces, interactive voice response (IVR), feature phones and wearable sensors. Vibrent
Health will develop, test, maintain and upgrade the technology platform over a period of five years for the term
of the award using state of the art and secure data hosting from AWS GovCloud. NIH aims to begin enrolling
participants later this year and has set a goal of reaching one million volunteers within four to five years and
hopes to continue to enroll participants well beyond that mark.
“This $74 million funding over five years is one of the largest federal investments in a health technology
platform of its kind and we are honored to be selected for this mission-critical initiative,” said Vibrent Health
founder and CEO Praduman Jain. “It validates our platform and demonstrates customer confidence in our
technology innovation, security and maturity.”
The Vibrent Health ‘personalization engine’ is a central component of the platform that will tailor precise
experiences and data collection methods to meet the unique needs of every individual. The intention is to
catalyze a revolution in how to prevent, detect and treat diseases with a level of precision that is not possible
without the large and diverse dataset that the PMI research program will generate.
“This is the most far-reaching medical research initiative in the history of the United States,” said Eric
Topol, Director of the Scripps Translational Science Institute. “So for us at Scripps to get to work again with
Praduman Jain and his remarkably talented team at Vibrent Health, we’re set up for a truly exciting and
transformative collaboration.”
Vibrent Health has partnered with The Scripps Research Institute (TSRI), which will drive engagement
research, participant enrollment of direct volunteers over five years and validation of emerging wearables and
medical devices.
Vibrent has also partnered with CSC who will provide an important element of human touch that complements the technology approach to data donation. CSC will provide participant support for consent, engagement, retention outreach and technical support. “As a physician, for my patients and profession, I am excited to
partner with Vibrent Heath on this groundbreaking Precision Medicine Initiative that will advance healthcare
discoveries and improve health,” said Dr. Robert Wah, Global Chief Medical Officer at CSC.
Vibrent Health looks forward to enabling other large scale implementations for advocacy, clinical research,
public health, global health, clinical care and medical device organizations using the Vibrent Health Precision
Medicine Platform. President Obama Quote “Precision medicine gives us the chance to marry what’s unique
about America - our spirit of innovation, our courage to take risks, our collaborative instincts - with what’s
unique about Americans - every individual’s distinctive genetic makeup, lifestyles, and health needs. In doing
so, we can keep ourselves, our families, and our nation healthier for generations to come,” said President Barack
Obama in an op-ed for the Boston Globe. NIH Director Francis S. Collins Quote “This range of information
at the scale of one million people from all walks of life will be an unprecedented resource to understand all of
the factors that influence health and disease,” said NIH Director Francis S. Collins, M.D., Ph.D. “Over time,
data provided by participants will help us answer important health questions, such as why some people with
elevated genetic and environmental risk factors for disease still manage to maintain good health, and how
people suffering from a chronic illness can maintain the highest possible quality of life. The more we understand
about individual differences, the better able we will be to effectively prevent and treat illness.”
91
For more information on the Precision Medicine Initiative (PMI), see https://www.whitehouse.gov/
precision-medicine . For more information on the PMI Cohort Program, see https://www.nih.gov/
precision-medicine-initiative-cohort-program About Vibrent Health Vibrent Health provides an
evidence-based B2B2C technology infrastructure called ‘Adaptive Platform for Precision Engagement’ that enables longitudinal data collection, engagement and insights. This infrastructure is supported by the three
pillars of technology, science and customer support, each of which continuously improves through a process
of “measure-learn-iterate”. Such an approach enables adaptability and personalization to ensure long-term
success of population level programs. To learn more visit: http://www.vibrenthealth.com/ View source
NewsRx LLC
(2016-07-25), Vibrent Health Funded by the National Institutes of Health to Support Historic U.S. Precision Medicine Initiative Research Program, Journal of Engineering, 1496, ISSN: 1945-872X, BUTTER® ID:
012095584
Delirium and Genetics
Young African-American adults are less susceptible to delirium in ICU than Caucasians
By a News Reporter-Staff News Editor at Mental Health Weekly Digest – INDIANAPOLIS – The first study to
evaluate the relationship between race and intensive care unit delirium has found that African-American ICU
patients age 18 to 50 are less susceptible to delirium than similarly aged Caucasians or than either AfricanAmerican or Caucasian ICU patients age 50 or older.
Delirium is a state of confusion that comes on suddenly and is associated with longer ICU and hospital stays,
increased costs of care and higher death rate. Known risk factors for developing delirium in the ICU include
age, pre-existing cognitive impairment, and sedation (often used in conjunction with mechanical ventilation).
Prior to the new study by researchers from the Regenstrief Institute and the Indiana University Center for
Aging Research, the relationship between race and delirium had not been systematically evaluated.
“Relationship between African-American Race and Delirium in the Intensive Care Unit” is published online
ahead of print in the journal Critical Care Medicine.
“Since African-Americans tend to have higher disease severity in the ICU, we were surprised to find that
race could be a protective factor for younger African-American adults,” said Regenstrief Institute investigator
and IU Center for Aging Research scientist Babar A. Khan, M.D., M.S., the first author of the study.
“We now know that race should be considered among the risk factors for developing delirium for Caucasians
of all ages but only for African-Americans if they are 50 or older. Clearly, different groups have different risk
profiles for delirium.”
A total of 2,087 adults, admitted to a medical or surgical ICU, 48 percent of whom were African-Americans,
participated in the study. The majority participated in an indigent care program or state Medicaid program
and were thus considered to be of similar economic status. All had ready access to healthcare services and
delivery at Eskenazi Health, a healthcare system that puts special emphasis on vulnerable populations in the
metropolitan Indianapolis area. Dr. Khan is the medical director of the Eskenazi Health Critical Care Recovery
Center.
Based on patient data from the Regenstrief Medical Record System, the African-Americans and Caucasians
had comparable death rates, illness severity and similar rates of mechanical ventilation and dementia diagnoses, however smoking and depression diagnosis were greater among Caucasians. More drugs associated with
delirium were prescribed for Caucasians than African-Americans.
The reasons why African-Americans have lower rates of delirium are unclear according to Dr. Khan and
will need exploration in future studies.
“If you look at various studies, there are certain medications to which African-Americans respond better
and from which they have better outcomes compared to Caucasians,” Dr. Khan said. “If in the near future we
are able to learn more about delirium at a basic or molecular level utilizing genetics and biomarkers, we may
be able to come up with better preventive and treatment strategies based on personalization of therapy. Our
findings provide the kind of information we need to conduct precision medicine.”
Dr. Khan notes that even in the short run the clinical implications of the study findings should raise awareness among physicians and other healthcare providers who can provide targeted interventions to decrease
delirium burden.
Delirium presents in several ways. Individuals with delirium may be agitated and aggressive (hyperactive)
or lethargic and withdrawn (hypoactive) or be both hyperactive and hypoactive. While many patients could
92
have delirium upon admission to the ICU, some are able to communicate or follow commands when admitted.
They are still at risk for developing delirium during their ICU stay, which once developed render them unable
to pay attention or communicate.
According to the American Geriatrics Society approximately 7 million hospitalized Americans experience
delirium every year.
NewsRx LLC
(2016-06-27), Young African-American adults are less susceptible to delirium in ICU than Caucasians, Mental Health Weekly Digest, 312, ISSN: 1543-6608, BUTTER® ID: 011948554
93
Chapter 7
Government Agencies Offices and
Entities
Illumina, Inc.
Illumina Applauds FDA’s Issuance of Draft Guidances to Advance Precision Medicine Using
Next Generation Sequencing
By a News Reporter-Staff News Editor at Journal of Engineering – Illumina, Inc. (NASDAQ: ILMN) announced
that it supports the Food and Drug Administration (FDA) in their efforts to provide a flexible and streamlined
approach to the oversight of next generation sequencing (NGS) diagnostic tests, as represented by FDA’s release
of two new draft guidance documents. The FDA draft guidances are part of the larger Precision Medicine
Initiative announced by President Obama in his 2015 State of the Union address.
“This is an important step forward,” said Francis deSouza, President and CEO of Illumina. “We support
the proactive actions the FDA is taking to recognize the benefits of next generation sequencing and provide
appropriately flexible and adaptive regulatory oversight of these tests, while accommodating the rapid evolution
of NGS technologies.”
Next generation sequencing can examine millions of DNA variants at a time, reading a patient’s DNA to
detect genomic variations that determine whether a person has or is at risk of disease and informing treatment
decisions. Illumina will continue to partner with customers and work with FDA to collectively enable the
promise of genetic medicine by driving the adoption of next generation sequencing.
The two FDA draft guidance documents, Use of Standards in FDA Regulatory Oversight of Next Generation
Sequencing Based In Vitro Diagnostics (IVDs) Used for Diagnosing Germline Diseases and Use of Public Human Genetic Variant Databases to Support Clinical Validity for Next Generation Sequencing Based In Vitro
Diagnostics, were released July 6th. About Illumina Illumina is improving human health by unlocking the
power of the genome. Our focus on innovation has established us as the global leader in DNA sequencing and
array-based technologies, serving customers in the research, clinical and applied markets. Our products are
used for applications in the life sciences, oncology, reproductive health, agriculture and other emerging segments. To learn more, visit www.illumina.com and follow @illumina. View source version on businesswire.com:
NewsRx LLC
(2016-07-18), Illumina Applauds FDA’s Issuance of Draft Guidances to Advance Precision Medicine Using
Next Generation Sequencing, Journal of Engineering, 544, ISSN: 1945-872X, BUTTER® ID: 012061734
94
Terrapinn Inc.
Keynote announced for World Precision Medicine Congress USA 2016 - FDA Commissioner
Dr. Robert Califf to tackle precision medicine
By a News Reporter-Staff News Editor at Biotech Business Week – Dr. Robert Califf, the recently appointed
Commissioner of the U.S. Food and Drug Administration (FDA), will be presenting the opening address on the
second day of the inaugural World Precision Medicine Congress USA 2016 (WPMC) presented by Terrapinn
Inc. His talk will be at 9:00 AM on November 15(th), 2016.
Download the preview agenda now at http://goo.gl/rp4xSd
The FDA recently developed a platform called precisionFDA as their portion of the federal Precision Medicine
Initiative. They will be showcasing their efforts towards the new scientific paradigm in the keynote address, as
well as highlighting some of the exciting work behind the precisionFDA platform.
This comes on the heels of the brand new efforts announced by the White House several weeks ago towards
advancing precision medicine across several agencies, including the NIH, FDA and ONC.
“The interface between the FDA and the private sector will be vitally important if precision medicine as
envisioned by President Obama is to become a reality in our lifetime,” says Chris Hackett, Executive Director of
the WPMC and Project Director of Life Sciences at Terrapinn. “I am absolutely honored to have Commissioner
Califf participating and working with us at our inaugural show, and being willing to interface with a rapidly
changing industry. I look forward to evolving our partnership in the coming weeks.”
The Commissioner will be joining a roster of expert speakers on topics ranging from cell and gene therapy
manufacturing to the internet-of-things (IoT) and cloud computing in healthcare. A few highlight speakers
including Gary Palmer, the Chief Medical Officer of NantHealth, Dr. Murray Brilliant from the Marshfield
Clinic and Mike West, CEO of BioTime.
The event is being held November 14-15(th) 2016 at the Hyatt Washington Capitol Hill in Washington, D.C.
Download the preview agenda now at http://goo.gl/rp4xSd
NewsRx LLC
(2016-06-13), Keynote announced for World Precision Medicine Congress USA 2016 - FDA Commissioner
Dr. Robert Califf to tackle precision medicine, Biotech Business Week, 51, ISSN: 1543-6861, BUTTER® ID:
011822366
Infectious Diseases
NIH names Johns Hopkins a Center of Excellence for bioethics research
By a News Reporter-Staff News Editor at Politics & Government Week – The National Human Genome Research Institute of the National Institutes of Health (NIH) has awarded the Johns Hopkins Berman Institute
of Bioethics a “Center of Excellence” grant to study the ethical, legal and social implications (ELSI) of applying
genomics to research on, and the prevention and treatment of, infectious disease. This builds on three years of
work of an exploratory Center of Excellence in ELSI Research (CEER) at the Berman Institute, the first such
project to focus attention on genomic ELSI issues in the context of infectious disease.
The new grant establishes the Johns Hopkins program as a Specialized CEER, with over $4 million in
funding over four years. Only seven other such centers have been established across the country. The CEER
brings together a multidisciplinary team from across Johns Hopkins University and Johns Hopkins Medicine,
led by co-principal investigators Gail Geller, ScD, MHS and Jeffrey Kahn, PhD, MPH of the Berman Institute.
Infectious diseases account for a significant proportion of illness and death worldwide. “Recent research
has suggested that a person’s genes can play a significant role in the susceptibility to infection, its severity and
transmissibility, and the response to treatment,” Geller notes.
“The promise of applying genomic information to the prevention and treatment of disease is driving the
NIH’s Precision Medicine Initiative, and there are important benefits to be realized in the application of the
tools of precision medicine to infectious disease, but also unique ethical, legal, and policy issues,” Kahn says.
The Johns Hopkins CEER team members include experts in genomics, immunology and infectious disease,
bioethics, epidemiology, public health preparedness, education, and health policy, in keeping with the intention
that CEERs create opportunities for transdisciplinary research.
“Johns Hopkins is uniquely suited to examine the ethical, legal, social and policy issues at the intersection
of genomics and infectious disease, with a deep bench of global leaders in all of the relevant disciplines,” Geller
says.
95
The CEER’s transdisciplinary research plan has three specific program areas, each with a unique pilot
project. The program on “implications for research” will address the impact of discoveries related to genetic
variation in HIV and HCV transmission on cohorts of at-risk urban populations. The program on “implications
for public health policy” will analyze the role and impact of advances in vaccinomics for informing populationbased prevention in the context of a pandemic. The program on “implications for clinical practice” will assess the
application of genomics in the clinical management of acute, high consequence infectious diseases like MRSA
and Ebola.
NewsRx LLC
(2016-06-02), NIH names Johns Hopkins a Center of Excellence for bioethics research, Politics & Government Week, 358, ISSN: 1944-270X, BUTTER® ID: 011788367
University of Minnesota
Pioneering study will establish the legal framework for genomic medicine
By a News Reporter-Staff News Editor at Politics & Government Week – The National Institutes of Health
(NIH) has awarded the first-ever grant dedicated to laying the policy groundwork needed to translate genomic
medicine into clinical application. The project - LawSeqSM - will convene legal, ethics and scientific experts
from across the country to analyze what the state of genomic law is and create much-needed guidance on what
it should be.
NIH has declared the adoption of genomic medicine by clinicians to be a top priority to improve both individual and public health. The federal Precision Medicine Initiative (PMI), announced by President Obama and
currently being launched, aims to use genomics and other analyses to accelerate development of more powerful
and tailored treatments for cancer and other diseases. Yet U.S. federal and state genomics law is unclear and
poorly understood, presenting a major obstacle to progress.
As NIH Director Francis S. Collins, MD, and National Cancer Institute Director Harold Varmus, MD, have
written, “Achieving the goals of precision medicine will . . . require advancing the nation’s regulatory frameworks.” Leading LawSeqSM is a team of three principal investigators:
These co-leaders will be joined by a group of 22 top experts - from academia, industry, and clinical care who will collaborate over the course of this 3-year project to clarify current law, address gaps, and generate the
forward-looking recommendations needed to create the legal foundation for successfully translating genomics
into clinical care.
NewsRx LLC
(2016-06-30), Pioneering study will establish the legal framework for genomic medicine, Politics & Government Week, 355, ISSN: 1944-270X, BUTTER® ID: 011971300
Publicly funded cancer researchers present trial results at ASCO 2016
By a News Reporter-Staff News Editor at Clinical Trials Week – PORTLAND, OR - Investigators from SWOG,
the National Cancer Institute (NCI) funded clinical trials group, will make 21 presentations in Chicago next
week at the 2016 Annual Meeting of the American Society of Clinical Oncology (ASCO), the world’s leading
professional organization for physicians who care for people with cancer.
At ASCO 2016, SWOG investigators will report on clinical trials involving treatments for a variety of cancers, including lung, prostate, breast, ovarian, colorectal, pancreatic, and melanoma. SWOG researchers will
also present on cancer genetics, tumor biology, and patient and survivor care. Of the 21 presentations, 10
focus on SWOG-led trials, including Lung-MAP, the precision medicine trial which tests targeted treatments
and the latest immunotherapies for people with advanced squamous cell lung cancer. Now enrolling patients
nationwide, Lung-MAP is conducted in partnership with the NCI’s National Clinical Trials Network (NCTN),
Friends of Cancer Research, and the Foundation for the National Institutes of Health.
In addition, SWOG Executive Officer Dr. Julie Gralow, an internationally renowned breast cancer physician
and researcher, is part of a team whose work will be highlighted at the June 5th ASCO plenary session, a spot
reserved for the most high-impact cancer trials. Results from this Canadian Cancer Trials Group trial will
focus on the use of aromatase inhibitors in treating postmenopausal women with early-stage breast cancer.
96
“Our presence at ASCO illustrates the power of the NCI’s cancer research network,” said Dr. Charles D.
Blanke, SWOG group chair and professor of medicine at the Knight Cancer Institute at Oregon Health & Science University. “When you connect thousands of researchers at academic medical centers, cancer centers, and
community hospitals across the U.S. and Canada, you can ask bigger, bolder questions about cancer. By working cooperatively, we do trials no other organization can - and can really move the needle in cancer medicine.”
Some highlights of SWOG’s ASCO 2016 presentations:
NewsRx LLC
(2016-06-13), Publicly funded cancer researchers present trial results at ASCO 2016, Clinical Trials Week,
130, ISSN: 1543-6764, BUTTER® ID: 011823248
SolveBio
SolveBio Awarded SBIR Phase I Grant from NIH for Genomic Intelligence Genetic Variant
Analysis and Visualization Tool
By a News Reporter-Staff News Editor at Biotech Business Week – SolveBio, a contextual knowledge hub, announced that the National Institutes of Health (NIH) has funded further development of the Variant Explorer,
a genetic variation analysis and visualization system. SolveBio received a Phase I Small Business Innovation
Research Grant from the National Institute of General Medical Sciences for the research and development
grant, R43GM117644, entitled “The Variant Explorer: a cloud-based data integration and visualization system for improving clinical interpretation of sequenced genetic variants”. The funds will be used to develop the
Variant Explorer as a decision support tool for clinical geneticists, molecular pathologists, and scientists in the
pharmaceutical industry and at academic medical centers.
“The interpretation and analysis of interesting variants is now the single most intractable bottleneck in
the next generation sequencing-based workflow.” said Dandan Xu, chief scientific officer at SolveBio and the
principal scientist for the project. “The Variant Explorer is an interface to the SolveBio contextual knowledge hub for genetic variants that need to be evaluated and classified. The combination of curated and actionable data and an enjoyable user interface allows the scientist and clinician to evaluate genetic variants
more efficiently and effectively. We see this advancement in information visualization and analysis as being
integral to realizing the promise of precision medicine.” About SolveBio SolveBio, founded in 2013, is a contextual knowledge hub focused on enabling pharma, biotech, and diagnostic organizations to effectively connect and use external and internal genomic information. SolveBio extracts, evaluates, and unifies complex,
disparate biomedical data for life science enterprises. SolveBio is based in New York and is backed by Andreessen Horowitz, Max Levchin, Nat Turner, SV Angel, Faridan Capital and other leading life sciences and
technology investors. For more information, visit www.solvebio.com. View source version on businesswire.com:
NewsRx LLC
(2016-06-20), SolveBio Awarded SBIR Phase I Grant from NIH for Genomic Intelligence Genetic Variant
Analysis and Visualization Tool, Biotech Business Week, 100, ISSN: 1543-6861, BUTTER® ID: 011908379
The Translational Genomics Research Institute
TGen and international team find new avenues of precision medicine for treating cancer
By a News Reporter-Staff News Editor at Politics & Government Week – PHOENIX, Ariz. – An international
team of scientists, including those at the Translational Genomic Research Institute (TGen), have discovered
new avenues of potential treatments for a rare and deadly cancer known as Adrenocortical Carcinoma, or ACC.
In a study published in the scientific journal Cancer Cell, researchers conducted an extensive genomic profile
of ACC, a cancer of the adrenal glands, which are located above the kidneys. Current treatment options for
ACC have not changed in decades and are not curative.
“This is one of the most comprehensive genomic characterizations ever done of this rare tumor type,” said
Dr. Timothy Whitsett, an Assistant Professor in TGen’s Cancer and Cell Biology Division, and one of the study’s
authors. “This study should provide rationale and validation for new therapeutic strategies and clinical studies,
providing potentially better treatments for ACC patients.”
97
The study is part of The Cancer Genome Atlas (TCGA), a program overseen by the National Institutes of
Health (NIH) that aims to generate comprehensive, multi-dimensional maps of the key molecular changes in
major types of cancer.
“This data has implications for diagnosing and predicting outcomes of adrenal cancer. It also allows us to
probe deep into the biology of the disease to understand how these new gene mutations contribute to adrenal
cancer progression and formation,” said senior author Dr. Gary D. Hammer, the Millie Schembechler Professor
of Adrenal Cancer at the University of Michigan Comprehensive Cancer Center.
The ACC study examined 91 tumor samples from six countries across four continents, providing a global
look at this disease.
One of the major findings of this study is the identification of a third class, or subtype, of ACC. The study
showed that the three subtypes of ACC hold significantly different outcomes for patients, and – based on their
distinct molecular biomarkers – could help determine the best course of treatment for each patient.
“Clinical implementation of this three-class grading system would represent a true advance for patient care,”
the report said.
Another key finding was that many adrenal tumors undergo whole genome doubling – a phenomenon in
which each chromosome in the gene replicates and creates a second copy. This reflects instability of the cancer
genome, which is particularly prominent in adrenal cancer.
The study also identified additional genes that may drive the formation and progression of ACC tumors. And
based on a review of clinical trials and FDA-approved cancer drugs, the study identified 51 genetic alterations
that could provide targets for new therapeutics.
“We hope these results illustrate how integrating molecular and clinico-pathologic data can inform more
precise therapeutic decision making for ACC patients,” said Dr. Whitsett, who along with TGen colleagues
provided analyses of ACC’s cellular pathways, clinical pathology data and overall disease expertise.
Other than surgery and radiation, a standard treatment for ACC is the use of a compound called mitotane,
a chemical relative of DDT, which the U.S. banned as an insecticide in 1972. The adrenal glands are responsible for making several critical hormones, including those needed to respond to stress and maintain normal
blood sugar levels. While use of mitotane in ACC patients reduces tumors, it does not provide a cure and has
significant side effects. New and better therapies are needed for these patients.
The study published in Cancer Cell, titled Comprehensive Pan-Genomic Characterization of Adrenocortical
Carcinoma, was coordinated by the National Cancer Institute and the National Human Genome Research
Institute, and funded by the NIH.
In another study of ACC published March 10 in the journal PLOS ONE, TGen researchers used a new
method of investigating DNA methylation – one of many mechanisms that cells use to control how genes are
expressed – to identify new genes and cellular pathways associated with cancer of the adrenal glands.
“Using the ‘discretization method,’ which groups samples according to the DNA methylation levels, we were
able to more precisely learn which DNA methylation changes result in aberrant gene and cellular-pathway
expression in ACC,” said Dr. Bodour Salhia, an Assistant Professor of TGen’s Integrated Cancer Genomics
Division, and the study’s senior author. “This analysis of our data revealed new information about how genes
are modulated and specifically how important tumor-suppressor genes can be turned off and allow the cancer
to grow.”
This study in PLOS ONE was able to use data unearthed during the Cancer Cell study to further understanding of the potential drivers of ACC, Dr. Salhia said.
The study in PLOS ONE, titled Pathway Implications of Aberrant Global Methylation in Adrenocortical
Cancer, was funded in part by the TGen Foundation through its ATAC Research Fund, the Kirsten’s Legacy
Fund, and contributions by the Virginia G. Piper Charitable Trust and an individual contributor, Mr. Ray
Thurston.
NewsRx LLC
(2016-05-26), TGen and international team find new avenues of precision medicine for treating cancer, Politics & Government Week, 270, ISSN: 1944-270X, BUTTER® ID: 011732887
98
Scripps Research Institute
TSRI awarded $20 million for first year of precision medicine initiative cohort program
By a News Reporter-Staff News Editor at Journal of Engineering – LA JOLLA, CA - As part of the most ambitious medical research program in the history of American medicine, The Scripps Research Institute (TSRI)
has received an initial award of $20 million for its role in a national precision medicine initiative, the National
Institutes of Health (NIH) announced.
“We are thrilled to have the opportunity to help lead this far-reaching, transformative program of one million or more U.S. participants with long-term follow up,” said the new grant’s principal investigator Eric Topol,
MD, who is director of the Scripps Translational Science Institute (STSI), professor of genomics at TSRI and
chief academic officer at Scripps Health. “Our focus at STSI for the decade of its existence has been to advance
individualized medicine. Using genomics, mobile apps and biosensors and providing data back to each participant, this study will set the foundation for new medical knowledge and ways of engaging people in research as
citizen-scientists.”
“This grant, which will total almost $120 million over five years, is one of the largest federal grants ever
awarded to TSRI,” said TSRI CEO Peter Schultz, PhD, “and it represents the expanding leadership role that
Eric and TSRI will play in this new era of biomedical research.”
The five-year award is part of the Cohort Program of President Obama’s Precision Medicine Initiative (PMI);
the PMI Cohort Program is a landmark longitudinal research effort that aims to engage one million or more
U.S. participants to improve the ability to prevent and treat disease based on individual differences in lifestyle,
environment and genetics.
“STSI already has played a pivotal role in a digital medicine revolution that is radically transforming the
way we think about and provide health care,” said Chris Van Gorder, president and CEO of Scripps Health,
which operates STSI in partnership with TSRI. “This grant ensures the continuation of that work for years to
come on a scale that will benefit the entire nation.”
The new grant will support a Participant Technologies Center through STSI that will play a key role in
enrolling participants in the study, as well as providing mobile applications. In parallel, the center will develop
platforms to deliver these same functions to those without smartphones and work with various technology
organizations to increase smartphone accessibility.
STSI has partnered with the NIH-funded Vibrent Health of Fairfax, Virginia, which will provide the technology platform to enroll and engage participants through mobile apps, web app, interactive voice response,
feature phones and wearable sensors; Vibrent Health will develop, test, maintain and upgrade the technology
platform to enroll, consent, collect data from, communicate with and retain participants throughout the grant
period.
In addition, Seattle-based Sage Bionetworks, an integral partner in the design, development and data hosting for many of mobile-app-based research studies on Apple’s open source ResearchKit platform, will be responsible for developing symptom measurements from phone, wearable and other sensors, as well as community
outreach and participant engagement efforts with the Participant Technologies Center.
The initiative also includes an extensive network of high-profile partners including Walgreens and PatientsLikeMe. Working collaboratively with other entities, the Scripps Participant Technologies Center will be responsible for enrollment of at least 350,000 participant-volunteers.
“These partners provide unparalleled outreach and engagement, along with data privacy and security capabilities,” Topol said.
The team aims to begin initial enrollment into the study this year, with the aim of meeting its enrollment
goal by 2020.
Participants will be invited to answer questions about their health history and status, share their genomic
and other biological information through simple blood and urine tests and grant access to their clinical data
from electronic health records. In addition, mobile health devices and apps will provide lifestyle data and
environmental exposures in real time. All of this will be accomplished with essential privacy and security
safeguards. As partners in the research, participants will have ongoing input into study design and implementation, as well as access to a wide range of their individual and aggregated study results.
“This range of information at the scale of 1 million people from all walks of life will be an unprecedented
resource for researchers working to understand all of the factors that influence health and disease,” said NIH
Director Francis S. Collins, M.D., Ph.D. “Over time, data provided by participants will help us answer important
health questions, such as why some people with elevated genetic and environmental risk factors for disease
still manage to maintain good health, and how people suffering from a chronic illness can maintain the highest
99
possible quality of life. The more we understand about individual differences, the better able we will be to
effectively prevent and treat illness.”
The knowledge gained from the PMI Cohort Program will extend successes of precision medicine in some
cancers to many other diseases. Importantly, the program will focus not just on disease, but also on ways to
increase an individual’s chances of remaining healthy throughout life.
NewsRx LLC
(2016-07-18), TSRI awarded $20 million for first year of precision medicine initiative cohort program, Journal of Engineering, 1076, ISSN: 1945-872X, BUTTER® ID: 012062306
Data Visualization
UC San Diego to participate in White House’s National Microbiome Initiative
By a News Reporter-Staff News Editor at Information Technology Newsweekly – On May 13, the White House
Office of Science and Technology Policy (OSTP) announced a new National Microbiome Initiative , a coordinated
effort to better understand microbiomes – communities of microorganisms that live on and in people, plants,
soil, oceans and the atmosphere – and to develop tools to protect and restore healthy microbiome function.
OSTP is launching the initiative with a combined federal agency investment of more than $121 million. The
University of California San Diego is a key participant in this effort, investing $12 million in its own microbiome
research efforts.
The National Microbiome Initiative puts the spotlight on UC San Diego’s Microbiome and Microbial Sciences
Initiative, a concerted research and education effort initiated in October 2015 by Chancellor Pradeep K. Khosla
to leverage the university’s strengths in science, medicine, engineering and the humanities. The campuswide undertaking encompasses a research-focused Center for Microbiome Innovation and a student-centered
Microbial Sciences Graduate Research Initiative.
Three UC San Diego leaders in the microbiome field were invited to attend the May 13 event in Washington,
D.C.: Rob Knight, PhD, director of the UC San Diego Center for Microbiome Innovation, professor of pediatrics
in the School of Medicine and professor of computer science and engineering in the Jacobs School of Engineering;
Pieter Dorrestein, PhD, professor of pharmacy in the Skaggs School of Pharmacy and Pharmaceutical Sciences
and professor of pharmacology and pediatrics in the School of Medicine; and Embriette Hyde, PhD, assistant
project scientist and project manager of the American Gut Project in the School of Medicine.
The UC San Diego Center for Microbiome Innovation has also received letters of support from a number of
companies committed to innovation in this field, including GE, Illumina, Janssen R&D, MO BIO Laboratories,
Biota, Sirenas, GALT and ChuckAlek. Participation in the White House’s National Microbiome Initiative and
this network will help the Center for Microbiome Innovation target the human microbiome as a means to better
manage diseases such as asthma, diabetes, obesity and psychiatric illnesses – and explore the microbiome as a
source for new drugs and potential tool for precision medicine. UC San Diego researchers will also collaborate
to advance research on soil, aquatic and other environmental microbiomes, and help other scientists use that
information to address global challenges to agricultural sustainability, biofuel development and climate change
mitigation.
“This ambitious undertaking cannot be accomplished by individual laboratories working in isolation –
developing advanced microbiome tools and treatments requires new collaborations among many disciplines.
Advancing this relatively new field also depends on attracting and training multidisciplinary networks of scientists and engineers,” Chancellor Khosla said.
“We are very fortunate that the White House and OSTP recognize the importance of microbiome research
and supports these needs, and we’re grateful that they recognize the leadership of UC San Diego faculty in
this field. We look forward to working with the White House and scientific leaders around the country on this
initiative, which we expect will rapidly lead to a variety of new scientific insights, technological advances and
economic opportunities that will benefit society, human health and the environment for decades.”
UC San Diego’s leadership in the microbiome field
UC San Diego became a part of the driving force behind the National Microbiome Initiative after Knight
helped organize a July 2013 scientific meeting in Washington, D.C., that focused on the future of human microbiome science. That meeting led to invitations to testify before Congress on the importance of the field and to
meet with new leaders in the White House’s Office of Science and Technology Policy. In October 2015, Knight
and Dorrestein joined a group of top scientists to co-author a Science paper that specifically called for a national
initiative to coordinate and accelerate microbiome research across the U.S.
100
More than 100 UC San Diego Center for Microbiome Innovation faculty – spanning disciplines from engineering to medicine to ecology – are involved in standards efforts, such as Microbiome Quality Control and
the Genomic Standards Consortium; large-scale crowdsourced projects, such as the Earth Microbiome Project,
American Gut, Global Natural Products; and a collaborative network spanning thousands of investigators at
other institutions. Participation in the National Microbiome Initiative will allow these researchers to further
these efforts, building new bridges between technology developers and end-users in fields ranging from pharmaceuticals to agriculture.
The UC San Diego Center for Microbiome Innovation is already advancing the three main goals of the White
House OSTP National Microbiome Initiative:
Support interdisciplinary microbiome research
“Microbes pervade all kinds of processes – from our bodies to our planet to industrial fermentation and
drug synthesis. Working closely with other researchers in the White House’s National Microbiome Initiative
will help us unravel the fundamental science so we can understand how microbes do all these things, and help us
improve the speed and accuracy in which we can ‘read out’ microbes,” said Knight, who is known for developing a
genetic sequencing technique that allows researchers to differentiate unknown microbes in hundreds of samples
at once.
In one example project that takes advantage of interdisciplinary expertise provided by the Center for Microbiome Innovation, UC San Diego physicians, microbiome researchers, chemists, genomics experts and bioinformaticians are collaborating to build a 3D map of the chemistry associated with cystic fibrosis and how it shapes
the lung microbiome. Their goal is to develop more effective, highly personalized treatments for potentially fatal
lung infections that frequently affect people with this disease.
In another multidisciplinary project, UC San Diego researchers are sequencing the gut microbiomes of
people with inflammatory bowel diseases (IBD), such as Crohn’s disease, and comparing them to healthy gut
microbiomes. The team aims to use this information to subtype Crohn’s disease patients into distinct populations, going beyond classifications based on disease site such as ileal and colonic Crohn’s. Here, the goal
is to instead identify microbiomarkers associated with response to different treatments. This approach may
allow for more specific diagnoses, prognoses, and perhaps more personalized treatments for IBD. This project
brings together UC San Diego clinical gastroenterologists and genetic sequencing experts, as well as computer
scientists who are developing the high-performance computing, metagenomic assembly and data visualization
infrastructure needed to read out microbiomes and analyze the results.
Develop platform technologies – with academic-industry partnerships
The UC San Diego Center for Microbiome Innovation serves as a platform for industry-university collaborations on the most pressing microbiome challenges.
“I am extremely excited about the many ways that leading-edge microbiome science can be translated to the
real world,” said Knight. “Health, animal care, agriculture, personal care products, industrial processes and
environmental remediation are just a few of the applications that are ripe for disruption. Improving microbiome
technologies is another area where academic-industry partnerships will yield extraordinary advances.”
Through their microbiome center, Dorrestein and Knight are leading a UC San Diego-wide effort to provide
government agencies, health care providers, industry partners and others with a rapid response system for
microbiome sample readout and analysis that streamlines the process into a matter of days or even hours – a
more clinically useful timeframe than the weeks or months it can typically take to obtain this type of data.
“UC San Diego is fast becoming the go-to place to read out microbes – mapping which species live where and
in what quantities – and understand what they are doing,” said Dorrestein, who is a leader in metabolomics
and data visualization, two technologies that are being increasingly applied to study the chemical environments
surrounding microbiomes and to enable rapid interpretation of the results.
Expand the microbiome workforce
Developing a disruptive microbiome workforce is another key UC San Diego effort. In the second branch of
the UC San Diego Microbiome and Microbial Sciences Initiative, Kit Pogliano, PhD, professor in the Division of
Biological Sciences, directs a student-centered Microbial Sciences Graduate Research Initiative. The graduate
program is campus-wide, meaning students can work with any affiliated faculty member in any department.
“We aim to train the next generation of cross-disciplinary researchers with strong quantitative skills that
can be applied to microbiome studies, and encourage innovative ideas that will disrupt almost every industry,
most notably food, energy and medicine,” Pogliano said.
NewsRx LLC
(2016-05-31), UC San Diego to participate in White House’s National Microbiome Initiative, Information
Technology Newsweekly, 324, ISSN: 1944-1800, BUTTER® ID: 011778179
101
Veracyte
Veracyte Releases Statement on CMS’s Final PAMA Rule
By a News Reporter-Staff News Editor at Politics & Government Week – Veracyte, Inc. (NASDAQ: VCYT), a
molecular diagnostics company pioneering the field of molecular cytology, issued the following statement in
response to the Centers for Medicare and Medicaid Services’ (CMS) release of its final rule outlining how the
agency will establish Medicare reimbursement rates for advanced genomic tests such as Veracyte’s Afirma(®)
Gene Expression Classifier (GEC) under the Protecting Access to Medicare Act (PAMA), beginning January 1,
2018.
“We are pleased that CMS has released the final PAMA rule and will now move forward to implement
market-based pricing for advanced genomic tests such as the Afirma GEC,” said Bonnie Anderson, Veracyte
president and chief executive officer. “We believe PAMA implementation will bring welcome transparency and
certainty to Medicare pricing. This in turn should help fuel innovation in diagnostics, which is transforming
patient care and helping to make precision medicine a reality. Of course, details matter and we look forward
to reviewing the PAMA rule closely and continuing to engage with CMS on its implementation.”
The Afirma GEC has helped tens of thousands of patients avoid unnecessary thyroid surgery following
indeterminate biopsy results. Under the new PAMA rule, Medicare reimbursement for advanced diagnostic
laboratory tests (ADLTs) such as the Afirma GEC will be based on the median price paid by commercial payers. Most private payers currently cover the Afirma GEC at a rate that is at or above the current Medicare
reimbursement rate of $3,200. Under PAMA, the new market-based Medicare rates will override any prior
rates.
Veracyte aims to achieve Medicare coverage for the Percepta(®) Bronchial Genomic Classifier, which is used
to improve lung cancer diagnosis, in 2016. The company plans to seek coverage and a rate determination for
Percepta under the Palmetto GBA MolDx program as it did for the Afirma GEC. At a time the company deems
appropriate, it will secure a unique CPT code for Percepta and transition Medicare rates to the PAMA process.
NewsRx LLC
(2016-07-07), Veracyte Releases Statement on CMS’s Final PAMA Rule, Politics & Government Week, 270,
ISSN: 1944-270X, BUTTER® ID: 012007447
Veracyte
Veracyte Statement on CMS’s Preliminary Gapfill Rate for the Afirma® Gene Expression
Classifier
By a News Reporter-Staff News Editor at Politics & Government Week – Veracyte, Inc. (NASDAQ: VCYT), a
molecular diagnostics company pioneering the field of molecular cytology, issued the statement below on the
Centers for Medicare and Medicaid Services’ (CMS) preliminary “gapfill” Medicare reimbursement rate for
Veracyte’s Afirma Gene Expression Classifier (GEC), which the agency released this afternoon.
Medicare currently reimburses $3,200 per test for the Afirma GEC, which is used to help patients avoid an
unnecessary thyroid surgery when their biopsy results are ambiguous. The preliminary proposed gapfill rate,
which is based on the median proposed rate submitted by all Medicare Administrative Contractors (MACs)
- whether they currently administer claims for the test (based on geography) or not - is $2,240.16. Medicare
represents approximately 20 percent of Afirma GEC test volume.
“We are disappointed by the proposed gapfill rate, which we believe does not accurately reflect the value that
the Afirma GEC delivers to patients and the healthcare system,” said Bonnie Anderson, Veracyte’s president
and chief executive officer. “Further, our test was one of an entire group of precision medicine diagnostic tests
whose preliminary reimbursement rates were reduced. We plan to engage vigorously with CMS - both directly
and with our industry partners through The Coalition for 21(st) Century Medicine - and are optimistic that the
final Medicare reimbursement rate for the Afirma GEC will match the current rate of $3,200. We believe that
to do otherwise would be a significant step backwards for innovation, which is at the core of the White House’s
Precision Medicine Initiative.”
Through the gapfill process, the price for a test is determined by the median price submitted by each of
the agency’s MACs. In the case of the Afirma GEC, the MAC (Noridian) that currently processes all Medicare
claims for the genomic test proposed a rate that matches the test’s current rate of $3,200; other MACs, which
do not have experience with the test, submitted lower proposed rates.
102
The proposed Medicare reimbursement rates will be open to public comment for 30 days, with the final
rates expected to be published later this year and to become effective January 1, 2017. The gapfill rates will be
effective until CMS re-prices the Afirma GEC and other advanced genomic tests using market-based pricing,
which is expected to happen in 2018 as part of the agency’s implementation of PAMA (the Protecting Access to
Medicare Act).
NewsRx LLC
(2016-06-30), Veracyte Statement on CMS’s Preliminary Gapfill Rate for the Afirma® Gene Expression
Classifier, Politics & Government Week, 272, ISSN: 1944-270X, BUTTER® ID: 011971240
103
Chapter 8
Hospital
Inflammatory Bowel Diseases
A new tool for forecasting the behavior of the microbiome
By a News Reporter-Staff News Editor at Journal of Mathematics – A team of investigators from Brigham and
Women’s Hospital and the University of Massachusetts have developed a suite of computer algorithms that
can accurately predict the behavior of the microbiome - the vast collection of microbes living on and inside the
human body. In a paper published in Genome Biology, the authors show how their algorithms can be applied
to develop new treatments for serious diarrheal infections, including Clostridium difficile, and inflammatory
bowel disease. The team also shows how to identify bacteria most crucial for a healthy and stable microbial
community, which could inform the development of probiotics and other therapies.
The open source software package the researchers have designed, known as Microbial Dynamical Systems
INference Engine (MDSINE), uses advanced machine learning technologies to accurately predict how microbial communities in the gut will grow and interact over time. The team validated MDSINE using extensive
computational simulations, and also applied the software to infection experiments to evaluate the dynamics of
C. difficile, a bacterial species that is the most common cause of infection in the hospital and can cause serious illness and even death in patients. The team also analyzed the effects of a “probiotic cocktail” of various
bacterial strains that is being developed to treat inflammatory diseases, predicting the contributions of each
member of the cocktail to maintain the stability of the bacterial community when diet was altered.
“Every person has their own bacterial ecosystem living within them. There’s a lot of exciting new evidence
that giving patients bacteriotherapies, or cocktails of bacteria, may be effective in treating or preventing a
variety of major diseases including infections, arthritis, inflammatory bowel disease, and cancer. MDSINE
is the first tool we’ve developed under the new BWH Precision Medicine Initiative, and we’re releasing it as
open source software in that hope that it will help to advance the bacteriotherapy field,” said senior author
Georg Gerber, MD, PhD, MPH, Assistant Professor in Computational Pathology at BWH and Co-Director of
the Massachusetts Host-Microbiome Center at BWH.
“Advanced computational methods like MDSINE are essential for understanding how to design and evaluate
bacteriotherapies or ‘bugs as drugs,’ and to tailor them to individual patients since everyone’s microbiome is different. Our results have given us insights into new bacteriotherapies for C. difficile infection and inflammatory
bowel disease, and moreover suggest general strategies for developing these therapies for many other diseases,”
said Vanni Bucci, PhD, first author of the study and an assistant professor at the University of Massachusetts
at Dartmouth.
NewsRx LLC
(2016-07-12), A new tool for forecasting the behavior of the microbiome, Journal of Mathematics, 88, ISSN:
1945-8746, BUTTER® ID: 012038029
104
Clearbridge BioMedics
BGI and Clearbridge BioMedics Partner to Develop China CTC Liquid Biopsy Market
towards Precision Medicine
By a News Reporter-Staff News Editor at Journal of Engineering – BGI, the world’s largest omics organization,
has signed a partnership agreement with Singapore-based oncology research and diagnostics company Clearbridge BioMedics (CBB). This aims to develop the CTC (circulating tumor cell) liquid biopsy market in China,
including Mainland China, Hong Kong and Macao. BGI will work together with CBB to solely distribute and
market the ClearCell(®) FX1 solution and related products in China, extend this technology into clinical and
cutting-edge research applications, and develop clinical products. The ClearCell(®) technology is a label-free,
automated system that is able to isolate and enrich intact, viable rare cells from a patient’s blood sample.
This joint collaboration aims to develop an integrated CTC liquid biopsy solution by leveraging BGI’s
strengths from genomics to trans-omics and CBB’s expertise in rare cell separation, in order to provide more
actionable clinical insights through a simple blood draw. BGI is establishing a dedicated team to ensure this
trans-omics science and technology is successfully translated into precision medicine. This team will provide
an innovative end-to-end solution for omics analysis or genetic profiling at the single cell level from extremely
rare cells in clinical samples.
The collaborative research agreement focuses on major types of cancer in China, such as lung, breast, and
liver. BGI and CBB will collaborate to drive clinical adoption by working with key opinion leaders in leading
hospitals in order to expand the CTC technology and genomic applications. Possible applications include cancerrelated targeted gene analysis, drug-related gene analysis, single cell analysis and cancer immunotherapy.
“We believe that liquid biopsy plays a key role in clinical diagnostics and personalized medicine and can
potentially even aid in immunotherapy PD-L1 treatment management. BGI is a global leader in the genomics
and precision medicine space, and this collaboration supports our efforts to bring our ClearCell(®) FX1 System
into the China research and clinical markets. We are convinced their strong network, track record and excellent
capabilities complement our execution and align with our strategic vision for China,” said Mr Johnson Chen,
Chairman and Founder of Clearbridge BioMedics.
“BGI’s goal is to make state-of-the-art genomics highly accessible to the global research community and
clinical markets by integrating an array of leading technologies. This includes BGISEQ series sequencers for
genomics, economies of scale for sample storage and bioinformatics for IT and computing resources. Liquid
biopsy, a critical cancer sample format which enables access to extremely rare cells such as CTC, will enable us
to study cancer genome and diseases at the single cell level. Having worked closely with Clearbridge BioMedics
for the last few years, we are very confident that the combination of their rare cell enrichment technology with
BGI’s genomic and trans-omics technology, and our strong collaboration networks including joint labs in top
hospitals and global alliances based on BGISEQ sequencers, will result in better cancer management. We hope
to accelerate the adoption of precision medicine in China, to better serve the needs of patients,” said Dr. Xun
Xu, Executive Vice President of BGI and Director of BGI Research.
NewsRx LLC
(2016-05-30), BGI and Clearbridge BioMedics Partner to Develop China CTC Liquid Biopsy Market towards
Precision Medicine, Journal of Engineering, 442, ISSN: 1945-872X, BUTTER® ID: 011758142
City of Hope
City of Hope and Providence Agree to Expand Access to Cancer Care
By a News Reporter-Staff News Editor at Politics & Government Week – City of Hope and two Providence
Southern California medical centers announced they will partner to expand access to leading-edge, high-quality
cancer care and services within the communities served by Providence Little Company of Mary Medical Center
Torrance and Providence Holy Cross Medical Center in Mission Hills.
The partnership will focus on caring for patients in the South Bay and the San Fernando and Santa Clarita
valleys with the convenience and benefit of Providence’s long-standing reputation for excellence and compassionate care and City of Hope’s internationally-recognized cancer care and research expertise. With the affiliation, City of Hope will open a multi-disciplinary cancer center in the future Providence Advanced Ambulatory
Care Center in Torrance, providing radiation oncology, medical and surgical oncology, chemotherapy and other
cancer-related services. Additional oncology services will be provided there by Providence Little Company
of Mary Medical Center Torrance’s cancer program, which will relocate its imaging, outpatient surgery and
105
breast center services to the new center. The Providence Advanced Ambulatory Care Center is now in the
design stages and due to open in 2018.
In the San Fernando and Santa Clarita valleys, the relationship will broaden City of Hope’s existing presence
at Providence Holy Cross Medical Center and expand care for cancer patients in the Santa Clarita Valley.
“Cancer care today can be extraordinarily complex,” said Harlan Levine, M.D., chief executive of City of
Hope Medical Foundation. “With City of Hope and Providence partnering in this way, our innovative research
in genomic and precision medicine can bring new hope and treatment opportunities to cancer patients who
might not otherwise have the access they need.”
The relationship will enable City of Hope’s research-driven best practices to be made available in the communities served by the two Providence hospitals, providing oncology integration and expertise within the communities.
“Providence is a mission-driven industry leader in Los Angeles, dedicated to quality and compassionate care
in the communities we serve. Our patients will benefit greatly by remaining close to home while benefiting, as
well, from this advanced level of cancer expertise in their own communities,” said Bernard Klein, M.D., chief
executive of Providence Holy Cross Medical Center.
“City of Hope is the gold standard for advanced cancer care and will bring to this partnership the latest
research and innovative treatments for our patients,” added Mary Kingston, chief executive, Providence Little
Company of Mary Medical Centers Torrance and San Pedro.
City of Hope is one of just 45 National Cancer Institute-designated comprehensive cancer centers. It is
recognized for research and treatment protocols that have advanced cancer care across the nation, and around
the globe.
Providence, a leading Catholic not-for-profit health care organization, is anchored in Southern California
by six hospitals and is the second largest health care provider in the Greater Los Angeles Area. Providence
has been recognized with numerous national awards for quality and patient safety, including being the first
and only health system in California to have all eligible hospitals receive Healthgrades’ Distinguished Hospital
Award for Clinical Excellence two consecutive years.
“Today, all health care providers are being held to higher standards of accountability. We embrace this
challenge,” said Vijay Trisal, M.D., community practices medical director at City of Hope. “All patients deserve
access to the most-advanced cancer treatment options and expertise available, and their health care providers
should be accountable for providing patient-centered care that is affordable and conveniently located. This is
what City of Hope and Providence will achieve together.”
Providence Holy Cross primarily serves the northern San Fernando Valley and the Santa Clarita Valley
while Providence Little Company of Mary Medical Center Torrance serves the South Bay region.
“Our partnership with City of Hope will ensure Providence patients have the best cancer care, close to home,”
said Erik Wexler, chief executive, Providence Southern California. About City of Hope City of Hope is an independent research and treatment center for cancer, diabetes and other life-threatening diseases. Designated
as one of only 45 comprehensive cancer centers, the highest recognition bestowed by the National Cancer Institute, City of Hope is also a founding member of the National Comprehensive Cancer Network, with research
and treatment protocols that advance care throughout the world. City of Hope is located in Duarte, California,
just northeast of Los Angeles, with community clinics throughout Southern California. It is ranked as one of
“America’s Best Hospitals” in cancer by U.S. News & World Report. Founded in 1913, City of Hope is a pioneer
in the fields of bone marrow transplantation, diabetes and numerous breakthrough cancer drugs based on technology developed at the institution. For more information about City of Hope, follow us on Facebook, Twitter,
YouTube or Instagram. About Providence Health & Services: Providence Health & Services, Southern California, is a Catholic not-for-profit, mission-driven healthcare system. Providence Southern California operates
six award-winning hospitals and a comprehensive, fully-integrated network of primary care clinics, urgent care
centers, home care, Providence TrinityCare and Providence TrinityKids Care hospice, the Roy and Patricia Disney Family Cancer Center and the John Wayne Cancer Institute, as well as Providence High School. Providence
is anchored locally by Providence Holy Cross Medical Center in Mission Hills, Providence Saint Joseph Medical
Center in Burbank, Providence Saint John’s Health Center in Santa Monica, Providence Tarzana Medical Center and Providence Little Company of Mary Medical Centers in Torrance and San Pedro. With more than 3,400
physicians, Providence provides coordinated primary and specialty care through an array of physician groups
and individual providers including Providence Medical Institute and physician groups in the South Bay, the
West Valley and Santa Clarita. Providence affiliate, Facey Medical Group, provides primary and specialized
care in the San Fernando and Santa Clarita valleys. For more information, visit California.Providence.org View
106
NewsRx LLC
(2016-06-02), City of Hope and Providence Agree to Expand Access to Cancer Care, Politics & Government
Week, 227, ISSN: 1944-270X, BUTTER® ID: 011788247
Columbia University Medical Center
Four NYC medical centers receive new NIH precision medicine grant
By a News Reporter-Staff News Editor at Ivy League Week – NEW YORK, NY, Columbia University Medical
Center (CUMC) and Weill Cornell Medicine, in collaboration with NewYork-Presbyterian and NYC Health +
Hospitals/Harlem, have been awarded a grant from the NIH for approximately $4 million in fiscal year 2016 to
enroll participants in the Cohort Program of President Barack Obama’s Precision Medicine Initiative (PMI)–a
large-scale research effort to improve our ability to prevent and treat disease based on individual differences in
lifestyle, environment and genetics. The five-year award is estimated to total $46.5 million, pending progress
reviews and availability of funds.
CUMC is one of several medical centers that will provide expertise and infrastructure needed to launch the
PMI Cohort Program. This landmark research effort aims to engage 1 million or more U.S. volunteers from the
diversity of America in a significant research effort to improve our ability to advance precision medicine. The
program seeks to extend the success of precision medicine in some cancers many other diseases. Importantly,
the program will focus not just on disease, but on ways to increase an individual’s chances of remaining healthy
throughout life.
“Columbia’s university-wide commitment to pioneering research and clinical care in precision medicine coincides perfectly with the national priority established by President Obama to improve health and save lives, and
we are deeply enthusiastic about being selected to help lead this effort,” said Columbia University President
Lee C. Bollinger. “We believe that in years to come, our society will benefit immeasurably from the advances in
medical science that will emerge from our collaboration with this team of outstanding New York-based medical
centers.”
“Cornell University has a distinguished legacy of leading scientific discoveries that address our greatest
healthcare challenges,” said Hunter R. Rawlings III, interim president of Cornell University. “The launch of
this collaboration marks a turning point in our effort to conquer disease and to translate research discoveries
into life-changing impact for communities in New York and around the world.”
“The PMI Cohort Program aligns perfectly with our own precision medicine effort, which we launched in
2015 in partnership with NewYork-Presbyterian and faculty from across Columbia University,” said Lee Goldman, MD, Dean of the Faculties of Health Sciences and Medicine and Chief Executive, CUMC. “This award,
in collaboration also with NewYork-Presbyterian, Weill Cornell, and our long-standing colleagues at New York
City Health + Hospitals/Harlem, will extend our ongoing successes in taking an individualized approach to
treating some cancers and rare genetic diseases to a broader range of human illnesses across the ethnically,
culturally, and socioeconomically diverse population we serve. It will also enable us to make sure that research
findings benefit our local population and beyond as quickly as possible.”
“Precision medicine has the power to fundamentally change the way we understand and treat some of the
world’s most challenging diseases,” said Dr. Augustine M.K. Choi, interim dean of Weill Cornell Medicine.
