title: Pharmacological treatment of ALS.
abstract: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease resulting from chronic and selective loss of motor neurons in the brain and spinal cord. In 1993, the etiology of ALS has been clarified for a small sub-group of patients with an autosomal-dominant form of this disease. About 10 percent of familial ALS patients have been associated with more than 50 mutations of the gene of the Cu/Zn superoxide dismutase (SOD1). Mutations in the SOD1 gene account for 1 percent of all ALS patients and have therefore limited epidemological and clinical relevance; however, they are of fundamental importance for the understanding of the ALS pathogenesis, and the development of neuroprotective strategies. In two double-blind and placebo-controlled studies the membrane stabilisator riluzole has been shown to be the first neuroprotective compound with a significant effect on survival of ALS patients. The neuroprotective approach reduced therapeutic nihilism in ALS and is a first step in the treatment of this devastating disease.
authors.affiliation: Department of Neurology, University of Ulm, Germany.
authors.forename: C
authors.initials: C
authors.lastname: Münch
authors.forename: A C
authors.initials: AC
authors.lastname: Ludolph
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D002986:Clinical Trials as Topic; D006801:Humans; D009046:Motor Neurons / Q000473:pathology; D018696:Neuroprotective Agents / Q000627:therapeutic use*; D017354:Point Mutation / Q000235:genetics; D019782:Riluzole / Q000627:therapeutic use*; D013482:Superoxide Dismutase / Q000235:genetics
chemical_list: D018696:Neuroprotective Agents; D019782:Riluzole; D013482:Superoxide Dismutase