A groundbreaking study has revealed that Ustekinumab, a drug initially developed for treating psoriasis, may offer new hope for children and adolescents with early-stage type-1 diabetes. Funded by a collaborative partnership between the National Institute for Health Research (NIHR) and the Medical Research Council (MRC), the research provides promising evidence that this drug could help preserve insulin production in young diabetes patients.
Published in Nature Medicine, the Cardiff University-led study highlights Ustekinumab’s potential in managing type-1 diabetes without the immediate need for insulin. Ustekinumab, an established immunotherapy since 2009, is administered via injection and has been used to treat over 100,000 patients with various immune conditions, including severe psoriasis, psoriatic arthritis, Crohn’s disease, and ulcerative colitis.
The clinical trial evaluated the drug’s effectiveness on 72 adolescents aged 12 to 18 who had recently been diagnosed with type-1 diabetes. Dr. Danijela Tatovic from Cardiff University’s School of Medicine explained that type-1 diabetes results from the immune system attacking and destroying insulin-producing cells in the pancreas. This destruction typically leads to a lifelong dependency on insulin injections. By targeting this immune response early, it is possible to preserve insulin-producing cells and potentially reduce or eliminate the need for insulin therapy.
The study’s findings demonstrate that Ustekinumab not only helps preserve vital insulin-producing cells but also identifies the specific immune cells responsible for their destruction. This advancement allows for more precise and targeted treatments that maximize therapeutic benefits while minimizing side effects.
Professor Tim Tree of King’s College London noted that Ustekinumab effectively reduces the levels of Th17.1 cells, a rare subset of immune cells implicated in the destruction of insulin-producing cells. “These cells constitute just one in a thousand of blood immune cells but play a significant role in the autoimmune attack on insulin-producing cells. Ustekinumab’s targeted approach minimizes side effects by focusing on these problematic cells while preserving the majority of the immune system,” Tree explained.
Professor Colin Dayan, a Clinical Professor at Cardiff University’s School of Medicine, emphasized the advantage of using Ustekinumab at an earlier stage of diabetes development. “While this study was conducted on children already needing insulin, the optimal approach would involve treating them before insulin dependence develops. Ustekinumab’s favorable safety profile makes it a viable option for early intervention.”
The research showed that after 12 months of Ustekinumab treatment, C-peptide levels—an indicator of insulin production—were 49% higher in participants, highlighting the drug’s role in preserving insulin production. This is the first clinical evidence demonstrating the involvement of Th17 cells in type-1 diabetes.
Additionally, the study underscores the potential of combining early screening with Ustekinumab treatment. Dr. Peter Taylor from Cardiff University’s Systems Immunity Research Institute pointed out that advances in early detection through simple antibody tests could allow for preventative measures before insulin dependence occurs. “Integrating early screening with Ustekinumab treatment offers a promising strategy for preventing type-1 diabetes progression,” Taylor said.
The study also involved collaboration with researchers from King’s College London, Swansea University, and the University of Calgary. Funded by the Efficacy and Mechanism Evaluation (EME) Programme, a joint initiative of the NIHR and MRC, the USTEKID study paves the way for further research to confirm these findings.
For additional information about the study, visit the NIHR and MRC Funding and Awards website.
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