Investigational Drug, Teplizumab, May Help Delay Onset of Type 1 Diabetes

A two-year delay in the onset of type 1 diabetes could make a big difference for people with the disease. And researchers say a new drug may make that postponement possible.

Researchers gave the drug teplizumab or a placebo to a small group of people who were nearly certain to develop type 1 diabetes, based on genetics and certain symptoms. Those given a placebo, or dummy drug, progressed to type 1 diabetes in an average of just over 24 months. Those given the drug developed the autoimmune disease in an average of 48 months.

It's at least a few years until this drug could possibly be approved for use outside of a clinical trial, said Jessica Dunne, senior director of research at JDRF (formerly the Juvenile Diabetes Research Foundation).

Still, "keeping kids off insulin as long as possible is really exciting," Dunne said. "This study shows with just a short course of therapy, it's feasible to give people two years less of insulin shots, finger pricks and daily monitoring."

Dunne also noted that a delay means a child might be more mature and able to help manage the condition. "The difference between a 12-year-old and a 14-year-old being diagnosed is huge," she said.

A delay in diagnosis also means two fewer years with the disease. That might translate into fewer complications later in life, according to the JDRF.

"This is the first study to show any drug can delay type 1 diabetes diagnosis a median of two years in people at high risk," said study lead author Dr. Kevan Herold, a Yale University professor, in a news release from the school.

Type 1 diabetes occurs when the body's immune system mistakenly attacks insulin-producing cells called beta cells in the pancreas. Insulin is a hormone that helps usher the sugar from foods into the body's cells to be used as fuel.

These people must replace that lost insulin through daily injections or an insulin pump. Up to 1.5 million Americans have type 1 diabetes, according to the study authors.

Teplizumab targets specific types of immune system cells, interfering with the destruction of beta cells.

The study included 76 people older than 8 years. Most were under 18 years old. They were at very high risk of developing type 1 diabetes. They had a relative with type 1 diabetes and evidence that their own immune systems might already be attacking beta cells (auto-antibodies). The study volunteers also showed signs that their bodies weren't processing blood sugar normally.

Slightly more than half of this group received teplizumab for two weeks intravenously. The others were given a placebo by IV. Study participants were followed until they developed type 1 diabetes. The average study follow-up for most was three years, though some of the study participants haven't yet developed diabetes and are still being followed.

In addition to delaying the average onset of type 1 diabetes by two years, the researchers found that only 43% in the treatment group developed type 1 diabetes compared to 72% in the placebo group.

The findings were presented Sunday at the American Diabetes Association meeting, in San Francisco. They were published simultaneously in the New England Journal of Medicine.

Dr. Clifford Rosen, from the Maine Medical Research Institute, and Dr. Julie Ingelfinger, from Massachusetts General Hospital, wrote an editorial in the same issue of the journal.

"The results of this trial are striking, with several caveats," they wrote.

An important caveat, they said, is that these findings shouldn't be interpreted as a cure. But the findings do provide clues to potential causes of type 1 diabetes, and suggest ways to possibly try to modify the course of the disease.

The editorialists also noted that the study was small, and involved just a two-week course of treatment.

JDRF's Dunne agreed that the study raises new questions to be explored, such as what would happen if people were given more than one treatment? Or what if this drug was used with another?

But she said, "This study is particularly compelling because it was just a single course of the drug."

Funding for this study was provided by the U.S. National Institutes of Health, JDRF and the American Diabetes Association.

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