ALS drug development: reflections from the past and a way forward.
Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) have provided a rich pipeline of drugs for clinical trialists. At least 32 unique compounds have been tested. Nevertheless, riluzole is currently the only treatment that prolongs survival. We present a critical overview of past clinical trials, how therapies are selected for testing in people, challenges with ALS clinical trial design and conduct, and ways to best move forward.
Aggarwal
Swati
S
Department of Neurology, Massachusetts General Hospital, Neurology Clinical Trials Unit, Charlestown, Massachusetts 02129, USA. spaggarwal@partners.org
Cudkowicz
Merit
M
D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy / Q000473:pathology / Q000628:therapy*; D000818:Animals; D002986:Clinical Trials as Topic; D006801:Humans; D018696:Neuroprotective Agents / Q000627:therapeutic use; D012107:Research Design; D019782:Riluzole / Q000627:therapeutic use; D016896:Treatment Outcome
D018696:Neuroprotective Agents; D019782:Riluzole