Scientists have found a drug that can delay the onset of type 1 diabetes in children who are at high risk of developing the autoimmune disease.
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 also published in the New England Journal of Medicine, according to NBC News.
Some experts are calling this finding a significant milestone in type 1 diabetes research.
PREVIOUSLY: Baby Takes Part In Trial To Prevent Diabetes
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.
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, NBC News reports.
“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 caused quite a stir in 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.”
According to the American Diabetes Association, about 1.25 million people in the U.S. have type 1 diabetes, with nearly 18,000 new cases diagnosed annually in people under age 20. The life expectancy of a type 1 diabetic is a decade shorter than those without the disease. 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.
Provention Bio Inc is developing the drug, but additional studies will be required before regulatory agencies approve it. Currently, the immuno-therapeutic drug is not approved by the FDA.