Updates from the Teplizumab Trial: You’re Not Going to Believe the Results

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Last year, at the American Association for Diabetes (ADA) Scientific Sessions, a drug, called teplizumab, was able to significantly delay—for over two years—the onset of type 1 diabetes (T1D) in participants with a high risk of developing the disease. This was the first ever study in humans to show a delay in the onset of T1D. But it doesn’t stop there. JDRF has updated results, and this is big. The investigators were able to follow up on the individuals who didn’t develop clinical diabetes, and the effect of teplizumab had been sustained: 50% of those treated with teplizumab remain diabetes-free, compared to only 22% of those taking placebo, and the delay in diabetes onset was close to 3 years (35 months). These were presented by Emily Sims, M.D., an assistant professor of pediatrics at Indiana University and a JDRF-funded investigator, at ADA’s Scientific Sessions.

Additionally, the participants, both the teplizumab and placebo groups, had a progressive decline in the biomarker that measure’s the body’s ability to produce insulin—C-peptide—preceding the trial. But the teplizumab team found that production of C-peptide actually went up following treatment with teplizumab, especially in the first 6 months after treatment. Like, teplizumab could reverse the downward trajectory of C-peptide loss that was there before the trial.* Um, wow. And there’s more: the increases in C-peptide were correlated with induction of a state of “exhaustion” in “bad” immune cells. Like, they literally took a punch and lost the ammo to keep on attacking the beta cells. Wow (again).

So, could the exhaustion of “bad” immune cells result in not getting T1D or significantly slowing down the time to getting it? Well, we’re not there yet, but we’re close, and closer to a day when T1D might be prevented, forever.

JDRF Leadership: JDRF funded one of the first clinical trials of this drug, results of which were integral to the teplizumab prevention trial. The study in this report was conducted by TrialNet, a T1D trial consortium funded by NIDDK and by the NIH’s Special Diabetes Program for which JDRF is the leading advocate. Multiple studies involving samples and data from teplizumab trials have continued to receive JDRF funding. Teplizumab (now called PRV-031) is currently being tested in a phase 3 clinical trial in people recently diagnosed with T1D, headed by Provention Bio, a company with an investment from the JDRF T1D Fund. If successful, this could become the first immune therapy approved for T1D.

*In the placebo group, C-peptide still declined.