An immunotherapy delayed the progression of type 1 diabetes for an average of two years in high-risk children and adults, according to a study published this month in the New England Journal of Medicine. Teplizumab, classified as an anti-CD3 drug, appeared to successfully disrupt the process of beta cell destruction for an average of two years, the study found.
These findings were announced by researchers connected with TrialNet, a collective research organization focused on researching therapies to prevent type 1 diabetes. The study was a highlight of the American Diabetes Association 79th Scientific Sessions in San Francisco, and the stock of Provention Bio, Inc., the company attempting to develop this therapy, soared upon release of the news.
For the Phase 2 study, researchers enrolled 76 participants, 72 percent of whom were 18 years old or younger. Each participant had two or more type 1 diabetes-related autoantibodies and abnormal blood sugar levels at time of enrollment. The researchers then divided participants into two groups – one group of 44 participants received a 14-day course of teplizumab, while the other 32 participants received a placebo. The researchers then tracked the progression of beta cell destruction in the participants with oral glucose-tolerance tests over six-month intervals.
What the researchers found was that those who had received the teplizumab treatment delayed progression towards the onset of type 1 diabetes. The average time of diagnosis among those who received the treatment was 48.4 months, compared to an average of 24.4 months for the placebo group.
During the study period, type 1 diabetes was diagnosed in 43% of the participants who received teplizumab, and in 72% of those who received the placebo. The researchers, however, were careful only to say that teplizumab showed promise in delaying the onset of type 1 diabetes, not in preventing the onset of the condition altogether.
The trial results might represent a redemptive milestone for a drug therapy that had once been abandoned as ineffective. In 2010, Eli Lilly and Company and MacroGenics announced that a Phase III trial of teplizumab had failed to reduce HbA1c or insulin dosing requirements of people who already had been diagnosed with type 1 diabetes. The project was subsequently abandoned.
According to a BioSpace report, Provention CEO Ashleigh Palmer says the company will now evaluate a path toward regulatory approval of the drug to treat type 1 diabetes. A trial currently is also underway to see if the therapy might help pediatric patients who had been very recently diagnosed with type 1 diabetes.
T1D Exchange is not connected with this research effort, and cannot enroll participants in the study. For more information about the current trial, click here.