Relatives of patients with type 1 diabetes (T1D) are at an increased risk for developing the disease themselves. In a recent study, researchers found that the use of teplizumab may delay the progression to disease among this patient population.
“Type 1 diabetes is a chronic autoimmune disease that leads to destruction of insulin-producing beta cells and dependence on exogenous insulin for survival,” wrote the researchers, whose study was published in The New England Journal of Medicine and presented at the American Diabetes Association’s 79th Scientific Sessions in San Francisco. “Some interventions have delayed the loss of insulin production in patients with type 1 diabetes, but interventions that might affect clinical progression before diagnosis are needed.”
The randomized, placebo-controlled, double-blind trial included patients with a family member who had T1D. Patients were randomized to receive either teplizumab—an Fc receptor–nonbinding anti-CD3 monoclonal antibody—or placebo; for 14 days, patients received a single daily dose. Oral glucose-tolerance tests were conducted every six months to test for progression to T1D.
“Previous clinical research funded by the NIH found that teplizumab effectively slows the loss of beta cells in people with recent onset clinical type 1 diabetes, but the drug had never been tested in people who did not have clinical disease,” said lead study author Kevan C. Herold, MD, of Yale University, in a press release. “We wanted to see whether early intervention would have a benefit for people who are at high risk but do not yet have symptoms of type 1 diabetes.”
Teplizumab Delays Disease Onset by Nearly Double
Of the 76 patients included in the trial, 55 (72%) were aged ≤ 18 years; 44 received teplizumab, and 32 were given placebo. The median time to diagnosis in the teplizumab group was nearly twice that of the placebo group (48.4 months vs. 24.4 months). In total, 19 (43%) teplizumab and 23 (72%) placebo patients were diagnosed with T1D.
“The hazard ratio for the diagnosis of type 1 diabetes (teplizumab vs. placebo) was 0.41 (95% confidence interval, 0.22 to 0.78; P=0.006 by adjusted Cox proportional-hazards model),” the researchers wrote, adding, “The annualized rates of diagnosis of diabetes were 14.9% per year in the teplizumab group and 35.9% per year in the placebo group.”
Adverse events included rash and transient lymphopenia.
The trial was conducted by Type 1 Diabetes Trialnet, which researches strategies to delay or prevent T1D.
“The difference in outcomes was striking. This discovery is the first evidence we’ve seen that clinical type 1 diabetes can be delayed with early preventive treatment,” according to Lisa Spain, PhD, Project Scientist from the National Institute of Health (NIH)’s National Institute of Diabetes and Digestive and Kidney Diseases, and sponsor of TrialNet. “The results have important implications for people, particularly youth, who have relatives with the disease, as these individuals may be at high risk and benefit from early screening and treatment.”