BGU and UC Researchers Find Potential ALS Therapy
March 24, 2015
The Jerusalem Post — A collaborative research study recently published in the journal Neuron by researchers from BGU and the University of California, San Diego (UCSD), describes the identification of a novel molecular mechanism which could lead to the future development of new therapies for amyotrophic lateral sclerosis (ALS).
The study was conducted by BGU’s Dr. Adrian Israelson from the Department of Physiology and Cell Biology and Prof. Don W. Cleveland from UCSD.
ALS, also known as Lou Gehrig’s disease, is a fatal neurodegenerative disease characterized by progressive weakness and paralysis due to muscle atrophy. This leads to difficulty in speaking, swallowing, and eventually breathing.
There is no cure or effective treatment for ALS. Its cause is unknown in the vast majority of cases, but about 10% of the cases are genetically inherited. Patients with the disease usually only survive for a few years after onset.
About 20% of the genetic cases are due to mutations in the superoxide dismutase (SOD1) gene. Multifaceted research projects have demonstrated that mutations in this gene (more than 165 different mutants are now known) provoke selective killing of motor neurons by their acquisition of some form of toxicity. However, the basis for this selective toxicity has not been identified.
The researchers report the identification of a factor that is able to inhibit accumulation of misfolded SOD1. Since the misfolding of proteins is a common toxic mechanism among neurodegenerative disorders, this discovery holds the potential to help treat ALS and many other neurodegenerative diseases which are linked to accumulation of misfolded proteins.