Psoriasis drug shows promise for treating type 1 diabetes

Daisy Abbs, who lives with type 1 and took part in the USTEKID clinical trial.

Results from a clinical trial named USTEKID, published in the journal Nature Medicine, suggest that ustekinumab may be effective in treating the early stages of type 1 diabetes in children and adolescents.

Protecting insulin-making cells

Co-funded by JDRF, the study demonstrated that ustekinumab is effective in preserving the body’s ability to produce insulin in type 1 diabetes, bringing the goal of managing type 1 diabetes without insulin a step closer. After 12 months, the participants who were taking ustekinumab had C-peptide levels – a sign that the body is producing insulin – that were 49% higher than the group who were taking a placebo.

The study showed that ustekinumab reduced the destructive impact of specific immune cells on insulin-making beta cells. The drug treats the underlying immune process that causes type 1 diabetes, making it different from insulin injections that top up the lack of insulin for people with type 1.

Reducing the need for insulin

Dr Danijela Tatovic, who led the research project, said: “Type 1 diabetes occurs when the body’s immune system attacks and destroys the cells of the body that produce insulin. This eventually leaves the person dependent on insulin injections. Researchers are now developing ways to slow or halt the immune system attack. If such treatments can be started early, before all the insulin-making cells are lost, this could prevent or reduce the need for insulin.”

The USTEKID clinical trial

Led by researchers at Cardiff University, the clinical trial tested ustekinumab in 72 young people between 12 and 18 years old who were within 100 days of being diagnosed with type 1 diabetes. At universities and hospitals across the UK, the teenagers were given injections of either ustekinumab or a placebo seven times over 44 weeks. The participants also gave blood and urine samples and completed three short questionnaires.

What is ustekinumab?

Ustekinumab is an established immunotherapy, meaning it targets the immune cells in the body. It is used by more than 100,000 people in the UK living with immune conditions, including severe psoriasis, psoriatic arthritis, severe Crohn’s disease and severe ulcerative colitis. The drug is given as an injection, which people can give themselves at home.

A precision medicine

Professor Tim Tree, who led the research team at King’s College London, said: “We have found that Ustekinumab reduces the level of a tiny group of immune cells in the blood. These cells make up only 1 in 1,000 of blood immune cells, but they seem to play an important role in destroying insulin-producing cells. This explains why ustekinumab has so few side effects. It targets the trouble-making cells, while leaving 99% of the immune system intact – a great example of precision medicine.”

Crucial JDRF funding

The USTEKID trial provides the first clinical trial-based evidence for the role of this type of immune cell in type 1 diabetes. Identifying the specific immune cells that cause damage to the insulin-making beta cells could pave the way for precise and targeted therapies to maximise benefits and minimise side effects for people living with type 1.

Professor Tim Tree said: “JDRF funding was instrumental in the biomarker experiments in the project where we examined blood samples from the trial participants. This was the key to linking a reduction in these cells with a good outcome in those treated with the drug. This offers opportunities to personlise therapy to those most likely to benefit or test newer therapies that might target this population of cells more quickly and potently.”

More research needed

While the trial demonstrates the benefit of using ustekinumab to treat type 1 diabetes, further clinical trials are required to confirm this finding and to work out which patients would benefit most from the treatment. The researchers also hope to test whether this drug could be used to treat individuals at earlier stages of type 1, before they ever need insulin therapy.