Novel Strategies for Development of Therapeutics for Neurodegenerative Disorders

Abstract

Neurodegenerative diseases are particularly challenging from both a research and a drug development perspective. A deeper understanding of the genetics of neurogenerative disease may reveal novel therapeutic targets. Alternatively, identification of common mechanisms underlying neural protection, repair, inflammation, and regeneration could provide therapeutic targets broadly applicable to neurodegenerative diseases. Beyond understanding the etiology of neurodegenerative diseases, there is a need for easily measured biomarkers that can be reliable predictors for the rate of progression in individuals and biomarkers that can be leveraged to measure clinical efficacy of new therapeutics. Despite advances, there are no novel disease-modifying therapies that provide significant benefit for patients who suffer from these debilitating disorders. As we look to the future, gene therapy has the potential to transform treatment for a wide range of neurological diseases; creating therapies that could slow, halt or even restore normal function.