annual report 2014 - Diabetes Research Institute

annual report 2014 >
MAKING
PROGRESS
POSSIBLE
Thank you to every individual, family,
foundation, and business that has given
generously over the last year and
through the years.
Highlights of the Year
2
National Board of Directors
11
Executive Officers' Message
4
Regional Boards of Directors
12
Financial Summary
6
DRI Foundation Staff
13
To our Donors
8
Heritage Society
10
Highlights
of the
Year
Throughout the year, Diabetes Research Institute scientists continued
to build upon critical research programs while launching new scientific
initiatives necessary for developing the DRI BioHub, a bioengineered
mini organ that mimics the native pancreas to restore natural insulin
production. It was an exciting year in which we witnessed progress
across the three main research challenges of the Site, Sustainability
and Supply, with some projects advancing to or nearing the clinical trial
phase of testing. Below is a summary of the year’s research highlights
that were made possible by your generous support.
Dr. Alice Tomei, assistant professor of surgery and cell transplantation, together with
collaborators at the École Polytechnique Fédérale de Lausanne (EPFL) in Switzerland,
demonstrated that their unique conformal coating process allows efficient encapsulation
of islets without compromising viability and function of the cells. The team's novel method
encases the islets within complete, uniform, and thin capsules of similar density, and
has been designed to specifically address what are considered to be the limitations of
traditional cell encapsulation strategies. The results of their study earned the cover
position in the prestigious journal Proceedings of the National Academy of Sciences.
Dr. Luca Inverardi and his team have continued their work with Myeloid-Derived Suppressor
Cells (MDSCs), a special population of immunomodulatory cells that help tumors escape
destruction by the immune system. MDSCs prevent tumor rejection by recruiting Regulatory
T cells to surround the cancer cells. The researchers are investigating the potential of MDSCs
to protect insulin-producing cells from autoimmune destruction using a similar mechanism.
MDSCs are usually harvested from the bone marrow, but this past year the team was the
first to discover and characterize a novel subset of MDSCs that they have named fibrocyteMyeloid-Derived Suppressor Cells (f-MDSC). The cells were isolated from the umbilical cord
blood of healthy newborn babies and have a powerful immunosuppressive effect. These
particular cells are also easy to grow, expand, and bank. The team’s discovery and results of
their studies were published in the European Journal of Immunology and Genomic Data.
Their efforts are now focused on developing ideal conditions for expanding and preserving
f-MDSC for their possible clinical use in achieving tolerance to transplanted insulinproducing cells in those with T1D.
THE SITE
The Food and Drug Administration (FDA) approved
the DRI’s submission to initiate a Phase I/II clinical
trial that will test islets transplanted in a new site
in the body called the omentum, an apron-like lining
inside the abdomen. The omentum closely replicates
the physiological drainage of insulin from the
pancreas and has many other beneficial properties.
In this trial, human donor islet cells will be
transplanted within a “biodegradable scaffold,” a
DRI BioHub platform. The biodegradable scaffold
uses a patient’s own plasma, the liquid part of
the blood, together with thrombin, a commonlyused, clinical-grade enzyme. Several patients
have completed the islet transplantation
screening process and have been selected as
candidates for the transplant.
Human islet cells embedded in the
biodegradable scaffold. The magnification
of the mesh shows the fibrin fibers that
hold the islets in place.
Dr. Peter Buchwald, director of drug discovery, and his team are targeting a recently
identified signaling pathway that leads to autoimmune destruction of insulin-producing
cells. They have had promising results in experimental models demonstrating that
new-onset diabetes can be reversed by blocking this pathway with a protein known
as Smad 7. There is also scientific evidence supporting his theory that the use of
Smad 7 not only controls the autoimmune destruction of the islet cells, but can also
lead to islet regeneration. Dr. Buchwald and his team are investigating the possible
beta cell-enhancing effects of this treatment with the goal of quickly translating
this research to clinical therapies.
In its Phase I/II clinical trial, the DRI
is testing the omentum as a new
transplant site. The omentum is rich
with blood vessels, is easily accessed
surgically, and has the same insulin
drainage characteristics of the
pancreas, among other benefits.
SUSTAINABILITY
Drs. Alberto Pugliese and Thomas Malek have been collaborating with Paris-based
Dr. David Klatzmann, who recently conducted clinical trials using low-dose IL-2 (Interleukin-2)
in patients with type 1 diabetes (T1D). The study compared three low doses of IL-2 to
determine whether these would be safe and result in increased Regulatory T cells (Tregs),
which regulate the immune system and suppress autoimmunity. Results of these
collaborative studies were published this year in several peer-reviewed journals, including
Diabetes and the Journal of Autoimmunity. New trials are now enrolling patients with
recent onset type 1 diabetes (within 3 months from diagnosis) to determine whether
low-dose IL-2 can preserve or improve the ability of the pancreas to produce insulin.
