Hope For The Future
Duncan
Olivia
Foundation
for Prader-Willi Research
www.pwsresearch.org -- Coming Soon!
"Harnessing the Power of Research to make Prader-Willi Syndrome powerless in the lives of those we love"
The Foundation for Prader-Willi Research is a non- profit organization dedicated to furthering a better understanding of the causes of Prader-Willi Syndrome through the financial support of research. The FPWR was founded and is maintained by parents and family members of persons with PWS. We hope that our efforts will lead to more effective treatments and an eventual cure for PWS. The FPWR is a registered, 501(3)c organization. All donations are fully tax-deductible to the extent allowed by law. All publicly donated funds are directed towards the support of biomedical research.
Octreotide: One Small Step Closer… or is this a Giant Leap?
by
Lisa Graziano
Executive Director
Prader-Willi California Foundation
Not long after
our son Cameron was born and we began to learn more about Prader-Willi syndrome
(PWS), I made two predictions: First, that someday babies born with Prader-Willi
syndrome would be able to receive growth hormone treatment without having to
undergo the quite unpleasant stimulation test we had to watch our baby go
through. And second, that a medication to combat the symptom of
unrelenting hunger would be found within his lifetime.
Thankfully, my
first prediction came true less than eighteen months later when, in 2000 the FDA
determined that PWS is an eligible condition for treatment with growth hormone,
and that growth hormone deficiency testing is no longer required for children
with PWS and growth failure.
That same
year, 2000, scientists made the connection between the newly discovered hormone
ghrelin (derived from the root “gre” for “growth” and the suffix
“relin” for “releasing hormone”) and its regulation of hunger.
Ghrelin levels ebb and tide over the course of the day; when they are
high we feel hungry, and they fall after we eat.
Ghrelin levels in individuals with PWS don’t appear to ebb and tide,
but rather they are always high. In
fact, they are about four to five times higher than the general population.
I just love the analogy PWCF member Jeff Higgens once used to
describe PWS ghrelin levels: he said “ghrelin in the body of someone with PWS
is like a broken fire hydrant.” Could
it be that when we find a way to “cap” this gushing ghrelin, we may finally
have a successful treatment for the symptom of hunger?
At this year’s
PWSA (USA) National Conference, Andrea Haqq, M.D. (Department of
Pediatrics at Duke University Medical Center) reported on exciting new research
she and other scientists, including David Cummings, M.D. (University of
Washington’s Department of Medicine) are working on involving ghrelin and a
potential appetite suppression medication called octreotide.
Dr. Haqq reported that octreotide (a somatostatin agonist that binds to the receptors, disallowing it to be taken up by the hypothalamus – in other words, it works as a “ghrelin cap!”) was recently studied to determine whether it would decrease ghrelin levels in children with PWS. Four children with PWS were studied over the course of about seven days. Ghrelin levels, in addition to a number of other parameters, were measured before, during, and after octreotide treatments (subcutaneous injections). The results of the study showed that fasting ghrelin levels in the children decreased by approximately 67% after 5-7 days of octreotide treatment – meaning octreotide did lower ghrelin levels. Side effects were minimal: transient (temporary) diarrhea and abdominal discomfort. Dr. Haqq also noted that one parent reported fewer tantrums over denial of food during the intervention period.
Do we have a successful satiety medication yet? Not quite – further studies are needed to determine the efficacy and safety of octreotide. But I have faith in my second prediction. One small step after another, we move closer to realizing our hopes and dreams for our children.