40 Years of Research,
Education & Support
Summer 2015
The Tribulations and Triumphs of Transplant:
Maggie Williamson’s Journey
By Maggie Williamson and Siri Vaeth Dunn, MSW
Maggie Williamson was diagnosed with cystic fibrosis (CF) at 13
months old. In the spring of 2013, Maggie was 24 years old,
married and living in California, when she experienced a severe
downturn in her health. She was listed for transplant, and
nearly 13 months later, received the gift of new lungs. In a
recent interview, Maggie spoke candidly about the unexpected
tribulations and powerful triumphs post-transplant.
Tell me about the weeks prior to transplant.
That was my toughest month. I was not working or in school; I
only concentrated on my health. I got extremely sick and was put
on full-time oxygen. I pretty much did nothing – even driving and
running errands were too hard. I started ordering groceries online
and the rare times Tom and I went out, I used a wheelchair. It
was very hard for Tom to watch me deteriorate. What was it like to take your first breath with your new lungs?
I never had that “aha” moment with my breath, as my lungs
were slow to expand, but other physical changes brought tears
to my eyes. Seeing my oxygen saturation reach 100 percent
two days after surgery was a moment I won’t forget. I marveled
at walking down the hospital hallway ten days out of surgery
and standing tall for the first time after years of hunching over
due to heavy breathing. The first time I looked in the mirror and
saw the huge scar running down my chest I burst into tears,
recognizing my life had changed for good. It was such a joyful
Maggie WIlliamson canoeing on her 27th birthday, five months post transplant
moment to know I was given the ultimate gift of life because of my
donor. It was a profoundly grateful moment. Tell me about Tom and your journey as a couple.
I met Tom in 2009, when I was 21. I was more independent in our first
three years, but during the last three, Tom has had a much bigger
Continued on page 4
Measuring CFTR Function in Humans
with a Simple Sweat Test
By Jeffrey Wine, PhD
Jeffrey Wine, PhD
As everyone in the cystic fibrosis (CF) community knows, CF treatment is being revolutionized.
For the first time, defective CFTR molecules, the root cause of all CF symptoms, can be
targeted directly. Remarkably, the new medicines are easily taken daily pills that fix CFTR
in all parts of the body – not only the lungs. Ivacaftor (Kalydeco) is the first such medicine;
better follow-on drugs are being developed. These exciting research breakthroughs have
created a huge surge of hope, but also great impatience to get these life-saving drugs
as soon as possible. Drug discovery is accelerating because innovations such as robotic
testing allow millions of compounds to be screened on cells grown in the laboratory. But
it is still necessary to test potential drugs in humans to look for side effects and to see how
well they work.
Continued on page 10
CFRI Community
Summer 2015
Editorial Chief
Sue Landgraf
Editors
Bridget Barnes
Siri Vaeth Dunn, MSW
Contributing Writers
Bridget Barnes
Isa Stenzel Byrnes, MSW, MPH
Mary Convento
Siri Vaeth Dunn, MSW
Bill Hult
Sue Landgraf
John Mark, MD
Jane Mitchell
Maggie Williamson
Jeffrey Wine, PhD
Layout and Design
Marina Michaelian Ward
Board of Directors
Bill Hult, President
Jessica Martens, Vice President
Mike Roanhaus, Secretary
Oscar Flamenco, CPA, Treasurer
Brad Baugh, JD
Francine Bion
Elyse Elconin-Goldberg
Doug Modlin, PhD
Kristin Shelton, RRT
Executive Director
Sue Landgraf
Programs & Outreach
Manager
Siri Vaeth Dunn, MSW
Programs & Outreach
Supervisor
Mary Convento
Letter from the
Executive Director
“Alone we can do so little, together we can do so much..”
Dear Friends,
— Helen Keller
What an exciting year! CFRI has experienced a period of unprecedented growth in every sense of the word. In addition to
increased research funding, we have launched numerous new
programs, including the Partners in Living Initiative, which offers
wonderful support to our community. The successful Pediatric
Conference and Mindfulness Based Stress Reduction sessions Sue Landgraf
have concluded, but a Caregivers Support Group and counseling
services are available to individuals nationwide through the Cystic Fibrosis Quality of Life Program,
A Living Legacy of Peter Judge. I strongly encourage those touched by cystic fibrosis (CF) to take
advantage of these resources. They will strengthen you, and in turn strengthen our community.
We are gearing up for the 28th National Cystic Fibrosis Family Education Conference, which will
be held July 31 to August 2, 2015 in Redwood City, California. Some of the most noted experts in
the field will travel from New York, Ontario (Canada), Washington, New Jersey, and throughout
California to address topics including the CF pipeline, mental health and adherence, transition, and
the treatment of emerging pathogens. We are shifting the schedule this year to expand access
to this vital information. The conference will conclude on Sunday with a new sit-down luncheon
featuring an exciting multi-media presentation from a dynamic member of the CF community,
as well as a wide array of support groups, yoga, art, and journaling workshops. I look forward to
seeing everyone there.
I also extend a warm invitation to adults with CF and their friends and families to attend the CF
Summer Retreat, “Animate Your Summer!,” from August 2 to August 9, 2015 at Vallombrosa in
Menlo Park, California. Thanks to CFRI’s stringent infection control policies, those with CF have a
rare opportunity to come together for educational presentations, camaraderie, support, and fun.
