The Path To A Resilient Character Fall 2014

Fall 2014
The Path To A Resilient Character
By Elyse Elconin-Goldberg
“Resilience is the capacity to respond to
pressures and tragedies quickly, adaptively
and effectively,” shared Isabel Stenzel
Byrnes, MSW, MPH, during her inspirational
presentation, “A Resilient Character:
Adapting Well to Changes In Our CF,” at
the 27th National Cystic Fibrosis Family
Education Conference. Isabel, a 41-yearold social worker with cystic fibrosis (CF)
who had a double lung transplant 10 years
ago, emphasized the important role that
resilience plays in adapting to changes that
develop when living with CF, noting,
“Change is inevitable, but how one reacts
to that change has a significant impact
on the emotional well being of every
individual.” When living with CF, shared
Isabel, we must ask ourselves how we
can react to change in a healthier and
more resilient way. “How can resilience be
fostered within ourselves and in families
coping with CF?”
Thanks to neuroplasticity, our brains are
capable of re-wiring how we respond to
stress. “Opening to possibility,” a favorite saying of
Isabel’s, suggests having
a
a more positive attitude,
versus being stuck in the
negative. When we are
faced with adversity,
as those living with CF
frequently are, Isabel
suggested that we ask
ourselves if our responses
to stressors are working
for or against us. Isabel
quoted Linda Graham,
MFT, who writes about
the “5 C’s” of coping with
change: “Calm (keep calm
and carry on); Clarity
(having perspective);
Isabel Stenzel Byrnes, MSW, MPH
Connection (finding a
network); Competence (I did, so I can!);
vulnerability, hope (optimism), and social
and Courage (do one new thing a day).”
support (being understood). She cited a
2014 study conducted with teens with CF,
Isabel stated that in CF, there are three
Continued on page 4
things required for resilience: emotional
CFRI ~ Research for Living ~ Partners for Life By Beate Illek, PhD
For almost 40 years, CFRI’s investment
in new ideas has enabled clinicians and
researchers at Children’s Hospital Oakland
Research Institute (CHORI) to gain new
insights for the treatment of CF. I first
learned of CFRI as a visiting scholar from
Germany in Dr. Terry Machen’s laboratory
at UC Berkeley, where I developed my
interest in CF research. CFRI’s support of
a vibrant CF research community had an
enormous impact on my education and
training as a post-doctoral researcher. CFRI
provided a local network that facilitated the
formation of successful collaborations, the
establishment of a CF research laboratory
at CHORI, and the achievement of basic
Beate Illek, PhD
research findings throughout my entire
career. CFRI’s support of research – both
directly and indirectly – has been vital to
our scientific success and has resulted in
many peer-reviewed publications involving
electrophysiological experiments that were
carried out in my laboratory.
These contributions include key discoveries
that pioneered the regulation of the CFTR
chloride channel in airway epithelial cells
and its conductivity to bicarbonate and other
anions. The early discovery of genistein as
an activator of mutant G551D CFTR in CF
patients was made at CHORI, and paved
the way for a new drug treatment of the
Continued on page 18
CFRI Community
Fall 2014
Editorial Chief
Sue Landgraf
Editors & Writers
Bridget Barnes
Siri Vaeth Dunn, MSW
Contributing Writers
Darlene Batchelder
Linda Burks, EdD
Mary Convento
Meg Dvorak, LCSW
Elyse Elconin-Goldberg
Eric Frisbee
Bill Hult
Beate Illek, PhD
Sue Landgraf
Anna Modlin, MA
Layout and Design
Marina Michaelian Ward
Photography
Craig Burleigh
Board of Directors
Bill Hult, President
Jessica Martens, Vice President
Mike Roanhaus, Secretary
Oscar Flamenco, CPA, Treasurer
Francine Bion
Elyse Elconin-Goldberg
Ann Jones
Doug Modlin
Kristin Shelton, RRT
Executive Director
Sue Landgraf
Director of Programs
and Operations
David Soohoo
Program and Outreach
Coordinator
Notes from the
Executive Director
Dear Friends,
As I write to you, I am sitting in my office, reflecting on the
meaningful connections made with our CF community last
summer. The busy days of our powerful “Zen” Teen & Adult
Retreat and 27th National Cystic Fibrosis Family Education
Conference, “The Changing Faces of Cystic Fibrosis: Inspiring
Hope,” left us with the joy of camaraderie and a wealth of
information from our excellent presenters.
Looking at the brilliant prayer flags encircling my office, handmade by individuals in our community
who loved and lost someone with CF, I am reminded of the urgent need to expand our programs.
To that end, I am honored to announce the extraordinarily generous gift of $105,000 to CFRI’s
2015 Cystic Fibrosis Research Challenge Circle by Suzanne Freiley, in memory of her niece, Jessica
Fredrick, who lost her battle with CF this year at the age of 21. Ms. Freiley hopes that her gift,
which was made possible by a one-time double matching gift program from Lam Research, and
constitutes the largest single individual donation in CFRI’s history, will inspire others to give
generously to fund CF research and support. We are honored that Ms. Fredrick’s family would
pay tribute to her by helping to seek a cure for others. More details will be presented in the next
edition of CFRI Community.
CFRI is in this together with you – as we have been for nearly 40 years. We are experiencing an
exciting and hopeful time, as those with the double Delta F508 mutation are on the brink of
receiving medications that address the basic underlying cause of the disease. Our new tagline,
“Research for Living ~ Partners for Life,” reminds us that CFRI stands for hope and longevity;
daily, and into the future.
S CF Chef S
Warmly,
Mary Convento
Resource Development
Associate
Siri Vaeth Dunn, MSW
Resource Development
Assistant
Sue Landgraf | Executive Director
and mother of an adult daughter with CF
Scott Wakefield
Partnership with AbbVie a recipe for success!
Thanks to all who contributed recipes.
We will share the final results from this exciting
project in the next CFRI Community.
News from the Board
CFRI Community is published
and distributed to friends of
CFRI for free.
Send address corrections and
other correspondence to CFRI:
1731 Embarcadero Road, Suite 210
Palo Alto, CA 94303
Phone: Toll Free 1.855.cfri.now
(1.855.237.4669)
Fax: 650.404.9981
Email: cfri@cfri.org
Website: www.cfri.org
Dear CFRI Community,
The CFRI Board of Directors is pleased to share that our CF
Research Challenge has been met! Thanks to our generous
CF Research Challenge Circle donors who gifted $75,000
to match designated donations dollar for dollar, we raised
$150,000 to fund three additional CF research projects for
2014/2015! Our many thanks to the CF Research Challenge
Circle co-chairs, Paul Quinton, a preeminent CF researcher
living with CF, and Jim and Barbara Curry, longtime friends
of CFRI and grandparents to Cameron, who has CF. Our
With our permission, you may
reproduce original information
from this newsletter with credits
to CFRI Community and the author.
Bill Hult, President
2
Sue Landgraf, Executive Director
These programs strengthened our commitment to increase
funding for CF researchers to keep scientists committed to the search for a cure, while we expand
the breadth of our educational programs. We must reach more people touched by CF, including
families of the newly diagnosed and young children, non-English speaking families, and individuals
in need of psychosocial support. We are initiating new programs, while investigating ways to
effectively bring vital information about this challenging disease to those who need it the most.
Continued on page 3
cfri | Fall 2014
New CF Drugs Advance Hope and Quality of Life
for Patients and Their Families
By Bridget Barnes
“The pipeline holds more promise than
ever,” declared Dr. Ahmet Uluer, referring
to the progress of new therapies for the
treatment of cystic fibrosis (CF). In his presentation at this summer’s Cystic Fibrosis Family
Education Conference, “Cystic Fibrosis Drug
Pipeline: Past, Present and Future,” Dr.
Uluer, Director of the Adult Cystic Fibrosis
Program at the Boston Children’s and
Brigham and Women’s Hospital CF Center,
and Assistant Professor of Medicine at
Harvard University, covered many of the
existing and potential treatments that
modulate the dysfunctional CFTR protein
responsible for CF, restore airway surface
liquid, alter mucus, target inflammation,
treat infection, and augment nutrition.
Advances in the understanding
of the disease and the development of these new therapies
have led to improved quality of
life and life expectancy for those
living with CF. “The combined
efforts of organizations such as
CFRI, the Cystic Fibrosis Foundation, and many individuals
have contributed to this growing
knowledge base and are helping
to develop the next generation
of CF therapies,” Dr. Uluer
elaborated.
New therapies for this multiorgan disease that affects the
lungs, colon, sweat glands,
pancreas, bone, and efferent
ducts must go through multiphase drug trials to assess both
safety and efficacy before being
approved by the US Food and
Drug Administration (FDA), a
process that takes 6 to 18 years,
explained Dr. Uluer.
Dr. Ahmet Uluer sharing updates on the CF Drug Pipeline.
News from the Board
Continued from page 2
deepest gratitude to the CF Research Circle
donors listed on page 19.
CFRI is certainly living our tagline, Research
for Living ~ Partners for Life, through a
vigorous focus on funding research and
working with the CF medical community
to ensure access to the most current CF
medical/healthcare news and protocols.
To that end, we are honored to announce
the formation of our new Medical Advisory
Board, comprised of some of the most
cfri | Fall 2014
While reviewing the “cycle of
inflammation and infection”
esteemed members of our medical and
research community. Please go to page 6
for a list of the Advisory Board’s prestigious
members. We are honored by their
participation and support.
which causes CF lung disease, Dr. Uluer
highlighted several available drugs that are
significantly improving the quality of life
for those with CF. They include aquaDEKs®
and improved enzymes for good nutrition,
inhaled TOBI®, azithromycin, Cayston®,
and TIP (tobramycin inhaled powder) for
fighting infection, ibuprofen for reducing
inflammation, Pulmozyme® for liquefying
mucus, hypertonic saline to restore airway
surface liquid, and Kalydeco® for CFTR
modulation. In addition, a drug application
for Liprotamase, a pancreatic enzyme that
improves fat absorption, is under FDA
review, while numerous promising trials
for other drugs are underway.
