World-first trial offers new hope for type 1 diabetes

(Press-News.org) Researchers at St Vincent’s Institute of Medical Research (SVI) in Melbourne have shown that a commonly prescribed rheumatoid arthritis drug can suppress 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.  

About St Vincent’s Institute  

St Vincent’s Institute of Medical Research (SVI) is a Melbourne-based, independent medical research institute that conducts biomedical research into the cause, prevention and treatment of high-impact diseases such as cancer, heart disease, diabetes, obesity, bone diseases and Alzheimer’s. SVI is affiliated with St. Vincent’s Hospital and the University of Melbourne. www.svi.edu.au.  

Background information  

About type 1 diabetes   

Type 1 diabetes is an autoimmune disease in which the body’s immune system mistakenly attacks and kills the cells in the pancreas that produce insulin.  

People with type 1 diabetes are dependent on externally administered insulin in order to survive.  

Type 1 diabetes is most commonly diagnosed in children, but adults can also be diagnosed with the disease.  

More than 130,000 Australians have type 1 diabetes – we have one of the highest rates of type 1 diabetes in the world. In 2022, around 4,000 people were newly diagnosed with type 1 diabetes in Australia.  

The estimated health care cost in Australia for treating people with type 1 diabetes in 2012 was $570 million annually (latest figures available).  

  More about 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 attached videos demonstrate the ‘life and death of a beta cell’, and what happens in the lab setting when a similar drug is used in cell culture with insulin-producing cells incubated together with immune cells. In the first video, without the treatment, the immune cells attack and kill the insulin-producing cells. When the drug is included in the second video, the immune cells are disabled and are unable to kill the insulin-producing cells.   

https://youtu.be/yPovcqYu8a4  

https://youtu.be/Cs7UgvQcrko  

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