Drug Targeting Immune System Delays Type 1 Diabetes in High Risk Patients

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By : dLife Editors

A drug that targets the immune system can delay the onset of Type 1 diabetes an average of two years in children and adults at high risk, according to findings presented at the American Diabetes Association meeting in San Francisco this past weekend.

With 28 study sites around the world, it is the largest clinical trial network ever assembled to discover ways to delay and prevent Type 1 diabetes.

“The key point for the millions of people at risk to develop this disease is we now have the first immunotherapy that significantly delays the onset of Type 1 diabetes,” says Dr. Bill Russell, TrialNet principal investigator, and director of the Division of Pediatric Endocrinology and Diabetes at Vanderbilt University Medical Center.

VUMC was the largest contributor to the study, providing more than 20% of the participants.

According to Russell, eighty-five percent of the almost 3,000 patients in the Children’s Diabetes Program at Vanderbilt have Type 1 diabetes.

“And we know that relatives of people with Type 1 diabetes are 15 times more likely to develop the disease than the general population, so this is particularly good news for them as well,” he adds.

Type 1 diabetes is an autoimmune disease that occurs when the body’s immune system attacks and destroys insulin-producing beta cells in the pancreas, causing abnormal blood glucose levels.

Teplizumab is an immunotherapy drug designed to interfere with the body’s immune destruction of its own beta cells. The drug is being developed by Provention Bio Inc. and is not yet approved by the FDA.

Previous studies showed teplizumab prolonged insulin production in people recently diagnosed with Type 1 diabetes, but this is the first study to test it in people at high risk for the disease.

“This is the first study to show any drug can delay Type 1 diabetes diagnosis a median of two years in people at high risk,” says Dr. Kevan Herold, Teplizumab Prevention study chair and professor of Immunobiology and Internal Medicine at Yale University. “As anyone with Type 1 diabetes will tell you, every day you can delay this disease is important.”

How was the Trial Designed?

Participants chosen for the study each had a lifetime risk of clinical diagnosis nearing 100%. Fifty-five of the 76 study participants were under age 18 and all had a relative with Type 1 diabetes, two or more Type 1 diabetes autoantibodies, and abnormal blood sugar levels.

“The subject population all had positive diabetes antibodies in their circulation, indicating that their immune systems were targeting their beta cells in the pancreas,” Russell indicates. “They also had impaired glucose tolerance, meaning their glucose response to an oral glucose tolerance test was not normal but it wasn’t yet in the diabetic range.”

He says this is now called “stage 2” of Type 1 diabetes.

Russell says people who are in stage 2, (antibodies plus impaired glucose tolerance) have an 85% likelihood of developing diabetes (stage 3) within five years.

Participants were randomly assigned to either the treatment group, which received a 14-day course of teplizumab, or the control group, which received a placebo.

All participants regularly received glucose tolerance tests until the study was completed, or until they developed clinical Type 1 diabetes – whichever came first.

Seventy-two percent of people in the control group developed clinical diabetes, compared to only 43% of the teplizumab group.

The median time for people in the control group to develop clinical diabetes was just over 24 months, while the median time for the treatment group was 48 months.

TrialNet researchers will conduct additional studies to look for ways to extend the benefits of teplizumab.

“We are now exploring a full-blown prevention trial in people even earlier in the disease process,” Russell says. “And also looking at delaying or preventing progression to stage 3.”

The Teplizumab Prevention Study was funded by the National Institutes of Health (NIH), with additional support from JDRF, the leading global organization funding Type 1 diabetes research.

MacroGenics/Provention Bio donated the study drug and provided funds for additional site monitoring.

The study has been published in the New England Journal of Medicine.

Vanderbilt University Medical Center.


  1. Vanderbilt University Medical Center. Study drug delays Type 1 diabetes in high-risk children and adults. (2019, June 10.) EurekAlert! Retrieved: https://www.eurekalert.org/pub_releases/2019-06/vumc-sdd061019.php