A small-molecule inhibitor developed by Innovimmune Biotherapeutics has prevented multiple sclerosis from developing in mice and shown promise in treating other autoimmune diseases.
In a new study appearing in the Journal of Neurological Sciences, Innovimmune's retinoic acid receptor-related orphan receptor ROR gamma t prevented disease progression of murine experimental autoimmune encephalomyelitis, a CNS disorder used as an animal model for brain inflammation. The findings could eventually lead to a new class of therapeutics to treat the debilitating disease, which currently has no cure.
T helper 17 (TH17) cells and their production of IL-17 cytokines play a primary role in the pathogenesis of experimental autoimmune encephalomyelitis in animals as well as several human autoimmune disorders like multiple sclerosis. ROR gamma t is a nuclear hormone receptor that specifically regulates TH17 cells by acting as a control switch for TH17 differentiation and function. Essentially, ROR gamma t acts as the master regulator of human TH17 cells.
The ROR gamma t inhibitor is part of the Brooklyn, NY, startup's INV-17 portfolio of lead compounds, which the company is concurrently advancing for potential therapeutic applications in multiple ROR gamma t-regulated autoimmune diseases with significant unmet medical need, including multiple sclerosis.
"This is a remarkable finding in that a novel therapeutic approach targeting pathogenic T helper 17 cells provides superior preclinical efficacy. These results, which demonstrate disease prevention in the absence of toxicity, may provide a novel disease-modifying treatment strategy with an oral INV-17 drug to patients with MS and other TH17-mediated autoimmune diseases," said investigator Dr. Daniel Rosenbaum, a professor of medicine and chair of the neurology department at the State University of New York Downstate Medical Center.
In 2012, Innovimmune received a $600,000 NIH grant to develop novel autoimmune compounds.
- here's the study
- read the press release