“This NIH grant, and our critical work with colleagues from Columbia, NYC Health + Hospitals/Harlem and
NewYork-Presbyterian, will ensure that we are better able to understand the key genetic and other biological
drivers of disease and ultimately improve the lives of our patients. We are incredibly honored to be selected for
this grant, and grateful to President Obama and the NIH for their bold vision.”
“It’s an incredible honor for our physicians and researchers to be a part of this historic initiative,” said Dr.
Steven J. Corwin, president and CEO of NewYork-Presbyterian. “As we delve into new research and discover
new prevention and treatment options, this grant gives us a tremendous opportunity to continue to excel in our
collective fight against cancer and all life-threatening diseases.”
“The ‘patient-powered’ research that will result from our partnership with CUMC promises to help transform the way we achieve our mission to deliver equitable and culturally responsive care to the city’s most
vulnerable populations,” said Ram Raju, MD, President and CEO of NYC Health + Hospitals. “Our collaboration with CUMC also underscores the critical role that the public hospital system plays in medical education
and cutting-edge research to benefit the communities we serve.”
“We are pleased and excited that the NIH has chosen the Columbia/Weill Cornell/NewYork-Presbyterian and
Harlem Hospital collaboration as one of the partners in this ambitious and fundamentally important program,”
107
said Tom Maniatis, PhD, Director of the Columbia/NewYork-Presbyterian Precision Medicine Initiative and cofounder of the New York Genome Center. Dr. Maniatis is also the Isidore S. Edelman Professor and Chair
of the Department of Biochemistry and Molecular Biophysics at CUMC. “This award is a validation of our
commitment to realize the vision of precision medicine, which identifies relationships between genetic, lifestyle,
and environmental differences in individuals, and the prevention, diagnosis, and treatment of human diseases.
This grant also recognizes the successful establishment of the Institute of Genomic Medicine (IGM) at Columbia
by its Director, Dr. David Goldstein, who has demonstrated the reality of a precision medicine-based approach
to treating children with rare, previously undiagnosed genetic disorders.”
“As doctors and scientists, we are committed to providing our patients with the very best, most cuttingedge care to ensure that illness isn’t a barrier in their everyday lives,” said Dr. Mark Rubin, director of the
Englander Institute for Precision Medicine and the Homer T. Hirst III Professor of Oncology in Pathology at
Weill Cornell Medicine, and director of the precision medicine program at Weill Cornell Medicine and NewYorkPresbyterian/Weill Cornell Medical Center. “The PMI Cohort Program will enable scientists to detect and
delineate the key drivers of disease across the diverse population of patients we serve – and move us closer to
fulfilling the promise of precision medicine.”
CUMC is one of four centers that have been designated as a regional PMI Cohort Program Healthcare
Provider Organization (HPO). As an HPO, CUMC and its partners seek to enroll at least 150,000 volunteers
by 2021. By engaging with a number of community organizations throughout New York City, this multicenter
collaboration will help to ensure that participants in the PMI Cohort Program represent the geographic, ethnic,
racial and socioeconomic diversity of the country that the NIH is hoping to achieve.
NewsRx LLC
(2016-07-26), Four NYC medical centers receive new NIH precision medicine grant, Ivy League Week, 101,
ISSN: 0000-0000, BUTTER® ID: 012105984
Cancer Treatment Centers of America
Largest Study of Genomic Profiling in Colorectal Neuroendocrine Tumors Shows Promise
for Providing Oncologists with Precision Treatment Options for Patients
By a News Reporter-Staff News Editor at Cancer Vaccine Week – Cancer researchers have identified precision treatment options for patients with increasingly aggressive colorectal tumors. Based on genomic profiles,
oncologists developed a personalized treatment approach that achieved a rapid and dramatic response in patients, according to a study published in the June 2016 edition of Cancer Discovery - “BRAF(V600E) Mutations
in High-Grade Colorectal Neuroendocrine Tumors May Predict Responsiveness to BRAF-MEK Combination
Therapy.”
Bruce Gershenhorn, D.O., a medical oncologist at Cancer Treatment Centers of America® (CTCA) at Midwestern Regional Medical Center (Midwestern), along with other researchers, looked at 108 patient cases to
identify effective treatment options for neuroendocrine tumors (NET) of the digestive tract. These solid tumors are increasing with an estimated annual incident rate of 3.65/100,000, according to the National Cancer
Institute Surveillance, Epidemiology and End Results (SEER) registry.
The study, which identifies recurrent somatic BRAF alterations in high-grade colorectal NETs, is highlighted by the dramatic response of two patients within the group. The patients, both female, were diagnosed
with treatment-refractory metastatic high-grade rectal NET harboring a BRAF(V600E) substitution. For each
patient, to investigate further therapeutic options, a comprehensive genomic profile was conducted.
“This is the largest published colorectal NET series and the first reported oncogenic BRAF mutation in highgrade NET,” Gershenhorn said. “What we have demonstrated in this study is the ability of genomic profiling to
identify therapeutically relevant alterations in this aggressive disease with no standard treatment approach.”
With a tailored treatment approach, based on the findings of the genomic profiling, each patient achieved a
rapid and dramatic response to combination BRAF-MEK inhibition-directed therapy.
Patient A, a 70-year old female diagnosed with a poorly differentiated high-grade (grade 3) rectal NET, received neoadjuvant chemoradiation, followed by a low anterior resection and end colostomy showed metastatic
disease, which was confirmed by a liver biopsy as high-grade NET. Follow-up scans confirmed disease progression and she reported abdominal pain with 3 months of her treatment. After her genomic profiling revealed
BRAF(V600E) substitution at a mutant allele frequency (MAF) of 26 percent, she was transitioned to two different drugs, and her symptoms resolved within 10 days of treatment.
108
Patient B, a 39-year old female was found to have a locally advanced high-grade rectal NET. She was treated
with chemotherapy and radiation but developed progressive disease with metastases, and worsening rectal
pain. After her genomic profiling revealed BRAF(V600E) substitution at an (MAF) of 53 percent, she was
transitioned to two other drugs, and within three days her pain issue was resolved, followed by dramatic radiographic response.
“Finding driver mutations, which are the engines of the cancer cell, in uncommon cancers can sometimes
provide a unique treatment option for those patients,” Gershenhorn said. “This case is a great example of the
benefits of NGS in patients with rare tumors, by finding a common and ‘targetable’ driver, extrapolating from
what is known about the mutation in another cancer type, and using a powerful, precision drug combination
to target that engine.”
You can obtain additional information about this study by using the following link. For additional information about neuroendocrine cancers and treatment options, please visit cancercenter.com.
About Cancer Treatment Centers of America® Cancer Treatment Centers of America Global, Inc. (CTCA),
headquartered in Boca Raton, Fla., is a national network of five hospitals that serves adult patients who are
fighting cancer. CTCA® offers an integrative approach to care that combines advancements in genomic testing
and precision cancer treatment, surgery, radiation, immunotherapy and chemotherapy, with evidence-based
supportive therapies designed to help patients physically and emotionally by enhancing their quality of life
while managing side effects both during and after treatment. CTCA serves patients from around the world at
its hospitals in Atlanta, Chicago, Philadelphia, Phoenix and Tulsa. Consistently rated among U.S. hospitals
that deliver the highest quality of care and patient experience, CTCA provides patients and their families
with comprehensive information about their treatment options and encourages their active participation in
treatment decisions. For more information, visit cancercenter.com, http://Facebook.com/cancercenter
and http://Twitter.com/cancercenter .
About Cancer Treatment Centers of America® at Midwestern Regional Medical Center Cancer Treatment
Centers of America® (CTCA) at Midwestern Regional Medical Center (Midwestern) is a landmark in cancer
care that uses advanced technology and precision medicine to offer treatment for adult patients battling cancer.
The 72-bed hospital combines innovative conventional medical treatments with evidence-based integrative oncology services to deliver whole-person care. CTCA® at Midwestern is a Magnet Recognized® hospital, ranking
among the top centers in the nation for nursing excellence. The hospital is accredited by the Foundation for
the Accreditation of Cellular Therapy (FACT) at the University of Nebraska Medical Center for demonstrating
compliance with the FACT-JACIE International Standards for Cellular Therapy Product Collection, Processing
and Administration. CTCA at Midwestern also holds high honors as a Certified Quality Breast Center of Excellence™, recognized by the National Quality Measures for Breast Centers Program™ (NQMBC®). For more
information, please visit http://www.cancercenter.com/midwestern .
NewsRx LLC
(2016-06-20), Largest Study of Genomic Profiling in Colorectal Neuroendocrine Tumors Shows Promise for
Providing Oncologists with Precision Treatment Options for Patients, Cancer Vaccine Week, 25, ISSN: 15436802, BUTTER® ID: 011910097
Biologics
Lattice Biologics Ltd. Reports Second Quarter 2016 Highlights
By a News Reporter-Staff News Editor at Biotech Week – NOT FOR DISSEMINATION IN THE US OR
THROUGH US NEWSWIRE SERVICES
Lattice Biologics Ltd. (TSX VENTURE: LBL) (“Lattice Biologics” or the “Company”) announces financial
results for the second quarter ended March 31, 2016:
Second Quarter Financial Results (all figures denoted in USD):
With 50 new surgeons added to the Company’s user base during the first two quarters of 2016, the Company
is rapidly expanding into new markets and with cutting edge new products. Lattice Biologics’ revenue was
$1,006,975 at 27% gross margin in the three months ended March 31, 2016 compared to $1,065,920 at 18%
gross margin in the three months ended March 31, 2015. The Company expects gross margins to continue to
increase as it shifts the sales mix across the new, significantly higher-margin product lines.
Also during the first two quarters of 2016, Lattice Biologics increased its total number of direct hospital
approvals from 18 to 27, with another approximate 200 hospital approvals currently pending.
The Company continues to make significant improvements on the balance sheet, including an upsurge in
cash from $50,000 to $518,000, a 90% increase. The previously disclosed commitments to convert $1,400,000
109
of debt to equity have been increased to $1,733,000 of debt, which is expected to be converted to equity (at a
weighted average price of CDN $0.23/share). This is in addition to the initial $500,000 (at a weighted average
price of CDN $0.21/share) converted at the time of going public, for a total of $2,233,000 of debt committed to
be converted to equity during the first two quarters of 2016. The $1,733,000 being converted represents 42% of
the Company’s long term debt as of March 31, 2016.
Lattice Biologics maintains its commitment to honoring the gift of donation by implementing a stronger
quality control environment for the recovery and processing of donors. As the Company’s revenues are still
largely concentrated in the musculoskeletal market (representing 84% of the Company’s sales mix in the three
months ended March 31, 2016 compared to 90% in the three months ended March 31, 2015), revenue is highly
dependent on the number of donors the Company is able to recover, and, in turn, process.
As indicated above, the Company continues efforts to diversify the sales mix across the new product lines
added in 2015: Lattice Biologics’ Acellular Dermal Matrix scaffold (AdMatrix) and Demineralized Bone Matrix
putty. As at March 31, 2016, the Company had built adequate inventory levels of the new product lines in
preparation for the growth in sales. 68% of the Company’s $2,232,390 inventory is comprised of both finished
and unfinished goods for the new product lines.
2016 Business Outlook:
The Company remains focused on raising additional capital to: assist with its working capital constraint;
fulfill open purchase orders; launch of the Company’s new product lines; and support R&D efforts. Appropriate
working capital conditions will allow the Company to expand current operations by accepting and processing a
greater number of donors each month and also to launch new high-margin product lines. If Lattice Biologics is
successful in its financing efforts, the Company expects to be cash flow positive by the end of fiscal 2016.
Furthermore, Lattice Biologics is proactively working to complete all additions to its Scientific Advisory
Boards (SABs) which are comprised of strategic Key Opinion Leaders (KOLs) in each of the Company’s targeted
surgical specialties. These professionals play a significant role in influencing industry perception and opinion
and can be instrumental in the recruitment of new surgeon partners and generation of critical clinical data.
“I am extremely pleased with our efforts to build out our SAB team as well as our M&A and R&D efforts to
further our growth trajectory,”states CEO Guy Cook. “EBITDA will be a key indicator of our positive growth
position while key surgeon recruitment will help make Lattice a leader in delivering top-notch care in precision
medicine.”
Lattice Biologics Ltd.
NewsRx LLC
(2016-06-15), Lattice Biologics Ltd. Reports Second Quarter 2016 Highlights, Biotech Week, 120, ISSN:
1537-4699, BUTTER® ID: 011842969
University of California - Los Angeles Health Sciences
National Institutes of Health awards $69.6 million to support research partnership
By a News Reporter-Staff News Editor at Health & Medicine Week – The National Institutes of Health has
awarded $69.6 million to the UCLA Clinical and Translational Science Institute, a research partnership of
UCLA, Cedars-Sinai Medical Center, Charles R. Drew University of Medicine and Science, and the Los Angeles
Biomedical Research Institute.
The new five-year grant follows the consortium’s initial $83.1 million Clinical and Translational Science
Award in 2011, bringing the total NIH support for the project to more than $152.7 million.
Both grants support the continued development of the prestigious consortium, one of more than 60 such
research partnerships established by the NIH to enhance biomedical research. The consortiums accelerate the
translation of laboratory discoveries into more effective treatments for patients; actively engage communities
in clinical research; and train future generations of researchers to work across scientific disciplines to improve
health. The mission of the UCLA Clinical and Translational Science Institute (CTSI) is to bring biomedical
innovations to bear on the greatest health needs of Los Angeles, the largest county in the country, and one of
the most diverse.
“The NIH’s generous investment is further enabling UCLA and its consortium partners to improve the
health of Los Angeles in a meaningful way and to enhance the quality of life in our community,” said Dr. John
C. Mazziotta, vice chancellor of UCLA Health Sciences and chief executive officer of UCLA Health. “As we go
forward and make continued improvements in translating research into better treatments for those diseases
that cause the most disability and early deaths in Los Angeles County, our experiences and successes will
provide a model for health improvement nationwide.”
110
To date, the CTSI has supported the work of more than 1,600 investigators, including basic scientists,
clinicians and population researchers. CTSI infrastructure has supported 40,000 clinical research visits in
outpatient, inpatient and community settings.
CTSI-supported researchers have published more than 1,600 scientific articles and been awarded 370 pilot
grants, enabling them to generate data that they leveraged into $58 million in additional outside funding.
The CTSI itself also has:
“This award supports our comprehensive translational research programs dedicated to advancing scientific
discoveries for the direct benefit our patients and the community,” said Dr. Shlomo Melmed, executive vice
president and dean of the medical faculty at Cedars-Sinai. “As the largest academic medical center in California, Cedars-Sinai is pleased to partner with our UCLA-wide colleagues in this multidisciplinary endeavor to
improve the health of our community and enhance our diagnosis and treatment of disease.”
David Meyer, president and chief executive officer of the Los Angeles Biomedical Research Institute, said
that with the renewal of this grant, crucial funding will flow to accelerate the development the next generation
of devices, diagnostics and therapies, and to advance the field of precision medicine.
“For more than six decades, innovations at LA BioMed have placed tools in the hands of physicians and
healthcare workers,” Meyer said. “Through the CTSI partnership and the benefits that it brings to LA BioMed,
we will be able to ensure that future discoveries will move out of the laboratory and even beyond the bedside to
improve the health of our diverse community and worldwide.”
Dr. Steven M. Dubinett, director of the CTSI, Senior Associate Dean for Translational Research and Associate Vice Chancellor for Research at the David Geffen School of Medicine at UCLA, said the CTSI grants
allow for the creation of “a borderless clinical and translational research institute that brings UCLA CTSI
innovations and resources to bear on the greatest health needs of Los Angeles.”
“This funding is helping us develop new means to retain, recruit and empower scientists to work together
across disciplines, departments, institutions and geography,” Dubinett said.
Said Dr. David M. Carlisle, president and chief executive officer of Charles R. Drew University of Medicine
and Science: “The continuation of the Clinical and Translational Science Award will further the work that
has progressed by the consortium of institutions for the next five years. The NIH’s significant investment in
Los Angeles will help to facilitate the our mission and help to improve the capacity of universities, academic
medical centers, clinicians and researchers to collaborate with each other and with the community to advance
health science research in ways that will reduce health disparities while advancing individual, community and
population health.”
NewsRx LLC
(2016-07-15), National Institutes of Health awards $69.6 million to support research partnership, Health &
Sickle Cell Anemia and Genetics
New approach could make bone marrow transplants safer
By a News Reporter-Staff News Editor at Cancer Gene Therapy Week – Harvard Stem Cell Institute (HSCI)
scientists have taken the first steps toward developing a treatment that would make bone marrow - blood stem
cell - transplantation safer and, as a result, more widely available to the millions of people living with blood
disorders like sickle cell anemia, thalassemia, and AIDS.
Bone marrow transplantation currently is the only curative therapy for these blood diseases. But, for the
new, transplanted stem cells to do their work, the faulty stem cells must first be “evicted” or killed. Accomplishing that requires patients endure chemotherapy and radiation – a vicious assault on the body with life-long
consequences.
In a study recently published in the journal Nature Biotechnology, HSCI researchers at Harvard University
and Massachusetts General Hospital (MGH), in collaboration with Boston Children’s Hospital and Dana Farber
Cancer Institute, have developed a non-toxic transplantation procedure using antibodies to specifically target
blood stem cells in mice, an approach they hope will make blood stem cell transplants for these patients far less
toxic.
The new treatment removes more than 98% of blood stem cells, making it as effective as chemotherapy and
radiation.
“Instead of using non-targeted drugs that have lots of collateral damage we thought we could take advantage
of the precision of the immune system, in particular, antibodies,” said David Scadden, MD, Co-director of HSCI,
the Gerald and Darlene Jordan Professor of Medicine at Harvard University, and senior author on the paper.
111
As part of the immune system, antibodies naturally seek and destroy foreign agents in the body. Rahul
Palchaudhuri, a postdoctoral fellow in Scadden’s lab and first author on the paper, armed CD45-targeting
antibodies with a payload that destroys only existing blood cells. The payload kills cells by means other than
genetic destruction, in contrast to the current standard treatments.
“Antibodies are remarkably specific in what they target,” said Palchaudhuri, a chemist by training, with a
background in cancer research. “We can direct them to CD45, a cell marker which is exclusively expressed in
the blood system. That way we avoid toxicities to non-blood tissues.”
Unlike chemotherapy and radiation – which indiscriminately damage cells and tissues, healthy or otherwise
– the CD45-targeting antibodies leave the thymus and the bone marrow, environments critical to the formation of T cells and innate immune cells, unharmed. Animals receiving the antibody treatment were able to
withstand infection that was lethal to mice treated with radiation. Currently, infections after transplant are
common and may be severe, causing death in a substantial number of people.
About one in ten patients do not survive transplantation following the standard treatments. Those who
do may suffer from stunted growth and intellectual development, infertility, and damaged DNA; at present,
patients can only attempt a curative transplant by increasing their risk of developing cancer later.
Because of this, families and doctors often shrink from transplant options, particularly when it comes to
treating children, and it will limit the extent to which the breakthroughs in gene therapy and gene editing will
be applied, explained Scadden, who is a practicing hematologist at MGH and chairman of Harvard’s Department of Stem Cell and Regenerative Biology.
Animals that received the antibody treatment had a broad ten-day window within which they could accept
a bone marrow transplant, and individuals that did not receive a bone marrow transplant were able to fully
recover without adverse effects. Furthermore, mice suffering from sickle cell anemia were successfully transplanted using the antibody method and cured of their anemia. Should the same hold true for humans, what
amounts to months of recovery in a hospital bed may be replaced by an outpatient procedure, and a failed
transplant would not be fatal.
“If this approach works in humans, it will really change the conversation that providers have with patients,”
Scadden said, especially for those “who have these underlying genetic disorders and for who the new geneediting and gene therapy techniques are being developed.”
The scientists are now trying to identify antibodies that would be effective in humans, and a company has
been formed to move the work towards translation and determine which models are most useful in a preclinical
setting.
“It brings precision medicine into the area of transplant in a way that hasn’t been there and is needed,”
Scadden said.
NewsRx LLC
(2016-06-20), New approach could make bone marrow transplants safer, Cancer Gene Therapy Week, 26,
ISSN: 1543-6837, BUTTER® ID: 011909049
NIH/Office of the Director
NIH awards $55 million to build million-person precision medicine study
By a News Reporter-Staff News Editor at Journal of Engineering – The White House will be issuing a fact
sheet announcing the awardees called The Precision Medicine Cohort Program Awards Announcement. It will
be followed up by press events tomorrow.
The National Institutes of Health announced $55 million in awards in fiscal year 2016 to build the foundational partnerships and infrastructure needed to launch the Cohort Program of President Obama’s Precision
Medicine Initiative (PMI). The PMI Cohort Program is a landmark longitudinal research effort that aims to engage 1 million or more U.S. participants to improve our ability to prevent and treat disease based on individual
differences in lifestyle, environment and genetics. The awards will support a Data and Research Support Center, Participant Technologies Center and a network of Healthcare Provider Organizations (HPO). An award to
Mayo Clinic, Rochester, Minnesota, to build the biobank, another essential component, was announced earlier
this year. All awards are for five years, pending progress reviews and availability of funds. With these awards,
NIH is on course to begin initial enrollment into the PMI Cohort Program in 2016, with the aim of meeting its
enrollment goal by 2020.
The PMI Cohort Program is one of the most ambitious research projects in history and will set the foundation
for new ways of engaging people in research. PMI volunteers will be asked to contribute a wide range of health,
environment and lifestyle information. They will also be invited to answer questions about their health history
112
and status, share their genomic and other biological information through simple blood and urine tests and grant
access to their clinical data from electronic health records. In addition, mobile health devices and apps will
provide lifestyle data and environmental exposures in real time. All of this will be accomplished with essential
privacy and security safeguards. As partners in the research, participants will have ongoing input into study
design and implementation, as well as access to a wide range of their individual and aggregated study results.
“This range of information at the scale of 1 million people from all walks of life will be an unprecedented
Director Francis S. Collins, M.D., Ph.D. “Over time, data provided by participants will help us answer important
health questions, such as why some people with elevated genetic and environmental risk factors for disease
still manage to maintain good health, and how people suffering from a chronic illness can maintain the highest
possible quality of life. The more we understand about individual differences, the better able we will be to
effectively prevent and treat illness.”
The knowledge gained from the PMI Cohort Program will extend successes of precision medicine in some
cancers to many other diseases. Importantly, the program will focus not just on disease, but also on ways to
increase an individual’s chances of remaining healthy throughout life.
“As someone who has personally benefited from precision medicine, I am excited for this study to intersect
with other fundamental changes in medicine and research to empower people to live healthier lives,” added
PMI Cohort Program Director Eric Dishman. “What potential participants need to know is that we are equally
interested in learning how we can prevent illness in the first place, but when we do get ill, which treatment
options are going to work best for each of us individually.”
These initial awards bring together the major elements through a variety of new partnerships that are
needed to launch the PMI Cohort Program later this year. “This is an incredibly complex study requiring new
kinds of strategic and operational partnerships – this can’t be business as usual,” said Kathy L. Hudson, Ph.D.,
NIH Deputy Director for Science, Outreach, and Policy who helped orchestrate the PMI Cohort Program. “We
are excited to break new ground in engaging people in research and building a study of this scale and scope.”
The infrastructure will be assembled with the following organizations and is intended to expand over time
as needed to support the growth of the cohort:
The Data and Research Support Center has been awarded to Vanderbilt University Medical Center,
Nashville, Tennessee, working with the Broad Institute, Cambridge, Massachusetts, and Verily Life Sciences
(formerly Google Life Sciences), Mountain View, California. This center will acquire, organize and provide secure access to what will be one of the world’s largest and most diverse datasets for precision medicine research.
They will also provide research support for the scientific data and analysis tools for the program, helping to
build a vibrant community of researchers from community colleges to top healthcare research institutions and
industries, and including citizen scientists, who can propose studies using this information.
Enrollment of PMI Cohort Program participants will be through two distinct approaches. One leverages the
strengths of HPOs that have existing relationships with potential participants, and the other will be through
the Participant Technologies Center, which will support direct enrollment. The Participant Technologies Center has been awarded to the Scripps Research Institute, San Diego, and Vibrent Health, Fairfax, Virginia.
The center will also develop, test, maintain and upgrade, as needed, PMI Cohort Program mobile applications.
These mobile apps will be used to enroll, consent, collect data from and communicate with PMI Cohort Program
participants. Importantly, the center will need to develop parallel platforms to deliver these same functions to
those without smartphones, and work with various technology organizations to increase smartphone accessibility.
NIH will build a network of HPOs over time to ensure that participants in the research represent the geographic, ethnic, racial and socioeconomic diversity of the country. The network will include regional and
national medical centers, community health centers and medical centers operated by the U.S. Department of
Veteran Affairs (VA). The following organizations have been selected as the initial set of HPOs with another
funding opportunity in the coming months. These HPOs will engage their patients in the PMI Cohort Program,
help build the research protocols and plans, enroll interested individuals and collect essential health data and
biological specimens. The regional medical centers are:
These awardees have sub-awards with organizations that extend the geographic reach of the HPO network.
In addition, NIH collaborated with the Health Resources & Services Administration (HRSA) to select six Federally Qualified Health Centers (FQHCs), which are community-based HPOs that reach underserved areas and
populations. This award supports a pilot program to determine infrastructure needs that will enable a wide
variety of FQHCs to participate as HPOs. FQHCs will be critical for bringing underserved individuals, families and communities into the cohort, especially those historically underrepresented in biomedical research.
Recipients are:
113
Another significant and important partner in the HPO network is the VA, which has medical centers across
the United States that provide care to America’s veterans. By collaborating with the VA, the NIH will ensure
America’s former servicemen and women have the opportunity to participate in the PMI Cohort Program. The
VA Healthcare System serves a demographically and geographically diverse population, which strengthens
NIH’s capacity to enroll people of all races and socioeconomic backgrounds in the cohort. The VA will leverage
the experience and infrastructure gained from its Million Veteran Program, which partners with U.S. veterans
receiving care at VA medical centers to study how genes affect health, to help enroll veterans in the PMI Cohort
Program.
In May of 2016, Mayo Clinic was awarded the task of building the PMI Cohort Program Biobank. The
biobank will support the collection, analyses, storage and distribution for research use of biological samples
known as biospecimens. Data from laboratory analyses of biospecimens will be combined with an array of
other lifestyle and health information provided by volunteers to help researchers continue to unravel individual
differences that contribute to disease and response to treatments.
NewsRx LLC
(2016-07-25), NIH awards $55 million to build million-person precision medicine study, Journal of Engineering, 790, ISSN: 1945-872X, BUTTER® ID: 012094818
Health Outcomes Sciences
Precision Medicine Solutions Made Easier and More Affordable to Healthcare Providers
By a News Reporter-Staff News Editor at Cardiovascular Week – Health Outcomes Sciences (HOS), serving
hospitals and health systems across the country, has achieved new milestones in its journey to deliver personalized predictive analytics for cardiovascular patients. As a provider of online healthcare technology solutions,
the month of June 2016 marks the company’s release of its patented next generation ePRISM® platform along
with brand new subscription plans that make adoption of its precision medicine solutions easier and more
affordable.
com/news/home/20160613005051/en/ Dr. John Spertus explains how a predictive model is used in the
ED for stroke patients. “We’re seeing a groundswell of interest in the use of predictive analytics to support
personalized care and shared decision-making,” says Dr. John Spertus, HOS co-founder and Chief Scientific
Officer. “However, we know that cost can be a barrier to rapid adoption, as can the IT burden associated with
implementing new technology. That’s why we’ve made it possible for hospitals and health systems to be up
and running with our next generation ePRISM® platform in just a few days’ time, and they can use any of the
predictive models in our library for just a few hundred dollars a month.”
The company’s new ePRISM® Level 1 and Level 2 options offer hospitals and health systems their own
private online web portal along with a subscription to any predictive model in the company’s library. Differences
between the two options include the number of user logins, the ability to save data and download it for localized
use, and the opportunity for branding with a logo. Both options include no-cost educational webinars and
US-based phone support. Although the new ePRISM® Level 1 and Level 2 options reduce the operational
requirements of a major IT project, the company will continue to offer a more robust option that fully integrates
with all major electronic medical record (EMR) systems.
The company’s partnership with the American College of Cardiology (ACC) has contributed to a strong track
record of client success in cardiovascular care. Its suite of predictive models for percutaneous coronary intervention (PCI) and implantable cardioverter defibrillator (ICD) are derived directly from ACC NCDR® research,
and are regarded as the gold standard. Within the next few months, the company will expand its library to
include suites of predictive models for orthopedics and oncology, expressly in support of CMS initiatives addressing Comprehensive Care for Joint Replacement (CJR) and Screening for Lung Cancer with Low Dose
Computed Tomography.
Spertus offers, “Thousands of predictive models have been published in scientific annals, but HOS is careful
to select only those that offer high impact to patient outcomes. We work closely with clinical care teams to
embed the predictive models seamlessly into routine workflow.”
Earlier this year, HOS added new predictive models to its library with the assistance of Saint Luke’s Health
System in Kansas City. Spertus describes in this video how one of the models is being used at Saint Luke’s
Hospital as part of the RESOLVE research study, an American Heart Association (AHA) initiative that seeks
to improve outcomes for stroke patients. The company worked with Drs. Arnold and Cohen at Saint Luke’s Mid
114
America Heart Institute to launch a model that addresses outcomes for transcatheter aortic valve replacement,
or TAVR (otherwise known as transcatheter aortic valve implantation, or TAVI). The company is currently
offering TAVR/TAVI teams around the world access to the model via a free online risk calculator.
“Our solutions play a pivotal role in healthcare quality management,” Spertus explains. “The Wall Street
Journal recently reported on the prevalence of medical errors, and the toll on lives lost. Personalized precision medicine can help overcome that growing epidemic.” About Health Outcomes Sciences Health Outcomes
Sciences is propelling the practice of precision medicine through its patented content enablement platform,
ePRISM®, which translates scientific models into automated evidence-based decision support solutions. By
helping health and life science organizations – as well as allied technology partners – deliver the power of
personalized predictive science at the point of care, the company facilitates significant and measurable improvements in clinical and financial outcomes, variations in care, appropriate use and rational consumption of
resources. The company’s solutions are specialty agnostic, scalable at the enterprise level and employ a cloudhosted software-as-a-service (SaaS) business model. For additional information on Health Outcomes Sciences,
visit www.h-outcomes.com. View source version on businesswire.com: http://www.businesswire.com/
news/home/20160613005051/en/
NewsRx LLC
(2016-06-27), Precision Medicine Solutions Made Easier and More Affordable to Healthcare Providers, Cardiovascular Week, 69, ISSN: 1543-6845, BUTTER® ID: 011945928
Thermo Fisher Scientific Inc.
Thermo Fisher Scientific and West China Hospital Partner on Joint Research Platform for
Precision Medicine
By a News Reporter-Staff News Editor at Journal of Engineering – Thermo Fisher Scientific Inc. (NYSE: TMO),
the world leader in serving science, announced a partnership with West China Hospital of Sichuan University,
one of the largest single-site hospitals in the world, to develop a joint platform to research precision medicine.
The partners shared the news during the seventh round of the China-U.S. High-Level Consultation on Peopleto-People Exchange (CPE) held in Beijing on June 6-7.
“Our company mission - to enable our customers to make the world healthier, cleaner and safer - is perfectly
aligned with the priorities in China’s 5-year plan,” said Marc N. Casper, president and chief executive officer of
Thermo Fisher. “We share a commitment to advancing precision medicine, and we’re excited about strengthening our collaboration with West China Hospital to provide technologies and expertise that will ultimately
improve patient care.”
“We are exploring opportunities to collaborate with renowned, industry-leading international companies to
harness and capitalize on our strength in pathology,” said Li Weimin, president of West China Hospital. “With
our partners at Thermo Fisher, we are working to enhance the quality of pathological research and clinical
diagnosis. We are also exploring an opportunity to extend our partnership in Western China by accelerating
the development of precise pathological diagnoses that will ultimately help improve people’s lives.”
Richard Stengel, U.S. undersecretary of state for public diplomacy and public affairs, said, “In recent years,
we have witnessed several achievements resulting from public and private partnerships between the U.S. and
China - all of which aim to improve the well-being of people in both countries. The collaboration between
Thermo Fisher and West China Hospital is a perfect example of America’s efforts to strengthen ties with China
in science, technology and health. The agreement reinforces the spirit and goals of the CPE.”
The two parties have agreed to collaborate closely to make West China Hospital a leading global molecular
diagnosis center. The joint efforts include construction of training bases for standardized pathology qualitycontrol and standardized pathologic diagnosticians and the development of advanced laboratories with efficient, safe and integrated business procedures. About West China Hospital of Sichuan University West China
School of Medicine/West China Hospital of Sichuan University (WCSM/WCH) is a prestigious medical center
located on the banks of the Jinjiang River in Chengdu, the capital of Sichuan province and one of China’s
most famous historical and cultural cities. WCH is one of the largest single-site hospitals in the world and
a leading medical center of West China, treating complicated and severe cases, especially in the fields of living donor liver transplantation, severe acute pancreatitis, and clinical anesthesia. WCH aims to become the
national center for medical scientific research and technology innovation. It has one State Laboratory, two
ministry-level key laboratories, one Cooperative Research Centre online system under the Ministry of Education, and 26 open laboratories. Since 2009, the hospital has been ranked second on the China Best Hospital
115
List on three occasions. In 2011, it was ranked 3rd in the Most Popular 3A Hospital List and 2nd in the
Top 50 Chinese public general hospitals by measure of its social contribution. In 2007 and 2008, WCH won
Golden Awards for hospital management from the industry body Hospital Management Asia. About Thermo
Fisher Scientific Thermo Fisher Scientific Inc. is the world leader in serving science, with revenues of $17
billion and more than 50,000 employees in 50 countries. Our mission is to enable our customers to make the
world healthier, cleaner and safer. We help our customers accelerate life sciences research, solve complex analytical challenges, improve patient diagnostics and increase laboratory productivity. Through our premier
brands - Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific and Unity Lab Services - we
offer an unmatched combination of innovative technologies, purchasing convenience and comprehensive support. For more information, please visit www.thermofisher.com. View source version on businesswire.com:
NewsRx LLC
(2016-06-27), Thermo Fisher Scientific and West China Hospital Partner on Joint Research Platform for
Precision Medicine, Journal of Engineering, 1569, ISSN: 1945-872X, BUTTER® ID: 011953764
West China Hospital of Sichuan University
The Seventh Annual US-China High-Level Consultation on People-to-People Exchange was
held in Beijing
By a News Reporter-Staff News Editor at Asia Business Newsweekly – The seventh annual US-China HighLevel Consultation on People-to-People Exchange (CPE) was recently held in Beijing. Over 200 representatives
from CPE members of both sides and other participating organizations attended the meeting. The consultation
between both sides covered the fields of education, science & technology, culture, health, sports, and women.
Different programs implemented in the CPE framework were concluded and planned.
http://photos.prnewswire.com/prnvar/20160706/386977
Among the 135 topics for which common understandings have been reached during this round of consultation, 6 representative ones were chosen to be included in the signing ceremony, and the joint precision medical
platform built together by West China Hospital, Sichuan University and Thermo Fisher Scientific was one of
them. This signature agreement is regarded as a part of cooperation and exchange activities between China
and the US, which will help promote the development of precision medicine and pathological studies in western
China and benefit the public.
In the morning of June 6, Li Weimin, President of West China Hospital, Sichuan University and Gianluca
Pettiti, Thermo Fisher Scientific country president of China, signed a cooperation memorandum for building
the joint precision medical research platform in Beijing Westin Hotel. West China Hospital, Sichuan University
and Thermo Fisher Scientific will cooperate comprehensively in a number of fields and make joint efforts to
create a world leading molecular diagnosis center for the hospital. The specific cooperation programs include
building a joint modern clinical pathological laboratory, establishing a hospital-industry cooperative platform
for promoting tissue bank, proteomics, molecular diagnosis, and pathological quality control, creating a professional and modern pathological diagnostician training center, and setting up efficient, safe, and integrated
business flows.
It is reported that as early as December 1, 2015, the joint modern clinical pathological laboratory built by
West China Hospital, Sichuan University and Thermo Fisher Scientific was unveiled. In addition to serving
scientists and postgraduate students from hospitals and scientific research organizations affiliated to Sichuan
University, it will provide scientific research services for the public.
According to President Li Weimin, West China Hospital, Sichuan University is seeking close cooperation
with leading scientific & technical enterprises across the globe to reinforce its advantages in pathology. Cooperation with Thermo Fisher Scientific is expected to improve quality of pathological study and clinical diagnosis. Such cooperation will continue to expand to accelerate development of precision pathological diagnosis in
western China and to benefit health of the public.
Photo - http://photos.prnewswire.com/prnh/20160706/386977 .
SOURCE West China Hospital of Sichuan University
Photo: http://photos.prnewswire.com/prnh/20160706/386977
NewsRx LLC
(2016-07-19), The Seventh Annual US-China High-Level Consultation on People-to-People Exchange was
held in Beijing, Asia Business Newsweekly, 68, ISSN: 1938-1808, BUTTER® ID: 012070159
116
University of Illinois at Chicago
UIC to enroll participants in president’s Precision Medicine Initiative
By a News Reporter-Staff News Editor at NewsFile – The University of Illinois at Chicago, Northwestern University, the University of Chicago and their affiliated hospitals and clinics have been selected to enroll 150,000
Illinoisans in the national Precision Medicine Initiative Cohort Program. The Illinois consortium is one of several such groups across the nation that will help bring one million or more U.S. participants over the next five
years into a research effort to improve the prevention and treatment of disease based on individual differences
in lifestyle, environment and genetics.
The Illinois Precision Medicine Initiative Cohort Program consortium will receive $4.3 million in fiscal year
2016 and a total of approximately $45 million over 5 years pending progress and availability of funds from the
National Institutes of Health to meet its participant enrollment goal.
The Precision Medicine Initiative, announced by President Obama in his 2015 State of the Union address,
launched in 2016 with a $215 million budget. It aims to enable a new era of medicine in which researchers,
providers and research participants work together to develop individualized care.
Most prevention strategies and medical treatments are designed for the average patient. This one-size-fitsall approach means that strategies to stay healthy and treatments for illnesses are successful for some people,
but not others.
Precision medicine is an emerging approach for disease treatment and prevention that takes into account
individual variability in genes, environment and lifestyle for each person. While some advances in precision
medicine have been made, the practice is not currently commonplace.
“Illinois, as just a single state, very closely resembles the rest of the U.S. combined because we have such
diversity in terms of our populations and types of communities,” says Dr. Robert Winn, associate vice chancellor
for community based practice at the University of Illinois Hospital & Health Sciences System, director of the
University of Illinois Cancer Center and a principal investigator on the grant.
Dr. Robert Barish, vice chancellor for health affairs at UIC, says that “for precision medicine to work for
everyone, we need to know how prevention strategies, medicines and therapies work in a broad range of populations. With UIC’s unique ability to reach diverse communities through our clinics throughout Chicago, and
through our regional College of Medicine campus in Peoria, we are well-positioned to help answer the questions
that will help bring precision medicine to everyone.”
UI Health’s 13 Mile Square Health Centers are federally qualified neighborhood clinics located in some of
Chicago’s most underserved communities, said Winn.
“Our Mile Square Health Centers reach into communities that carry an exceptionally heavy burden of disease,” said Winn, noting they serve African-American neighborhoods on the city’s south and west sides where
cancer, diabetes, heart disease and asthma rates can be more than twice as high as in other populations.
Volunteer participants in the Precision Medicine Initiative Cohort Program will be asked to contribute a
wide range of health, environment and lifestyle information. The participants will also answer questions about
their health history and status, share their genomic and other biological information through simple blood
and urine tests, and grant access to their clinical data from electronic health records. In addition, mobile
health devices and apps will provide lifestyle data and environmental exposures in real time. All this will be
accomplished with essential privacy and security safeguards. As partners in the research, participants will
have ongoing input into study design and implementation, as well as access to a wide range of their individual
and aggregated study results.
The knowledge gained from the Precision Medicine Initiative Cohort Program will extend the success of
precision medicine in some cancers to many other diseases. Importantly, the program will focus not just on
disease, but also on ways to increase an individual’s chances of remaining healthy throughout life.
“The range of information at the scale of one million people from all walks of life will be an unprecedented
Director Dr. Francis Collins.
Dr. Martha Daviglus and Denise Hynes of the UIC College of Medicine are co-principal investigators on
the grant. Dr. Jerry Krishnan, Maria Argos, Dr. Frederick Behm, Dr. Ben Gerber, Timothy Johnson, Robin
Mermelstein, Dr. Terry Vanden Hoek and Karriem Watson of the UIC College of Medicine and Marcelo Bento
Soares of the University of Illinois at Peoria College of Medicine are co-investigators on the grant.
UIC will coordinate efforts to enroll individuals at UI Hospital; Mile Square Health Center; University
of Illinois at Peoria College of Medicine; Southern Illinois University Healthcare; Memorial Medical Center,
Springfield; Blessing Health System, Quincy; Sarah Bush Lincoln Health Center, Matoon; OSF Saint Francis
Medical Center, Peoria; Cook County Health and Hospitals System; and Mount Sinai Hospital, Chicago.
117
NewsRx LLC
(2016-07-25), UIC to enroll participants in president’s Precision Medicine Initiative, NewsFile, 164, ISSN:
0000-0000, BUTTER® ID: 012091674
Atrial Fibrillation
UK first heart operations using novel system at Leicester
By a News Reporter-Staff News Editor at Computer Weekly News – The UK’s first heart operations using a
novel software platform to pinpoint the source of the heart condition have been carried out in Leicester thanks
to research at the University of Leicester.
Professor Andre Ng, Professor of Cardiac Electrophysiology at the University of Leicester and Consultant
Cardiologist and Electrophysiologist at Leicester’s Hospitals, has carried out three operations since November
2015.
The patients suffered from a condition known as atrial fibrillation (AF) – the commonest heart rhythm
disturbance affecting more than 1 million people in the UK.
All three patients have now returned home following the operations which were completed successfully.
AF is a condition that causes the upper chambers of the heart (atria) to beat very fast and irregularly due
to chaotic electrical activity. As a result the atria do not beat in an organised way and pump less efficiently,
increasing the likelihood of stroke and heart failure.
Electrical activity in the heart is an area of specialist research at the University of Leicester, spearheaded
by Professor Ng and his research team in the University’s Department of Cardiovascular Sciences. Professor
Ng has been researching this field for several years and this latest technique that he is applying on a patient
will help to further enhance his research.
Professor Ng said the UK first at Leicester exemplifies how research at the University of Leicester was
providing benefits for patients thanks to the partnership between the University and Leicester’s Hospitals and
the support of the NIHR Leicester Cardiovascular Biomedical Research Unit.
Commenting on the heart condition known as AF, Professor Ng said: “Initial treatment for AF is with
medication to control the heart rate or reduce AF episodes using drugs. In many patients, AF fails to be
controlled by medication and continues to cause debilitating symptoms.
“Catheter ablation has been increasingly used in patients with AF over the past decade or so. The procedure involves inserting electrical wires called catheters into the heart to ablate (or ‘burn’) the abnormal areas
in the heart that are causing or sustaining AF. In patients who are in an early phase of the condition, this
procedure has been shown to be reasonably effective requiring limited burning in well-defined heart regions
(e.g. pulmonary veins).
“However, the results of AF ablation in patients with more advanced form of the condition is less than ideal
with variable results and patients often need to undergo several procedures with suboptimal long term outcome.
The conventional approach involves extensive ablation on many different atrial locations due to the seemingly
chaotic electrical activity seen. More recent data support the presence of focal or rotational sources (or rotors)
which may be driving the chaotic process.”
Describing his research and the technique that he deployed for the first time in the UK, Professor Ng added:
“Topera is a new software platform developed to “decode” the chaotic electrical signals and represent the activity
in the form of rotor maps – allowing us to see the rotors and the centres of rotation analogous to the “eye of the
storm”.
“The location of these rotors are different in different patients and hence this new software platform allows
a personalised or precision approach to target localised sources for ablation rather than having to ablate over
a wide area in the atrial chambers. The initial results from studies conducted in USA and some European
centres e.g. Germany are promising and suggest better efficacy than the conventional approach with extensive
ablation.
“We are very pleased to have used this new system at Leicester for the first time in UK. We managed to
use the Topera system to analyse the electrical activity during AF in our patients. We are very pleased that
AF stopped when we did the ablation and the patients returned to normal rhythm, which was the best possible
result and desired procedural outcome for our patients.
“This approach is related to our ongoing research at the University of Leicester aimed at understanding
the substrate underlying recurrent persistent atrial fibrillation. The map generated with the Topera system is
quite unique and there is much research to be done to fully understand the different behaviour of these maps
in different patients. Having access to this new software algorithm allows us to examine the response to AF
118
ablation using this approach in our patients first hand. It is hoped that we will achieve better results with more
focussed targets and therefore less ablation required.
“At the University of Leicester and Leicester’s Hospitals, we are very pleased to be the first UK centre, based
at Glenfield Hospital, with access to this new technology. AF ablation in patients with more advanced form of
the condition is challenging using the current approach, often needing extensive ablation.
“This new software platform uses mathematical processing techniques to ‘decode’ the chaotic behaviour to
reveal the underlying focal or rotational activity which are believed to be the driver of the rhythm disturbance.
Hence it is hoped that targeting these localised critical circuits would lead to better results and less ablation
required which should be translated to better patient outcome.
“The variable results from the current approach to AF ablation highlights that there is a significant component of individual difference between patients which needs a personalised or precision medicine approach to
identify the unique characteristics in the particular patient that we are treating. This new platform appears
to be able to go some way towards this aim and we are very keen to be able to establish more evidence with
future specific research studies at the University.”
NewsRx LLC
(2016-06-01), UK first heart operations using novel system at Leicester, Computer Weekly News, 770, ISSN:
1944-1606, BUTTER® ID: 011785191
Cancer Prevention
University of Maryland Greenebaum Cancer Center earns NCI’s highest designation
By a News Reporter-Staff News Editor at VerticalNews Health & Science – BALTIMORE - . The University of
Maryland Marlene and Stewart Greenebaum Cancer Center has been awarded the National Cancer Institute’s
highest designation as a Comprehensive Cancer Center. The prestigious distinction recognizes the cancer center’s high caliber of scientific leadership and robust programs in basic, clinical and population science research,
placing it in the top tier of cancer centers nationwide. The new name of the center is the University of Maryland
Marlene and Stewart Greenebaum Comprehensive Cancer Center (UMGCCC).
The cancer center was granted NCI-designated Cancer Center status in 2008 and applied last fall to become
a Comprehensive Cancer Center. NCI awarded the center the new designation after a rigorous review, which
included a three-day site visit by 22 NCI reviewers in late February. The reviewers cited the cancer center’s
“impressive progress” over the past five years and rated the center “outstanding.” The new designation goes
into effect at the start of the cancer center’s next grant cycle August 1.
“We are extremely proud to have met the NCI’s exacting standards to be recognized as a Comprehensive
Cancer Center and to be ranked in the very top echelon of cancer centers in the country,” says Kevin J. Cullen,
MD, the Marlene and Stewart Greenebaum Distinguished Professor of Oncology at the University of Maryland
School of Medicine and the cancer center’s director. “This designation is a tremendous achievement for our
entire team and will significantly enhance our ability to translate discoveries in the laboratory into better
treatments for cancer patients in Maryland and beyond.”
The Greenebaum Comprehensive Cancer Center is one of only 46 NCI-designated Comprehensive Cancer
Centers in the United States. There are a total of 69 NCI-designated Cancer Centers in 35 states and Washington, D.C.
“The Greenebaum family could not be more pleased that the Marlene and Stewart Greenebaum Cancer Center has achieved this important milestone,” says Michael Greenebaum, President, Greenebaum Enterprises,
Inc. “To see the cancer center reach the highest echelon under the direction of Dr. Cullen is truly a dream come
true for my Mom and Dad.”
As a result of the new designation, the cancer center’s grant will increase 50 percent, to $1.5 million, and
the center will be eligible for other funding from the NCI and other public and private sources.
“We have made significant strides in expanding our basic and clinical research to include a strong population
science program to help reduce disparities in both cancer treatment and prevention that threaten the health of
minority populations,” Dr. Cullen says. “About 33 percent of the patients who take part in our clinical trials are
African-American, reflecting our cancer center’s unique position and mission to involve the minority community
in state-of-the-art clinical and translational research.”
Dr. Cullen adds that the cancer center has also developed a comprehensive education and training program
to educate the next generation of clinicians and scientists.
NCI Comprehensive Cancer Centers have comprehensive, well-integrated programs in population health,
education and cancer prevention as well as outstanding basic, clinical and translational research programs.
119
Maryland Gov. Larry Hogan, who was treated for Stage III non-Hodgkin lymphoma at the cancer center
in 2015, has advocated for the center’s efforts to achieve Comprehensive Cancer Center status. “Our state
takes enormous pride in the Greenebaum Cancer Center’s accomplishments and commitment to helping reduce
cancer risks, increase access to care and improve the health of all Marylanders,” Mr. Hogan says. “The cuttingedge research being conducted at the cancer center has changed the ways cancer is treated, not only here in
Maryland but around the world.
“Personally, I could not be more grateful to have been the recipient of the outstanding medical care that the
center is known for,” Mr. Hogan says. “And it is because of this expert and compassionate care, combined with
a lot of support and prayers, that I am proud to say that I am now in complete remission and cancer-free.”
“This is a significant achievement for the Greenebaum Cancer Center, and one that perfectly reflects the
research-intensive ethos and culture of the School of Medicine,” says E. Albert Reece, MD., PhD, MBA, vice
president for medical affairs at the University of Maryland and the John Z. and Akiko K. Bowers Distinguished
Professor and dean of the University of Maryland School of Medicine. “With our commitment to discovery-based
medicine, this designation further supports the culture and research productivity of our faculty in developing
major breakthroughs in cancer that will benefit patients in our community and around the world.”
“This designation reflects the commitment to scientific discovery, precision medicine and cancer prevention
that makes the Greenebaum Comprehensive Cancer Center a world-class institution, known for its innovative
research but also its compassionate patient care,” says Jay A. Perman, MD, president of the University of
Maryland, Baltimore (UMB). “Faculty members at the School of Nursing and other professional schools at
UMB work very closely with the cancer center on a number of major research initiatives, including exploring
methods to eradicate debilitating cancer-related pain.”
The UMGCCC is part of both the University of Maryland School of Medicine and the University of Maryland Medical Center. All of the physicians and the majority of the basic scientists are employees and faculty
members of the School of Medicine. The cancer center also is at the heart of the University of Maryland Cancer
Network, which includes cancer centers at several community hospitals in the University of Maryland Medical
System (UMMS) - the University of Maryland St. Joseph Medical Center, the University of Maryland Upper
Chesapeake Medical Center, and the University of Maryland Baltimore Washington Medical Center.
“This designation marks a significant milestone for the Greenebaum Cancer Center, further advancing
the extraordinary levels of clinical services and research available to the people of Maryland and the region,”
says Robert A. Chrencik, president and chief executive officer of the University of Maryland Medical System.
“Through the University of Maryland Cancer Network, our affiliated cancer centers treat Marylanders with the
innovative and outstanding care they would expect from an academic cancer center - but closer to their homes.”
NCI-designated Cancer Centers are the backbone of the NCI’s programs to study and control cancer. About
three-quarters of NCI’s grants for investigator-initiated research are awarded to NCI-designated Cancer Centers, and many new therapies are available to patients as part of clinical trials. Studies have shown that
patients treated at NCI-designated Cancer Centers have increased survival rates.
NewsRx LLC
(2016-06-19), University of Maryland Greenebaum Cancer Center earns NCI’s highest designation, NewsRx
Health & Science, 17, ISSN: 1944-2602, BUTTER® ID: 011858685
120
Chapter 9
Oncology
Amoy Diagnostics Co, Ltd.
AmoyDx ROS1 Diagnostics Test Contributed to a Phase 2 Clinical Study of Crizotinib in
Lung Cancer
By a News Reporter-Staff News Editor at Clinical Trials Week – Amoy Diagnostics Co, Ltd. (AmoyDx), a
leading provider of cancer molecular diagnostic products and services, announced that its AmoyDx® ROS1 test
successfully supported OxOnc Development LP (“OxOnc”) in the completion of a Phase 2 study of crizotinib (the
“OxOnc Study”) in East Asian patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC). The
OxOnc Study met its primary objective of demonstrating a high overall response rate for crizotinib. It enrolled
127 ROS1-positive patients in the People’s Republic of China (PRC), Japan, Taiwan and South Korea by using
AmoyDx® ROS1 gene fusions test, and is the largest ROS1-positive NSCLC cohort reported to date. Results
from the OxOnc Study were presented at the recent 2016 American Society of Clinical Oncology (ASCO) Annual
Meeting. ( http://oxonc.com/pdfs/OxOncRelease020713.pdf ). ROS1 gene rearrangements occur in
about 2.4 percent of Asian patients with NSCLC. An estimated 1.5 million new cases of NSCLC are diagnosed
worldwide each year.
“We expect NSCLC patients with ROS1 translocations to benefit significantly from the OxOnc Study results. AmoyDx aims to provide our customers with superior and innovative products and services to improve
healthcare and patient well-being, and in particular, contribute to the development of new cancer therapies
and precision medicine,” said Dr. Zheng, AmoyDx’s CEO and President.
