“These study results demonstrating that we can delay the onset of type 1 diabetes are of great worldwide importance for persons impacted by Type 1 diabetes, especially the relatives of people with Type 1 diabetes who are themselves at 15 times greater risk of developing the disease than the general U.S. population,” said Linda DiMeglio, MD, MPH, TrialNet Principal Investigator and professor of pediatrics at Indiana University School of Medicine.
IU School of Medicine is one of 28 sites that participated in the study conducted by TrialNet, the largest clinical trial network ever assembled to discover ways to delay and prevent Type 1 diabetes.
“This is the first study to show any drug can delay type 1 diabetes diagnosis a median of 2 years in people at high risk,” explains Teplizumab Prevention Study Chair Kevan Herold, MD, professor of immunobiology and internal medicine at Yale University. “As anyone with type 1 diabetes will tell you, every day you can delay this disease is important.”
Type 1 diabetes is an autoimmune disease that occurs when the body’s immune system attacks and destroys insulin-producing beta cells in the pancreas, causing abnormal blood glucose (sugar) levels. Teplizumab is an immunotherapy drug designed to interfere with the body’s immune destruction of its own beta cells.
While previous studies showed teplizumab prolonged insulin production in people recently diagnosed, this is the first study to test it in people at high risk for the disease.
Of the 76 participants in the study, 55 were under age 18, and all had a relative with Type 1 diabetes. All participants had two or more Type 1 diabetes autoantibodies and abnormal blood sugar levels, as identified by TrialNet screening. These individuals are thought to have a lifetime risk of clinical diagnosis nearing 100 percent.
Participants were randomly assigned to either the treatment group, which received a 14-day course of teplizumab, or the control group, which received a placebo. All participants regularly received glucose tolerance tests until the study was completed, or until they developed clinical Type 1 diabetes – whichever came first.
Seventy-two percent of people in the control group developed clinical diabetes, compared to only 43 percent of the teplizumab group. The median time for people in the control group to develop clinical diabetes was just over 24 months, while the median time for the treatment group was 48 months.
Samples collected during the trial are being studied to help researchers understand why certain people responded to teplizumab better than others. Next, TrialNet researchers hope to conduct additional studies to look for ways to extend the benefits of teplizumab. TrialNet is currently conducting two other trials to see if other immune therapy can delay Type 1 diabetes.
TrialNet Chair Carla Greenbaum, MD, director of the diabetes research program at Benaroya Research Institute in Seattle, said, “In addition to being able to accurately predict who will develop T1D, we have now found a way to delay it. This is an incredible advancement that gets us one step closer to our ultimate goal: a future without T1D. Relatives of people with T1D are urged to get screened for risk at TrialNet.org.”
“We are excited that Indiana University was able to contribute to this groundbreaking research with its potential to impact people at risk for Type 1 diabetes,” said DiMeglio “These findings highlight that Type 1 diabetes can be delayed with therapies that influence the immune system.”
The Teplizumab Prevention Study was funded by the National Institutes of Health (NIH), primarily through the Special Diabetes Program, and NIH’s National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), with additional support from the Juvenile Diabetes Research Foundation, the leading global organization funding Type 1 diabetes research. MacroGenics/Provention Bio donated the study drug and provided funds for additional site monitoring.
IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability.
The largest clinical trial network ever assembled to change the course of type 1 diabetes, TrialNet offers free risk screening for relatives of people with type 1 diabetes and innovative clinical studies testing ways to maintain insulin production before and after diagnosis. There’s also the option to get a test kit in the mail to complete screening at home or take to a local lab. Visit www.trialnet.org.