Type 1 Diabetes Drug, Teplizumab, Has Additional Benefits, New Study Shows

Teplizumab is a monoclonal antibody that modifies T cells to prolong the pancreas’ ability to create insulin. The drug is specific to a molecule called CD3, which is the “cognate” component of the T cell, Dr. Herold explains. This modulates the immune cells and prevents them from attacking the cells in the pancreas that produce insulin.

The medication is delivered through a daily infusion for 12 days and does not need to be taken continuously, which is another benefit as many treatments need to be done every day, Dr. Herold says.

“Diabetes is a disease that is with you literally every minute of the day. You don’t sleep, exercise, or eat without thinking about your metabolic control,” says Dr. Herold. “People with diabetes will tell you that any time without the disease is a gift, particularly for young children and their parents. Some ask, ‘If you’re still going to get diabetes, what’s the big deal?’ But when you’re an eight-year-old child, if the time at which you need to take insulin, follow a prescribed diet, and monitor your blood sugar is delayed for two more years, that’s huge. Half of our patients have experienced a delay of even greater periods of time—even as long as 11 years.”

In childhood, those extra years matter—a high-schooler, for instance, may be better equipped to handle the burden of a chronic disease than a middle-schooler. The delay also reduces the time a patient is exposed to the high blood sugars that cause a wide range of health complications. As therapies for diabetes continue to evolve, there will likely be more improved and convenient options for patients.

The PROTECT trial included 328 patients ages 8 to 17 who were randomly assigned to receive teplizumab or a placebo for two 12-day courses 26 weeks apart. (The pandemic lengthened the time to 52 weeks for some patients.)

The trial showed that the course of medication was successful in preserving beta cell function. Participants who received teplizumab also were able to reduce their use of insulin, had a lowered risk of severe hypoglycemia (low blood sugar), and were more likely to spend more time in their target glucose range. Furthermore, those who received the drug were more likely to reach the clinical threshold for disease remission, and they showed improvement in quality-of-life measures.

Dr. Herold says the promising news with teplizumab is that it is the starting point to prevent the disease from occurring or even to restore lost beta cells. Combining treatments may prolong and enhance responses in those at risk for type 1 diabetes. In addition, replacing the insulin-producing cells that have been destroyed—even with stem cell-derived beta cells, together with teplizumab, may be an effective combination, he adds.

“This is an exciting step toward our goal of eliminating type 1 diabetes,” says Dr. Herold, who is also a member of Yale’s Human and Translational Immunology Program, which works to accelerate the application of developments in the field of immunology to treat diseases.

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