“We are pleased to collaborate with AmoyDx, and appreciate the excellent performance of the ROS1 diagnostic test which was critical to the success of our study completion,” said Dr. Wenn Sun, OxOnc’s Managing
Partner. In February 2013, OxOnc announced that it had entered into a co-development agreement with Pfizer
to conduct the OxOnc Study. ( http://oxonc.com/pdfs/OxOncRelease020713.pdf )
AmoyDx has obtained CE-mark for its ROS1 kit detecting 14 ROS1 gene fusions in Europe in November
2013 and received Chinese FDA (CFDA) approval in September 2014. This highly sensitive assay uses realtime PCR technology to analyze tumor messenger RNA from human tumor tissue or tumor cells and can derive
results within 2.5 hours. For NSCLC precision medicine, AmoyDx also provides EGFR, KRAS, BRAF, ALK,
ALK/ROS1, EGFR/ALK/ROS1 detection kits, which are both CE certified and CFDA approved. About AmoyDx:
Amoy Diagnostic Co., Ltd. is an R&D based provider of state-of-the-art companion diagnostic products and
services in the area of human precision oncology. Its customers include healthcare providers, pharmaceutical
companies and academic institutions. The company has a rich portfolio of oncology molecular testing products
which can be used for the detection of gene mutation/fusions of different cancer types including but not limited to
lung cancer, colorectal cancer, breast cancer, cervical cancer and melanoma. AmoyDx is one of market leaders in
the field of oncology diagnostics for precision medicines and its products have been widely used in more than 40
countries.(www.amoydx.com) View source version on businesswire.com: http://www.businesswire.com/
news/home/20160617005218/en/
NewsRx LLC
(2016-07-04), AmoyDx ROS1 Diagnostics Test Contributed to a Phase 2 Clinical Study of Crizotinib in Lung
Cancer, Clinical Trials Week, 178, ISSN: 1543-6764, BUTTER® ID: 011985383
121
University of Michigan Health System
Bare Your Blues for Father’s Day
By a News Reporter-Staff News Editor at Cancer Weekly – Does your dad or husband need another tie for
Father’s Day? Probably not.
Experience the interactive Multimedia News Release here: http://www.multivu.com/players/
English/7815451-blue-boxer-prostate-cancer/
This year, give the gift of making a difference and take on prostate cancer by baring your blues and supporting the Blue Boxer Fund for prostate cancer research.
One in seven men will hear the words “it’s prostate cancer” in his lifetime. While research progress has been
made, prostate cancer remains the second deadliest cancer among men.
With your support, we can take on prostate cancer and give the men in our lives hope. Hope for new treatments, as well as detection and prevention strategies that pave the way for a long, healthy life.
Inspired to make a difference, a group of patients, including Gary Andrus and John Loussia, established
the Blue Boxer Prostate Cancer Research Fund at the University of Michigan Comprehensive Cancer Center.
Using blue boxers as a symbol of solidarity in the face of prostate cancer, the goal is to beat the disease through
accelerated research and discovery at U-M with one of the world’s top prostate cancer research teams.
Using a pioneering approach to cancer called precision medicine, U-M’s prostate cancer team is identifying
new methods to determine the unique and complex nature of each instance of the disease. This will transform
the way prostate cancer is treated around the world.
Scientists use each tumor’s genomic profile to identify the specific gene mutations that started the disease,
the way the particular genes are clustered, and the stage and rate of the cancer’s progression. Researchers are
also studying the specific biological events that fuel variations in the cancer’s behavior.
Building on these efforts, U-M’s multidisciplinary experts have undertaken an ambitious and comprehensive
research initiative to distinguish between different kinds of prostate cancers; to develop targeted therapies that
precisely attack them at the molecular level; and to bring the vision of personalized oncology to fruition within
the next decade.
Now, we are asking you to join us and Absopure, who has committed to match dollar-for-dollar all donations
made to the Blue Boxer Fund from Wednesday, June 15, through Monday, June 20, 2016. Every contribution no matter the size - matters as we raise awareness and much-needed funds for prostate cancer research.
This Father’s Day, consider donating to support groundbreaking research, share with friends, or launch
your own online fundraising page TODAY to benefit the Blue Boxer Fund.
Don’t be modest ... bare YOUR blues! Together, we can take on – and uncover cures for – prostate cancer.
NewsRx LLC
(2016-06-28), Bare Your Blues for Father’s Day, Cancer Weekly, 37, ISSN: 1532-4567, BUTTER® ID:
011958479
122
Pediatric Cancer
Data on Pediatric Cancer Described by Researchers at Cancer Hospital (Biorepository for
Pediatric Cancer with Minimal Resources: Meeting the Challenges)
By a News Reporter-Staff News Editor at Pediatrics Week – Current study results on Pediatric Cancer have
been published. According to news reporting originating from Cairo, Egypt, by VerticalNews correspondents,
research stated, “Conducting high throughput -omics research requires high quality, data-rich biospecimens
to unravel factors underlying childhood cancers; this is an extra burden in a limited resources country. For
this purpose, Children’s Cancer Hospital (CCHE), the largest pediatric cancer hospital worldwide, established
a cutting-edge Biorepository and Biospecimen Research Facility (CCHE-BBR).”
Our news editors obtained a quote from the research from Cancer Hospital, “To present a step-by-step
guide to establishing a hospital-based biorepository with limited resources, and working in collaboration with
different hospital facilities to supply the research community with high quality data-rich biospecimens fit for
a wide range of research purposes. This approach will foster research in the era of personalized precision
medicine. CCHE-IRB approved the collection and storage of biospecimens from patients and parents for future
research. We focused on staff training, recruiting qualified scientists, and establishing the infrastructure. The
CCHE Biorepository developed strict standardized procedures for sample acquisition, processing, annotation,
storage, and distribution based on ISBER Best Practices and CAP-accreditation guidelines. We collect samples
at different clinical time points (e.g., at remission and/or relapse) as well as parents’ samples for genetic studies.
Using CaTissue®, an electronic storage management system, allowed sample annotation and full integration
with clinical data and the cancer registry. In 2 years, we succeeded in establishing a well-designed biorepository
within our regulations, bylaws, and SOPs, and with a minimal budget. We store high quality blood derivatives,
CSF, and malignant/normal tissue samples. Building a high quality biorepository with minimal-resources to
encourage research is possible.”
According to the news editors, the research concluded: “Having the suitable infrastructure with a significant
number of clinically annotated samples can play a major role in international research projects, sharing samples
and/or data with other groups.”
For more information on this research see: Biorepository for Pediatric Cancer with Minimal Resources: Meeting the Challenges. Biopreservation and Biobanking , 2016;14(1):9-16. (Mary Ann Liebert,
Inc. - www.liebertpub.com; Biopreservation and Biobanking - http://www.liebertpub.com/overview/
biopreservation-and-biobanking-formerly-cell-preservation-technology/110/ )
The news editors report that additional information may be obtained by contacting R.M. Labib, 1 Biorepository and Biospecimen Research, Children’s Cancer Hospital , Cairo, Egypt. Additional authors for this research
include M.M. Mostafa, A.S. Alfaar, D. Yehia, M. Alaa, M.G. Elzayat, M. Adel, S. Ezzat and S. Abo El-Naga.
NewsRx LLC
(2016-07-16), Data on Pediatric Cancer Described by Researchers at Cancer Hospital (Biorepository for
Pediatric Cancer with Minimal Resources: Meeting the Challenges), Pediatrics Week, 36, ISSN: 1944-2645,
BUTTER® ID: 012054668
University of Manchester
European cancer scientists converge on Manchester to share latest research
By a News Reporter-Staff News Editor at Biotech Business Week – Cancer experts from around Europe will
converge on Manchester this weekend (July 9-12) to share expertise with the city’s world-leading scientists at
a special Congress.
Organisers say Manchester was chosen to host the European Association for Cancer Research because it is
a global centre of excellence for cancer research.
The Congress, which forms part of Manchester City of Science, will see experts share work spanning from
basic research to precision medicine - where treatments can be tailored towards individual patients’ differing
tumours.
Scientists from Cancer Research UK Manchester Institute and The University of Manchester will be
amongst those showcasing their work.
European Association for Cancer Research President Professor Richard Marais, who is also Director of
Cancer Research UK Manchester Institute, based at The University of Manchester, said the Congress was an
important event for the city.
123
Professor Marais said: “Manchester is a city of medical and healthcare pioneers and today provides an
environment where innovation can grow and prosper.
“It is a hub for top global pharmaceutical, medtech and biotech companies - there are 250 life sciences
companies in the region, as well as four universities with a strong track record of working with industry.
“This Congress features an exciting scientific programme, with world-class speakers covering all of the
most innovative current research topics from basic research into the biology and understanding of cancer right
through to precision medicine.
“This is an emerging important concept in cancer research and treatment today and one of the key focuses
of the Manchester Cancer Research Centre,- a partnership between Cancer Research UK, The University of
Manchester and The Christie NHS Foundation Trust.
“Researchers here in Manchester and from all over Europe will be able to share their latest work and make
new contacts and collaborations that will support important cancer research work in the future.”
The Congress will open with a speech from Sir Harpal Kumar, Cancer Research UK’s chief executive who
suggests the best way to predict the future is to invent it.
Manchester has been chosen as European City of Science for 2016 - testament to its important scientific
heritage. The Congress will take place at Manchester Central on 9-12 July.
NewsRx LLC
(2016-07-18), European cancer scientists converge on Manchester to share latest research, Biotech Business
Leukemias
Findings from University of Washington in the Area of Chronic Myelogenous Leukemia
Reported (Management of Advanced-Phase Chronic Myelogenous Leukemia)
By a News Reporter-Staff News Editor at Hematology Week – New research on Leukemias is the subject of a
report. According to news reporting originating from Seattle, Washington, by NewsRx editors, the research
stated, “Chronic myelogenous leukemia represents the poster child of successful precision medicine in cancer,
with amazing survival results achieved with targeted tyrosine kinase inhibitors (TKIs) in many patients with
chronic-phase disease.”
Our news editors obtained a quote from the research from the University of Washington, “Unfortunately,
however, this good news has not extended to patients in blast crisis, for whom survival has not clearly been
improved with TKIs. During his presentation at the NCCN 21st Annual Conference, Jerald P. Radich, MD,
briefly explored the biology behind advanced-stage disease and several of the molecular findings in disease
progression.”
According to the news editors, the research concluded: “He also reviewed some of the therapeutic options
in advanced disease, emphasizing that transplantation, although fraught with some difficulties, offers the best
long-term prognosis for patients in blast crisis.”
For more information on this research see: Management of Advanced-Phase Chronic Myelogenous
Leukemia. Journal of the National Comprehensive Cancer Network , 2016;14(5.5):669-671. Journal of the National Comprehensive Cancer Network can be contacted at: Harborside Press, 37 Main St, Cold Spring Harbor,
NY 11724, USA.
The news editors report that additional information may be obtained by contacting J.P. Radich, University
of Washington, Fred Hutchinson Canc Res Center, Sch Med, Seattle, WA 98195, United States.
NewsRx LLC
(2016-06-27), Findings from University of Washington in the Area of Chronic Myelogenous Leukemia Reported (Management of Advanced-Phase Chronic Myelogenous Leukemia), Hematology Week, 29, ISSN: 15436721, BUTTER® ID: 011948064
124
Colon Cancer
Genetic mutations found linked to rare cases of multiple bowel tumors
By a News Reporter-Staff News Editor at Cancer Weekly – Researchers have identified genetic mutations affecting the immune system which may lead to the development of more than one bowel tumour at the same
time. Understanding how these cancers develop could improve targeting of therapies, according to the study
published in Nature Communications.
Most people with colorectal cancer will develop one primary tumour, which may then multiply and spread.
However, in around two to five per cent of colorectal cancer cases, two primary tumours will originate and
develop independently, known as synchronous colorectal cancer.
The study, led by researchers from King’s College London, investigated whether the synchronous tumours
had the same or different mutations and what genetic alterations could be predisposing people to develop more
than one colorectal tumour. They analysed 20 synchronous colorectal cancers from 10 patients, and compared
their genetics to people with only one colorectal cancer and to healthy people.
The genetic analysis found the synchronous tumours were not related to each other – they contained a variety
of different genetic mutations that had led to cancer. This genetic variation makes synchronous colorectal
cancers more difficult to treat, because therapies targeted at specific genetic aberrations in a person’s cancer
may not work if the other tumour has different genetic mutations.
Researchers also found synchronous colorectal cancer patients had a higher occurrence of inherited damaging mutations in immune-related genes. Three-quarters of synchronous colorectal cancer patients were found
to have rare damaging mutations in genes related to immune system function.
These patients had differences in the composition of their gut immune cells, which could lead to inflammation in the gut. The study authors conclude that synchronous colorectal cancer patients have inherited
damaging alterations of immune-related genes, which may cause an inflammatory environment in the gut and
increase the frequency of independent cancer-initiating events.
Dr Francesca Ciccarelli, senior author from the Division of Cancer Studies at King’s College London, said:
‘Between two and five per cent of all bowel cancer patients develop more than one primary tumour, called
synchronous tumours. Prior to this study, it was unclear whether these multiple tumours started because of
the same faulty genes. Now we know that these tumours are as different as cancers from two different people
and patients inherit mutations in genes of the immune system that potentially have damaging effect. These
mutations are usually very rare in healthy people, but they occur at a much higher frequency in this patient
group.
‘Currently, colorectal cancer patients with synchronous tumours receive the same type of treatments as
other cancer patients, but we now know that each of their multiple cancers are likely to respond to therapy
and develop resistance in a different way. Therefore, their treatment should be tailored accordingly. This is an
extreme example of precision medicine - normally, precision medicine targets a therapy to match a patient’s
cancer mutation, but in the case of multiple tumours in one patient we need to give the right drug to the right
cancer in order to maximise the response.’
‘We still do not know whether the damaging mutations in immune system genes of these patients have a
direct effect on the composition of the gut immune cells. To unravel this aspect, we will do further studies to
profile the gene expression pattern of these cells and, in case we find differences with healthy people, this can
be used as a biomarker to predict the development of synchronous tumours.’
Dr Matteo Cereda, author from the Division of Cancer Studies at King’s College London, said: ‘These patients also have an abnormal composition of immune cells of the gut and in the tumours. Our hypothesis is
that these individuals develop multiple tumours because they have an abnormal immune and inflammatory
response that favours tumour initiation.’
NewsRx LLC
(2016-07-19), Genetic mutations found linked to rare cases of multiple bowel tumors, Cancer Weekly, 232,
ISSN: 1532-4567, BUTTER® ID: 012066869
125
Genomic Health Announces Multiple Oncotype DX® Presentations at ASCO 2016
Showcasing Industry-leading Commitment to Bringing Precision Medicine to Cancer
Patients
By a News Reporter-Staff News Editor at Women’s Health Weekly – Genomic Health, Inc. (Nasdaq: GHDX)
announced results from seven Oncotype DX(®) studies being presented at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting. Results include four new analyses from the National Cancer Institute’s
(NCI) Surveillance, Epidemiology, and End Results (SEER) Registry with more than 44,600 breast cancer patients. The SEER analyses reconfirmed through prospective outcomes data that the Oncotype DX(®) Breast Recurrence Score™ is an accurate predictor of five-year survival in patients with node-positive and node-negative
disease in contemporary “real-world” clinical practice and revealed disparities in Oncotype DX testing use and
patient outcomes.
“The results of the new analyses of SEER Registry data reinforce the specific value of Oncotype DX in nodepositive disease and in older women,” said Steven Shak, M.D., chief scientific officer, Genomic Health. “These
new data add to unprecedented evidence that Oncotype DX provides critical information to ensure improved
patient outcomes and less harm, and should be recommended and used as standard of care in node-negative
disease and certain patients with node-positive disease.”
– In node-positive disease, the Breast Recurrence Score added considerable
additional independent prognostic value for five-year breast cancer
survival when reported separately for patients with micrometastases,
one, two or three positive nodes (Abstract 6575).
– In node-negative disease, worse breast cancer survival was observed in
older patients (over age 70) who were tested and had an intermediate or
high Breast Recurrence Score result, contrary to the general perception
that older women tend to have favorable outcomes. Patients age 70 or
older also had lower reported chemotherapy use, supporting continued
examination of the often reported issue of under-treatment of the
NewsRx LLC
(2016-06-23), Genomic Health Announces Multiple Oncotype DX® Presentations at ASCO 2016 Showcasing
Industry-leading Commitment to Bringing Precision Medicine to Cancer Patients, Women’s Health Weekly, 175,
ISSN: 1532-4729, BUTTER® ID: 011932736
Ignyta, Inc.
Ignyta Appoints Dr. Christian V. Kuhlen as General Counsel
By a News Reporter-Staff News Editor at Pharma Business Week – Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, announced that Dr. Christian V. Kuhlen has been
appointed to serve as its General Counsel and Secretary, replacing Matt Onaitis. Since 2007, Dr. Kuhlen
served as General Counsel, Vice President and Secretary of Genoptix, Inc., where he led the legal and corporate governance functions from its initial public offering in 2007, through its successful acquisition by Novartis
in 2011. Following the completion of Genoptix’s acquisition, Dr. Kuhlen continued to serve the company in
these capacities, as well as leading its internal public policy function. Prior to joining Genoptix, Dr. Kuhlen
was an attorney in private practice with Cooley LLP from October 2004 to September 2007. Dr. Kuhlen holds
a B.S. in Biochemistry and Cell Biology and a B.A. in Economics from the University of California, San Diego,
and a J.D. and M.D. from the University of Southern California.
“Chris has extensive legal experience with development-stage and publicly-traded, commercial-stage precision medicine companies, coupled with a strong medical and scientific background that is rare amongst General
Counsel,” said Jonathan Lim, M.D., Chairman and CEO of Ignyta. “We expect that his background in advising on complex business transactions, as well as his years of experience supporting operational teams, will
strengthen our management team as we strive to benefit patients with entrectinib and our other cancer precision medicines in development. Speaking on behalf of the team at Ignyta, we also would like to thank Matt for
his excellent contributions to Ignyta, and wish him all the best in the next stage of his career.” About Ignyta,
Inc. At Ignyta, we fight cancer - a formidable opponent that manifests as thousands of different molecularly
defined diseases and takes away millions of lives globally every year. In this fight, our vision is not just to shrink
126
tumors but to eradicate residual disease - the source of cancer relapse and recurrence - in precisely defined patient populations by 2030. We will work tirelessly to achieve this vision by pursuing an integrated therapeutic
(Rx) and companion diagnostic (Dx) strategy for treating cancer patients. Our Rx efforts are focused on discovering, in-licensing or acquiring, then developing and commercializing, molecularly targeted therapies that,
sequentially or in combination, are foundational for eradicating residual disease. Our Dx efforts aim to pair
these product candidates with biomarker-based companion diagnostics that are designed to precisely identify,
at the molecular level, the patients who are most likely to benefit from the therapies we develop. We believe
that only through this integrated Rx/Dx approach can we succeed in this fight.
NewsRx LLC
(2016-07-18), Ignyta Appoints Dr. Christian V. Kuhlen as General Counsel, Pharma Business Week, 27,
ISSN: 1543-6667, BUTTER® ID: 012060856
Prostate Cancer Foundation
Inherited Genetic Mutations Found in 12% of Men with Metastatic Prostate Cancer
By a News Reporter-Staff News Editor at Women’s Health Weekly – One in nine (12%) men with metastatic
prostate cancer carry inherited mutations in DNA damage repair (DDR) genes, reports a new study funded in
part by the Prostate Cancer Foundation (PCF). These practice-changing findings suggest that all metastatic
prostate cancer patients should undergo screening for DDR defects, and that families of men found to have
these mutations seek genetic counseling.
These results were published, July 6, 2016 in the New England Journal of Medicine.
“The implications of these groundbreaking findings are profound,” said Jonathan W. Simons, MD, president
and CEO, Prostate Cancer Foundation. “Not only will they advance precision medicine for prostate cancer by
identifying who may benefit from targeted treatment, but also new recommendations for screening can help
determine who is at the greatest risk for this disease so we can intervene. Also, we estimate more than 12,000
U.S. families facing prostate cancer are carrying these genes in their children and grandchildren.”
“I think every man today with metastatic prostate cancer should have genetic testing, regardless of age or
family history,” said Peter Nelson, MD, of Fred Hutchinson Cancer Research Center, and Professor of Medical
Oncology at the University of Washington and the Genitourinary Oncology Clinical Research Director of the
Seattle Cancer Care Alliance. Nelson led the study on behalf of the PCF International Prostate Cancer Dream
Team.
Breaks in DNA occur thousands of times in each cell cycle, and normal cells have about half a dozen ways
to combat DNA damage. However, cells with mutated DDR genes have deficient repair pathways, leading to
the accumulation of mutations that can promote tumor formation.
Most notable of these are defects in BRCA1/2 genes, which are infamous for increasing a woman’s risk of
breast and ovarian cancer. In the study, Nelson and colleagues found that BRCA2 specifically represented 44%
of all identified mutations, making it the most common heritable mutation in men with advanced prostate
cancer.
Prostate cancer patients with DDR mutations may benefit from targeted treatment with a relatively new
class of drugs known as PARP inhibitors. Olaparib, a PARP inhibitor initially approved for the treatment of
BRCA-mutated ovarian cancer, received breakthrough status for the treatment of advanced prostate cancer
earlier this year. DDR-mutated tumors may also be sensitive to platinum chemotherapy, such as cisplatin and
carboplatin.
The presence of hereditary mutations in metastatic prostate cancer patients may warrant screening of family members, who may also carry these oncogenic defects. Genetic counseling of male and female relatives can
help determine their own risk and recourse for prostate, breast, ovarian, and pancreatic cancers. The type of
testing required by patients and their relatives will vary on an individual basis.
Importantly, due to the relative rarity of germline DDR defects in the general population, the study authors
caution against the widespread implementation of genetic screening. At this time, they do not recommend
genetic testing for men with localized, low-intermediate risk prostate cancer without a strong family history
of prostate, breast, or other cancers. Moreover, they do not suggest that male relatives who test positive for
DDR mutations undergo prophylactic prostatectomies, which can have many side effects that degrade quality of
life. Rather, male carriers should get more intensive screening for prostate cancer beginning at a younger age.
Keywords for this news article include: Oncology, Gynecology, Women’s Health, Genetic Counseling, Prostate
Cancer Foundation.
127
NewsRx LLC
(2016-07-21), Inherited Genetic Mutations Found in 12% of Men with Metastatic Prostate Cancer, Women’s
Health Weekly, 122, ISSN: 1532-4729, BUTTER® ID: 012076893
Association for Molecular Pathology
International Lung Cancer Experts Seek Public Comments on Revised Molecular Testing
Guideline to Improve Patient Selection and Targeted Therapies
By a News Reporter-Staff News Editor at Cancer Weekly – The College of American Pathologists (CAP), the
International Association for the Study of Lung Cancer (IASLC), and the Association for Molecular Pathology (AMP) announced the open comment period for the revised evidence-based guideline, “Molecular Testing
Guideline for Selection of Lung Cancer Patients for EGFR and ALK Tyrosine Kinase Inhibitors.”
The open comment period begins today and will close on August 2, 2016. The online format provides an
opportunity for public review of new draft recommendations for several key topics, as well as recommendation
statements that have been reaffirmed since the initial guideline was jointly published online in April 2013 by
Archives of Pathology & Laboratory Medicine, The Journal of Thoracic Oncology, and The Journal of Molecular
Diagnostics.
“In an era of precision medicine, our collective ability to not only understand but leverage the latest breakthroughs in lung cancer research will enable us to provide more precise targeted treatment options. The revised
guideline provides a clear framework based on evidence from recently published clinical literature,” said Philip
T. Cagle, MD, FCAP, medical director of pulmonary pathology in the Department of Pathology and Genomic
Medicine at Houston Methodist Hospital, editor-in-chief of Archives of Pathology & Laboratory Medicine, and
CAP member.
The guideline revisions are designed to provide state-of-the-art molecular testing of lung cancer recommendations for pathologists, oncologists, and other cancer and molecular diagnostic laboratory professionals.
The revisions are all based on evidence from an unbiased review of published experimental literature since
2013 and include the recommendations from an expert panel of renowned worldwide leaders in the field. The
final recommendations will be approved and jointly published after consideration of the public comments, further panel discussion, and a complete evidence analysis. For more information and to provide comments, visit
http://www.amp.org/LBGOCP .
“The revised guideline reinforces the importance of molecular testing, establishes molecular testing standards, and helps guide targeted therapies for lung cancer,” said Yasushi Yatabe, MD, PhD, chief of the Department of Pathology and Molecular Diagnostics at Aichi Cancer Center in Nagoya, Japan, and IASLC member.
“We strongly encourage our peers to provide feedback to ensure our draft recommendations are sound, practical,
and implementable, so we can all support best clinical practices and provide optimal care to our patients.”
“The final manuscript will serve as both an update and extension of the 2013 practice guideline that established evidence-based identification and best practices for molecular biomarker testing for patients diagnosed
with non-squamous, non-small cell lung cancer of all stages,” said Neal I. Lindeman, MD, director of Molecular
Diagnostics Laboratory at Brigham and Women’s Hospital, associate professor of pathology at Harvard Medical
School, and AMP member. “This guideline will continue to be updated as appropriate, and guidelines for other
biomarkers associated with lung cancer will be added as more research becomes available.”
NewsRx LLC
(2016-07-12), International Lung Cancer Experts Seek Public Comments on Revised Molecular Testing
Guideline to Improve Patient Selection and Targeted Therapies, Cancer Weekly, 62, ISSN: 1532-4567, BUTTER® ID: 012031547
128
Lung Cancer
Lung cancer experts seek public comments on revised molecular testing guideline
By a News Reporter-Staff News Editor at Cancer Weekly – BETHESDA, MD. – The College of American Pathologists (CAP), the International Association for the Study of Lung Cancer (IASLC), and the Association for Molecular Pathology (AMP) announced the open comment period for the revised evidence-based guideline, “Molecular
Testing Guideline for Selection of Lung Cancer Patients for EGFR and ALK Tyrosine Kinase Inhibitors.”
The open comment period begins today and will close on August 2, 2016. The online format provides an
opportunity for public review of new draft recommendations for several key topics, as well as recommendation
statements that have been reaffirmed since the initial guideline was jointly published online in April 2013 by
Archives of Pathology & Laboratory Medicine, The Journal of Thoracic Oncology, and The Journal of Molecular
Diagnostics.
“In an era of precision medicine, our collective ability to not only understand but leverage the latest breakthroughs in lung cancer research will enable us to provide more precise targeted treatment options. The revised
guideline provides a clear framework based on evidence from recently published clinical literature,” said Philip
T. Cagle, MD, FCAP, medical director of pulmonary pathology in the Department of Pathology and Genomic
Medicine at Houston Methodist Hospital, editor-in-chief of Archives of Pathology & Laboratory Medicine, and
CAP member.
The guideline revisions are designed to provide state-of-the-art molecular testing of lung cancer recommendations for pathologists, oncologists, and other cancer and molecular diagnostic laboratory professionals. The
revisions are all based on evidence from an unbiased review of published experimental literature since 2013 and
include the recommendations from an expert panel of renowned worldwide leaders in the field. The final recommendations will be approved and jointly published after consideration of the public comments, further panel
discussion, and a complete evidence analysis. For more information and to provide comments, visit https://
www.iaslc.org/articles/capiaslcamp-molecular-testing-guideline-open-comment-period .
“The revised guideline reinforces the importance of molecular testing, establishes molecular testing standards, and helps guide targeted therapies for lung cancer,” said Yasushi Yatabe, MD, PhD, chief of the Department of Pathology and Molecular Diagnostics at Aichi Cancer Center in Nagoya, Japan, and IASLC member.
“We strongly encourage our peers to provide feedback to ensure our draft recommendations are sound, practical,
and implementable, so we can all support best clinical practices and provide optimal care to our patients.”
“The final manuscript will serve as both an update and extension of the 2013 practice guideline that established evidence-based identification and best practices for molecular biomarker testing for patients diagnosed
with non-squamous, non-small cell lung cancer of all stages,” said Neal I. Lindeman, MD, director of Molecular
Diagnostics Laboratory at Brigham and Women’s Hospital, associate professor of pathology at Harvard Medical
School, and AMP member. “This guideline will continue to be updated as appropriate, and guidelines for other
biomarkers associated with lung cancer will be added as more research becomes available.”
Together with a multi-disciplinary expert panel, Co-chairs Cagle, Yatabe, and Lindeman formulated new
draft recommendations addressing these key questions:
In conjunction with revising the guideline, CAP, IASLC, and AMP will develop clinical tools and resources
for pathologists and oncologists that summarize the findings and recommendations. The organizations expect
to develop a patient guide for further understanding, including questions for patients to ask their physicians.
NewsRx LLC
(2016-07-12), Lung cancer experts seek public comments on revised molecular testing guideline, Cancer
129
Scientific and Technical Instrument Companies
MultiModal Molecular Imaging Institute at Maastricht University Joins Waters Centers of
Innovation Program
By a News Reporter-Staff News Editor at Cardiovascular Business Week – At a ceremony and symposium at
Maastricht University, The Netherlands, Waters Corporation (NYSE:WAT) officially welcomed the Maastricht
MultiModal Molecular Imaging Institute M4I into the Waters Centers of Innovation (COI) Program. Under
the direction of Professor Ron Heeren, the institute conducts research into applications for mass spectrometry
in three main areas: oncology, neurology and cardiovascular medicine.
“Prof. Heeren is a true visionary and we are extremely pleased to welcome him into our COI Program,” said
Eric Fotheringham, Director of the Waters Centers of Innovation Program. “The fantastic work being done
by the MultiModal Molecular Imaging Institute with the help of imaging mass spectrometry gives me a lot of
optimism about medical science and the future of health care.”
For its research, the Institute employs three forms of mass spectrometry - mass spectrometry imaging (MSI)
coupled with ion mobility and high spectral mass resolution. The scope of Prof. Heeren’s use of imaging mass
spectrometry is wide-ranging. He uses it to determine where molecules of all types - lipids, intact proteins,
endogenous peptides, drug metabolites - are localized within a tissue sample - and which are found in high
concentrations and which are not - all in an effort to improve the understanding of disease progression and
regression.
In an interview published in 2014, Prof. Heeren said, “I dare to predict that in ten years’ time, this imaging
technique will be a standard diagnostic tool and patients will be assessed on their individual molecular status.
We’re already well on the way in oncology, neurology and cardiovascular medicine.”
Precision or personalized medicine is the development of treatments that are attuned to the specific biology
of an individual patient. More specifically, by understanding the molecular heterogeneity of tumors and their
different cellular phenotypes, precise treatments can be formulated that offer a patient the best chance of a
long and healthy life.
To salute the work of Prof. Heeren and the Maastricht MultiModal Molecular Imaging Institute, Waters
Corporation sponsored a symposium titled Translational Imaging MS: Molecular Pathology Towards Precision
Medicine. The event featured presentations by noted experts in imaging mass spectrometry including Markus
Stoekli, Novartis Institutes for BioMedical Research; Prof. Klaus Dreisewerd University of Munster; Prof.
Michel W. F. Nielen, Wageningen University; Dr. Liam McDonnell, Leiden University Medical Center; Professor Zoltan Takats, Imperial College. About the MultiModal Molecular Imaging Institute The M4I Division
of Imaging Mass Spectrometry is one of the world leaders in high resolution molecular imaging of biological
surfaces. The division targets the development and application of state-of-the-art mass spectrometry based
molecular imaging approaches for biomedical cellular and tissue research. Main research aim for the coming
years is to develop and apply mass spectrometry as a tool for personalized medicine. About Waters Corporation
(www.waters.com) Waters Corporation (NYSE:WAT) develops and manufactures advanced analytical and material science technologies for laboratory dependent organizations. For more than 50 years, the company has
pioneered a connected portfolio of separations science, laboratory information management, mass spectrometry
and thermal analysis systems.
Waters is a trademark of Waters Corporation. View source version on businesswire.com: http://www.
businesswire.com/news/home/20160622006005/en/
NewsRx LLC
(2016-07-05), MultiModal Molecular Imaging Institute at Maastricht University Joins Waters Centers of
Innovation Program, Cardiovascular Business Week, 4, ISSN: 1552-2512, BUTTER® ID: 011995617
130
Oncology
New Findings Reported from Uniformed Services University of the Health Sciences Describe
Advances in Prostate Cancer (A novel genomic alteration of LSAMP associates with
aggressive prostate cancer in African American men)
By a News Reporter-Staff News Editor at Cancer Weekly – Fresh data on Oncology are presented in a new
report. According to news reporting originating from Bethesda, Maryland, by NewsRx correspondents, research stated, “Evaluation of cancer genomes in global context is of great interest in light of changing ethnic
distribution of the world population. We focused our study on men of African ancestry because of their disproportionately higher rate of prostate cancer (CaP) incidence and mortality.”
Our news editors obtained a quote from the research from the Uniformed Services University of the Health
Sciences, “We present a systematic whole genome analyses, revealing alterations that differentiate African
American (AA) and Caucasian American (CA) CaP genomes. We discovered a recurrent deletion on chromosome
3q13.31 centering on the LSAMP locus that was prevalent in tumors from AA men (cumulative analyses of
435 patients: whole genome sequence, 14; FISH evaluations, 101; and SNP array, 320 patients). Notably,
carriers of this deletion experienced more rapid disease progression. In contrast, PTEN and ERG common
driver alterations in CaP were significantly lower in AA prostate tumors compared to prostate tumors from CA.
Moreover, the frequency of inter-chromosomal rearrangements was significantly higher in AA than CA tumors.”
According to the news editors, the research concluded: “These findings reveal differentially distributed
somatic mutations in CaP across ancestral groups, which have implications for precision medicine strategies.”
For more information on this research see: A novel genomic alteration of LSAMP associates with aggressive
prostate cancer in African American men. Ebiomedicine , 2015;2(12):1957-64.
The news editors report that additional information may be obtained by contacting G. Petrovics, Center
for Prostate Disease Research, Dept. of Surgery, Uniformed Services University of the Health Sciences and
Walter Reed National Military Medical Center, Bethesda, MD 20814, United States. Additional authors for this
research include H. Li, T. Stumpel, S.H. Tan, D. Young, S. Katta, Q. Li, K. Ying, B. Klocke, L. Ravindranath, I.
Kohaar, Y. Chen, D. Ribli, K. Grote, H. Zou, J. Cheng, C.L. Dalgard, S. Zhang, I. Csabai, J. Kagan and Takeda.
NewsRx LLC
(2016-07-05), New Findings Reported from Uniformed Services University of the Health Sciences Describe
Advances in Prostate Cancer (A novel genomic alteration of LSAMP associates with aggressive prostate cancer
in African American men), Cancer Weekly, 299, ISSN: 1532-4567, BUTTER® ID: 011995820
University of Southampton
New insight into cancer genes could lead to personalized treatments
By a News Reporter-Staff News Editor at Hematology Week – Research, involving scientists and doctors at the
University of Southampton and Royal Bournemouth Hospital, has identified a network of genes that are likely
to be shared by all patients who have chronic lymphocytic leukaemia (CLL).
Furthermore, the study has identified gene networks that are associated with patient survival, which could
be identified as targets for treatment.
World-leading cancer researcher, Dr Christoph Bock, from the CeMM Research Center for Molecular
Medicine of the Austrian Academy of Sciences, Vienna, led the project, in collaboration with Professor Jonathan
Strefford from the University of Southampton and CLL clinician, Professor David Oscier from the Royal
Bournemouth Hospital.
It has been published in Nature Communications.
The study was part of a group of work by an international consortium of biomedical researchers led by
the CeMM Research Center, testing the feasibility of epigenetic analysis for clinical diagnostics and precision
medicine.
Epigenetic changes occur in all cancers, and in various other diseases. Building upon years of technology
development in laboratories around the world, this series of research studies shows the accuracy and robustness
of epigenetic tests. Going forward, clinical researchers will be able to apply these methods for specific diseases,
and it is expected that epigenetic tests will be become widely used for selecting personalized treatments in
cancer and other diseases.
131
The UK team, made up of clinicians and scientists from Southampton and Bournemouth, and funded by
Bloodwise and Bournemouth Leukemia Fund, contributed to an international effort to perform the first largescale analysis of the chromatin landscape (a combination of DNA and proteins) in human tumours, focusing on
this occasion on patients with CLL.
Professor Strefford comments: “Our study, has been able to dissect the variability that exists in the
epigenome of CLL patients, and helped to identify disease-specific changes, which will hopefully be informative
for distinguishing disease subtypes or identifying suitable treatments. Epigenetics can offer a useful doorway
into ways of improving disease diagnosis and more personalized treatment choices for patients.”
Dr Matt Kaiser, Head of Research at Bloodwise, says: “Chronic lymphocytic leukaemia develops at different
rates in different patients and some will respond better to treatment than others. While testing for the presence
of specific genetic faults is being developed to better predict a patient’s prognosis, it is clear that other biological
factors influence outcome. This exciting study shows how looking at the bigger genetic picture - the broader
spectrum of how genes behave and interact in the cancer cells - could be integrated to more accurately tailor
treatment plans for individual patients.”
NewsRx LLC
(2016-07-11), New insight into cancer genes could lead to personalized treatments, Hematology Week, 117,
ISSN: 1543-6721, BUTTER® ID: 012022004
Leukemias
New Leukemia Study Findings Reported from New York University (Progress and Prospects
in Pediatric Leukemia)
By a News Reporter-Staff News Editor at Pediatrics Week – A new study on Leukemias is now available. According to news reporting out of New York City, New York, by VerticalNews editors, research stated, “Pediatric
leukemia is the single most common malignancy affecting children, representing up to 30% of all pediatric
cancers. Dramatic improvements in survival for acute lymphoblastic leukemia (ALL) have taken place over the
past 4 decades with outcomes approaching 90% in the latest studies.”
Our news journalists obtained a quote from the research from New York University, “However, progress
has been slower for myeloid leukemia and certain subgroups like infant ALL, adolescent/young adult ALL, and
relapsed ALL. Recent advances include recognition of molecularly defined subgroups, which has ushered in
precision medicine approaches.”
According to the news editors, the research concluded: “We discuss the current understanding of the biology
of the various childhood leukemias, recent advances in research, and future challenges in this field.”
For more information on this research see: Progress and Prospects in Pediatric Leukemia. Current Problems in Pediatric and Adolescent Health Care , 2016;46(7):229-241. Current Problems in Pediatric and Adolescent Health Care can be contacted at: Elsevier Science Inc, 360 Park Ave South, New York, NY 10010-1710,
USA. (Elsevier - www.elsevier.com; Current Problems in Pediatric and Adolescent Health Care - http://www.
journals.elsevier.com/current-problems-in-pediatric-and-adolescent-health-care/ )
Our news journalists report that additional information may be obtained by contacting P.P. Madhusoodhan,
New York University, Div Pediat Hematol Oncol, Dept. of Pediat, Perlmutter Canc CenterLangone Med Center,
New York, NY, United States. Additional authors for this research include W.L. Carroll and T. Bhatla.
NewsRx LLC
(2016-07-16), New Leukemia Study Findings Reported from New York University (Progress and Prospects
in Pediatric Leukemia), Pediatrics Week, 253, ISSN: 1944-2645, BUTTER® ID: 012054792
132
Oncology
New Prostate Cancer Study Results Reported from University of Oxford (The Utility of
Molecular Imaging in Prostate Cancer)
By a News Reporter-Staff News Editor at Drug Week – Investigators discuss new findings in Oncology. According to news reporting from Oxford, United Kingdom, by NewsRx journalists, research stated, “Prostate
cancer is the commonest solid-organ cancer diagnosed in males and represents an important source of morbidity and mortality worldwide. Imaging plays a crucial role in diagnosing prostate cancer and informs the
ongoing management of the disease at all stages.”
The news correspondents obtained a quote from the research from the University of Oxford, “Several novel
molecular imaging technologies have been developed recently that have the potential to revolutionise disease
diagnosis and the surveillance of patients living with prostate cancer. These innovations include hyperpolarised
MRI, choline PET/CT and PSMA PET/CT. The major utility of choline and PSMA PET/CT currently lies in their
sensitivity for detecting early recurrence after radical treatment for prostate cancer and identifying discrete
lesions that may be amenable to salvage therapy.”
According to the news reporters, the research concluded: “Molecular imaging is likely to play a future role
in characterising genetic and biochemical signatures in individual tumours, which may be of particular significance as cancer therapies move into an era of precision medicine.”
For more information on this research see: The Utility of Molecular Imaging in Prostate Cancer. Current Urology Reports , 2016;17(3):26. (Springer - www.springer.com; Current Urology Reports - http://www.
springerlink.com/content/1527-2737/ )
Our news journalists report that additional information may be obtained by contacting A. Leiblich, Dept. of
Urology, Churchill Hospital, University of Oxford NHS Trust, Old Road, Headington, Oxford, UK. Additional
authors for this research include D. Stevens and P. Sooriakumaran.
NewsRx LLC
(2016-07-22), New Prostate Cancer Study Results Reported from University of Oxford (The Utility of Molecular Imaging in Prostate Cancer), Drug Week, 470, ISSN: 1532-4575, BUTTER® ID: 012082476
Oncology
New Prostate Cancer Study Results Reported from University of Oxford (The Utility of
Molecular Imaging in Prostate Cancer) (The Utility of Molecular Imaging in Prostate
Cancer)
By a News Reporter-Staff News Editor at Ivy League Week – Investigators discuss new findings in Oncology.
According to news reporting from Oxford, United Kingdom, by NewsRx journalists, research stated, “Prostate
cancer is the commonest solid-organ cancer diagnosed in males and represents an important source of morbidity
and mortality worldwide. Imaging plays a crucial role in diagnosing prostate cancer and informs the ongoing
management of the disease at all stages.”
The news correspondents obtained a quote from the research from the University of Oxford, “Several novel
molecular imaging technologies have been developed recently that have the potential to revolutionise disease
diagnosis and the surveillance of patients living with prostate cancer. These innovations include hyperpolarised
MRI, choline PET/CT and PSMA PET/CT. The major utility of choline and PSMA PET/CT currently lies in their
sensitivity for detecting early recurrence after radical treatment for prostate cancer and identifying discrete
lesions that may be amenable to salvage therapy.”
According to the news reporters, the research concluded: “Molecular imaging is likely to play a future role
in characterising genetic and biochemical signatures in individual tumours, which may be of particular significance as cancer therapies move into an era of precision medicine.”
For more information on this research see: The Utility of Molecular Imaging in Prostate Cancer. Current Urology Reports , 2016;17(3):26. (Springer - www.springer.com; Current Urology Reports - http://www.
springerlink.com/content/1527-2737/ )
Our news journalists report that additional information may be obtained by contacting A. Leiblich, Dept. of
Urology, Churchill Hospital, University of Oxford NHS Trust, Old Road, Headington, Oxford, UK. Additional
authors for this research include D. Stevens and P. Sooriakumaran.
NewsRx LLC
133
(2016-07-19), New Prostate Cancer Study Results Reported from University of Oxford (The Utility of Molecular Imaging in Prostate Cancer) (The Utility of Molecular Imaging in Prostate Cancer), Ivy League Week, 189,
ISSN: 0000-0000, BUTTER® ID: 012072663
Ovarian Cancer
Ovarian cancer study provides painstaking look at inner workings of tumors
By a News Reporter-Staff News Editor at Politics & Government Week – In what is believed to be the largest
study of its kind, scientists at the Pacific Northwest National Laboratory, Johns Hopkins University and their
collaborators from institutions across the nation have examined the collections of proteins in the tumors of 169
ovarian cancer patients to identify critical proteins present in their tumors.
By integrating their findings about the collection of proteins (the proteome) with information already known
about the tumors’ genetic data (the genome), the investigators report the potential for new insights into the
progress of the most malignant form of the disease. The work is published June 29, 2016, in the advance online
edition of Cell.
The researchers say their achievement illustrates the power of combining genomic and proteomic data - an
approach known as proteogenomics - to yield a more complete picture of the biology of a cancer that accounts
for three percent of all cancers in women and is the fifth leading cause of cancer deaths among women in the
United States.
“Historically, cancer’s been looked at as a disease of the genome,” said Karin Rodland, a senior author of the
study and chief scientist for biomedical research at PNNL, a U.S. Department of Energy laboratory. “But that
genome has to express itself in functional outcomes, and that’s what the proteomic data adds, because proteins
do the actual work of the genome.”
Daniel W. Chan, the study’s other senior author, who led the team at the Johns Hopkins University School
of Medicine, said, “Correlating our data with clinical outcomes is the first step toward the eventual ability
to predict outcomes that reflect patient survival, with potential applications for precision medicine and new
targets for pharmaceutical interventions. But just like anything in medicine, clinical validation will be a long
and rigorous process.”
The authors say that with the findings, researchers expect to be better able to identify the biological factors
defining the 70% of ovarian cancer patients who suffer from the most malignant form of ovarian cancer, called
high-grade serous carcinoma. Currently, only one in six such patients lives five or more years beyond diagnosis.
The power of collaboration
The work draws on the efforts of physicians, scientists and patients who have worked together to understand
ovarian cancer. The investigators say the effort requires collaboration among physicians as well as patients
willing to take part in research to benefit others with the disease or even to prevent others from ever developing
cancer.
Under the leadership of the National Cancer Institute, scientists around the nation have worked together to
create the Cancer Genome Atlas (TCGA), a collaborative effort to map cancer’s genetic mutations. The task for
ovarian cancer was completed in 2011. In the current study, the PNNL and JHU teams each studied subsets
of 169 high-grade serous carcinoma tissue samples and accompanying genomic and clinical data drawn from
that study.
The Johns Hopkins team initially selected 122 of the samples based on those tumors’ ability to repair damaged DNA - known as homologous recombination deficiency - and characterized by changes in genes including
BRCA1, BRCA2 and PTEN, mutations long linked to increased cancer risk and severity.
“We chose to examine these samples because patients with changes in these genes already are benefiting
from a specific drug regimen for breast cancer, so if we could find similar changes in ovarian cancer genomes
and proteomes, those patients would likely benefit from the same regimen,” said Chan, a professor of pathology
and oncology at JHU. Chan is one of the inventors of the OVA1 ovarian cancer detection test, which is licensed
to Vermillion Inc. of Austin, Texas.
The PNNL team initially selected 84 samples based on overall patient survival times. “We examined the
data for the shortest-surviving patients and the longest-surviving patients hoping to pinpoint biological factors
associated with extremely short survival or better-than-average, longer survival,” said Rodland.
Then, through their participation in the Clinical Proteomic Tumor Analysis Consortium (CPTAC), another
program of the National Cancer Institute which funded both teams, the two groups combined their efforts.
Using protein measurement and identification techniques based on mass spectrometry, the teams identified
9,600 proteins in all the tumors, and pursued study on 3,586 proteins common to all 169 tumor samples.
Beyond the genome
134
While many people are familiar with the role our genes play in the development of cancer, the genes are often
just a starting point, for patients and researchers alike. Genes are transcribed into RNA, the genetic material
that makes proteins, which are the workhorses of cells. The activity of the proteins varies dramatically, with
many undergoing changes that affect their impact and interactions with other proteins.
A detailed look at the activity of proteins in cancer biology gives researchers insight into specific molecular
events that would otherwise remain unknown.
A hallmark of cancer, and particularly high grade serous carcinoma, is when genetic instructions go awry.
One form is the appearance of more copies of certain regions of the genome. These so-called copy number
alterations can lead to changes in protein abundance. When the researchers compared known regions of copy
number alterations, they found that parts of chromosomes 2, 7, 20 and 22 led to changes in abundance of more
than 200 proteins. A more careful study of those 200 proteins revealed that many are involved in cell movement
and immune system function, both processes implicated in cancer progression, the researchers said.
“Adding the information about the proteome on top of the genome provides an entirely new dimension of
information that has enabled the discovery of new biological insights to ovarian cancer, while creating a valuable
resource that the scientific community can use to generate new hypotheses about the disease, and how to treat
it,” said Rodland.
“High grade serous carcinoma is such a challenging disease, requiring complex clinical care to achieve longterm survival. This new knowledge gives us new directions to test in the lab and clinic,” said study author
Douglas A. Levine, director of gynecologic oncology at the Laura and Isaac Perlmutter Cancer Center of NYU
Langone Medical Center. “This proteogenomic analysis will help us improve patient outcomes and quality of
life.”
In addition to large teams of scientists from PNNL and Johns Hopkins, contributors included colleagues
from Stanford University School of Medicine, Vanderbilt University School of Medicine, University of California
at San Diego, New York University School of Medicine, Virginia Tech, the National Cancer Institute’s Office of
Cancer Clinical Proteomics Research, as well as CPTAC investigators.
The proteomic analyses performed by the PNNL team were done at EMSL, the Environmental Molecular
Sciences Laboratory, a DOE Office of Science user facility.
NewsRx LLC
(2016-07-14), Ovarian cancer study provides painstaking look at inner workings of tumors, Politics & Government Week, 325, ISSN: 1944-270X, BUTTER® ID: 012043264
Ovarian Cancer
Ovarian cancer study uncovers new biology
By a News Reporter-Staff News Editor at Politics & Government Week – In what is believed to be the largest
study of its kind, scientists at The Johns Hopkins University and the Pacific Northwest National Laboratory
led a study that examined the proteomes of 169 ovarian cancer patients to identify critical proteins expressed
by their tumors.
By integrating their findings with the tumors’ genetic data – the cancer genome – the investigators report
the potential for new insights into the progress of the most malignant form of the disease.
“Correlating our data with clinical outcomes is the first step toward the eventual ability to predict outcomes
that reflect patient survival, with potential applications for precision medicine and new targets for pharmaceutical interventions,” says Daniel W. Chan, Ph.D., a professor of pathology and oncology who led the team at the
Johns Hopkins University School of Medicine. “But just like anything in medicine, clinical validation will be a
long and rigorous process.”
In a summary of the work published in the advance online edition of Cell on June 29, 2016, the researchers
say their achievement illustrates the power of combining genomic and proteomic data to potentially yield a
more complete picture of the biology of a cancer that accounts for 3 percent of all cancers in women and is the
fifth leading cause of cancer deaths among women in the United States.
“With this knowledge, researchers expect to be better able to identify the biological factors defining the 70
percent of ovarian cancer patients who suffer from the most malignant form of ovarian cancer, called high-grade
serous carcinoma,” says Chan. Currently, only one in six patients lives five or more years beyond diagnosis.
“Historically, cancer’s been looked at as a disease of the genome,” says Karin Rodland, Ph.D., chief scientist
for biomedical research at the Pacific Northwest National Laboratory. “But that genome has to express itself
in functional outcomes, and that’s what the proteomic data add, because proteins are what get the actual work
of the genome done.”
135
That work starts, Rodland says, when genes are transcribed into RNA, the genetic material that makes
protein, the full set of which is referred to as the transcriptome.
The biological complexity of cancer is due in significant part to the fact that not every part of the transcriptome leads to proteins, and not every protein stays stable, with many undergoing changes that affect their
impact and interactions with other proteins.
In their effort to better explain ovarian cancer biology, each team studied subsets of 169 high-grade serous
carcinoma tissue samples and accompanying genomic and clinical data drawn from The Cancer Genome Atlas
(TCGA), a collaborative effort to map cancer’s genetic mutations. The task for ovarian cancer was completed in
2011.
The Johns Hopkins team initially selected 122 of the samples based on those tumors’ ability to repair damaged DNA – known as homologous recombination deficiency – and characterized by changes in genes, including
BRCA1, BRCA2 and PTEN, mutations long linked to increased cancer risk and severity.
“We chose to examine these samples because patients with changes in these genes already are benefiting
from a specific drug regimen for breast cancer, so if we could find similar changes in ovarian cancer genomes
and proteomes, those patients would likely benefit from the same regimen,” says Chan.
The Pacific Northwest team initially selected 84 samples based on overall patient survival times. “We examined the data for the shortest-surviving patients and the longest-surviving patients, hoping to pinpoint
biological factors associated with extremely short survival or better-than-average, longer survival,” says Rodland.
Then, through their participation in the Clinical Proteomic Tumor Analysis Consortium (CPTAC), a program of the U.S. National Cancer Institute that funded both teams, the two teams combined their efforts.
Using protein measurement and identification techniques, such as mass spectrometry, the teams identified
9,600 proteins in all the tumors and pursued study on 3,586 proteins common to all 169 tumor samples.
A hallmark of cancer, and particularly high-grade serous carcinoma, is the appearance of more copies of
certain regions of the genome. These so-called copy number alterations can lead to changes in protein abundance. When the researchers compared known regions of copy number alterations, they found that parts of
chromosomes 2, 7, 20 and 22 led to changes in abundance of more than 200 proteins. A more careful study
of those 200 proteins revealed that many are involved in cell movement and immune system function, both
processes implicated in cancer progression, the researchers say.
“By comparing data for overlapping patient samples and finding comparable measurements of protein analysis at both institutions, we think our findings indicate excellent scientific rigor and reproducibility,” says
Rodland.
This work should give researchers everywhere a wealth of credible information, she continues, because of the
number of samples investigated, the number of proteins investigated in each sample, and the ability to crossreference and correlate the new protein data with the TCGA genome and transcriptome data. “In brief, adding
the proteome on top of the genome – what we call proteogenomics – provided a new dimension of information
that enabled discovery of new biological insights into ovarian cancer while creating a valuable resource for the
scientific community,” says Rodland.
“High-grade serous carcinoma is such a challenging disease, requiring complex clinical care to achieve longterm survival. This new knowledge gives us new directions to test in the lab and clinic,” says Douglas A. Levine,
M.D., director of gynecologic oncology at the Laura and Isaac Perlmutter Cancer Center at NYU Langone
Medical Center. “This proteogenomic analysis will help us improve patient outcomes and quality of life.”
NewsRx LLC
(2016-07-14), Ovarian cancer study uncovers new biology, Politics & Government Week, 327, ISSN: 1944270X, BUTTER® ID: 012043265
136
Owlstone Medical Ltd
Owlstone Medical Closes $7M £4.9M Investment to Commercialize Disease Breathalyzer
By a News Reporter-Staff News Editor at Biotech Business Week – Owlstone Medical Ltd, a diagnostics company, has been spun out of parent company, Owlstone Inc, to develop and commercialize a breathalyzer for use
in clinical diagnostics and precision medicine with applications in cancer, inflammatory and infectious disease.
Based in Cambridge, UK, the Company announced that it has successfully raised $7 million (£4.9 million) financing. The investment round was led by Medtekwiz Advisory Ltd and will be used to fund ongoing clinical
trials of the breathalyzer in lung and colon cancer screening.
Owlstone Medical has been created to leverage proprietary and proven Field Asymmetric Ion Mobility Spectrometry (FAIMS) technology for the advancement of a disease breathalyzer. FAIMS measures volatile organic
compound (VOCs) metabolites in patient’s breath or bodily fluids which are specific to disease. Measurement
of VOC biomarkers allows diagnosis of disease at a very early stage, to enable more effective treatment and
better patient outcomes.