We have so much to accomplish together as a community. We are still seeking the elusive cure
for our loved ones with CF, and eagerly await the arrival of powerful new drugs that treat the
underlying defect in those living with the double delta F508 mutation. While I believe that alone
each of us can achieve important goals, I agree with Helen Keller’s observation that, “together we
can do so much.” I am delighted to do so much in partnership with you.
Programs & Operations
Associate
Warmly,
Programs & Operations
Assistant
Sue Landgraf | Executive Director and mother of an adult daughter with CF
Scott Wakefield
France Redmayne
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
News from the Board
Dear CFRI Community,
I hope that this finds you well. As we approach the halfway
mark for 2015, I am pleased to share that CFRI is continuing
the growth trend that began in 2014. Our recently
completed audit shows that we are fiscally strong. In fact,
due to the success of our fundraising efforts last year, our
revenue growth in 2014 exceeded our 2013 total by nearly
34%, allowing us to provide new education and support
programs and increase our research awards. We provided
a second year of funding to five CF researchers from 2013
Phone: Toll Free 1.855.cfri.now
(1.855.237.4669)
Fax: 650.404.9981
Email: cfri@cfri.org
Website: www.cfri.org
With our permission, you may
reproduce original information
from this newsletter with credits
to CFRI Community and the author.
2
Bill Hult, President
Continued on page 3
cfri | Summer 2015
Cystic Fibrosis and Complementary and Alternative Medicine:
An Integrative/Holistic Approach
John D. Mark, MD
For those with cystic fibrosis (CF), the current
system of conventional healthcare can be
enormously frustrating. Rather than simply
keeping their symptoms in check, many
people with CF seek to reclaim wellbeing and
vitality. The holistic approach does not reject
conventional medicine, but considers the
entire picture of health and uses the best
and most appropriate options for healing.
Complementary and alternative medicine
(CAM) is increasingly used by patients with
CF. CAM covers a broad range of healing
philosophies, approaches and therapies
that can be used alone, in combination
with other alternative therapies, or along
with conventional medicine.
The major classes of CAM include alternative
medical systems such as homeopathic,
naturopathic, traditional Chinese, and
Ayurvedic medicine. Mind-body medicine
incorporates interventions that enhance the
body’s capacity to affect bodily functions
and symptoms. Biologically based therapies
include dietary supplements, foods, herbs,
vitamins, and natural substances. Manipulative and body-based methods include
osteopathy, chiropractic and massage.
Energy therapies include biofield therapies
and bio-electromagnetic therapy.
Complementary and alternative therapies can be very helpful to those with CF
followed by mind-body medicine (49%) and
manipulative therapies (14.5%).
As a starting point, making lifestyle choices
that include avoidance of allergens, tobacco
smoke and air irritants, good sleep hygiene,
and regular exercise can have a positive
impact upon one’s health. Exercise is a
valuable tool for CF patients as it often
stimulates coughing, thereby helping
with airway clearance while improving
cardiovascular health. When planning one’s
In a study of CAM use in children and adults
exercise routine, seek advice from your CF
with CF, 75% of patients used some form of
care team.
alternative medicine, and over two-thirds
of the respondents felt CAM was useful.
Nutrition is vitally important to CF health.
The majority (53%) used biologically based Because diet is the major source of antitherapies such as dietary supplements,
oxidants, patients with cystic fibrosis should
maintain a high calorie, high protein diet,
while increasing their consumption of fruits
and vegetables, omega-3 fatty acids, and
other antioxidant-rich foods including fish,
while decreasing omega-6 fatty acid intake.
Mind-body therapies or cognitive behavioral
therapies include relaxation therapy, breathing exercises, biofeedback, hypnosis, and
guided imagery, and are based on the theory
that the nervous system’s response to stress
and strong emotions increases cytokine
levels that are contributors to airway
inflammation. Self hypnosis, breathing
exercises, yoga, and relaxation techniques
have all been studied in CF and indicate that
Continued on page 5
News from the Board
Continued from page 2
and funded six new CF researchers in 2014, stability as well as its flexibility to respond
bringing our total to 11 researchers in 2014 to our CF community’s emerging needs.
– an increase of four.
Last year the Board of Directors partiThe credit for this must be shared amongst cipated in an in-depth strategic planning
process. A summary of the resulting
our entire CFRI community, from donors,
to corporate and foundation funders, to plan is included in the attached Annual
Report. I encourage you to read this
members of the Board, and our agency
document, which will help to guide our
staff. We are fortunate to have the
organizational leadership of Sue Landgraf, actions in the year ahead as we continue
to embody our tagline, “Research for
and I can assure you that the Board of
Directors is fully committed to both CFRI’s Living ~ Partners for Life.” You are a part
cfri | Summer 2015
of this strategic plan. Every member of
our community has a role to play. I thank
you for your engagement and support,
as we seek to improve and enhance the
lives of those with cystic fibrosis.
