TrialNet Study - Immunotherapy can delay type 1 diabetes onset

In spring 2019, results of an exciting clinical trial revealed that the use of an immunotherapy drug called Teplizumab delayed the onset of type 1 diabetes (T1D) for an average of two years in children and adults at high risk. The innovative study, which provided the first concrete evidence that prevention of T1D may be possible, was conducted by TrialNet and the findings were presented at the American Diabetes Association’s 79th Scientific Sessions and published in the New England Journal of Medicine. In 2020, continued follow-up of trial participants showed that the drug can in fact delay the onset of T1D for even longer – as much as three years.

Immunotherapy is a promising form of treatment that could one day cure T1D. It involves the use of drugs that target the immune system to prevent or reverse autoimmunity while leaving the body’s ability to fight infectious diseases or cancer intact.

All participants in TrialNet’s Teplizumab study were relatives of people with T1D and were identified through blood tests to have close to a 100% chance of developing the disease in their lifetime. Of the 76 high-risk individuals who took part in the trial, 55 were under the age of 18. Funded by JDRF and the US National Institutes of Health (primarily through the Special Diabetes Program) this research represents a major breakthrough in the area of T1D prevention. While previous studies showed Teplizumab prolonged insulin production in people recently diagnosed with T1D, this is the first to demonstrate that a drug can delay onset of the disease in high-risk individuals.

“Delaying the onset of type 1 diabetes is the only approach in the short-term to impact the incidence and prevalence of the disease,” said Dave Prowten, JDRF Canada’s president and chief executive officer. “This study marks an incredible advancement in research that bring us closer to our goal of turning type one into type none.”

In August 2019, Teplizumab was granted a Breakthrough Therapy Designation by the US FDA, a pathway that expedites the advancement and review of new drugs for serious diseases. Provention Bio, the company developing the drug, is supported by JDRF through the T1D Fund. Further studies of Teplizumab are in progress, including the PROTECT study – a clinical trial supported by Provention Bio that is testing whether the drug can prevent progression of T1D in children who have just been diagnosed. The PROTECT study includes Canadian sites and will begin enrolment in 2020.