title: Emerging drugs for amyotrophic lateral sclerosis.
abstract: BACKGROUND
  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.
  
  
  METHODS
  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.
  
  
  CONCLUSIONS
  The search for effective drug therapy faces important challenges in the areas of basic science and animal research, translation of these results into human clinical trials, inherent bias in human studies and issues related to delays in clinical diagnosis. How these issues may be addressed and why ALS research constitutes fertile grounds for drug development not only for this devastating disease, but also for other more prevalent neurodegenerative diseases, is discussed in this review.
authors.affiliation: The Neurological Institute of Columbia University, Eleanor and Lou Gehrig MDA/ALS Center, NY 10032, USA. hm264@columbia.edu
authors.forename: Ali Aamer
authors.initials: AA
authors.lastname: Habib
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Mitsumoto
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy*; D000818:Animals; D002986:Clinical Trials as Topic; D004353:Drug Evaluation, Preclinical / Q000379:methods; D006801:Humans; D016032:Randomized Controlled Trials as Topic