In a landmark achievement, researchers have reported successfully delaying the onset of type 1 diabetes in high-risk patients through an eight-year trial, according to Science. Just two weeks of treatment with the drug delayed the onset of diabetes by an average of two years.
Since scientists first discovered they could predict risk for diabetes based on family history, they’ve searched for interventions to prevent the disease in individuals known to be at risk.
By the time diabetes is usually diagnosed, the beta cells responsible for producing insulin have already been lost. But before this occurs, immune cells called T cells have been attacking the pancreas for some time. Antibody markers indicate when this is occurring, and during this process, beta cells in the pancreas are mostly still functional. This suggests that the right treatment could potentially prevent further damage.
But previous trials, using treatments such as oral insulin and high doses of B vitamins, were not effective in staving off diabetes.
With the new trial, just 14 days of treatment with an experimental drug called teplizumab delayed the onset of diabetes for a year or more. It was a small, phase two trial with 76 participants between the ages of 8 and 49. They were considered at high risk for diabetes, having relatives with the disease, as well as tests showing the antibody markers indicating that T cells are attacking the pancreas.
A total of 44 received the treatment, out of which 43 percent developed diabetes. A placebo was given to the remaining 32 people, out of which 72 percent ultimately developed diabetes. On average, the disease also took effect later in the group that had been given teplizumab.
“This is the first successful trial to show that you can delay and possibly prevent, type 1 diabetes,” according to the lead author of the study, Yale University professor of immunology and endocrinology, Dr. Kevan Herold. “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.”
The primary side effects were a rash and a temporary decline in levels of lymphocytes, a type of white blood cell.
“Not having diabetes is a big deal. Anything that can prevent the disease will bring huge excitement. We are anxious to hear from the FDA what the regulatory path is moving forward,” Herold said.
The results were presented at a meeting of the American Diabetes Association in San Francisco, California, and were published in the The New England Journal of Medicine (NEJM).