Peace and good health,
Bill Hult | Board President
3
The Tribulations and Triumphs of Transplant
Continued from front cover
role in taking care of me. My mom flew in from Chicago to help
out for the first two months post transplant, and my dad and
brother came out to help for as long as their work allowed, but
since then it has been all on Tom. As most parents know, being
a caregiver is extremely hard. Going through something as major
as a transplant takes its toll on everyone. We talk a lot about how
our situation is not a “normal” couple situation. This journey has
been difficult. I don’t sugar coat it because I think more couples
should speak out about the challenges chronic illness brings to
their relationship. How hard was the recovery?
The initial recovery was almost too easy. For me, the hard work
came when I was discharged. Going in for hospital appointments
three to four days a week started to take its toll and became
exhausting and overwhelming. Things have settled a bit, but I
would be okay with not seeing a hospital for a few months.
What was unexpected post transplant?
We have this great miracle, but a lot of new problems come with
it. I was an expert in my CF, but still don’t have much of a clue
about this new disease and body that I live with. Many people
think the only concern is rejection of the lungs, but there are other
serious complications. In addition to a (now healed) wound and
blood infection, pneumothorax, and frightening virus, I have a
lot of stomach issues because nerves that help with digestion
were severed during surgery. People who don’t know much
about transplant perceive me as cured. This is far from a cure. It
is a way to prolong and improve my quality of life, but I still have
a chronic illness.
What have the greatest challenges been post transplant?
Learning a new normal. Rediscovering life. I was essentially reborn,
and am revisiting the developmental stages. In the beginning
I needed help with everything like a newborn. I feel like I’m in
my preteen phase now, testing limits and boundaries, gaining
independence, and trying to ignore the fact that I occasionally
still need support. Prednisone gives you a false sense of reality,
and I’ve come up with crazy ideas like wanting to rock climb
four months post-transplant. Most significantly, it has been a
very long time since I’ve had a focus besides my health, and I
feel a bit lost as to where I will go in life. Defining myself outside
of sickness will be my biggest challenge.
Maggie and Tom Williamson, soon afer her transplant.
What advice do you have for others who are pondering
when to get listed?
Listen to your gut. After I was approved to be listed I gave myself
an extra three months, and at that point my mind and body
were ready. Everyone is different so don’t read the bad stories
and think it will happen to you. This truly is each person’s unique
journey. And remember, you have nothing to lose. At end stage
disease there is nowhere to go but down, and transplant offers
the hope of a better quality of life. What has been a “wow” moment for you post transplant?
Dancing and singing! Also, the endurance I have. Being able to
take a shower, get dressed, and do my hair without having to
stop for long periods of time between each task. Who knew you
could wash your hair and shave your legs in the same showering
session!? Tom and I are moving to England in the near future –
something that was unimaginable just a few months ago.
Oftentimes it hits me that these are not my lungs. That will hit me
like a ton of bricks some days, and it just brings me to a quiet place
of sheer gratitude. I am living because of someone else. That is
the biggest “wow” moment that keeps coming back to me.
Funding Inspired CF Research
David Cornfield, MD
4
Carlos Milla, MD
CFRI’s Board of Directors has awarded research grant funding to two Stanford
physicians and their post-doctoral research fellows through CFRI’s Elizabeth
Nash Memorial Fellowship Program. Principal Investigator, David Cornfield, MD,
Professor of Pulmonary Medicine and Chief, Pulmonary and Critical Care Medicine
at Lucile Packard Children’s Hospital Stanford, along with his post doctoral fellow,
Michael Tracy, MD, have received a $40,000 grant for their CF research project, while
Carlos Milla, MD, Associate Professor of Pediatrics (Pulmonary Medicine) Stanford
University Medical School, and his post-doctoral fellow, Andrey Malkovskiy, PhD,
were granted $40,000 for their research project. CFRI is honored to support their
inspired research.
cfri | Summer 2015
Breakthrough Therapy for Most
Common CF Mutation Recommended
for Approval by FDA Subcommittee
By Bridget Barnes
In May, the Pulmonary-Allergy Drugs
Advisory Committee of the US Food and
Drug Administration (FDA) voted 12 to 1
in favor of recommending that the FDA
approve a single pill combining the drugs
Ivacaftor and Lumacaftor for the treatment
of cystic fibrosis in patients age 12 years and
older who have two copies of the F508del
mutation. Developed by Vertex Pharmaceuticals and targeting the root cause of the
disease, the new combination, called
“Orkambi,” combines Ivacaftor, a
“potentiator,” that improves the transport
of chloride ions across the cell membrane,
and Lumacaftor, a “corrector,” that moves
defective CFTR protein to the proper place in
the airway cell membrane and improves its
An Integrative/Holistic Approach
Continued from page 3
they may decrease shortness of breath and
chronic symptoms such as cough, and may
help with lung expansion.
While herbal supplements are the most
common CAM therapy reportedly used
by those with CF, their use is consistently
under-reported to CF physicians. This is
cause for concern, because some herbs
may have negative interactions with
conventional medications and other
supplements. Dietary supplements do
not need to meet the same safety and
effectiveness standards as pharmaceutical
medications, and there are reports of some
supplements being contaminated with
metals, unlabeled prescription drugs, and
microorganisms, or containing none of the
herbs listed on their labels.
