title: Clinical trials: present and future.
abstract: The past decade has seen a major expansion of clinical trials in amyotrophic lateral sclerosis (ALS). However, the perfectly-designed ALS trial remains elusive. Attempts to track the progression of the disease are affected by continual improvements in the care of patients. Comparing the effectiveness of different drugs is difficult because different primary endpoints are used in different studies. We also need to decide how much benefit we are aiming to achieve when studying a new treatment. The interpretation of animal models has also proved problematic, with results not being replicated in human studies. Moreover, promising phase I/II trial results have often not been confirmed by phase III studies. Our patients, meanwhile, are anxious to try any medication that may help. The ALS research community has learned a great deal from past trials and this will be greatly beneficial when evaluating the novel and combination therapies currently being developed. Effort must also be directed towards the search for objective markers for ALS.
authors.affiliation: Eleanor & Lou Gehrig MDA/ALS Center, Columbia-Presbyterian Medical Center, New York, NY, USA.
authors.forename: H
authors.initials: H
authors.lastname: Mitsumoto
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy*; D000818:Animals; D002986:Clinical Trials as Topic / Q000639:trends*; D004791:Enzyme Inhibitors / Q000627:therapeutic use*; D006801:Humans
chemical_list: D004791:Enzyme Inhibitors