Owlstone Medical is managed by a highly experienced team and supported by world-renowned experts in
lung cancer diagnosis. Billy Boyle, Co-founder and CEO at Owlstone Medical, is an engineering graduate from
Cambridge University and one of the original co-founders of Owlstone Inc, which was spun out of Cambridge
University in 2004 and has raised $28M in investment and won in excess of $25M in engineering grants largely
from the US Military.
Billy Boyle commented: “Securing this funding is further validation of our technology, and we are excited to
progress our vision to revolutionize the detection and diagnosis of cancer, infectious and inflammatory diseases.
The breathalyzer we are developing provides clinicians with a highly sensitive, non-invasive diagnostic, which
will enable early detection and improve patient outcomes. We are also working with pharma partners to develop
non-invasive companion diagnostics to better match patients to treatment for emerging personalised therapies.”
Regius Professor Christofer Toumazou, advisor to Medtekwiz and recently appointed to the Owlstone Medical Board, said: “I am delighted to have joined the Board and to be involved at such a milestone in the development of the Company. With the investment, I look forward to seeing a step change in the way serious disease
can be diagnosed and monitored, and particularly for colon and lung cancer, which are two of the biggest cancer
killers worldwide.”
Owlstone Inc CEO and Owlstone Medical board member, Bret Bader commented: “We are very pleased
to have completed this transaction with Medtekwiz. Owlstone Inc has developed a revolutionary platform
technology that promises to change the delivery of chemical detection solutions in markets that range from
defense to medical diagnostics. This transaction and the creation of Owlstone Medical is a template for our
strategy of creating market specific spin outs in order to take a focused approach to solve profound problems.”
The potential of the technology to enable rapid detection of disease, without the need for costly, invasive medical procedures, has seen Owlstone Medical recently awarded the Business Weekly Astra Zeneca-MedImmune
Life Science Innovation award. In February, the Company won an NHS contract for STRATA, adapting its
disease breathalyzer technology for precision medicine and companion diagnostics and prior to that, it was
awarded a $1.4 million (£1 million) NHS contract for LuCID (Lung Cancer Indicator Detection) to use FAIMS
technology in the early detection of lung cancer.
To find out more about Owlstone Medical visit: www.owlstonemedical.com View source version on businesswire.com: http://www.businesswire.com/news/home/20160628005938/en/
NewsRx LLC
(2016-07-11), Owlstone Medical Closes $7M £4.9M Investment to Commercialize Disease Breathalyzer,
137
Perthera, Inc.
Perthera Secures $8.7 Million Investment from Pilot Growth Equity to Expand Global
Leadership in Precision Cancer Analysis “PCA”
By a News Reporter-Staff News Editor at Journal of Engineering – Perthera, Inc. announced the completion
of an $8.7 Million Series A financing. Pilot Growth Equity, a technology growth equity firm with offices in
San Francisco, New York and Washington, DC, led the round and is the sole institutional investor. Perthera
is the market and technology leader in Precision Cancer Analysis (“PCA”), which provides physicians a more
accurate understanding of a patient’s cancer and thereby the ability to identify prospective treatment options
personalized to the individual patient. At the same time, a PCA provides individuals access to a world-class
actionable therapy plan wherever in the world they may be.
“With Operation Moonshot and other initiatives worldwide exploring how new medical technologies can
finally put an end to cancer, we think current practices are going to evolve and change dramatically,” said
Perthera CEO Andrew Mignatti. “One major change in the foreseeable future will be that a PCA will be the
first step undertaken by people diagnosed with cancer just as today an MRI is the first step of those who need
detailed pictures of a body’s structural and organic systems. That detailed level of analysis will, in turn, lead
to the identification of specific therapies, including those in clinical trials, appropriate for individual patients.
One major result will be that oncologists, families and patients can make more informed decisions about their
care,” he said.
Emmanuel “Chip” Petricoin, PhD, who co-founded the company with well-known investor and tech executive
Dendy Young, explained, “The rapid progress being achieved in the field of precision medicine offers the very
real promise of more successful new treatments for cancer. But a precise understanding of an individual’s
particular cancer tumor is a prerequisite to the use of precision medicines, and that’s what a PCA can provide,”
he said. “We do that by using the most sophisticated and comprehensive molecular analysis available. Although
genomic testing has proven to be a breakthrough technology, an even more comprehensive portrait of a patient’s
cancer can be obtained through our multi-omic analysis. Our unique workflow protects tissue, utilizes best-inclass molecular testing, and leverages our unique and growing databank of previous treatment histories and
patient outcomes. The PCA is becoming a critical part of the future of cancer treatment,” Petricoin added.
Neil Callahan, a co-founder and managing director of Pilot Growth Equity, who has joined Perthera’s board,
said, “Perthera’s growth over the last four years has been compelling. Hundreds of oncology practices, including many of the most respected cancer treatment centers and hospitals across the United States, have ordered
the PCA. Adoption of the PCA as the oncologist’s and patient’s first step in developing a cancer treatment
strategy is accelerating at a rapid pace. Perthera’s proprietary software platform automates the creation of
the PCA, integrates the data from all previous analyses and patient history, produces a detailed personalized analysis of the patient’s unique cancer, and delivers an improved patient experience plus an increasingly
higher probability of medical success. In addition to its technology innovations, Perthera has formed a world
class Virtual Tumor Board made up of leading oncologists who provide the treating physicians and patients
with a ‘virtual second opinion’ based on their expertise in concert with the Perthera PCA,” he said. “We think
Perthera has already changed cancer analysis forever and is at the very forefront of personalized and precision healthcare. We are honored to be their partner on this mission.” About Perthera, Inc. Perthera is the
market and technology leader in developing Precision Cancer Analysis (“PCA”) used to provide detailed full
molecular information about cancer tumors. The PCA is a first step in the use of “precision” (sometimes called
“personalized”) medical treatments for cancer patients. It is based in the Tysons Corner area of Northern Virginia. For more information, see www.Perthera.com About Pilot Growth Equity Pilot Growth Equity (“Pilot
Growth”) is a technology growth equity firm with offices in San Francisco, New York and Washington, DC.
Pilot Growth invests in growth-stage technology companies that require capital and strategic resources to accelerate their growth. Founded by entrepreneurs, Pilot Growth provides its portfolio companies a combination
of world class operating, company-building, and advisory expertise, as well as global access to private and
public sector customers. Please visit us at www.pilotgrowth.com. View source version on businesswire.com:
NewsRx LLC
(2016-07-11), Perthera Secures $8.7 Million Investment from Pilot Growth Equity to Expand Global Leadership in Precision Cancer Analysis “PCA”, Journal of Engineering, 960, ISSN: 1945-872X, BUTTER® ID:
012026590
138
Medical, Healthcare
Predicine and CloudHealth to Offer Blood-Based Lung and Prostate Cancer Tests in China
By a News Reporter-Staff News Editor at Journal of Engineering – Predicine, Inc. and CloudHealth Genomics
Ltd announced at the American Society of Clinical Oncology (ASCO) 2016 meeting, to offer Predicine’s liquid
biopsy tests to patients with lung (PrediSeq-Lung) or prostate (PrediSeq-Prostate) cancer in China.
Lung cancer is the number one cause of mortality in China. Prostate cancer on the other hand is the fastest
growing cancer type in China and the most common and second leading cause of cancer death in the western
world. Earlier this year, Dr. Winston Patrick Kuo, President of Predicine, introduced their PrediSeq-Lung
liquid biopsy test at the 23rd International Molecular Medicine Tri-Conference, which took place in San Francisco. A month later, Predicine presented data introducing PrediSeq-Prostate, the world’s first liquid biospy
NGS test for precision medicine in prostate cancer, at the American Association of Cancer Research (AACR) annual meeting in New Orleans. Both tests are based on Predicine’s patented NGS technology, detecting genetic
alterations (mutations, copy number, indel, rearrgangement and splice variants) in circulation.
“We are committed to developing the first- and best-in-class non-invasive NGS-based cancer tests to enable
personalized cancer care and global drug development,” said Dr. Shidong Jia, Founder and CEO of Predicine.
“We are glad to work with industry leaders like CloudHealth so as to serve more patients in China in a timely
manner,” Dr. Jia added. Predicine plans to launch additional tests for major cancer types in China later this
year.
The tests have been rigorously validated and are now being adopted by leading pharmaceutical companies
in the US and China. “The recognition of PrediSeq cancer tests by top-notch pharmaceutical companies is a
testament of the science and quality behind Predicine,” Dr. Jason Jin, CEO and Co-founder of CloudHealth, said
in a statement. “We are excited to move forward with Predicine to offer cancer patients and drug development
companies instant access to the state-of-the-art liquid biopsy technology,” Dr. Jin added.
NewsRx LLC
(2016-06-20), Predicine and CloudHealth to Offer Blood-Based Lung and Prostate Cancer Tests in China,
Journal of Engineering, 1116, ISSN: 1945-872X, BUTTER® ID: 011915897
Prostate Cancer Foundation
Prostate Cancer Foundation Announces 24 New Young Investigator Awards
By a News Reporter-Staff News Editor at Cancer Vaccine Week – The Prostate Cancer Foundation (PCF) is
pleased to announce 24 new Young Investigator (YI) Awards to pioneer increasingly precise treatments and
faster cures for prostate cancer. Members of the Class of 2016 were selected from a pool of 128 applicants from
77 institutions across 15 countries.
Read More About the Class of 2016 PCF Young Investigators
“Our 2016 Young Investigators encapsulate the future of cancer care, and we take great pride in our investment in these remarkable researchers,” said Jonathan W. Simons, MD, president and CEO of PCF. “By
leveraging the most current advances in biotechnology, they are poised to break new ground in immunotherapy and precision medicine, and establish novel modalities for optimizing existing therapies for better patient
outcomes in the near term.”
Successful proposals in the Class of 2016 forecast new paradigms for the understanding and management
of prostate cancer and other genetically complex diseases. These include increased reliance on genomics to
accurately predict prognosis and personalize treatments. Several YIs are working on game-changing new immunological approaches that redirect a patient’s own immune cells to fight cancer. Advances in these areas
may one day circumvent treatment resistance and eliminate the need for systemic treatments that can be toxic.
Projects also predominantly concentrate on treatment resistant prostate cancers. This work will not only
identify new vulnerabilities in aggressive forms of prostate cancer, but will also help prevent the onset of–and
possibly reverse–resistance to first-line therapies.
Other YIs address the relationship of lifestyle factors, such as obesity, and aggressive disease, a burgeoning
research area that has gained momentum in recent months.
139
The YI Program has been a pillar of PCF’s Research Enterprise since its inception in 2007. Awards make
3-year investments in early career scientists with the skills and expertise to rapidly advance high impact discoveries that can be translated to clinical practice. As part of the YI program, awardees are mentored by leaders
in prostate cancer research. Each award is matched dollar-for-dollar by the investigator’s institution.
To date, PCF has provided funding to 202 YIs, representing a total investment of $42.3 million in 10 countries. Awardees have been responsible for innovative discoveries related to prostate cancer diagnostics, disease
progression, and the development of treatment resistance.
NewsRx LLC
(2016-07-04), Prostate Cancer Foundation Announces 24 New Young Investigator Awards, Cancer Vaccine
Quintiles
Quintiles Introduces Precision Enrollment to Enhance Patient Recruitment in Oncology
Trials
By a News Reporter-Staff News Editor at Clinical Trials Week – Quintiles, the world’s largest provider of
product development and integrated healthcare services, announced its Precision Enrollment offering. The
new enrollment model is designed to significantly accelerate site start-up and patient recruitment in oncology
clinical trials working with the company’s network of investigative sites across the U.S and leveraging secondary
data (e.g. electronic health records-EHRs.) In this new model, the traditional site start-up pathway is realigned
so a site is only opened after a patient is identified.
com/news/home/20160531005279/en/
“The rise of precision medicine, scarcity of patients and investigators, combined with increasingly complex
trial protocols, requires innovative approaches to clinical trial implementation,” said Cynthia Verst, PharmD,
president, Clinical Operations at Quintiles. “This solution harnesses Quintiles’ deep therapeutic expertise in
oncology, robust oncology investigator relationships and our demonstrated operational excellence to identify
and recruit the right patient, from the right investigator, for the right oncology trial at the right time.”
Today, three out of five oncology treatments are targeted therapies, with efficacy in niche patient subpopulations.1 These factors can contribute to higher screen failure rates and pose critical challenges for recruiting
patients that fit a particular trial profile. Conventional models for targeting these sub-populations and prescreening of these patients are time- and cost-intensive, making it difficult for oncologists to find the study that
fits the individual patient.
With Precision Enrollment, Quintiles has built a network of oncology sites with master contracts as well
as centralized IRB capabilities to streamline patient identification and start-up. The program will enable preidentification of patients based on study and biomarker criteria, across broad geographic areas, using EHRs
and other secondary data sources before a site joins a study. By using this rapid start-up model, these preidentified patients are quickly matched to specific protocol inclusion and exclusion criteria and site activation
is designed to take less than 21 days.
“Our premier site network and strong relationships with investigators is an integral part of Precision Enrollment, and this approach can provide value to both pharmaceutical companies and patients alike,” said Jeanne
Hecht, senior vice president and global head, Site & Patient Networks at Quintiles. “By opening a site only
after a patient has been identified, we’re able to reduce zero-enrolling sites and associated time and costs, and
most importantly, provide patients with quicker access to potentially life-saving treatments.”
To learn more about Quintiles Precision Enrollment, view the video and visit http://quintiles.com/
precisionenrollment . About Quintiles Quintiles (NYSE: Q) helps biopharma and other healthcare companies improve their probability of success by connecting insights from our deep scientific, therapeutic and
analytics expertise with superior delivery for better outcomes. From advisory through operations, Quintiles is
the world’s largest provider of product development and integrated healthcare services, including commercial
and observational solutions. Conducting operations in approximately 100 countries, Quintiles is a member
of the FORTUNE 500 and has been named to FORTUNE’s list of the “World’s Most Admired Companies.” To
learn more, visit www.quintiles.com. 1 McKesson Specialty Health/US Oncology Network. 2013
Click here to subscribe to Mobile Alerts for Quintiles. View source version on businesswire.com: http:
140
NewsRx LLC
(2016-06-13), Quintiles Introduces Precision Enrollment to Enhance Patient Recruitment in Oncology Trials, Clinical Trials Week, 488, ISSN: 1543-6764, BUTTER® ID: 011823574
Colon Cancer
Researchers: Specific Gene Could Predict Colon Cancer Outcomes
By a News Reporter-Staff News Editor at Gastroenterology Week – Those studying and treating colon cancer
will have a new way of looking at the disease thanks to research results from a study led by Timothy J. Yeatman,
MD, President and Director of Gibbs Cancer Center & Research Institute at Spartanburg Regional Healthcare
System.
The study, published in prestigious medical scientific journal Nature Communications in June 2016, entitled
“A multigene mutation classification of 468 colorectal cancers reveals a prognostic role for APC,” focuses on the
Adenomatous Polyposis Coli (APC), a gene that has long been considered the “gatekeeper” in the development
of colon cancer.
Yeatman, lead investigator at the Gibbs Cancer Center & Research Institute, along with Dr. Michael J.
Schell of Moffitt Cancer Center in Tampa, Fla., performed extensive analysis of DNA sequencing from a large,
human colon cancer database to identify a new, clinically-relevant role for APC, that is mutated in more than
70 percent of colon cancer cases.
“The fact that APC mutation is so common would suggest that it wouldn’t have any other role than simply
to initiate colon cancer,” Yeatman said. “But we found this not to be the case.”
Starting with analyzing 1,321 cancer genes, the study results found that gene sequencing of APC and other
associated genes reveals a prognostic effect that could help physicians better predict long term outcomes. Previously, APC had not been commonly sequenced in many clinical panels.
“This advances personalized or precision medicine for colon cancer,” Yeatman said.
Precision medicine promotes personalized medical care, with decisions, practices, and/or products tailored
to each patient, often based on genetic testing. Precision medicine considers “individual variability in genes,
environment and lifestyle for each person,” according to the National Institutes of Health.
The precision of gene sequencing is key. A cancerous colon tumor may be caused by a number of mutations.
All genes have two copies, one from the mother and one from the father. These pairs are classified by four
variations: (1) wild type (normal), with no associated mutations in either copy; (2) one mutation present, one
copy missing; (3) one mutation present, one normal copy present; or (4) both copies mutated.
As common as APC mutation is in colon cancer, tumors with normal APC genes produce some of the worst
outcomes, along with tumors containing two mutations, according to study results.
Also in play for prognostic differences are the locations of mutations within the gene, types and numbers of
mutations associated with each tumor, and mutations in genes with which APC partners.
“No one has ever related these specific mutations to clinical outcomes – the chances that you’ll live or die,”
Yeatman said.
The results that Yeatman and colleagues discovered were possible because of the ability to sequence a lot of
tumors. In fact, one of the world’s largest molecular and clinical databases on colon cancer is the basis for the
research. The database was originally created as a collaboration between Merck and the Moffitt Cancer Center
and more recently, a collaboration with the Gibbs Center & Research Institute.
“That’s why it’s so important for patients to participate in research and for institutions to collaborate,”
Yeatman said.
Yeatman, a surgeon who specializes in surgical oncology, liver cancer and colorectal cancer, performs research at the state-of-the-art, 10,000-square-foot lab at Gibbs Cancer Center & Research Institute, which oversees as many as six research projects at any given time.
Because Gibbs is the next generation “hybrid academic/community” cancer center, its focus is on translational research and clinical care, also known as “lab bench to patient bedside.” This approach promises to bring
discoveries from the lab to the patient in a much faster time frame, according to Yeatman. The gene sequencing
highlighted in the article has already been taking place at Gibbs for a year.
“I suspect that more clinicians will start looking at this gene, and consider including it in their sequencing
panels,” said Yeatman, who has spent a lifetime researching colon cancer and has published papers in Science,
Nature Medicine, Nature Genetics, and Nature Cancer Review, among others. His research has been federally
funded without interruption since 1994. This study was funded by a grant from the National Cancer Institute.
141
The next step: Yeatman and other researchers will continue to try to identify clinical uses for the sequencing
of this gene, and many others in the hopes of predicting drug responses and other applications. At some point,
Yeatman suspects that the APC gene sequencing discovery could impact decisions made regarding the proper
treatment of patients with colon cancer.
NewsRx LLC
(2016-07-04), Researchers: Specific Gene Could Predict Colon Cancer Outcomes, Gastroenterology Week,
321, ISSN: 1543-6748, BUTTER® ID: 011986410
Ovarian Cancer
Simplifying access to gene testing for women with ovarian cancer
By a News Reporter-Staff News Editor at Cancer Weekly – A new streamlined approach to genetic testing for
women with ovarian cancer provides testing rapidly and affordably, allowing many more patients to benefit
from personalised cancer management and their relatives to benefit from cancer prevention strategies.
The new approach offers cancer patients the opportunity to get gene testing at one of their routine cancer
clinic appointments instead of having to be referred to a separate genetic testing clinic.
As well as offering a more streamlined patient journey, the pathway is less resource intensive for health
systems and could save the NHS millions of pounds per year, if implemented nationally.
The new testing pathway was developed by researchers at The Institute of Cancer Research, London, as
part of the Wellcome Trust-funded Mainstreaming Cancer Genetics programme.
It was piloted for BRCA gene testing in 207 women with ovarian cancer at The Royal Marsden NHS Foundation Trust, in a study supported by the NIHR Biomedical Research Centre at The Royal Marsden and The
Institute of Cancer Research (ICR).
The study, published (Wednesday) in the journal Scientific Reports, showed the new testing approach was
welcomed by patients; all 207 ovarian cancer patients accepted the offer of BRCA gene testing and the post-test
feedback was very positive.
The new testing pathway reduced hospital visits for patients and substantially reduced the time taken for
testing to be completed, ensuring the results were able to be incorporated into clinical decision making.
The test results were useful in deciding medical management of four-fifths of the patients who were receiving
cancer treatment. This included 32 women found to have a BRCA mutation, many of whom became eligible for
new precision medicines only suitable for women with BRCA-related ovarian cancer.
In the new pathway cancer patients gave consent for testing by a cancer doctor or nurse who completed a
30-minute online training module designed by the research team. All patients found to have a BRCA mutation
automatically get an appointment with the genetics team to discuss the implications for themselves and their
families in detail.
For each patient identified with a BRCA mutation, on average three family members also decided to see
a geneticist to discuss the implications for them. BRCA mutations increase the risk of breast and ovarian
cancer occurring and testing in the relatives allowed individualised cancer risk information to be given to them.
Relatives that also have a BRCA mutation have various options available to them to improve early detection or
prevention of cancer.
The new testing pathway has now become standard at The Royal Marsden and is being adopted by other
hospitals in the UK and internationally. Many more women with ovarian cancer have received BRCA testing
as a result.
Almost all ovarian cancer patients are eligible for BRCA testing under current national recommendations,
but provision of testing has been patchy across the NHS. It is estimated that less than a third of ovarian cancer
patients have actually been getting testing.
The simplicity and efficiency of the new testing approach would, the researchers believe, make it practical
for all eligible ovarian cancer patients across the UK to be offered testing within existing resources. The researchers estimate that rolling out the new pathway across the NHS would save £2.6M per year compared with
the current standard process.
A companion study led by the same researchers in collaboration with DRG Abacus and Astra Zeneca found
that the new approach to testing was extremely cost-effective, because many healthy relatives at high risk of
cancer make choices that reduce their chance of developing the disease.
If all 7,000 women diagnosed with epithelial ovarian cancer in the UK each year were offered testing, just a
single year’s testing is likely, over time, to prevent hundreds of breast and ovarian cancers and dozens of deaths
in their relatives, the study found.
142
The researchers showed that national implementation for all ovarian cancer patients would cost £4,339 per
quality-adjusted life year - far below the £20,000 threshold that is used to decide what tests and treatments
the NHS should offer.
Study leader Professor Nazneen Rahman, Head of Cancer Genetics at The Institute of Cancer Research,
London, and The Royal Marsden NHS Foundation Trust, said:
“We know BRCA gene testing can be greatly beneficial for women with ovarian cancer, allowing their care to
be tailored to their individual genetic information, and improving the cancer risk information we can provide
to their families.
“Our new gene testing pathway is faster, simpler and better designed for cancer patients’ needs than the
standard NHS process. Our study has shown that it is not only a feasible way of providing BRCA testing to
all eligible women with ovarian cancer, but could also prevent cancers and save the NHS millions of pounds a
year.”
Professor Martin Gore, Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust, said:
“The new genetic testing pathway has been a huge success in the clinic. It all runs very smoothly and I
know that patients and their families are really most appreciative.”
Preeti Dudakia, 49, an ovarian cancer patient at The Royal Marsden who took part in the study said:
“My cancer team explained why the test was useful very clearly and when it came back positive I was able to
have a treatment only given to women with a BRCA mutation. If my mother had been able to have this testing
when she got ovarian cancer I would have known I was at risk sooner. My situation could have been completely
different.”
Professor Paul Workman, Chief Executive of The Institute of Cancer Research, London, said:
“Twenty years ago the BRCA2 gene was identified at the ICR. This study is an excellent example of how
science such as this can be turned into something very practical that can improve the patient care and save
lives. We hope the new model for genetic testing will be rolled out across the NHS.”
NewsRx LLC
(2016-07-26), Simplifying access to gene testing for women with ovarian cancer, Cancer Weekly, 177, ISSN:
1532-4567, BUTTER® ID: 012100179
Oncology
Studies from Fred Hutchinson Cancer Research Center Yield New Information about
Prostate Cancer (Biallelic Inactivation of BRCA2 in Platinum-sensitive Metastatic
Castration-resistant Prostate Cancer)
By a News Reporter-Staff News Editor at Cancer Weekly – Research findings on Oncology are discussed in
a new report. According to news reporting out of Seattle, Washington, by NewsRx editors, research stated,
“Understanding the molecular underpinnings of sensitivity to specific therapies will advance the goal of precision medicine in prostate cancer (PCa). We identified three patients with metastatic castration-resistant PCa
(mCRPC) who achieved an exceptional response to platinum chemotherapy (not first-line treatment for PCa),
despite disease progression on prior standard therapies.”
Our news journalists obtained a quote from the research from Fred Hutchinson Cancer Research Center,
“Using targeted next-generation sequencing on the primary and metastatic tumors, we found that all three
patients had biallelic inactivation of BRCA2, a tumor suppressor gene critical for homologous DNA repair.
Notably, two had germline BRCA2 mutations, including a patient without compelling family history who was
diagnosed at age 66 yr. The third patient had somatic BRCA2 homozygous copy loss.”
According to the news editors, the research concluded: “Biallelic BRCA2 inactivation in mCRPC warrants
further exploration as a predictive biomarker for sensitivity to platinum chemotherapy.”
For more information on this research see: Biallelic Inactivation of BRCA2 in Platinum-sensitive Metastatic
Castration-resistant Prostate Cancer. European Urology , 2016;69(6):992-995. European Urology can be contacted at: Elsevier Science Bv, PO Box 211, 1000 Ae Amsterdam, Netherlands. (Elsevier - www.elsevier.com;
European Urology - http://www.journals.elsevier.com/european-urology/ )
Our news journalists report that additional information may be obtained by contacting H.H. Cheng, Fred
Hutchinson Canc Res Center, Human Biol Div, 1124 Columbia St, Seattle, WA 98104, United States. Additional
authors for this research include C.C. Pritchard, T. Boyd, P.S. Nelson and B. Montgomery.
NewsRx LLC
143
(2016-06-21), Studies from Fred Hutchinson Cancer Research Center Yield New Information about Prostate
Cancer (Biallelic Inactivation of BRCA2 in Platinum-sensitive Metastatic Castration-resistant Prostate Cancer), Cancer Weekly, 563, ISSN: 1532-4567, BUTTER® ID: 011921276
Oncology
Studies in the Area of Lung Cancer Reported from M. Saito and Co-Researchers (Gene
aberrations for precision medicine against lung adenocarcinoma)
By a News Reporter-Staff News Editor at Clinical Trials Week – Fresh data on Oncology are presented in a new
report. According to news reporting originating from Barcelona, Spain, by NewsRx correspondents, research
stated, “Lung adenocarcinoma (LADC), the most frequent histological type of lung cancer, is often triggered by
an aberration in a driver oncogene in tumor cells. Examples of such aberrations are EGFR mutation and ALK
fusion.”
Our news editors obtained a quote from the research, “Lung adenocarcinoma harboring such mutations can
be treated with anticancer drugs that target the aberrant gene products. Additional oncogene aberrations,
including RET, ROS1, and NRG1 fusions, skipping of exon 14 of MET, and mutations in BRAF, HER2, NF1,
and MEK1, were recently added to the list of such druggable driver oncogene aberrations, and their responses
to targeted therapies are currently being evaluated in clinical trials. However, approximately 30% and 50% of
LADCs in patients in Japan and Europe/USA, respectively, lack the driver oncogene aberrations listed above.
Therefore, novel therapeutic strategies, such as those that exploit the vulnerabilities of cancer cells with nononcogene aberrations, are urgently required.”
According to the news editors, the research concluded: “This review summarizes the current status of research on precision medicine against LADC and enumerates the research priorities for the near future.”
For more information on this research see: Gene aberrations for precision medicine against lung adenocarcinoma. Cancer Science , 2016;107(6):713-720. Cancer Science can be contacted at: Wiley-Blackwell, 111
River St, Hoboken 07030-5774, NJ, USA. (Wiley-Blackwell - http://www.wiley.com/ ; Cancer Science http://onlinelibrary.wiley.com/journal/10.1111/(ISSN)1349-7006 )
The news editors report that additional information may be obtained by contacting M. Saito, Inst Predict &
Personalized Med Canc, Canc Genome Biol Grp, Barcelona, Spain. Additional authors for this research include
K. Shiraishi, H. Kunitoh, S. Takenoshita, J. Yokota and T. Kohno.
NewsRx LLC
(2016-07-25), Studies in the Area of Lung Cancer Reported from M. Saito and Co-Researchers (Gene aberrations for precision medicine against lung adenocarcinoma), Clinical Trials Week, 720, ISSN: 1543-6764, BUTTER® ID: 012090598
Oncology
University of Michigan Reports Findings in Head and Neck Cancer (Fibroblast growth factor
family aberrations as a putative driver of head and neck squamous cell carcinoma in an
epidemiologically low-risk patient as defined by targeted sequencing)
By a News Reporter-Staff News Editor at Life Science Weekly – Current study results on Oncology have been
published. According to news reporting originating from Ann Arbor, Michigan, by NewsRx correspondents,
research stated, “Targeted sequencing of patients with epidemiologically low-risk (ELR) head and neck squamous cell carcinoma (HNSCC) could help identify novel drivers or lost suppressors leading to precision medicine
protocols and improved survival rates. A patient with ELR-HNSCC was selected for targeted sequencing.”
Our news editors obtained a quote from the research from the University of Michigan, “We then assessed next
generation sequencing cohorts from the Oncomine Powertool Database, which contains pan-cancer data from
The Cancer Genome Atlas (TCGA). Targeted sequencing revealed fibroblast growth factor receptor-1 (FGFR1)
amplifications as a putative driver of the patient’s tumor. Patients with HNSCC from TCGA data demonstrated
fibroblast growth factor (FGF) family mutations, rearrangements, or amplifications in over 35% of HNSCC
cases, with a statistically significant higher frequency in African American populations. FGF alterations were
unique from activating phosphatidylinositol 3-kinase (PIK3CA) mutations.”
According to the news editors, the research concluded: “Together, these data suggest that FGF signaling may
be critical for a subset of patients with HNSCC independent of other known pathways and provides rationale
for leveraging patients with ELR-HNSCC to define molecular subsets of high-risk HNSCC.”
144
For more information on this research see: Fibroblast growth factor family aberrations as a putative driver
of head and neck squamous cell carcinoma in an epidemiologically low-risk patient as defined by targeted
sequencing. Head and Neck-Journal for the Sciences and Specialties of the Head and Neck , 2016;38():E1646E1652. Head and Neck-Journal for the Sciences and Specialties of the Head and Neck can be contacted at:
Wiley-Blackwell, 111 River St, Hoboken 07030-5774, NJ, USA.
The news editors report that additional information may be obtained by contacting B.N. Tillman, University
of Michigan, Sch Med, Center Comprehens Canc, Ann Arbor, MI 48109, United States. Additional authors
for this research include M. Yanik, A.C. Birkeland, C.J. Liu, D.H. Hovelson, A.K. Cani, N. Palanisamy, S.
Carskadon, T.E. Carey, C.R. Bradford, S.A. Tomlins, J.B. McHugh, M.E. Spector and J.C. Brenner.
NewsRx LLC
(2016-05-31), University of Michigan Reports Findings in Head and Neck Cancer (Fibroblast growth factor
family aberrations as a putative driver of head and neck squamous cell carcinoma in an epidemiologically lowrisk patient as defined by targeted sequencing), Life Science Weekly, 8345, ISSN: 1552-2474, BUTTER® ID:
011773776
145
Chapter 10
Additional Research
Cure Forward
Alicia Staley, Patient Advocacy Leader, Selected to Participate in Vice President Joe
Biden’s Cancer Moonshot Summit
By a News Reporter-Staff News Editor at Cancer Weekly – Alicia Staley, a three-time cancer survivor, founder
of #BCSM, a leading breast cancer social community, and Patient Advocacy Manager for Cure Forward, an
online precision medicine platform for cancer patients, has been selected to participate in the National Cancer
Moonshot Summit in Washington, D.C. on June 29.
Hosted by Vice President Joe Biden, the summit is designed to facilitate interaction among cancer researchers, oncologists, philanthropists, patients, advocates, and other experts. Attendees will participate in
presentations, working sessions and a variety of digital activities in order to collaborate and address ways to
achieve the goal of doubling the rate of progress in the prevention, diagnosis, treatment, and care of cancer.
“The Moonshot cannot be achieved by one person, one organization, one discipline, or even one collective
approach,” Vice President Biden said. “Solving the complexities of cancer will require the formation of new
alliances to defy the bounds of innovation and accelerate the prevention, diagnosis, treatment, and - ultimately
- a cure. It’s going to require millions of Americans speaking up and contributing what they’re able. That’s
what the Cancer Moonshot Summit is all about.”
Staley was invited as a patient advocate representative to provide insight from the patient community.
Diagnosed with Hodgkin’s Lymphoma at the age of nineteen, later overcoming breast cancer twice in her 30s,
and more recently as a leader of online communities, Staley is well aware of the emotions and challenges that
patients face at all stages of diagnosis and treatment.
“In the last several years, I have seen social media emerge as an indispensable resource for cancer patients.
Communities can help individuals to find their way through the confusing world of cancer care, and can tackle
some of the fundamental issues that patients face today. Making those communities happen has been my
mission,” explains Staley. “Recently, I have turned that mission into a career with my role at Cure Forward.
We need to keep driving this, and being invited to represent patients at the Moonshot Summit is a wonderful
opportunity to bring attention to patient perspectives as the nation rallies against cancer.”
In 2008, Staley joined Twitter to find and connect with other cancer survivors. In 2011, Staley co-founded
#BCSM, which attracts over 250 global participants each week to its scheduled online discussions. Since its
launch, #BCSM has been showcased at SXSW in 2013, 2014, and 2015, and is recognized as the gold standard
for disease-specific Twitter chats.
Today, Staley is the Patient Advocacy Manager for Cure Forward, an online precision medicine platform
made for cancer patients and their families. Cure Forward helps individuals explore precision medicine testing,
obtain their cancer diagnostic data, and use it to find care options including clinical trials. The company’s
platform includes a social network for cancer patients that is rooted in science.
“We are thrilled for Alicia to have been selected to participate in this landmark event,” said Martin Naley,
Cure Forward’s Founder. “She has made connecting with and advocating for cancer patients a significant part
of her life. At Cure Forward, she works directly with our patient community, as a ‘mentor of mentors’, shaping a
new type of patient-to-patient dialogue, where people use science to solve their disease and where clinical trials
are considered at every step of a person’s care.” About Cure Forward Cure Forward bridges the gap between
patients and precision medicine, beginning in cancer. Cure Forward provides a simple-to-use platform that
allows patients to obtain their genomic data and use it to identify treatment options including clinical trials,
146
solving critical business needs for diagnostic laboratories, health care providers and clinical trial recruiters.
Cure Forward is based in Cambridge, Massachusetts and was incubated and launched by Apple Tree Partners,
New York City. The company’s platform is in beta release at www.cureforward.com. View source version on
NewsRx LLC
(2016-07-12), Alicia Staley, Patient Advocacy Leader, Selected to Participate in Vice President Joe Biden’s
Cancer Moonshot Summit, Cancer Weekly, 193, ISSN: 1532-4567, BUTTER® ID: 012031648
Ariel Precision Medicine, LLC
Ariel Precision Medicine, LLC Files SEC Form D, Notice of Exempt Offering of Securities
(May. 27, 2016)
By a News Reporter-Staff News Editor at NewsFile – According to news reporting originating from Washington, D.C., by NewsRx journalists, a U.S. Securities and Exchange Commission (SEC) filing by Ariel Precision
Medicine, LLC (Form D) was posted on May 27, 2016.
The SEC file number is 0001674590-16-000002.
The contact information for this company is 4638 CENTRE AVENUE, PITTSBURGH PA 15213,
2563279569.
Our editors provided additional information about Form D: Companies selling securities in reliance on a
Regulation D exemption or a Section 4(6) exemption from the registration provisions of the ‘33 Act must file a
Form D as notice of such a sale. The form must be filed no later than 15 days after the first sale of securities.
For additional information on Regulation D and Section 4(6) offerings, ask for a copy of the Regulation and the
pamphlet entitled: “Q & A: Small Business and the SEC” from the Commission’s Publications Unit or see the
Small Business Section of the Commission’s Web Site.
A U.S. Securities and Exchange Commission filing is a formal document or financial statement submitted
to the SEC by publicly-traded companies.
For additional information on this SEC filing see: http://www.sec.gov/Archives/edgar/data/
1674590/0001674590-16-000002-index.html .
NewsRx LLC
(2016-06-13), Ariel Precision Medicine, LLC Files SEC Form D, Notice of Exempt Offering of Securities
(May. 27, 2016), NewsFile, 964, ISSN: 0000-0000, BUTTER® ID: 011825813
Biotechnology Companies
BioNJ’s Third Annual Diagnostics & Precision Medicine Summit
By a News Reporter-Staff News Editor at Biotech Business Week – BioNJ’s Third Annual Diagnostics & Precision Medicine Summit, scheduled for Tuesday, June 21, 2016, at Rutgers Business School, Livingston Campus,
Piscataway, NJ, will bring together professionals in the fields of Diagnostics and Precision Medicine to collectively share the latest in technologies and trends, and assess how these and many other dynamics are driving
the advancement of better individualized patient outcomes.
“BioNJ’s Diagnostics & Precision Medicine Summit reinforces New Jersey’s vital role in the advancement
of Precision Medicine,” said BioNJ President and CEO Debbie Hart. “We have a stellar lineup of speakers, all
at the forefront of this evolving area of medical innovation, as well as a rich roster of events that will encourage
deep discussion on how Precision Medicine can translate to better health at the patient level.”
Thought leaders from the industry, academia, NIH and investor community will lead discussions on the
status of innovation, policy changes, the role of Big Data, the evolving value proposition and Precision Medicine
in practice. The robust full-day schedule will include Keynote presentations, plenary sessions, networking,
exhibits, poster sessions and company presentations.
NewsRx LLC
(2016-06-20), BioNJ’s Third Annual Diagnostics & Precision Medicine Summit, Biotech Business Week, 23,
ISSN: 1543-6861, BUTTER® ID: 011908317
147
Certara
Certara and The University of Manchester Co-host Inaugural Model-based Personalized
Drug Dosing in Healthcare Conference
By a News Reporter-Staff News Editor at Drug Week – Certara®, the global biosimulation technology-enabled
drug development company, in partnership with The University of Manchester, successfully organized the firstever Health Care Summit on Model-based Precision Dosing on May 19-20 in the UK. Precision dosing is a key
and proactive step toward achieving the goals of precision medicine, a global objective supported by world
leaders, including President Obama under the Precision Medicine Initiative.
“Through its modeling and simulation work, Certara has been working toward the goal of precision dosingproviding the right drug dose to maximize therapeutic benefit, while reducing risk for each individual patient,”
said Certara Chief Executive Officer Edmundo Muniz, MD, PhD. “Through this Summit and the 15 examples
which were presented that showcased the successful use of modeling and simulation in the hospital research
environment, we are confident that precision dosing in healthcare will become mainstream shortly.”
Professor Kay Marshall, head of the Manchester Pharmacy School and a speaker at the conference, said:
“Everyone is different and this means that they react to drugs in different ways. The emerging precision dosing
field harnesses the explosion of genomic data and various markers of bodily functions using mathematical
modeling to ensure that individuals get the best possible treatment.”
Speakers from eight countries, representing research institutions, academia, pharmaceutical companies,
former regulators, and legal authorities spoke at the Summit. Sessions were organized around special populations representing the most fragile and complex population cohorts, including oncology, HIV, pediatric,
obese, renally-impaired, cell transplant, and adolescent psychiatric patients and pregnant women. In addition
to demonstrating how modeling and simulation facilitated individualized dosing, the conference focused on
linking personalized dosing and its impact on public health.
Certara Chief Scientific Officer Amin Rostami, who also serves as the chair of systems pharmacology at
The University of Manchester, said, “We now understand more fully the sources of variability, the individual
characteristics of each patient, which can drive dosing decisions. The successful case studies shared at this
Summit demonstrate that we are now ready to take the next step toward individualized dosing in everyday
health care.” About Certara Certara is a global biosimulation and regulatory writing company, committed
to optimizing drug development decisions. Its clients include hundreds of international biopharmaceutical
companies, leading academic institutions, and key regulatory agencies. Certara’s solutions, which span drug
discovery through patient care, increase the probability of regulatory and commercial success by using the most
scientifically-advanced modeling and simulation technologies and regulatory strategies. For more information,
visit www.certara.com. View source version on businesswire.com: http://www.businesswire.com/news/
home/20160525005153/en/
NewsRx LLC
(2016-06-10), Certara and The University of Manchester Co-host Inaugural Model-based Personalized Drug
Dosing in Healthcare Conference, Drug Week, 95, ISSN: 1532-4575, BUTTER® ID: 011897205
Depression
Children of depressed parents at high risk of adverse consequences into adulthood
By a News Reporter-Staff News Editor at Psychology & Psychiatry Journal – New York, NY ()–The latest report
from a 30-year study of families at high- and low-risk for depression reveals that the offspring of depressed
parents have a higher risk for depression, morbidity and mortality that persists into middle age. While major
depression typically begins during adolescence in both high- and low-risk individuals, children with a family
history are more likely to have recurrent episodes of depression and poor outcomes as they mature.
The findings were published in the American Journal of Psychiatry.
Previous reports from this longitudinal study were issued at the 10- and 20-year follow-up periods, when the
offspring were adolescents or young adults. This latest analysis confirms that children of depressed parents
continue to have a three-fold increase in the risk of major depression or anxiety. High-risk offspring with
early-onset depression also had a higher risk of a recurrence after age 20. The low-risk group did not have an
increased risk of recurrence.
148
The study began in 1982, and the last interviews were completed in 2015. There were six waves of interviews,
at baseline and 2, 10, 20, 25, and 30 years. The current analysis included 263 biological children from 91
families who entered the study at wave 1 or 2 and were assessed at wave 5 or 6.
Although the high- and low-risk groups did not show differences in education, employment status, or income
at the 30-year follow-up, those in the high-risk group were more likely to be separated or divorced and had fewer
children. They also received more treatment over a longer period of time, received more continuous treatment
for emotional problems, and had worse overall functioning than those in the low-risk group.
“These findings indicate that a simple family history of assessment of major depression can help identify
individuals at long-term risk for depression,” said Myrna Weissman, PhD, the Diane Goldman Kemper Family
professor of epidemiology (in psychiatry) at Columbia University Medical Center, chief of the division of epidemiology at New York State Psychiatric Institute, and one of the lead authors of the paper. “It has been shown
that even highly efficacious prevention programs for previously depressed adolescents were less effective if the
parent was depressed. Our previous work has shown that treatment of the depressed parent to remission can
reduce the symptoms of depression for both parent and child.”
Ongoing research with this cohort includes neuroimaging studies to better understand the biological underpinnings of depression. In the era of precision medicine, these studies are designed to bring the identification
and treatment of depressed individuals to new levels of accuracy and effectiveness.
NewsRx LLC
(2016-05-28), Children of depressed parents at high risk of adverse consequences into adulthood, Psychology
& Psychiatry Journal, 9, ISSN: 1944-2726, BUTTER® ID: 011743782
Frost & Sullivan
Clinical Grade Wearables Accelerate Growth Opportunities for Internet of Medical Things
IoMT Solutions
By a News Reporter-Staff News Editor at Journal of Engineering – Due to newly commercialized solutions and
pent up demand, wearables dedicated to chronic disease monitoring and other clinical applications are expected
to transform care provision models.
New analysis from Frost & Sullivan, Wearable Technologies in Clinical and Consumer Health ( http://
bit.ly/23X0kj5 ), finds the global healthcare wearable devices market earned revenues of $5.1 billion in 2015
and estimates this to reach $18.9 billion in 2020, at a compound annual growth rate (CAGR) of 29.9 percent.
Meanwhile, consumer health wearables are expected to grow at a CAGR of 27.8 percent, and medical and
clinical-grade wearables are expected to grow at a CAGR of 32.9 percent.
To learn more about Frost & Sullivan’s strategic recommendations and competitive considerations for this
rapidly expanding market opportunity, please visit: http://frost.ly/9e .
“Breakthrough technological innovations in wearable electronics, sensors, alternate power sources and wireless platforms are enabling novel applications that would not have been feasible even five years ago” said Frost
& Sullivan’s Transformational Health Research Director, Venkat Rajan. “Moving beyond basic consumer centric applications, newer wearable devices with more robust and reliable feature sets open a wide spectrum of
clinical use cases.”
While the market for consumer fitness technologies provide large market opportunities and simplified paths
to market, these devices face high degrees of competition and narrow product lifecycles before being supplanted
by the next buzzworthy device. Recognizing these dynamics, developers are investing greater focus towards
medical grade gadgets that provide greater relevancy and reliability in health management.
Clinical grade wearables technologies enable care anywhere-anytime support paradigms. Market dynamics
in high-acuity or other medical use cases dictate attention towards interoperability, affordability and data
accuracy.
“Clinical wearables must concurrently justify their value to payers, patients and clinicians to gain market
foothold,” noted Rajan. “Confidence in the accuracy of collected data is paramount to the utility of information
in any medical decision support.”
Despite this tremendous opportunity, many wearable launches have struggled or failed to achieve expected
traction. One common pitfall, are systems that are over engineered or unnecessarily complex. The effort required on the user side to understand, maintain or properly operate the device would often lead to high abandonment rates after a few months.
149
Wearable Technologies in Clinical and Consumer Health is part of the Visionary Healthcare ( http://
ww2.frost.com/research/industry/transformational-health/visionary-healthcare/ ) Growth
Partnership Service program, which also includes research in the following markets: artificial intelligence,
healthcare patient engagement, retail care environments, aging in place, precision medicine. All research services included in subscriptions provide detailed market opportunities and industry trends evaluated following
extensive interviews with market participants.
NewsRx LLC
(2016-05-30), Clinical Grade Wearables Accelerate Growth Opportunities for Internet of Medical Things
IoMT Solutions, Journal of Engineering, 509, ISSN: 1945-872X, BUTTER® ID: 011758208
PHEMI
Cloud-Based Big Data Warehouses Deliver Unparalleled Benefits for Healthcare
By a News Reporter-Staff News Editor at Information Technology Newsweekly – PHEMI, developer of
enterprise-grade big data privacy, governance, and management solutions, released a new white paper,
Healthcare Big Data Warehouse in the Cloud. Produced in conjunction with healthcare cloud computing
firm, ClearDATA, the white paper illustrates the benefits cloud-based big data warehouses offer healthcare organizations. The Healthcare Big Data Warehouse in the Cloud white paper can be downloaded at:
http://bit.ly/29zggad .
“Healthcare organizations aiming to use all of their data face multiple disparate data silos, which frequently
impedes their ability to locate, access, protect, and derive meaningful insights from that data. By combining
big data solutions that address governance and privacy concerns and consolidate all types of data, with cloud
solutions designed for Healthcare, IT organizations can minimize complexity, and achieve significant bottomline benefits,” said Adam Lorant, vice president, product and solutions, PHEMI. “This white paper shows why
it’s time to bring the speed, scale, and economics of the cloud to healthcare data, enabling organizations to
both uncover actionable insights faster, and ensuring the governance and privacy protections essential to this
industry.”
Data has become increasingly important to clinical, diagnostic, financial, and research operations, as well
as for precision medicine and value-based care initiatives. Modern cloud-based big data solutions incorporate innovative mechanisms simplifying privacy and governance, automating de-identification of Protected
Health Information (PHI) in accordance with Safe Harbor guidelines and the Health Information Trust Alliance (HITRUST) framework. Healthcare Big Data Warehouse in the Cloud reveals how implementing a costefficient cloud-based big data warehouse solution accelerates research and analytics activities, allowing data
to reach researchers and other users quicker, while maintaining data privacy, security, and governance. The
13-page white paper also explores key challenges, the need to balance privacy and data utility, and a checklist
of considerations for selecting a provider.
“Compared with on-premise solutions, cloud-based big data solutions like PHEMI Central in the Cloud and
ClearDATA’s Dynamic Cloud Platform for AWS provide significant advantages and quantifiable benefits, as
detailed in the white paper,” said Scott Whyte, SVP Growth & Innovation, ClearDATA. “Healthcare organizations implementing big data cloud technologies can realize reductions in infrastructure capital costs and a
shorter time-to-insight cycle, in turn optimizing clinical operations and improving quality of care.”
To download the Healthcare Big Data Warehouse in the Cloud white paper, visit http://bit.ly/29zggad
. For more information on PHEMI, follow @PHEMISystems on Twitter, or PHEMI on LinkedIn. About PHEMI
PHEMI develops enterprise-grade big data privacy, governance, and management solutions for healthcare and
other industries. Its flagship product, PHEMI Central, helps organizations capitalize on the explosion of data
types and volumes available to their organization today, driving discovery, innovation, and performance, while
lowering costs, improving outcomes, and allowing better decisions faster. View source version on businesswire.com: http://www.businesswire.com/news/home/20160712006068/en/
NewsRx LLC
(2016-07-26), Cloud-Based Big Data Warehouses Deliver Unparalleled Benefits for Healthcare, Information
Technology Newsweekly, 60, ISSN: 1944-1800, BUTTER® ID: 012106268
150
Cure Forward
Cure Forward Announces First Trial Recruitment Collaboration That Will Expand Patient
Access to Innovative Treatments
By a News Reporter-Staff News Editor at NewsFile – Cure Forward, an online precision medicine platform
for cancer patients and their care teams, and Novartis Pharmaceuticals Corporation announced that Cure
Forward’s unique Clinical Trial Exchange will support Novartis in identifying potential patients for all of its
current oncology trials.
The collaboration further validates Cure Forward’s approach for real-time patient sourcing in clinical trials.
By utilizing its patent pending Clinical Trial Exchange, Cure Forward facilitates connections between patients
and drug development companies, based on mutual criteria and preferences, comparable to popular online
dating platforms.
There is a significant efficiency gap in the clinical trial process today, placing undue burden on patients
and caregivers to navigate the vast, complex landscape of medical research options - often at stages in which
their time is most valuable. Drug developers are concurrently developing investigational treatments, but often
struggle to find the right patients to receive these investigational treatments.
Cure Forward’s platform empowers patients to govern their own care plans, providing accurate, easily accessible information to help drive individual choices.
“Today’s patient is active in his or her care, accessing data to make important treatment decisions,” said
Martin Naley, Founder and Chief Strategy Officer, Cure Forward. “Cure Forward makes clinical trial enrollment easier and more accessible. Novartis’ bold approach of activating searches for oncology trials on our
platform will help patients find possible investigational treatments for their care.”
For more information, please visit CureForward.com. About Cure Forward Cure Forward bridges the gap
between patients and precision medicine, beginning in cancer. Cure Forward provides a simple-to-use platform
that allows patients to obtain their genomic data and use it to identify treatment options including clinical trials, solving critical business needs for diagnostic laboratories, health care providers and clinical trial recruiters.
Cure Forward is based in Cambridge, Massachusetts and was incubated and launched by Apple Tree Partners,
New York City. The company’s platform is in beta release at www.cureforward.com. View source version on
NewsRx LLC
(2016-07-11), Cure Forward Announces First Trial Recruitment Collaboration That Will Expand Patient
Access to Innovative Treatments, NewsFile, 382, ISSN: 0000-0000, BUTTER® ID: 012022625
Rheumatology
Data on Rheumatology Reported by Researchers at Education and Clinical Center
(Biomarkers to guide clinical therapeutics in rheumatology?)
By a News Reporter-Staff News Editor at Journal of Engineering – Investigators publish new report on Rheumatology. According to news reporting originating from Palo Alto, California, by VerticalNews correspondents,
research stated, “The use of biomarkers in rheumatology can help identify disease risk, improve diagnosis
and prognosis, target therapy, assess response to treatment, and further our understanding of the underlying
pathogenesis of disease. Here, we discuss the recent advances in biomarkers for rheumatic disorders, existing
impediments to progress in this field, and the potential of biomarkers to enable precision medicine and thereby
transform rheumatology.”
Our news editors obtained a quote from the research from Education and Clinical Center, “Although significant challenges remain, progress continues to be made in biomarker discovery and development for rheumatic
diseases. The use of next-generation technologies, including large-scale sequencing, proteomic technologies,
metabolomic technologies, mass cytometry, and other single-cell analysis and multianalyte analysis technologies, has yielded a slew of new candidate biomarkers. Nevertheless, these biomarkers still require rigorous
validation and have yet to make their way into clinical practice and therapeutic development. This review focuses on advances in the biomarker field in the last 12 months as well as the challenges that remain. Better
biomarkers, ideally mechanistic ones, are needed to guide clinical decision making in rheumatology. Although
the use of next-generation techniques for biomarker discovery is making headway, it is imperative that the
roadblocks in our search for new biomarkers are overcome to enable identification of biomarkers with greater
diagnostic and predictive utility.”
151
According to the news editors, the research concluded: “Identification of biomarkers with robust diagnostic
and predictive utility would enable precision medicine in rheumatology.”
For more information on this research see: Biomarkers to guide clinical therapeutics in rheumatology? Current Opinion In Rheumatology , 2016;28(2):168-75. (Lippincott Williams and Wilkins - www.lww.com; Current
Opinion In Rheumatology - http://journals.lww.com/co-rheumatology/pages/default.aspx )
The news editors report that additional information may be obtained by contacting W.H. Robinson, aDivision
of Immunology and Rheumatology, CCSR 4135, Stanford bGeriatric Research Education and Clinical Center,
Veterans Affairs Palo Alto Healthcare System, Palo Alto, California, United States.
NewsRx LLC
(2016-06-27), Data on Rheumatology Reported by Researchers at Education and Clinical Center (Biomarkers to guide clinical therapeutics in rheumatology?), Journal of Engineering, 459, ISSN: 1945-872X, BUTTER®
ID: 011952596
Definiens
Definiens to Speak on Big Data Panel at BIO 2016
By a News Reporter-Staff News Editor at Clinical Trials Week – Definiens, the pioneer in Tissue Phenomics
solutions for diagnostics development and commercialization, announced that the company will be a participant in a panel discussing big data operations and R&D efficiencies at the BIO International Convention on
Thursday, June 9th.
Merrilyn Datta, chief commercial officer, Definiens, will be a featured speaker on the panel, entitled “Using
Big Data for Personalizing Medicine: Insights from Genomic Data and Real World Evidence.”
“Big data will be essential to unlocking precision medicine,” said Datta. “By combining tissue data, genomics, and clinical outcomes, we will be able to better match therapies with patients. I look forward to engaging with the other participants at BIO in order to help move this important conversation forward.”
