New Therapy Prevents Type 1 Diabetes in Mice

Medically Reviewed by Brunilda Nazario, MD on March 30, 2004
From the WebMD Archives

March 30, 2004 -- A synthetic compound may one day save people at risk for type 1 diabetes from a lifetime of insulin injections, according to new research.

Preliminary tests show the compound prevented the disease in mice by blocking a pathway involved in inflammation, a process involved in the development of type 1 diabetes.

If further studies confirm these results, researchers say the compound, known as ISO-1, may be developed into a long-acting drug that would work like a vaccine to protect people against the disease.

About 1 million people in the U.S. have type 1 diabetes, formerly known as insulin-dependent or juvenile diabetes. The disease occurs when insulin-producing cells in the pancreas are destroyed by the immune system.

Researchers say recent studies have shown the that several proteins are involved in the immune system's inflammatory responses that lead to the development of type 1 diabetes, and this new therapy targets one of those proteins in order to prevent the disease.

The results of the study were presented this week at the national meeting of the American Chemical Society in Anaheim, Calif.

Compound May Prevent Diabetes

In the study, researchers gave ISO-1 via injection to a group of mice that were also given a chemical that induces type 1 diabetes. The study showed that the compound completely protected the mice from the disease, while mice treated only with the diabetes-inducing chemical developed type 1 diabetes.

In another experiment, researchers injected ISO-1 in mice that were genetically bred to develop diabetes, and the compound prevented the disease in 90% of the animals treated with the compound.

Researchers say they believe the compound works by blocking a protein called MIF, which appears to be involved in the inflammatory process linked to the destruction of insulin-producing cells.

Because the compound is a relatively small chemical, researchers say it could be developed into a pill that could work like a vaccine to provide a lifetime of protection from diabetes for people at risk of the disease. Blood tests can identify people at risk of type developing type 1 diabetes.

"We believe this is the most promising compound to date for preventing type 1 diabetes," says researcher Yousef Al-Abed, PhD, an associate investigator at North Shore-Long Island Jewish Research Institute in Manhasset, N.Y., in a news release. "If it works, it will be especially beneficial for young people, who often have a difficult time managing their diabetes with daily insulin injections."

Show Sources

SOURCES: American Chemical Society 227th National Meeting and Exposition, Anaheim, Calif., March 28 - April 1, 2004. News release, American Chemical Society.

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