In a first, researchers have found that a treatment affecting the immune system effectively slowed the progression to clinical Type-1 diabetes in high risk individuals by two years or more. "The results have important implications for people, particularly youth, who have relatives with the disease, as these individuals may be at high risk and benefit from early screening and treatment," said Lisa Spain, Project Scientist from US National Institutes of Health's National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).
The study, involving treatment with an anti-CD3 monoclonal antibody (teplizumab), was conducted by Type 1 Diabetes TrialNet, an international collaboration aimed at discovering ways to delay or prevent Type-1 diabetes. Researchers enrolled 76 participants aged 8-49 years who were relatives of people with Type-1 diabetes, had at least two types of diabetes-related autoantibodies (proteins made by the immune system), and abnormal glucose (sugar) tolerance.
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 received glucose tolerance tests regularly until the study was completed, or until they developed clinical Type-1 diabetes - whichever came first.
During the trial, 72 per cent of the people in the control group developed clinical diabetes, compared to only 43 per cent of the teplizumab group. The median time for people in the control group to develop clinical diabetes was just over 24 months, while those who developed clinical diabetes in the treatment group had a median time of 48 months before progressing to diagnosis.
"The difference in outcomes was striking. This discovery is the first evidence we've seen that clinical Type-1 diabetes can be delayed with early preventive treatment," Spain added.
Type-1 diabetes develops when the immune system's T cells mistakenly destroy the body's own insulin-producing beta cells. Insulin is needed to convert glucose into energy. Teplizumab targets T cells to lessen the destruction of beta cells.
The effects of the drug were greatest in the first year after it was given, said the study published online in The New England Journal of Medicine.
