This is big. No, wait, this is ENORMOUSLY HUGE. Published today in the New England Journal of Medicine, an immune therapy demonstrated a significant delay in the time to onset of type 1 diabetes (T1D) in high-risk relatives of people with the disease. A significant delay meaning 2+ years! This is the first ever study in humans to show a delay in the onset of T1D.

“This is an exciting and important advance that shows we can change the course of type 1 diabetes,” said Aaron J. Kowalski, Ph.D., Breakthrough T1D President and CEO. “Our goal of cures for and prevention of type 1 diabetes is making real strides forward.”

The drug is called teplizumab, and it is an anti-CD3 monoclonal antibody. CD3 is a blood marker that helps to activate the immune cells—called T cells—which are thought to be responsible for T1D. Breakthrough T1D funded one of the first clinical trials of this drug in new onset disease, results of which were integral to the teplizumab prevention trial discussed here.

Here are the details:

  • TrialNet did a phase 2 randomized trial and recruited 76 subjects who had not been diagnosed with the disease, but who exhibited at least 2+ autoantibodies and abnormal blood sugar (called dysglycemia). Antibodies against one’s own proteins are a marker for T1D
  • Median (average) time to T1D: 48.4 months for the teplizumab-treated subjects vs. 24.4 months for placebo-treated subjects—that means it delayed T1D for two years!
  • Moreover, 57% of teplizumab-treated subjects remained T1D-free at the conclusion of the study, compared with 28% of the placebo group—meaning that teplizumab-treated subjects were half as likely to develop T1D compared to the placebo group

TrialNet is the largest clinical trial network for T1D, funded by the National Institutes of Health, Breakthrough T1D and the American Diabetes Association.

Carla Greenbaum, M.D., TrialNet Chair and Diabetes Program Director, Benaroya Research Institute at Virginia Mason in Seattle, WA, said it best: “This is an incredible advancement that gets us one step closer to our ultimate goal: a future without T1D.”

We hope so.

The antibody will now go through a phase 3 clinical trial for newly onset disease, headed by Provention Bio, a company with an investment from the Breakthrough T1D T1D Fund for development of an enterovirus vaccine.