A study that was presented at the American Diabetes Association’s Scientific sessions on Monday showed that treatment with autoantigen specific therapies, such as Alum-GAD, is safe to give to children with an increased risk for developing type 1 diabetes, however, it does not prevent the development of the disease.
According to the investigators, glutamate decarboxylase, or glutamic acid decarboxylase (GAD), which is an enzyme that is targeted by autoantibodies in people who later develop type 1 diabetes, earlier showed that it may preserve some insulin production for 30 months in people with type 1 diabetes. However, this had not been confirmed in larger scale studies. This double-blind, randomized, investigator-initiated study was set to show the safety and the efficacy of alum-formulated GAD65 (Alum-GAD), on the progression to type 1 diabetes in children with ongoing persistent beta-cell autoimmunity as indicated by multiple positive islet cell autoantibodies.
DIAPREV-IT is the first prevention study where Alum-GAD was given before the onset of type 1 diabetes. The study aimed to test if it is safe to use Alum-GAD on children who are at high risk for developing the disease in an attempt to induce immune-tolerance earlier during the autoimmune process, and thereby prevent or delay the process leading to clinical type 1 diabetes. Known risk factors for type 1 diabetes include family history, genetics, geography and age, with the highest incidence of onset occurring in children between 4-7 years and 10-14 years.
The study was conducted from April 2009 to January 2017 in Sweden. The trial enrolled 50 children, aged 4-18 years, who did not yet have type 1 diabetes, and who had positive GAD-antibodies and at least one additional type 1 diabetes-associated autoantibody (IA-2Ab, ZnT8R/W/QAb or IAA).
Since the study shows that Alum-GAD treatment is safe, the investigators wanted to further explore this treatment—either in different doses or in combination with other drugs—in future prevention studies that may impact patient care, said chief investigator Helena Elding Larsson, associate professor of pediatric endocrinology and diabetes at Lund University in Sweden. “For example, one option could be to use repeated increasing small doses as is common in classic immune tolerance treatment for allergic symptoms.”
“We collected a large number of immunological samples from the study that have yet to be analyzed to understand the possible mechanistic effects of Alum-GAD,” Larsson explained. “These results will be important to consider in developing new studies with combination therapies performed.”