BIO 2016 will be held in San Francisco from June 6-9th, 2016. The panel will take place on June 9th in
room West 3006 from 10:30-11:45am. For more information about the panel, visit www.mybio.org. For more
information about Definiens’ technology, visit www.definiens.com. About Definiens Definiens is the pioneering
provider of Tissue Phenomics® solutions for biomarker and companion diagnostics development and commercialization. Definiens’ technology empowers smarter tissue-based diagnostics by leveraging quantitative tissue
readouts and other big data sources. By enabling the development of powerful and precise assays for patient
stratification and clinical trial enrollment, Definiens aims to dramatically improve patient outcomes. Definiens’
Tissue Phenomics approach was awarded the 2013 Frost and Sullivan Company of the Year Award for Global
Tissue Diagnostics and Pathology Imaging. For more information, please visit: www.definiens.com View source
NewsRx LLC
(2016-06-13), Definiens to Speak on Big Data Panel at BIO 2016, Clinical Trials Week, 247, ISSN: 1543-6764,
BUTTER® ID: 011823355
152
Frost & Sullivan
Emerging Technologies Driving Disruption and Transformation in Health and Wellness
By a News Reporter-Staff News Editor at Drug Week – TechVision, Frost & Sullivan’s technology consulting
division, has identified new technologies impacting the health and wellness sector. These technologies offer a
number of opportunities for innovative pharmaceutical and biotechnology companies to deliver their product to
the market. Innovation plays a key role for companies attempting to find the optimal customer niche. Therefore,
investments in research and development continue leading mainstream strategies.
http://photos.prnasia.com/prnvar/20160611/8521603810
Photo - http://photos.prnewswire.com/prnh/20160610/378012
New TechVision analysis from Frost & Sullivan, Health & Wellness Top 10 Technologies ( http://utm.
io/256638 ), profiles the top 10 technologies impacting the health and wellness sector. New business models
and emerging trends, involving new players and collaboration, are assessed between large pharmaceutical and
medical devices firms.
For complimentary access to more information on this research, please visit: http://frost.ly/h1 .
Medical device and drug manufacturing companies enter long-term partnerships aiming to combine their
drug delivery devices with the most active therapeutics, thereby bringing down the operational costs and saving
a significant amount of time during the process. Similarly, life sciences research, cloud-based bioinformatics
and nanotech-based companies are also partnering with giant pharmaceutical firms to help innovate in new
customer niches and service segments.
More than 8.2 million people died of cancer, while more than 5 million people died of diabetes and other
related disorders in 2015. Poor diet and factors such as pollution, smoking and high stress levels lead to greater
disease burden. The global disease burden and excessive healthcare spending have resulted in numerous research and discovery programs. Increasing application of big data analytics has led to better management of
genomic data, aiding in biomarker discovery and disease understanding.
“A higher concern and commitment among the scientific and clinical communities to provide improved facilities and therapies is essential. The aging population and chronic diseases continue to exhibit a dramatic rise,
spurring the demand for novel solutions in the health and wellness sector,” said Frost & Sullivan TechVision
Industry Analyst Cecilia Van Cauwenberghe. “This is driving public and private support in the funding and
investment of therapeutic areas and breakthrough technologies.”
Early adoption of operational analytics, gene editing, 3D printing, telemedicine, cloud-based computing and
advanced biosensors are poised to ramp innovation efforts to a new level. Enhanced techniques for accelerating
drug discovery have improved target selection, lead identification, preclinical tests, clinical trials, chemical
synthesis, formulations studies and product management.
“Precision medicine has the ability to target diseases with specialized therapies, driving promising solutions. Last-generation transdermal drug delivery technology provides patients with more accurate and precise
therapeutics delivery and monitoring,” noted Van Cauwenberghe. “However, the rapid advancement of micro
and nanotechnologies creates a confident environment among venture capitals and private investors inclined
to fund precision medicine-related innovations.”
Survive and Thrive in an Unpredictable Future! Schedule a Growth Strategy Dialog with the Tech Vision
Global Team to discuss your strategic growth development and discover growth opportunities impacting your
business, here: http://ww2.frost.com/consulting/
Health & Wellness Top 10 Technologies, part of the TechVision subscription, offers a detailed account of the
various emerging manufacturing technologies and their impact on medical device manufacturing in the next
five years. This research service provides trends, insights, and technologies of focus for stakeholders in this
segment. It also presents an overview of technology Mega Trends affecting medical devices. It identifies the
emerging market needs that are likely to shape R&D efforts in the near future.
NewsRx LLC
(2016-07-01), Emerging Technologies Driving Disruption and Transformation in Health and Wellness, Drug
153
Findings from Children’s National Medical Center Update Understanding of Pulmonary
Hypertension (Challenges, priorities and novel therapies for hypoxemic respiratory failure
and pulmonary hypertension in the neonate)
By a News Reporter-Staff News Editor at Clinical Trials Week – Investigators discuss new findings in Lung Diseases and Conditions. According to news reporting out of Washington, District of Columbia, by NewsRx editors,
research stated, “Future priorities for the management of hypoxemic respiratory failure (HRF) and pulmonary
hypertension include primary prevention of neonatal lung diseases, ‘precision medicine’ and translating promising clinical and preclinical research into novel therapies. Promising areas of investigation include noninvasive ventilation strategies, emerging pulmonary vasodilators (for example, cinaciguat, intravenous bosentan,
rho-kinase inhibitors, peroxisome proliferator-activated receptor-gamma agonists) and hemodynamic support
(arginine vasopressin).”
Our news journalists obtained a quote from the research from Children’s National Medical Center, “Research challenges include the optimal timing for primary prevention interventions and development of validated biomarkers that predict later disease or serve as surrogates for long-term respiratory outcomes. Differentiating respiratory disease endotypes using biomarkers and experimental therapies tailored to the underlying pathobiology are central to the concept of ‘precision medicine’ (that is, prevention and treatment strategies
that take individual variability into account). The ideal biomarker should be expressed early in the neonatal
course to offer an opportunity for effective and targeted interventions to modify outcomes. The feasibility of
this approach will depend on the identification and validation of accurate, rapid and affordable point-of-care
biomarker tests. Trials targeting patient-specific pathobiology may involve less risk than traditional randomized controlled trials that enroll all at-risk neonates. Such approaches would reduce trial costs, potentially with
fewer negative trials and improved health outcomes.”
According to the news editors, the research concluded: “Initiatives such as the Prematurity and Respiratory Outcomes Program, supported by the National Heart, Lung, and Blood Institute, provide a framework to
develop refined outcome measures and early biomarkers that will enhance our understanding of novel, mechanistic therapeutic targets that can be tested in clinical trials in neonates with HRF.”
For more information on this research see: Challenges, priorities and novel therapies for hypoxemic respiratory failure and pulmonary hypertension in the neonate. Journal of Perinatology , 2016;36():S32-S36.
Journal of Perinatology can be contacted at: Nature Publishing Group, 75 Varick St, 9TH Flr, New York,
NY 10013-1917, USA. (Nature Publishing Group - http://www.nature.com/ ; Journal of Perinatology http://www.nature.com/jp/ )
Our news journalists report that additional information may be obtained by contacting J.L. Aschner, Childrens Natl Med Center, Dept. of Pediat, Washington, DC 20010, United States. Additional authors for this
research include J. Gien, N. Ambalavanan, J.P. Kinsella, G.G. Konduri, S. Lakshminrusimha, O.D. Saugstad
and R.H. Steinhorn.
NewsRx LLC
(2016-06-20), Findings from Children’s National Medical Center Update Understanding of Pulmonary Hypertension (Challenges, priorities and novel therapies for hypoxemic respiratory failure and pulmonary hypertension in the neonate), Clinical Trials Week, 74, ISSN: 1543-6764, BUTTER® ID: 011909316
Findings from Harvard University in the Area of Meningeal Neoplasms Reported (Genomic
and Epigenomic Landscape in Meningioma)
By a News Reporter-Staff News Editor at Pain & Central Nervous System Week – Current study results on
Nervous System Diseases and Conditions have been published. According to news reporting out of Boston,
Massachusetts, by NewsRx editors, research stated, “Meningiomas are the most common primary intracranial
neoplasms in adults.”
Our news journalists obtained a quote from the research from Harvard University, “Despite their prevalence, their biologic underpinnings remain incompletely described. The recent application of unbiased nextgeneration sequencing and epigenomic approaches has implicated a new array of candidate biomarkers and
oncogenic drivers.”
154
According to the news editors, the research concluded: “These insights may serve to craft a molecular taxonomy for meningiomas and highlight putative therapeutic targets in an era of biology-informed precision
medicine.”
For more information on this research see: Genomic and Epigenomic Landscape in Meningioma. Neurosurgery Clinics of North America , 2016;27(2):167-179,8. Neurosurgery Clinics of North America can be contacted at: W B Saunders Co-Elsevier Inc, 1600 John F Kennedy Boulevard, Ste 1800, Philadelphia, PA 191032899, USA. (Elsevier - www.elsevier.com; Neurosurgery Clinics of North America - http://www.journals.
elsevier.com/neurosurgery-clinics-of-north-america/ )
Our news journalists report that additional information may be obtained by contacting W. Linda, Harvard
University, Sch Med, Dana Farber Canc Inst, Dept. of Canc Biol, 44 Binney St, Boston, MA 02115, United
States. Additional authors for this research include Y. Mei, P.K. Agarwalla, R. Beroukhim and I.F. Dunn.
NewsRx LLC
(2016-05-30), Findings from Harvard University in the Area of Meningeal Neoplasms Reported (Genomic
and Epigenomic Landscape in Meningioma), Pain & Central Nervous System Week, 352, ISSN: 1532-4672,
BUTTER® ID: 011744657
Findings from Harvard University in the Area of Meningeal Neoplasms Reported (Genomic
and Epigenomic Landscape in Meningioma) (Genomic and Epigenomic Landscape in
Meningioma)
By a News Reporter-Staff News Editor at Ivy League Week – Current study results on Nervous System Diseases
and Conditions have been published. According to news reporting out of Boston, Massachusetts, by NewsRx
editors, research stated, “Meningiomas are the most common primary intracranial neoplasms in adults.”
Our news journalists obtained a quote from the research from Harvard University, “Despite their prevalence, their biologic underpinnings remain incompletely described. The recent application of unbiased nextgeneration sequencing and epigenomic approaches has implicated a new array of candidate biomarkers and
oncogenic drivers.”
According to the news editors, the research concluded: “These insights may serve to craft a molecular taxonomy for meningiomas and highlight putative therapeutic targets in an era of biology-informed precision
medicine.”
For more information on this research see: Genomic and Epigenomic Landscape in Meningioma. Neurosurgery Clinics of North America , 2016;27(2):167-179,8. Neurosurgery Clinics of North America can be contacted at: W B Saunders Co-Elsevier Inc, 1600 John F Kennedy Boulevard, Ste 1800, Philadelphia, PA 191032899, USA. (Elsevier - www.elsevier.com; Neurosurgery Clinics of North America - http://www.journals.
elsevier.com/neurosurgery-clinics-of-north-america/ )
Our news journalists report that additional information may be obtained by contacting W. Linda, Harvard
University, Sch Med, Dana Farber Canc Inst, Dept. of Canc Biol, 44 Binney St, Boston, MA 02115, United
States. Additional authors for this research include Y. Mei, P.K. Agarwalla, R. Beroukhim and I.F. Dunn.
NewsRx LLC
(2016-05-31), Findings from Harvard University in the Area of Meningeal Neoplasms Reported (Genomic
and Epigenomic Landscape in Meningioma) (Genomic and Epigenomic Landscape in Meningioma), Ivy League
155
Findings from Harvard University Yields New Findings on Secondary Headache Disorders
(Spreading Depression in Primary and Secondary Headache Disorders)
By a News Reporter-Staff News Editor at Pain & Central Nervous System Week – Researchers detail new data
in Nervous System Diseases and Conditions. According to news originating from Boston, Massachusetts, by
NewsRx correspondents, research stated, “Purpose of Review Spreading depression (SD) is a wave of simultaneous and near-complete depolarization of virtually all cells in brain tissue associated with a transient ‘depression’
of all spontaneous or evoked electrical activity in the brain. SD is widely accepted as the pathophysiological
event underlying migraine aura and may play a role in headache pathogenesis in secondary headache disorders
such as ischemic stroke, subarachnoid or intracerebral hemorrhage, traumatic brain injury, and epilepsy.”
Our news journalists obtained a quote from the research from Harvard University, “Here, we provide an
overview of the pathogenic mechanisms and propose plausible hypotheses on the involvement of SD in primary and secondary headache disorders. SD can activate downstream trigeminovascular nociceptive pathways to explain the cephalgia in migraine, and possibly in secondary headache disorders as well. In healthy,
well-nourished tissue (such as migraine), the intense transmembrane ionic shifts, the cell swelling, and the
metabolic and hemodynamic responses associated with SD do not cause tissue injury; however, when SD occurs in metabolically compromised tissue (e.g., in ischemic stroke, intracranial hemorrhage, or traumatic brain
injury), it can lead to irreversible depolarization, injury, and neuronal death. Recent non-invasive technologies
to detect SDs in human brain injury may aid in the investigation of SD in headache disorders in which invasive
recordings are not possible. SD explains migraine aura and progression of neurological deficits associated with
other neurological disorders.”
According to the news editors, the research concluded: “Studying the nature of SD in headache disorders
might provide pathophysiological insights for disease and lead to targeted therapies in the era of precision
medicine.”
For more information on this research see: Spreading Depression in Primary and Secondary Headache
Disorders. Current Pain and Headache Reports , 2016;20(7):3-10. Current Pain and Headache Reports can be
contacted at: Springer, 233 Spring St, New York, NY 10013, USA. (Springer - www.springer.com; Current Pain
and Headache Reports - http://www.springerlink.com/content/1531-3433/ )
The news correspondents report that additional information may be obtained from S.P. Chen, Harvard
University, Sch Med, Dept. of Neurol, Massachusetts Gen HospNeurosci Intens Care Unit, Boston, MA 02115,
United States.
NewsRx LLC
(2016-07-25), Findings from Harvard University Yields New Findings on Secondary Headache Disorders
(Spreading Depression in Primary and Secondary Headache Disorders), Pain & Central Nervous System Week,
174, ISSN: 1532-4672, BUTTER® ID: 012088679
Findings from Harvard University Yields New Findings on Secondary Headache Disorders
(Spreading Depression in Primary and Secondary Headache Disorders) (Spreading
Depression in Primary and Secondary Headache Disorders)
By a News Reporter-Staff News Editor at Ivy League Week – Researchers detail new data in Nervous System Diseases and Conditions. According to news originating from Boston, Massachusetts, by NewsRx correspondents, research stated, “Purpose of Review Spreading depression (SD) is a wave of simultaneous and
near-complete depolarization of virtually all cells in brain tissue associated with a transient ‘depression’ of all
spontaneous or evoked electrical activity in the brain. SD is widely accepted as the pathophysiological event
underlying migraine aura and may play a role in headache pathogenesis in secondary headache disorders such
as ischemic stroke, subarachnoid or intracerebral hemorrhage, traumatic brain injury, and epilepsy.”
Our news journalists obtained a quote from the research from Harvard University, “Here, we provide an
overview of the pathogenic mechanisms and propose plausible hypotheses on the involvement of SD in primary and secondary headache disorders. SD can activate downstream trigeminovascular nociceptive pathways to explain the cephalgia in migraine, and possibly in secondary headache disorders as well. In healthy,
well-nourished tissue (such as migraine), the intense transmembrane ionic shifts, the cell swelling, and the
156
metabolic and hemodynamic responses associated with SD do not cause tissue injury; however, when SD occurs in metabolically compromised tissue (e.g., in ischemic stroke, intracranial hemorrhage, or traumatic brain
injury), it can lead to irreversible depolarization, injury, and neuronal death. Recent non-invasive technologies
to detect SDs in human brain injury may aid in the investigation of SD in headache disorders in which invasive
recordings are not possible. SD explains migraine aura and progression of neurological deficits associated with
other neurological disorders.”
According to the news editors, the research concluded: “Studying the nature of SD in headache disorders
might provide pathophysiological insights for disease and lead to targeted therapies in the era of precision
medicine.”
For more information on this research see: Spreading Depression in Primary and Secondary Headache
Disorders. Current Pain and Headache Reports , 2016;20(7):3-10. Current Pain and Headache Reports can be
contacted at: Springer, 233 Spring St, New York, NY 10013, USA. (Springer - www.springer.com; Current Pain
and Headache Reports - http://www.springerlink.com/content/1531-3433/ )
The news correspondents report that additional information may be obtained from S.P. Chen, Harvard
University, Sch Med, Dept. of Neurol, Massachusetts Gen HospNeurosci Intens Care Unit, Boston, MA 02115,
United States.
NewsRx LLC
(2016-07-26), Findings from Harvard University Yields New Findings on Secondary Headache Disorders
(Spreading Depression in Primary and Secondary Headache Disorders) (Spreading Depression in Primary and
Secondary Headache Disorders), Ivy League Week, 77, ISSN: 0000-0000, BUTTER® ID: 012105957
Health and Medicine
Findings from State University of New York Broaden Understanding of Experimental
Biology and Medicine (Multiomic candidate biomarkers for clinical manifestations of sickle
cell severity: Early steps to precision medicine)
By a News Reporter-Staff News Editor at Health & Medicine Week – Investigators publish new report on Health
and Medicine. According to news originating from Syracuse, New York, by NewsRx correspondents, research
stated, “In this review, we provide a description of those candidate biomarkers which have been demonstrated
by multiple-omics approaches to vary in correlation with specific clinical manifestations of sickle cell severity.
We believe that future clinical analyses of severity phenotype will require a multiomic analysis, or an omics
stack approach, which includes integrated interactomics.”
Our news journalists obtained a quote from the research from the State University of New York, “It will
also require the analysis of big data sets. These candidate biomarkers, whether they are individual or panels of functionally linked markers, will require future validation in large prospective and retrospective clinical
studies. Once validated, the hope is that informative biomarkers will be used for the identification of individuals most likely to experience severe complications, and thereby be applied for the design of patient-specific
therapeutic approaches and response to treatment.”
According to the news editors, the research concluded: “This would be the beginning of precision medicine
for sickle cell disease.”
For more information on this research see: Multiomic candidate biomarkers for clinical manifestations of
sickle cell severity: Early steps to precision medicine. Experimental Biology and Medicine , 2016;241(7):772781. Experimental Biology and Medicine can be contacted at: Sage Publications Ltd, 1 Olivers Yard, 55 City
Road, London EC1Y 1SP, England.
The news correspondents report that additional information may be obtained from S.R. Goodman, State
University of New York, Dept. of Psychiat & Behav Sci, Syracuse, NY 13210, United States. Additional authors
for this research include B.S. Pace, K.C. Hansen, A. D’alessandro, Y. Xia, O. Daescu and S.J. Glatt.
NewsRx LLC
(2016-06-03), Findings from State University of New York Broaden Understanding of Experimental Biology
and Medicine (Multiomic candidate biomarkers for clinical manifestations of sickle cell severity: Early steps to
precision medicine), Health & Medicine Week, 4471, ISSN: 1532-4605, BUTTER® ID: 011792224
157
Pharmacoeconomics
Findings in Pharmacoeconomics Reported from University of Western Ontario (Cost
Implications of Value-Based Pricing for Companion Diagnostic Tests in Precision Medicine)
By a News Reporter-Staff News Editor at Economics Week – New research on Pharmacoeconomics is the subject of a report. According to news reporting originating from London, Canada, by VerticalNews editors, the
research stated, “Many interpretations of personalized medicine, also referred to as precision medicine, include discussions of companion diagnostic tests that allow drugs to be targeted to those individuals who are
most likely to benefit or that allow treatment to be designed in a way such that individuals who are unlikely to
benefit do not receive treatment. Many authors have commented on the clinical and competitive implications of
companion diagnostics, but there has been relatively little formal analysis of the cost implications of companion
diagnostics, although cost reduction is often cited as a significant benefit of precision medicine.”
Our news editors obtained a quote from the research from the University of Western Ontario, “We investigate
the potential impact on costs of precision medicine implemented through the use of companion diagnostics.
We develop a framework in which the costs of companion diagnostic tests are determined by considerations
of profit maximization and cost effectiveness. We analyze four scenarios that are defined by the incremental
cost-effectiveness ratio of the new drug in the absence of a companion diagnostic test.”
According to the news editors, the research concluded: “We find that, in most scenarios, precision medicine
strategies based on companion diagnostics should be expected to lead to increases in costs in the short term
and that costs would fall only in a limited number of situations.”
For more information on this research see: Cost Implications of Value-Based Pricing for Companion Diagnostic Tests in Precision Medicine. Pharmacoeconomics , 2016;34(7):635-644. Pharmacoeconomics can be
contacted at: Adis Int Ltd, 5 The Warehouse Way, Northcote 0627, Auckland, New Zealand.
The news editors report that additional information may be obtained by contacting G.S. Zaric, University
of Western Ontario, Ivey Business Sch, London, ON N6A 3K7, Canada.
NewsRx LLC
(2016-07-15), Findings in Pharmacoeconomics Reported from University of Western Ontario (Cost Implications of Value-Based Pricing for Companion Diagnostic Tests in Precision Medicine), Economics Week, 86,
ISSN: 1945-6913, BUTTER® ID: 012051155
Intestinal Disease
Findings on Intestinal Disease Detailed by Investigators at Pasteur Institute
(Gastrointestinal tract: the leading role of mucosal immunity)
By a News Reporter-Staff News Editor at Gastroenterology Week – A new study on Intestinal Disease is now
available. According to news reporting originating from Lille, France, by NewsRx correspondents, research
stated, “An understanding of mucosal immunity is essential for the comprehension of intestinal diseases that
are often caused by a complex interplay between host factors, environmental influences and the intestinal
microbiota. Not only improvements in endoscopic techniques, but also advances in high throughput sequencing
technologies, have expanded knowledge of how intestinal diseases develop.”
Our news editors obtained a quote from the research from Pasteur Institute, “This review discusses how the
host interacts with intestinal microbiota by the direct contact of host receptors with highly conserved structural
motifs or molecules of microbes and also by microbe-derived metabolites (produced by the microbe during adaptation to the gut environment), such as short-chain fatty acids, vitamins, bile acids and amino acids. These
metabolites are recognised by metabolite-sensing receptors expressed by immune cells to influence functions of
macrophages, dendritic cells and T cells, such as migration, conversion and maintenance of regulatory T cells
and regulation of proinflammatory cytokine production, which is essential for the maintenance of intestinal
homeostasis and the development of intestinal diseases, such as inflammatory bowel diseases. First interventions in these complex interactions between microbe-derived metabolites and the host immune system for the
treatment of gastrointestinal diseases, such as modification of the diet, treatment with antibiotics, application
of probiotics and faecal microbiota transplantation, have been introduced into the clinic. Specific targeting of
metabolite sensing receptors for the treatment of gastrointestinal diseases is in development.”
According to the news editors, the research concluded: “In future, precision medicine approaches that consider individual variability in genes, the microbiota, the environment and lifestyle will become increasingly
important for the care of patients with gastrointestinal diseases.”
158
For more information on this research see: Gastrointestinal tract: the leading role of mucosal immunity.
Swiss Medical Weekly , 2016;146():36-48. Swiss Medical Weekly can be contacted at: E M H Swiss Medical
Publishers Ltd, Farnsburgerstr 8, Ch-4132 Muttenz, Switzerland.
The news editors report that additional information may be obtained by contacting A. Steinert, Inst Pasteur,
Center Infect & Immun Lille, Lille, France. Additional authors for this research include K. Radulovic and J.H.
Niess.
NewsRx LLC
(2016-05-30), Findings on Intestinal Disease Detailed by Investigators at Pasteur Institute (Gastrointestinal
tract: the leading role of mucosal immunity), Gastroenterology Week, 117, ISSN: 1543-6748, BUTTER® ID:
011749969
Frontage Laboratories, Inc.
Frontage Laboratories Announces Partnership with Quanterix and Validation of Simoa HD-1
Analyzer and Software to Support Biomarker Analysis
By a News Reporter-Staff News Editor at Computer Weekly News – Frontage Laboratories, Inc., a Contract
Research Organization based in Pennsylvania, New Jersey and China, announced that they are the first CRO to
complete a GLP validation of the Simoa HD-1 Analyzer and software along with the interface to Watson LIMS.
Data will be generated, processed and stored in a secure environment meeting the compliance requirements
needed to support biomarker analysis for regulated studies.
In February 2016, Frontage became Quanterix’ Preferred Partner in biomarker assay development and
testing services in the regulated space. Validation entailed assessing the system against both business and
regulatory requirements, including 21 CFR Part 11, working with Quanterix on how to best use the available
features in a regulated environment, developing specific user requirements and test scripts, and successfully
executing the validation test scripts. The Simoa software application includes compliance features such as audit
trails, controlled user access, configurable security roles, a secure data base and a Simoa Desktop Shield add-on
which limits a users’ ability to access workstation files. “This is another important milestone for our partnership
with Frontage and reinforces both parties’ commitment to ensuring compliancy with regulatory standards for
biomarker analysis,” said Kevin Hrusovsky, CEO and Executive Chairman at Quanterix. “In collaboration with
Frontage, we look forward to providing organizations with the tools and support necessary to better determine
the efficacy of disease treatment, ultimately supporting meaningful advancements in precision medicine.”
“We are excited to announce that the Quanterix Simoa HD-1 Analyzer and software are now fully validated
in Frontage, which allows the biomarker analysis to support drug discovery and development not only in an
exploratory manner, but also in the regulated environment. We are happy to be one of the preferred partners of Quanterix by incorporating Simoa’s ultrasensitive single molecule measurement into our platforms of
biomarker services, which allows us to meet the biomarker services needs of our sponsors,” said Dr. John Lin,
Senior Vice President, Bioanalytical and Biologics Services, Frontage Laboratories, Inc. Keywords for this news
article include: Software, Frontage Laboratories Inc..
NewsRx LLC
(2016-06-29), Frontage Laboratories Announces Partnership with Quanterix and Validation of Simoa HD-1
Analyzer and Software to Support Biomarker Analysis, Computer Weekly News, 297, ISSN: 1944-1606, BUTTER® ID: 011968742
159
Guardant Health
Guardant Health Announces Project LUNAR, An Ambitious Multi-arm, Multi-site Umbrella
Trial to Study Blood Test for Early Cancer Detection
By a News Reporter-Staff News Editor at Clinical Trials Week – Guardant Health announced the launch of an
ambitious initiative, Project LUNAR, which will extend the technology behind the company’s market-leading
comprehensive liquid biopsy, Guardant360, into early-stage cancer detection.
https://photos.prnewswire.com/prnvar/20140212/SF64352LOGO
The company is partnering with leading academic, industrial, and patient advocacy groups to study what
would be a novel, first-in-class technology.
“The existence of a minimally invasive, highly sensitive and specific test for early-stage cancer holds the
promise of a universal screening test that could change the face of cancer treatment forever,” said Guardant
Health co-founder and CEO Helmy Eltoukhy, PhD. “In fact, the development of such a test has been Guardant
Health’s goal from day one. We started with late stage disease, and over the last four years have worked
methodically through iterative technology development to arrive at this point. We understand that with any
promise of this magnitude comes a responsibility to fully understand its implications, so we continue to bring
in experts from advocacy, academia, industry, and public health to support our efforts.”
Oncologists and researchers have long sought a minimally invasive, highly sensitive and specific multicancer test as a more effective way to catch cancer in its earliest stages, when treatment and prognosis are
most promising.
“As a BRCA-carrier with a family history of pancreatic cancer, I know just how important a highly sensitive
and specific test can be for a patient,” said Dr. Pamela Munster, a UC San Francisco clinical oncologist and
co-director of the Center for BRCA Research at UCSF, and one of the Principal Investigators of the LUNAROvarian study. “Particularly for cancers where prophylactic surgeries may not be acceptable, early detection
can save lives and can also alleviate anxiety, as well as guide specific screening. Cancer prognosis is nearly
always better when the disease is detected early.”
LUNAR is an umbrella protocol that Guardant Health is launching with researchers from the Massachusetts
General Hospital, Perelman School of Medicine at the University of Pennsylvania, the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, UC San Francisco, Samsung Medical Center, the University of Colorado Anschutz Medical Campus, and other institutions that will study the ability of Guardant
Health’s technology to detect cancer at early stages in high-risk populations. Guardant Health has already
collected samples from multiple trial sites in breast, ovarian, lung, pancreatic, and colorectal cancers, with
pilot data expected in the second half of 2016. Guardant expects to enroll thousands of patients in multi-site,
multi-arm prospective clinical trials that will demonstrate first the feasibility and then efficacy of early detection of the deadliest cancers, through the integrated use of cell free DNA, imaging, germline risk assessment,
and other highly complementary technologies.
“The LUNAR technology, with its great sensitivity necessary for early detection, will establish a new frontier in cancer diagnostics, allowing use of a biological signal instead of standard imaging, and possibly one
day replacing invasive procedures,” said Dr. Massimo Cristofanilli, Associate Director for Precision Medicine
and Translational Research at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University
and Professor of Medicine at Northwestern University Feinberg School of Medicine, and one of the Principal
Investigators of the LUNAR-Breast study. “The potential applications in breast cancer and other solid tumors
can go far beyond early detection to monitor the efficacy and detect resistance to systemic therapies in adjuvant
and neo-adjuvant settings.”
The company presented an early proof-of-concept study to the American Association for Cancer Research in
April that demonstrated its ability to detect cancer in 86% of early-stage colorectal cancer patients with ultrahigh specificity. Project LUNAR (an acronym for Liquid biopsy Using NGS to Assay high-Risk patients) will
use a newer, more sensitive version of the company’s technology than was used in that trial.
“The knowledge gained from processing tens of thousands of Guardant360 patient samples has enabled
us to iteratively push the performance limits of our technology to develop an affordable LUNAR assay that
can detect a single mutated DNA fragment among hundreds of thousands of genome copies,” said Guardant
Health President and co-founder AmirAli Talasaz, PhD. “Such a test may potentially have a clinical sensitivity
approaching 90% for many cancer types, and could be priced in the hundreds of dollars.”
Guardant Health has established its advanced cancer product, Guardant360, as best in class in terms of
both sensitivity and specificity, with validated analytical specificity of 99.9999%. High specificity is critical
when screening asymptomatic people who are at a high risk for developing cancer.
160
“False positive findings cause real harm, both in terms of anxiety and the unnecessary invasive procedures
they often result in, and have been the downfall of many screening methodologies, especially for lung cancer,”
said Dr. Anil Vachani, Associate Professor of Medicine at the Perelman School of Medicine at the University of
Pennsylvania, and one of the Principal Investigators of the LUNAR-Lung study. “Guardant’s ctDNA approach
may provide the positive predictive value we need to assist in the management of suspicious findings frequently
identified using currently employed imaging studies in oncology practice.”
There are two reasons why Guardant Health expects its approach to have superb positive predictive value
for cancer. First, while many screening signals, like dense tissue on a scan, or an elevated protein biomarker
like CEA or PSA, are loosely correlated with a particular cancer, oncogenically-altered DNA is considered to be
the major universal pathogenic driver of the disease. It is causal, not correlative. Second, Guardant Health’s
digital sequencing platform uses advanced molecular biology, novel chemistry, and a bioinformatics digital
inference engine to maximize nucleic acid yield and nearly eliminate the errors intrinsic to sequencing.
The potential for extremely high specificity is exciting for the many patient advocacy groups that are learning
more about project LUNAR. Eltoukhy said, “The voice of the patient is critical in developing screening methodologies that are not only clinically and scientifically sound, but reflect the real-world needs of patients. That’s
why Guardant Health has built an advisory committee with the Bonnie J. Addario Lung Cancer Foundation,
The BRCA Foundation, Bright Pink, HeritX, and the Lung Cancer Alliance.”
Guardant Health will be kicking off Project LUNAR at an event in San Francisco on May 26 that will
feature dozens of leading experts and advocates in oncology including a panel conversation between Dr. Eric
Topol, endowed Chair of Innovative Medicine at Scripps, and many of the project’s collaborators.
NewsRx LLC
(2016-06-06), Guardant Health Announces Project LUNAR, An Ambitious Multi-arm, Multi-site Umbrella
Trial to Study Blood Test for Early Cancer Detection, Clinical Trials Week, 467, ISSN: 1543-6764, BUTTER®
ID: 011861243
Intertrust Technologies Corporation
Haystack Bio and Intertrust Genecloud Partner for Precision Medicine Analytics
By a News Reporter-Staff News Editor at Journal of Engineering – Intertrust Technologies Corporation and
Haystack Bio, a pioneer in immuno-technologies, announced that Haystack will use Intertrust’s Genecloud
platform for trusted healthcare analytics. Under the partnership, Haystack Bio will use Genecloud to securely
store, analyze and share data collected from Haystack Bio’s advanced single-cell discovery platform.
Immunotherapies and genomic medicines are transforming how we treat cancer, infectious disease, and
autoimmune diseases. Drugs and vaccines that harness the immune system are rapidly emerging as the foundation for the next generation of precision and cellular medicines. Founded by leaders in the field of single-cell
genomics from MIT and Berkeley, Haystack Bio has developed a revolutionary analysis platform that incorporates breakthrough cell imaging, genomics and advanced bioinformatics. Building on this platform, Haystack
is creating a unique immunology knowledge-base and discovery engine that is yielding novel insights to drive
the next generation of therapies.
Genecloud is a distributed Big Data platform for trusted storage and analysis of genomic and other health
information. Intertrust built Genecloud to enable collaboration within and between institutions to address
issues such as genomic privacy by allowing users to interact with sensitive data only through secured computer programs - trusted analytics - that are governed by stakeholder policies. Genecloud ensures that all
customer data is persistently protected and accessible only to those with demonstrable rights to interact with
it. Sophisticated auditing mechanisms guarantee that all accesses are logged to support customer compliance
efforts.
“Haystack Bio’s platform will be used by biopharma partners and research institutions around the world. We
believe that Genecloud is the best way to store, analyze and collaborate around data, maximizing productivity
while protecting privacy. Data governance is essential for genomics, and nobody addresses this challenge as
deeply or as systematically as Genecloud,” said Jim Flanigon, CEO of Haystack Bio.
“We are very excited to partner with Haystack Bio in their mission to bring cutting edge single-cell analysis
into mainstream drug development,” said Knox Carey, vice president of technology initiatives at Intertrust
and general manager of Genecloud. “Our partnership will allow Haystack and its customers to collaborate securely and develop novel insights with firm assurances that their data and intellectual property are absolutely
protected.” About Intertrust Intertrust provides trusted computing products and services to leading global
161
corporations - from mobile and CE manufacturers and service providers to enterprise software platform companies. These products include the world’s leading digital rights management, software tamper resistance and
privacy-driven data platforms for media and entertainment, ad tech, healthcare and IoT.
Founded in 1990, Intertrust is based in Silicon Valley, with regional offices in London and Beijing. The
Company has a legacy of invention, and its fundamental contributions in the areas of computer security and
digital trust are globally recognized. Intertrust holds hundreds of patents that are key to Internet security,
trust and privacy management components of operating systems, trusted mobile code and networked operating
environments, web services and cloud computing. Additional information is available at intertrust.com. Follow
Intertrust on Twitter or LinkedIn. About Haystack Bio Haystack Bio is an immuno-discovery and engineering
company for precision cellular medicines. We employ a fully integrated and proprietary single-cell platform
that includes breakthrough single-cell profiling, cloud-based data processing and analytics. The result is an
immuno-knowledgebase and discovery engine that uniquely enables us to generate novel insights from complex
samples-needles in haystacks-and then use this information to help stratify and engineer more durable immune
medicines. Additional information is available at haystackbio.com. View source version on businesswire.com:
NewsRx LLC
(2016-05-30), Haystack Bio and Intertrust Genecloud Partner for Precision Medicine Analytics, Journal of
Engineering, 958, ISSN: 1945-872X, BUTTER® ID: 011758687
HIV/AIDS
HIV study confirms clinically viable vaccine paving the way for future treatments
By a News Reporter-Staff News Editor at AIDS Weekly – A new scientific study conducted by a team of leading
AIDS scientists reveal results that lead the way to the development of an effective human vaccine against
human immunodeficiency virus (HIV). In the study published in Nature Medicine, researchers worked with a
species of Old World monkeys, rhesus macaques to reproduce the trial results of RV144, the only HIV vaccine
that has been tested and shown to reduce the rate of HIV acquisition in a phase III clinical trial.
Researchers were interested in not only recapitulating the findings from the RV144 trial, but also determining if replacing the alum adjuvant–a substance commonly found in non-living vaccines known to induce
antibody-mediated immunity–with a different adjuvant, MF59 would decrease simian immunodeficiency virus
(SIV) acquisition at an increased rate and yield a more efficacious vaccine. Although MF59 is known to help
stimulate the human body’s immune response, the idea that it could lead to greater vaccine efficacy is a popular
working theory shared by scientists worldwide.
Rafick-Pierre Se?kaly, PhD, the Richard J. Fasenmyer Professor of Immunopathogensis, is a pioneering
AIDS researcher, co-director of the CFAR Proteomics and Systems Biology Core, a professor of pathology at
Case Western Reserve University School of Medicine. He helped to lead this study which simulated potential
HIV infection by rectally challenging the macaques with SIV, the primate form of HIV. Simulation in macaques
has been a critical step in progressing HIV research, since the virus does not replicate well in most other model
organisms.
With this simulation complete, researchers were able to successfully reproduce the RV144 trial results in
macaques, ultimately finding that the RV144 vaccine combined with the alum adjuvant reduced the risk of
acquisition by 44 percent, which is within the range of the 31 percent efficacy previously measured in humans.
However, researchers were surprised to learn that changing the adjuvant to MF59 did not result in a significantly better vaccine, but instead rendered the altered vaccine unable to prevent SIV acquisition at a greater
rate and triggered an adaptive immune response only at the site of infection, thereby disproving the commonly
believed hypothesis. Importantly, researchers were able to uncover more about the alum group’s mode of action in the RV144 vaccine which, to-date, is not fully understood. Researchers discovered a unique association
between an intercellular pathway known as Ras-Raf-MEK-ERK (RAS) and the efficacy of RV144. Ten of twelve
genes associated with the RAS pathway were expresses within the vaccine and have been shown to trigger
several subsets of innate and adaptive responses that, in turn, are associated with a decreased risk of SIV acquisition in the alum-vaccine group. Whether RAS activation is an important hallmark of HIV-vaccine efficacy
in humans is an area for further study.
According to Dr. Sekaly, “These fascinating clinical results effectively dispel our previous belief that the
RV144 vaccine could possibly become more effective with the MF59 adjuvant. Instead, we found that the modified vaccine actually triggered the recruitment of innate cells in the site of infection. Through this research, we
162
were able to confirm the efficacy of the current RV144 vaccine in preventing infection by HIV/SIV in macaques,
creating an even clearer pathway to the near-term development of this vaccine for human use.”
Based on the pre-vaccination RNA expression of the macaque, Dr. Sekaly and his team were also able to
accurately predict whether or not the animal was going to respond to the RV144 vaccine in alum, correctly
forecasting the response in two thirds of the macaques tested. These findings have tremendous implications for
the use of personalized medicine in vaccinology, an exciting exploration of genomics that is already underway
in the scientific community.
“We have seen that pre-selecting subjects that will benefit from a therapy based on their predicted response
or risk of disease is an excellent way to improve potential outcomes,” said Dr. Sekaly. “These study results
strongly support the notion that personalized and predictive vaccinology will soon become a reality, including
in HIV–a disease area for which this type of precision medicine is desperately needed but has not yet been
extensively studied.”
NewsRx LLC
(2016-07-25), HIV study confirms clinically viable vaccine paving the way for future treatments, AIDS
Hortonworks, Inc.
Hortonworks Initiates Precision Medicine Consortium to Explore Next Generation Genomics
Open Source Platform
By a News Reporter-Staff News Editor at Journal of Engineering – (Hadoop Summit) – Hortonworks, Inc.( ®)
(NASDAQ: HDP), a leading innovator of open and connected data platforms, announced the formation of a new
consortium to define and develop an open source genomics platform to accelerate genomics-based precision
medicine in research and clinical care. Other founding members include Arizona State University, Baylor
College of Medicine, Booz Allen Hamilton, Mayo Clinic, OneOme and Yale New Haven Health.
http://photos.prnewswire.com/prnvar/20140227/SF73721LOGO
Harnessing the full potential of precision medicine demands a platform to store a massive volume of genomic
information, an analytic interface and real time querying at scale, among other requirements. The consortium
will further define these requirements and address the limitations of current technologies through an open and
collaborative approach using Design Thinking. It will also leverage knowledge across multiple domains including medical genetics, programming, high performance computing and big data to ensure that organizations of
any size have the opportunity to store and analyze genomics in a single platform. At Hadoop Summit in San
Jose, CA, the consortium members will launch the collaborative effort to define an open-source platform for
genomics.
The announcement furthers Hortonworks’ support for the White House’s Precision Medicine Initiative. Hortonworks is committed to providing its expertise in Connected Data Platforms and building open communities
to address the challenges and opportunities of precision medicine.
“Unleashing the power of data through open community and collaboration is the right approach to solve a
complex problem like precision medicine,” said DJ Patil, chief data scientist, White House Office of Science and
Technology Policy. “Initiatives like this one will break data silos and share data in an open platform across
industries to speed genomics-based research and ultimately save lives.”
“The scale and challenges of bringing precision medicine to life requires a united front across the technology,
research and healthcare industries,” said Vishal Dhanuka, vice president of Innovation and Strategy, Hortonworks. “Hortonworks is proud to help define and design an open source genomics platform to save lives through
genomics-based medicine.”
“This open-source solution will accelerate genomic processing, further enabling precision medicine,” states
Kevin Vigilante, chief medical officer, Booz Allen. “Booz Allen is dedicating our health experts, data scientists,
and technologists to tackle the challenge of bringing innovation to healthcare.”
“To truly achieve precision medicine in everyday patient care, we need solutions that can manage and analyze massive amounts of genomics and clinical information in near real-time,” states Jason Ross, senior director
of EMR Integration, OneOme. “We believe that this consortium and open-source platform will help accelerate
personalized healthcare solutions like pharmacogenomics into routine clinical care.”
“The joint efforts of the leading thought leaders from genomics research and open source technology will
enable the personalized medicine revolution through a diverse collection of analytic tools,” said Jay Etchings,
director of operations for research computing and senior HPC architect at Arizona State University. “This
163
defining strategy is going to fuel novel genomic analysis and near-seamless integration of multidimensional
molecular and clinical data, and we are extremely excited to be part of this consortium.”
NewsRx LLC
(2016-07-11), Hortonworks Initiates Precision Medicine Consortium to Explore Next Generation Genomics
Open Source Platform, Journal of Engineering, 660, ISSN: 1945-872X, BUTTER® ID: 012026289
Ignyta, Inc.
Ignyta Secures $42 Million Term Loan Facility
By a News Reporter-Staff News Editor at NewsFile – Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company
focused on precision medicine in oncology, announced that it has secured a $42 million term loan facility from
Silicon Valley Bank and Oxford Finance. Under the loan facility, the company received initial funding of $32
million, substantially all of which was used to repay the company’s prior loan with Silicon Valley Bank, and
has a conditional option to receive an additional $10 million.
“This loan facility strengthens our balance sheet, enabling us to aggressively pursue the development of
our lead program, entrectinib, and continue the development of the rest of our precision medicine oncology
pipeline,” said Jacob Chacko, M.D., CFO of Ignyta. “We appreciate the support Silicon Valley Bank and Oxford
Finance are providing to us as we continue to grow.”
The loan agreement, in addition to customary conditions, provides that the second tranche of $10 million
may be drawn down by Ignyta at any time from April 7, 2017 to August 31, 2017, provided that Ignyta has
satisfied certain conditions to trigger the second tranche.
Borrowings under the credit facility will bear interest at a rate of Prime + 4.35%, and have interest only
payments for twenty-four months, followed by an amortization period of thirty-six months. The interest only
period will be extended by an additional six months in the event Ignyta has satisfied certain conditions to trigger
the six-month extension. In the event that the interest only period is extended, then the amortization period
will be reduced to thirty months. In connection with entering the credit facility, Ignyta also agreed to issue
warrants to Silicon Valley Bank and Oxford Finance equal to 1.5% of the funded amount, with an exercise
price equal to the lower of the ten-day average price of Ignyta’s common stock prior to funding or the price
per share on the day prior to funding. About Ignyta, Inc. At Ignyta, we fight cancer - a formidable opponent
that manifests as thousands of different molecularly defined diseases and takes away millions of lives globally,
every year. In this fight, our vision is not just to shrink tumors but to eradicate residual disease - the source
of cancer relapse and recurrence - in precisely defined patient populations by 2030. We will work tirelessly
to achieve this vision by pursuing an integrated therapeutic (Rx) and companion diagnostic (Dx) strategy for
treating cancer patients. Our Rx efforts are focused on discovering, in-licensing or acquiring, then developing
and commercializing, molecularly targeted therapies that, sequentially or in combination, are foundational
for eradicating residual disease. Our Dx efforts aim to pair these product candidates with biomarker-based
companion diagnostics that are designed to precisely identify, at the molecular level, the patients who are most
likely to benefit from the therapies we develop. We believe that only through this integrated Rx/Dx approach
can we succeed in this fight.
NewsRx LLC
(2016-07-11), Ignyta Secures $42 Million Term Loan Facility, NewsFile, 524, ISSN: 0000-0000, BUTTER®
ID: 012022794
164
Biotechnology Companies
Illumina Announces Initial Customer Orders for the Global Screening Array
By a News Reporter-Staff News Editor at Biotech Business Week – Illumina, Inc. (NASDAQ: ILMN) announced
that it has signed deals with 12 customers for its new Infinium® Global Screening Array (GSA). In total, the
company has received orders for more than 3 million samples of the new consortia-developed array. Initial
customers include human disease researchers at The Broad Institute and deCODE Genetics, health systems
Avera Health, Codigo46, Diagnomics, Eone Diagnomics Genome Center (EDGC), Sanford Health and UCLA
Health System, genomic service providers Centre National de Genotypage, Human Genomics Facility HuGeF,
Erasmus MC, Life and Brain, and consumer genomics company 23andMe, Inc.
“The array content includes highly predictive hand-curated content, as well as high value markers for translational research applications and sample quality control (QC) designed to be useful across a broad range of
applications, populations and diseases,” said Benjamin Neale, PhD, Assistant Professor, Analytic and Translational Genetics Unit, Massachusetts General Hospital and The Broad Institute, who led the predictive content
selection for the consortia.
“We were impressed that the GSA included content applicable to a range of clinical research activities across
our healthcare ecosystem. For the moment, this is an exceptional research opportunity,” said Dan Geschwind,
MD, PhD, Gordon and Virginia MacDonald Distinguished Professor in Neurology, Psychiatry and Human Genetics and Senior Associate Dean and Associate Vice Chancellor for Precision Medicine, at UCLA. “As genetics
and genomics becomes incorporated into clinical practice in the future, we expect to be able to use these data
to make the care that we deliver in UCLA health more personalized.”
The GSA is a highly economical tool for genetic risk screening of large global populations. With volume
discounts enabling price points below $40 per sample, it offers unparalleled genomic coverage and imputation performance across 26 continental populations and features approximately 50,000 hand-curated variants
relevant to clinical research, including markers for pharmacogenomics, newborn screening research, risk profiling and confirmation of putative clinical associations. Leveraging the 24-sample Infinium format, the array
includes 660,000 markers, and allows for the cost-effective addition of up to 50,000 custom markers.
“The early adoption of the GSA, represented by these deals, illustrates the widespread market demand for
genotyping products and the continued relevance of arrays in human disease and translational research,” said
Rob Brainin, Vice President and General Manager, Applied Genomics at Illumina. “We expect that the value of
the content on this array will lead to widespread use in clinical research, including precision medicine programs,
predictive risk screening, large scale genome-wide association studies, and in biobank sample characterization
and quality control.”
NewsRx LLC
(2016-06-27), Illumina Announces Initial Customer Orders for the Global Screening Array, Biotech Business
Interpreta, Inc.
Interpreta to Exhibit at 2016 AHIP Institute & Expo and Demonstrate How Healthcare
Analytics Delivered Daily Improves Care Management for Health Plans
By a News Reporter-Staff News Editor at Journal of Engineering – (AHIP Booth #358) - Interpreta, Inc., an
analytics service company that delivers population and single member analytics for population health and
precision medicine, will be exhibiting at AHIP Institute & Expo in Las Vegas from June 15 - 17.
Company representatives will demonstrate for health plans how daily re-computation of member and population data, combined with genomic data, delivers clinical analytics that identify prospective (Healthcare
Effectiveness Data and Information Set) HEDIS and pay for performance (P4P) gaps in care, as well as assist
with Medicare risk adjustment. When delivered to care teams as daily updates in real time, care managers
can quickly identify, prioritize and manage member care needs for better coordinated care between members,
payers and caregivers. Workflow is improved and more efficient so care managers can efficiently prioritize
intervention.
Superior medication reconciliation is also enabled by compiling an accurate list of the medications a member
is taking - including drug name, dosage, frequency, and route - and comparing that list against admission,
transfer, and/or discharge orders to ensure the member is receiving the correct medications. Access to drug
effectiveness and safety information improves improve clinical outcomes.
165
Interpreta was founded by Ahmed Ghouri, M.D., Gary Rayner, and Raghu Sugavanam, three seasoned
and successful technology entrepreneurs. Additional biographical details are available here. Their combined
experience in clinical analytics, information technology and growth of profitable companies is generating substantial momentum among leading organizations in key healthcare verticals. Interpreta will be announcing
major customers in the near future.
To set up a meeting with Interpreta at AHIP Institute & Expo, contact Beth@clearpointagency.com. About
Interpreta, Inc. Interpreta delivers on the promise of precision medicine today. Through daily re-computation
of individual member clinical and full-sequence genomic data, Interpreta’s fifth-generation healthcare analytics provides a prospective clinical interpretation that enables true personalization of therapy, and prevents
clinical gaps before they occur. Interpreta’s daily dynamic guidance, updated at the single member level,
maximizes quality scores, enables precision medicine, reduces medical costs and improves outcomes. See the
daily difference at www.Interpreta.com. Twitter: @InterpretaInc View source version on businesswire.com:
NewsRx LLC
(2016-06-27), Interpreta to Exhibit at 2016 AHIP Institute & Expo and Demonstrate How Healthcare Analytics Delivered Daily Improves Care Management for Health Plans, Journal of Engineering, 716, ISSN: 1945872X, BUTTER® ID: 011952850
Interpreta
Interpreta to Exhibit at AHIP 2016
By a News Reporter-Staff News Editor at Journal of Engineering – Press Kit Materials are Available at: http:
//www.tradeshownews.com/events/ahip-2016/Interpreta-Press-Kit/ Company:
Interpreta Booth/Stand: 358 Event: AHIP 2016
Jun 15 - 17, 2016
Las Vegas, NV, US Web:
http://www.interpreta.com/ Twitter:
https://twitter.com/
InterpretaInc LinkedIn: https://www.linkedin.com/company/interpreta About Interpreta Interpreta delivers on the promise of precision medicine today. Through daily re-computation of individual member
clinical and genomic data, Interpreta provides a prospective clinical interpretation that prevents gaps in patient care. Daily, dynamic guidance updated at the single member level maximizes quality scores for HEDIS,
P4P, and Risk Adjustment, while reducing medical costs and improving outcomes. Interpreta was founded
by Ahmed Ghouri, M.D., Gary Rayner, and Raghu Sugavanam, three seasoned and successful technology
entrepreneurs. Their combined experience in clinical analytics, information technology, novel drug development, and growth of profitable companies is generating substantial momentum among leading organizations
in key healthcare verticals. View source version on businesswire.com: http://www.businesswire.com/
news/home/20160613006555/en/
NewsRx LLC
(2016-06-27), Interpreta to Exhibit at AHIP 2016, Journal of Engineering, 717, ISSN: 1945-872X, BUTTER®
ID: 011952851
166
Oncology
Investigators at University Health Network Detail Findings in Prostate Cancer (Clonality of
localized and metastatic prostate cancer)
to news reporting out of Toronto, Canada, by NewsRx editors, research stated, “Purpose of reviewThe influence
of the long life-history of prostate cancer on the temporal and spatial variability of the tumour genome is now
being elucidated.”
Our news journalists obtained a quote from the research from University Health Network, “Multiregion
sequencing to identify spatio-genomic differences in prostate tumour mutation profiles combined with computational approaches can map the evolution and transit of tumour cells throughout an individual patient.Recent
findingsA series of recent studies have demonstrated that a prostate tumour is often composed of different subclones, with varying genetic similarity. As such, a single biopsy specimen may be insufficient to make accurate
clinical predictions from molecular biomarkers, greatly complicating the application of biopsy-based tools for
According to the news editors, the research concluded: “In addition, subclones that arise outside of the
primary tumour can seed new metastases and circulate between sites within a patient.SummaryThe mutational complexity of multiple tumour clones within the same individual, which respond differently to specific
treatments, suggests the need for multimodal interventions.”
For more information on this research see: Clonality of localized and metastatic prostate cancer. Current
Opinion in Urology , 2016;26(3):219-224. Current Opinion in Urology can be contacted at: Lippincott Williams
& Wilkins, Two Commerce Sq, 2001 Market St, Philadelphia, PA 19103, USA. (Lippincott Williams and Wilkins
- www.lww.com; Current Opinion in Urology - http://journals.lww.com/co-urology/pages/default.
aspx )
Our news journalists report that additional information may be obtained by contacting P.C. Boutros, Univ
Hlth Network, Lab Med Program, Dept. of Pathol, Toronto, ON, Canada. Additional authors for this research
include M. Fraser, T. van der Kwast and R.G. Bristow.
NewsRx LLC
(2016-06-07), Investigators at University Health Network Detail Findings in Prostate Cancer (Clonality of
localized and metastatic prostate cancer), Cancer Weekly, 439, ISSN: 1532-4567, BUTTER® ID: 011875027
Veterinary Research
Investigators from Roslin Institute Target Veterinary Research (Veterinary oncology:
Biology, big data and precision medicine)
By a News Reporter-Staff News Editor at Veterinary Week – Current study results on Veterinary Research have
been published. According to news reporting originating from Midlothian, United Kingdom, by VerticalNews
correspondents, research stated, “Despite significant advances in both the understanding and the treatment
of cancer, the disease remains one of high mortality and morbidity causes in all species. Increases in survival
times in human cancer have increased significantly in the past 25 years but most of these increases have been
through small incremental changes.”
