Drug delays type one diabetes in vulnerable 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 at an American Diabetes Association meeting in San Francisco and 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 eight 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. Among the 44 volunteers randomly assigned to receive the drug, 19, or 43 percent, developed diabetes, with the disease appearing within 48.4 months in half of them.

By comparison, among the 32 people who received a placebo, 23, or 72 percent, developed diabetes, with half the patients developing it within 24.4 months. When the study was stopped, the percentage of diabetes-free participants was twice as high in the teplizumab group, 57 percent, as in the placebo group, 28 percent.

The chief side effects were temporarily low levels of lymphocytes — a type of white blood cell — and a rash.

“Not having diabetes is a big deal. Anything that can prevent the disease will bring huge excitement,” Herold said. “We are anxious to hear from the FDA what the regulatory path is moving forward.”

The long awaited results have re-energized the diabetes research community.

Dr. Clifford Rosen of the Maine Medical Center Research Institute and Journal deputy editor Dr. Julie Ingelfinger wrote in an editorial, “We can finally say that there has been substantial progress in modulating the early course of type 1 diabetes.”

And Dr. Fernando Ovalle, director of the division of endocrinology, diabetes and metabolism at the University of Alabama at Birmingham, who was not involved in the study, noted that while this approach is unlikely to fully prevent or reverse type 1 diabetes, it may be used in combination with other approaches that may some day lead to a cure.

“I would argue that drugs like Verapamil, which have been shown to preserve beta cell function in adults with newly diagnosed type 1 diabetes, can play a significant synergistic role in combination with these immune modulator therapies, and deserves further study,” Ovalle said.

Currently, the treatments for type 1 diabetes include: Taking insulin via injection, an insulin pump, or artificial pancreas; Carbohydrate, fat and protein counting; Frequent blood sugar monitoring; and Eating healthy foods, and Exercising regularly and maintaining a healthy weight.

The promise is that teplizumab, which works by modifying the white blood cells from the immune system that kill insulin-producing cells in the pancreas, would delay or even prevent the disease in children who are at high risk so they do not have to make these life altering changes.