title: The Dilemma of the Clinical Trialist in Amyotrophic Lateral Sclerosis: The Hurdles to Finding a Cure.
abstract: Amyotrophic lateral sclerosis can be described as a disease with a poorly understood pathophysiologic mechanism and no treatment that dramatically impacts the course of the disease. Clinical trialists are faced with finding small treatment effects against a background of multiple potential treatments, a past history of failed trials, and heterogenous clinical outcomes. This article summarizes this environment and provides a rationale for drug development going forward.
authors.affiliation: Department of Neurology, Forbes Norris MDA/ALS Center, California Pacific Medical Center, 2324 Sacramento Street, Suite 111, San Francisco, CA 94115, USA. Electronic address: Katzjs@sutterhealth.org.
authors.forename: Jonathan S
authors.initials: JS
authors.lastname: Katz
authors.affiliation: Department of Neurology, The University of Kansas Medical Center, 3901 Rainbow Boulevard, Mail Stop 2012, Kansas City, KS 66160, USA.
authors.forename: Richard J
authors.initials: RJ
authors.lastname: Barohn
authors.affiliation: Department of Neurology, The University of Kansas Medical Center, 3901 Rainbow Boulevard, Mail Stop 2012, Kansas City, KS 66160, USA.
authors.forename: Mazen M
authors.initials: MM
authors.lastname: Dimachkie
authors.affiliation: Department of Neurology, Eleanor and Lou Gehrig MDA/ALS Research Center, Columbia University Medical Center, 710 West 168th Street, New York, NY 10032, USA.
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Mitsumoto
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy*; D002986:Clinical Trials as Topic / Q000379:methods; D006801:Humans; D012108:Research Personnel / Q000523:psychology*
keywords: Amyotrophic lateral sclerosis; Clinical trialists; Drug development; Treatment