More than five years after receiving an experimental immunotherapy drug, half of a group of people at high risk of developing type 1 diabetes remained disease-free, compared with 22% of those who received a placebo. This is evident from a new study by the Yale School of Medical Researchers.
And those who got diabetes did so an average of about five years after receiving the new drug teplizumab, compared with 27 months for those who received the placebo.
The study, carried out in collaboration with researchers from Indiana University, was published March 3 in the journal Science Translational Medicine.
“If approved for use, it will be the first drug to delay or prevent type 1 diabetes, ”said Kevan Herold, CNH full professor of immunobiology and medicine (endocrinology) at Yale and co-senior author of the paper.
The drug, developed by biotech company Provention, has been classified as a breakthrough by the U.S. Food and Drug Administration and could be approved for general use by the summer, Herold said.
In the study, an analysis of 76 subjects showed decreased T-cell damage in response to the drug and improved function of insulin-producing beta cells in those who received teplizumab.
The subjects in the study had a mean age of 13 years and had relatives with type 1 diabetes.
The new study is the result of 30 years of Herold’s lab working to find new treatments for type 1 diabetes. The results are a continuation of another clinical trial organized by TrialNet, an international coalition dedicated to studying the disease. This study, published in 2019, showed a delay in the onset of type 1 diabetes in those who received teplizumab.
Type 1 diabetes is an autoimmune disease in which a person’s own T cells attack insulin-producing beta cells in the pancreas. Those diagnosed will need lifelong insulin treatment and are at greater risk of death and diseases that affect the heart, kidneys, and eyesight. The disease is often diagnosed in childhood or adolescence.
Herold emphasized that it was not known whether some of the patients who received teplizumab would never develop type 1 diabetes. However, delaying the onset of the disease could have a huge impact on the development of those at risk.
“Any time without diabetes is important, especially for children who might have the chance to grow up without diabetes, ”he said.
The study was funded by the National Institute for Diabetes and Digestive and Kidney Diseases.
Emily Sims of Indiana University School of Medicine is the lead author on the study.
The study was sponsored by the Type 1 Diabetes TrialNet Study Group, a network funded by the National Institutes of Health and JRDF.