In milestone trial, experimental drug delays type 1 diabetes

Marking the culmination of a 33-year odyssey, scientists today report a milestone in type 1 diabetes: the first time the disease has been markedly delayed in young people at high risk. Presenting at the American Diabetes Association meeting in San Francisco and publishing simultaneously in the New England Journal of Medicine, researchers found that 2 weeks of an experimental intravenous drug held off disease by an average of about two years.

The mainstay of type 1 diabetes treatment is insulin, discovered 97 years ago. These results open a new chapter, says Jeffrey Bluestone, an immunologist at the University of California, San Francisco, and part of the research team. “On the one hand,” the outcome is “pretty exciting,” Bluestone says. “On the other hand, now the real hard work begins.” That will mean considering how to move this treatment forward and probing whom it’s most likely to help.

The clinical trial began 8 years ago and included 76 people, the youngest of whom were 8 years old and the oldest in their 40s. Nearly three-quarters were 18 and under. Each had an extremely high risk of type 1 diabetes. In this autoimmune disease, the body attacks cells in the pancreas that make insulin, which helps keep blood glucose levels in check. By the time diabetes is diagnosed, most of these insulin-producing cells, called beta cells, are gone.

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