Posted by Ellen on June 03, 1999 at 12:45:32:
In Reply to: Another DRI report posted by Sandy Donchess on June 03, 1999 at 11:00:37:
By STEPHEN SMITH
Herald Health Writer
For people with the most severe type of diabetes, the reminders of their
disease are constant and painful. There's the jab of a needle delivering
life-sustaining insulin, the dread from standing in an ice-cream shop
empty-handed while all your friends savor a sugary treat.
On Wednesday, diabetes researchers at the University of Miami revealed
findings of a landmark drug study and dared to utter the word scientists
are most reticent to speak: cure.
Not right now. But the doctors at UM's Diabetes Research Institute
believe the medicine they tested in monkeys offers the best hope ever of
allowing diabetics to lead lives free of needles and ostracism.
There are few places in the nation where such a breakthrough would mean
more than in South Florida. Diabetes disproportionately targets African
Americans, Hispanics, Native Americans and Asian Americans. That's why
South Florida could benefit so much: The region has an estimated 400,000
diabetics.
``As a transplant immunologist, this is one of the most exciting
developments I've seen in 20 years,'' said Norma S. Kenyon, the UM
researcher who is the lead author of the study, which will appear in the
journal Proceedings of the National Academy of Sciences. ``But as a
mother of a daughter with diabetes, I do not want to raise false
hopes.''
Still, Kenyon and her colleagues are bursting with optimism after their
studies of a drug with a moniker straight out of Star Wars:
anti-CD154(hu5c8).
In people with the most pernicious form of diabetes, Type 1, the body
destroys the cells in the pancreas that make insulin, which helps
convert food into energy. They're called islet cells, and the sickest
diabetics have been getting transplants of islets since 1985.
To trick the body into embracing these donor cells, patients must take
antirejection drugs for the rest of their lives, drugs that can make
them seriously ill. And, even then, only 10 percent of transplant
patients are totally independent from insulin shots or pumps a year
later.
So scientists have been looking for a drug that's less toxic and more
effective. That's where anti-CD154(hu5c8) comes in. It falls into a
class of drugs known as monoclonal antibodies.
The scientists who discovered the medicine at a Cambridge, Mass.,
biotech company called Biogen believed it worked by shackling part of
the immune response that typically rejects transplanted islets. And they
believed it accomplished that without the nasty side effects associated
with traditional antirejection drugs.
Before testing it in humans, they needed to see how it would work in
monkeys. So researchers at UM removed the pancreases from six young
monkeys and seeded islet cells in their livers. When the monkeys were
given anti-CD154(hu5c8), the islets kept working. And working. In all
six monkeys. So far, the record holder is one primate who has gone 483
days since getting the transplanted cells.
In many ways, this is what medicine will look like in the 21st Century,
when the emphasis will shift from controlling disease to curing it by
routinely swapping out faulty body parts for healthy ones.
``This could open a new era in transplantation,'' said Dr. Camillo
Ricordi, scientific director at the Diabetes Research Institute.
By this summer, the UM researchers intend to begin studying the drug in
humans, who, like monkeys, have donor islets transplanted into the
liver, a better home for new islets than the very delicate pancreas.
``You could come in as an outpatient,'' Kenyon said, ``a catheter would
be placed through your skin into a vein into the liver, and you simply
take the bag of islets, drain them by gravity into the liver and that's
it.''
Almost. You'd still probably have to take regular, perhaps monthly,
doses of the experimental medicine.
For families like the Ullmans of Boca Raton, that would amount to
liberation. Now, Zachary Ullman, 11, must wear an insulin pump every
hour, every day. He has to prick his finger, to make sure his blood
sugar is at equilibrium. He must always carry an emergency stash of
food, lest his blood-sugar level get out of whack.
``I would hope this drug becomes a cure because then Mom wouldn't have
to go on all my overnight trips,'' Zachary said. ``My life would be a
lot easier without diabetes.''
As for Kenyon, the UM scientist, she has a personal stake in this
research. Her daughter Laura, now 6, was 14 months old when she was
diagnosed with diabetes, already the focus of Kenyon's laboratory
labors.
So, she was asked, if the new drug is proved safe and effective in
humans, would you allow your daughter to participate in the study?
Absolutely, the mother and scientist said.
For more information about participating in the study: contact the
University of Miami's Diabetes Research Institute at 305-243-5557.
e-mail: sfsmith_AT_herald.com