For medications that target bacteria
associated with worsening lung disease,
a 24-week extension study of inhaled
Levofloxacin in CF patients with chronic
Pseudomonas aeruginosa showed
prolonged improvement in lung function
and quality of life. In addition, interim
analysis of a long-term study of Arikace
(inhaled Amikacin) in patients with
Pseudomonas also appears to show longterm improvement in FEV1. “As with
Pseudomonas, we need studies on how
to eradicate MRSA,” Dr. Uluer stated.
AeroVanc®, an inhaled dry-powder
addressing persistent Methicillin-resistant
Staphylococcus aureus (MRSA) is currently
in Phase 2 clinical trials and shows a reduction in MRSA colonies and improved FEV1.
Final results from this study will be
available in 2015.
Continued on page 4
CF community at Smart Patients is growing
and includes parents and young adults
affected by CF. The CFRI Board invites
you to join at www.smartpatients.com/
cfri and become part of an even larger
CF community.
We are your partners for life, and want to
provide you with resources that will help
on this journey with CF. CFRI now has an
Peace and good health,
online community for CF patients and caregivers! Smart Patients is where patients and
caregivers affected by complex illnesses
learn from each other about treatments,
challenges, and shared experiences. The Bill Hult | Board President
3
New CF Drugs Advance Hope
levels. Incredible!” Vertex is planning
to submit an application to the FDA for
review, with possible approval in 2015. Continued from page 3
Anti-inflammatories Alpha 1 Anti-Trypsin
and KB001-A are both in Phase 2 trials and
appear to “clean up the cascade of infection
and mucus build up caused by the secretion
of neutrophils,” Dr. Uluer reported.
Bronchitol (inhaled Mannitol) helps clear
mucus in the lungs by restoring surface
liquid in the airways. While approved in
Europe and other countries, it awaits
approval in the U.S.
CFTR modulators are potentially the most
effective, as they target the underlying
causes of cystic fibrosis rather than
the symptoms. One of these, Ataluren,
addresses “nonsense” mutations, and
as of August 2014, CF patients are being
recruited for a redesigned Phase 3 trial.
Another potential treatment, developed
by Vertex Pharmaceuticals, is Kalydeco®
combined with Lumacaftor (VX-809) for
people with two copies of the Delta F508
A Resilient Character
Continued from front cover
in which their perceived stress was low
and correlated with higher resilience, in
contrast to a non-CF teenage population.
The resiliency of the teens with CF was
attributed to their accelerated maturity,
taking of responsibility, acceptance of
prognosis, regaining of control, redefining
normality, social support, and problemsolving skills.
Reflecting on her own experiences growing up, Isabel shared how her parents
fostered resilience in her identical twin
sister, Ana, and herself. With age came
changes in the level of responsibility their
parents expected of them. As Isabel and
Ana transitioned from childhood to young
adulthood, responsibilities such as learning
to wash the dishes were accompanied by
taking over cleaning and disinfecting their
nebulizers. Learning to drive became an
opportunity to drive themselves to medical
appointments. Isabel shared study results
that identified key factors influencing
resilience in children, including a secure
attachment to their parent/caregiver, their
children’s internal make-up, self-calming
abilities, positive emotions, a positive family
environment, an external support system,
and a sense of control and mastery.
4
For people with one copy of the Delta
F508 mutation, Dr. Uluer imparted recent
news that the Kalydeco®/Lumacaftor
combination did not demonstrate a
significant improvement in lung function.
Dr. Uluer went on to say that Phase 2 data
of Kalydeco® in combination with VX-661
“looks promising for homozygotes with a
Delta F508 mutation and might be effective
for Delta F508 heterozygotes using higher
doses, but still has a way to go.”
Dr. Uluer and Paul Quinton, PhD
mutation. Phase 3 clinical trials for this
combination were recently completed, and
showed significant improvement in lung
function and other key measures of health
and, as noted by Dr. Uluer, “there was a
clinically significant drop in sweat chloride
In addition to the promise of new discoveries, Dr. Uluer reiterated, “We must continue
to develop therapies and strategies to deliver
current treatments more efficaciously.”
Concluding, he shared important advice
from his 70 and 80-year-old patients on keys
to staying healthy: “Good luck, exercise,
and fill your life with happiness.”
Both Ana and Isabel were hospitalized
frequently throughout their lives. Isabel
detailed how she and Ana always tried to
find a mental outlet, whether in or out of
the hospital, to express their emotions
while dealing with the daily challenges
of living with CF. This included drawing,
painting, photography, and writing.
As her disease progressed, Isabel
discovered that, “advanced disease was
uninvited, but became an opportunity.”
This opportunity presented itself in the
form of therapeutic narrative writing. Isabel
shared that through writing and telling
her story, her resilience grew, noting,
“Writing is a way to link the right and left
brain’s responses to challenges, and fosters
self-awareness, insights into behavior,
understanding of others’ struggles, and a
sense of one’s own growth. Additionally,
sharing one’s writing fosters reciprocity,
empathy and a special kind of intimacy.”
Isabel became a character in her own
dramatic life story: both Isabel and Ana
received double lung transplants, which
Isabel shared was the “ultimate redemption
and reward.” In 2013, Isabel’s strength and
resiliency were put to the test with the
loss of her sister Ana. In honoring Ana and
her memory, Isabel continues to share
Isabel Stenzel Byrnes at the Transplant Games
their journey of living with CF, and the
challenges and opportunities it presents.
Change in the life of a person living with
CF is a given, but how one faces those
changes is up to the individual. Isabel’s
insights on resiliency exemplified courage,
tenderness, sadness, and loss, and also her
triumph as a survivor.
cfri | Fall 2014
Cystic Fibrosis and Cancer:
The Need for Awareness and Screening
By Siri Vaeth Dunn, MSW
While very limited research has been
conducted on cancer in cystic fibrosis
patients due to the small numbers involved,
Dr. Van Loon shared the results of a 2012
study which analyzed data from over
41,000 cystic fibrosis patients followed
at U.S. CF centers between 1990 and
2009 and concluded that, “CF patients
have an increased
risk of digestive tract
cancers – including
esophago-gastric,
biliary tract, small
bowel, and colon
cancers – compared
to the general U.S.
population.” The
study also reported
that CF patients
demonstrated an
increased risk of
testicular cancer and
lymphoid leukemia,
but a decreased
risk of melanoma.
Dr. Van Loon noted
that, “the increased
risk for digestive
tract cancers was
most pronounced
in patients who had
previously undergone
lung
transplantation,
Dr. Katherine Van Loon is studying the incidence and implications of
with most cancers
gastrointestinal cancer in those with CF.
arising in the bowel.”
Be Done,” at CFRI’s 27th National Cystic
While colonoscopies to screen for preFibrosis Family Education Conference,
cancerous polyps are recommended for
Katherine Van Loon, MD, MPH, Assistant
the general population beginning at the
Clinical Professor and clinician at the
age of 50, Dr. Van Loon expressed concern
Helen Diller Family Comprehensive Cancer
that this may be too late for patients with
Center at the University of California, San
Francisco, raised awareness of a significant cystic fibrosis. Dr. Van Loon noted that
a literature review “identified multiple
health issue for those with CF. Dr. Van
cases of colon cancer diagnosed in CF
Loon discussed the increased risk for
patients between the ages of 13 and 45,
cancers of the digestive tract in patients
suggesting that this patient population
with CF, and the need to assess whether
may be at risk from a much earlier age.”
new screening protocols should be
She also discussed a study of over 28,000
implemented for this population. Dr. Van
patients with CF in the U.S. and Canada, in
Loon, a gastrointestinal cancer specialist,
which the mean age of onset of digestive
was inspired to study the incidence and
tract cancers was 32 years, while those
implications of gastrointestinal cancer in
between the ages of 20 and 29 had a
those with CF by several of her patients
notable increase in risk.
facing this dual diagnosis.
Thanks to advances in the understanding
and treatment of cystic fibrosis (CF),
people with CF are living longer; but as they
age, other health complications emerge
that may not be overtly CF related. In her
illuminating presentation, “CF and Cancer:
Who Should Be Concerned and What Can
cfri | Fall 2014
Dr. Van Loon stressed that both patients
and care providers need more information
about the increased risk of digestive tract
cancers for those with CF. Despite studies
indicating that patients with CF have a
higher risk and are diagnosed at a younger
age than their non-CF peers, there are
currently no screening protocols that take
these factors into consideration. Dr. Van
Loon emphasized, “because this population
suffers from chronic gastrointestinal
complications of CF, many early warning
signs and symptoms of gastrointestinal
malignancy – for example, abdominal
pain, constipation, and anemia – may be
masked or attributed to other etiologies,
resulting in delays to diagnosis.” In an
effort to develop new cancer screening
protocols for CF patients, Dr. Van Loon
is currently enrolling subjects for a study
of adults with CF, aged 30 and older,
who will undergo colonoscopies and
screening. Support for her research has
been provided through a cystic fibrosis
oncology research fund established in
memory of Anabel Stenzel, who had CF and
lost her battle with small bowel cancer
in 2013. Results of this important and
timely study will guide the development
of recommendations for gastrointestinal
cancer screening for those with CF.
Anabel Mariko Stenzel
5
CFRI Announces
Formation of
Medical Advisory
Board
Beate Illek, PhD
Bill Hult
Julie Desch, MD
Honoring the Extraordinary: CFRI’s Annual
Awards Recognize Our Community Heroes
By Siri Vaeth Dunn, MSW
Each year at our National Cystic Fibrosis Family Education Conference, CFRI honors individuals that have made extraordinary contributions to the cystic fibrosis (CF) community.