Our news editors obtained a quote from the research from Roslin Institute, “For some cancers, e.g. pancreatic cancer, survival times have not increased significantly in over 100 years. In veterinary oncology, there
have been major shifts in the management of cancer in companion animals. Increased availability of specialist
centres, coupled with changing attitudes in owners and veterinarians, have meant improvements in veterinary cancer care borne from market pressures and increased awareness and understanding. In this review the
changing face of cancer biology over the past 25 years will be examined, and the barriers to clinical progress in
veterinary medicine considered.”
According to the news editors, the research concluded: “Finally, an optimistic view of the future will be
presented with the prospect of greater control over this devastating disease.”
For more information on this research see: Veterinary oncology: Biology, big data and precision medicine.
Veterinary Journal , 2016;213():38-45. Veterinary Journal can be contacted at: Elsevier Sci Ltd, The Boulevard,
Langford Lane, Kidlington, Oxford OX5 1GB, Oxon, England. (Elsevier - www.elsevier.com; Veterinary Journal
- http://www.journals.elsevier.com/veterinary-journal/ )
167
The news editors report that additional information may be obtained by contacting L.Y. Pang, Roslin Inst,
Roslin EH25 9RG, Midlothian, United Kingdom.
NewsRx LLC
(2016-07-18), Investigators from Roslin Institute Target Veterinary Research (Veterinary oncology: Biology,
big data and precision medicine), Veterinary Week, 64, ISSN: 1944-2785, BUTTER® ID: 012066579
Ketones
Investigators from University of Cincinnati Release New Data on Ketones (Design
considerations for a pilot trial using a novel approach for evaluating smoking-cessation
medication in methadone-maintained smokers)
By a News Reporter-Staff News Editor at Clinical Trials Week – Investigators discuss new findings in Ketones.
According to news reporting originating from Cincinnati, Ohio, by NewsRx correspondents, research stated,
“The prevalence of smoking in methadone-maintained (MM) patients is over 80% and smoking-abstinence
rates are strikingly low, even with the use of first-line smoking-cessation medications. Research has found
that cigarettes increase the reinforcing properties of methadone; this interaction may be an additional, daily,
challenge to smoking cessation in MM-smokers.”
Our news editors obtained a quote from the research from the University of Cincinnati, “This paper describes
a novel approach in which patients who experience a particular barrier to achieving smoking abstinence are
selected, and the impact of smoking-cessation medications on the identified barrier is evaluated. This is a 7week, outpatient, randomized, within-subject, placebo-controlled, crossover trial with a follow-up visit at week
8. MM-smokers, who smoke-40% of their total daily cigarettes in the 4-h post-methadone-dosing period, as
assessed with a Quitbit electronic cigarette lighter, will be recruited from a methadone program in Cincinnati,
Ohio. Eligible participants will be randomized to receive four interventions (nicotine nasal spray (1 mg per
dose, up to 40 times per day), placebo nicotine nasal spray, varenicline (2 mg/day), and varenicline placebo) in
one of four orders to mitigate potential order effects. The primary outcome analysis will consist of two sets of
statistical analyses, one comparing the effect of nicotine nasal spray to its placebo, and one comparing the effect
of varenicline to its placebo, on the proportion of daily cigarettes smoked during the 4-h post-methadone-dosing
period.”
According to the news editors, the research concluded: “This trial is of interest both as an efficient, precision
medicine-based approach to testing smoking-cessation interventions and as a specific strategy for identifying
effective smoking-cessation treatment for MM-smokers.”
For more information on this research see: Design considerations for a pilot trial using a novel approach for
evaluating smoking-cessation medication in methadone-maintained smokers. Contemporary Clinical Trials
, 2016;47():334-339. Contemporary Clinical Trials can be contacted at: Elsevier Science Inc, 360 Park Ave
South, New York, NY 10010-1710, USA. (Elsevier - www.elsevier.com; Contemporary Clinical Trials - http:
//www.journals.elsevier.com/contemporary-clinical-trials/ )
The news editors report that additional information may be obtained by contacting T. Winhusen, University
of Cincinnati, Coll Med, Addict Sci Div, Dept. of Psychiat & Behav Neurosci, 3131 Harvey Ave, Cincinnati, OH
45229, United States. Additional authors for this research include J. Theobald and D. Lewis.
NewsRx LLC
(2016-06-06), Investigators from University of Cincinnati Release New Data on Ketones (Design considerations for a pilot trial using a novel approach for evaluating smoking-cessation medication in methadonemaintained smokers), Clinical Trials Week, 516, ISSN: 1543-6764, BUTTER® ID: 011861287
168
Oncology
Investigators from University of Michigan Report New Data on Prostate Cancer (Targeting
PARP in Prostate Cancer: Novelty, Pitfalls, and Promise)
By a News Reporter-Staff News Editor at Cancer Weekly – Data detailed on Oncology have been presented.
According to news reporting from Ann Arbor, Michigan, by NewsRx journalists, research stated, “Metastatic
prostate cancer remains a highly lethal disease with no curative therapeutic options. A significant subset of
patients with prostate cancer harbor either germline or somatic mutations in DNA repair enzyme genes such
as BRCA1, BRCA2, or ATM.”
The news correspondents obtained a quote from the research from the University of Michigan, “Emerging
data suggest that drugs that target poly(adenosine diphosphate [ADP]-ribose) polymerase (PARP) enzymes
may represent a novel and effective means of treating tumors with these DNA repair defects, including prostate
cancers. Here we will review the molecular mechanism of action of PARP inhibitors and discuss how they target
tumor cells with faulty DNA repair functions and transcriptional controls. We will review emerging data for
the utility of PARP inhibition in the management of metastatic prostate cancer.”
According to the news reporters, the research concluded: “Finally, we will place PARP inhibitors within the
framework of precision medicine based care of patients with prostate cancer.”
For more information on this research see: Targeting PARP in Prostate Cancer: Novelty, Pitfalls, and
Promise. Oncology-New York , 2016;30(5):377-385. Oncology-New York can be contacted at: Ubm Medica,
535 Connecticut Ave, Ste 300, Norwalk, CT 06854, USA.
Our news journalists report that additional information may be obtained by contacting P.L. Palmbos, University of Michigan, Dept. of Internal Med, Div Hematol Oncol, 1500 E Med Center Dr7216 CC, Ann Arbor,
MI 48109, United States.
NewsRx LLC
(2016-06-14), Investigators from University of Michigan Report New Data on Prostate Cancer (Targeting
PARP in Prostate Cancer: Novelty, Pitfalls, and Promise), Cancer Weekly, 86, ISSN: 1532-4567, BUTTER® ID:
011833725
Sleep Research
Latest sleep research to be presented June 11-15 at APSS annual meeting in Denver
By a News Reporter-Staff News Editor at Health & Medicine Week – DARIEN, IL - Sleep clinicians and scientists from around the world will discuss current practices in sleep medicine and the latest findings in sleep and
circadian research at SLEEP 2016, the 30th anniversary meeting of the Associated Professional Sleep Societies
LLC (APSS), which will be held June 11-15 at the Colorado Convention Center in Denver.
Preceded by postgraduate courses on June 11, the scientific program begins at 1 p.m. on Sunday, June
12. General sessions include abstract presentations, symposia, clinical workshops and discussion groups on
topics ranging from circadian rhythms and metabolism to telemedicine and precision medicine. Clinical sleep
specialists also will discuss current practices in the diagnosis and treatment of sleep disorders such as insomnia,
narcolepsy and obstructive sleep apnea. The final program is available at http://www.sleepmeeting.org .
Among the highlights:
NewsRx LLC
(2016-06-10), Latest sleep research to be presented June 11-15 at APSS annual meeting in Denver, Health
169
Pharmaceutical Companies
Lilly and American Migraine Foundation Announce $1 Million Grant for Launch of First
Patient Registry to Advance Research and Development in Migraine
By a News Reporter-Staff News Editor at Pharma Business Week – Eli Lilly and Company (NYSE: LLY) and the
American Migraine Foundation announced that Lilly will provide a $1 million grant to the American Migraine
Foundation to sponsor the development of the American Registry for Migraine Research (ARMR), a largescale, publicly-accessible headache registry and biological repository that will contain clinical, biologic and
neuroimaging data to further advance the research and discovery of new treatments for migraine.
Migraine is the third-most prevalent and sixth-most disabling medical disorder in the world, and it is the
leading cause of all disability attributed to neurological diseases worldwide.(1,2) Other primary and secondary
headache disorders are also highly prevalent and cause substantial burden for people with these diseases,
including impaired quality of life and financial cost.(1)
Through this registry, the American Migraine Foundation aims to establish repositories and enroll patients
with primary and secondary headache disorders to collect imaging data, genetics information and biologic samples. The American Migraine Foundation will also work to establish and train a larger network of investigative
sites to enroll subjects, submit data and access all collected data for specific studies. Data from the registry
will be made accessible to all researchers, with grant applications available for high-priority projects.
“We are enormously grateful for the vision and very generous support from Lilly to help the American
Migraine Foundation build and sustain a patient registry and biorepository for migraine and other headache
disorders,” said American Migraine Foundation Chairman Dr. David Dodick. “We believe the knowledge gained
from this registry and the potential to identify clinical, laboratory, imaging and genetic biomarkers will usher
in an era of precision medicine and generate new targets for generations of patients to come.”
As a platinum sponsor, Lilly will serve as a member of the Corporate Registry Roundtable, working with
the American Migraine Foundation and other members to propose topics and analysis areas for the registry,
which will be approved by the ARMR Scientific Review Board.
“Despite the prevalence and overwhelming impact they cause, migraine and other headache disorders historically are some of the most underfunded areas of clinical research,” said Dr. Robert Conley, Distinguished
Lilly Scholar and leader of Lilly’s pain development platform. “We are proud to support the American Migraine
Foundation’s efforts to establish a registry that will allow researchers to evaluate and compare real-world data
that can help transform research in headache disorders, and ultimately help healthcare providers improve
outcomes for people living with these devastating disorders.”
NewsRx LLC
(2016-06-20), Lilly and American Migraine Foundation Announce $1 Million Grant for Launch of First Patient Registry to Advance Research and Development in Migraine, Pharma Business Week, 122, ISSN: 15436667, BUTTER® ID: 011914199
Pharmaceutical Companies
Merrimack Announces a Leading-edge Biomarker-Selected, Multi-Arm Basket Trial that
Matches Patients with Most Appropriate Combination Regimens
By a News Reporter-Staff News Editor at Clinical Oncology Week – Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) announced the initiation of a biomarker-selected, multi-arm Phase 1 clinical study in metastatic
colorectal, non-small cell lung, and head and neck cancers. Merrimack’s first-of-its-kind basket study will use
a combination of genetic and nongenetic biomarkers to match patients to appropriate novel combinations of
investigational drug regimens based on their cancer’s molecular signature. This approach is expected to enable more than 95 percent of eligible patients to qualify for enrollment to one of the investigational arms of the
study. Merrimack utilized its systems biology approach to identify the biomarkers and selection criteria of the
study.
“This innovative trial design is a major step towards applying precision medicine in the earliest phases of
clinical testing, while also ensuring that virtually every patient that enrolls in the study receives one of the
investigational regimens,” said Christopher Lieu, M.D., Investigator at the CU Cancer Center and Assistant
Professor of Medical Oncology at the University of Colorado School of Medicine. “The difficulty with most basket
studies is that they identify their patient population solely on genetic mutations, which often results in a very
small percentage of patients qualifying for the investigational therapy. By targeting a combination of genetic
170
markers and frequently activated resistance pathways, the vast majority of biopsied patients will be matched
to a biomarker-selected study arm.”
Merrimack’s approach to biomarker profiling and subsequent patient assignment builds on the foundation
of previous basket studies. This study design has broadened its scope by also identifying molecular features
in the tumor and its surrounding environment that are believed to indicate which signaling mechanisms are
responsible for tumor growth and drug resistance. By exploring the dynamics of multiple molecular features,
and by including multiple combinations of investigational therapies from its oncology pipeline, Merrimack
expects to investigate a personalized drug regimen in almost all of the basket trial’s eligible patient population.
“We are committed to implementing precision medicine at every stage of clinical development,” said Gavin
MacBeath, Ph.D., Merrimack’s Senior Vice President of Translational Medicine. “At Merrimack, our research
has shown that most cancers have multiple mechanisms supporting tumor growth and we are developing a
number of investigational agents that are designed to block key drivers of tumor growth and resistance. We
know enough about the biology of these pathways to prospectively assign patients to the investigational regimen that we believe most closely matches their specific disease. We believe this is the first trial to rationally
combine novel regimens based upon specific multi-dimensional characteristics of the patient’s tumor. This
unique approach is expected to provide a path forward in developing improved outcomes in difficult to treat
patient populations.”
NewsRx LLC
(2016-05-30), Merrimack Announces a Leading-edge Biomarker-Selected, Multi-Arm Basket Trial that
Matches Patients with Most Appropriate Combination Regimens, Clinical Oncology Week, 305, ISSN: 15436780, BUTTER® ID: 011747537
Muses Labs
Metabolon and Muses Labs Partner to Apply Metabolomics to Alzheimer’s Disease
By a News Reporter-Staff News Editor at Journal of Engineering – Muses Labs announced it has partnered
with Metabolon, Inc., the global leader in metabolomics, to study and apply metabolomics in individuals with
pre- and early-symptomatic cognitive decline and Alzheimer’s disease. The companies expect to bring new
clinical trial services and updated care protocols to the market in 2016. Metabolomics is the study of small
molecules called metabolites and is a powerful phenotyping technology for precision medicine. It measures
changes in metabolite levels and maps them to the appropriate biochemical pathways to give clinicians a better
understanding of health and the influences of genes, microbiome, diet, lifestyle and drug treatment.
“Muses Labs’ protocols utilize a patient’s genome, blood tests, medical history and lifestyle to direct care.
Metabolomics adds additional significant insight into a patient’s biological status to improve identification of
active pathologies and enable further refinement of patient care programs,” said Vik Chandra, CEO of Muses
Labs. “Metabolomics has the potential to significantly expand the effectiveness of combination therapy protocols for Alzheimer’s and other complex diseases. Our collaboration with Metabolon has the power to advance
precision medicine in this area.”
Muses Labs’ MEND™ Protocol is a data driven, technology enabled, personalized methodology to correctly
identify and simultaneously treat the dozens of pathology drivers of age-related cognitive decline. It is designed
to help patients who are beginning to have symptoms of age-related cognitive decline, those with a family
history of dementia and patients with mild Alzheimer’s disease. The Protocol recommends optimal medical
interventions personalized for each patient and has the potential to improve memory and cognitive function.
“We’re working together to expand the knowledge we can apply toward overcoming Alzheimer’s disease,”
said John Ryals, PhD, CEO of Metabolon. “Our goal is to use our proprietary technology in conjunction with
Muses Labs’ personalized Protocols to provide clinicians with a more accurate health assessment of patients
experiencing cognitive decline as well as more actionable information. Metabolomics may help answer questions
that surround complex, neurodegenerative diseases and refine personalized treatment.”
“While research into metabolomics and Alzheimer’s disease is in its early stages, we believe that by using
this technology, the connections between metabolism and the underlying pathologies of Alzheimer’s disease
will become better understood,” said Dr. Marwan Sabbagh, Research Professor of Neurology, University of
Arizona College of Medicine, Chief Medical-Scientific Advisor, Muses Labs. “Metabolomics can guide the clinical care of Alzheimer’s disease today while laying the foundation for new interventions and protocols.” About
Metabolon Metabolon, Inc. is the world’s leading health technology company focused on the use of metabolomics
171
in precision medicine. This powerful tool for assessing health is based on proprietary platforms and informatics that deliver biomarker discoveries, innovative diagnostic tests and ground-breaking partnerships in
genomics-based health initiatives. Metabolon’s expertise is also accelerating research and product development across the pharmaceutical, biotechnology, consumer products, agriculture and nutrition industries, as
well as academic and government organizations. Founded in 2000 and headquartered in Research Triangle
Park, North Carolina, the company has conducted more than 5,000 independent and collaborative studies,
resulting in over 550 peer-reviewed publications. For more information, please visit www.metabolon.com or
follow us on LinkedIn or Twitter. About Muses Labs Muses Labs brings together 30 years of scientific research, hundreds of published clinical studies, and a team of highly-respected Alzheimer’s researchers, doctors and advanced technology. The company applies its clinical precision medicine platform and technologyenabled protocol to collect individual patient medical and behavioral data, identify underlying causes of agerelated cognitive decline (such as Alzheimer’s disease and memory loss), and design personalized treatment
plans. A comprehensive adherence program supports individuals and their caregivers to improve patient outcomes. This first personalized protocol is called the MENDTM Protocol (Metabolic Enhancement for NeuroDegeneration). For more information, visit www.museslabs.com. View source version on businesswire.com:
NewsRx LLC
(2016-07-25), Metabolon and Muses Labs Partner to Apply Metabolomics to Alzheimer’s Disease, Journal
of Engineering, 774, ISSN: 1945-872X, BUTTER® ID: 012094805
NantHealth, LLC
NantHealth Files Registration Statement for Proposed Initial Public Offering
By a News Reporter-Staff News Editor at Health & Medicine Week – NantHealth, LLC, a leading nextgeneration, evidence-based, personalized healthcare company enabling improved patient outcomes and more
effective treatment decisions for critical illnesses, announced that it has publicly filed a registration statement
on Form S-1 with the U.S. Securities and Exchange Commission (SEC) relating to a proposed initial public
offering of shares of its common stock. All shares of common stock to be sold in the offering will be offered
by NantHealth. The number of shares to be offered and the price range for the proposed offering have not yet
been determined. NantHealth intends to list its common stock on the NASDAQ Global Market under the ticker
symbol “NH.”
Jefferies LLC and Cowen and Company, LLC are acting as joint book-running managers for the offering,
and First Analysis Securities Corporation and FBR Capital Markets & Co. are acting as co-managers.
A registration statement relating to these securities has been filed with the SEC, but has not yet become
effective. These securities may not be sold, nor may offers to buy be accepted, prior to the time the registration
statement becomes effective. This press release shall not constitute an offer to sell or the solicitation of an offer
to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such
offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any
such state or jurisdiction.
The offering will be made only by means of a prospectus. When available, copies of the preliminary
prospectus may be obtained from: Jefferies LLC, Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 547-6340 or by email at Prospectus Department@Jefferies.com; or Cowen and Company, LLC, c/o Broadridge Financial Services, Attention: Prospectus
Department, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (631) 274-2806 or by fax
at (631) 254-7140. About NantHealth NantHealth, a member of the NantWorks ecosystem of companies, is
a leading next-generation, evidence-based, personalized healthcare company enabling improved patient outcomes and more effective treatment decisions for critical illnesses. Our unique systems-based approach to
personalized healthcare applies novel diagnostics tailored to the specific molecular profiles of patient tissues
and integrates this molecular data in a clinical setting with large-scale, real-time biometric signal and phenotypic data to track patient outcomes and deliver precision medicine. For nearly a decade, we have developed an
adaptive learning system, CLINICS, which includes our unique software, middleware and hardware systems
infrastructure that collects, indexes, analyzes and interprets billions of molecular, clinical, operational and
financial data points derived from novel and traditional sources, continuously improves decision-making and
further optimizes our clinical pathways and decision algorithms over time. As a pioneer in the era of big data
and augmented intelligence, we believe we are uniquely positioned to benefit from multiple significant market
172
opportunities as healthcare providers and payors transition from fee-for-service to value-based reimbursement
models and accelerate their pursuit of evidence-based clinical practice.
Our mission is to empower providers to seamlessly act on the best evidence-based information available to
better fulfill their roles as caregivers rather than as financial managers, to provide payors with the necessary
tools to better fulfill their roles as stewards of an increasingly complex and rapidly evolving healthcare system,
to facilitate biopharmaceutical companies to accelerate development of drugs for critical illnesses based upon
the unique biology and specific health conditions of patients, and to empower patients with the knowledge
to enable active participation in the management of their own health, or self-care. View source version on
NewsRx LLC
(2016-05-27), NantHealth Files Registration Statement for Proposed Initial Public Offering, Health &
Biometrics
New Biometrics Study Findings Have Been Reported from Dana-Farber Cancer Institute
(Optimal Bayesian adaptive trials when treatment efficacy depends on biomarkers)
By a News Reporter-Staff News Editor at Clinical Trials Week – New research on Biometrics is the subject
of a report. According to news originating from Boston, Massachusetts, by NewsRx correspondents, research
stated, “Clinical biomarkers play an important role in precision medicine and are now extensively used in
clinical trials, particularly in cancer. A response adaptive trial design enables researchers to use treatment
results about earlier patients to aid in treatment decisions of later patients.”
Our news journalists obtained a quote from the research from Dana-Farber Cancer Institute, “Optimal
adaptive trial designs have been developed without consideration of biomarkers. In this article, we describe
the mathematical steps for computing optimal biomarker-integrated adaptive trial designs. These designs
maximize the expected trial utility given any pre-specified utility function, though we focus here on maximizing
patient responses within a given patient horizon. We describe the performance of the optimal design in different
scenarios. We compare it to Bayesian Adaptive Randomization (BAR), which is emerging as a practical approach
to develop adaptive trials. The difference in expected utility between BAR and optimal designs is smallest
when the biomarker subgroups are highly imbalanced. We also compare BAR, a frequentist play-the-winner
rule with integrated biomarkers and a marker-stratified balanced randomization design (BR). We show that, in
contrasting two treatments, BR achieves a nearly optimal expected utility when the patient horizon is relatively
large.”
According to the news editors, the research concluded: “Our work provides novel theoretical solution, as
well as an absolute benchmark for the evaluation of trial designs in personalized medicine.”
For more information on this research see: Optimal Bayesian adaptive trials when treatment efficacy
depends on biomarkers. Biometrics , 2016;72(2):414-421. Biometrics can be contacted at: Wiley-Blackwell,
111 River St, Hoboken 07030-5774, NJ, USA. (Wiley-Blackwell - http://www.wiley.com/ ; Biometrics http://onlinelibrary.wiley.com/journal/10.1111/(ISSN)1541-0420 )
The news correspondents report that additional information may be obtained from Y.F. Zhang, Dana Farber
Canc Inst, Dept. of Biostat & Computat Biol, Boston, MA 02115, United States. Additional authors for this
research include L. Trippa and G. Parmigiani.
NewsRx LLC
(2016-07-25), New Biometrics Study Findings Have Been Reported from Dana-Farber Cancer Institute (Optimal Bayesian adaptive trials when treatment efficacy depends on biomarkers), Clinical Trials Week, 385,
ISSN: 1543-6764, BUTTER® ID: 012090280
173
Huntington Disease
New brain map could enable novel therapies for autism and Huntington’s disease
By a News Reporter-Staff News Editor at Mental Health Weekly Digest – USC scientists have mapped an
uncharted portion of the mouse brain to explain which circuit disruptions might occur in disorders such as
Huntington’s disease and autism.
Hong-Wei Dong, an associate professor of neurology at the USC Mark and Mary Stevens Neuroimaging and
Informatics Institute, and his colleagues have been sketching a road map of the mouse brain for the past decade.
Their most recent study, published in the journal Nature Neuroscience on June 20, looked at the connections
of a part of the brain that is responsible for motor learning, the dorsal striatum, which is stationed near the
front of the brain.
Scientists injected fluorescent molecules into about 150 mouse brain structures and used a high-resolution
microscope to document the molecules as they moved through the brain’s “cellular highways,” which need to
be in tip-top shape for different parts of the brain to communicate and coordinate behaviors. In the previously
unsurveyed dorsal striatum, USC researchers were able to identify 29 distinct areas responsible for things such
as eye movement, mouth and facial movements and pain information processing. They also located hubs that
coordinate complex limb movements.
Parkinson’s disease, obsessive compulsive disorder, attention deficit hyperactivity disorder and many other
movement disorders involve connections of this brain region, said Dong, senior author of the study. Researchers
in the Dong Lab followed circuitry paths from beginning to end in the same way someone might pick up a ball
of yarn and slowly unravel it.
“This study moves researchers to the next level to help them understand how the brain circuit is disrupted,”
Dong said. “Previously the dorsal striatum was one huge thing. It’s almost like telling someone they should
come visit you in California. Where should they go? We have really narrowed it down - I live in North Hollywood
in this apartment building. That will help people in the future to really understand the pathways for diseases
with specific symptoms.”
Early symptoms of Huntington’s disease, for example, include slow or abnormal eye movement and stuttering. Most patients experience depression and have psychiatric issues such as social withdrawal and insomnia,
Dong said. His lab has subdivided the dorsal striatum so scientists can better predict problem areas in this
brain structure and focus their research there.
“Of course, humans and mice are different, but they are both mammals,” Dong said. “The biggest differences
reside in high-level cognition. So we can use the organization of the mouse brain to understand how human
brains are organized.”
After all, most scientific research begins at the mouse level. Understanding the mouse brain is of critical
importance and will potentially lead to the development of new drugs and medical therapies.
The cerebral cortex is the brain’s CEO; it regulates higher-order functions such as motor learning and attention through its connections to brain structures such as the dorsal striatum, which is historically divided
into just four regions. Today, USC researchers said they are the first to create the most comprehensive map of
connections between the dorsal striatum and the cerebral cortex that is available for any mammal.
“If you have one big structure, it’s very difficult to know which part is the problem area,” said Houri Hintiryan, lead author and assistant professor of research at the Laboratory of Neuro Imaging at the Keck School
of Medicine of USC. “This study shows 29 different parts of this brain region receiving information from the
cerebral cortex. We’re providing a structural basis for studies seeking to understand which part of the brain
does what.”
The big data project is publicly available at the Mouse Connectome Project and will aid in the leap toward
precision medicine, Dong said. Next on his team’s mapping list is the hippocampus, which is the center of
emotion, memory and the autonomic nervous system. A detailed atlas of this area could further Alzheimer’s
disease research.
“With our brain map, researchers could look for circuitry-specific drug discovery,” Dong said. “Now we
provide a very clear map to help people do stem cell research. They know exactly where to put stem cells.”
Dong chose to map the mouse brain so his lab could examine individual neurons and axons, gateways to
neighboring neurons. In contrast, the human brain would look pixelated if the scientists zoom in for the detail
needed to develop their meticulous maps.
Many USC scientists use big data to find answers to intractable problems such as Alzheimer’s disease and
PTSD. USC houses the Global Alzheimer’s Association Interactive Network (GAAIN), which is the first usable
online platform that provides scientists with Alzheimer’s disease research data and tools. GAAIN contains
data on over 366,000 subjects and is home to the largest brain-mapping project in the world.
174
NewsRx LLC
(2016-07-04), New brain map could enable novel therapies for autism and Huntington’s disease, Mental
Health Weekly Digest, 145, ISSN: 1543-6608, BUTTER® ID: 011986818
Oncology
New Breast Cancer Study Findings Have Been Reported by Researchers at University of
Helsinki (Identification of selective cytotoxic and synthetic lethal drug responses in triple
negative breast cancer cells)
By a News Reporter-Staff News Editor at Cancer Weekly – Investigators publish new report on Oncology. According to news reporting out of Helsinki, Finland, by NewsRx editors, research stated, “Triple negative breast
cancer (TNBC) is a highly heterogeneous and aggressive type of cancer that lacks effective targeted therapy.
Despite detailed molecular profiling, no targeted therapy has been established.”
Our news journalists obtained a quote from the research from the University of Helsinki, “Hence, with the
aim of gaining deeper understanding of the functional differences of TNBC subtypes and how that may relate
to potential novel therapeutic strategies, we studied comprehensive anticancer-agent responses among a panel
of TNBC cell lines. The responses of 301 approved and investigational oncology compounds were measured in
16 TNBC cell lines applying a functional profiling approach. To go beyond the standard drug viability effect
profiling, which has been used in most chemosensitivity studies, we utilized a multiplexed readout for both cell
viability and cytotoxicity, allowing us to differentiate between cytostatic and cytotoxic responses. Our approach
revealed that most single-agent anti-cancer compounds that showed activity for the viability readout had no
or little cytotoxic effects. Major compound classes that exhibited this type of response included anti-mitotics,
mTOR, CDK, and metabolic inhibitors, as well as many agents selectively inhibiting oncogene-activated pathways. However, within the broad viability-acting classes of compounds, there were often subsets of cell lines
that responded by cell death, suggesting that these cells are particularly vulnerable to the tested substance.
In those cases we could identify differential levels of protein markers associated with cytotoxic responses. For
example, PAI-1, MAPK phosphatase and Notch-3 levels associated with cytotoxic responses to mitotic and proteasome inhibitors, suggesting that these might serve as markers of response also in clinical settings. Furthermore, the cytotoxicity readout highlighted selective synergistic and synthetic lethal drug combinations that
were missed by the cell viability readouts. For instance, the MEK inhibitor trametinib synergized with PARP
inhibitors. Similarly, combination of two non-cytotoxic compounds, the rapamycin analog everolimus and an
ATP-competitive mTOR inhibitor dactolisib, showed synthetic lethality in several mTOR-addicted cell lines.”
According to the news editors, the research concluded: “Taken together, by studying the combination of
cytotoxic and cytostatic drug responses, we identified a deeper spectrum of cellular responses both to single
agents and combinations that may be highly relevant for identifying precision medicine approaches in TNBC
as well as in other types of cancers.”
For more information on this research see: Identification of selective cytotoxic and synthetic lethal drug
responses in triple negative breast cancer cells. Molecular Cancer , 2016;15():8-23. Molecular Cancer can be
contacted at: Biomed Central Ltd, 236 Grays Inn Rd, Floor 6, London WC1X 8HL, England. (BioMed Central
- http://www.biomedcentral.com/ ; Molecular Cancer - www.molecular-cancer.com)
Our news journalists report that additional information may be obtained by contacting P. Gautam, University of Helsinki, Inst Mol Med Finland, Helsinki, Finland. Additional authors for this research include L.
Karhinen, A. Szwajda, S.K. Jha, B. Yadav, T. Aittokallio and K. Wennerberg.
NewsRx LLC
(2016-06-14), New Breast Cancer Study Findings Have Been Reported by Researchers at University of
Helsinki (Identification of selective cytotoxic and synthetic lethal drug responses in triple negative breast cancer cells), Cancer Weekly, 279, ISSN: 1532-4567, BUTTER® ID: 011833876
175
Capillary Electrophoresis
New Capillary Electrophoresis Findings from N.A. Guzman et al Outlined (An emerging
micro-scale immuno-analytical diagnostic tool to see the unseen. Holding promise for
precision medicine and P4 medicine)
By a News Reporter-Staff News Editor at Life Science Weekly – Current study results on Capillary Electrophoresis have been published. According to news reporting originating in East Brunswick, New Jersey, by
NewsRx journalists, research stated, “Over the years, analytical chemistry and immunology have contributed
significantly to the field of clinical diagnosis by introducing quantitative techniques that can detect crucial
and distinct chemical, biochemical and cellular biomarkers present in biosamples. Currently, quantitative
two-dimensional hybrid immuno-analytical separation technologies are emerging as powerful tools for the sequential isolation, separation and detection of protein panels, including those with subtle structural changes
such as variants, isoforms, peptide fragments, and post-translational modifications.”
The news reporters obtained a quote from the research, “One such technique to perform this challenging task
is immunoaffinity capillary electrophoresis (IACE), which combines the use of antibodies and/or other affinity
ligands as highly selective capture agents with the superior resolving power of capillary electrophoresis. Since
affinity ligands can be polyreactive, i.e., binding and capturing more than one molecule, they may generate
false positive results when tested under mono-dimensional procedures; one such application is enzyme-linked
immunosorbent assay (ELISA). IACE, on the other hand, is a two-dimensional technique that captures (isolation and enrichment), releases, separates and detects (quantification, identification and characterization) a
single or a panel of analytes from a sample, when coupled to one or more detectors simultaneously, without the
presence of false positive or false negative data. This disruptive technique, capable of preconcentrate on-line
results in enhanced sensitivity even in the analysis of complex matrices, may change the traditional system of
testing biomarkers to obtain more accurate diagnosis of diseases, ideally before symptoms of a specific disease
manifest.”
According to the news reporters, the research concluded: “In this manuscript, we will present examples of
the determination of biomarkers by IACE and the design of a miniaturized multi-dimensional IACE apparatus
capable of improved sensitivity, specificity and throughput, with the potential of being used as a point-of-care
instrument and holding promise for precision medicine and P4 medicine.”
For more information on this research see: An emerging micro-scale immuno-analytical diagnostic tool to see
the unseen. Holding promise for precision medicine and P4 medicine. Journal of Chromatography B-Analytical
Technologies in the Biomedical and Life Sciences , 2016;1021():14-29. Journal of Chromatography B-Analytical
Technologies in the Biomedical and Life Sciences can be contacted at: Elsevier Science Bv, PO Box 211, 1000
Ae Amsterdam, Netherlands.
Our news correspondents report that additional information may be obtained by contacting N.A. Guzman,
AffinityCE LLC, East Brunswick, NJ 08816, United States.
NewsRx LLC
(2016-06-21), New Capillary Electrophoresis Findings from N.A. Guzman et al Outlined (An emerging microscale immuno-analytical diagnostic tool to see the unseen. Holding promise for precision medicine and P4
medicine), Life Science Weekly, 2138, ISSN: 1552-2474, BUTTER® ID: 011922960
Oncology
New Colon Cancer Study Findings Reported from M. Jesinghaus and Co-Authors
(Genotyping of colorectal cancer for cancer precision medicine: Results from the IPH
Center for Molecular Pathology)
By a News Reporter-Staff News Editor at Cancer Weekly – Investigators publish new report on Oncology. According to news reporting originating in Heidelberg, Germany, by NewsRx journalists, research stated, “Cancer
precision medicine has opened up new avenues for the treatment of colorectal cancer (CRC). To fully realize its
potential, high-throughput sequencing platforms that allow genotyping beyond KRAS need to be implemented
and require performance assessment.”
The news reporters obtained a quote from the research, “We comprehensively analyzed first-year data of
202 consecutive formalin-fixed paraffin embedded (FFPE) CRC samples for which prospective genotyping at
our institution was requested. Deep targeted genotyping was done using a semiconductor-based sequencing
176
platform and a self-designed panel of 30 CRC-related genes. Additionally, microsatellite status (MS) was determined. Ninety-seven percent of tumor samples were suitable for sequencing and in 88% MS could be assessed.
The minimal drop-out rates of 6 and 25 cases, respectively were due to too low amounts or heavy degradation
of DNA. Of 557 nonsynonymous mutations, 90 (16%) have not been described in COSMIC at the time of data
query. Forty-three cases (22%) had double- or triple mutations affecting a single gene. Sixty-four percent had
genetic alterations influencing oncological therapy. Eight percent of patients (MSI phenotype: 6%; mutated
POLE: 2%) were potentially eligible for treatment with immune checkpoint inhibitors. Of 56% of KRASwt CRC
that potentially qualified for anti-EGFR treatment, 30% presented with mutations in BRAF/NRAS. Mutated
PIK3CA was detected in 21%.”
According to the news reporters, the research concluded: “We here present real-life routine diagnostics data
that not only demonstrate the robustness and feasibility of deep targeted sequencing and MS-analysis of FFPE
CRC samples but also contribute to the understanding of CRC genetics. Most importantly, in more than half
of the patients our approach enabled the selection of the best treatment currently available.”
For more information on this research see: Genotyping of colorectal cancer for cancer precision medicine:
Results from the IPH Center for Molecular Pathology. Genes Chromosomes & Cancer , 2016;55(6):505-521.
Genes Chromosomes & Cancer can be contacted at: Wiley-Blackwell, 111 River St, Hoboken 07030-5774, NJ,
USA.
Our news correspondents report that additional information may be obtained by contacting M. Jesinghaus,
Natl Center Tumor Dis NCT, HSO, Heidelberg, Germany. Additional authors for this research include N. Pfarr,
V. Endris, M. Kloor, A.L. Volckmar, R. Brandt, E. Herpel, A. Muckenhuber, F. Lasitschka, P. Schirmacher, R.
Penzel, W. Weichert and A. Stenzinger.
NewsRx LLC
(2016-06-07), New Colon Cancer Study Findings Reported from M. Jesinghaus and Co-Authors (Genotyping
of colorectal cancer for cancer precision medicine: Results from the IPH Center for Molecular Pathology), Cancer
Encephalitis
New Findings from Autonomous University Update Understanding of Progressive Multifocal
Leukoencephalopathy (Precision medicine in multiple sclerosis: biomarkers for diagnosis,
prognosis, and treatment response)
By a News Reporter-Staff News Editor at Health & Medicine Week – Investigators publish new report on Encephalitis. According to news reporting out of Barcelona, Spain, by NewsRx editors, research stated, “This
article highlights recent studies on MRI and body fluid molecular biomarkers with potential implications in
diagnosis, prognosis, and response to treatment in patients with multiple sclerosis (MS). In the MS diagnostic
process, a reappraisal of well known MRI findings, including symptomatic and spinal cord lesions, and incorporation of newer, more specific, features of MS lesions, such as detection of central veins, are in a testing phase
aiming to increase diagnostic accuracy.”
Our news journalists obtained a quote from the research from Autonomous University, “Lesion counts on
T2-weighted scans and gadolinium uptake on postcontrast T1-weighted scans are well established prognostic
brain MRI biomarkers. Evidences supporting the implementation of brain volume measures for disease and
treatment monitoring are increasing, maybe within a ‘zero tolerance’ scheme in the overarching no evidence of
disease activity concept. Cerebrospinal fluid (CSF) chitinase 3-like 1 and the light subunit of neurofilaments
are consolidating their prognostic role in patients with clinically isolated syndrome. Blood CD62L and CSF
IgM oligoclonal bands may become clinically useful biomarkers for progressive multifocal leukoencephalopathy
risk stratification in MS patients treated with natalizumab.”
According to the news editors, the research concluded: “Although more studies are needed, current and
emerging imaging and molecular biomarkers in MS may contribute to outline the concept of precision medicine
in MS.”
For more information on this research see: Precision medicine in multiple sclerosis: biomarkers for diagnosis, prognosis, and treatment response. Current Opinion in Neurology , 2016;29(3):254-262. Current Opinion in Neurology can be contacted at: Lippincott Williams & Wilkins, Two Commerce Sq, 2001 Market St,
Philadelphia, PA 19103, USA. (Lippincott Williams and Wilkins - www.lww.com; Current Opinion in Neurology - http://journals.lww.com/co-neurology/pages/default.aspx )
177
Our news journalists report that additional information may be obtained by contacting M. Comabella, Autonomous University of Barcelona, Center Esclerosi Multiple Catalunya Cemcat, Serv Neurol Neuroimmunol,
Inst Recerca Vall dHebron VHIRHosp Univ Vall dHe, E-08193 Barcelona, Spain. Additional authors for this
research include J. Sastre-Garriga and X. Montalban.
NewsRx LLC
(2016-06-03), New Findings from Autonomous University Update Understanding of Progressive Multifocal Leukoencephalopathy (Precision medicine in multiple sclerosis: biomarkers for diagnosis, prognosis, and
treatment response), Health & Medicine Week, 1755, ISSN: 1532-4605, BUTTER® ID: 011791237
Science
New Findings on Science Described by Investigators at University of California (Overcoming
mTOR resistance mutations with a new-generation mTOR inhibitor)
By a News Reporter-Staff News Editor at Clinical Trials Week – New research on Science is the subject of a
report. According to news reporting originating in Berkeley, California, by NewsRx journalists, research stated,
“Precision medicines exert selective pressure on tumour cells that leads to the preferential growth of resistant
subpopulations, necessitating the development of next-generation therapies to treat the evolving cancer. The
PIK3CA-AKT-mTOR pathway is one of the most commonly activated pathways in human cancers(1), which has
led to the development of small-molecule inhibitors that target various nodes in the pathway.”
The news reporters obtained a quote from the research from the University of California, “Among these
agents, first-generation mTOR inhibitors (rapalogs) have caused responses in ‘N-of-1’ cases, and secondgeneration mTOR kinase inhibitors (TORKi) are currently in clinical trials(2-4). Here we sought to delineate
the likely resistance mechanisms to existing mTOR inhibitors in human cell lines, as a guide for next-generation
therapies. The mechanism of resistance to the TORKi was unusual in that intrinsic kinase activity of mTOR
was increased, rather than a direct active-site mutation interfering with drug binding. Indeed, identical drugresistant mutations have been also identified in drug-naive patients, suggesting that tumours with activating
MTOR mutations will be intrinsically resistant to second-generation mTOR inhibitors. We report the development of a new class of mTOR inhibitors that overcomes resistance to existing first-and second-generation
inhibitors.”
According to the news reporters, the research concluded: “The third-generation mTOR inhibitor exploits
the unique juxtaposition of two drug-binding pockets to create a bivalent interaction that allows inhibition of
these resistant mutants.”
For more information on this research see: Overcoming mTOR resistance mutations with a new-generation
mTOR inhibitor. Nature , 2016;534(7606):272-276,345-285. Nature can be contacted at: Nature Publishing
Group, Macmillan Building, 4 Crinan St, London N1 9XW, England. (Nature Publishing Group - http://
www.nature.com/ ; Nature - http://www.nature.com/nature/ )
Our news correspondents report that additional information may be obtained by contacting V.S. RodrikOutmezguine, University of California, Dept. of Chem, Berkeley, CA 94720, United States. Additional authors
for this research include M. Okaniwa, Z. Yao, C.J. Novotny, C. McWhirter, A. Banaji, H. Won, W. Wong, M.
Berger, E. de Stanchina, D.G. Barratt, S. Cosulich, T. Klinowska, N. Rosen and K.M. Shokat.
NewsRx LLC
(2016-06-27), New Findings on Science Described by Investigators at University of California (Overcoming
mTOR resistance mutations with a new-generation mTOR inhibitor), Clinical Trials Week, 154, ISSN: 15436764, BUTTER® ID: 011946591
178
New Proteomics Data Have Been Reported by Researchers at University of Florence
(Allostasis and Resilience of the Human Individual Metabolic Phenotype)
By a News Reporter-Staff News Editor at Life Science Weekly – Research findings on Life Science Research are
discussed in a new report. According to news reporting out of Sesto Fiorentino, Italy, by NewsRx editors, research stated, “The urine metabotype of 12 individuals was followed over a period of 8-10 years, which provided
the longest longitudinal study of metabolic phenotypes to date. More than 2000 NMR metabolic profiles were
analyzed.”
Our news journalists obtained a quote from the research from the University of Florence, “The majority
of subjects have a stable metabotype. Subjects who were exposed to important pathophysiological stressful
conditions had a significant metabotype drift. When the stress conditions ceased, the original metabotypes
were regained, while an irreversible stressful condition resulted in a permanent metabotype change. These
results suggest that each individual occupies a well-defined region in the broad metabolic space, within which
a limited degree of allostasis is permitted. The insurgence of significant stressful conditions causes a shift of
the metabotype to another distinct region. The spontaneous return to the original metabolic region when the
stressful conditions are removed suggests that the original metabotype has some degree of resilience.”
According to the news editors, the research concluded: “In this picture, precision medicine should aim at
reinforcing the patient’s metabolic resilience, that is, his or her ability to revert to his or her specific metabotype
rather than to a generic healthy one.”
For more information on this research see: Allostasis and Resilience of the Human Individual Metabolic
Phenotype. Journal of Proteome Research , 2015;14(7):2951-62. (American Chemical Society - www.acs.org;
Journal of Proteome Research - http://www.pubs.acs.org/journal/jprobs )
Our news journalists report that additional information may be obtained by contacting V. Ghini, †Magnetic
Resonance Center (CERM), University of Florence, Via L Sacconi 6, 50019 Sesto Fiorentino, Italy. Additional
authors for this research include E. Saccenti, L. Tenori, M. Assfalg and C. Luchinat.
NewsRx LLC
(2016-05-31), New Proteomics Data Have Been Reported by Researchers at University of Florence (Allostasis
and Resilience of the Human Individual Metabolic Phenotype), Life Science Weekly, 5436, ISSN: 1552-2474,
BUTTER® ID: 011771320
Peptides and Proteins
New tool brings personalized medicine closer
By a News Reporter-Staff News Editor at Life Science Weekly – Scientists from EPFL and ETHZ have developed
a powerful tool for exploring and determining the inherent biological differences between individuals, which
overcomes a major hurdle for personalized medicine.
One of the biggest obstacles in successfully treating metabolic disorders such as diabetes, obesity, fatty liver
etc, is the variation in the way patients respond to medication. The key to this variation lies in the inherent
biological differences between individuals, which cannot all be explained genetically. At the same time, this
variation makes it very difficult to develop “standard” treatments for certain diseases. In a groundbreaking
initiative, EPFL and ETHZ scientists have developed a strategy that can define and explain metabolic differences between individuals, essentially paving the way for precision medicine. The work, which also highlights
significant issues with animal-based drug studies, is published in Science.
We are increasingly learning that medical interventions can be more successful when they are tailored to
the specific profile of the individual patient. The problem is that defining that profile is extremely difficult, as
it involves information on the person’s genome, proteins, fats, and all sorts of other layers of biology that make
up their tissues and body. And so far, the only differences that we have been seriously taking into account are
those found between genes.
This is what the labs of Johan Auwerx at EPFL and Ruedi Aebersold at ETH Zurich set out to solve with
their recently published study. Looking at 40 different mice strains, the researchers successfully connected the
variation between individuals’ genomes to the variation between their proteomes – their full set of proteins. In
this way, they took a giant leap in profiling the biology of a particular individual.
179
“There is a black box between a patient’s genome and their disease,” says Johan Auwerx, whose lab handled
the genome side of the study. “What we have done here is find a way to fill the black box by obtaining information
on the patient’s proteome.”
“It’s much more complex to measure the set of proteins than to sequence the entire genome,” says Yibo
Wu, co-lead author of the study. The scientists used protein data from mice, which they obtained from a new
mass spectrometry technique that Aebersold’s group developed, known as SWATH-MS. This is a technique that
combines the advantages of high-throughput mass spectrometry with high reproducibility and consistency. In
short, it is able to identify thousands of different proteins in hundreds of samples much faster and accurately
than conventional techniques, allowing the researchers to measure the concentrations of a broad spectrum of
liver proteins in the mice.
The researchers measured a total of 2,600 different proteins from tissue samples of 40 mice strains, all of
which came from the same two ancestors, meaning that they were genetically related to each other. “We wanted
to keep the genetics simple in order to observe differences in the impact of environment (diet) on the proteome,
other layers of biology, and their predisposition to develop diseases,” says Auwerx.
The mice were divided into groups of representing each of the 40 strains, and the groups were fed either
a high-fat diet - essentially junk food - or a healthy, low-fat diet. Over a few weeks, the scientists charted the
mice’s physiological data, and tested how fast they could gain weight on the junk-food diet and lose weight by
exercising. Despite their similar genetic make-up, the mice on the high-fat diet showed varied responses to diet
and exercise. For example, some developed metabolic disorders like fatty liver, while others did not.
The researchers then combined the physiological data with data for their genome, their proteome, and their
transcriptome, which is essentially their full set of RNAs – another biological “layer” in the black box. Through
this combination, the scientists were able to better understand the role several proteins play when it comes to
metabolizing fat and producing energy from it.
“Like the mouse strains in this study, each patient with a disease is genetically different”, says Ruedi Aebersold. “The approach we used in the mouse cohort can now be applied one-for-one in research on human
diseases, and particularly for personalized medicine.” The SWATH-MS approach is now ready to use in human
cohort studies, as the researchers in his group have produces a corresponding database for thousands of human
proteins.
“The aim here is to be able to customise medical intervention for each patient based on their individual
biological makeup, the ‘black box’,” says Auwerx. In this vein, his group is now looking at specific drugs that
can be used more effectively with this approach to treat metabolic disorders.
NewsRx LLC
(2016-06-28), New tool brings personalized medicine closer, Life Science Weekly, 937, ISSN: 1552-2474, BUTTER® ID: 011959485
Vanderbilt University Medical Center
NIH awards $11.6 million to Vanderbilt, Miami and Meharry for Precision Medicine Center
By a News Reporter-Staff News Editor at NewsFile – Researchers at Vanderbilt University Medical Center
(VUMC), the University of Miami and Meharry Medical College were recently awarded a five-year, $11.6 million
grant to launch a new center that will enable research using precision medicine to eradicate health disparities,
specifically those among African-Americans and Latinos.
The Vanderbilt-Miami-Meharry Center of Excellence in Precision Medicine and Population Health will
leverage unique institutional assets and resources to develop novel methods and catalyze approaches to advance population health.
The center will be led by principal investigators with complementary expertise: Consuelo H. Wilkins, M.D.,
MSCI, Executive Director, Meharry-Vanderbilt Alliance; Nancy J. Cox, Ph.D., Director, Vanderbilt Genetics
Institute; Maria de Fatima Lima, Ph.D., Dean, School of Graduate Studies and Research, Meharry Medical
College; and Roy E. Weiss, M.D., Ph.D., Chair, Department of Medicine, University of Miami Miller School of
Medicine.
“The future is now ... and this award represents unparalleled possibilities in health care delivery as it will
allow us to link genetic data with contextual data to learn why different populations experience different health
outcomes. Discovering the answers to these questions aligns directly with Meharry’s mission. I am confident
that the research coming from this collaboration with the University of Miami and Vanderbilt University School
of Medicine will ultimately help transform health care in a way that allows every individual to live life healthier,”
said James E. K. Hildreth, Ph.D., M.D., President and Chief Executive Officer, Meharry Medical College.
180
“Team science is truly the best way to advance precision medicine. We are excited about the potential for
new discoveries and understandings in how diseases can be prevented, diagnosed earlier and treated with new
therapies that target social and genetic drivers...all leading to better outcomes and more hope,” said Steven
Altschuler, M.D., CEO of UHealth - the University of Miami Health System.
The foundation of precision medicine is that prevention and treatment strategies take individual variability
into account, which has the potential to eliminate disparities. Yet, to date, when developing new ways to treat
disease, investigational therapies are often insufficiently tested in minorities, even though efficacy and safety
can be highly variable in certain races and/or ethnicities.
“For precision medicine to reach its full potential we must develop new ways to integrate social, cultural,
environmental and biological data to accurately identify strategies to prevent and treat disease among all
populations, especially those with disproportionately poor health outcomes,” said Wilkins.
The center, one of three new awards from the National Institute on Minority Health and Health Disparities,
will include core programs focused on building and sustaining a regional consortium and developing strategies
to speed implementation, diffusion and adoption of precision medicine discoveries. “We will also create two
novel cores. One to improve access to the genetic and clinical data of minorities and another to cross-train
researchers in health disparities and precision medicine techniques,” said Lima.
Three projects are funded as part of the center. In a study led by University of Miami’s Sunil Rao, Ph.D.
and Erin Kobetz, Ph.D., MPH, we will develop new statistical methods to predict risk of disparities, starting
with cervical cancer. Dr. Cox will examine genetic risk factors contributing to health disparities and will
characterize the relative contribution of genetic risk factors for asthma and pre-term birth, two conditions
with substantial disparities in health outcomes among African- Americans and Latinos. Derek Griffith, Ph.D.,
Associate Professor of Medicine, Health and Society, Vanderbilt University, will lead a study of person-specific
obesity treatment for African- American and Latino men.
“This grant creates exciting opportunities for our institutions to collaborate in new ways, applying the power
of precision medicine to help answer important questions about diseases that are disproportionately affecting
the health of minority populations. Our longstanding partnership with Meharry Medical College continues to
result in advancements in knowledge that are positively impacting the health of citizens across the Southeast.
I want to welcome colleagues with the University of Miami as they join with us in this new endeavor and look
forward to their contributions as we seek to improve the health of the populations we serve,” said Jeff Balser,
M.D., Ph.D., President and CEO of VUMC and Dean of the Vanderbilt University School of Medicine.
Engaging African- American and Latino communities in the center will be vital to its success. According to
Wilkins, community members in Nashville and Miami provided key input to help shape the center’s proposal
and will continue to be involved as part of the consortium and on the Ethics Advisory Board.
“If precision medicine will be used to improve population health and lead to health equity, we must address
ongoing barriers to research including issues around trust and genomic health literacy,” said Wilkins.
NewsRx LLC
(2016-06-06), NIH awards $11.6 million to Vanderbilt, Miami and Meharry for Precision Medicine Center,
NewsFile, 199, ISSN: 0000-0000, BUTTER® ID: 011863547
Omicia Inc.
Omicia Raises $23 Million in Series B Funding
By a News Reporter-Staff News Editor at Journal of Engineering – Omicia, Inc., a leading provider of clinical
genome interpretation and reporting software, announced the completion of a $23 million Series B financing
round. Several new investors participated in the round, including UPMC Enterprises, Roche Venture Fund,
LDV Partners, Ping An Ventures, and a large genomics investor, as well as existing investors ARTIS Ventures,
Acadia Woods Partners and Buchanan Investments.
Omicia’s latest financing will be used to fuel acceleration in product development and an expansion in sales,
sales support, marketing and operations. In addition, the company plans to extend variations of its flagship
OpalTM Clinical platform for use in new geographies and new verticals including life science research, clinical
trial support, population health management and consumer applications.
“With new investment from one of the nation’s leading health systems, a premier life sciences company, and
China’s largest insurance company, we are well positioned to expand our team and extend our technologies,
products and services into new markets,” said Matt Tindall, CEO of Omicia. “As evidenced by the work we’re
doing with Genomics England, the world’s leading national genome project, Omicia is filling a critical gap
between sequencing tools and clinical decision making, delivering on the promise of precision medicine.”
181
Omicia’s software provides leading healthcare system stakeholders with fast, accurate and scalable next
generation sequence (NGS) interpretation and reporting capabilities to drive improved clinical outcomes. Further, the platform provides customers with an option to scale production in a HIPAA-compliant, cloud-enabled
environment.
