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Phase 1 Study Shows Promising Results In Type 1 Diabetes

POSTED: 5:04 pm EDT May 14, 2008
UPDATED: 5:53 pm EDT May 14, 2008

The following is a transcript of a report by medical editor Marilyn Brooks that first aired May 14, 2008, on WTAE Channel 4 Action News at 5 p.m.


Researchers at Children's Hospital might have a way to reverse type 1, or insulin dependent, diabetes.

The treatment cures mice and has not moved on to human trials.

The treatment is for people who have just developed the disease. It is so promising that TrialNet, a national network of diabetes research institutions, has agreed to a national trial using the approach to test for safety. If it proves safe, they'll move on to Phase 2.

Two weeks ago, the mice had type 1 diabetes. They were cured by a treatment that reverses the condition in new onset patients. It begins with leukophoresis.

"Leukophoresis is just to extract the white cells, giving back to the patient the red, the serum and everything," said Dr. Massimo Trucco. "When we have just the white cells, we grow in our petri dish until we have a sufficient number."

Those cells are then treated and returned to the patient with the ability to block the immune system’s attack on insulin-producing beta cells in the pancreas.

"When we will do the real therapy to do to children that still have some insulin-producing cells and are young enough to produce new beta cells," said Trucco.

Leukophoresis is tough on small children. In anticipation of larger human trials, one researcher developed a microsphere-based vaccine to make the process easier.

"They will still need an injection very close to the site where the pancreas is, but it will be under the skin injection, very safe, and it's no different than doing an allergy test," said Dr. Nick Giannoukakis.

Before that therapy is offered, it must be proven safe.

Ryan Cupps, 19, is the first of 14 volunteers in the Phase 1 trial.

"I was excited," he said. "I mean, over 20 million people have diabetes. If I can help those 20 million people, then I'm going to do it."

Cupps was diagnosed with type 1 diabetes in 2000. He takes six insulin injections every day.

Again, the findings of the Phase 1 study shows the vaccine prevents and reverses new onset diabetes in mice. The early human trials are expected to be finished by the end of the year. Trucco expects to be able to move into the Phase 2 study next year.


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