Amyotrophic Lateral Sclerosis (ALS)

Computer Error Results in Wrong Participants Getting Placebo in Tirasemtiv Trial

South San Francisco biotechnology company Cytokinetics announced July 8, 2013, that a computer program error occurred in its phase 2b BENEFIT-ALS clinical trial designed to evaluate the safety, tolerability and potential efficacy of tirasemtiv in people with amyotrophic lateral sclerosis (ALS)

A ‘Transport Problem’ May Cause Motor Neuron Death

Axonal transport is the process that is responsible for moving nutrients, proteins and other vital cellular cargo to and from the cell bodies in motor neurons – the muscle-controlling nerve cells that are lost in amyotrophic lateral sclerosis (ALS).

ALS: Misfolded TDP43 Appears to Spread

A team of researchers based at institutions in the United States and Germany has described the path amyotrophic lateral sclerosis (ALS) appears to take as it progresses through different areas of the central nervous system (the brain and spinal cord).

Generic Riluzole on the Market

The U.S. Food and Drug Administration (FDA) granted approval June 18, 2013, to Apotex Corp., Glenmark Generics and Sun Pharmaceutical Industries to market generic versions of the prescription amyotrophic lateral sclerosis (ALS) drug riluzole.

Stem Cell Screen Identifies Target in ALS

Using a stem-cell-based screening method, a team of researchers based at institutions in Massachusetts and Connecticut identified a small molecule called kenpaullone as a potential therapy for amyotrophic lateral sclerosis (ALS).

Further studies revealed that the molecule appears to work by inhibiting the activity of two proteins, GSK3 and HGK. This data suggests the proteins may be potential targets at which to aim therapies in ALS.

ALS Briefs: New Findings About Three ALS-Associated Genes

Recent research in amyotrophic lateral sclerosis (ALS) includes findings involving the CRMP4, FUS and TDP43 genes.

Rare variant in the CRMP4 gene is associated with ALS

A study conducted in France and Sweden has identified an association between a rare variant in the gene for the collapsing response mediator protein 4 (CRMP4) protein and ALS.

ALS — Bridge-to-Industry Training Program

MDA launched its innovative Bridge-to-Industry (B2I) program with a $180,000 grant over three years to postdoctoral fellow Archi Joardar at the University of Arizona in Tucson, to develop two promising drug candidates for the treatment of amyotrophic lateral sclerosis (ALS).

MDA’s Bridge-to-Industry, or B2I, is a pilot project that trains promising researchers in translational research by providing experience both in academia and the biopharmaceutical industry.    

ALS — ALS Therapy Development Institute (ALS TDI)

MDA and the nonprofit biotech ALS Therapy Development Institute (ALS TDI) have extended their strategic research partnership through 2013. With the extension comes a $3.2 million MDA grant to help support the nonprofit biotech's continued progress toward developing treatments for amyotrophic lateral sclerosis (ALS).

ALS TDI, located in Cambridge, Mass., screens at least 25 potential ALS therapeutics every year.

ALS — National Institutes of Health (NIH) Laboratory

MDA has awarded $400,000 to National Institutes of Health (NIH) Laboratory of Neurogenetics researchers to perform exome sequencing on samples taken from 1,000 people with sporadic amyotrophic lateral sclerosis (ALS). The project will be led by neurologist Bryan Traynor, head of the Neuromuscular Diseases Research Group at the NIH in Bethesda, Md.

ALS Briefs: Disease Modifier, Growth Factors, Antioxidant Genes

Identification of the biological mechanisms that underlie amyotrophic lateral sclerosis (ALS) may provide scientists with important clues about the disease that may be used to identify biomarkers and potential biolo