In a groundbreaking development, researchers at the St Vincent’s Institute of Medical Research (SVI) in Melbourne have demonstrated that a commonly prescribed rheumatoid arthritis drug, baricitinib, could effectively impede the progression of type 1 diabetes.
The world-first human trial, led by SVI’s Professor Thomas Kay and published in the New England Journal of Medicine, showcased the drug’s ability to safely preserve the body’s insulin production and suppress the advancement of type 1 diabetes, offering hope for a novel disease-modifying treatment.Â
The study focused on individuals who initiated baricitinib treatment within 100 days of being diagnosed with type 1 diabetes. Professor Kay explained the rationale behind the research, stating, “When type 1 diabetes is first diagnosed, there is a substantial number of insulin-producing cells still present.
We wanted to see whether we could protect further destruction of these cells by the immune system.” The results indicated that baricitinib effectively slowed the progression of the disease in recently diagnosed individuals, offering a potential breakthrough in treatment options.Â
One key aspect of this research lies in the form of administration—baricitinib can be delivered as a tablet, marking a significant departure from traditional insulin therapies that require injections or infusion pumps. Professor Kay expressed excitement about this aspect, stating, “It is tremendously exciting for us to be the first group anywhere in the world to test the efficacy of baricitinib as a potential type 1 diabetes treatment.”Â
During the trial, participants receiving baricitinib demonstrated a reduced need for insulin treatment, providing a positive indicator of the drug’s impact on insulin production. Without treatment, immune cells typically attack and destroy insulin-producing cells, making management of type 1 diabetes an ongoing and demanding task for those diagnosed and their families.Â
Insulin therapy, while life-saving, poses challenges such as potential dangers if administered improperly and the risk of long-term complications, including heart issues, vision impairment, kidney disease, and nerve damage. Professor Helen Thomas, preclinical lead on the trial, expressed optimism about the potential impact of baricitinib, stating, “We are very optimistic that this treatment will become clinically available.
This would be a huge step-change in how type 1 diabetes is managed and we believe it shows promise as a fundamental improvement in the ability to control type 1 diabetes.” The randomized, double-blind, placebo-controlled trial involved 91 participants aged between 10 and 30 years old, all diagnosed with type 1 diabetes within 100 days of starting the trial.
The trial spanned one year, during which researchers closely monitored blood glucose and insulin production. Participants continued their prescribed insulin therapy while being assessed for key parameters such as total daily insulin dose, endogenous insulin production, blood glucose levels, and HbA1C levels—a measure of average blood glucose over the last two to three months.Â
Baricitinib, currently used to treat rheumatoid arthritis, functions by blocking an enzyme involved in immune system regulation and inflammation. The drug’s ability to dampen the immune response against insulin-producing cells in newly diagnosed type 1 diabetes patients suggests its potential to delay full-blown symptoms, enhance glucose control, and mitigate long-term health risks.Â
This research not only highlights the promise of baricitinib as a groundbreaking treatment for type 1 diabetes but also emphasizes the potential shift in diabetes management paradigms, offering hope for improved control and reduced complications for individuals living with this lifelong autoimmune disease.Â
Journal Reference Â
Baricitinib and β-Cell Function in Patients with New-Onset Type 1 Diabetes, New England Journal of Medicine (2023). DOI: 10.1056/NEJMoa2306691Â