Dr. David Klatzmann from the
Université Pierre et Marie Curie
in Paris, France, (second from left)
with the DRI’s Drs. Alberto Pugliese,
Jay Skyler, and Thomas Malek. The
researchers are collaborating to
conduct clinical studies using lowdose IL-2 to boost Treg function,
reverse autoimmunity, and
restore insulin production.
In this three-dimensional model of human Smad 7, each colored region is believed to interact with
a critical receptor (called TGF-ß) in this important pathway under study at the DRI.
SUPPLY
The exocrine, or non-insulin-producing, cells of the pancreas have been shown to give
rise to insulin-producing endocrine cells. However, previous attempts to achieve this
have thus far relied on the use of genetic manipulation, which remains a translational
hurdle for diabetes therapies. Drs. Juan Dominguez-Bendala and Ricardo Pastori and
their teams have been able to convert adult human exocrine tissue into insulin-producing
cells – and have done so using a single molecule that is already in clinical use for other
conditions. The DRI team is the very first group in the world to achieve this result using
human cells with a compound that is already FDA-approved. The non-genetic conversion
of human pancreatic exocrine to endocrine cells is a novel strategy and represents a safer
and simpler alternative to genetic reprogramming, while opening the door to the design
of new therapies.
The DRI team is very encouraged by these results and is now planning to conduct a
subsequent clinical trial in Miami that will begin to expand the window to longer time
frames post-diagnosis. They believe that patients may benefit from this type of therapy as
long as they maintain a certain level of stimulated insulin production. Data has shown that
such levels may be present in patients for years after type 1 diabetes develops. The trial being
planned will involve patients with residual insulin secretion after one year. They will also be
studying whether this therapy can be applicable to patients who receive a pancreas or islet
cell transplant as a means of halting the autoimmune attack that caused the onset of T1D.
Recent findings in mouse models also suggest that IL-2 may promote some level of beta cell
regeneration, in addition to improving immune regulation.
[diabetes research institute foundation] 2
3 [2014 annual report]
"There is no better clinical translational
group working on type 1 diabetes in
the world."
Executive
Officers’
Message
At the DRI Foundation, where the majority of volunteers and
professionals have a loved one with diabetes, our resolve
could not be stronger. We want a cure, period.
At the DRI, our team of researchers – many of whom are
also touched by this disease – are in lockstep with us.
They passionately share the same mission.
Our passion and commitment drive us, but alone they
will not defeat such a complex disease. It takes expertise,
experience, and vision to achieve something so challenging.
Thankfully, we have these, too, a fact that is evident to our
supporters, as well as to the DRI’s esteemed colleagues
throughout the scientific community.
On that point, several months ago we attended the two-day
meeting of the DRI’s Scientific Advisory Board (SAB), an
external council of distinguished investigators who meet
every three years to review, and make recommendations on,
the Institute’s research program. Their report is then presented
to the Dean of the University of Miami Miller School of
Medicine, of which the DRI is a part. The SAB’s members
offered a glowing review of the DRI’s work, reporting that,
“The committee was unanimous in its feeling that major
strides have been made in both basic and translational
research programs at the DRI…There is no better clinical
translational group working on type 1 diabetes in the world.”
By the very nature of our work at the Diabetes Research Institute Foundation,
we meet countless individuals and families who are personally affected by
diabetes. Some have been living with this disease for decades, while others are
relatively new to it. Throughout the course of their involvement with us, they
all come to realize the very same thing: the DRI and Foundation are, together,
quite a special place.
We are special for many reasons, chief among them being our unparalleled
commitment to see this job to the end by discovering a biological cure for
diabetes. The drive to fulfill this mission is palpable throughout our entire
organization. It underscores everything that we do.
[diabetes research institute foundation] 4
In other words, we rely on you and all of our generous
donors to help us meet this ongoing need. Highlights of
the progress that you helped our researchers achieve are
presented in this report.
This past year, many have made a significant investment in our
research program. Thousands have led and/or participated in
the various events held in our regions and other communities
across the country. Others have generously donated whatever
they could to help move the science along. We are grateful for
each and every gift, regardless of the amount, because we will
not get there any other way.
