GABA stands for gamma amino butyric acid and is produced in the body as glutamate is decarboxylased by the GAD enzyme. GABA is an important suppressive and “calming” neurotransmitter. Recent pre-clinical findings support the use of GABA as a synergistic component for treatment of autoimmune diabetes and beta cell regeneration as well as for controlling inflammation, such as in rheumatoid arthritis, metabolic syndrome and type 2 diabetes.
A recent paper (Tian, Dang, Kaufman, et al. Diabetes 2014;63:3128–3134) reports that newly diabetic NOD mice have successfully been treated and some even cured using a combination regimen consisting of GABA and Antigen Based Therapy (ABT). The ABT used in the study was pro-insulin formulated in alum, while similar results were obtained with GAD65 in alum as the ABT in a previous paper using an islet transplantation NOD model (Tian, Dang, Kaufman. PLoS One 2011;6:e25337). GAD65 is the active substance in Diamyd®, the Company’s ABT which is currently being tested in children for the treatment and prevention of type 1 diabetes.
The findings provide proof-of-principle that combining ABT with an anti-inflammatory agent, such as GABA, can effectively restore normoglycemia in newly diabetic mice. The therapy is reported to preserve insulin producing cells as well as to promote their replication. It is concluded in the new paper, that the use of GABA in combination with an ABT may hold promise for type 1 diabetes intervention in humans.
A group at the University of Alabama, Birmingham, in collaboration with Diamyd Medical, is planning a randomized, double-blind, placebo-controlled clinical study with the combination GABA and Diamyd as ABT in patients with new onset type 1 diabetes. Discussions with the FDA regarding the study design are ongoing.