The US Food and Drug Administration approves the first treatment to delay the onset of type 1 diabetes | CNN


A biological treatment that delays the onset of type 1 diabetes received approval from the US Food and Drug Administration on Thursday.

It is the first approved treatment for the prevention of type 1 diabetes.

The monoclonal antibody teplizumab, which will be marketed under the brand name Tzield, from ProventionBio and Sanofi is given through an intravenous infusion.

It is thought to work by preventing the body’s faulty attack on insulin-producing cells. The idea is that protecting these cells buys people more time before they become dependent on insulin to manage their condition.

In clinical trials, Tzield delayed progression to full-blown diabetes by just over two years. But the benefits lasted longer in some study participants.

One of them, Michaela Olsten, was screened for diabetes after her 9-year-old sister, Mia, suddenly had a life-threatening episode of diabetic ketoacidosis and was diagnosed with diabetes. There was no history of diabetes in the family, and Michaela was not ill, but she did have four of the five types of autoantibodies that doctors look for to assess a person’s risk.

“They tell us that when someone has many signs, it’s not if they’re going to get diabetes, but when,” her mom, Tracy, said.

Michaela was 15 years old when she joined the study and got teplizumab. She is now 21 years old and a student in college. She gets an annual batch of tests to check her pancreas and blood markers, and Tracey Olsten says her condition hasn’t progressed in six years.

According to a scientific statement from the Juvenile Diabetes Research Foundation, the Endocrine Society, and the American Diabetes Association, when a person has signs of autoimmune diseases and episodes of uncontrolled blood sugar, the risk of developing insulin-dependent diabetes over a five-year period is 75%. The lifetime risk of developing insulin-dependent diabetes is almost 100%.

So far, Michaela seems to be beating those odds.

Tracy said that for Mia, who is insulin dependent, managing her diabetes is a constant chore.

“She has a tremendous amount of juggling that her peers don’t have to do. She has to plan ahead when she has a basketball game or practices to make sure she’s upping her carbs and lowering her insulin levels,” Tracy said. Or a day without thinking about it nonstop, and being able to give Michaela the opportunity where she doesn’t have to think about it 24/7 is amazing.”

A major challenge in prescribing Zelda is finding people who need it, says Aaron Kowalski, CEO of the Juvenile Diabetes Research Foundation. The drug is approved for people who don’t have any symptoms of the disease and may not know they’re going to get it.

“Screening becomes a really big issue, because what we know is that about 85% of type 1 diagnoses today are in families with no known family history,” Kowalski said. “Our goal is to do a general screening of the population” with blood tests to look for signs of disease.

Tzield is approved for use in people 8 years of age or older who are in stage 2 type 1 diabetes. At this stage, doctors can measure antibodies that attack the insulin-producing beta cells in a person’s blood, and they have abnormal levels of sugar. in the blood, but their bodies can still produce insulin.

“The way that not only the industry but our medical system deals with autoimmune diseases, especially type 1 diabetes, is really suboptimal in today’s day and age,” said Ashley Palmer, co-founder and CEO of ProventionBio. What we do is we wait until the symptoms of the disease appear to the doctors, and then the doctors treat the patient’s symptoms chronically for life. The problem is that in type 1 diabetes, when symptoms first appear, it’s too late.”

Treatment comes in one cycle of injections for 14 days each lasting 30 to 60 minutes.

The most common side effects reported in participants were decreased white blood cells and lymphocytes, rash, and headache.

In type 1 diabetes, a person’s immune system attacks cells called beta cells in the pancreas that produce insulin, a hormone that helps blood sugar enter cells, where it is used for energy. An attack can occur for years before any symptoms of diabetes appear. Without insulin, blood sugar can build up in the bloodstream and break down body fat and muscle.

Palmer says Tzield stops disease before symptoms appear by stopping the autoimmune disease process and the underlying destruction of beta cells. The treatment essentially restarts the immune system, preserving beta cell function.

“We don’t really have any preventative measure for type 1 diabetes yet, though [the National Institutes of Health] said Dr. Robert Gabay, chief scientist and medical officer for the American Diabetes Association. “Finally, there is something that delays the onset of type 1 diabetes, which is very exciting.”

Unlike type 2 diabetes, which can be prevented with lifestyle changes like losing weight and exercising, type 1 is a genetic disease that hasn’t had any prevention options yet.

“For some reason, we don’t screen for type 1 diabetes, even though there are biomarkers available to show that the autoimmune disease process is really underway,” Palmer said. He added that hopes that the drug will motivate the medical system to start screening residents during routine childhood visits in order to intercept the disease and delay its onset.

With Tzield, doctors check family members of people with type 1 diabetes to see if they have those specific antibodies. If antibody levels are high and the person appears to be on the verge of developing diabetes, treatment will delay this process.

“If someone has type 1, a common question that arises is, ‘Well, what about my child?’ Will they develop type 1? “It’s only a 5% risk, and more often than not, they won’t, but if you can find people who will treat it, it can make a big difference,” Gabbay said.

A late diagnosis of type 1 diabetes can have a significant impact.

“Obviously, quality of life is greatly affected and negatively affected if you are diagnosed with type 1 diabetes. It is a disease that never goes away,” Palmer said.

People with type 1 diabetes must monitor their blood glucose levels around the clock, which affects how they exercise and eat. High blood sugar can lead to diabetic ketoacidosis, where the body begins to break down fat as its fuel, and can cause acids called ketones to build up in the bloodstream. This condition can lead to hospitalization, coma, or death.

As of 2019, about 1.9 million people have type 1 diabetes in the United States, according to the American Diabetes Association, including 244,000 children and teens. Type 1 affects 8% of people with diabetes.

“Type 1 infection occurs mainly in children and adolescents, and when you’re in adolescence, that’s when you just want to forget that you have it,” said Olivier Boguelot, president of Sanofi’s generics division in the US. “So when you have the potential for treatment to delay the onset of the disease, you can change the way quality of life is affected for families and those children.”

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