Many new drugs cost more than $100,000 per year.
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.
In those who did progress to a diagnosis of type 1 diabetes, development was significantly delayed in those who received teplizumab compared with placebo, with a median time to diagnosis of 48.4 months versus 24.4 months, respectively. "Developing immuno-modulatory drugs that don't require continuous treatment to impact autoimmune disease is a major paradigm shift", said Jeffrey Bluestone, PhD, A.W. and Mary Margaret Clausen Distinguished Professor of Metabolism and Endocrinology at the UC San Francisco (UCSF) Diabetes Center, President, CEO of the Parker Institute for Cancer Immunotherapy, and a Director of Provention Bio.
Still, "keeping kids off insulin as long as possible is really exciting", Dunne said.
Dunne also noted that a delay means a child might be more mature and able to help manage the condition. "The difference between a 12-year-old and a 14-year-old being diagnosed is huge", she said. "The 2-year delay in diagnosis is clinically important". That might translate into fewer complications later in life, according to the JDRF.
"This is the first study to show any drug can delay Type 1 diabetes diagnosis a median of two years in people at high risk", says Dr. Kevan Herold, Teplizumab Prevention study chair and professor of Immunobiology and Internal Medicine at Yale University.
"Our findings support the notion that type 1 diabetes is a chronic T-cell-mediated disease and suggest that immunomodulation before the development of clinical disease can be useful".
About 1.25 million people in the US have type 1 diabetes, with almost 18,000 new cases diagnosed annually in people under age 20, the American Diabetes Association says.
Teplizumab works by binding to the pro-inflammatory T cell co-receptor CD3, which in turn prevents them from activating the T cells that attack and kill the pancreas' beta cells. They were at very high risk of developing Type 1 diabetes.
The share price of Provention Bio doubled yesterday after it released data which showed its immunotherapy PRV-031 (teplizumab) could delay the onset of of type 1 diabetes in children and adults by a median of two years. Also, the volunteers all had tests showing diabetes-related autoantibodies that attack the pancreas, plus unhealthy blood sugar levels.
The treatment involved two weeks of daily infusions of a compound called teplizumab. The others were given a placebo by IV. Study participants were followed until they developed Type 1 diabetes. The theme of this year's Diabetes Week is to "See Diabetes Differently" as everyone is different, and we want everyone to see diabetes differently.
Seventy-two percent of people in the control group developed clinical diabetes, compared to only 43% of the teplizumab group.
Shares of biotech Provention Bio Inc (NASDAQ: PRVB) are trading sharply higher Monday following publication of the results of a National Institutes of Health, or NIH, -sponsored study online in the New England Journal of Medicine as well as presentation of the same at the 79th Annual American Diabetes Association Meeting.
"The results of this trial are striking, with several caveats", they wrote.