A look into the future of ALS research. Issue 6 (June 2016)

• Record Type: Journal Article
• Authors: Clerc, Pascaline; Lipnick, Scott; Willett, Catherine
• Is Part Of: Drug discovery today. Volume 21:Issue 6(2016)
• Page Start: 939
• Page End: 949
• Publication Date: 2016-06
• Subjects: Drugs -- Design -- Periodicals; Drugs -- Research -- Periodicals; 615.1
• DOI: 10.1016/j.drudis.2016.02.002
• Languages: English
• ISSNs: 1359-6446

Abstract

Highlights: Biomedical research relies heavily on animal models of human diseases. The high drug development failure rate reflects the lack of translation. There is an urgent need to reexamine our current research models. New technologies and advances in science have transformed the field of toxicology. Applying the same principles to biomedical research will fasten drug discovery. Abstract : Although amyotrophic lateral sclerosis (ALS), also referred as 'Lou Gehrig's Disease, ' was first described in 1869 and the first disease-associated gene was discovered almost 20 years ago, the disease etiology is still not fully understood and treatment options are limited to one drug approved by the US Food and Drug Administration (FDA). The slow translational progress suggests that current research models are not ideal to study such a complicated disease and need to be re-examined. Progress will require greater insight into human genes and biology involved in ALS susceptibility, as well as a deeper understanding of disease phenotype at the histological and molecular levels. Improving human disease outcome will require directing focus toward improved assessment technologies and innovative approaches. Teaser: A review of the current models, including animal models, used in ALS research and new models to accelerate drug discovery.