Australian World-First Trial Offers New Hope For Type 1 Diabetes

Researchers at St Vincent’s Institute of Medical Research (SVI) in Melbourne have shown that a commonly prescribed rheumatoid arthritis drug can stop the progression of type 1 diabetes.  

The world-first human trial, published today in the New England Journal of Medicine and led by SVI’s Professor Thomas Kay, showed that a drug called baricitinib can safely and effectively preserve the body’s own insulin production and suppress the progression of type 1 diabetes in people who initiated treatment within 100 days of diagnosis.    

“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. We showed that baricitinib is safe and effective at slowing the progression of type 1 diabetes in people who have been recently diagnosed,” said Professor Kay.  

This ground-breaking research shows promise as the first disease-modifying treatment of its kind for type 1 diabetes that can be delivered as a tablet.  

“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,” said Professor Kay.  

“Up until now, people with type 1 diabetes have been reliant on insulin delivered via injection or infusion pump. Our trial showed that, if started early enough after diagnosis, and while the participants remained on the medication, their production of insulin was maintained. People with type 1 diabetes in the trial who were given the drug required significantly less insulin for treatment.”   

Management of the lifelong autoimmune disease is incredibly burdensome on those diagnosed and their families, requiring meticulous glucose monitoring and insulin administration day and night to stay alive.   

Up until insulin’s discovery more than 100 years ago, type 1 diabetes was a fatal condition. Despite insulin’s life-saving role, the therapy itself is potentially dangerous if too much or too little is administered, and the condition still comes with long-term complications, including heart attack and stroke, vision impairment, kidney disease and nerve damage.   

“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,” said Professor Helen Thomas, preclinical lead on the trial.  

The clinical trial was funded by JDRF, the leading type 1 diabetes research, advocacy and community programs organisation, including through the JDRF Type 1 Diabetes Clinical Research Network. Partners included The Royal Melbourne Hospital, St Vincent’s Hospital Melbourne, The Royal Children’s Hospital and The Women’s and Children’s Hospital in Adelaide.