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Compound shows promise as vaccine-like drug for preventing type 1 diabetes

ANAHEIM, Calif., March 30 Researchers have identified a compound that shows promise in animal studies of becoming the first effective drug for the prevention of type 1 diabetes, or insulin-dependent diabetes, which afflicts about one million people in the United States and is on the rise worldwide.

The synthetic compound, called ISO-1, appears to work by blocking a pathway involved in inflammation. If successful as a vaccine-like drug, it could ultimately save lives, reduce health care costs and help prediabetic people avoid a lifetime of insulin injections, the researchers say. Their work was described today at the 227th national meeting of the American Chemical Society, the world's largest scientific society.

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

The compound targets prediabetic individuals, defined as those who have blood markers either antibodies or genetic markers that are predictive of the disease. Using certain blood tests, many of these individuals can now be identified long before they actually develop type 1 diabetes (also known as juvenile diabetes). More testing is needed before the compound is ready for human use, which could be several years, say Al-Abed and his associates, who hope to develop the compound into a long-acting oral drug.

When ISO-1 was given via injection over a ten-day period to mice that were also given a chemical designed to induce a form of type 1 diabetes, ISO-1 completely protected them from developing the disease. By contrast, all of the mice treated only with the diabetes-inducing chemical developed the disease. In a similar controlled experiment,
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