Treatment Delays Type 1 Diabetes Risk

A single course of treatment with teplizumab significantly slowed the progression of type 1 diabetes in high-risk individuals who had not yet deveoped the condition, according to a study in the New England Journal of Medicine.

Type 1 diabetes is an autoimmune condition in which insulin-producing beta cells are destroyed. It affects up to 1.5 million people in the U.S. where, despite treatment, most patients do not achieve healthy glycemic targets raising the risk of complications and death. So delaying disease onset, as done in this study, would be a benefit to patients.

"Our findings support the notion that type 1 diabetes is a chronic T-cell–mediated disease and suggest that immunomodulation before the development of clinical disease can be useful," wrote authors who were led by Kevan C. Herold, M.D., Yale University. The study was published Aug. 15.

This was a phase two randomized, placebo-controlled, double-blind trial of 76 high-risk individuals with at least one first-degree relative with type 1 diabetes. Most of the members of this trial (72 percent) were teens and children under the age of 18 years. 44 were randomonly assigned to a single 14-day course of teplizumab and 32 received the placebo. The treatment delayed the onset of type 1 diabetes by 48.4 months as compared to 24.4 months for the placebo group specifically affecting 19 members (43 percent) of the treatment group and 23 (72 percent) of the placebo group. The hazard ratio for diagnosis was 0.41 (95% confidence interval).

"Our observations among young persons who did not yet have clinical disease reflect the likely progression when two or more autoantibodies and dysglycemia are found and are consistent with our report of high rates of beta-cell death in these persons," the authors wrote.

The adverse events associated with this trial included rash and transient lymphopenia.

The authors noted several limitations, including the small number of participants so they cannot say for certain the degree of applicability to people without first-degree relatives with type 1 diabetes.

REFERENCE

Kevan C. Herold, M.D., Brian N. Bundy, Ph.D., S. Alice Long, Ph.D., et al. "An Anti-CD3 Antibody, Teplizumab, in Relatives at Risk for Type 1 Diabetes." NEJM. Aug. 15, 2019. DOI: 10.1056/NEJMoa1902226