“As an investor, UPMC is excited to work with Omicia to begin to address today’s extraordinary opportunities in precision medicine,” said Tal Heppenstall, president of UPMC Enterprises, the innovation and commercialization arm of UPMC, one of the country’s largest integrated healthcare provider and payor systems.
“We envision a future in which we can routinely deliver actionable genomic insights about individual patients
in real-time, within the workflow of our 3,500 physicians.”
In addition, Omicia’s OpalTM Clinical platform leverages a deep variant data warehouse to deliver highthroughput results to assist lab partners with automated diagnostic reporting. The easily deployed, white-label
solution can be used to replace manual, expensive and difficult to maintain in-house software pipelines to lower
costs and improve quality.
“Omicia has built a world-class software platform that can be applied at the intersection of genomic data
and more traditional healthcare information to generate insights powering both predictive and preventive
medicine,” said Stu Peterson, co-founder and senior partner at ARTIS Ventures, who in addition to contributing
to its latest round of financing was its first institutional investor, leading Omicia’s Series A. “Genome sequencing, combined with machine-learning algorithms and powerful data analytics, will provide physicians with
more complete, actionable insights at a reasonable cost.”
Another significant new development for the company is the strong support in the round from strategically
important overseas investors. With new partners in Asia and Europe, the company intends to continue its push
into these important markets.
“We are very proud to work with Omicia and its team to bring novel technology offerings to markets which
are driven by active health management initiatives,” said Jiang Zhang, Managing Director Ping An Ventures. “Omicia’s software is designed perfectly to address important population health management opportunities in Asia.” About Omicia Omicia delivers a market-leading software platform that enables analysis,
interpretation, and reporting on genomic data to expedite diagnosis and improve medical outcomes. Omicia’s products are in use at more than 500 world-class academic and clinical institutions worldwide. By accelerating understanding of the genetic basis of disease, drug response, and health, Omicia is unlocking the
potential of individualized medicine. Headquartered in Oakland, California with an office in the UK, Omicia was founded by renowned industry veterans with a deep understanding of technology, genomics, and diagnostics. Investors include ARTIS Ventures, Acadia Woods Partners, Bay City Capital, Buchanan Investments, Casdin Capital, LDV Partners, Ping An, Roche Venture Fund, UPMC Enterprises and Yuri Milner.
For additional information, visit www.omicia.com and follow us on Twitter @omicia. For employment inquiries, please contact us at http://www.omicia.com/careers/ . View source version on businesswire.com:
NewsRx LLC
(2016-06-20), Omicia Raises $23 Million in Series B Funding, Journal of Engineering, 1077, ISSN: 1945872X, BUTTER® ID: 011915861
Amendola Communications
Orion Health Engages Amendola Communications for Public Relations and Content
Creation
By a News Reporter-Staff News Editor at Journal of Engineering – Amendola Communications, a nationally
recognized, award-winning public relations, content creation and marketing firm specializing in healthcare and
health information technology (HIT), is pleased to announce that Orion Health™ has selected Amendola as its
PR agency of record for the United States providing media relations and content creation services.
http://photos.prnewswire.com/prnvar/20140404/MM99055LOGO
Orion Health, a large, global healthcare data technology firm with U.S. headquarters in Scottsdale, Ariz.,
is improving healthcare now while also anticipating what’s ahead with its scalable technology that drives interoperability. With more than 100 million patient records in its systems worldwide, Orion Health’s open-data
platform serves as an end-to-end solution for effective management of all types of clinical, genomic, social and
environmental data. This data can then be leveraged to identify and prioritize patients’ needs, effectively manage patient care, and proactively drive wellness.
182
“We chose Amendola Communications because of our synergy and cultural fit,” said Wayne Oxenham,
president-North America at Orion Health. “Amendola shares our vision of using health IT to improve our
healthcare system. They have the industry knowledge as well as media and analyst relationships to help shine
a light on the innovative work that we are doing at Orion Health to transform healthcare.”
Amendola Communications will provide broad PR and marketing services, including media research, aggressive media relations, and securing top-tier awards and speaking opportunities. The agency also will be
responsible for delivering a range of content demonstrating the thought leadership and expertise of Orion
Health’s subject matter experts, including bylined articles, blog posts, press releases and other materials.
“The work that Orion Health is doing to further precision medicine and the tools they provide are critical
in creating individualized, targeted treatment plans that engage patients and improve preventive health and
outcomes,” said Jodi Amendola, CEO of Amendola Communications. “Our deep experience in this area positions
us perfectly to strategically share with the market the details about Orion’s leadership and innovation – as well
as the many success stories of customers using their open-data platform.”
The timing couldn’t be better, Amendola said, given that precision medicine is such a high priority right
now. For example, the U.S. government’s newly launched Precision Medicine Initiative (PMI) calls for $215
million in fiscal year 2016 to support research in this area “with the goal of developing more effective ways to
prolong health and treat disease.”
NewsRx LLC
(2016-07-04), Orion Health Engages Amendola Communications for Public Relations and Content Creation,
Journal of Engineering, 978, ISSN: 1945-872X, BUTTER® ID: 011991252
PatientsLikeMe
PatientsLikeMe Names Marni Hall Senior Vice President
By a News Reporter-Staff News Editor at Pharma Business Week – PatientsLikeMe announced it has appointed
Marni Hall, PhD, MPH, as its new Senior Vice President of Research and Policy. A distinguished research
scientist and public policy expert, Hall will develop and direct the strategies and teams focused on expanding
the role of real-world evidence in precision medicine, and in the research agendas of PatientsLikeMe and its
customers.
PatientsLikeMe CEO Martin Coulter said Hall “will now apply her strategic research and operational expertise to help us work with our members and partners to use patient-reported data in new and innovative
ways, so that the patient experience can lead to even more significant developments and discoveries, such as
improved outcomes.”
According to Hall, the new opportunity allows her to continue to do research in a scientifically-rigorous
and patient-centered setting. “PatientsLikeMe has been a critical force in documenting and analyzing real-life
patient experiences and evolving the role of real-world evidence in clinical and public health research. My goal
is to extend its impact, so that the patient experience drives a future where healthcare is able to emphasize
individual needs and preferences. I’m thrilled to join a company that is so focused on helping people thrive each
day, while collecting data essential to this emerging field,” Hall said.
A research scientist by training, Hall has spent nearly two decades at the intersection of science and policy.
She started her career studying toxicology and molecular epidemiology at Columbia University. After serving
as Program Director in the Public Health Group of External Medical Affairs at Pfizer, Hall joined the FDA’s
Office of Planning and Informatics (OPI) in 2008 as a Principal Analyst. In this role, she initiated and led the
development of CDER’s data standards plan. She was appointed Director of Regulatory Science in 2011.
Hall holds bachelor of science degrees in chemistry and in society, technology, and policy from Worcester
Polytechnic Institute. She also holds a master’s degree in public health from Columbia University’s Mailman
School of Public Health as well as a master of science degree in biochemistry and a PhD in toxicology from
Columbia University’s Graduate School of Arts and Sciences. About PatientsLikeMePatientsLikeMe is a patient network that improves lives and a real-time research platform that advances medicine. Through the
network, patients connect with others who have the same disease or condition and track and share their own
experiences. In the process, they generate #dataforgood: data about the real-world nature of disease that help
researchers, pharmaceutical companies, regulators, providers and nonprofits develop more effective products,
services and care. With more than 400,000 members, PatientsLikeMe is a trusted source for real-world disease information and a clinically robust resource that has published more than 70 research studies. Visit us
183
at www.patientslikeme.com or follow us via our blog, Twitter or Facebook. View source version on businesswire.com: http://www.businesswire.com/news/home/20160711005661/en/
NewsRx LLC
(2016-07-25), PatientsLikeMe Names Marni Hall Senior Vice President, Pharma Business Week, 27, ISSN:
1543-6667, BUTTER® ID: 012093708
PHEMI
PHEMI Data Scientist Speaking at Upcoming Cloud and Big Data Expo 2016 in New York
City
By a News Reporter-Staff News Editor at Journal of Engineering – PHEMI, the company delivering big data
solutions with built-in privacy, security, and governance and enterprise-grade management, announced the
participation of its lead data scientist Sarbjit Sarkaria at the upcoming Cloud and Big Data Expo to be held
June 7-9 at the Javits Center in New York City, NY.
Mr. Sarkaria’s speaking session “Connecting the Dots: Driving New Business Value with Data Science”
will show how data science and big data technologies bring organizations significantly greater value and newly
discovered intelligence from their data. With examples from enterprise, precision medicine, and genomics,
this highly informative session sheds light on the latest advancements in big data management, analytics and
data science. EXHIBITION Cloud Expo 2016, June 7-9, 2016, Javits Center, New York City, NY SPEAKING
SESSION Connecting the Dots: Driving New Business Value with Data Science WHEN/WHERE Wednesday,
June 8, 3:40-4:15 PM ETRoom 1A02 SPEAKER BIO PHEMI’s lead data scientist Sarbjit Sarkaria is an expert in machine learning, data analytics, and modern software technologies, including Hadoop, Spark, Web
frameworks, REST, and SOA. He has domain skills in data science, neural networks, image processing, and
artificial intelligence, plus over 20 years experience building complex software systems. Cloud Expo is the
world’s largest exhibition covering all things related to cloud computing, with a noted focus on big data and a
vast selection of technical and strategic industry keynotes, general sessions, breakout sessions, and signature
power panels. About PHEMI PHEMI delivers privacy, security, governance and enterprise-grade management
for big data. Its flagship product, PHEMI Central, embeds and enforces consent, data sharing agreements and
privacy policies at the data level, removing a critical roadblock standing in the way of enterprises that aim to
become data-driven.
PHEMI Central lets organizations easily access and mine any variety of data at any volume to drive insights that lower costs, improve outcomes, and allow better decisions faster. PHEMI brings both privacy and
performance to big data. For more information visit http://www.phemi.com/ and follow us on Twitter. View
NewsRx LLC
(2016-06-20), PHEMI Data Scientist Speaking at Upcoming Cloud and Big Data Expo 2016 in New York
City, Journal of Engineering, 1091, ISSN: 1945-872X, BUTTER® ID: 011915874
Endocrinology
Recent Research from National Cancer Center Highlight Findings in Endocrine-Related
cancer (Evaluation of the methods to identify patients who may benefit from PARP
inhibitor use)
By a News Reporter-Staff News Editor at Clinical Trials Week – Current study results on Endocrinology have
been published. According to news reporting originating in Singapore, Singapore, by NewsRx journalists, research stated, “The effectiveness of poly (ADP-ribose) polymerase inhibitors (PARPi) in treating cancers associated with BRCA1/2 mutations hinges upon the concept of synthetic lethality and exemplifies the principles
of precision medicine. Currently, most clinical trials are recruiting patients based on pathological subtypes or
have included BRCA mutation analysis (germ line and/or somatic) as part of the selection criteria.”
The news reporters obtained a quote from the research from National Cancer Center, “Mounting evidence,
however, suggests that these drugs may also be efficacious in tumors with defects in other genes involved in the
homologous recombination repair pathway. Advances in molecular profiling techniques together with increased
research efforts have led to a better understanding of the molecular aberrations underlying this BRCA-like
184
phenotype and helped broaden the concept of BRCAness. Hence, it is likely that the list of predictive biomarkers
for PARPi therapy will increase in future. There is currently no gold standard method of testing for PARPi
response and no universal guidelines are in place on how to incorporate biomarker testing into routine clinical
diagnostics. In this review, we explore the concept of BRCAness and highlight the different methods that have
been used to identify patients who may benefit from the use of these anticancer agents.”
According to the news reporters, the research concluded: “The identification of predictive biomarkers is
crucial in improving patient selection and expanding the clinical applications of PARPi therapy.”
For more information on this research see: Evaluation of the methods to identify patients who may benefit
from PARP inhibitor use. Endocrine-Related Cancer , 2016;23(6):R267-R285. Endocrine-Related Cancer can
be contacted at: Bioscientifica Ltd, Euro House, 22 Apex Court Woodlands, Bradley Stoke, Bristol BS32 4JT,
England.
Our news correspondents report that additional information may be obtained by contacting D. Lim, Natl
Canc Center, Div Med Oncol, Canc Genet Serv, Singapore, Singapore.
NewsRx LLC
(2016-07-25), Recent Research from National Cancer Center Highlight Findings in Endocrine-Related cancer (Evaluation of the methods to identify patients who may benefit from PARP inhibitor use), Clinical Trials
Blood Cells
Reports from Beijing Institute of Genomics Describe Recent Advances in Blood Cells
(Reprogramming of blood cells into induced pluripotent stem cells as a new cell source for
cartilage repair)
By a News Reporter-Staff News Editor at Stem Cell Week – Investigators publish new report on Blood Cells.
According to news reporting from Beijing, People’s Republic of China, by NewsRx journalists, research stated,
“An attempt was made to reprogram peripheral blood cells into human induced pluripotent stem cell (hiPSCs)
as a new cell source for cartilage repair. We generated chondrogenic lineage from human peripheral blood via
hiPSCs using an integration-free method.”
The news correspondents obtained a quote from the research from the Beijing Institute of Genomics, “Peripheral blood cells were either obtained from a human blood bank or freshly collected from volunteers. After
transforming peripheral blood cells into iPSCs, the newly derived iPSCs were further characterized through
karyotype analysis, pluripotency gene expression and cell differentiation ability. iPSCs were differentiated
through multiple steps, including embryoid body formation, hiPSC-mesenchymal stem cell (MSC)-like cell expansion, and chondrogenic induction for 21 days. Chondrocyte phenotype was then assessed by morphological,
histological and biochemical analysis, as well as the chondrogenic expression. hiPSCs derived from peripheral blood cells were successfully generated, and were characterized by fluorescent immunostaining of pluripotent markers and teratoma formation in vivo. Flow cytometric analysis showed that MSC markers CD73 and
CD105 were present in monolayer cultured hiPSC-MSC-like cells. Both alcian blue and toluidine blue staining
of hiPSC-MSC-chondrogenic pellets showed as positive. Immunohistochemistry of collagen II and X staining
of the pellets were also positive. The sulfated glycosaminoglycan content was significantly increased, and the
expression levels of the chondrogenic markers COL2, COL10, COL9 and AGGRECAN were significantly higher
in chondrogenic pellets than in undifferentiated cells. These results indicated that peripheral blood cells could
be a potential source for differentiation into chondrogenic lineage in vitro via generation of mesenchymal progenitor cells.”
According to the news reporters, the research concluded: “This study supports the potential applications of
utilizing peripheral blood cells in generating seed cells for cartilage regenerative medicine in a patient-specific
and cost-effective approach.”
For more information on this research see: Reprogramming of blood cells into induced pluripotent stem
cells as a new cell source for cartilage repair. Stem Cell Research & Therapy , 2016;7():31. (BioMed Central http://www.biomedcentral.com/ ; Stem Cell Research & Therapy - stemcellres.com)
Our news journalists report that additional information may be obtained by contacting Y. Li, Key Laboratory of Genomic and Precision Medicine, Beijing Institute of Genomics, Chinese Academy of Sciences, Beijing,
100101, People’s Republic of China. Additional authors for this research include T. Liu, N. Van Halm-Lutterodt,
J. Chen, Q. Su and Y. Hai.
185
NewsRx LLC
(2016-05-30), Reports from Beijing Institute of Genomics Describe Recent Advances in Blood Cells (Reprogramming of blood cells into induced pluripotent stem cells as a new cell source for cartilage repair), Stem Cell
Bioinformatics
Reports from University College Highlight Recent Findings in Bioinformatics (Problems,
challenges and promises: perspectives on precision medicine)
By a News Reporter-Staff News Editor at Information Technology Newsweekly – Investigators discuss new
findings in Bioinformatics. According to news reporting out of Dublin, Ireland, by VerticalNews editors, the
research stated, “The ‘precision medicine (systems medicine)’ concept promises to achieve a shift to future
healthcare systems with a more proactive and predictive approach to medicine, where the emphasis is on disease prevention rather than the treatment of symptoms. The individualization of treatment for each patient
will be at the centre of this approach, with all of a patient’s medical data being computationally integrated and
accessible.”
Our news journalists obtained a quote from the research from University College, “Precision medicine is
being rapidly embraced by biomedical researchers, pioneering clinicians and scientific funding programmes
in both the European Union (EU) and USA. Precision medicine is a key component of both Horizon 2020 (the
EU Framework Programme for Research and Innovation) and the White House’s Precision Medicine Initiative.
Precision medicine promises to revolutionize patient care and treatment decisions. However, the participants
in precision medicine are faced with a considerable central challenge. Greater volumes of data from a wider
variety of sources are being generated and analysed than ever before; yet, this heterogeneous information must
be integrated and incorporated into personalized predictive models, the output of which must be intelligible to
non-computationally trained clinicians. Drawing primarily from the field of ‘oncology’, this article will introduce
key concepts and challenges of precision medicine and some of the approaches currently being implemented to
overcome these challenges.”
According to the news editors, the research concluded: “Finally, this article also covers the criticisms of
precision medicine overpromising on its potential to transform patient care.”
For more information on this research see: Problems, challenges and promises: perspectives on precision
medicine. Briefings in Bioinformatics , 2016;17(3):494-504. Briefings in Bioinformatics can be contacted at:
Oxford Univ Press, Great Clarendon St, Oxford OX2 6DP, England. (Oxford University Press - http://www.
oup.com/ ; Briefings in Bioinformatics - bib.oxfordjournals.org)
Our news journalists report that additional information may be obtained by contacting D.J. Duffy, University College Dublin, Syst Biol Ireland, Sci Link Bldg, Dublin 4, Ireland.
NewsRx LLC
(2016-07-05), Reports from University College Highlight Recent Findings in Bioinformatics (Problems, challenges and promises: perspectives on precision medicine), Information Technology Newsweekly, 140, ISSN:
1944-1800, BUTTER® ID: 012001792
186
Science
Reports Outline Cell Science Study Results from Y.X. Wang et al (A mutation abolishing
the ZMPSTE24 cleavage site in prelamin A causes a progeroid disorder)
By a News Reporter-Staff News Editor at Life Science Weekly – New research on Science is the subject of
a report. According to news originating from Atlanta, Georgia, by NewsRx correspondents, research stated,
“In 1994 in the Journal of Cell Science, Hennekes and Nigg reported that changing valine to arginine at the
endoproteolytic cleavage site in chicken prelamin A abolishes its conversion to lamin A. The consequences of
this mutation in an organism have remained unknown. We now report that the corresponding mutation in a
human subject leads to accumulation of prelamin A and causes a progeroid disorder.”
Our news journalists obtained a quote from the research, “Next generation sequencing of the subject and
her parents’ exomes identified a de novo mutation in the lamin A/C gene (LMNA) that resulted in a leucine to
arginine amino acid substitution at residue 647 in prelamin A. The subject’s fibroblasts accumulated prelamin
A, a farnesylated protein, which led to an increased percentage of cultured cells with morphologically abnormal
nuclei. Treatment with a protein farnesyltransferase inhibitor improved abnormal nuclear morphology.”
According to the news editors, the research concluded: “This case demonstrates that accumulation of
prelamin A, independent of the loss of function of ZMPSTE24 metallopeptidase that catalyzes processing of
prelamin A, can cause a progeroid disorder and that a cell biology assay could be used in precision medicine to
identify a potential therapy.”
For more information on this research see: A mutation abolishing the ZMPSTE24 cleavage site in prelamin
A causes a progeroid disorder. Journal of Cell Science , 2016;129(10):1975-1980. Journal of Cell Science can be
contacted at: Company Of Biologists Ltd, Bidder Building Cambridge Commercial Park Cowley Rd, Cambridge
CB4 4DL, Cambs, England.
The news correspondents report that additional information may be obtained from Y.X. Wang, MNG Labs,
Atlanta, GA 30342, United States. Additional authors for this research include U. Lichter-Konecki, K. AnyaneYeboa, J.E. Shaw, J.T. Lu, C. Ostlund, J.Y. Shin, L.N. Clark, G.G. Gundersen, P.L. Nagy and H.J. Worman.
NewsRx LLC
(2016-06-21), Reports Outline Cell Science Study Results from Y.X. Wang et al (A mutation abolishing the
ZMPSTE24 cleavage site in prelamin A causes a progeroid disorder), Life Science Weekly, 2794, ISSN: 15522474, BUTTER® ID: 011923524
Stem Cell Research
Reports Outline Stem Cell Research Study Findings from Johns Hopkins University School
of Medicine (Determination of Functional Activity of Human iPSC-Derived Hepatocytes by
Measurement of CYP Metabolism)
By a News Reporter-Staff News Editor at Gastroenterology Week – Data detailed on Stem Cell Research have
been presented. According to news reporting originating from Baltimore, Maryland, by NewsRx correspondents, research stated, “The advent of induced pluripotent stem cell (iPSC) technology has enabled the modeling of an array of specific human disease phenotypes, aiding in the increasingly important and indispensable
understanding of disease progression and pathogenesis. Pluripotent stem cell-derived hepatocytes present a
new avenue for drug screening and personalized drug testing toward precision medicine.”
Our news editors obtained a quote from the research from the Johns Hopkins University School of Medicine,
“CYP450 microsomal enzymes play a critical role in drug metabolism. Hence, CYP activity measurement of
iPSC-derived hepatocytes is a vital prerequisite, to ensure metabolic functionality before proceeding to drug
testing.”
According to the news editors, the research concluded: “Herein, we describe the protocol for measurement
of different CYP450 enzyme activities in human iPSC-derived hepatocytes.”
For more information on this research see: Determination of Functional Activity of Human iPSC-Derived
Hepatocytes by Measurement of CYP Metabolism. Methods In Molecular Biology , 2016;1357():383-94.
The news editors report that additional information may be obtained by contacting P. Chaudhari, Dept. of
Oncology, The Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine,
Baltimore, MD, 21205, United States. Additional authors for this research include N. Prasad, L. Tian and Y.Y
Jang.
187
NewsRx LLC
(2016-05-30), Reports Outline Stem Cell Research Study Findings from Johns Hopkins University School of
Medicine (Determination of Functional Activity of Human iPSC-Derived Hepatocytes by Measurement of CYP
Metabolism), Gastroenterology Week, 716, ISSN: 1543-6748, BUTTER® ID: 011750508
Immunology
Reports Summarize Allergy and Immunology Research Study Results from S.J. Galli and
Co-Researchers (Toward precision medicine and health: Opportunities and challenges in
allergic diseases)
By a News Reporter-Staff News Editor at Immunotherapy Weekly – Researchers detail new data in Immunology.
According to news reporting out of Stanford, California, by NewsRx editors, the research stated, “Precision
medicine (also called personalized, stratified, or P4 medicine) can be defined as the tailoring of preventive
measures and medical treatments to the characteristics of each patient to obtain the best clinical outcome for
each person while ideally also enhancing the cost-effectiveness of such interventions for patients and society.
Clearly, the best clinical outcome for allergic diseases is not to get them in the first place.”
Our news journalists obtained a quote from the research, “To emphasize the importance of disease prevention, a critical component of precision medicine can be referred to as precision health, which is defined herein
as the use of all available information pertaining to specific subjects (including family history, individual genetic and other biometric information, and exposures to risk factors for developing or exacerbating disease), as
well as features of their environments, to sustain and enhance health and prevent the development of disease.
In this article I will provide a personal perspective on how the precision health-precision medicine approach
can be applied to the related goals of preventing the development of allergic disorders and providing the most
effective diagnosis, disease monitoring, and care for those with these prevalent diseases.”
According to the news editors, the research concluded: “I will also mention some of the existing and potential
challenges to achieving these ambitious goals.”
For more information on this research see: Toward precision medicine and health: Opportunities and
challenges in allergic diseases. Journal of Allergy and Clinical Immunology , 2016;137(5):1289-1300. Journal of Allergy and Clinical Immunology can be contacted at: Mosby-Elsevier, 360 Park Avenue South, New
York, NY 10010-1710, USA. (Elsevier - www.elsevier.com; Journal of Allergy and Clinical Immunology http://www.journals.elsevier.com/journal-of-allergy-and-clinical-immunology/ )
Our news journalists report that additional information may be obtained by contacting S.J. Galli, Stanford
Center Genom & Personalized Med, Stanford, CA, United States.
NewsRx LLC
(2016-06-15), Reports Summarize Allergy and Immunology Research Study Results from S.J. Galli and
Co-Researchers (Toward precision medicine and health: Opportunities and challenges in allergic diseases),
Immunotherapy Weekly, 39, ISSN: 1532-463X, BUTTER® ID: 011842904
Diet and Nutrition Disorders
Research Conducted by C. Zoccali and Co-Authors Has Provided New Information about
Vitamin D Deficiency (Moderator’s view: Vitamin D deficiency treatment in advanced
chronic kidney disease: a close look at the emperor’s clothes)
By a News Reporter-Staff News Editor at Clinical Trials Week – Investigators publish new report on Diet and
Nutrition Disorders. According to news originating from Reggio di Calabria, Italy, by NewsRx correspondents,
research stated, “Two recent vitamin D supplementation (ergocalciferol) trials in stage G5D CKD patients with
vitamin D insufficiency showed that this sterol effectively increases serum 25-hydroxyvitamin D [25(OH)D] but
fails to modify serum PTH and other clinical outcomes. The Pro side of this polar view emphasizes that the
duration of these studies was too short to allow sensible analyses based on a clinical endpoint.”
Our news journalists obtained a quote from the research, “Furthermore, he notes that in the second study,
the use of active forms of vitamin D, phosphate binders and cinacalcet could have hindered appreciation of the
effect of ergocalciferol supplementation per se. The Con side produces an updated meta-analysis showing that
inactive vitamin D forms largely fail to reduce serum PTH and affect various relevant endpoints, including
188
muscle strength, functional capacity, quality of life and hospitalization. Studies suggesting an effect of inactive vitamin D forms in advanced CKD are either very small and mainly based on sequential, uncontrolled
observations or inherently weak, simple pre/post studies.”
According to the news editors, the research concluded: “No biological or clinical evidence exists that 25(OH)D
may exert meaningful effects in CKD patients who are being treated with active forms of vitamin D. Careful a
etiologic studies based on the omics sciences, i.e. precise pathophysiological profiling of individual CKD patients
followed by consequential, well-targeted intervention(s) in the precision medicine scenario, will likely provide
a definitive answer to the lingering question of whether inactive vitamin D forms may have biological effects
beyond those produced by their proximate metabolite 1,25-dihydroxyvitamin D3.”
For more information on this research see: Moderator’s view: Vitamin D deficiency treatment in advanced chronic kidney disease: a close look at the emperor’s clothes. Nephrology Dialysis Transplantation
, 2016;31(5):714-716. Nephrology Dialysis Transplantation can be contacted at: Oxford Univ Press, Great
Clarendon St, Oxford OX2 6DP, England. (Oxford University Press - http://www.oup.com/ ; Nephrology
Dialysis Transplantation - ndt.oxfordjournals.org)
The news correspondents report that additional information may be obtained from C. Zoccali, Osped Riuniti
Reggio Calabria, Hypertens & Renal Transplantat Unit, Nephrol, Reggio Di Calabria, Italy.
NewsRx LLC
(2016-06-20), Research Conducted by C. Zoccali and Co-Authors Has Provided New Information about Vitamin D Deficiency (Moderator’s view: Vitamin D deficiency treatment in advanced chronic kidney disease: a
close look at the emperor’s clothes), Clinical Trials Week, 696, ISSN: 1543-6764, BUTTER® ID: 011909873
Health and Medicine
Research Data from Ernst-Moritz-Arndt University Update Understanding of Medical Ethics
(Clinical decision-making and secondary findings in systems medicine)
By a News Reporter-Staff News Editor at Journal of Mathematics – Investigators publish new report on Health
and Medicine. According to news reporting from Greifswald, Germany, by VerticalNews journalists, research
stated, “Systems medicine is the name for an assemblage of scientific strategies and practices that include
bioinformatics approaches to human biology (especially systems biology); ‘big data’ statistical analysis; and
medical informatics tools. Whereas personalized and precision medicine involve similar analytical methods
applied to genomic and medical record data, systems medicine draws on these as well as other sources of data.”
The news correspondents obtained a quote from the research from Ernst-Moritz-Arndt University, “Given
this distinction, the clinical translation of systems medicine poses a number of important ethical and epistemological challenges for researchers working to generate systems medicine knowledge and clinicians working
to apply it. This article focuses on three key challenges: First, we will discuss the conflicts in decision-making
that can arise when healthcare providers committed to principles of experimental medicine or evidence-based
medicine encounter individualized recommendations derived from computer algorithms. We will explore in
particular whether controlled experiments, such as comparative effectiveness trials, should mediate the translation of systems medicine, or if instead individualized findings generated through ‘big data’ approaches can
be applied directly in clinical decision-making. Second, we will examine the case of the Riyadh Intensive Care
Program Mortality Prediction Algorithm, pejoratively referred to as the ‘death computer,’ to demonstrate the
ethical challenges that can arise when big-data-driven scoring systems are applied in clinical contexts. We
argue that the uncritical use of predictive clinical algorithms, including those envisioned for systems medicine,
challenge basic understandings of the doctor-patient relationship. Third, we will build on the recent discourse
on secondary findings in genomics and imaging to draw attention to the important implications of secondary
findings derived from the joint analysis of data from diverse sources, including data recorded by patients in an
attempt to realize their ‘ quantified self.’ Summary: This paper examines possible ethical challenges that are
likely to be raised as systems medicine to be translated into clinical medicine. These include the epistemological challenges for clinical decision-making, the use of scoring systems optimized by big data techniques and
the risk that incidental and secondary findings will significantly increase.”
According to the news reporters, the research concluded: “While some ethical implications remain still
hypothetical we should use the opportunity to prospectively identify challenges to avoid making foreseeable
mistakes when systems medicine inevitably arrives in routine care.”
For more information on this research see: Clinical decision-making and secondary findings in systems
medicine. BMC Medical Ethics , 2016;17():12-23. BMC Medical Ethics can be contacted at: Biomed Central Ltd,
189
236 Grays Inn Rd, Floor 6, London WC1X 8HL, England. (BioMed Central - http://www.biomedcentral.
com/ ; BMC Medical Ethics - http://www.biomedcentral.com/bmcmedethics/ )
Our news journalists report that additional information may be obtained by contacting T. Fischer, Ernst
Moritz Arndt Univ Greifswald, Fac Theol, Greifswald, Germany. Additional authors for this research include
K.B. Brothers, P. Erdmann and M. Langanke.
NewsRx LLC
(2016-06-21), Research Data from Ernst-Moritz-Arndt University Update Understanding of Medical Ethics
(Clinical decision-making and secondary findings in systems medicine), Journal of Mathematics, 429, ISSN:
1945-8746, BUTTER® ID: 011928266
Molecular Psychiatry
Researchers at Rush University Have Reported New Data on Molecular Psychiatry (Brain
connectomics predict response to treatment in social anxiety disorder)
By a News Reporter-Staff News Editor at Psychology & Psychiatry Journal – New research on Molecular Psychiatry is the subject of a report. According to news reporting originating in Chicago, Illinois, by VerticalNews
journalists, research stated, “We asked whether brain connectomics can predict response to treatment for a
neuropsychiatric disorder better than conventional clinical measures. Pre-treatment resting-state brain functional connectivity and diffusion-weighted structural connectivity were measured in 38 patients with social
anxiety disorder (SAD) to predict subsequent treatment response to cognitive behavioral therapy (CBT).”
The news reporters obtained a quote from the research from Rush University, “We used a priori bilateral
anatomical amygdala seed-driven resting connectivity and probabilistic tractography of the right inferior longitudinal fasciculus together with a data-driven multivoxel pattern analysis of whole-brain resting-state connectivity before treatment to predict improvement in social anxiety after CBT. Each connectomic measure improved
the prediction of individuals’ treatment outcomes significantly better than a clinical measure of initial severity,
and combining the multimodal connectomics yielded a fivefold improvement in predicting treatment response.
Generalization of the findings was supported by leave-one-out cross-validation. After dividing patients into
better or worse responders, logistic regression of connectomic predictors and initial severity combined with
leave-one-out cross-validation yielded a categorical prediction of clinical improvement with 81% accuracy, 84%
sensitivity and 78% specificity.”
According to the news reporters, the research concluded: “Connectomics of the human brain, measured by
widely available imaging methods, may provide brain-based biomarkers (neuromarkers) supporting precision
medicine that better guide patients with neuropsychiatric diseases to optimal available treatments, and thus
translate basic neuroimaging into medical practice.”
For more information on this research see: Brain connectomics predict response to treatment in social
anxiety disorder. Molecular Psychiatry , 2016;21(5):680-685. Molecular Psychiatry can be contacted at: Nature
Publishing Group, Macmillan Building, 4 Crinan St, London N1 9XW, England. (Nature Publishing Group http://www.nature.com/ ; Molecular Psychiatry - http://www.nature.com/mp/ )
Our news correspondents report that additional information may be obtained by contacting S. WhitfieldGabrieli, Rush University, Medical Center, Dept. of Psychiat, Chicago, IL 60612, United States. Additional
authors for this research include S.S. Ghosh, A. Nieto-Castanon, Z. Saygin, O. Doehrmann, X.J. Chai, G.O.
Reynolds, S.G. Hofmann, M.H. Pollack and J.D.E. Gabrieli.
NewsRx LLC
(2016-06-04), Researchers at Rush University Have Reported New Data on Molecular Psychiatry (Brain
connectomics predict response to treatment in social anxiety disorder), Psychology & Psychiatry Journal, 720,
ISSN: 1944-2726, BUTTER® ID: 011808286
190
Bacterial Infections and Mycoses
Researchers at Shantou University Have Reported New Data on Sepsis (Defining
immunological dysfunction in sepsis: A requisite tool for precision medicine)
By a News Reporter-Staff News Editor at Life Science Weekly – Current study results on Bacterial Infections
and Mycoses have been published. According to news reporting originating from Guangdong, People’s Republic
of China, by NewsRx correspondents, research stated, “Immunological dysregulation is now recognised as a
major pathogenic event in sepsis. Stimulation of immune response and immuno-modulation are emerging
approaches for the treatment of this disease.”
Our news editors obtained a quote from the research from Shantou University, “Defining the underlying
immunological alterations in sepsis is important for the design of future therapies with immuno-modulatory
drugs. Clinical studies evaluating the immunological response in adult patients with Sepsis and published in
PubMed were reviewed to identify features of immunological dysfunction. For this study we used key words
related with innate and adaptive immunity. Ten major features of immunological dysfunction (FID) were identified involving quantitative and qualitative alterations of [antigen presentation](FID1), [T and B lymphocytes]
(FID2), [natural killer cells] (FID3), [relative increase in T regulatory cells] (FID4), [increased expression of PD1 and PD-ligand1](FID5), [low levels of immunoglobulins](FID6), [low circulating counts of neutrophils and/or
increased immature forms in non survivors](FID7), [hyper-cytokinemia] (FID8), [complement consumption]
(FID9), [defective bacterial killing by neutrophil extracellular traps](FID10). This review article identified ten
major features associated with immunosuppression and immunological dysregulation in sepsis.”
According to the news editors, the research concluded: “Assessment of these features could help in utilizing
precision medicine for the treatment of sepsis with immuno-modulatory drugs.”
For more information on this research see: Defining immunological dysfunction in sepsis: A requisite tool
for precision medicine. Journal of Infection , 2016;72(5):525-536. Journal of Infection can be contacted at: W
B Saunders Co Ltd, 32 Jamestown Rd, London NW1 7BY, England. (Elsevier - www.elsevier.com; Journal of
Infection - http://www.journals.elsevier.com/journal-of-infection/ )
The news editors report that additional information may be obtained by contacting J.F. Bermejo-Martin,
Shantou University, Coll Med, Int Inst Infect & Immun, 22 Xinling Rd, Shantou 515041, Guangdong, People’s
Republic of China. Additional authors for this research include D. Andaluz-Ojeda, R. Almansa, F. Gandia, J.I.
Gomez-Herreras, E. Gomez-Sanchez, M. Heredia-Rodriguez, J.M. Eiros, D.J. Kelvin and E. Tamayo.
NewsRx LLC
(2016-05-31), Researchers at Shantou University Have Reported New Data on Sepsis (Defining immunological dysfunction in sepsis: A requisite tool for precision medicine), Life Science Weekly, 6702, ISSN: 1552-2474,
BUTTER® ID: 011772392
Molecular Research
Researchers at University of Puerto Rico Have Reported New Data on Molecular Research
(Biobanking of Exosomes in the Era of Precision Medicine: Are We There Yet?)
By a News Reporter-Staff News Editor at Health & Medicine Week – Investigators discuss new findings in
Molecular Research. According to news reporting originating from San Juan, Puerto Rico, by NewsRx correspondents, research stated, “The emerge of personalized medicine demands high-quality human biospecimens
with appropriate clinical annotation, especially in complex diseases such as cancer, neurodegenerative, cardiovascular, and metabolic alterations in which specimen heterogeneity and individual responses often complicate
the development of precision therapeutic programs. In the growing field of extracellular vesicles (EVs) research,
exosomes (EXOs)–a particular type of EVs–have been proposed as an advantageous diagnostic tool, as effective
delivery vehicles and as therapeutic targets.”
Our news editors obtained a quote from the research from the University of Puerto Rico, “However, the lack
of consensus on isolation methods and rigorous criteria to characterize them puts the term EXO into question
at the time that might explain some of the controversial results found in the literature. A lack of response in
the biobank network to warrant standard optimized procedures for the isolation, characterization, and storage
of EXOs will undoubtedly lead to a waste of resources and failure.”
According to the news editors, the research concluded: “This review is aimed at highlighting the increasing
importance of EXOs for the clinic, especially in the cancer field, and at summarizing the initiatives taken to
191
improve current isolation procedures, classification criteria, and storage conditions of EXOs as an effort to identify technological demands that biobank platforms face for the incorporation of EXOs and other extracellular
vesicle fractions as valuable biospecimens for research.”
For more information on this research see: Biobanking of Exosomes in the Era of Precision Medicine: Are
We There Yet? International Journal of Molecular Sciences , 2016;17(1):.
The news editors report that additional information may be obtained by contacting E.M. Mora, Dept. of
Surgery, School of Medicine, Medical Sciences Campus, University of Puerto Rico, San Juan 00936, Puerto
Rico. Additional authors for this research include S. Alvarez-Cubela and E. Oltra.
NewsRx LLC
(2016-05-27), Researchers at University of Puerto Rico Have Reported New Data on Molecular Research
(Biobanking of Exosomes in the Era of Precision Medicine: Are We There Yet?), Health & Medicine Week, 947,
ISSN: 1532-4605, BUTTER® ID: 011735917
Autoimmune Diseases and Disorders
Researchers from Technion-Israel Institute of Technology Report Findings in Multiple
Sclerosis (Shifting paradigms in multiple sclerosis: from disease-specific, through
population-specific toward patient-specific)
By a News Reporter-Staff News Editor at Clinical Trials Week – Investigators publish new report on Autoimmune Diseases and Disorders. According to news originating from Haifa, Israel, by NewsRx correspondents,
research stated, “In recent years we notice paradigm shifts in the understanding of multiple sclerosis (MS),
leading to important transition in the patients’ management. This review discusses some of the recent findings
and developments underlying the conceptual changes being translated from ‘treating the disease’ to ‘treating
the patient’ with MS (PwMS).”
Our news journalists obtained a quote from the research from the Technion-Israel Institute of Technology, “Applying advanced technologies combined with cross-disciplinary efforts in the fields of neuropathology,
neuroimmunology, neurobiology, and neuroimaging, together with clinical neurology provided support for the
notion that MS is not a single disease but rather a spectrum. Predictive markers of disease subtypes, disease activity and response to therapy are being developed; some already applied to practice, allowing informed
management. In parallel, population-specific issues, some genetic-driven, others caused by environmental (sunexposure, life-style, etc.), gender-related (hormones) and epigenetic factors, are being elucidated. Additionally,
patient empowerment-based approaches, including integration of patient-reported outcome measures (PRO)
as well as tools to enhance patients’ adherence to medications, are being developed, some already provided as
part of emerging mobile-health technologies.”
According to the news editors, the research concluded: “Developments in the MS field, elucidating disease
subtypes and interpopulation diversities, together with integration of patient-centered approaches, allow transition toward precision medicine in MS clinical trials and patient care.”
For more information on this research see: Shifting paradigms in multiple sclerosis: from disease-specific,
through population-specific toward patient-specific. Current Opinion in Neurology , 2016;29(3):354-361. Current Opinion in Neurology can be contacted at: Lippincott Williams & Wilkins, Two Commerce Sq, 2001 Market
St, Philadelphia, PA 19103, USA. (Lippincott Williams and Wilkins - www.lww.com; Current Opinion in Neurology - http://journals.lww.com/co-neurology/pages/default.aspx )
The news correspondents report that additional information may be obtained from D. Golan, Technion Israel
Inst Technol, Ruth & Bruce Rappaport Fac Med, Haifa, Israel. Additional authors for this research include E.
Staun-Ram and A. Miller.
NewsRx LLC
(2016-05-30), Researchers from Technion-Israel Institute of Technology Report Findings in Multiple Sclerosis (Shifting paradigms in multiple sclerosis: from disease-specific, through population-specific toward patientspecific), Clinical Trials Week, 422, ISSN: 1543-6764, BUTTER® ID: 011747989
192
Skin Diseases and Conditions
Researchers from Transylvania University Report Findings in Atopic Dermatitis (Precision
medicine in patients with allergic diseases: Airway diseases and atopic
dermatitis-PRACTALL document of the European Academy of Allergy and Clinical ...)
By a News Reporter-Staff News Editor at Respiratory Therapeutics Week – New research on Skin Diseases and
Conditions is the subject of a report. According to news reporting from Brasov, Romania, by NewsRx journalists,
research stated, “In this consensus document we summarize the current knowledge on major asthma, rhinitis,
and atopic dermatitis endotypes under the auspices of the PRACTALL collaboration platform. PRACTALL
is an initiative of the European Academy of Allergy and Clinical Immunology and the American Academy of
Allergy, Asthma & Immunology aiming to harmonize the European and American approaches to best allergy
practice and science.”
The news correspondents obtained a quote from the research from Transylvania University, “Precision
medicine is of broad relevance for the management of asthma, rhinitis, and atopic dermatitis in the context of a
better selection of treatment responders, risk prediction, and design of disease-modifying strategies. Progress
has been made in profiling the type 2 immune response-driven asthma. The endotype driven approach for nontype 2 immune response asthma, rhinitis, and atopic dermatitis is lagging behind. Validation and qualification
of biomarkers are needed to facilitate their translation into pathway-specific diagnostic tests. Wide consensus
between academia, governmental regulators, and industry for further development and application of precision
medicine in management of allergic diseases is of utmost importance.”
According to the news reporters, the research concluded: “Improved knowledge of disease pathogenesis
together with defining validated and qualified biomarkers are key approaches to precision medicine.”
For more information on this research see: Precision medicine in patients with allergic diseases: Airway diseases and atopic dermatitis-PRACTALL document of the European Academy of Allergy and Clinical Immunology and the American Academy of Allergy, Asthma & Immunology. Journal of Allergy
and Clinical Immunology , 2016;137(5):1347-1358. Journal of Allergy and Clinical Immunology can
be contacted at: Mosby-Elsevier, 360 Park Avenue South, New York, NY 10010-1710, USA. (Elsevier www.elsevier.com; Journal of Allergy and Clinical Immunology - http://www.journals.elsevier.com/
journal-of-allergy-and-clinical-immunology/ )
Our news journalists report that additional information may be obtained by contacting A. Muraro, Transylvania Univ Brasov, Dept. of Allergy & Clin Immunol, Brasov, Romania. Additional authors for this research
include R.F. Lemanske, P.W. Hellings, C.A. Akdis, T. Bieber, T.B. Casale, M. Jutel, P.Y. Ong, L.K. Poulsen, P.
Schmid-Grendelmeier, H.U. Simon, S.F. Seys and I. Agache.
NewsRx LLC
(2016-06-13), Researchers from Transylvania University Report Findings in Atopic Dermatitis (Precision
medicine in patients with allergic diseases: Airway diseases and atopic dermatitis-PRACTALL document of the
European Academy of Allergy and Clinical ...), Respiratory Therapeutics Week, 88, ISSN: 1543-6664, BUTTER®
ID: 011828085
Enzymes and Coenzymes
Researchers from University of Missouri Describe Findings in Enzymes and Coenzymes
(Induced Structural Disorder as a Molecular Mechanism for Enzyme Dysfunction in
Phosphoglucomutase 1 Deficiency)
By a News Reporter-Staff News Editor at Life Science Weekly – Data detailed on Enzymes and Coenzymes
have been presented. According to news reporting originating in Columbia, Missouri, by NewsRx journalists,
research stated, “Human phosphoglucomutase 1 (PGM1) plays a central role in cellular glucose homeostasis,
mediating the switch between glycolysis and gluconeogenesis through the conversion of glucose 1-phosphate
and glucose 6-phosphate. Recent clinical studies have identified mutations in this enzyme as the cause of
PGM1 deficiency, an inborn error of metabolism classified as both a glycogen storage disease and a congenital
disorder of glycosylation.”
The news reporters obtained a quote from the research from the University of Missouri, “Reported here are
the first crystal structures of two disease-related missense variants of PGM1, along with the structure of the
wild-type enzyme. Two independent glycine-to-arginine substitutions (G121R and G291R), both affecting key
193
active site loops of PGM1, are found to induce regions of structural disorder, as evidenced by a nearly complete
loss of electron density for as many as 23 aa. The disordered regions are not contiguous in sequence to the site of
mutation, and even cross domain boundaries. Other structural rearrangements include changes in the conformations of loops and side chains, some of which occur nearly 20 A away from the site of mutation. The induced
structural disorder is correlated with increased sensitivity to proteolysis and lower-resolution diffraction, particularly for the G291R variant. Examination of the multi-domain effects of these G– >R mutations establishes
a correlation between interdomain interfaces of the enzyme and missense variants of PGM1 associated with
disease.”
According to the news reporters, the research concluded: “These crystal structures provide the first insights
into the structural basis of enzyme dysfunction in PGM1 deficiency and highlight a growing role for biophysical
characterization of proteins in the field of precision medicine.”
For more information on this research see: Induced Structural Disorder as a Molecular Mechanism for
Enzyme Dysfunction in Phosphoglucomutase 1 Deficiency. Journal of Molecular Biology , 2016;428(8):14931505. Journal of Molecular Biology can be contacted at: Academic Press Ltd- Elsevier Science Ltd, 24-28
Oval Rd, London NW1 7DX, England. (Elsevier - www.elsevier.com; Journal of Molecular Biology - http:
//www.journals.elsevier.com/journal-of-molecular-biology/ )
Our news correspondents report that additional information may be obtained by contacting K.M. Stiers,
University of Missouri, Dept. of Biochem, 117 Schweitzer Hall, Columbia, MO 65211, United States. Additional
authors for this research include B.N. Kain, A.C. Graham and L.J. Beamer.
NewsRx LLC
(2016-05-31), Researchers from University of Missouri Describe Findings in Enzymes and Coenzymes (Induced Structural Disorder as a Molecular Mechanism for Enzyme Dysfunction in Phosphoglucomutase 1 Deficiency), Life Science Weekly, 1283, ISSN: 1552-2474, BUTTER® ID: 011767805
Researchers from Washington University School of Medicine Describe Findings in Nucleic
Acids Research (DGIdb 2.0: mining clinically relevant drug-gene interactions)
By a News Reporter-Staff News Editor at Clinical Trials Week – Research findings on Life Science Research
are discussed in a new report. According to news reporting from St. Louis, Missouri, by NewsRx journalists,
research stated, “The Drug-Gene Interaction Database (DGIdb, www.dgidb.org) is a web resource that consolidates disparate data sources describing drug-gene interactions and gene druggability. It provides an intuitive
graphical user interface and a documented application programming interface (API) for querying these data.”
The news correspondents obtained a quote from the research from the Washington University School of
Medicine, “DGIdb was assembled through an extensive manual curation effort, reflecting the combined information of twenty-seven sources. For DGIdb 2.0, substantial updates have been made to increase content and
improve its usefulness as a resource for mining clinically actionable drug targets. Specifically, nine new sources
of drug-gene interactions have been added, including seven resources specifically focused on interactions linked
to clinical trials. These additions have more than doubled the overall count of drug-gene interactions. The total number of druggable gene claims has also increased by 30%. Importantly, a majority of the unrestricted,
publicly-accessible sources used in DGIdb are now automatically updated on a weekly basis, providing the most
current information for these sources. Finally, a new web view and API have been developed to allow searching
for interactions by drug identifiers to complement existing gene-based search functionality.”
According to the news reporters, the research concluded: “With these updates, DGIdb represents a comprehensive and user friendly tool for mining the druggable genome for precision medicine hypothesis generation.”
For more information on this research see: DGIdb 2.0: mining clinically relevant drug-gene interactions.
Nucleic Acids Symposium Series , 2016;44(D1):D1036-44. (Oxford University Press - http://www.oup.com/
; Nucleic Acids Symposium Series - nass.oxfordjournals.org)
Our news journalists report that additional information may be obtained by contacting A.H. Wagner, McDonnell Genome Institute, Washington University School of Medicine, St Louis, MO 63108, United States.
Additional authors for this research include A.C. Coffman, B.J. Ainscough, N.C. Spies, Z.L. Skidmore, K.M.
Campbell, K. Krysiak, D. Pan, J.F. McMichael, J.M. Eldred, J.R. Walker, R.K. Wilson, E.R. Mardis, M. Griffith
and O.L Griffith.
NewsRx LLC
194
(2016-05-30), Researchers from Washington University School of Medicine Describe Findings in Nucleic
Acids Research (DGIdb 2.0: mining clinically relevant drug-gene interactions), Clinical Trials Week, 1644,
ISSN: 1543-6764, BUTTER® ID: 011749127
SCIEX
SCIEX Announces High Throughput, Industrialized Omics Solutions at ASMS 2016
By a News Reporter-Staff News Editor at Journal of Engineering – SCIEX, a global leader in life science analytical technologies, announced their latest proteomics solution advancements, which address the challenges
of throughput, reproducibility and robustness faced by Academic Labs working to advance precision medicine.
The sample sets involved in this research are getting larger and increasingly unmanageable through traditional
proteomics research approaches, and significant workflow improvements are required to scale omics analysis.
SCIEX has now advanced their platforms to industrialized performance levels, adding new tools for customers
to analyze larger sample sets, faster and with more confidence.
The SCIEX Next-Generation Proteomics (NGP) platform offers solutions for large-scale quantitative proteomics with technologies such as the TripleTOF® 6600 system with SWATH® 2.0 Acquisition, to perform
quantitative proteomics research by solving the missing data problem, enhancing data processing and results
visualization, with retrospective analysis, and OneOmics™ cloud computing. Bringing these technologies together has set the standard for truly comprehensive data with the most confident and reproducible protein
quant for thousands of proteins in hundreds of samples.
To advance to the scale required for Precision Medicine studies, researchers need to understand disease
at the molecular level, and they need to generate comprehensive, reproducible biological results over large
sample cohorts. Often this work involves complex samples preparation, as researchers compare healthy and
diseased samples in pursuit of potential biomarkers, which could be genes, proteins or metabolites. This work
requires higher levels of reproducibility and greater sample throughput than typically associated with basic
omics research.
Additions to the SCIEX NGP platform for industrialized proteomics includes: New sample prep automation
with the Beckman Coulter® Biomek NX, powered by Protein Preparation Kits: The workhorse of the industry,
the Biomek NX powered by Protein Preparation Kits, automates manual tedious steps, for improved reproducibility. Microflow SWATH Acquisition: Offers a higher flow rate for significant time savings and improved
robustness, while maintaining the high quantitative quality of SWATH acquisition. SWATH Performance Kits:
Facilitates a quick start-up by enabling customers to check the performance of their SWATH Acquisition and
monitor performance across their sample sets. OneOmics AutoUploader: Featured in CloudConnect, AutoUploader moves data files automatically to the cloud right after acquisition to further streamline the quantitative
proteomics workflow. SWATH to MRM Builder: This new Cloud enables researchers to easily transition from
SWATH to MRM workflows, bridging the gap between discovery and targeted validation.
“Life science research is constantly evolving, and the tools we need must also evolve. With our increasing
focus on precision medicine, we have needed to scale up workflows in our lab by running larger studies while
still controlling the accuracy and precision of our quantitative results,” said, Jenny Van Eyk, Director, Advanced Clinical Biosystems Research Institute, Cedars Sinai Medical Center. “SCIEX’s Industrialized Omics
solutions provide the automation and increased throughput that enables researchers like me to scale up while
still generating high quality data that help us understand disease.”
“For SCIEX, it’s all about providing the technologies our customers need to address the extremely complex
work associated with translational research academic lab. Our industrialized proteomics solutions provide the
speed, sensitivity, and quality data needed to advance life science research and help our customers advance
precision medicine. And our efforts are not limited to proteomics. We employed the same principles in the
recently-launched Lipidyzer™ Platform for industrialized lipidomics research”, said Mark Cafazzo, Director of
Academic Business at SCIEX. “Our mass spec-based solutions enable quantitative biology at an industrialized
level that no other mass spec provider can match.” The Lipidyzer Platform and its role in understanding lipids
and disease and Metabolomics for personalized medicine, among other topics, will be discussed in the SCIEX
users meetings held on Sunday June 5th, 2016 - Register for the user meeting > About SCIEX SCIEX helps
to improve the world we live in by enabling scientists and laboratory analysts to find answers to the complex
analytical challenges they face. The company’s global leadership and world-class service and support in the
capillary electrophoresis and liquid chromatography-mass spectrometry industry have made it a trusted partner to thousands of the scientists and lab analysts worldwide who are focused on basic research, drug discovery
and development, food and environmental testing, forensics and clinical research.
195
With over 40 years of proven innovation, SCIEX excels by listening to and understanding the ever-evolving
needs of its customers to develop reliable, sensitive and intuitive solutions that continue to redefine what is
achievable in routine and complex analysis. For more information, please visit sciex.com.
SCIEX social: Twitter: @SCIEXnews, LinkedIn and Facebook.
For Research Use Only. Not for use in diagnostic procedures.
AB Sciex is operating as SCIEX. SCIEX and Beckman Coulter® are Danaher Corporation companies.
