An international consortium involving clinicians and researchers from Australia, North America and Europe recently completed a randomised controlled clinical trial to determine if the immune therapy teplizumab could delay the development of type 1 diabetes.
The study published today in the New England Journal of Medicine ran for five years and involved 76 children and young adults at very high risk of developing type 1 diabetes. The findings revealed that teplizumab delayed the onset of diabetes by two years.
Clinician scientist at the Institute and The Royal Melbourne Hospital Associate Professor John Wentworth, who led the Australian arm of the Type 1 Diabetes TrialNet Study, said the result was a game changer for people living with type 1 diabetes.
“These results are incredibly encouraging. We have known for over three decades how to identify children who are destined to develop type 1 diabetes. Now, for the first time, we have something that will improve their prospects and delay the need to start insulin injections to control blood sugar levels,” he said.
The researchers identified the 76 high-risk study participants by screening more than 200,000 people with a family connection to type 1 diabetes from around the world. Of these, 44 received a 14-day course of teplizumab injections and 32 received placebo injections. Participants were then monitored to see how quickly they developed diabetes.
Over five years, 72 per cent of people who received the placebo developed diabetes compared to only 43 per cent who received teplizumab. The development of diabetes also occurred two years later in the teplizumab group.
“This extremely important finding demonstrates that immune therapy can delay type 1 diabetes. This will provide us with the evidence to perform more clinical trials so that we can develop better immune therapies for type 1 diabetes.
“This result also provides hope to families living with type 1 diabetes. Previously, we could offer a blood test that would indicate whether a child was likely to develop diabetes. Now we can actually do something to help these at-risk children and delay their diabetes.”
The Royal Melbourne Hospital and Walter and Eliza Hall Institute’s long-term pioneering research expertise into type 1 diabetes is an important contribution to the Type 1 Diabetes TrialNet Study.
Associate Professor Wentworth said the outcomes of the trial would continue with further research at the Institute. “Samples from the study will help us develop better ways to identify the ‘immune storm’ that we believe pushes people into a diagnosis of type 1 diabetes. A better test will help us work out the best time to treat people with immune therapy and to monitor its effectiveness,” he said.
This trial is being run in conjunction with several other trials that will likely have major changes for diabetes worldwide.
The study is supported by the Type 1 Diabetes TrialNet Study Group, a clinical trials network funded by the National Institutes of Health (NIH) through the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of Allergy and Infectious Diseases, and The Eunice Kennedy Shriver National Institute of Child Health and Human Development.
For information about type 1 diabetes trials, please contact research nurses Felicity Healy and Leanne Redl at The Royal Melbourne Hospital: +61 3 9342 7063.
M: +61 475 751 811