We are all a part of this special place. Our mission
is to find a cure, and we need each and every one of
you to join us. On behalf of all of us at the DRI and
Foundation and the millions counting on us to cross
the finish line, thank you for your continued support,
trust, and friendship.
Sincerely,
Receiving this impressive validation from such a distinguished
panel of experts reinforces our belief that we have invested
our time and resources in the right place. Yet for all of the
accolades, we know there is still much work to be done,
because tomorrow is not soon enough to cure this disease.
That is why we would be remiss if we did not persistently ask
ourselves: how do you take something that is clearly special
and make it better? That is precisely what we are charged with
doing, in the interest of our loved ones, each of you, and the
millions of people living with diabetes.
One answer to that question is to continuously employ the
highest standards of financial stewardship and accountability.
Over the years, we have gone to great lengths to maintain
expenses at acceptable levels, to meet the rigorous guidelines
established by various non-profit oversight groups, and to
provide the necessary transparency about our operations.
Another answer is to ensure that research progress
continues, allowing us to keep moving toward our ultimate
goal. Much of that depends on our ability to fund the DRI’s
research initiatives.
5
[2014 annual report]
Harold G. Doran, Jr.
Joshua W. Rednik
Chairman
President and CEO
Financial
Summary
Diabetes Research Institute Foundation
Statement of Activities for the Year
ended June 30, 2014.
Through the support of private
philanthropy, the Diabetes Research
Institute Foundation has funded
six chairs totaling almost $14 million.
Support and Revenue
The J. Enloe and Eugenia J. Dodson
Chair in Diabetes Research
Contributions
Reimbursement Contracts
Special Events, Net of Expenses
Investment Income
Total Support and Revenue
5,300,455
139,017
3,757,157
2,055,447
$11,252,076
Expenses and Fund Balances
Program Services
Research (Provided to the Diabetes
Research Institute)
Community Education
6,792,936
930,994
Total Program Services
$7,723,930
Support Services
Administration and General
Fundraising
Total Support Services
Change in Net Assets
Net Assets, Beginning of Year
Net Assets, End of Year
Research Funding is Critical
955,229
1,409,158
$2,364,387
1,163,759
26,307,331
$27,471,090
Fundraising Percentage
The Diabetes Research Institute has become the world leader it is today as a
result of the substantial funding provided by the Diabetes Research Institute
Foundation (DRIF). This funding stream is at the heart of the DRI's ability to
make significant strides toward a cure. Supported by your donations, the
DRIF ensures that our scientists can jump-start new ideas while continuing
innovative, cure-focused research projects. Our mission – to provide the DRI
with the funding necessary to cure diabetes now – is testament to the belief
that tomorrow is not soon enough to cure this disease.
[diabetes research institute foundation] 6
Fundraising Expense as a
Percentage of Support and Revenue
7
[2014 annual report]
12.5%
Stacy Joy Goodman Chair in
Diabetes Research
Mary Lou Held Chair for Diabetes Research
Martin Kleiman Endowed Investigatorship
Daniel H. Mintz Visiting Professorship
Ricordi Family Chair in Transplant
Immunobiology.
To our
donors
“We need to continue
to bring this cause and our
mission to find a cure to the
forefront for the millions who
suffer with diabetes, including
my son.”
– Doug Donaldson (left)
[
To Our Generous Donors with Deepest Gratitude...
The Diabetes Research Institute and Foundation wish to gratefully
acknowledge all of our donors and volunteers, who are enabling us
to make great strides toward a biological cure for diabetes.
Thank you to every individual, family, foundation, and business, many of whom are pictured
within this report, that have given generously over the last year and throughout the years.
We would not have been able to come this far without you.
[diabetes research institute foundation] 8
>
HERITAGE
SOCIETY
NATIONAL BOARD
OF DIRECTORS
The Heritage Society of the Diabetes Research Institute Foundation recognizes
individuals who have generously made provisions in their estate plans,
through life insurance, charitable remainder trusts and gift annuities, or other
deferred giving vehicles to ensure that critical funding for the Diabetes Research
Institute continues into the future.
Over the years, planned giving programs have enabled many donors to make
substantial gifts to the DRI in ways that have complemented their individual
financial objectives. Heritage Society members have chosen to create their
own personal legacies and perpetuate their philanthropic goals for all those
affected by diabetes.
Chairman
Harold G. Doran, Jr.
President and CEO
Joshua W. Rednik
Immediate Past Chairman
Thomas D. Stern
Directors
Diane Beber
Marlene Berg
Ronald Maurice Darling, Jr.
