Experimental drug delays type 1 diabetes in high risk children

For the first time, scientists have found a drug that can delay the onset of type 1 diabetes in those who are at high risk of developing the autoimmune disease, a finding some experts are calling a milestone in type 1 diabetes research.

In high-risk people, 14 days of therapy with the experimental drug teplizumab delayed development of the disease by a year or more, according to results from a study presented Sunday at an American Diabetes Association meeting in San Francisco. The results of the study were simultaneously published in the New England Journal of Medicine.

The phase 2 trial, which studies a drug’s effectiveness in a relatively small number of people, is the first to show that immunotherapy can be used to delay the onset of an inherited disease.

“This is a huge milestone. We’ve had trials that have been going on for a couple decades, but they have not been able to prevent diabetes. It was a very disappointing result in the field,” said lead study author Dr. Kevan Herold, professor of immunology and endocrinology at Yale University. “This is the first successful trial to show that you can delay and possibly prevent, type 1 diabetes.”

The 76 study participants, who ranged in age from 8 to 49, faced a high risk of type 1 diabetes in part because their relatives had the disease, which kills the beta cells in the pancreas that make and release insulin. Also, the volunteers all had tests showing diabetes-related autoantibodies that attack the pancreas, plus unhealthy blood sugar levels.