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For the first time, scientists have discovered a drug that can delay the onset of type 1 diabetes in people at high risk of developing autoimmune disease. Some experts call this milestone a milestone in type 1 diabetes research.
In high-risk patients, 14 days of treatment with the experimental drug, teplizumab, delayed the development of the disease by one year or more, according to a study presented Sunday at a meeting of the American Diabetes Association in San Francisco. The results of the study were published simultaneously in the New England Journal of Medicine.
The Phase 2 trial, which studies the effectiveness of a drug in a relatively small number of people, is the first to show that immunotherapy can be used to delay the disease. appearance of a hereditary disease.
"It's an important step. Trials have been under way for a few decades, but they have not prevented diabetes. This is a very disappointing result on the ground, "said Dr. Kevan Herold, lead author of the study, professor of immunology and endocrinology at Yale University. . "This is the first successful attempt to show that it is possible to delay and possibly prevent type 1 diabetes."
The 76 study participants, aged 8 to 49 years, were at high risk for type 1 diabetes, in part because their loved ones had the disease, which destroys the pancreatic beta cells that make the disease. and release insulin. In addition, the volunteers all had tests showing diabetes-related autoantibodies that attack the pancreas, as well as unhealthy blood sugar levels.
Of the 44 volunteers randomly assigned to receive the drug, 19, or 43%, developed diabetes, with the disease appearing within 48.4 months in half of them.
In comparison, among the 32 people who received a placebo, 23, or 72%, developed diabetes, with half of the patients developing it within 24.4 months.
When the study was stopped, the percentage of participants without diabetes was twice as high in the teplizumab group (57%) as in the placebo group (28%).
The main adverse effects were temporarily low levels of lymphocytes – a type of white blood cell – and a rash.
"Not having diabetes is a big problem. Anything that can prevent the disease will cause great excitement, "said Herold. "We look forward to hearing from the FDA on regulatory developments."
The results of the test excited not only the team of Herold, but also the entire scientific community.
Dr. Clifford Rosen, of the Maine Medical Center Research Institute, and Journal Editor Dr. Julie Ingelfinger, wrote in an editorial, "We can finally say that there is had substantial progress in modulating the early course of type 1 diabetes. "
And Dr. Fernando Ovalle, director of the Division of Endocrinology, Diabetes and Metabolism at the University of Alabama in Birmingham, who did not participate in the study, noted that this approach would probably not prevent or completely reverse type 1 diabetes, but could be used in combination with other approaches to eventually achieve a cure.
"I would say that drugs like verapamil, which has been proven to preserve beta cell function in adults with newly diagnosed type 1 diabetes, can play a significant synergistic role in combination with these immunomodulatory treatments, and deserve to be deepened, "Ovalle said.
In the United States, about 1.25 million people have type 1 diabetes, and nearly 18,000 new cases are diagnosed every year in people under 20, the American Diabetes Association announced. The life expectancy of a type 1 diabetic is ten years less than that of unaffected people.
Currently, treatments for type 1 diabetes include:
- Taking insulin by injection, insulin pump or artificial pancreas
- Counting carbohydrates, fats and proteins
- Frequent monitoring of blood glucose
- Eat healthy foods, and
- Exercise regularly and maintain a healthy weight
The promise is that teplizumab, which modifies the white blood cells of the immune system that kill insulin-producing cells in the pancreas, would delay or even prevent the disease in high-risk children, so they do not have to take them. changing life changes.
"We are talking about not having diabetes. It may be the difference between having the disease at 8 or 12 years old or even after high school, so these results are of considerable importance, "Herold told NBC News.
"This is the first immune modulator to change the progression of the disease. This could open a whole new area of investigation on the use of immunotherapy to treat other autoimmune diseases, he added.
The most important impact of this drug seems to have been observed in the first year after treatment, while only 7% of them developed type 1 diabetes, compared to 44% under placebo.
Another set of therapies could further delay the development of diabetes and "that's what we hope to do," said Herold.
He said that there was a reluctance to give the drug in the long run lest it slow down the immune system too much.
"It was felt that to have a really effective treatment, you needed a drug that was administered over a short period of time so that people would not be immunodepressed chronically," he said.
Provention Bio Inc is developing the drug, but additional studies will be needed before regulatory agencies approve it. Currently, the immunotherapeutic drug is not approved by the FDA.
Many new drugs cost more than $ 100,000 a year. If teplizumab was to delay the development of type 1 diabetes for about a year, Herold said, people might think it's worth it.
"You have to talk to a person with diabetes," he said. "I think most people would tell you that a day without diabetes is a great day, because this disease is a disease 24 hours a day, 7 days a week. You can not sleep, you can not eat you can not walk without knowing it. And three-quarters of the people here are children. We are talking about a critical moment in their development. "
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