Studies of antioxidants such as Vitamin
C, selenium, and fish oil/omega 3 fatty
acids used by those with CF have had
variable results. Creatine has been studied
in CF and shown to improve short-term
muscle strength. Other beneficial dietary
supplements for CF include vitamins A,
B, D, and E, calcium, magnesium, ginger,
gingko, garlic, lipoic acid, baker’s yeast,
green tea, and ginseng. Probiotics are
beneficial for those with CF, as they may
cfri | Summer 2015
function as a chloride channel. Phase 3 clinical
trials were conducted at approximately 200
clinical trial sites in North America, Europe
and Australia, and more than 1,100 people
with two copies of the F508del mutation,
ages 12 and older, participated in the studies.
The trials tested two different doses of
Ivacaftor and Lumacaftor. Compared with
those on placebo, participants who took the
combination treatment showed statistically
significant and consistent improvement in
lung function and other important health
measures, including weight gain, and a reduction in the rate of pulmonary exacerbations. Sue Landgraf, CFRI’s executive director,
travelled to Maryland to address the
Pulmonary-Allergy Drugs Advisory Commit-
A Vertex scientist in the lab
tee during oral presentations, along with
members of the nationwide CF community. She noted that the testimony of those living
with the disease was extremely impactful,
adding, “The committee’s recommendation
for approval brings us one step closer to a
drug therapy that targets the double Delta
F508 mutation. For now, this is the closest
thing to a cure that we have. CFRI was
honored to be a part of this momentous
day.” The application will now go to the
FDA for approval, with an estimated
announcement by July 5th.
help improve intestinal barrier function
and modify the immune response. Taurine,
an essential amino acid, may improve fat
absorption and weight gain in CF, while
N-acetylcysteine (NAC) may soon be a
standard of care for the antioxidant properties it provides.
Manipulative therapies include massage,
osteopathic and chiropractic manipulation.
Small studies of children with CF and their
parents have found that massage therapy
led to a reduction in anxiety, improved mood
and increased peak flow measurements.
Traditional Chinese Medicine is based upon
the belief in an unseen vital energy, or “qi,”
that affects patients’ health. This energy’s
flow is monitored and manipulated using
acupuncture, Asian herbs, diet, and
physical therapy. One study at Boston’s
Children’s Hospital showed acupuncture to
be effective in decreasing pain complaints
in patients with CF. Homeopathic medicine
utilizes the “Law of Similars,” in which
small, highly diluted quantities of medicinal
substances are given to cure symptoms,
when the same substances given at
higher or more concentrated doses would
actually cause those symptoms.
In order to put all the pieces of one’s CF
care together, it is useful to develop an
John D. Mark, MD
Integrative Medical Plan for CF, which
includes discussions about medications,
adherence, lifestyle changes including
exercise and diet, supplements (including
herbal remedies), and the use of Traditional
Chinese Medicine, manipulative therapies
and/or homeopathy. The most important
component is education and teamwork
in regard to all aspects of the healthcare
plan. If the CF medical team sets CAM in
opposition to conventional medicine, it
could create a barrier to optimal self-care
management. The use of complementary
and alternative medicine does not indicate
unrealistic expectations of a cure; rather,
it demonstrates a desire to have better
control and self-management of one’s
chronic condition.
5
The Jessica Fredrick
Memorial CF Research
Challenge Circle:
Supporting the
Search for a Cure in
Memory & In Honor
of Those with CF
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.
“Strength does not come from
physical capacity.
It comes from an indomitable will.”
— Gandhi
Jessica Fredrick
CFRI is very pleased to have broadened
its research funding in 2014, thanks to
members of our Jessica Fredrick Memorial
CF Research Challenge Circle, and the
generosity of those who contributed to the
fund. CFRI needs your help to continue this
forward momentum. Please join with us to
improve the lives of those battling cystic
fibrosis. The Research Challenge Circle has
set a goal to raise $80,000 in 2015, to be
used to match contributions designated for
research. Please consider joining the Circle,
or making a gift to the Research Challenge
Fund. Unless otherwise notified, all gifts
received through this newsletter will be
matched by the Circle and will be used to
fund vital CF research projects. Now is an
exciting time to give, as your gift to CFRI
will be matched 1-to-1 by members of
the Jessica Fredrick Memorial CF Research
Challenge Circle, thereby doubling the
impact of your donation!
Those with CF spend hours each day trying
to slow the inevitable progression of the
disease. In addition to lung disease, many
cope with liver issues, diabetes, pancreatic
insufficiency, and other complications. We
still have much more work to do to improve
and save the lives of our loved ones. But we
cannot do it without you. Please give today
and help us match the Circle’s fund so that
we can bring hope to those with CF and
their loved ones.
Working together, with an indomitable will,
we can inspire hope and find a cure.
To contribute to the Jessica Fredrick Memorial
CF Research Challenge Circle, please call
650.404.9975, or email cfri@cfri.org
6
CFRI Gives Thanks to Members
of the Jessica Fredrick Memorial
CF Research Challenge Circle
The Elizabeth Nash Foundation
Millward Brown, Inc.
