A look into the future of ALS research

Pascaline Clerc; Scott Lipnick; Catherine Willett

doi:10.1016/j.drudis.2016.02.002

A look into the future of ALS research

Drug Discov Today. 2016 Jun;21(6):939-49. doi: 10.1016/j.drudis.2016.02.002. Epub 2016 Feb 6.

Authors

Pascaline Clerc¹, Scott Lipnick², Catherine Willett³

Affiliations

¹ The Humane Society of the United States, 700 Professional Drive, Gaithersburg, MD 20879, USA. Electronic address: pclerc@humanesociety.org.
² Massachusetts General Hospital, Harvard Medical School, Department of Medicine, 55 Fruit Street, Boston, MA 02114, USA.
³ The Humane Society of the United States, 700 Professional Drive, Gaithersburg, MD 20879, USA.

PMID: 26861067
DOI: 10.1016/j.drudis.2016.02.002

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.

Publication types

Review

MeSH terms

Amyotrophic Lateral Sclerosis*
Animals
Biomedical Research
Disease Models, Animal
Humans