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Drug that delays onset of type 1 diabetes gets approval in US

In an ongoing trial, the drug teplizumab delayed the onset of type 1 diabetes in children and adults by nearly three years, compared with a placebo

Health 18 November 2022

A person with diabetes injecting insulin

A person with diabetes injecting insulin

kevajefimija/Getty Images/iStockphoto

A new kind of therapy that could delay the start of type 1 diabetes in children by up to three years has been approved for use in the US.

The treatment, called teplizumab, works by partially blocking the immune system’s attack on insulin-making cells in the pancreas and is thought to be the first to postpone the start of any autoimmune condition.

Delaying the onset of type 1 diabetes should make it easier for children to cope with the condition and reduce its toll on their health, says Kevan Herold at Yale University, who was involved in the drug’s development.

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Type 1 diabetes tends to start in children and young adults. Those affected must inject insulin with their meals to prevent their blood sugar levels rising too high. They must also frequently measure their blood sugar levels and control what they eat.

“This is a 24/7 disease – you don’t go to sleep, you don’t eat, without consideration of the disease. Any time without it is of value,” says Herold.

The immune attack happens over several years and involves many aspects of the immune system. If a child is suspected to be at risk, they can be monitored with blood tests for antibodies that damage pancreas cells.

Teplizumab, made by New Jersey-based firm Proventionbio, has been approved for people aged 8 and over who have these antibodies but don’t yet have blood sugar levels so high that they are classed as having diabetes.

The drug is thought to work by lowering the activity of a subset of immune cells called T-cells, which are involved in killing the insulin-making pancreas cells. It is given by infusions in hospital once a day for two weeks.

An ongoing 76-person trial was the basis for the US approval. In the trial, teplizumab is given to people aged between 8 and 49 who don’t have type 1 diabetes, but have a relative with the condition. Among the participants who have developed type 1 diabetes so far, teplizumab delayed the condition’s onset by nearly 3 years, compared with those receiving placebo infusions.

Teplizumab has caused a higher rate of side effects, such as a rash, than the placebo infusions. Despite teplizumab targeting some immune cells, the number of everyday infections is similar between the two groups.

In separate trials, the drug was given to several hundred people with newly diagnosed type 1 diabetes to determine whether it reduces the condition’s severity. So far, the US Food and Drug Administration (FDA) has only approved teplizumab for delaying type 1 diabetes among people who do not yet have the condition.

The new approach opens the possibility that type 1 diabetes could be delayed further by adding in other drugs that affect the immune system, says Herold. “No one is saying this is the final word. Maybe we could give this drug followed by something else.”

The trials recruited children and young adults who had a close relative with the condition, as well as the tell-tale antibodies. This approach wouldn’t pick up everyone who develops type 1 diabetes because many of those affected don’t have a close relative with the condition.

Other screening methods are being investigated, including genetic tests. Those found to be at higher risk would still need periodic tests to see if the immune attack had started.

“If you use multiple genes, you are going to capture a lot more children and adults who at risk of developing type 1 diabetes,” says Rachel Besser at the University of Oxford.

Screening programmes mean children can be diagnosed and start insulin injections earlier. “There’s a great advantage in being able to identify children early,” says Besser. “It prevents hospitalisations, which carry with them trauma and distress.”

Teplizumab is under review by drug regulators in the UK and continental Europe under fast-track pathways designed for particularly innovative new medicines.

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