Professor Tom Kay explains the research

World-first research offers new hope for type 1 diabetes

SVI researchers have shown that a commonly prescribed rheumatoid arthritis drug can suppress the progression of type 1 diabetes. 

The world-first human trial, led by Professors Thomas Kay and Helen Thomas, 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.  

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.

Find out more about type 1 diabetes and the results of the clinical trial

About the research

  • Type 1 diabetes

    Type 1 diabetes affects more than 130,000 Australians. 

     Every day, seven Australians are diagnosed with this condition, many of them children.    

    Type 1 diabetes occurs when the body’s immune system, which normally protect us from infection, mistakenly kill the cells in the pancreas that produce insulin. Once they have been killed, these cells are gone forever.   

     Once diagnosed, people with type 1 diabetes are dependent on insulin – given by injection or infusion pump – for the rest of their lives.  

     Insulin, while effective, has considerable limitations. This is because it is not able to mimic the key properties of a healthy pancreas – where the insulin-producing cells can detect changes in circulating blood glucose levels almost immediately, and react instantaneously.   

     Reliance on insulin as a solution creates significant issues – not so much because of insulin itself, but because of the difficulty in monitoring and managing glucose levels appropriately and effectively.   

    The science

    On average, bringing a new drug to market costs $2-3 billion and takes more than 13 years of intense study and clinical trials. Many drugs fall at one or other of the hurdles put before them – they have unintended side effects, or are toxic, or patients don’t show the benefits that have been seen in animal models of disease. Because of the great investment in time, money and resources spent on getting a drug to the clinic, researchers are increasingly combing through the medicine chest of approved drugs to identify those that might work for a different indication. 

    The randomised, double-blind, placebo-controlled human trial of the drug baricitinib monitored the blood glucose and insulin production of 91 participants over the course of one year.  Of these, 60 were given baricitinib and 31 were given a placebo. All trial participants were aged between 10 years old and 30 years old and started on the trial within 100 days of having been diagnosed with type 1 diabetes. Participants continued with their prescribed insulin therapy throughout the duration of the study. Researchers monitored participants’ total daily dose of insulin, the amount of insulin produced endogenously (by their own pancreas), their blood glucose levels, and their HbA1C levels. HbA1c, or glycated haemoglobin, is a measure of average blood glucose (sugar) levels for the last two to three months. 

    Baricitinib blocks an enzyme which normally helps to transmit signals that regulate the immune system and inflammation. The drug is currently prescribed for the treatment of rheumatoid arthritis, which is another autoimmune disease. It is thought that the drug similarly dampens down the immune response that is mounted against insulin-producing cells in people with newly diagnosed type 1 diabetes, thus delaying the onset of full-blown symptoms of the disease, improving glucose control and reducing potential for detrimental longer term health effects.  

     

    The team

    SVI

    Professor Tom Kay
    Trial Lead

    Professor Helen Thomas
    Lead Scientist

    Associate Professor Balasubramanian Krishnamurthy
    Senior clinician researcher

    Dr Michaela Waibel
    Project Manager

    Dr Michelle So

    Site investigators

    Associate Professor John Wentworth
    Lead Clinician (The Royal Melbourne Hospital)

    Professor Fergus Cameron
    Site Leader (The Royal Children’s Hospital Melbourne)

    Professor Jenny Couper
    Site Leader (Adelaide Women’s and Children’s Hospital)

    Professor Richard MacIsaac
    Site Lead (St Vincent’s Hospital Melbourne)

    Professor Peter Coleman
    Royal Melbourne Hospital

    Dr Gabby Atlas
    Royal Children’s Hospital

    Dr Jess Harbison
    University of Adelaide

    Professor Len Harrison
    WEHI

    Publications
    1. Waibel M, Wentworth JM, So M, Couper JJ, Cameron FJ, MacIsaac RJ, Atlas G, Gorelik A, Litwak S, Sanz-Villanueva L, Trivedi P, Ahmed S, Martin FJ, Doyle ME, Harbison JE, Hall C, Krishnamurthy B, Colman PG, Harrison LC, Thomas HE, Kay TWH; BANDIT Study Group. Baricitinib and β-Cell Function in Patients with New-Onset Type 1 Diabetes. N Engl J Med. 2023 Dec 7;389(23):2140-2150. doi: 10.1056/NEJMoa2306691. PMID: 38055252.
    2. Donaldson LE, Vogrin S, So M, Ward GM, Krishnamurthy B, Sundararajan V, MacIsaac RJ, Kay TWH, McAuley SA. Continuous Glucose Monitoring-Based Composite Metrics: A Review and Assessment of Performance in Recent-Onset and Long-Duration Type 1 Diabetes. Diabetes Technol Ther. 2023 Jul;25(7):497-506. doi: 10.1089/dia.2022.0563. Epub 2023 May 8. PMID: 37010375.
    3. Waibel M, Thomas HE, Wentworth JM, Couper JJ, MacIsaac RJ, Cameron FJ, So M, Krishnamurthy B, Doyle MC, Kay TW. Investigating the efficacy of baricitinib in new onset type 1 diabetes mellitus (BANDIT)-study protocol for a phase 2, randomized, placebo controlled trial. Trials. 2022 May 23;23(1):433. doi: 10.1186/s13063-022-06356-z. PMID: 35606820; PMCID: PMC9125350.