Emerging drugs for amyotrophic lateral sclerosis

Introduction: Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disease that results in increasing disability and that is uniformly fatal. Since its approval in the 1990s, riluzole remains the sole treatment for ALS offering modest survival benefit. While significant advances have been made in the symptomatic management of the disease, more effective drug therapy targeting disease progression is sorely needed. Areas covered: Advances in the understanding of pathogenic mechanisms involved in disease development and progression have provided multiple avenues for developing effective treatment strategies. This review highlights recent discoveries relating to these diverse mechanisms and their implications for the development of drug therapy. Previous human clinical trials that have targeted these pathways are mentioned and ongoing drug trials are discussed. Expert opinion: The search for effective drug therapy faces important challenges in the areas of basic science and animal re...