health & wellness
Cure Kids First
Michigan pays lead role in conference for kids with Inflammatory Bowel Disease.
CARE
KID
fir
Ruthan Brodsky
I Contributing Writer
ichigan's Pediatric IBD
Foundation is the lead co-
sponsor for a third meeting of
federal regulators, the European Medicines
Agency, medical industry representatives,
academia, patients and researchers for col-
laborative, stakeholder discussions March
30-31.
Federal regulators include the Food
and Drug Administration (FDA) and the
National Institutes of Health (NIH).
A major agenda item for the meeting
addresses the issue of how to expedite
the approval process of IBD medicines
for children and to efficiently monitor
patient safety by starting a disease-specific
National Public Registry available to all
providers and patients.
Pediatric Inflammatory Bowel Disease
M
36
March 5 • 2015
is a group of chronic disorders that cause
pain, swelling and inflammation of the
small and large intestine. The two most
common of these disorders are Crohn's
Disease and Ulcerative Colitis.
The problem is that despite universal
coverage, more than 70 percent of the
medications used in children with IBD
are prescribed off-label because they were
tested for adults and not for children.
Insurance companies can deny parents
insurance reimbursement for these pre-
scribed off-label medications sometimes
resulting in IBD medications costs of over
$75,000-$100,000 per year.
"The Pediatric IBD Foundation par-
ticipated in GREAT1 (Gastroenterology
Regulatory Endpoints and the
Advancement of Therapeutics) 18 months
ago, then we were a co-sponsor for
GREAT2 and now we are the lead co-spon-
sor for GREAT3," says Dr. Eric Zuckerman
of Bloomfield Hills, board chairman of the
Pediatric IBD Foundation. Zuckerman,
also a FDA Patient Representative, will be
presenting a model for a public National
IBD Registry at GREAT3.
"The Pediatric IBD Foundation has
come a long way:' continues Zuckerman.
"We are doing all the administrative
responsibilities for GREAT3 including reg-
istration, travel and hotel arrangements,
and helping with the agenda. I think we
were selected because we are a nonprofit
in the right place at the right time and
receive our funds from professional orga-
nizations, parents and other foundations.
"Although we are the smallest group at
this meeting, we are highly regarded by
those attending and are honored to take
on these tasks:'
Zuckerman is one of the founding
members of the Pediatric IBD Foundation,
established in 2008 by a group of volunteer
parents. He has held the position of board
chairman since the foundation's inception
helping to set the foundation's ground
work and infrastructure through advocacy,
research and education. Today, five of
the foundation's six board members are
located in Michigan.
"Of the 1.7 million IBD patients in the
U.S., one-third had their disease diagnosed
in childhood and 50 percent had symp-
toms before age 18:' explains Zuckerman.
"Unfortunately there is a disproportional
amount of resources dedicated to studying
this disease when it first strikes in chil-
dren because they often provide a better
opportunity than adults to understand
what triggers these diseases in genetically
susceptible individuals.
"Interestingly, cases of pediatric Crohn's
Disease have tripled in the last decade
continues Zuckerman. "In addition, the
incidence of IBD is higher in patients of
Jewish descent. What's more alarming is
the increase of IBD in children under age
6 and a larger number diagnosed during
their first year. The Foundation is focused
on finding safer, more effective treatments
and to "Cure Kids First" as healthier chil-
dren make healthier adults:'
Advocacy For Kids
"The Pediatric IBD Foundation is in a
growth period and moving forward con-
structively as it adjusts to increasing its
collaborative efforts with other profes-
sional groups and fostering interest in
other foundations:' says Alan Singer of
Bloomfield Hills.
"I've been friends with Eric for many
years and was very much aware of the
family's struggles when their daughter was
diagnosed with IBD disease as an adoles-
cent. I understand many of their problems
because I've been dealing with Crohn's dis-
ease myself since my early 40s. I became
a foundation board member four years
ago so I could use my experience both as a
patient as well as serving on many Jewish
and other nonprofit boards.
"There are many great programs in the
works as we expand our network of getting
others to help us while maintaining a vol-
unteer parent organization that uniquely
focuses on children and is supported by
parents and by other foundations inter-
ested in our mission:' says Singer.
Research For A Cure
The foundation supports the oldest and
one of the largest collections of pediat-
ric IBD tissue and blood samples in a
biorepository at Massachusetts General
Hospital. This resource is used by
researchers and industry pursuing new
diagnostic tests for IBD.
"One of our foundation's research initia-
tives, called the Biome-Genome Study, is
analyzing the more than 1,000 samples of
tissue and blood located in the repository
freezer, looking for which bacteria triggers
which gene says Zuckerman.
"For instance, we already know that
what triggers the disease in Iowa is differ-
ent than what triggers it in Israel. We are
also looking for which gene is carried in
certain families by using saliva samples for
DNA detecting:'
Coordination With Israel
According to
Zuckerman, more than
800 physicians partici-
pated in the foundation's
medical education pro-
gram in its first year.
The foundation is also
in discussions with an
Israeli IBD organiza-
Dr. Eric
tion
to collaborate on
Zuckerman
educational and research
initiatives between the U.S. and Israel
through the Michigan-Israel Business
Bridge.
"The hardest part for me was to real-
ize that children are the most neglected
demographic in IBD research and
have no voice says Amanda Fisher of
Birmingham, IBD Foundation board
member. "Parents see their children in
pain and are unable to provide medication
because most are considered off-label and
not approved for use in this age group.
Philanthropy runs in my blood, especially
when it's something I feel passionate
about. I'm going to do whatever I can to
make an impact and help:'
"We in Michigan have this opportunity
to lead a collective effort and help chil-
dren and their families who live with this
disease says Zuckerman. "We believe our
Michigan group can serve as a catalyst for
important discussions about pediatric IBD
on a national level. We invite parents who
are interested in learning more about us or
in supporting our mission to contact the
Pediatric IBD Foundation at www.pedsibd.
org."
❑
Donations can be made online or
mailed to: Pediatric IBD Foundation,
ao Tucker, Saltzman and Dyer, 50
Congress St., Suite 430, Boston, MA
02109.