In people at high risk for type 1 diabetes, 14 days of therapy with the experimental drug teplizumab delayed development of the disease by a year or more, according to results from a mid-stage study presented Sunday.
The 76 study participants, who ranged in age from 8 to 49, faced a high risk of type 1 diabetes in part because their relatives had the autoimmune disease, which kills the beta cells in the pancreas that make and release insulin. Also, the volunteers all had tests showing diabetes-related autoantibodies that attack the pancreas, plus unhealthy blood sugar levels.
Among the 44 volunteers randomly assigned to receive the drug, 19, or 43%, developed diabetes, with the disease appearing within 48.4 months in half of them.
By comparison, among the 32 people who received a placebo, 23, or 72%, developed diabetes, with half the patients developing it within 24.4 months.
When the study was stopped, the percentage of diabetes-free participants was twice as high in the teplizumab group (57%) as in the placebo group (28%).
The chief side effects were temporarily low levels of lymphocytes – a type of white blood cell – and rash.
Provention Bio Inc is developing the drug. The trial was financed by the National Institutes of Health and the Juvenile Diabetes Research Foundation. The findings were reported at a meeting of the American Diabetes Association in San Francisco and in The New England Journal of Medicine.
“After repeated failures, this is the first time anyone’s been able to delay the onset of type 1 diabetes,” chief author Dr. Kevan Herold of Yale University in New Haven, Connecticut told Reuters Health by phone. “The rate of development of diabetes was reduced by half.”
Dr. Clifford Rosen of the Maine Medical Center Research Institute and Journal deputy editor Dr. Julie Ingelfinger wrote in an editorial, “We can finally say that there has been substantial progress in modulating the early course of type 1 diabetes.”
Additional studies will be required before regulatory agencies approve the drug.
About 1.25 million people in the U.S. have type 1 diabetes, with nearly 18,000 new cases diagnosed annually in people under age 20, the American Diabetes Association says.
Teplizumab works by modifying the white blood cells from the immune system that kill insulin-producing cells in the pancreas.
The drug’s greatest impact seemed to be in the first year after treatment, when only 7% developed type 1 diabetes, compared with 44% who received placebo.
Another round of therapy might further delay diabetes development and “that’s what we’re hoping to do,” said Herold.
He said there would be reluctance to give the drug long-term for fear it might throttle back the immune system too much.
“It’s been felt that to have a really effective treatment, you need a drug you give for a short period of time so people are not chronically immunosuppressed,” he said.
Many new drugs cost more than $100,000 per year. If teplizumab were to delay development of type 1 diabetes for a year or so, said Herold, people might think that’s worth it.
“You have to talk to someone who has diabetes,” he said. “I think most people would tell you a day without diabetes is terrific because the disease is a 24/7 disease. You can’t sleep, you can’t eat, you can’t walk without considering it. And three quarters of the people here are children. We’re talking about a critical time in their development.”
Type 1 diabetes typically takes more than a decade off a person’s life.