Nominations for the CFRI Professional of the Year and the Dave Stuckert Memorial
Volunteer of the Year were received from across the nation, and a panel of judges comprised of prior award winners selected the outstanding honorees. This year, CFRI’s Board
of Directors also introduced the CFRI Partners in Living Award in Memory of Anabel Stenzel.
The 2014 Professional of the Year Award was presented to Beate Illek, PhD, a brilliant
researcher at Children’s Hospital Oakland Research Institute (CHORI) who has achieved
key discoveries including: the conductivity of CFTR to bicarbonate and other anions in
airway cells; the discovery of genistein as an activator of G551D mutant CFTR activity; the
characterization and establishment of novel CF bronchial epithelial cell models; and the
discovery of a malfunctioning bacterial defense mechanism in CF airway cells. In addition
to these accomplishments, Dr. Illek has established a CF research student program at
CHORI to inspire new leaders in the field. Dr. Illek exemplifies outstanding dedication and
commitment to the CF community.
Bill Hult, CFRI’s intrepid Board president, was awarded the 2014 Dave Stuckert Memorial
Volunteer of the Year Award, created in honor of this dedicated CFRI volunteer who lost
his battle with CF in 1989. Since joining CFRI’s Board of Directors in 2004, and his election as
President in 2008, Bill has helped to expand CFRI’s capacity to meet the needs of a broadening CF community, while ensuring CFRI’s financial and organizational health. Serving as
Board liaison on the Research Advisory Committee (RAC) and developing strategies related
to CFRI’s long-term vision, Bill exemplifies an unflagging commitment to CFRI’s mission.
CFRI’s Board of Directors inaugurated a new award this year, the CFRI Partners in Living
Award in Memory of Anabel Stenzel. Ana, a former CFRI Board member, was a beloved
member of the CFRI community; a strong woman with CF who lost her battle with cancer
last year. The recipient of this new award is selected by CFRI’s Board of Directors. Nominees
must have CF, have supported CFRI through volunteer and/or fundraising efforts, and embody
the qualities exemplified by Ana, including courage, initiative, determination, adherence
to medical regimen, community service, and positive coping.
The first recipient of the CFRI Partners in Living Award in Memory of Anabel Stenzel was Dr.
Julie Desch. Dr. Desch received her medical degree in pathology from Stanford University
School of Medicine. She has been a dedicated CFRI volunteer for many years, previously
serving on the Board of Directors, and sharing her medical and scientific knowledge through
membership on the Research Advisory Committee. She has been a presenter at CFRI’s
Discovery Series, and has authored articles for this newsletter. Dr. Desch is the founder of New
Day Wellness, a nonprofit organization with a mission to coach and train wellness to people
with chronic illness. Dr. Desch exemplifies Anabel’s spirit through her courage, positive
attitude, and volunteer service to CFRI.
Congratulations to these outstanding individuals!
6
CFRI is honored to announce the
formation of our new Medical
Advisory Board, comprised of some of
the most esteemed members of our
medical and research community. We
are grateful to these individuals for
sharing their expertise and experience
so as to ensure CFRI has vital
information and access to the most
current CF-related news and protocols.
Julie Desch, MD
Founder, New Day Wellness
Deborah “DJ” Kaley
RN, MSN, AE-C, CPN
Pulmonary Nurse Case Manager
Bay Area Pediatric Pulmonary
University of California San Francisco
Benioff Children’s Hospital Oakland
Thomas G. Keens, MD
Director, Cystic Fibrosis Care Center
Children’s Hospital Los Angeles
Professor of Pediatrics
Physiology and Biophysics
Keck School of Medicine
University of Southern California
Paul Quinton, PhD
Nancy Olmsted Professor in
Pediatric Pulmonology
Professor of Biomedical Sciences
University of California Riverside
Department of Pediatrics
Rady Children’s Hospital
University of California San Diego
School of Medicine
Kristin Shelton, RRT
Respiratory Coordinator
CF/Pulmonary Clinic
Stanford Children’s Health
Ahmet Uluer, MD, DO
Director, Adult Cystic Fibrosis Program
Division of Pulmonary and
Respiratory Diseases
Boston Children’s Hospital and
Brigham and Women’s Hospital
Assistant Professor of Medicine
Harvard Medical School
cfri | Fall 2014
The CF Journey:
Where We Have Been and
Where We are Going
By Linda Burks, EdD
Taking us on a time travel trip from medieval Europe to the 21st century, Thomas
G. Keens, MD, Director of the Cystic Fibrosis
Care Center at Children’s Hospital Los
Angeles, and Professor of Pediatrics,
Physiology and Biophysics at the Keck
School of Medicine at the University of
Southern California, engagingly presented,
“Where We Have Been and Where We are
Going” in the field of cystic fibrosis (CF)
at CFRI’s National Cystic Fibrosis Family
Education Conference. With the median life
expectancy for those with CF now in the
early 40’s, medical advances have come a
long way since CF was first noted in German
folklore as
a hex, when
tasting salt
while kissing
a newborn
was seen as
a sign the
baby would
soon die.
In 1938, life
expectancy
for those with
CF was less
than one year.
Two years
later, CF was
recognized as
an autosomal
recessive
disorder.
Incorporation
of the Cystic
Fibrosis
Foundation
in 1955 raised
Thomas G. Keens, MD
research
awareness,
while the establishment of the first CF
Centers in 1961 improved care and increased
the median age of survival to 10 years. CFRI
was established in 1975. At that time, few
with CF survived their teens.
An historic milestone was reached in 1989
when the CF gene was identified. With the
cfri | Fall 2014
discovery
of the cystic
fibrosis
transmembrane
conductance
regulator
protein
(CFTR), it
seemed a
cure was close by. That trip has taken longer
than first expected.
CFTR regulates chloride transfer in and
out of the cell. The improper function of
the chloride channel creates sticky mucus
and an excess of salty sweat, and many
organ systems
are negatively
affected.
Pancreatic
enzymes are
produced,
but the
thick mucus
prevents
them from
reaching the
intestine to
aid digestion.
Scarring
occurs in the
pancreas and
liver. The
heavy mucus
weighs down
cilia in the lung
lining, enabling
bacteria to
develop,
resulting in
damaged lungs. These
symptoms
have been treated by new therapies –
including Pulmozyme®, azithromycin,
TOBI®, and Cayston® – that have generally
increased overall lung function. A primary
focus now is preventing acute exacerbations, which typically result in dramatic
loss of lung function. Researchers seek to
understand what causes the dip in FEV1
following an exacerbation, and whether it
is gradual or sudden. With newborn screening for CF now
standard in all 50 states, many mutations
(nearly 2,000 thus far) have been
identified, and as Dr. Keens explained, this
enormous number further complicates the
search for a cure. Mutations are classified
in two main categories: one where little
to no CFTR protein is found on the cell
surface; the second where CFTR is on the
cell surface, but fails to function properly.
Observing that disease severity may be
predicted by the percent of functioning
CFTR, the goal of new therapies is to
modify CFTR so that 30% of the protein
functions properly. Recent research on CFTR modifiers has
demonstrated exciting results. The
potentiator Ivacaftor (VX770 or Kalydeco®)
has been shown to produce 50% CFTR
function for those with the G551D mutation.
For those with two copies of the Delta
F508 gene, the combination of Ivacaftor
with Lumacaftor (VX809), a corrector that
brings CFTR to the surface, is promising.
Initial results from Phase 3 studies indicate
significant improvement in FEV1, body mass
and quality of life. Unfortunately, little
difference was found for those with one
copy of the Delta F508 gene. It is an exciting time. As new treatments
to prevent disease progression are
discovered, the possibility of further lung
and pancreatic damage can be kept at
bay. As Dr. Keens compellingly shared,
we are on the cusp of a new generation of
treatments for CF and are converging on
a cure. 7
There in the Room:
From Survival to Healthiness with Cystic Fibrosis
By Darlene Batchelder
Mary ElizaBeth Peters is a survivor. The
33-year-old theater artist and CF educator
from Boston, Massachusetts, received a
double lung transplant in 2010.
In her moving presentation,
“More than Survival: A Pursuit
of Healthiness,” given at CFRI’s
27th National Cystic Fibrosis
Family Education Conference,
Peters used the stage to give
voice to her own unique
cystic fibrosis (CF) experience,
as well as to the journeys and
stories of others touched by CF.
When she isn’t on stage,
Peters works with children
with disabilities, publishes
a blog, directs theatre
productions, and writes. Her
book, Unto the East: Words in
Waiting, is the story of her wait
for a double lung transplant.
She began her opening
night presentation by asking
everyone to
answer the
questions,
“What can you
do that others
cannot?” and
“What can’t you
do that others
can?” In doing so,
she invited us all
to consider that
regardless of our
varied abilities, we
each have unique
stories to share and
it matters that we
share them.
Confessing that she was “bored with her
own biography,” Peters shared what
others with CF suggested she explore in
her presentation, including: “Acceptance
of the disease, the emotional effects, as
well as physical;” “How freaking tired
we are – I am not lazy, I am exhausted;”
“The psychological effects;” and, “This is
8
or noon on a Sunday in the hospital when
no one is around and you can’t get a pass
off the floor to go buy a Snickers bar and
your family is at home eating lunch after
church. We are there, in
the room…We are there in
the room when a person
is diagnosed with cystic
fibrosis. We are there in
the room at a graduation
party we worried wouldn’t
come. We are there in the
room when it’s time to start
using supplemental oxygen.
We are there in the room
when you win a swim meet.