Sereno Group of Willow Glen
Spiritus Project (Robert Link)
John and Sallie Best
Randall Colombo
Michael and Pat Flynn
Suzanne Freiley
Bill and Vicci Hult
Shannon Jorgenson
Sue and Marc Landgraf
Fred and Linda Milanovich
Doug and Robin Modlin
Frank S. Morsman, Jr.
Marvin and Bune Primack
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 Road
Suite 210
Palo Alto, CA 94303
CauseVox
Research
Campaign Hits
100% of Goal!
Thanks to our generous donors,
CFRI’s crowdfunding campaign
to raise $5,000 in support of
CF research was a success.
Contributions made through the
30-day campaign were matched
one-to-one by members of
CFRI’s Jessica Fredrick Memorial
CF Research Challenge Circle,
thereby doubling the impact of
each gift. CFRI is grateful to all who
supported this important campaign.
cfri | Summer 2015
January 1, 2015 — April 30, 2015
Henry Aulus Allison
Gianna R. Altano
Ginni Andres
David Armknecht
Jodi Armknecht
Ann M. Baldwin
Gloria Bellefleur
Anne C. Beltrame
Kristina Benson
Ezequiel Bondy-Villa
Lara Borowski
Wendy Bosarge
Alicia Brogle
Sonya Chartrand
Gail Christensen
Ryan Coelho
K. Cole
Robert Cowden
Rachel Crocker
Bette Dambacher
Charles Delgado
Neva L. DeVore
Steven Elconin
Jessica A. Fredrick
Mildred Fredrick
Jade Frota
R. M. Gabbard
John “Jack” Girvin
Jenise Giuliano
Lawrence “ Larry”
Goldberg
Diana Goodman
Heather E. Gottlieb
Bill Haig
Kathleen Halsey
Warren A. Harden
Sharon Hill
Sean Hyland
James A. Irwin
Jason Jean
Jordan Jean
Alec Jenkins
Kathy Judge
Peter Judge
Blake Kelly
Norman King
Lori Kipp
Bridget Klein
Kelly M. Knight
Raymond Kulik
Al Ladendorf
Dawn Longero
Justine M. Marocco
Jason Marshall
Jean Marshall
Jordan Marshall
Frances Martinelli
Nahara Mau
David McAfee
Juanita McDonald
Robert McFadden
Helen McGowan
Barbara A. McKee
Ronald McMullen
Arthur Messinger
Loretta Morris
Lee Nichols
Gretchen Nordlund
Thaddeus Novak
Michele D. Olson
Jennifer Ortman
Dellene Ott
Lisa Pearne
Paula Peterson
Barbra A. Petitt
Kevin Pira
Jon Prater
Timothy Prater
Richard Richards
Peter Riddle
Tom Rolefson
Edith Schlotterbeck
Joseph M. Sinnaeve
Tammy Smerber
Dawn Smith
Yolanda “Lindy”
Stagnaro Dunn
Anabel M. Stenzel
Robin Stephenson
Robert C. Stewart
Charlie Stockley
David Stuckert
Laurie Stuckert
Delinda Syme
Bill Tidball
Louis Trigueiro
Ralph Turner
Erik Van Ness
Tom Walton
Tara Weir
Dawn Wernli
Ray Wilkens
Sharon Williams
Kelly A. Wilson
Cynthia Witman
Hannah Mitchell
Anna Modlin
Kate Nelson
Jessica Nett
Scott Parks
Brie Peters
Jereme Pierce
Hollie Pratt
Robyn Primack
Paul Quinton
Pauline Richard
Rebecca Roanhaus
Kaeti Roberts
Ann Robinson
Elizabeth Rogers
Taylor Rolefson
Alanah Rosenbloom
Corey Sarkis
Lindsay Schwab
Ken Semel
Gina Serrato
Rachel Silver
Matt Spadia
Alma G. St. Lawrence
Rebecca Swank
Heidi Tegner Geyer
Adam Thompson
Katherine Thompson
Pina Toccagino
Carol D. Umber
Devin Wakefield
Scott Wakefield
Christopher Walton
Jason Webster
Pamela Whitehurst
Kareese Wilson
Jeff Wine
Nina Wine
January 1, 2015 — April 30, 2015
Chelsa Aboud
Sadie Anderson
Kyle Baker
Susie Baldwin
Bridget Barnes
Lucy Barnes
Brandon Begin
Aidan Biggar
Oliver Biggar
Erin Brassfield
Lauren Catron
Maureen Coffy
Michelle Compton
Barbara H. Curry
Cheri DeWilde
Tess Dunn
Jessica Eller
Daniel Ellett
Thomas Evans
cfri | Summer 2015
Emily Fredrick
Sean Gallagher
Mark E. Gerow
Larissa Giuliano
Mark Glisson
Elyse E. Goldberg
Jen Goodwin
Elsie Hamer
Alex Hampton
Liz Hampton
Kerry Harrison
George M. Hawley
Christian Heavner
Tyler Heavner
Kathleen G. Henshaw
Melanie Henshaw
James Heyboer
Shawn Hidy
Vincent Holmquist
Thomas Horal
Bill Hult
Deion Jefferson
Carroll P. Jenkins
Michelle Jones
Peggy B. Jones
Stan Kelly
Franny Kiles
Kristin Kovolinka
Santosh M. Krishnan
Daniel Lagasse
Douglas Lagasse
Kody Lawrence
Joseph Librers
Alyson Lowery
Emily F. Lucas
Larissa Marocco
Eric Marten
Carly L. McReynolds
7
CFRI’s Partners in Living Initiative:
Celebrating A Mind-Body Success
By Siri Vaeth Dunn, MSW
The Partners in Living Initiative – A Holistic
Approach to Life with Cystic Fibrosis (CF),
was designed to help members of the CF
community achieve optimal physical and
mental wellbeing, in recognition of their
significantly higher rates of depression
and anxiety, which if left unaddressed are
detrimental to overall health. Launched in
January 2015, the pilot six-month multidisciplinary initiative – funded by a $50,000
new program grant from Vertex Pharmaceuticals – successfully strengthened the
link between good mental health and a
better physical quality of life for those
who participated.
