New Therapy Prevents Type 1 Diabetes in Mice
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
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
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
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
"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."