We are there in the room
when you have to go in the
hospital the Wednesday
before the prom. We are
there in the room when
you get married. We are
in the room when you get
divorced. We are in the
room when your wife/
husband leaves you a year
after transplant and you
wonder why this happens
Mary ElizaBeth Peters
so often with so many
people you know. We are there in the room
we are uniquely linked by
many common experiences. when you first get listed for transplant. We
Peters shared her distincare there in the room when your kid goes
tive CF experiences as
to kindergarten. We are there. In the room.
part of an online CF
We are present. ”
women’s group. Despite
In closing, Peters read her moving and
the challenges placed
autobiographical poem, “My Sister Died
upon those with CF to
on a Sunday,” noting that she decided to
join together, Peters
share it, “not to make us all sad and cry,
movingly conveyed the
though that might be the case. I chose to
deep connections that
present it because it highlights an important
can be forged online,
fact – your life is not someone’s movie. It’s
and the support and
your real life. It’s happiness and it’s sadness,
sense of community
and the good days and bad days are all your
that results. The following excerpt
real, real life. And that’s your story and you
was created by the women in the group,
inspired in part by the Vagina Monologues should tell it.”
poem, “I was there in the room.”
Beth eloquently encouraged us to reflect
on and tell our unique stories. Everyone
“We meet online and we are . . . present…
has one; and the authentic expression of
We are in a tiny internet room where
it, amidst the sadness and challenges of
we can share with each other at 4 in the
morning when we can’t sleep or 9 in the
CF, can lead to acceptance, connection,
morning when the spouse left for work
better health, and happiness.
an invisible illness – you might not look sick
but you can still be really sick.” The varied
responses made it clear that while our
individual experiences with CF are unique,
cfri | Fall 2014
CFRD: Current Issues & Future Strategies
By Siri Vaeth Dunn, MSW
Cystic fibrosis-related diabetes (CFRD)
impacts nearly 40% of young adults with
cystic fibrosis (CF). CF causes scarring to
the pancreas, thereby destroying a large
percentage of the insulin-secreting cells.
Left uncontrolled, CFRD can
have a significantly negative
impact on lung function and
mortality. Yet as Antoinette
Moran, MD, Professor and
Division Chief of Pediatric
Endocrinology at the
University of Minnesota
compellingly shared during
her presentation, “The
Sweet Evolution of CFRelated Diabetes,” at
CFRI’s 27th National Cystic
Fibrosis Family Education
Conference, early diagnosis
and insulin intervention can
reverse this trend, while
new “corrector” and
“potentiator” drugs in
the CF treatment pipeline
might “fix” the problem by
increasing insulin production.
high blood glucose levels create an ideal
environment for the growth of opportunistic bacteria in the lungs of CF patients.
Early diagnosis is key, and Dr. Moran
questioned whether earlier screening
Insulin helps the body to use
nutrients in food to produce
Dr. Antoinette Moran
energy, gain weight, and
build muscle. As noted by
should be implemented, because, “in the
Dr. Moran, “Essentially all cystic fibrosis
‘pre-diabetic’ period – years before the
patients are insulin insufficient,” leading
actual diagnosis of CFRD – nutritional
to “muscle and fat breakdown, difficulty
gaining or maintaining weight (particularly status and pulmonary function begin to
decline.” Early CFRD symptoms include
muscle), and impacting lung function.”
increased thirst and urination, the inability
The insufficient amounts of insulin are
to gain or maintain weight, poor growth
even more detrimental during times of
in children, and an unexplained and rapid
infection and steroid use.
drop in lung function. But Dr. Moran
Many studies have documented the
stressed, “There may be no obvious
negative impact of diabetes on survival
symptoms!” While oral glucose tolerance
in CF. Both hyperglycemia (high blood
testing (OGTT) is usually conducted
glucose) and insulin insufficiency have a
annually after a patient turns 6, there
negative impact on CF lung disease. As
are indications that many children have
explained by Dr. Moran, “There is normally
impaired glucose at a much younger age.
no glucose in airway secretions, but in CF,
Dr. Moran discussed a study in which
airway secretion glucose is elevated when
OGTT is conducted with infants beginning
plasma glucose is above 144 milligrams
at three months old that may provide new
per deciliter.” A study of CFRD patients
insights related to the ideal age to initiate
undergoing constant glucose monitoring
glucose testing.
found they spent nearly 50% of each day
above this level. This is significant, because Dr. Moran stressed, “Insulin provides a clear
cfri | Fall 2014
benefit and is the definitive treatment for
all patients with CFRD (with or without
fasting hyperglycemia).” Currently, 41% of
children with CF tested at ages 6 to 10 years
have abnormal glucose tolerance, leading
Dr. Moran to ask, “Should we be treating
pre-diabetes with insulin?”
A new, multi-center placebocontrolled study will explore
whether this is the case.
Dr. Moran raised the
possibility that the new
“potentiator” and
“corrector” drugs to
treat CF could help those
with CFRD, citing a small
study that showed that
“Ivacaftor increased
insulin secretion by 66% –
178% in four out of five CF
patients ages 6 to 52 years
with the G551D mutation.”
Dr. Moran pondered
whether those with
“pre-diabetic” glucose
intolerance levels would
also be candidates for
early treatment with these
new therapies.
Dr. Moran stressed that
while high mortality rates
for females with CFRD used to be the
“worst scenario,” this has been reversed
at her center because, “controlling CFRD
Those with CF-related diabetes must closely
monitor their blood glucose levels.
makes the difference.” She added, early
intervention and aggressive insulin therapy
“reverses chronic nutritional and pulmonary
decline in CFRD, thus improving survival.”
9
The CFRI Teen and Adult Retreat: A Zen
Experience!
By Anna Modlin, MA
This year’s CFRI Teen and
Adult Retreat was a great
success. The retreat included
several Zen-themed activities
-– Tai Chi, SoulCollage®
and guided imagery. We
were fortunate to have Dr.
Steve Singh and Dr. Elena
Sandoval, surgeons from
CFRI’s “Zen” Teen and Adult Retreat provided a place for
St. Luke’s Lung Transplant
reflection, camaraderie, information, and fun.
Center in Houston, lead
a question and answer session, and we
have lost, no matter how long ago.
also had a session on adoption. Rap
Unfortunately, this year we said goodbye
sessions were scattered throughout the
to some of our longtime friends, Ana S.,
retreat, during which attendees provided
Steve P., and Nahara M., along with many
emotional and social support to one
friends from past retreats and CF camp.
another. The week was filled with sharing,
They are missed but never forgotten, and
fun and laughter, all while observing
the memorial was a moving tribute. We
stringent cross-infection guidelines. We
ended the week with a hilarious talent
participated in entertaining games of
show and skit night. The snack room was
Wiffle ball, and some people even gloved
overflowing with goodies: special thanks
up to throw around a football. Most
to the Johsts at Grocery Outlet and to
attendees picked secret buddies at the
Whole Foods for their donations. During
beginning of the retreat, and presented
the week, new friendships were made, and
awards to their “Buddha buddies” at the
bonds deepened with old friends. Despite
awards ceremony. We held our annual
being a little tired from all the fun, I left
candlelight memorial, at which we lit
the retreat hopeful, emotionally filled,
candles and told stories about those we
refreshed, and recharged!
Donate Life:
Even Those With CF Can Donate!
By Siri Vaeth Dunn, MSW
Many members of the cystic fibrosis
community have received the gift of lung
transplantation. But many more are waiting.
It is extraordinarily easy to become an
organ donor, as every state has an online
registry. Most importantly, express your
wish to be a donor to your family, friends
and physicians, and include your wishes in
an advance directive or will. You do not
need to be young to be a donor: In 2013,
35% of deceased donors were over 50
years old, and of these, many were over
the age of 65.
You also do not need to be in perfect health.
In 2013, Gianna Altano lost her battle with
CF while waiting for a lung transplant. She
10
in turn, donated her kidneys and corneas.
That same year, Ana Stenzel lost her battle
with small bowel cancer after living with
CF and receiving two lung transplants.
She also donated her corneas. Tissue Bank
International has selected Ana to be an
honoree on Donate Life America’s float
at the annual Rose Parade in Pasadena,
California on New Year’s Day, 2015. By being
an organ donor, these women – and many
others – have helped extend and improve
the lives of people in need.
CFRI’s 27th
National
Cystic Fibrosis
Family
Education
Conference
Thank you to our generous
sponsors and supporters who
helped to make this year’s
event a tremendous success.
Sustaining Sponsors
Genentech, Inc.
Novartis Pharmaceuticals
Vertex Pharmaceuticals
Chiesi USA, Inc.
Platinum Level
AbbVie
Boomer Esiason Foundation
Gilead Sciences
Gold Level
Forest Laboratories
Foundation Care Pharmacy
Cystic Fibrosis Services Pharmacy
Silver Level
Electromed, Inc.
RespirTech
Med Systems
Cystic Fibrosis Pharmacy – Florida
Modern Health Pharmacy
Hill Rom
Supporters
Pari
Kendal Hall and Brad Huntzinger
Prodigy Press, Inc.
Sofitel San Francisco Bay
If you have not yet registered to be an organ
donor, go to www.organdonor.gov for the
latest information and links to register in
your state. The design for Donate Life America’s
2015 Rose Parade float
cfri | Fall 2014
California’s CF Newborn Screening:
Lessons Learned and Future Implications
By Eric Frisbee
Attendees at CFRI’s 27th National Cystic
Fibrosis Family Education Conference were
honored to have Martin Kharrazi, PhD,
MPH, present “Reflections on Detection
of CF and CRMS in California Newborn
Screening.” Dr. Kharrazi, Research Scientist
Martin Kharrazi, PhD, MPH
Supervisor with the California Department
of Public Health Genetic Disease Screening
Program, was instrumental in the design,
implementation, and evaluation of California’s unique 3-step newborn screening
model for cystic fibrosis (CF).
All 50 states in the U.S. include cystic fibrosis
in their newborn screening (NBS) programs,
as early detection of CF offers the opportunity for early intervention and improved
outcomes. NBS methods and models
differ by state. Each state program checks
for a chemical produced by the pancreas
called immunoreactive trypsinogen (IRT) as
a first step. When IRT levels are elevated,
most states’ CF screening programs test
for a panel of 23 mutations in the CF
transmembrane conductance regulator
cfri | Fall 2014
(CFTR) gene. Newborns with one or more
mutations are referred for sweat chloride
testing. The California program selectively
tests for 40 severe and ethnically diverse
mutations on its panel, and further tests
the blood spot by DNA sequencing when
a single panel mutation is found to look
for additional mutations.
