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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.

More than a million people in the United States have type 1 diabetes, which requires constant attention to blood sugar levels and insulin injections to stay alive. The condition carries a risk of long-term complications, including heart disease, blindness, and kidney failure. (People with the more common type 2 diabetes generally produce their own insulin, but their bodies can't use it properly.)

Over time, scientists have learned that type 1 diabetes begins years before it's diagnosed. Subtle attacks on the pancreas are led by the sentries of the immune system, T cells. Those attacks are detectable via antibody markers in the blood. During this quiet battle, beta cells in the pancreas are still largely intact, offering a crucial window in which to intervene and save them.

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