© 2016 AB Sciex. The trademarks mentioned herein are the property of the AB Sciex Pte. Ltd. or their
respective owners. AB Sciex™ is being used under license.
RUO-MKT-12-4073-A View source version on businesswire.com: http://www.businesswire.com/
news/home/20160606005088/en/
NewsRx LLC
(2016-06-20), SCIEX Announces High Throughput, Industrialized Omics Solutions at ASMS 2016, Journal
of Engineering, 1324, ISSN: 1945-872X, BUTTER® ID: 011916121
Breast Cancer
Scripps Florida scientists pioneer a breakthrough approach to breast cancer treatment
By a News Reporter-Staff News Editor at Women’s Health Weekly – JUPITER, FL - In a development that could
lead to a new generation of drugs to precisely treat a range of diseases, scientists from the Florida campus of
The Scripps Research Institute (TSRI) have for the first time designed a drug candidate that decreases the
growth of tumor cells in animal models in one of the hardest to treat cancers–triple negative breast cancer.
“This is the first example of taking a genetic sequence and designing a drug candidate that works effectively
in an animal model against triple negative breast cancer,” said TSRI Professor Matthew Disney. “The study
represents a clear breakthrough in precision medicine, as this molecule only kills the cancer cells that express
the cancer-causing gene–not healthy cells. These studies may transform the way the lead drugs are identified–
by using the genetic makeup of a disease.”
The study, published online ahead of print the week of May 9, 2016, by the journal Proceedings of the National Academy of Sciences, demonstrates that the Disney lab’s compound, known as Targaprimir-96, triggers
breast cancer cells to kill themselves via programmed cell death by precisely targeting a specific RNA that
ignites the cancer.
Short-Cut to Drug Candidates
While the goal of precision medicine is to identify drugs that selectively affect disease-causing biomolecules,
the process has typically involved time-consuming and expensive high-throughput screens to test millions of
potential drug candidates to identify those few that affect the target of interest. Disney’s approach eliminates
these screens.
The new study uses the lab’s computational approach called Inforna, which focuses on developing designer
compounds that bind to RNA folds, particularly microRNAs.
MicroRNAs are short molecules that work within all animal and plant cells, typically functioning as a “dimmer switch” for one or more genes, binding to the transcripts of those genes and preventing protein production.
Some microRNAs have been associated with diseases. For example, microRNA-96, which was the target of the
new study, promotes cancer by discouraging programmed cell death, which can rid the body of cells that grow
out of control.
In the new study, the drug candidate was tested in animal models over a 21-day course of treatment. Results
showed decreased production of microRNA-96 and increased programmed cell death, significantly reducing
tumor growth. Since targaprimir-96 was highly selective in its targeting, healthy cells were unaffected.
In contrast, Disney noted, a typical cancer therapeutic targets and kills cells indiscriminately, often leading
to side effects that can make these drugs difficult for patients to tolerate.
“In the future we hope to apply this strategy to target other disease-causing RNAs, which range from incurable cancers to important viral pathogens such as Zika and Ebola,” added Research Associate Sai Pradeep
Velagapudi, the first author of the study and a member of the Disney lab.
NewsRx LLC
(2016-05-26), Scripps Florida scientists pioneer a breakthrough approach to breast cancer treatment,
Women’s Health Weekly, 79, ISSN: 1532-4729, BUTTER® ID: 011732319
196
Oncology
Studies from Johns Hopkins University Add New Findings in the Area of Prostate Cancer
(Novel mechanism-based therapeutics for androgen axis blockade in castration-resistant
prostate cancer)
By a News Reporter-Staff News Editor at Biotech Week – New research on Oncology is the subject of a report.
According to news reporting out of Baltimore, Maryland, by NewsRx editors, research stated, “Understanding
the mechanisms by which castration-resistant prostate cancer (CRPC) progresses provides an opportunity to
identify novel therapeutic strategies to treat this disease. This understanding has led to approaches to attack
prostate cancer’s androgen axis in unique ways.”
Our news journalists obtained a quote from the research from Johns Hopkins University, “This review will
examine the classes of novel therapies for androgen axis blockade in CRPC, with a particular focus on the unique
characteristics of drugs in various stages of clinical development. The success of abiraterone and enzalutamide
has stimulated multiple investigations into novel approaches to attack the androgen-signaling pathway. Drugs
under development include cytochrome P17 inhibitors with 17,20-lyase specificity, androgen receptor antagonists that are active against mutated and constitutively active splice variant forms of the protein, androgen
receptor degraders, and bromodomain/bromodomain extra-terminal inhibitors that prevent chromatin binding
of activated receptors. The clinical development of several of these experimental agents is reviewed. Given the
unique mechanisms of action for drugs in development, and the possibility that the novel agents may be active
in the setting of common resistance mechanisms, treatment options for patients are likely to expand greatly in
the coming years.”
According to the news editors, the research concluded: “Future studies should prioritize combinations of
agents with unique mechanisms of action to optimize outcomes for patients, and should rely on precisionmedicine approaches to target known molecular alterations.”
For more information on this research see: Novel mechanism-based therapeutics for androgen axis
blockade in castration-resistant prostate cancer. Current Opinion in Endocrinology Diabetes and Obesity ,
2016;23(3):279-290. Current Opinion in Endocrinology Diabetes and Obesity can be contacted at: Lippincott
Williams & Wilkins, Two Commerce Sq, 2001 Market St, Philadelphia, PA 19103, USA.
Our news journalists report that additional information may be obtained by contacting B.A. Teply, Johns
Hopkins University, Sch Med, Dept. of Oncol, Baltimore, MD 21205, United States.
NewsRx LLC
(2016-06-01), Studies from Johns Hopkins University Add New Findings in the Area of Prostate Cancer (Novel mechanism-based therapeutics for androgen axis blockade in castration-resistant prostate cancer),
Biotech Week, 607, ISSN: 1537-4699, BUTTER® ID: 011783950
Oncology
Studies from National Institute of Science and Technology Have Provided New Information
about Breast Cancer (Toward precision medicine of breast cancer)
By a News Reporter-Staff News Editor at Cancer Weekly – Fresh data on Oncology are presented in a new report. According to news reporting originating from Rio de Janeiro, Brazil, by NewsRx correspondents, research
stated, “In this review, we report on breast cancer’s molecular features and on how high throughput technologies are helping in understanding the dynamics of tumorigenesis and cancer progression with the aim of
developing precision medicine methods. We first address the current state of the art in breast cancer therapies
and challenges in order to progress towards its cure.”
Our news editors obtained a quote from the research from the National Institute of Science and Technology,
“Then, we show how the interaction of high-throughput technologies with in silico modeling has led to set up
useful inferences for promising strategies of target-specific therapies with low secondary effect incidence for
patients. Finally, we discuss the challenge of pharmacogenetics in the clinical practice of cancer therapy.”
According to the news editors, the research concluded: “All these issues are explored within the context of
For more information on this research see: Toward precision medicine of breast cancer. Theoretical Biology & Medical Modelling , 2016;13():7. (BioMed Central - http://www.biomedcentral.com/ ; Theoretical
Biology & Medical Modelling - www.tbiomed.com)
197
The news editors report that additional information may be obtained by contacting N. Carels, Laboratorio
de Modelagem de Sistemas Biologicos, National Institute of Science and Technology for Innovation in Neglected
Diseases (INCT, IDN, CNPq), Centro de Desenvolvimento Tecnologico em Saude, Fundacao Oswaldo Cruz, Rio
de Janeiro, Brazil. Additional authors for this research include L.B. Spinasse, T.M. Tilli and J.A Tuszynski.
NewsRx LLC
(2016-06-07), Studies from National Institute of Science and Technology Have Provided New Information
about Breast Cancer (Toward precision medicine of breast cancer), Cancer Weekly, 730, ISSN: 1532-4567, BUTTER® ID: 011875258
Cardiovascular Diseases and Conditions
Studies from University of Glasgow Further Understanding of Hypertension (Use of
Biomarkers in the Evaluation and Treatment of Hypertensive Patients)
By a News Reporter-Staff News Editor at Cardiovascular Week – Research findings on Cardiovascular Diseases
and Conditions are discussed in a new report. According to news reporting out of Glasgow, United Kingdom,
by NewsRx editors, research stated, “The current definition of hypertension is based on blood pressure values,
and blood pressure also drives treatment decisions, is the most important treatment monitoring tool and helps
estimating risk of hypertension-related organ damage. In an era of precision medicine, additional biomarkers
are needed in the diagnosis and management of patients with hypertension.”
Our news journalists obtained a quote from the research from the University of Glasgow, “In this review, we
outline the areas in which functional, imaging and circulating biomarkers could help in a more individualised
definition of hypertension and associated risk. We will cover biomarkers for diagnosis; of pathophysiology and
prediction of hypertension; response to treatment, organ damage; and to monitor treatment.”
According to the news editors, the research concluded: “A clear focus is on the vasculature, the heart and
the kidneys, whereas we see a need to further develop biomarkers of cerebral function in order to diagnose
cognition deficits and monitor changes in cognition in the future to support addressing the growing burden of
hypertension-associated vascular dementia.”
For more information on this research see: Use of Biomarkers in the Evaluation and Treatment of Hypertensive Patients. Current Hypertension Reports , 2016;18(7):37-49. Current Hypertension Reports can be contacted
at: Springer, 233 Spring St, New York, NY 10013, USA. (Springer - www.springer.com; Current Hypertension
Reports - http://www.springerlink.com/content/1522-6417/ )
Our news journalists report that additional information may be obtained by contacting G. Currie, University
of Glasgow, BHF Glasgow Cardiovasc Res Center, Inst Cardiovasc & Med Sci, 126 Univ Pl, Glasgow G12 8TA,
Lanark, United Kingdom.
NewsRx LLC
(2016-07-25), Studies from University of Glasgow Further Understanding of Hypertension (Use of Biomarkers in the Evaluation and Treatment of Hypertensive Patients), Cardiovascular Week, 278, ISSN: 1543-6845,
BUTTER® ID: 012089732
198
Health and Medicine
Studies from University of North Carolina Yield New Information about Medical Ethics
(Citizen science or scientific citizenship? Disentangling the uses of public engagement
rhetoric in national research initiatives)
By a News Reporter-Staff News Editor at Biotech Week – Fresh data on Health and Medicine are presented in
a new report. According to news reporting from Chapel Hill, North Carolina, by NewsRx journalists, research
stated, “The language of ‘participant-driven research,’ ‘crowdsourcing’ and ‘citizen science’ is increasingly being
used to encourage the public to become involved in research ventures as both subjects and scientists. Originally,
these labels were invoked by volunteer research efforts propelled by amateurs outside of traditional research
institutions and aimed at appealing to those looking for more ‘democratic,’ centric,’ or ‘lay’ alternatives to the
professional science establishment.”
The news correspondents obtained a quote from the research from the University of North Carolina, “As
mainstream translational biomedical research requires increasingly larger participant pools, however, corporate, academic and governmental research programs are embracing this populist rhetoric to encourage wider
public participation. We examine the ethical and social implications of this recruitment strategy. We begin by
surveying examples of ‘citizen science’ outside of biomedicine, as paradigmatic of the aspirations this democratizing rhetoric was originally meant to embody. Next, we discuss the ways these aspirations become articulated
in the biomedical context, with a view to drawing out the multiple and potentially conflicting meanings of ‘public
engagement’ when citizens are also the subjects of the science. We then illustrate two uses of public engagement rhetoric to gain public support for national biomedical research efforts: its post-hoc use in the ‘care.data’
project of the National Health Service in England, and its proactive uses in the ‘Precision Medicine Initiative’
of the United States White House. These examples will serve as the basis for a normative analysis, discussing
the potential ethical and social ramifications of this rhetoric. We pay particular attention to the implications
of government strategies that cultivate the idea that members of the public have a civic duty to participate in
government-sponsored research initiatives. We argue that such initiatives should draw from policy frameworks
that support normative analysis of the role of citizenry.”
According to the news reporters, the research concluded: “And, we conclude it is imperative to make visible
and clear the full spectrum of meanings of ‘citizen science,’ the contexts in which it is used, and its demands
with respect to participation, engagement, and governance.”
For more information on this research see: Citizen science or scientific citizenship? Disentangling the
uses of public engagement rhetoric in national research initiatives. BMC Medical Ethics , 2016;17():1-17.
BMC Medical Ethics can be contacted at: Biomed Central Ltd, 236 Grays Inn Rd, Floor 6, London WC1X
8HL, England. (BioMed Central - http://www.biomedcentral.com/ ; BMC Medical Ethics - http://www.
biomedcentral.com/bmcmedethics/ )
Our news journalists report that additional information may be obtained by contacting J.P. Woolley, University of North Carolina, 333 MacNider HallCampus Box 7240, Chapel Hill, NC 27599, United States. Additional
authors for this research include M.L. McGowan, H.J.A. Teare, V. Coathup, J.R. Fishman, R.A. Settersten, S.
Sterckx, J. Kaye and E.T. Juengst.
NewsRx LLC
(2016-06-29), Studies from University of North Carolina Yield New Information about Medical Ethics (Citizen science or scientific citizenship? Disentangling the uses of public engagement rhetoric in national research
initiatives), Biotech Week, 584, ISSN: 1537-4699, BUTTER® ID: 011968412
Health and Medicine
Study Results from University of Washington Provide New Insights into Medical Decision
Making (A Framework for Prioritizing Research Investments in Precision Medicine)
By a News Reporter-Staff News Editor at Investment Weekly News – Current study results on Health and
Medicine have been published. According to news reporting from Seattle, Washington, by VerticalNews journalists, research stated, “The adoption of precision medicine (PM) has been limited in practice to date, and yet
its promise has attracted research investments. Developing foundational economic approaches for directing
proper use of PM and stimulating growth in this area from multiple perspectives is thus quite timely.”
The news correspondents obtained a quote from the research from the University of Washington, “Building
on our previously developed expected value of individualized care (EVIC) framework, we conceptualize new
199
decision-relevant metrics to better understand and forecast the expected value of PM. Several aspects of behavior at the patient, physician, and payer levels are considered that can inform the rate and manner in which PM
innovations diffuse throughout the relevant population. We illustrate this framework and the methods using
a retrospective evaluation of the use of OncotypeDx genomic test among breast cancer patients. The enriched
metrics can help inform many facets of PM decision making, such as evaluating alternative reimbursement
levels for PM tests, implementation and education programs for physicians and patients, and decisions around
research investments by manufacturers and public entities. We replicated prior published results on evaluation
of OncotypeDx among breast cancer patients but also illustrated that those results are based on assumptions
that are often not met in practice. Instead, we show how incorporating more practical aspects of behavior
around PM could lead to drastically different estimates of value. We believe that the framework and the methods presented can provide decision makers with more decision-relevant tools to explore the value of PM. There
is a growing recognition that data on adoption is important to decision makers.”
According to the news reporters, the research concluded: “More research is needed to develop prediction
models for potential diffusion of PM technologies.”
For more information on this research see: A Framework for Prioritizing Research Investments in Precision Medicine. Medical Decision Making , 2016;36(5):567-580. Medical Decision Making can be contacted
at: Sage Publications Inc, 2455 Teller Rd, Thousand Oaks, CA 91320, USA. (Sage Publications - http:
//www.sagepub.com/ ; Medical Decision Making - mdm.sagepub.com)
Our news journalists report that additional information may be obtained by contacting A. Basu, University
of Washington, Dept. of Econ, 1959 NE Pacific StBox 357660, Seattle, WA 98195, United States. Additional
authors for this research include J.J. Carlson and D.L. Veenstra.
NewsRx LLC
(2016-07-02), Study Results from University of Washington Provide New Insights into Medical Decision
Making (A Framework for Prioritizing Research Investments in Precision Medicine), Investment Weekly News,
718, ISSN: 1945-8185, BUTTER® ID: 011981670
Research and Markets
Technologies Impacting the Future of the Healthcare Sector - Increasing Trend Toward
Personalized & Precise Medical Treatments - Research and Markets
By a News Reporter-Staff News Editor at Journal of Engineering – Research and Markets has announced the
addition of the “Technologies Impacting the Future of the Healthcare Sector” report to their offering.
Technological advancements are disrupting the healthcare industry. There is an increasing trend toward
personalized and precise medical treatments, such as personalized genomics, which provide outputs at a much
lower cost and at a faster speed than has ever been possible. Precision medicine has also driven the development
of targeted drug delivery, which improves the efficacy of drugs, while reducing side effects.
The increasing occurrence of auto-immune disorders has spurred research interest in microbiome therapies,
which focus on using the body’s own microbiota as a defense against pathogens. Another hot technology area
revolutionizing neuroscience is optogenetics, which attempts to rewire the brain through the use of optics and
genetics. The success of optogenetics in vision recovery has provided an impetus for funding in this space.
The rising incidence of chronic and lifestyle disorders, along with the difficulty of finding donors, has led to
research in printing live tissue, currently used in pharma for toxicology testing. Long-term R&D in this space
is geared toward the development of 3D printed organs, which truly represent the next frontier in healthcare
advancement.
However, just as technologies are disrupting healthcare, the reverse can be said to hold true. Healthcare
is moving toward increased patient empowerment, leading to the development of wearables, mHealth and
telemedicine. Advances in Big Data and Cloud Computing have enabled more real-time healthcare solutions
such as biosensors, leading to greater patient engagement. Digital transformation has also been key to enabling
path-breaking development in genomics platforms, such as rapid genome sequencing.
Organizations are moving toward new care delivery models that increasingly focus on delivering patient
value, leading to redefined revenue pathways. This research service provides a deep-dive analysis into some of
the most disruptive technologies, trends and business models impacting healthcare today, providing decisionmakers with key insights for strategic planning. Key Topics Covered: 1. Executive Summary
2. Industry Overview
3. Technology and Market Trends
200
4. The biggest Game Changers in Healthcare
For
more
information
visit
http://www.researchandmarkets.com/research/nk6qf4/
technologies View source version on businesswire.com: http://www.businesswire.com/news/
home/20160523005994/en/
NewsRx LLC
(2016-06-06), Technologies Impacting the Future of the Healthcare Sector - Increasing Trend Toward Personalized & Precise Medical Treatments - Research and Markets, Journal of Engineering, 2233, ISSN: 1945872X, BUTTER® ID: 011869376
SCIEX
The University of Manchester and SCIEX Opened the £18m Stoller Biomarker Discovery
Centre on June 14th
By a News Reporter-Staff News Editor at Journal of Engineering – The University of Manchester opened the
multi-million pound Stoller Biomarker Discovery Centre on June 14th 2016. It focuses on biomedical research
including cancer, psoriasis and arthritis, utilizing mass spectrometry-based proteomics solutions from SCIEX.
The opening of the Centre was marked with a special two-day scientific event, the Stoller Biomarker Discovery
Centre Symposium, which took place on June 14th-15th 2016.
SCIEX’s proteomics systems have been selected to deliver the comprehensive data that is so urgently needed
in precision medicine. SCIEX’s patented SWATH® technology enables the quantification of thousands of proteins across large sample sets with a level of data completeness, quantitative accuracy, and reproducibility that
was only previously achievable with ‘gold-standard’ targeted multiple reaction monitoring (MRM) methods. The
SWATH next-generation proteomics technology will allow University scientists to collaborate with health companies and the National Health Service (NHS) to produce a greater number of tests and treatments, ultimately
accelerating the process of eliminating many of the most serious illnesses faced today.
Professor Tony Whetton is the Director of the new Centre, and said: “The Centre is a major step forward
in Precision Medicine. Essentially this is the future of healthcare - getting the right treatment to the right
person at the right time and in the right dose. Without biomarkers we won’t be able to identify which people
will benefit from certain medicines, so this new centre underpins everything we’re doing in precision medicine
in Manchester and beyond.”
The Stoller Biomarker Centre is located at CityLabs Manchester, in the midst of biotechnology companies,
the Central Manchester University Hospitals, National Health Service (NHS) Foundation Trust and The University of Manchester. The new Centre houses a large suite of high-end SCIEX mass spectrometers for targeted
next-generation proteomics, including TripleTOF® 6600 Systems with SWATH Acquisition, QTRAP® 6500+
Systems, and the SCIEX LipidyzerTM Platform. The University of Manchester has also invested in a number of
liquid chromatography and automated sample preparation components for the Centre, from SCIEX and other
Danaher life science companies, such as Beckman Coulter’s Biomek NXP Laboratory Automated Workstation.
“SCIEX’s mission of innovating integrated, reliable analytical tools to gain scientific understandings that
lead to better health, enables our customers to advance precision medicine with scale and speed like never before,” states Jean-Paul Mangeolle, President of SCIEX. “And it takes more than providing great instruments to
be part of a movement as important as Precision Medicine; it takes strong collaborations with customers, partnerships with industry leaders and teamwork with our colleagues at other Danaher Corporation life companies,
to establish and deploy the most comprehensive proteomics solutions.”
The Centre was officially opened at the event attended by Professor Dame Nancy Rothwell, President and
Vice-Chancellor of The University of Manchester, Sir Norman Stoller and trustees of the Stoller Charitable
Trust, and Dan Daniel, Executive Vice President of Danaher, with a ceremony during the Stoller Biomarker
Discovery Centre Symposium. At the conference, leading speakers from around the globe shared their insights
into key topics surrounding precision medicine, including pioneers within the field such as Dr Leroy Hood, Dr
Leigh Anderson, and Professor Jennifer Van Eyk. Professor Rothwell said: “Manchester has become a major
hub for precision medicine and proteomics and we are very grateful to the funders who have backed the cuttingedge work that is carried out by our scientists. As a result of their generosity, The Stoller Biomarker Discovery
Centre will start work on addressing some of the biggest issues in medicine in an environment where these
discoveries can move quickly to improve people’s lives.” About SCIEX SCIEX helps to improve the world we
live in by enabling scientists and laboratory analysts to find answers to the complex analytical challenges they
face. The company’s global leadership and world-class service and support in the capillary electrophoresis
201
and liquid chromatography-mass spectrometry industry have made it a trusted partner to thousands of the
scientists and lab analysts worldwide who are focused on basic research, drug discovery and development, food
and environmental testing, forensics and clinical research.
With over 40 years of proven innovation, SCIEX excels by listening to and understanding the ever-evolving
needs of its customers to develop reliable, sensitive and intuitive solutions that continue to redefine what is
achievable in routine and complex analysis. For more information, please visit sciex.com.
SCIEX social: Twitter: @SCIEXnews, LinkedIn and Facebook.
For Research Use Only. Not for use in diagnostic procedures.
AB Sciex is operating as SCIEX.
© 2016 AB Sciex. The trademarks mentioned herein are the property of the AB Sciex Pte. Ltd. or their
respective owners. AB Sciex™ is being used under license. RUO-MKT-12-4288-A View source version on businesswire.com: http://www.businesswire.com/news/home/20160628005047/en/
NewsRx LLC
(2016-07-11), The University of Manchester and SCIEX Opened the £18m Stoller Biomarker Discovery Centre on June 14th, Journal of Engineering, 1268, ISSN: 1945-872X, BUTTER® ID: 012026916
Transgenomic, Inc.
Transgenomic Reports First Quarter 2016 Financial Results
By a News Reporter-Staff News Editor at Journal of Engineering – Transgenomic, Inc. (NASDAQ: TBIO) reported financial results for the first quarter ended March 31, 2016, and provided a business update. Business
UpdateDuring the first quarter of 2016 Transgenomic (TBIO) continued to work to realign its core activities
around the commercialization of ICE COLD-PCR™ (ICP), an innovative technology that enables the use of DNA
liquid biopsies for better, safer and less costly diagnosis and treatment of many diseases. Broader commercialization of ICP is expected to provide a foundation for expansion of the Company’s licensing and partnering
strategy in order to maximize the value of this broadly enabling technology.
As part of its strategic focus on broadly commercializing ICP, TBIO is exiting lower growth legacy businesses,
a process that is now largely complete. TBIO conducted a strategic review of the Patient Testing Business Unit
in the first quarter of 2016 resulting in a decision to suspend testing at its CLIA laboratory in New Haven,
Connecticut, and consolidate all remaining CLIA activities in the Company’s laboratory in Omaha, Nebraska.
Divestiture options for these legacy Patient Testing assets are being pursued. Suspension of these Patient
Testing activities and closure of the New Haven lab have significantly reduced expenses, resulting in savings
of over $1 million a month.
The drive to exit legacy businesses has had major effects on the Company’s operations and financial results. The Genetic Assays and Platforms and Patient Testing businesses have been classified as discontinued
operations. Information presented for current and prior quarter periods in the financial statements has been
modified to reflect them as discontinued operations.
Paul Kinnon, Transgenomic President and Chief Executive Officer, commented, “We are now nearing completion of our plan to exit our low growth, unprofitable legacy businesses, thereby releasing management to
focus solely on commercialization and adoption of our ICP technology. As stated in our conference call last
month, we are optimistic that the foundation being developed for ICP will begin to bear fruit over the remainder of 2016. The recent release of additional compelling concordance data is an essential element in providing
potential partners and customers with the validation they need to demonstrate that ICP can deliver on its
promise to enable and simplify the detection of genetic alterations, which is central to implementation of personalized and precision medicine. We believe that the ICP platform has the potential to make TBIO a leader in
the rapidly emerging liquid biopsy market, and we have now turned our full attention to further development
of our ICE COLD-PCR technology. Having alternatives to tissue-based biopsies is expected to fundamentally
change how we diagnose and treat cancer and other disorders. The terrific results from our concordance study
have re-affirmed our excitement about the near and long-term potential for this broadly-enabling technology,
and we look forward to continued progress in the months ahead.” First Quarter Financial Results from Continuing OperationsNet sales for the first quarter of 2016 were $0.2 million as compared with $0.7 million for the
same period in 2015. The $0.5 million decrease reflects phasing of contracts in the first quarter due to client
sample availability issues, a situation the Company expects to be transitory.
Gross profit was a negative $0.3 million, compared with gross profit of $0.3 million for the same period in
2015. The negative gross profit in the first quarter of 2016 is due to the lower revenues noted above coupled with
a substantial one-time milestone expense associated with product commercialization. A significant portion of
202
Cost of Goods Sold is fixed costs associated with the operation of Transgenomic’s laboratory. The Company
anticipates that gross profit percentages will increase as revenues from ICP-based products and services rise.
Operating expenses were $2.0 million during the first quarter of 2016 as compared to $2.3 million in the
first quarter of 2015. The $0.3 million decrease in operating expenses was due to lower professional fees in the
first quarter of 2016 as compared to the same period in 2015.
The net loss from continuing operations for the first quarter of 2016 was $2.1 million or $0.10 per share,
compared with a net loss of $2.3 million or $0.28 per share for the first quarter of 2015. Modified EBITDA,
which is a non-GAAP measure that Transgenomic views as an appropriate and sound measure of the Company’s
results, was a loss of $2.1 million for the first quarter of 2016, compared to a loss of $1.8 million for the same
period in 2015. A reconciliation of Net Loss to Modified EBITDA is presented below.
NewsRx LLC
(2016-06-06), Transgenomic Reports First Quarter 2016 Financial Results, Journal of Engineering, 2269,
ISSN: 1945-872X, BUTTER® ID: 011869409
VIDA Diagnostics, Inc.
VIDA and Olympus Expand Strategic Partnership
By a News Reporter-Staff News Editor at Health & Medicine Week – VIDA Diagnostics, Inc. (“VIDA”), the leader
in precision pulmonary imaging, announced an expanded collaboration with Olympus Respiratory America, a
leading developer of devices for the treatment of acute and chronic conditions of the lung and a subsidiary
of Olympus Corporation, at the 2016 American Thoracic Society Meeting. Under the agreement, VIDA and
Olympus will incorporate precision informatics into current and future diagnostic and therapeutic procedural
workflows to manage patients with pulmonary diseases.
“Olympus’ goals are to develop more comprehensive technologies and tools to localize, diagnose and treat
the small pulmonary nodule,” said Katsuyuki Saito, Executive Officer, Olympus Corporation. “By unifying
advanced procedural selection and planning techniques using VIDA, we aim to offer a high value, efficient
solution for the physician and patient.”
The collaboration covers North America and Europe and will include marketing and development programs
in interventional pulmonology, tightly integrating imaging informatics software and services to drive diagnostic
and therapeutic procedures.
“Precision medicine requires precision information and we are delighted to expand our partnership with
Olympus to advance interventional pulmonology,” said Susan A. Wood, Ph.D., President and CEO of VIDA. “The
partnership enables integrated and streamlined procedural workflows, essential for higher-value precision care
and builds on the successes of our Olympus partnership to date.”
NewsRx LLC
(2016-06-03), VIDA and Olympus Expand Strategic Partnership, Health & Medicine Week, 3316, ISSN:
1532-4605, BUTTER® ID: 011791792
203
Walgreens
Walgreens to Participate in U.S. Precision Medicine Initiative Cohort Program through The
Scripps Research Institute
By a News Reporter-Staff News Editor at NewsFile – Walgreens will participate in the U.S. Precision Medicine
Initiative (PMI) Cohort Program through an initial $20 million grant awarded to The Scripps Research Institute
(TSRI) by the National Institutes of Health (NIH). The company will help support patient enrollment and
participation in the program through its daily interactions with more than 8 million customers in stores, online
and through its mobile app. The award will total almost $120 million over five years and is part of President
Obama’s Precision Medicine Initiative, one of the most ambitious medical research programs in the history
of American medicine aimed at revolutionizing how researchers, providers and research participants work
together to develop individualized care.
“As a trusted health care resource to the millions of customers and patients we see every day, Walgreens
will help launch this important program that can lead to delivering the right treatments at the right time to
help Americans live longer, happier and healthier lives,” said Co-Chief Operating Officer of Walgreens Boots
Alliance Alex Gourlay.
With this funding award, Walgreens will offer customers enrollment in the PMI Cohort Program at its stores,
at Walgreens Healthcare Clinics, online through Walgreens.com and through the Walgreens mobile app. For
those who enroll, Walgreens also can schedule their initial appointment for the program.
“Walgreens has a long commitment to implementing innovative digital tools to improve the health and wellbeing of our customers we serve every day across the country,” said Brad Fluegel, Walgreens senior vice president, chief healthcare commercial market development officer. “Our participation in the Precision Medicine
Initiative Cohort Program will help shape the future of pharmacy by developing more precise medications,
based on a person’s genetic and other biological information and lifestyle, that can reduce side effects and drug
interactions while increasing their effectiveness.”
The PMI Cohort Program is a landmark longitudinal research effort that aims to engage 1 million or more
U.S. participants to improve the ability of preventing and treating disease based on individual differences in
lifestyle, environment and genetics. Participants will share their health history and status, genomic and other
biological information, and grant access to their clinical data from electronic health records. In addition, mobile
health devices and apps will provide lifestyle data and environmental exposures in real time. The knowledge
gained from the program is expected to extend successes of precision medicine in some cancers to many other
diseases and also increase an individual’s chances of remaining healthy throughout life. About Walgreens
Walgreens (www.walgreens.com), one of the nation’s largest drugstore chains, is included in the Retail Pharmacy USA Division of Walgreens Boots Alliance, Inc. (NASDAQ: WBA), the first global pharmacy-led, health
and wellbeing enterprise. More than 8 million customers interact with Walgreens each day in communities
across America, using the most convenient, multichannel access to consumer goods and services and trusted,
cost-effective pharmacy, health and wellness services and advice. Walgreens operates 8,173 drugstores with a
presence in all 50 states, the District of Columbia, Puerto Rico and the U.S. Virgin Islands. Walgreens digital
business includes Walgreens.com, drugstore.com, Beauty.com and VisionDirect.com. More than 400 Walgreens
stores offer Healthcare Clinic or other provider retail clinic services. View source version on businesswire.com:
NewsRx LLC
(2016-07-18), Walgreens to Participate in U.S. Precision Medicine Initiative Cohort Program through The
Scripps Research Institute, NewsFile, 391, ISSN: 0000-0000, BUTTER® ID: 012059055
204
WuXi NextCODE
WuXi NextCODE Brings the Global Standard Platform for Genomic Big Data to Microsoft
Azure
By a News Reporter-Staff News Editor at NewsFile – WuXi NextCODE, a global genomic information and precision medicine company, announced that its comprehensive genomics platform is now available on Microsoft
Azure. By bringing its platform to Azure, WuXi NextCODE will enable an even broader community of healthcare institutions and companies to take advantage of its powerful capabilities for applying genome sequence
data to deliver and develop better medicine. The system will be demonstrated at the Microsoft booths at the Develop, Innovate, Advance (DIA) conference in Philadelphia June 28-30 and the Festival of Genomics in Boston
June 28-29.
“Launching our genomics platform on Microsoft Azure marks another exciting step in our development as
the standard platform on which precision medicine will be built and delivered,” said Hannes Smarason, chief
operating officer, WuXi NextCODE. “Microsoft Corp. is a leader in delivering powerful cloud technology. We
are thrilled to work with Microsoft experts and customers worldwide to use the genome to benefit patients.”
Today, WuXi NextCODE is the only platform that makes it possible to use large-scale genomic data in full
resolution over an ordinary internet connection. That means that a pediatrician in the Midwest can send the
DNA of a rare disease patient to be sequenced and uploaded to the WuXi NextCODE system on the Azure cloud
platform; a clinical geneticist in Boston can use WuXi NextCODE’s clinical interface to identify the variant
causing the illness; and the doctor, the geneticist and the parents can then review the entire process stepby-step in real time. They can see the child’s genome sequence in the context of global reference data in full
resolution on their desktops a thousand miles apart, without ever having to send big data files back and forth
online or on disk.
The same capability enables teams of scientists to link and instantly query tens of thousands of genomes
from many institutions to find new links between genes and diseases. Doing this on Azure means every researcher can access and query all of the same data using WuXi NextCODE’s renowned discovery interface
and scalable computing power. That eliminates the need to replicate and store multiple copies, and allows IT
teams to focus on advanced applications with real scientific and medical impact, rather than managing basic
infrastructure.
“Applying genomics to medicine is a big data challenge, and WuXi NextCODE is meeting this challenge with
a scalable solution on Microsoft Azure,” said David Heckerman, distinguished scientist, Microsoft Genomics.
“We are pleased to bring the power and reach of the WuXi NextCODE platform to Microsoft Azure as an integral
part of our growing genomics ecosystem.”
NewsRx LLC
(2016-07-11), WuXi NextCODE Brings the Global Standard Platform for Genomic Big Data to Microsoft
Azure, NewsFile, 1034, ISSN: 0000-0000, BUTTER® ID: 012023472
205
Index
Acute Lymphoblastic Leukemia, 63, 132
Adalimumab, 64
Addiction Research, 83
Adenocarcinomas, 9
Adrenocortical Carcinoma, 97, 98
Age-Related Macular Degeneration, 80, 81
Agriculture, 94, 101, 172
Algorithms, 37, 44, 50, 54, 104, 172, 182, 189
Amendola Communications, 182, 183
American Heart Association, 36, 114
American Society of Human Genetics, 74
Amino Acids, 158
Amoy Diagnostics Co, 121
Amplion, 44
Angiography, 19
Antibodies, 18, 111, 112, 176
Antidepressants, 57, 58
Aortic Valve, 115
ArcherDX, 9, 10
Ariel Precision Medicine, LLC, 147
Association for Molecular Pathology, 128, 129
Asthma, 100, 117, 181, 193
Atopic Dermatitis, 193
Atrial Fibrillation, 40, 118
Autism, 174
Autoimmune Diseases and Disorders, 192
Brain Diseases, 75
Brain Injury, 156, 157
Brasov, 193
BRCA2, 13, 14, 127, 134, 136, 143, 169
Breast Cancer, 13, 20, 24, 27, 31, 35, 54, 55, 96, 121,
126, 134, 136, 146, 160, 175, 196, 197, 200
Brigham and Women’s Hospital, 104, 128, 129
British Columbia, 62
Cancer, 5, 8–35, 44–46, 49, 51–55, 57–60, 62, 63, 65,
66, 68–71, 73, 80, 84, 87, 96–98, 104–109, 111, 112,
117, 119–129, 131–139, 141–144, 146, 151, 153,
160, 161, 164, 167, 169, 170, 173, 175–178, 181,
184, 185, 187, 191, 196, 197, 201, 202
Cancer Detection, 8, 160
Cancer Diagnostics, 160
Cancer Gene Therapy, 45, 111
Cancer Prevention, 18, 119, 120, 142
Cancer Treatment Centers of America, 108, 109
Canon BioMedical, 50
Capillary Electrophoresis, 176, 195, 201
Carcinogenesis, 18
Carcinoma, 12, 53
Cardiology, 39–42, 47, 50, 114
Cardiovascular, 11, 29, 36–42, 51, 72, 114, 118, 130,
191, 198
Cardiovascular Diseases, 38
Bacterial Infections and Mycoses, 46, 191
Cardiovascular Diseases and Conditions, 37, 198
Biochemical, 17, 23, 24, 64, 65, 133, 171, 176, 185
Cardiovascular Research, 40, 41
Biochemistry, 18, 23, 76–78, 108, 126, 183
Case Western Reserve University, 162
Bioengineering, 6, 7
CeMM Research Center for Molecular Medicine of the
Austrian Academy of Sciences, 10, 131
Biogerontology Research Foundation, 2
Bioinformatics, 5, 10, 16, 31, 73, 79, 87, 105, 153, 161, Certara, 52, 53, 148
186, 189
Cetuximab, 69, 70
Biologics, 3, 52, 109, 110, 159
Chemicals, 18
Biometrics, 173
Chemistry, 6, 10, 50, 66, 76, 101, 161, 176, 183
BioNJ, 147
Chemotherapy, 9, 20, 50, 54, 56, 58, 69, 70, 105, 109,
Biotechnology, 1–6, 11, 45, 111, 139, 172
111, 112, 126, 127, 143
Biotechnology Companies, 3, 153, 201
Children’s Hospital of Philadelphia, 79
Biotechnology Company, 17, 46, 51, 63, 81, 126, 164
China, 9, 10, 23, 30, 54, 65, 66, 72, 75, 83–85, 105, 115,
Biotechnology Innovation Organization, 2
116, 121, 139, 159, 181, 185, 191
Blindness, 72
Chronic Myelogenous Leukemia, 124
Blood, 8, 9, 15, 16, 20, 26, 34, 42, 55, 68, 72, 73, 76, 82, City of Hope, 105, 106
91, 98, 99, 105, 111–113, 117, 123, 154, 171, 177, Clearbridge BioMedics, 105
185, 198
Clinical Oncology, 6, 8, 12, 14, 24, 33, 46, 54, 55, 57,
Blood Cells, 73, 112, 185
62, 96, 121, 126, 139, 170
Boston Children’s Hospital, 20, 111
Clinical Research, 9, 10, 16, 17, 35, 44, 47, 90, 91, 110,
Brain Cancer, 60
111, 119, 127, 165, 170, 195, 202
206
Clinical Science, 12, 13
Colon Cancer, 29, 137, 141, 142
Colorectal, 8, 34, 35, 96, 108, 121, 125, 141, 160, 170,
176, 177
Columbia University Medical Center, 47, 49, 107, 149
Combinatorial, 53
Coronary Artery Disease, 19, 39
Corvidia Therapeutics, 51
Critical Care Medicine, 86, 92
Crohn’s Disease, 64, 101
Cure Forward, 62, 146, 147, 151
Cystic Fibrosis, 88, 101
Data Visualization, 101
Definiens, 152
Delirium, 92, 93
Dementia, 92, 171, 198
Dementia and Mental Illness, 77
Depression, 47, 61, 84, 92, 148, 149, 156, 157, 174
Diabetes, 15, 40, 47, 48, 67, 68, 72, 89, 100, 106, 117,
153, 179, 197
Diet and Nutrition Disorders, 188
Digestive System Diseases and Conditions, 23
District of Columbia, 154, 204
DNA Repair, 13, 14, 143, 169
DNA Research, 84
Drug Development, 8, 9, 31, 40, 43, 44, 52, 60, 66, 139,
148, 151, 161, 166
Drugs, 3, 13–15, 20, 21, 24, 26, 33, 37, 57, 60, 62, 63,
65–67, 69–71, 75, 76, 82, 83, 86, 88, 92, 98, 100,
104, 106, 108, 109, 111, 118, 127, 144, 148, 158,
161, 169, 173, 174, 180, 184, 191, 196, 197, 200
Drugs and Therapies, 61, 67
Duke University, 53, 65, 82, 84, 85
East Brunswick, 176
ECOG-ACRIN Cancer Research Group, 21
Economics, 19, 39, 126, 150, 158
Eli Lilly and Company, 170
Encephalitis, 177
Endocrinology, 90, 184, 197
Engineering, 6, 7, 16, 36, 65, 73, 87, 91, 94, 99–101,
105, 112, 115, 137–139, 149, 151, 161–163, 165,
166, 171, 181, 182, 184, 195, 200–202
Enzymes and Coenzymes, 193
Epidemiology, 95, 108, 126, 149, 183
Epidermal Growth Factor Receptor, 55
Epilepsy, 75, 156
Erlotinib, 55, 56
Eye Diseases, 78
Eye Diseases and Conditions, 78, 80
Frontage Laboratories, 159
Gastroenterology, 23, 30, 64, 141, 158, 187
Gastrointestinal Diseases, 158
Genetic Counseling, 90, 127
Genetic Engineering, 29
Genetics, 3, 9, 10, 16, 18, 26, 30, 33, 50, 71, 72, 74,
79, 83, 85, 87, 90–92, 96, 99, 107, 112, 117, 125,
141–143, 163, 165, 170, 177, 180, 200, 204
Genomic Health, 15, 16, 54, 55, 126, 181
George Mason University, 27
Glomerular Disease, 56
Guardant Health, 160, 161
Gynecology, 11, 12, 127
Harvard Medical School, 32, 128, 129
Health and Medicine, 90, 157, 189, 199
Health Outcomes Sciences, 114, 115
Health Policy, 95
Health Technology, 91, 171
Healthcare, 2, 3, 17, 22, 25, 36, 39, 43, 49, 50, 52, 58,
69, 74, 79, 80, 91, 92, 95, 102, 106–108, 111–115,
117, 121, 124, 138, 140, 141, 148–150, 152, 153,
161–163, 165, 166, 170, 172, 173, 182, 183, 186,
189, 200, 201, 204, 205
Heart Disease, 19, 40, 82, 117
Heart Disorders and Diseases, 40
Hematology, 9, 16, 20, 37, 43, 49, 59, 66, 68, 124, 131
Hemorrhage, 156, 157
Hepatocellular Carcinoma, 23
Hepatocytes, 187
Hereditary Disease, 81
Hormones, 29, 98, 192
Hortonworks, 163
Hospital, 11, 19, 30, 34, 35, 54, 59, 60, 71, 72, 79, 90,
92, 104, 106, 107, 109, 111, 112, 114–117, 119, 123,
128, 129, 131, 133, 142, 148, 160, 165
Huntington’s Disease, 174
Hypertension, 47, 82, 198
Idiopathic Pulmonary Fibrosis, 86
Ignyta, 17, 46, 126, 164
Ignyta, Inc, 17, 46, 126, 164
Illumina, 17, 94, 100, 165
Immunoglobulins, 191
Immunology, 30, 73, 95, 152, 161, 176, 188, 193
Immunotherapy, 8, 12, 27, 49, 58, 59, 68, 71, 105, 109,
139, 188
Indiana University, 59, 92
Induced Pluripotent Stem Cells, 40, 47, 185
Infectious Diseases, 18, 73, 95, 96
Inflammatory Bowel Diseases, 101, 158
Fibroblasts, 187
Information Technology, 5, 41, 43, 100, 150, 166, 182,
Fluorouracil, 54
186
Forentis Partners, 1
Institute of Cancer Research, 13, 14, 142, 143
Foundation Medicine, 12
Intensive Care, 81, 92, 189
Fred Hutchinson Cancer Research Center, 24, 127, Internal Medicine, 19, 40, 41, 56
143
207
International Association for the Study of Lung Can- Neurodegenerative Diseases, 171
cer, 55, 128, 129
Neuroendocrine Tumors, 108
Interpreta, 165, 166
Neuroimmunology, 192
Intertrust Technologies Corporation, 161
Neurology, 60, 130, 165, 171, 174, 177, 192
Intestinal Diseases, 158
Olfactory Neuroblastoma, 69, 70
Investing, 1, 100, 149
OmniSeq, 62
Johns Hopkins Medicine, 95
Oncology, 6, 9, 10, 12, 13, 16, 17, 19, 20, 24–26, 28, 32,
43, 45, 46, 49, 51–54, 58–60, 62, 66, 68–71, 88, 94,
Ketones, 168
105, 106, 108, 109, 114, 119, 121, 122, 126–131,
Kidney, 30, 40, 72, 189
133–136, 138, 140, 141, 143, 144, 148, 151, 161,
Kinase, 9, 46, 55, 79, 144, 154, 178
164, 167, 169–171, 175, 176, 186, 187, 197
King’s College London, 125
Open-Angle Glaucoma, 78
Ophthalmology, 81
Leber Congenital Amaurosis, 80, 81
Ovarian Cancer, 51, 52, 127, 134–136, 142, 143
Legislation, 2, 3
Owlstone Medical Ltd, 137
Leukemias, 9, 124, 132
Life Science Research, 84, 89, 179, 181, 194, 195
Pain, 19, 108, 109, 120, 154, 156, 157, 170, 174
Liquid Biotech USA, 18
Pancreas, 29
Lung Cancer, 8, 16, 20, 29, 35, 46, 55, 56, 68, 96, 102, Pathobiology, 56, 154
114, 121, 128, 129, 137, 139, 144, 161
Pathology, 28, 29, 32, 55, 56, 61, 73, 77, 98, 104, 108,
Lung Diseases, 154
115, 116, 128–130, 134, 135, 152, 162, 171, 177
Lung Diseases and Conditions, 86, 88, 154
Patient Care, 34, 52, 53, 57, 63, 69, 80, 98, 102, 115,
120, 143, 148, 163, 166, 171, 182, 186, 192
Machine Learning, 104, 184
PatientsLikeMe, 99, 183, 184
Macrogen, 81, 82
Pediatric Cancers, 132
Marketing, 1, 77, 181–183, 203
Pediatrics, 59, 73, 81, 88, 100, 123, 132
Mayo Clinic, 10, 38, 57, 58, 112, 114, 163
Peptides, 130
Medical, 1–3, 6–10, 12, 13, 15–17, 19, 21, 22, 24, 25, Personalis, 25
28, 29, 32, 34, 35, 41, 46–48, 51–53, 57, 59, 60, 64, Personalized Medicine, 3, 7, 8, 25, 27, 30, 62, 67, 105,
74, 79–81, 91, 92, 95, 97, 99, 105–111, 113–117,
130, 158, 163, 173, 179, 180, 191, 195
120, 124, 126–131, 135–138, 141–143, 147, 149, Perthera, 138
151, 153, 154, 159, 160, 163, 166, 170–172, 174, Pharmaceutical Companies, 121, 139, 140, 148, 183
179–183, 186, 188–190, 192, 195, 197, 199, 200, Pharmaceuticals, 6, 7, 51, 101, 151
204, 205
Pharmacodynamics, 8, 64, 67
Medical Devices, 91, 153
Pharmacoeconomics, 158
Medicare, 102, 103, 165
Pharmacogenetics, 50, 67, 197
Meningioma, 88, 155
Pharmacogenomics, 5, 17, 65, 67, 86, 163, 165
Mental Health, 61, 92, 174
Pharmacology, 52, 62, 65, 67, 83, 100, 148
Merrimack Pharmaceuticals, 170
Pharmacotherapy, 57, 58, 61, 83
Metastatic Prostate Cancer, 127, 167, 169
PHEMI, 150, 184
Methadone, 168
Phosphoglucomutase, 193, 194
Molecular Imaging, 130, 133
Plasma, 33–35, 81
Molecular Medicine, 27, 76, 139
Precision for Medicine, 43
Molecular Psychiatry, 190
Predictive Analytics, 1, 58, 114
Molecular Research, 191
Prefrontal Cortex, 61
Movement Disorders, 174
Progressive Multifocal Leukoencephalopathy, 177
Multiple Myeloma, 22
Prostate Cancer, 13, 14, 122, 127, 131, 133, 139, 140,
Multiple Myeloma Research Foundation, 22
143, 167, 169, 197
Multiple Sclerosis, 177, 192
Prostate Cancer Foundation, 13, 127, 139
Muses Labs, 171, 172
Protein Kinase Inhibitors, 18
Proteins, 6, 10, 12, 27, 30, 58, 59, 72, 75, 78, 79, 130,
Nanoparticles, 65
132, 134–136, 179, 180, 194, 195, 201
NantHealth, 49, 50, 58, 59, 95, 172
Proteome, 134–136, 179, 180
Nephrology, 56, 189
Proteomics, 27, 58, 59, 79, 116, 135, 162, 195, 201
Nervous System Diseases and Conditions, 87, 154–156
Psoriatic Arthritis, 86
Neuroblastoma, 20, 21
Psychiatry, 57, 58, 61, 65, 83, 148, 149, 165, 190
Neuroblastomas, 20, 21
208
Public Health, 33, 40, 41, 71, 72, 77, 83, 91, 95, 96,
101, 105, 106, 109, 113–116, 119, 121, 131, 137–
148, 160, 183
139, 148, 152, 153, 160, 161, 163, 166, 171, 172,
Pulmonary Hypertension, 154
182, 183, 187, 192, 200–202, 205
Pulmonology, 203
Technology Companies, 43, 138
Terrapinn Inc, 95
Quality of Care, 41, 109, 150
Therapeutics, 7, 8, 21, 46, 49, 51, 57–59, 63, 65, 67, 78,
Quebec, 51
86, 88, 98, 152, 153, 193, 197
Quintiles, 140
Therapy, 2, 8, 11, 13, 15, 16, 18, 20, 25–27, 29, 44, 45,
47, 51–56, 58–62, 64–71, 75, 81, 82, 88, 92, 95, 108,
Radiotherapy, 69, 70
109, 111, 112, 125, 133, 138, 151, 163, 166, 170,
RaNA Therapeutics, 63
171, 175, 177, 185, 187, 190, 192, 197
Rare Diseases, 44
Thermo Fisher Scientific Inc, 115, 116
Reggio di Calabria, 188, 189
Thrombosis, 37
Regulatory Agencies, 52, 53, 148
Transcranial Magnetic Stimulation, 61
Research and Markets, 200
Transgenomic, 33–35, 202, 203
Rheumatoid Arthritis, 75, 76
Transplantation, 20, 56, 106, 111, 112, 115, 124, 158,
Rheumatology, 151, 152
189
Rhinitis, 193
Tumors, 8, 15, 17, 19, 20, 25, 26, 28, 29, 44, 46, 54–56,
Rxight, 69
59, 60, 71, 90, 98, 108, 109, 127, 130, 131, 134–136,
138, 141, 143, 160, 164, 169, 184
Sarah Cannon Research Institute, 8, 9
Type 2 Diabetes, 47, 67, 71, 72
Sarcoma, 49
Tyrosine Kinase Inhibitors, 124, 128, 129
Science, 3, 14, 18, 22, 35, 36, 39, 46, 47, 50, 58, 60,
62, 63, 74, 75, 77, 79, 81, 82, 84, 85, 87, 89, 91, 92, U.S. Food and Drug Administration, 3, 55, 57, 76, 95
97, 99–101, 107, 110, 111, 113, 115, 116, 119, 123, University of Illinois at Chicago, 117
124, 130, 132, 135, 137, 139, 141, 143, 144, 146, University of Leicester, 118, 119
168, 176, 178, 179, 181, 183, 184, 187, 191, 193– University of Manchester, 52, 79, 123, 124, 148, 201
195, 199, 201
University of Maryland Medical Center, 120
Science And Technology, 69, 100, 105, 163, 197, 198
University of Michigan, 23, 24, 32, 56, 71, 98, 122, 144,
SCIEX, 195, 196, 201, 202
145, 169
Scripps Research Institute, 73, 91, 99, 113, 196, 204
University of Southampton, 11, 131
Secondary Headache Disorders, 156, 157, 170
University of Southern California, 126
Sepsis, 46, 191
Septic Shock, 46
Vanderbilt University Medical Center, 73, 113, 180
Serous Carcinoma, 134–136
Ventricular Tachycardia, 42
Seven Bridges, 17, 31, 32
Veracyte, 102
Sickle Cell Anemia, 111, 112
Veterinary, 167
Skin Diseases and Conditions, 193
Veterinary Research, 167
Sleep Medicine, 169
Vibrent Health, 91, 92, 99, 113
Software, 10, 14, 15, 17, 50, 104, 115, 118, 119, 138, VIDA Diagnostics, 203
159, 162, 172, 181, 182, 184, 203
Vitamin D Deficiency, 87, 189
SolveBio, 97
Southwest Research Institute, 6
Walgreens, 99, 204
Spinal Muscular Atrophy, 63
Waters Corporation, 130
Squamous Cell Carcinoma, 144, 145
Wellness, 3, 74, 87, 153, 182, 204
Station X, 16, 17
West China Hospital of Sichuan University, 115, 116
Stem Cell Research, 40, 79, 174, 185, 187
Women’s Health, 126, 127, 196
Strata Oncology, 32
WuXi NextCODE, 205
Sudden Cardiac Death, 42
Xenograft, 6, 53
Sunitinib, 69, 70
Surgery, 29, 52, 69, 70, 84, 98, 102, 105, 109, 131, 192
SWOG, 96, 97
Tamoxifen, 54, 55
Technology, 10, 11, 15, 16, 22, 25, 31–36, 39, 40, 43,
47, 51, 52, 63, 66, 69, 76, 81, 87, 91, 92, 97, 99,
209

Read the Report - Colorado BioScience Association

Transcription

Similar documents

Developed for maximum response at speed, Proxes T1

I`m helping to end MS by participating in the 2016 Jayman BUILT MS

The Board of Directors of Qatar Fuel (WOQOD) is pleased to invite

5 `S` Training Program at ABV-IIITM, Gwalior on 21.07.2016

Mikrotech® Mikrotech

Porky Expo Flyer

The NeuLion Report

Sept 30-Oct 2, 2016 Nov 4-6, 2016 @Camp Tadmor

4 star ALOE HOTEL, PAPHOS

the Guidelines for the registration on