Only newborns with two
or more mutations are
referred for diagnostic
follow up at CF Centers.
Dr. Kharrazi’s presentation gave insights on
the increasing number
of infants with CFTRrelated metabolic
syndrome (CRMS) that
are being identified
through NBS. In
California, CRMS is
diagnosed in infants
with a high IRT value
who have two CFTR
mutations (one CFTR
panel mutation, and
at least one CFTR
mutation from DNA
sequencing in trans), a
negative or intermediate
sweat test result of
sweat chloride values
<60 mmol/L, and no
clear CF symptoms. Some infants with CRMS
convert to CF in the first several years of
life. The others may be at higher risk of CF in
the future or of having problems
in the airways and sinuses,
intestines and pancreas, or the
reproductive tract as they age.
California started newborn screening for cystic fibrosis in July 2007.
The objectives for the design of
the California NBS program include:
using a 1 blood spot screening
model; focusing on severe cases
of CF; identifying at least 90%
of all Hispanic, Caucasian and
African American cases; reducing
the burden of false positives,
false negatives, sweat tests, and
costs, while maximizing case detection;
providing efficient reporting of NBS results;
diagnosing and following up on positive
screenings through accredited CF Centers.
Since the introduction of California’s CF
NBS, there have been over 3 million births
in the state. During this period, the CF NBS
program had a case detection rate of 92.7%,
with 421 cases detected by NBS out of 454
total CF cases. In addition, during this time,
NBS identified 539 CRMS cases (90 per
year), and 1,919 CF-carriers (320 per year).
“While an initial goal of the California NBS
was to focus on severe CF cases, with the
ultimate diversity of CF mutations – 400 in
total – and with the identification of many
newborns with CRMS, the California CF
NBS program continues to evolve,” stated
Dr. Kharrazi.
CF is not always a straight-forward diagnosis. Knowing your CF-causing mutations
is critical in getting the right treatments.
Disease-causing mutations with varying
clinical significance will require new
research to understand CFTR-related
disease manifestations over time. NBS
education will be refined, highlighting the
importance of genetic counselors trained
to help families deal with the uncertainties
of the NBS results, and the uncertain health
implications related to certain genotypes.
Additional resources can be found at the
following websites: California Newborn
Screening Program, www.cdph.ca.gov/NBS
and CFTR2 Project, www.cftr2.org
Many new CFTR mutations have been identified since the
implementation of California’s CF newborn screening program.
11
CFRI’s 27th National Cystic Fibrosis Family Educa
Save the Date:
July 31st –
August 2nd
2015
12
cfri | Fall 2014
ation Conference
cfri | Fall 2014
13
April 1, 2014 — August 31, 2014
Our “In Memory of ”
and
“In Honor of ”
pages
provide the
opportunity
to honor a
person, family,
or special event,
or
to remember
a loved one.
If you want your
donation to honor
or
remember
someone special,
please include
the person’s
name and address
with your
donation.
At your request,
we will send an
acknowledgement
of your gift to the
person you
designate.
Please mail your
contributions to:
CFRI
1731
Embarcadero Rd.
Suite 210
Palo Alto, CA
94303
14
Donald Aasen
Carol A. Adelman
Marcus Adelman
Sonya L. Akister
Henry G. Allison
Gianna R. Altano
Rosemary Altano
Mrs. Anderegg
Edward Andreini
David Armknecht
Jodi Armknecht
Richard Arnold
Kathleen C. Baldwin
Rose E. Balsley
Roger Baroody
Gino Barsanti
Betty Ann Beard
Anne C. Beltrame
Barbara Bennetti
Patricia Berndt
James W. Bertolini
Amy Bienenstock
Wendy Bosarge
Greg Brazil
Dave Brewer
Marty Brown
Ellis Bryant
Kyle Butler
Kenneth Cady
Carol Carey
John Carey
Sandra Cochan
Ryan Coelho
Kelly L. Colgan
George Colla
Leon Cramer
Rachel Crocker
Caroline Daly
Irene Day
Guido De Chellis
Matthew DeGregory
Charles Delgado
Neva L. DeVore
Jim Edwards
Jennifer Eisner
Trevor Fenn
Charles M. Fontana
Jerry Fox
Charles Fraker
Georgette Francis
Bob Franks
Jessica Fredrick
Jerry, friend of
Denise Morgan & family
Judy Gaal
Marie Gagliardi
Laura Gale
Aaron Gallagher
W. Erby George
Royce Goertzen
Diana Goodman
Robert Goodrich
Douglas Graham
Christina Groleau
Stephanie Halling
Virgil Hanson
Warren A. Harden
Vivian Hartman
Mark Holmes
Tracy Holmes
Jennifer House
Christopher Ireland
June L. Jackson
Jennifer M. Jameson
Orzella Jemas
Edward J. Jensen
Karen Johnson
Penelope Johnson
Mary Jones
Kathy & Peter Judge
Kurt Keonig
Kitty Kious
Lori Kipp
Bridget Klein
Jeffery Kreth
David Kroepfl
Jane Ellen Kulik
Carolyn S. Leavitt
Dawn Longero
Jennifer Longoria
Joseph A. Losier
Stephanie MacArthur
Robert Mackey
Christine Magee
Betten Marks
Jason Marshall
Jordan Marshall
Ken Martin
Paul Mathews
Nahara Mau
David McAfee
Karen Melvin
Nancy M. Melvin
Teresa Messer
Jennifer Mieuli
Matthew Mitchell
Jessica Mobley
Bob Moore
Brian Moore
Mike Moore
Loretta Morris
Roger Morris
Leanor Mott
Lynette Moulton
Dana Munsey
Kimberly Myers
Elizabeth Nash
Kim Nelson
Kim & Scott Nelson
Julie O’Leary
Jennifer Ortman
Dellene Ott
Matthew Ozoskey
Barbara Palys
Joseph J. Pausner
Lisa Pearne
Scott Petersen
Sean Peterson
Steven Peterson
Jon Prater
Timothy Quinton
Teresa Ravetto
Catherine Rawlings
Jaimee B. Renfrow
Melissa Reta
Winn Riske
Joshua Roberts
Katie Robinson
Pamela Rockhold
David Rolefson
Thelma Drogin Rosenthal
Marsha Rounds
Billy Ruffner
John Runge
Randy Rupracht
Siobhan H. Ryan
Michelle Sanderson
Maureen Sazio
Kathy Schaal-Wood
Dhea Schalles
Linda Scherschel
Joseph M. Sinnaeve
Tammy Smerber
Brian Smith
Anabel M. Stenzel
Robert C. Stewart
Charlie Stockley
Delinda Syme
Bill Syms
Christy Tegard
Steven Tegard
Patricia Thibault
Thibault Family
John Trask
Louis A. Trigueiro
Mary Tripp
Roy Tripp
James Tyrrell
Jennifer Uskoski
Rory & Jerry Vaeth
Jodi L. Voller
Aslaug Walters
Tom Walton
Sean Waltrip
Nicky Wastell
Irene Way
Delores Wegner
Tara Weir
Maurice Wernli
Hayley C. Wester
Beverly L. White
Sue White
Tina Wilkerson Crow
Cynthia Witman
cfri | Fall 2014
April 1, 2014 — August 31, 2014
Connor Acheson
Gordon P. Adelman
Lisa Allen
Sadie Anderson
Hatsuko Arima Stenzel
Beth Arvidson
Jessica Arvidson
Colby Babcock
Julie Baker
Kyle Baker
Robert Balsley
Casey Banos
Bridget Barnes
Lucy Barnes
Jamie Barry
Darrell Batchelder
Joseph Batchelder
Marilyn L. Bates
Makinnon Baugh
Marin Baugh
Baugh Family
Evelyn Baughman
Jamie C. Beasley-Killinger
Maggie-Faye Bendz
Brett Bennett
Amanda Bergman Moretto
Allison Best
Sallie Best
Aidan Biggar
Oliver Biggar
Brian Burks
Linda Burks
Isabel Stenzel Byrnes
Sean Campise
Andrew Carl
Ryan S. Carlstrom
Justin Carnie
Ruthie Carnie
Lauren Catron
Maureen Coffy
Eleanor Collins
Lauren Colonna
Cameron Cornell
James Cornell
Cornell Family
Barbara H. Curry
Carmen Curry
cfri | Fall 2014
McKenna H. David
Stacy Dean
Tracy Del Real
Gordon DeVore
Ben Dippner-Robertson
Josh Dodami
Jason Dolan
Tess Dunn
Tracey Efird
Daniel Ellett
Hayden Ellett
Kyle England
Timothy Estabrook
Janelle & Andre Estournes
Tommy Evans
Isaac Farfan
Kristin Favero Konvolinka
Billy Federal
Theresa Fenimore
Johnnie Figueira
Victoria Flamenco
Kathleen Flynn
Flynn Family
Jacob Fraker
Joseph Fraker
Cody Franks
Emily Fredrick
Jessica A. Fredrick
Rhonda Fredrick
Taylor Frisbee
Sean Gallagher
Jenise Giuliano
Larissa Giuliano
Mark Glisson
Elyse Elconin-Goldberg
Ruth Goldberg
Jen Goodwin
Emily Gorsky
Barbara S. Greenberg
Bonnie Grossman
Alejandra Guzman
Sonya Haggett
Hampton Twins
Jean Hanley
Julianna Harding
Monica Harding
David Hardy
Kathy Hardy
McKenna Hardy
T.J. Hardy
Alyssa Harvey
Lisa Hendley
Susan Hoffman
Jeremiah Holdaway
Joshua Holdaway
Courtney Hollis
Nicholas Hollis
Andrew Hotson
Drew & A. Hotson
Erinn Hoyt
Kristen Hoyt
Clark Huddleston
Noelle Hunt
Alexander Jenkins
Barbara Jensen
Lindsey Jensen
Darren Johst
Laine Jones
Michelle Jones
Peggy B. Jones
Wedding Guests of
Amanda & M. Jozkowski
Franny Kiles
Edward Kinney
Shae Kish
Ray Knudsen
Julianne Kobernus
Eleanor Kolchin
Jason Konkel
Kathy Konkel
Santosh M. Krishnan
Sally Kusalo
Steven Kusalo
Daniel Lagasse
Douglas Lagasse
Susan Lane
Stacey Lawn
Kody Lawrence
Barbara Lenssen
Joey Librers
Tara & S. Linehan
Michael Livingston
Ruth Livingston
Alyson Lowery
Emily F. Lucas
Joseph Madrigali
Larissa Marocco
Eric Marten
David Martin
Rose Martini
Gary Masching
Elizabeth A. Mayer
Tessa McCarthy
Beth McMullen
Rachael &
Rebecca McMullen
Kelly H. Melle
Stacy Melle
Jackie Merrill
Linda Meyer
Hannah Mitchell
Anna Modlin
Amanda Morelli
Amanda Moretto
Richard Moss
Chuck Nelson
Kate Nelson
Jessica Nett
Lindsey J. Nijmeh
Erin O’Dell
Scott Parks
Michelle Lynn Payton
Brie Peters
Kelley Phillips
Alex Prator
Todd Primack
Justin Raines
