Type 1 diabetes: Drug delays onset by 2 years

New research finds that the immunotherapy drug teplizumab delays the onset of type 1 diabetes by 2 years, on average, in high-risk individuals.
young man taking his blood sugar
New research has significant clinical implications, particularly for young people with a high risk of type 1 diabetes.

Type 1 diabetes is an autoimmune disease affecting about 1.25 million children and adults in the United States.

Some people have a higher risk of developing type 1 diabetes than others. Age influences risk; this condition is one of the most common chronic ones to emerge in childhood.

Males are more likely to develop type 1 diabetes than females, and having a family history of the disease also increases the chances of developing it.

Geography also seems to play a role in type 1 diabetes risk. For instance, Sweden, Finland, Norway, the United Kingdom, and Sardinia have the highest incidence of type 1 diabetes, whereas China and South American countries have the lowest.

For people whose risk is high, a new study brings some interesting and hopeful insights. Researchers — led by Dr. Kevan C. Herold, from Yale University, in New Haven, CT — have found that a drug called teplizumab can delay the onset of type 1 diabetes in people with a high risk.

Dr. Herold and the team published their findings in The New England Journal of Medicine and presented them at the American Diabetes Association’s Scientific Sessions, in San Francisco, CA.

Studying teplizumab in high-risk people

Teplizumab is an anti-CD3 monoclonal antibody. It affects the immune system by targeting effector T cells — a type of immune cell that, in type 1 diabetes, destroys insulin-producing beta cells.

Previous trials have shown that teplizumab reduces the loss of beta cells in people with new onset of type 1 diabetes.

In the new study, Dr. Herold and colleagues examined the effect of the drug on 76 participants who had relatives with type 1 diabetes and had at least two types of autoantibodies associated with diabetes.

Autoantibodies are proteins that the immune system produces.

The participants were 8–49 years old, and they also had abnormal blood sugar tolerance. The scientists randomly divided them into two groups.

One of the groups received teplizumab for 14 days, while the control group just received a placebo. The researchers tested the participants’ glucose tolerance regularly throughout the study.

Teplizumab delays onset by 24 months

By the end of the trial, 72% of the people in the placebo group had developed type 1 diabetes, whereas only 43% of the people in the teplizumab group had developed the condition.

Furthermore, in the control group, people developed diabetes over a median period of 24 months, whereas in the treatment group, participants developed the condition after a median of 48 months.

“The difference in outcomes was striking. This discovery is the first evidence we’ve seen that clinical type 1 diabetes can be delayed with early preventive treatment,” comments Lisa Spain, Ph.D., a project scientist at the National Institute of Diabetes and Digestive and Kidney Diseases, which is part of the National Institutes of Health (NIH).

The results have important implications for people, particularly youth[s] who have relatives with the disease, as these individuals may be at high risk and benefit from early screening and treatment.”

Lisa Spain, Ph.D.

The study’s lead author also comments on the findings, saying, “Previous clinical research funded by the NIH found that teplizumab effectively slows the loss of beta cells in people with recent-onset clinical type 1 diabetes, but the drug had never been tested in people who did not have clinical disease.”

“We wanted to see whether early intervention would have a benefit for people who are at high risk but do not yet have symptoms of type 1 diabetes,” he explains.

More research is necessary

However, the researchers also caution that the study has some limitations, such as the small number of participants, the fact that the study sample was not very ethnically diverse, and that all the participants had relatives with type 1 diabetes, which could mean that the study’s findings are not easy to generalize.

Also, the researchers need to dig deeper in order to understand why some people responded to treatment better than others. Certain immune system characteristics may play a role.

“While the results are encouraging, more research needs to be done to address the trial’s limitations, as well as to fully understand the mechanisms of action, long-term efficacy, and safety of the treatment,” says Spain.

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