Researchers from Cardiff University, King's College London, Swansea University, and the University of Calgary have discovered that ustekinumab, a drug widely used to treat psoriasis, could help preserve insulin production in children and young people with early-stage type 1 diabetes.
The clinical trial included 72 patients aged 12 to 18 with recent-onset type 1 diabetes. Of the 62 patients analyzed, 41 received ustekinumab and 21 received a placebo. After 12 months, researchers found that C-peptide levels—a biomarker indicating the body's insulin production—were 49% higher in the ustekinumab group compared to the placebo group.
Precision Medicine Approach
Professor Tim Tree of King's College London explained the mechanism behind the treatment's effectiveness: "We have found that ustekinumab reduces the level of a tiny group of immune cells in the blood called Th17.1 cells. These cells make up only one in 1,000 of blood immune cells, but they seem to play an important role in destroying insulin-producing cells."
This targeted approach represents a significant advancement in precision medicine for type 1 diabetes. "It targets the trouble-making cells, while leaving 99% of the immune system intact," Professor Tree added.
Ustekinumab, sold under the brand name Stelara, works by interfering with the body's inflammatory response by suppressing certain cytokines. It is currently approved for treating severe psoriasis, psoriatic arthritis, Crohn's disease, and ulcerative colitis.
Potential to Reduce Insulin Dependence
Type 1 diabetes occurs when the immune system attacks and destroys insulin-producing beta cells in the pancreas, eventually leaving patients dependent on insulin injections. An estimated 8% of the 5.6 million people with diabetes in the UK have type 1 diabetes.
Dr. Danijela Tatovic of Cardiff University School of Medicine highlighted the significance of the findings: "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 study, published in Nature Medicine, represents the first clinical trial-based evidence for the role of Th17 cells in type 1 diabetes and provides new insights into identifying the specific immune cells that cause the condition.
Early Intervention Potential
Professor Colin Dayan, Clinical Professor at Cardiff University's School of Medicine, emphasized the potential for early intervention: "We tested this treatment in children and adolescents who already needed insulin treatment. It would be better if we could treat them at an earlier stage, while the children are still well, and prevent them needing insulin."
The safety profile of ustekinumab makes it a promising candidate for early intervention. The drug is administered as an injection that patients can give themselves at home and has been used effectively in treating more than 100,000 patients with various immune conditions.
Future Directions
Dr. Peter Taylor of Cardiff University's Systems Immunity Research Institute pointed to promising future applications: "It is now possible with a simple finger-prick antibody test to detect children who will develop type 1 diabetes years before they need insulin. Combining screening in this way with early treatment with ustekinumab seems a very promising approach to preventing the need for insulin."
While the results are encouraging, researchers acknowledge that further clinical trials are needed to confirm these findings and determine which patients would benefit most from the treatment.
Dr. Jessica Farrow, research communications officer at Diabetes UK, commented on the study: "We would now like to see more research carried out in greater numbers of people exploring exactly how treatments like this work to slow or stop type 1, to find and tailor treatments that give each person the best outcome."
The research was funded by a partnership between the Medical Research Council and the National Institute for Health and Care Research, highlighting the collaborative effort to advance treatments for type 1 diabetes that address the underlying immune process rather than simply correcting insulin levels.