Briauna Red
Albert Reet
Cara Richardson
Rebecca Roanhaus
Ben Robertson
Abby Robinson
Ann Robinson
Carl Robinson
Robinson Family
Dale Roeder
Taylor Rolefson
Alanah Rosenbloom
Ben Sanford
Bridget Schmidt
Janice Shaul
Kerry Sheehan
Kandra L. Smith
David Soohoo
Ethan Spain
Lisa Steiding
Karlye Stein
Blaine Strohn
Brian Tacke
Alonzo Tatem
Heidi Tegner Geyer
Adam Thompson
Corky Thompson
Lauri Thompson
TJ
Lisa Tingley
Todd Trisch
Robert Turk-Bly
Siri Vaeth
Chris Vallee
William Vogt
Devin Wakefield
Hayley C. Webster
Jean C. Webster
Christopher Wernli
Pat Williams
Kareese Wilson
Nina Wine
Amanda Wood
15
Tips for Living an
Extraordinary Life:
Adult Panel
Discussion
At CFRI’s 27th National Cystic Fibrosis
Family Education Conference, during
a panel discussion, “Tips For Living An
Extraordinary Life,” five adults living with
cystic fibrosis (CF), ranging in age from
19 to 49 years, generously shared their
personal experiences and perspectives
on living life fully. Moderated by CFRI’s
former executive director, Carroll Jenkins,
MA, the panelists – Adriana Duffy-Hörling,
JD, Danielle Mandella, Anna Modlin, MA,
Sara Umber, and Robert “Bob” Wright –
explored issues related to employment,
health insurance, disclosing one’s
diagnosis, adherence to treatments, and
the pursuit of life goals. Members of the
audience had numerous questions for
the panelists, who provided a wealth
of information during their honest, and
often humorous, replies. CFRI is grateful
to our panel participants!
16
cfri | Fall 2014
Pain Free: Management Strategies in Cystic Fibrosis
By Bridget Barnes
In his lively talk, “Beyond the ‘Ouch Factor:’
Pain Management in Cystic Fibrosis,” at the
recent 27th National Cystic Fibrosis Family
Education Conference, Stephen Jones, MS,
RN, CPNP, ET, noted that many people living
with CF have chronic pain, and stressed the
importance of determining the underlying
cause and not just treating the symptoms.
Wearing numerous hats at Children’s Hospital, Albany Medical Center in New York,
Mr. Jones has worked for 31 years as a
Pediatric Nurse Specialist, Pediatric Nurse
Practitioner, and Enterostomal Therapist.
Over the past several years, he has focused
his attention on pain and discomfort in
CF patients.
and adults living with CF – interferes with
activities, lowers the quality of life, and
negatively impacts participation in daily
health care. Children with more frequent
pain experience worse physical and social
functioning and increased CF-specific
symptoms. Because pain is associated with
survival, assessment and treatment of
pain are crucial.
Mr. Jones reported that a 2012 study found
that pain is an equally common complaint
for acutely ill and clinically stable adults.
Pain intensity was consistent regardless of
disease severity and, “pain can come from
many sources because CF affects many
organs.” In determining the physiological
Stephen Jones, MS, RN, CPNP, ET
“In America we love to treat the symptom
and not the cause,” Mr. Jones explained,
“but what works for one person, doesn’t
work for another. Pain is a subjective
indicator that something is going on,” and
has sensory, emotional, cognitive, and
behavioral components that are interrelated
with environmental, developmental,
sociocultural, and contextual factors.
According to a 2007 study, children have
fewer adaptive skills when pain is present,
and pain early in life might lead to longterm consequences affecting growth
and development. Abdominal pain – the
most common discomfort in children
cfri | Fall 2014
and psychological components influencing
pain, Mr. Jones asked, “So the real
questions are: Is it physical pain? Or is it
fear, frustration, anger, or anxiety, because
narcotics won’t touch these – they are
psychological.” In order to determine pain
relief, it’s important to understand the
exact words used when a child or adult is
describing pain, as every patient is different,
with a different threshold and endpoint.
Whether one experiences chronic or acute
pain, Mr. Jones explained “it is important
to stay in tune with quality of life.” Chronic
pain is defined as lasting more than three
months and can include sleep disturbances,
and changes in appetite, activity and mood,
however, overt physiological symptoms are
often absent. Acute pain is sudden, sharp
and localized, and results in increased heart
rate, sweat, pallor, and increasing anxiety.
The Joint Commission on Accreditation of
Healthcare Organizations (JACHO) 2000
Standards for Pain Management considers
pain “a fifth vital sign,” and decrees,
“patients have the right to appropriate
assessment and management of pain.” In
2013, Haverman, et al, reviewed databases
from 1995 – 2012 and found 50% of patients
communicated about pain with their medical
team, and 25% communicated with their
friends or relatives. The transition from
acute to chronic
pain seemed to
occur around three
years of age. A 2008
study found that
abdominal pain is
caused by a myriad
of triggers including
constipation, irritable bowel disease,
enteric pathogens,
pancreatitis, peptic
ulcer disease, and
biliary issues.
Another study
found that over
70% of adults,
irrespective of
clinical status,
reported pain in
one or more areas
that interfered with
important physiotherapy treatments.
In children and adolescents with CF, the
effect of recurrent pain on their quality of life
significantly reduced physical functioning,
vitality, and overall perception of their
health. While these children had reduced
pulmonary function, their pain had a greater
impact on their quality of life. A 2014 study
at one CF center assessed the prevalence
of depression and identified risk factors in
children with CF. Of 190 patients, 9% had a
documented diagnosis of depression and
50% were on antidepressant medications.
“One thing led to another led to another,”
Continued on page 18
17
Pain Management
with breathing techniques, music therapy,
healing touch, guided imagery, and hypnosis. Pharmacological treatments are the
second line of defense, and include aspirin,
ibuprofen, naproxen, or acetaminophen,
followed by narcotics if necessary.
“Narcotics focus on the central brain and
have side effects such as constipation and
dependence.” Opioids might be required for
acute and chronic pain in severe advanced
illness and lung transplant.
Continued from page 17
Mr. Jones said, adding, “psychological
and emotional components are key in
pain assessment and management.”
Knowing that pain exists in CF, Mr. Jones
talked about pain scales, which are
designed as either uni-dimensional (i.e.
only one sensory component) or multidimensional, which are useful in looking at
the characteristics and impact of persistent
pain conditions when additional and more
in-depth information is needed.
Management of pain in CF “is a balancing
act.” For acute pain, Mr. Jones recommended non-pharmacological methods to
treat and control any underlying secondary
condition first, including therapeutic play
for children, distraction, relaxation along
Research for Living
Continued from front cover
underlying chloride channel defect in 4% of
the CF population. The recent characterization and establishment of novel cell
models for CF, such as CF epithelial cell
lines, conditionally reprogrammed CF cells,
and induced pluripotent stem cells will
continue to advance cell-based therapies
and personalized medicine approaches for
CF patients. Recently, I have focused on the
discovery of a multi-component bacterial
defense mechanism that does not work
properly in CF airway epithelial cells, and
our laboratory is exploring the idea that
CFTR and its partner in crime, bicarbonate,
are involved with the CF airways’ weak
natural defense. I am excited to study
this antimicrobial defense system and
test its utility to combat bacterial and
fungal infections, as it holds promise to
delay the onset of chronic infections in CF
children. The 2010 Annual Report of the CF
Foundation Patient Registry found that lung
infections occur in 40% of children with CF
ages 6 to 10, and 60% of teenagers up to
age 17. This new line of research is timely
and critical because it may translate to
the clinic and improve early treatment of
bacterial infections in young CF patients.
Personally, CFRI’s annual National Cystic
Fibrosis Family Education Conference,
which brings CF health care professionals
and research scientists together with
18
Management of pain is a balancing act.
In concluding, Mr. Jones listed important
“remember points” to consider when
managing pain. “Use appropriate
assessment tools, don’t under medicate,
remember there is no one ideal medication,
and consider the patient’s developmental
level, especially for children. We want to
keep everyone pain free.”
