Teplizumab May Prevent Type 1 Diabetes Development in High-Risk Individuals

type 1 diabetes
type 1 diabetes
In people at high risk of developing type 1 diabetes (T1D), teplizumab may delay or prevent disease progression.

SAN FRANCISCO — In people at high risk of developing type 1 diabetes (T1D), teplizumab may delay or prevent disease progression, according to study results presented at the American Diabetes Association 79th Scientific Sessions, held June 7 to 11, 2019.

Results from the phase 2 study were also published online in the New England Journal of Medicine on June 9.

There is a need for interventions that prevent the development of T1D in high-risk individuals, such as relatives of patients with T1D. Researchers conducted a randomized placebo-controlled trial of teplizumab, an anti-CD3 monoclonal antibody, to determine if the treatment alters the progression of T1D. Follow-up evaluations using oral glucose tolerance tests were performed at 6-month intervals.

Relatives of patients with T1D were randomly assigned to receive a 14-day course of teplizumab (n = 44) or placebo (n = 32). T1D was diagnosed in 19 participants (43%) of the teplizumab group with a median time to diagnosis of 48.4 months. In the control group, 23 participants (72%) were diagnosed with T1D after a median of 24.4 months.

Compared with placebo, participants receiving teplizumab were significantly less likely to progress to clinical T1D (adjusted hazard ratio, 0.41; 95% CI, 0.22-0.78; P =.006), with an annual rate for diagnosis of diabetes of 14.9% vs 35.9% in the placebo group.

Fewer cases of diabetes were diagnosed in patients who were HLA-DR3-negative, HLA-DR4-positive, or zinc transporter 8 antibody-negative.

Rash and transient lymphopenia were reported as adverse events.

Related Articles

The researchers noted that the study was limited by its small sample size and only a single course of medication. Repeated dosing may provide additional benefits, but the researchers did not investigate this in the study.

“The delay of progression to diabetes is of clinical importance, particularly for children, in whom the diagnosis is associated with adverse outcomes, and given the challenges of daily management of the condition,” wrote the researchers. “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.”


Herold KC, Bundy BN, Long SA, et al. An anti-CD3 antibody, teplizumab, in relatives at risk for type 1 diabetes [published online June 9, 2019]. N Engl J Med. doi:10.1056/NEJMoa1902226