John C. Doscas
Piero Gandini
Marc S. Goldfarb
Esther E. Goodman
Marc S. Goodman
Arthur Hertz
Glenn Kleiman
Eleanor Kosow
Sandra Levy
Vice Chairmen
William J. Rand, M.D.
Charles Rizzo
Treasurer
William J. Fishlinger
Secretary
Bonnie Inserra
We are exceptionally grateful to all of our Heritage Society donors, who
demonstrate the passion and vision to advance a cure beyond their lifetime.
[diabetes research institute foundation] 10
11
[2014 annual report]
Sean McGarvey
Shelia F. Natbony, D.O.
Allan L. Pashcow
Ramon Poo
Ricardo Salmon
David Sherr
Bruce A. Siegel
Kathy Simkins
Jill Viner
Bruce Waller
REGIONAL BOARDS
OF DIRECTORS
Florida Region
Northeast Region
Chairman
William J. Rand, M.D.*
Directors
Sari Addicott
Bernard Beber, M.D.
Diane Beber*
Crystal Blaylock Sanchez
Sabrina R. Ferris
Bruce Fishbein
Joel S. Friedman
Rene W. Guim
Javier Holtz
Norman Kenyon, M.D.
*Also member of National Board
of Directors
DRI FOUNDATION
STAFF
Executive Committee
William J. Fishlinger*
Marc S. Goodman*
Barbara Hatz
Bonnie Inserra*
Mary Revie
President and Chief Executive Officer
Executive Assistant
Deborah L. Chodrow
Laurie Cummings
Chief Operating Officer
Jeffrey Young
Chief Financial Officer
Meryl Lieberman
Allan L. Pashcow*
Charles Rizzo*
Barbara Tavrow
Senior Vice President
Tom Karlya
Vice President
Directors
Greg Besner
John Carrion
Diane Cohen
Delia DeRiggi-Whitton
Peter L. DiCapua
Kim Dickstein
Douglas R. Donaldson
Iris Feldman
Joan Fishlinger
Lindsey Inserra-Hughes
John Luebs
Louise Pashcow
Hon. C. Raymond Radigan
Marie Rizzo
Ricardo Salmon*
Samantha Shanken Baker
Northeast Region
Anthony E. Childs
Co-chairs
Marc S. Goldfarb*
Bruce A. Siegel*
Vito La Forgia
Sandra Levy*
Ramon Poo*
Cristina Poo
Deborah Rand
Michelle Robinson
Rosa Schechter
James Sensale
Jacci Seskin
Don Strock
Richard P. Tonkinson
Stephen Wagman
Rita Weinstein
Joshua W. Rednik
Thomas P. Silver
Bruce Waller*
Roberta Waller
Wendy Waller
Jill Shapiro Miller
Vice President of Gift Planning
Lori Weintraub, APR
Vice President of Marketing
and Communications
Lauren Schreier
Director of Marketing
and Communications
Aurora Nunez
Administrative Assistant
Oneida Osuna
Accounting Assistant
Mylinda Auguste
Data Entry Clerk
Marisol McKay
Date Entry Clerk
Eddy Garcia
Courier
Florida Region
Amy Epstein
Director of Special Events,
Manhattan Office
Lily Scarlett
Director of Special Events,
Jericho Office
Jill Salter
Development Manager
Melinda Megale
Special Events Coordinator
Tricia Pellizzi
Special Events Coordinator
Gloria Keyloun
Administrative Assistant
Sheryl Sulkin
Director of Special Events
Barbara Singer
Director of Special Projects
Nicole Otto
Associate Director of Special Events
Karen Paraboo
Administration and
Database Coordinator
Dena Kawecki
Joelle Parra
Sarah Mehan
Communications and
Social Media Coordinator
Special Events Coordinator
Melissa Peña
Development Coordinator
[diabetes research institute foundation] 12
Communications Assistant
Regional Director
13 [2014 annual report]
Special Events Manager
The organization of choice
for those who are serious,
passionate, and committed
to curing diabetes.
National Office
Florida Region
Northeast Region
200 South Park Road
410 Jericho Turnpike
Suite 100
Suite 201
Hollywood, FL 33021
Jericho, NY 11753
Telephone 954.964.4040
Telephone 516.822.1700
Toll-free 1.800.321.3437
Fax 516.822.3570
Fax 954.964.7036
Jericho Office
Manhattan Office
381 Park Avenue South
Suite 1118
New York, NY 10016
Telephone 212.888.2217
Fax 212.888.2219
DiabetesResearch.org