CF patients and
their families, has
taught me what it
means to live with
CF, and has put
faces behind the
disease. Based on
this, I developed
a passion to
train the next
generation of CF
scientists. In 2005,
I established the
summer student
program at CHORI
in partnership
with the Elizabeth
Nash Foundation,
to provide oneon-one training
and hands-on lab
experience in CF
research. To
Beate Illek, PhD, with fellow researcher Horst Fischer, PhD, and two
date, 16 college
participants in the 2013 CF Summer Research Program.
students have
participated, several of whom are personally to the SF Bay Area and matching them
impacted by CF. I am also honored to serve with the most promising collaborative
on CFRI’s Research Advisory Committee
CF-related projects. Thus far, 19 research
and as the Fellowship Administrator for
scientists and 32 fellows have participated
their Elizabeth Nash Memorial Fellowship in more than 30 CF research projects.
Program. Founded by the brilliant and
Thanks to CFRI’s research support, over
visionary Dr. Jonathan Widdicombe, the
200 publications have been produced.
program serves as an incubator to grow
CFRI brings hope of a cure for CF.
a new generation of CF scientists, by
Together we can do it!
attracting the best postdoctoral fellows
Research for living ~ Partners for life.
cfri | Fall 2014
CFRI’s CF Research
Challenge
Fund A Success!
Three New Projects Funded
Thanks to the Leadership
of the 2014 CF Research
Challenge Circle Donors
CFRI wishes to warmly acknowledge the
members of our 2014 CF Research Challenge
Circle, who generously contributed
$75,000 so as to match – dollar for dollar –
donations to our CF Research Challenge
Fund. Thanks to their generosity, and that
of our CF Research Challenge donors,
$150,000 will be designated for three
additional cystic fibrosis research projects
in 2014-2015.
For those of you who are inspired by the
leadership shown by our Circle members,
please join us! Together, we can support
the innovative research that will lead us to
new treatments, and ultimately, a cure.
2014 CF Research
Challenge Circle
Honorary Co-Chairs
Jim and Barbara Curry
Paul Quinton, PhD
Francine and Joel Bion
Dave and Kaye Cartnal
CFRI Board of Directors
Crossfit Ridgecrest Benefit
Suzanne Freiley
Insight Safari
Carroll P. Jenkins
Katie Knipp
Marc and Sue Landgraf
Fred and Linda Milanovich
Doug and Robin Modlin
Elizabeth Nash Foundation
David and Marcella Soohoo
Tim and Ann van Oppen
Vertex Pharmaceuticals, Inc.
Sandra K. Wilson Estate
Zumbathon Benefit
Christian and Darnelle Zimmerman
For information on how you can join
our 2015 CF Research Challenge Circle
please contact Sue Landgraf,
Executive Director, at 650.404.9975.
cfri | Fall 2014
Help Us Pursue Our Mission
Together We Can Provide
Support for Living and Research for Life
• DONATE TO THE 2015 CF RESEARCH CHALLENGE CIRCLE CFRI
has launched the CF Research Challenge Fund to raise funds to support
additional CF research projects. Become a member of the CF Research
Challenge Circle, and your gift will be used to encourage and match gifts
from donors and will be restricted to research programs.
• 40 Years – 40 Events: HOLD YOUR OWN FUNDRAISER Big or small,
we appreciate them all! Zumbathons, concerts, cupcake sales, dinner
parties with a special guest of honor, bocce ball tournaments – come up
with an idea and we will support you!
• MOTHERS’ DAY TEA Our Mothers’ Day Tea continues to be CFRI’s
largest fundraiser. Because every day is Mother’s Day, we have expanded
our fundraiser to take place year-round. Please contact us if you would
like to become a Tea Sender. For those who wish to use the Internet to
reach out to friends, colleagues and family members, you may also take
advantage of our Virtual Mothers’ Day Tea. It is fast, easy and fun!
• TRIBUTES IN HONOR OF, AND IN MEMORY OF Any gift to CFRI can
be made in honor or in memory of a loved one. Your loved one’s name
will appear in our newsletter, CFRI Community, and if requested, an
acknowledgement will be sent to the person you designate.
• GIVING GIFTS OF STOCK TO CFRI Giving a gift of appreciated stock
to CFRI is easy and rewarding. You will not pay capital gains tax on stock
that has appreciated over the years. You will receive an income tax
charitable deduction for the fair market value of the stock on the date
of the gift. If you hold stock certificates that you wish to donate to CFRI,
contact us for instructions on how to complete the transaction.
• CHARITABLE PLANNED GIVING Planned giving offers benefits for
donors that often include increased income and substantial tax savings,
while providing the opportunity to meet your philanthropic goals and
provide positive tax benefits.
• ATTEND A CFRI FUNDRAISING EVENT Whether you want to golf,
wine taste, or mingle while bidding on exclusive auction items, we have
an event for you! Upcoming events include:
— Wine for a Cure, a food and wine extravaganza at the Purple Orchid
Wine Country Resort and Spa in the Livermore Wine Country,
on October 19th, 2014.
— Sixty-Five Roses Benefit Concert with Tess Dunn and Special Guests at
Moe’s Alley, Santa Cruz, on December 14, 2014.
• VEHICLE DONATIONS If you have a car, boat, recreational vehicle,
or motorcycle that you no longer need, please consider donating
your vehicle to CFRI. This contribution is tax-deductible, and we will
coordinate the transfer of property. Visit our website for details on
making a donation.
For more information please contact
Mary Convento at 650-404-9975 or mconvento@cfri.org
19
Cystic Fibrosis Quality Of Life Program
Draws Focus To The Body-Mind Connection
By Meg Dvorak, LCSW
“If you are only treating the body,
you are treating half of the patient.”
– Norman Cousins, 1989
The Cystic Fibrosis Quality of Life (CFQofL)
Program: A Living Legacy of Peter Judge
was developed in 2008 to expand awareness of the body-mind interconnection in
chronic disease and to encourage treatment
for both. The CFQofL Program Advisory
Board, chaired by Dr. Richard Moss,
Professor Emeritus of Pediatrics at the
Stanford School of Medicine, undertook an
extensive literature review of the bodymind interconnection. The review, primarily
gleaned from research projects undertaken
by specialists in cancer and other chronic
disease, showed that improvement in the
psychological, social and spiritual wellbeing of patients improves their physical
condition, quality of life, and in some
situations, survival.
With the wealth of information available
from these studies, as well as ongoing
studies specific to CF, the CFQofL Program
brought together a consortium of chronic
disease specialists, professional caregivers,
patients, and family members to work
collaboratively on a variety of projects, with
the goal of educating our professional and
Connecting mind, body and spirit
lay communities on the need to include
psychosocial and spiritual counseling in
the overall treatment for cystic fibrosis.
The board has partnerships with the
Stanford School of Medicine at the Center
20
Psychological and social well-being is linked to improved health and quality of life.
for Education in Family and Community
Medicine, the Community Center for Health
and Wellness at Sofia University, and CFRI to
promote awareness and develop programs
to help CF patients.
with transportation or health barriers.
The CCHW also offers a monthly support
group for caregivers of CF patients. These
programs are currently under-utilized,
and CCHW hopes that more patients in the
SF Bay Area will take advantage of these
One such program, developed and managed
opportunities for support.
by the CFQofL Program, offers a sliding scale
for individualized counseling services at the Another of the board’s projects is The
Community Center for Health and Wellness Peter Judge Memorial Fund, established
(CCHW) in Palo Alto, California. The CCHW and maintained to assist adults with CF
counselors are
to pay for costs associated with getting
trained by
to Stanford hospital for care, including
experts in the
gas, tolls, meals, and lodging. For more
CF community
information about the fund, please contact
(including
Meg Dvorak at (650) 518-9976.
Dr. Moss and
Editors’ note: While the programs
others from the
Stanford teams) discussed above were created to serve
the cystic fibrosis community residing near
to understand
the Stanford CF Center in California, they
the complex
serve as an excellent model of effective
psychosocial
issues associated collaboration between cystic fibrosis
centers and community organizations to
with cystic
meet the needs of those with CF. There is
fibrosis – stress,
grief, depression, a vital need for programs that address the
psychological, social and spiritual well-being
body image,
of cystic fibrosis patients, which have been
insecurity,
documented to improve their physical
insurance and finances, problems with
health and quality of life. It is our hope
relationships, and addiction. No one in
that Ms. Dvorak’s article inspires others to
need of counseling is turned away due
to lack of funds, and counseling sessions explore the possibility of duplicating this
impactful model in their communities.
may be conducted by phone for those
cfri | Fall 2014
The 30th Annual Cystic Fibrosis Benefit Tournament
Raises over $60,000 for CFRI
The 30th annual Cystic Fibrosis Benefit
Tournament, held on August 4th, at the
illustrious Pasatiempo Golf Club in Santa
Cruz, California, raised over $60,000 to
support CFRI’s vital research and programs.
Spearheaded by Scott Hoyt, father of two
daughters with cystic fibrosis (CF) and
manager of Pasatiempo Golf Club, the
tournament brought together 170 people
committed to supporting the search for a
cure. “Many people associated with the
tournament deal with the disease on a daily
basis, and we are all so thankful for the
caring support,” Scott expressed.
One of the largest groups in attendance,
“Team Becca – Becca’s Bro’s,” was there
in support of Becca Roanhaus, daughter
of long-time CFRI Board member Mike
Roanhaus, and included many of Mike’s
fraternity brothers from college. Major
sponsors of the event included the Mike
and Dea Roanhaus family, HDR Architecture,
Star One Credit Union, the Kirkorian Family
Foundation, and the Scott and Anne
Hoyt family.
Becca Roanhaus surrounded by members of Team Becca, who golfed and raised funds in her honor.
Scott shared how meaningful it was to have
people with CF participating at the tournament and banquet. “It was very special, as
tournament participants see the impact
of their support. The life expectancy for
those diagnosed with CF has more than
doubled since the start of this benefit golf
tournament, and the quality of life for those
with the disease has drastically improved
due to the advancements in treatment
and medications.” The 31st Annual Cystic Fibrosis Benefit
Tournament will be held next summer on
August 3rd, 2015. Mark your calendars! In
addition to golfing at a “top-100” course,
you will help bring hope to those living
with cystic fibrosis.