program continues until August,
or until all funds are expended.
CFRI’s monthly peer-to-peer support group for caregivers is held
in Palo Alto, California, but participants may phone in, whether they
are in California, New York, or states
in between. The support group is
held on the fourth Tuesday of each
month at 7:00 pm PST.
CFRI’s Mindfulness-Based Stress Reduction
(MBSR) course, led by Dr. Julie Desch,
combined meditation, body awareness
and yoga to help people living with CF –
including caregivers – cope with anxiety,
depression, stress, and chronic pain. People
A very successful component of the initiative
from across the nation participated in this
has been the CF Quality of Life Program, a
online class, and the number of enrollees
Living Legacy of Peter Judge, in which CFRI
far exceeded our goal.
covers the cost of individual counseling for
The last component of the Partners in Living
those with CF and their parents/caregivers,
regardless of where they live in the U.S. This Initiative was the offering of an exciting
NNIVERSARY G
S 40th A
’
ALA
I
R
CF
A MAGICAL EVENING at CAROLANDS
September 25, 2015 | 7:00 pm – 10:00 pm
CF Pediatric Education Conference in May.
Spanish interpretation was provided, and
the conference was filmed and translated,
with the resulting DVDs distributed free of
charge to CF pediatric centers.
The Partners in Living Initiative has had
a measurable impact upon participants,
as outcome evaluations demonstrate
that participation in these programs has
improved participants’ wellbeing and
quality of life.
Tickets are $300 per person,
and expected to sell out.
Sponsorships are available.
A Magical Evening at Carolands is a
celebration of four decades of progress
in cystic fibrosis (CF) research. Enjoy
gourmet small plates, delectable desserts,
fine wine, fabulous entertainment
(Tammy Nelson of Beach Blanket Babylon),
the “CF Champion” award presentation,
and an exciting live auction. All funds
raised will support CFRI’s research and
education programs, to improve the lives
of those with cystic fibrosis.
For more information,
contact Siri Vaeth Dunn
650.404.9445 | svdunn@cfri.org
Sponsored by
• Vertex Pharmaceuticals
• NBC Bay Area • AbbVie • Chiesi USA
Those with CF cannot attend if they have:
Had a confirmed positive culture for Burkholderia
cepacia; Have cultured MRSA within the past 2 years;
Have had 2 or more consecutive positive cultures for
Nontuberculous mycobacteria in the past 12 months;
or currently culture positive for any bacteria resistant
to all antibiotics.
8
cfri | Summer 2015
CF Summer Retreat: Animate
progressed she sought a
Your Summer!
place where she could connect
By Isa Stenzel Byrnes, MSW / MPH
CFRI is excited to offer its 20th CF Summer
Retreat (formerly the CFRI Teen and Adult
Retreat),“Animate Your Summer!,” from
August 2–9th, 2015 at Vallombrosa Center
in Menlo Park. CFRI is a pioneer in creating
safe places for CF patients to socialize by
following a strict cross infection policy. The
retreat has been an emotional and social
sanctuary for many young adults with CF
over the years. Here they can exhale, be
themselves, open up, and share real stories
about what it’s like to grow up and live with
cystic fibrosis. Using educational seminars,
expressive arts workshops, and daily
exercise sessions such as Zumba® and yoga,
the CF Summer Retreat helps adults expand
their healing toolbox beyond the day-to-day
medical regimen. The retreat encourages CF
adults to live better with cystic fibrosis by
becoming empowered in self-management
of all aspects of CF care. The CF Summer
Retreat is indeed powerful medicine.
