Amyotrophic lateral sclerosis: a complex syndrome that needs an integrated research approach

• Published in: Neural regeneration research Vol. 14; no. 2; pp. 193 - 196
• Main Authors: Riancho, Javier, Gil-Bea, Francisco, Santurtun, Ana, López de Munaín, Adolfo
• Format: Journal Article
• Language: English
• Published: India Wolters Kluwer India Pvt. Ltd 01.02.2019; Medknow Publications and Media Pvt. Ltd; Medknow Publications & Media Pvt. Ltd; CIBERNED(Center for Networked Biomedical Research on Neurodegenerative Diseases, Ministry of Economy and Competitiveness, Institute Carlos Ⅲ), Madrid, Spain%CIBERNED(Center for Networked Biomedical Research on Neurodegenerative Diseases, Ministry of Economy and Competitiveness, Institute Carlos Ⅲ), Madrid, Spain; Department of Neurology, University Hospital Donostia, San Sebastián, Spain; Neurosciences Area, Institute Biodonostia, San Sebastián, Spain%Department of Physiology and Pharmacology, University of Cantabria-Idival, Santander, Spain%CIBERNED(Center for Networked Biomedical Research on Neurodegenerative Diseases, Ministry of Economy and Competitiveness, Institute Carlos Ⅲ), Madrid, Spain; Service of Neurology, Hospital Sierrallana-Idival, Torrelavega, Spain; Neurosciences Area, Institute Biodonostia, San Sebastián, Spain; Department of Neurosciences, University of the Basque Country, Universidad País Vasco-Euskal Herria Unibertsitatea, San Sebastián, Spain; Medknow Publications & Media Pvt Ltd; Wolters Kluwer Medknow Publications
• Subjects: Amyotrophic lateral sclerosis; amyotrophic lateral sclerosis; ALS; environment; epidemiology; genes; phenotype; therapy; Autoimmune diseases; Biomarkers; Body mass index; Clinical trials; Development and progression; Disease; Genes; Genetic aspects; Medical imaging; Medical research; Metabolism; Mutation; Nervous system diseases; Neurological research; Neurons; Neurophysiology; Research funding; Review; Greenland; phenotype; epidemiology; environment; genes; therapy; ALS; amyotrophic lateral sclerosis
• ISSN: 1673-5374; 1876-7958
• DOI: 10.4103/1673-5374.244783

Amyotrophic lateral sclerosis, the most common neurodegenerative disease affecting motor neurons, lacks an effective treatment. A small fraction of amyotrophic lateral sclerosis cases have a familial origin, related to mutations in causative genes, while the vast majority of amyotrophic lateral sclerosis cases are considered to be sporadic, resulting from the interaction between genes and environmental factors in predisposed individuals. During the past few years, dozens of drugs have been postulated as promising strategies for the disease after showing some beneficial effects in preclinical cellular and murine models. However, the translation into clinical practice has been largely unsuccessful and the compounds failed when were tested in clinical trials. This might be explained, at least partially, by the enormous complexity of the disease both from clinico-epidemiological and a pathogenic points of view. In this review, we will briefly comment on the complexity of the disease focusing on some recent findings, and we will suggest how amyotrophic lateral sclerosis research might be reoriented to foster the advance in the diagnostic and therapeutic questions.