Experimental Drug May Fight Type 1 Diabetes

By Amy Norton
HealthDay reporter

MONDAY, March 8, 2021 (HealthDay News) – Just two weeks of treatment with an investigational drug can delay the onset of type 1 diabetes by several years, researchers report.

The drug, called teplizumab, is already under review by the United States Food and Drug Administration based on previous evidence of its effectiveness.

If it gets the green light, it would become the first drug approved to delay type 1 diabetes in people at high risk.

In the previous study, researchers found that two weeks of teplizumab infusions generally kept the disease at bay for two years, compared to a placebo.

In this latest follow-up, investigators found that half of the patients receiving teplizumab were still diabetes-free five years later, compared to just 22% of the placebo group.

Initially, the patients in the study, most of whom were under 18, were almost certain they would develop type 1 diabetes: they had a relative with the disease and already had “autoantibodies” in their blood. , which is a sign the immune system was starting to attack the body’s own cells.


Type 1 diabetes occurs when the immune system mistakenly attacks cells in the pancreas that produce the hormone insulin. Insulin has the essential job of moving sugars from food to body cells for use as fuel.

To survive, people with type 1 diabetes must take synthetic insulin, either through daily injections or from a pump attached to the body.

How does the new drug work? Teplizumab is a laboratory-designed antibody that targets specific immune cells, interfering with their ability to deactivate and destroy insulin-producing cells.

It is not yet clear whether the drug could completely prevent type 1 diabetes in some people.

But it can prevent disease, which is essential, according to Sanjoy Dutta, vice president of research at JDRF, a nonprofit organization that funds research into type 1 diabetes.

The disease often strikes in childhood and after many years people frequently develop complications such as heart, kidney and eye disease, as well as severe nerve damage.

Delaying diabetes, especially in children, “is like money in the bank,” Dutta said.


The longer people can live with their own functioning beta cells (the cells that make insulin) the better, Dutta explained. They will have better long-term control of their blood sugar, and later this could reduce their risk of complications.

The teplizumab trial was sponsored by the Type 1 Diabetes TrialNet Study Group, a network funded by the US National Institutes of Health and JDRF.

The drug, developed by biotech company Provention, has received FDA “breakthrough” status, which means it will undergo expedited review. Approval could come as early as this summer, according to Dr. Kevan Herold, a professor at Yale University who led the trial.

These latest findings, published online on March 3 in the journal Scientific translational medicine, are based on 76 patients who were randomly assigned to two weeks of teplizumab (given intravenously) or to placebo.

Among the patients who received the real drug, the median time to diagnosis of diabetes was five years (meaning that half were still disease-free at that time). In the placebo group, that figure was just over two years, for a difference of almost three years.


Beyond that, the beta cell function of teplizumab patients improved.

According to Stephan Kissler, a researcher at the Joslin Diabetes Center in Boston who was not involved in the trial, “These results show not only that the drug can delay diabetes, but that it can also improve the function of the pancreas before the diabetes is not diagnosed. a convincing sign that the drug can stop the progression of the disease – at least for a significant period of time. “

There are still key questions that need to be answered.

Dutta said the trial, mostly involving children, turned conservative with the dose of teplizumab. So now researchers are wondering if a higher dose – perhaps giving a “booster”, similar to a vaccination – would be even more effective, he noted.

Kissler said the current study is “far from over,” as many participants remained diabetes-free.

“Teplizumab will be sufficient to completely prevent diabetes in some people is uncertain,” he said. “But these remarkable results give us hope that comprehensive prevention will one day be achievable.”


That, Kissler said, could come from teplizumab or other immune system therapies. He noted that researchers are developing drugs to protect beta cells from immune aggression and that it is possible that such therapies enhance the protective effect of teplizumab.

If teplizumab is approved, it will amplify the importance of identifying people with type 1 diabetes autoantibodies. Screening tests are available, but not yet widely performed.

According to JDRF, relatives of people with type 1 diabetes can get automatic antibody tests free of charge through the TrialNet study.

More information

JDRF has information on testing for type 1 diabetes autoantibodies.

SOURCES: Sanjoy Dutta, PhD, vice president, research, JDRF, New York; Stephan Kissler, Ph.D., research fellow, section of immunobiology, Joslin Diabetes Center, and associate professor, medicine, Harvard Medical School, Boston; Scientific translational medicine, March 3, 2021, online

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