The 2015 CF Summer Retreat honors the
memory of Kriss Benson, a beautiful 38-yearold woman who was a long-time retreat
attendee, often accompanied by her loving
husband, Arthur Yang. Kriss grew up in
isolation from CF peers, but as her disease
with others going through
the same experience. Once
Kriss attended, she said,
“the light bulb went on, and
I knew I needed CF friends
to help me survive.” Kriss
became such a passionate
supporter that she joined
the retreat committee, feverishly helping out with graphic
designs, scavenger hunts,
arts and crafts, and other
creative activities. CF retreat
helped Kriss cope with the
demands of advanced-stage
CF. In return, the retreat
community received a fun,
Kriss Benson with her husband, Arthur Yang
loving, creative, authentic,
and dedicated role model to
helping others experience it also. She would
inspire us to live our best lives with CF.
be so happy to know that the Memorial
Sadly, Kriss passed away in September,
Fund will be providing assistance for others
2014. Her family has generously established
to attend for years to come.” The entire CF
the Kriss Benson Memorial Retreat Fund to
Summer Retreat community misses Kriss
help those with financial limitations attend
tremendously and is deeply appreciative of
the retreat free of charge. Kriss Benson’s
the Benson family’s support.
husband Arthur Yang shared that, “Kriss
greatly benefited from the immeasurable We encourage those with CF – 18 years and
older – to join us at this year’s CF Summer
emotional support that the retreat
Retreat. Animate yourselves!
provided, and she was passionate about
CFRI’s 2015 CF Summer Retreat
August 2 – 9, 2015 | Vallombrosa Retreat Center, Menlo Park, California
The CF Summer Retreat provides a safe environment that enhances positive
coping skills, social support, and education for people who share common
experiences with CF. Adults with CF, 18 years and older, their family
members, friends, and health care providers may attend.
Please join us!
Registration:
$85 per participant/week;
$15 per participant or visitor/day
Accommodations:
$55 per participant/night
Scholarships are available.
To ensure good health for all, please use proper hygiene practices.
All participants/guests with CF must comply with CFRI’s Infection Control Guidelines. For specifics, please go to www.cfri.org.
Sponsored by Genentech and Gilead Sciences
cfri | Summer 2015
For more information go to www.cfri.org or call 1.855.cfri.now
9
Measuring CFTR Function in Humans
Continued from front cover
Tests of efficacy require that a symptom of
the disease, almost always the ‘FEV1’ test
of lung function, must show a significant
difference between large groups of subjects
on or off the medicine. This takes time,
and such tests are becoming more difficult
because improved clearance and antibiotic
treatment regimens are slowing the decline
in lung function in people with CF to such
an extent that it would take many years
to see a difference.
Fortunately, it is now possible to measure
CFTR function accurately in humans by
using a special sweat rate test. Our sweat
glands produce sweat in two ways. One
pathway depends linearly on CFTR function.
We call this kind of sweating C (for CFTRdependent) sweating. C-sweat rates are
zero in CF and are half normal in carriers.
This linear readout of CFTR function is
highly unusual and is not seen in most
other tests of CFTR function – for example,
FEV1 and conventional sweat chloride
measures are essentially the same in
carriers and healthy controls.
A second pathway does not require CFTR
and appears to be normal in people with CF.
We call this kind of sweating ‘M-sweating,’
for the drug that we use to produce it.
Our test measures both kinds of sweating
in each of about 50 sweat glands in a
little patch of skin. By comparing the two
types of sweating in the same glands, we
control for variables such as gland size.
And because we can return to the same
identified glands over and over again in
different conditions, we quickly amass large
data sets that allow us to quantify even
small changes in CFTR function.
We report an individual’s CFTR function
as the percent of average function in a
population of healthy control subjects.
Interestingly, we find a large range of values
in our healthy control subjects, as though
they too were expressing a range of CFTR
function. Healthy people are known to
express a range of CFTR mRNA transcripts
(the molecules that let the cell make
CFTR protein), but no prior test has been
sensitive enough to detect corresponding
differences in CFTR function. With support
from a CFRI New Horizons Grant and from
the CFFT Sweat Measures Consortium,
we are measuring C- and M-sweating in
a large cohort of healthy subjects, and
will attempt to correlate those rates
with CFTR transcript levels, as well as in
selected individuals the DNA sequence of
CFTR. The results will further our goal of
providing a fast and accurate assessment
of CFTR function over its entire range, thus
enabling more rapid human testing of
CFTR-directed therapeutics.
Mother’s Day is Every Day at CFRI!
Our heartfelt thanks go out to those of
you who participated in CFRI’s biggest
fundraiser of the year, the annual Mothers’
Day Tea, and helped brighten the future
for someone living with cystic fibrosis.
Since Mother’s Day is every day at CFRI,
10
we still need your help
to reach our goal of
$175,000 to support our
promising research,
education and outreach
to the CF community.
If you have not participated and would like to
invite your friends and
family to savor a cup of
tea while reflecting on
a loved one who lives
with cystic fibrosis,
please contact us
at CFRI and we will provide you with
everything you need, including beautiful
invitations, teabags and information
about CFRI. For those of you who wish
to participate in a virtual tea, go to
http://tinyurl.com/ousfddj
Save the Dates
CFRI 28th National CF
Family Education Conference
Partners in Progress
July 31 – August 2, 2015
Sofitel San Francisco Bay
Redwood City, CA
Register Now!
CFRI CF Summer Retreat
August 2 – 9, 2015
Vallombrosa Center
Menlo Park, CA
Register Now!
Cystic Fibrosis Benefit
Golf Tournament at
Pasatiempo Golf Course
August 3, 2015
Santa Cruz, CA
Register Now!
CFRI’s 40th
Anniversary Gala
A Magical Evening
at Carolands
September 25, 2015
Hillsborough, CA
Purchase Tickets Now!