Ask the Experts Panel: A Rare Opportunity
A highlight for many conference attendees
at our annual National Cystic Fibrosis
Family Education Conference is the “Ask
the Experts” panel, in which conference
speakers make themselves available to the
audience for 90 minutes to answer a wide
range of unscripted questions. This year’s
cfri | Fall 2014
moderator was CFRI’s Siri Vaeth Dunn,
MSW, the mother of a young adult with
CF. Participants on the panel included
(from left to right): Ahmet Uluer, MD, DO;
Katherine Van Loon, MD, MPH; Stephen
Jones, MS, RN, CPNP; Thomas Keens, MD;
Marty Kharrazi, PhD, MPH; and Isabel
Stenzel Byrnes, MSW, MPH. Expanding its
impact, the session was live streamed to
the online cystic fibrosis community, and
viewers were able to email their questions
to be asked in real time. The recording of
this informative panel will be posted on
CFRI’s website, www.cfri.org
21
27th National Cystic Fibrosis
Family Education Conference
The Changing Faces of Cystic Fibrosis:
Inspiring Hope
DVD Order Form
Presentations from CFRI’s 27th National Cystic Fibrosis Family Education conference are available on DVD format.
$5.00 for each disk and $20.00 for all 4 disks.
Order
Disk Topics / Speakers
D1
Mary ElizaBeth Peters, Theatre Artist and CF Educator, Boston, MA
More than Survival: Pursuit of Healthiness
D1
Thomas Keens, MD, Pediatric Pulmonology, Children’s Hospital Los Angeles, Los Angeles, CA
D2
Ahmet Z. Uluer, MD, DO, Division of Pulmonary and Respiratory Diseases, Boston Children’s Hospital, Boston, MA
D2
Antionette Moran, MD, Division of Pediatric Endocrinology Medical School, University of Minnesota, Minneapolis, MN
D2
The Resilient Character: Adapting Well To Changes in Our CF
Isabel Stenzel Byrnes, MSW, MPH, Social Worker, San Francisco Bay Area
D3
Beyond the Ouch Factor: Pain Management in CF
Stephen Jones, MS, RN, PNP, Children’s Hospital, Albany Medical Center, Albany, NY
D3
CF and Cancer: Who Should Be Concerned and What Can Be Done
Katherine Van Loon, MD, MPH, Gastrointestinal Surgery and Oncology Clinic, UCSF Medical Center, San Francisco, CA
D3
Reflections on Detection of CF and CFTR-Related Metabolic Syndrome (CRMS) in California Newborn Screening
Martin Kharrazi, PhD, MPH, California Department of Public Health, Richmond, CA
D4
Tips For Living an Extraordinary Life | Adults With CF Panel Discussion
Carroll Jenkins, MA (Moderator)
D4
Ask the Experts (Panel Discussion with Conference Speakers)
Thomas Keens, MD; Ahmet Uluer, MD, DO; Katherine Van Loon, MD, MPH; Martin Kharrazi, PhD, MPH;
Stephen Jones, MS, RN, PNP; and Isabel Stenzel Byrnes, MSW, MPH – Siri Vaeth Dunn, MSW (Moderator)
Where We Have Been and Where We Are Going
The Therapeutic Pipeline: CF Research Updates
The Sweet Evolution of CF-Related Diabetes (CFRD)
Send your order to: Cystic Fibrosis Research, Inc. (CFRI), 1731 Embarcadero Road, Suite 210, Palo Alto, CA 94303, USA
Fax: 650.404.9981, Phone: 1.855.cfri.now or order online at www.cfri.org. Thank you!
Name
Address
City
Phone
Total Number of Disks
22
ZIP
E-mail
Payment Method:
Credit Card Type / Number
Signature
State
Check
Credit
Cash Total Amount $
Expiration date
CF Relationship
cfri | Fall 2014
Thank You for
Your End-of-Year Support!
As autumn begins, and the end of the year draws nearer, many of us
begin to plan our year-end gifts to those charitable organizations that
are important to us. We hope you will consider CFRI when making
these decisions.
We are fortunate to have a dedicated and caring community that
believes in the work of CFRI and supports our mission to fund research,
provide educational and personal support, and to spread awareness
of cystic fibrosis.
Your end-of-year gift is greatly appreciated and central to the work we
do. This year you might think of giving through:
• the enclosed CFRI Community remit envelope
• a donation of stocks or other marketable securities
• our online donation program at www.cfri.org
• your company’s Workplace Giving program
• a vehicle donation
• the CFRI Special Gifts letter coming to you in December
• participation as a 2015 CF Research Challenge Circle Donor
Whatever you are able to give is greatly appreciated. Depending on
the type of gift, you may receive additional tax deductions. It is always
prudent to review your charitable giving with a financial advisor. Thank
you for your support this year, and for your continued commitment to
CFRI’s mission in 2015!
For more information, please contact Sue Landgraf,
Executive Director at 650.404.9975.
40
ars – For ty Eve
e
Y
y
nt s
rt
o
F
Stay Tuned!
In 2015, CFRI will celebrate 40 years of supporting
innovative research and services to the cystic fibrosis
(CF) community. In honor of this auspicious anniversary,
CFRI will soon launch an exciting “40 Years – 40 Events”
project. We hope to inspire a minimum of 40 separate
fundraising events across the country with CFRI as the
beneficiary. Please join us! The ideas are endless, including
concerts, bowl-a-thons, cook-offs, 10k runs, auctions,
car washes, and more! What’s your idea? Imagine the CF
research, education and support services we could fund
with this collective fundraiser. To get involved, please
email cfri@cfri.org, or call 650-404-9975.
cfri | Fall 2014
Save the Dates
CFRI’s Discovery Series
“Taking CF to College”
Cindy Marota
Director, Accessible Education Center
San Jose State University
Tuesday, October 14, 2014
6:00 pm – 7:30 pm
Crowne Plaza Hotel Palo Alto
CFRI’s Wine for a Cure
Sunday, October 19, 2014
Purple Orchid Wine Country Resort & Spa
in the wine country of Livermore, CA
Spectacular wine, food, music, auction, &
celebrities: A fundraiser for CFRI’s
research, education and support programs!
Book Signing
With Isabel Stenzel Byrnes:
The Power of Two Second Edition
Thursday, October 23, 2014
7:00 pm
Books Inc. (Town & Country Village)
855 El Camino Real, Palo Alto, CA
CFRI’s Discovery Series
“Gastrointestinal Challenges
in Cystic Fibrosis: Enzymes, Laxatives, and Beyond”
Julie Matel, MS, RD, CDE
Lucile Packard Children’s Hospital Stanford
Tuesday, November 11, 2014
6:00 pm – 7:30 pm
Crowne Plaza Hotel, Palo Alto, CA
Sixty-Five Roses Concert Event
With Tess Dunn & Special Guests
Music, Auction, Tacos, & More, Benefitting CFRI
Sunday, December 14, 2014
7:00 pm
Moe’s Alley, Santa Cruz, CA
www.moesalley.com
For more information about any of these events,
please call 1.855.cfri.now (1.855.237.4699)
or email cfri@cfri.org
23
Nonprofit
Organization
U.S. Postage
PAID
Palo Alto, CA
1731 Embarcadero Road . Suite 210
Palo Alto, California 94303
Permit #24
Change Service Requested
CFRI’s mailing list is confidential. We do not sell our list, nor do we give out any names or addresses under any circumstance.
CFRI Mission
Cystic Fibrosis Research, Inc.
exists to fund research,
to provide education and
personal support, and
to spread awareness of
cystic fibrosis,
a life-threatening
genetic disease.
CFRI Vision
As we work to find a cure
for cystic fibrosis,
CFRI envisions
informing, engaging
and empowering
the CF community to
help all who have this
challenging disease attain
the highest possible
quality of life.
Visit our website at:
www.cfri.org
for more information about us
and about cystic fibrosis.
Wine for a Cure – Please Join Us On October 19!
Wine for a Cure promises to be a crowd
pleaser. Held at the gorgeous Purple
Orchid Wine Country Resort and Spa in
Livermore, the event will bring together
some of the most notable vintners from
the region, including Steven Kent, La
Rochelle, McGrail Vineyards, Bodegas
Aguirre, and De Fina Family Cellars.
Bountiful gourmet food will be provided
by On the Vine, while live and silent
auctions, complete with jewelry, sports
tickets and luxury vacation packages,
will tempt guests to bid for a cure.
Special celebrities will be in attendance,
including Hollywood actor Todd
Purple Orchid Wine Country Resort and Spa
Giebenhain, who lives with CF; San
Francisco 49er great Bill Ring; drummer
Professor and Director of Stanford Medical
Center’s Pediatric Cystic Fibrosis Center, in
and percussionist Adam Topol; and singerrecognition of his inspirational dedication to
songwriter Tess Dunn, who lives with CF.
providing the highest quality of care to children
The event will be held on Sunday, October
and teens living with cystic fibrosis.
19, from 4:00 to 7:00 pm, with a special VIP
Generously sponsored by Vertex Pharmaceuticals,
reception from 3:00 to 4:00 pm, featuring
library wines from the Steven Kent portfolio,
AbbVie, Chiesi USA, Inc., and the Pleasanton
Lions Club, Wine for a Cure will support CFRI’s
the opportunity to mingle with event
vital programs, bringing hope to those living
celebrities, and an intimate performance by
recording artists, Adam Topol and Tess Dunn.
with cystic fibrosis.
The Caregiver of the Year Award will be
presented to Dr. Carlos Milla, Associate
To purchase tickets online, please visit:
www.cfri.org/wineforacure.shtml
For their generous support of CFRI Community, special thanks to:
Chiesi USA, Inc. • Genentech
Call toll free: 1.855.cfri.now
Cystic Fibrosis Research, Inc. a 501(c)(3) nonprofit organization Federal EIN# 51-0169988