New Epigenetic Therapy for Multiple Sclerosis

PROJECT DESCRIPTION Multiple sclerosisMSis one of the most disabling neuro inflammatory disorders that affects the brain and spinal cordAroundpeople are living with MS in the United States and approximatelymillion individuals worldwide suffer from this conditionThe exact mechanism that drives MS is not entirely understoodHoweverthe condition is generally thought as an autoimmune disease that attacks the myelin sheath in the brain and spinal cordwhich functions as the protective layer surrounding the nerves in the central nerve system that help electrical signals to transmit from the brain to the rest of the bodyOnce deterioratedthe disease can permanently damage the nervesleading to loss of ability to walkAt presentthere is no cure for MSand disease modifying therapies are among the limited treatment optionsThusnew therapy is urgently needed for effective treatment of MSFrom our recent studywe have discovered novel small molecule chemical agents that favor lineage differentiation of oligodendrocytesa key step for myelin repairOur lead compounds can inhibit over development of pro inflammatory Thcells in micewhich have recently been recognized as a major driving factor for the pathogenesis of MSMotivated by our recent discoverywe propose to optimize oral bioavailability and CNS activityand validate the therapeutic potential of our novel chemical epigenetic modulators as new effective treatment for MSas outlined in two Specific Aims of this projectThe results from this Phase I study will prepare us for a more efficient Phase II study of an extensive pre clinical evaluation of our most promising drug candidates for MS in human clinical trials PROJECT NARRATIVE Multiple Sclerosis is one of the most disabling neuro inflammatory disorders affecting overpeople in the United Statesand current treatment options are limitedWe recently discovered novel small molecule chemical agents that can facilitate the repair of cell damage caused by the diseaseIn this projectwe will optimize the therapeutic potential of our lead compounds and develop them into new effective and safe epigenetic treatment for multiple sclerosis