When Tyler Yun was three months
old he was diagnosed with a rare disease called Prader-Willi
Syndrome (PWS). The early diagnosis helped and he's now
a capable, hard-working teen � but the prognosis for him
is still grim. PWS patients can't stop eating, and eventually,
many of them literally eat themselves to death.
PWS is a genetic disorder that
afflicts one in every 15,000 newborns. Delayed mental
and physical development is part of the picture, but
probably the most disturbing characteristic of PWS is
in the obsessive need of patients to be constantly eating,
caused by a defect in the hypothalamus. Not surprisingly,
the stress the disease has on families is enormous.
At the Yun house food is completely locked away, which
may offer Tyler and his family a short reprieve, but
every trip out of the house is a struggle.
FOOD
WAR ON TWO FRONTS
PWS patients have been dealt a double whammy in the
food department. First, their bodies don't register
feelings of satiety � so they must endure constant cravings.
Tyler's mother describes the situation: "He'll eat food
off the floor or food he finds in the garbage. The impulse
just has a life of its own." What's more, PWS sufferers
have an altered metabolism that means they require 60%
fewer calories than normal people.
BIG
BROTHER'S CUPBOARD
Without the ability to suppress appetite, those with
PWS are at extreme risk of death from complications
resulting from obesity by the time they hit adolescence.
Parents must become food police and watch their children
grow up consumed with such a desire to eat that even
feces, dirt and their own bodies may be viewed as food
sources.
FREE
AT LAST?
Until now, constant surveillance was the only treatment
option. But Dr Jean-Pierre Chanoine, head of the Endocrinology
and Diabetes Unit at British Columbia Children's Hospital,
hopes he's found a much better treatment option. He's
designed a clinical trial to test a new, long-acting
form of the hormone somatostatin, marketed as Sandostatin
LAR. "An older version of the drug, octreotide, had
to be injected two or three times a day," says Dr Chanoine.
"In contrast, Sandostatin LAR is only injected once
a month."
Dr Chanoine has two hopes for his
trial. The first is to control ghrelin, the appetite-stimulating,
metabolism-slowing hormone which occurs at elevated
levels in PWS patients. The second is to produce a decrease
in appetite as the ghrelin level drops. He's optimistic
about both. The one-year trial will randomize 10 genetically-confirmed
PWS patients between 10-17 years of age to either Sandostatin
LAR or placebo. Dr Chanoine welcomes additional patients
� particularly from BC, and asks that physicians or
parents contact him at (604) 875-2624.
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