CF Discovery Series
September 8, 2015
October 13, 2015
November 10, 2015
6:00 pm – 7:30 pm PST
Crowne Plaza Cabana Hotel
Palo Alto, CA
Livestreamed!
CF Caregivers Support Group
Fourth Tuesday of Every Month
7:00 pm – 9:00 pm PST
CFRI office, Palo Alto, CA
Or participate by phone
Please RSVP
For more information
about any of these events,
please call 1.855.cfri.now
(1.855.237.4699)
or email cfri@cfri.org
cfri | Summer 2015
A Powerful Embrace: CFRI’s Retreat for Mothers of Those
with CF By Jane Mitchell
A group of 24 women gathered for CFRI’s
first retreat for mothers of children and
adults with CF, at the Vallombrosa Retreat
Center in Menlo Park, California, over the
weekend of April 17. Robin Modlin, MA,
mother of an adult daughter with CF and
long-time CFRI volunteer, brought the idea
for the retreat to CFRI, and “Embrace”
was born. The theme, “Embrace,” was
selected by Robin to encourage mothers
to, “embrace the journey, embrace the
community, embrace yourself.” Mothers
(and one adult sibling) traveled from several
states to participate. Our children ranged
in age from three years to 36; several
mothers had recently lost their daughters.
Vallombrosa provided the perfect backdrop
for sharing personal stories and experiences
along with our hopes and aspirations. CFRI
offered a weekend full of activities, including
a moving evening of diagnosis stories,
SoulCollage®, yoga, inspiring presentations
from experts, labyrinth walking, meditation,
and a powerful journaling workshop led by
Isabel Stenzel Byrnes, MSW, MPH. It was
a joyous time on Saturday evening when
singer-songwriter Nancy Cassidy performed
Embrace Retreat participants
that we will now continue the CF journey
together. The one question asked by every
mother who attended CFRI’s Embrace was,
Embrace is the perfect description for what
“When can we do this again?”
each of us experienced, as we shared our
unique bond of motherhood. As goodbyes Embrace was generously sponsored by
Genentech and Vertex, with additional
were shared on Sunday afternoon, there
was tremendous mutual excitement for this support from Modern Health Specialty
newly found community and the knowledge Pharmacy and Actavis.
at our evening celebration. The beautiful
music entranced everyone.
Become a CFRI Partner in Living! Your Support Can Change Lives
•ATTEND A CFRI FUNDRAISING EVENT No matter what
type of event you enjoy, we have one for you! In addition
to the Magical Evening at Carolands, we have concerts, golf
tournaments, and other events that are posted on our
website, Facebook page, and weekly eBlasts. Have fun while
supporting CFRI!
•COFFEE FOR A CURE Not a tea drinker? How about coffee?
Through Coffee for a Cure, you invite your family members,
friends and colleagues to join you for a virtual cup of
coffee, while supporting CFRI and its vital programs for
the CF community.
•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.
•HOLD YOUR OWN FUNDRAISER: 40 Years – 40 Events!
Big or small, we appreciate them all! Zumbathons, concerts,
cfri | Summer 2015
cupcake sales, dinner parties with a special guest of honor,
bocce ball tournaments – come up with an idea and we will
support you!
• TRIBUTES IN HONOR OF & 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.
•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 & provide positive tax benefits.
• 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
11
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.
CFRI’s 28th National Cystic Fibrosis
Family Education Conference
Partners in Progress
July 31 - August 2, 2015
Sofitel San Francisco Bay
Redwood City, CA
Our annual
conference brings together
experts in the field of CF to provide
the latest updates in research and care
to our diverse CF community.
Speakers: Tess Dunn – San Jose, California • Chris Kvam, Esq – Rochester, New York
Martha Markovitz, LCSW – University of Southern California • Kimberly Morse, LCSW –
Children’s Hospital Los Angeles • Elika Rad, MS, RN, NPC – Stanford Hospital & Clinics
Margaret Rosenfeld, MD – Seattle Children’s Hospital • Scott Russell, DPT – University
of Southern California • Greg Sawicki, MD, MPH – Boston Children’s Hospital
Lisa Saiman, MD – Columbia University Medical Center • Beth Sufian, Esq – Houston, Texas
Anna Tsang, MSO – St. Michael’s Hospital, Toronto
Early Bird Registration (on or before 6/30/15) - $180 per person
Regular Registration (after 6/30/15) - $215 per person
Registration includes meals, reference materials, and access to presentations & support groups.
To ensure good health for all, please use proper hygiene practices. All participants/guests with
CF must comply with CFRI’s Infection Control Guidelines. See www.cfri.org for specifics.
For more information, visit www.cfri.org or call 1.855.cfri.now
Visit our website at:
www.cfri.org
for more information about us
and about cystic fibrosis.
Call toll free: 1.855.cfri.now
Sponsored By: AbbVie • Boomer Esiason Foundation • Chiesi USA • Genentech
Gilead Sciences, Inc. • Novartis • Vertex Pharmaceuticals
For their generous support of CFRI Community, special thanks to:
Chiesi USA • Genentech • Gilead Sciences, Inc. • Vertex Pharmaceuticals
Cystic Fibrosis Research, Inc. a 501(c)(3) nonprofit organization Federal EIN# 51-0169988