Amyotrophic Lateral Sclerosis: Innovative Therapies for ALS under the Pipeline

Abstract

Over the last 5 decades, a multiple of experimental drugs compounds have been shown to dissuade disease progression in preclinical animal models of amyotrophic lateral sclerosis (ALS) but failed to show any efficacy in human clinical trials or are still waiting for approval under Phase I–III trials. Only 2 main drug compounds are discovered till date and approved by USA Food and Drug Administration for ALS treatment that show better efficacy, effective against ALS progression in early stages and enhances the survival rate of patients. The riluzole is a glutamatergic neurotransmission inhibitor and edaravone is act as an antioxidants. Various clinical trials carried out for ALS treatment but do not show any effective pharmacodynamic and pharmacokinetic data. We required further study on genetics and pathophysiology of ALS that associated with progression of disease. In this review, we focused on pathological aspects and some important drug molecules that participate in clinical trials.