Amyotrophic Lateral Sclerosis (ALS)

'Sugar' Molecule Delays Disease Progression in ALS Mice

Treatment with a small molecule called trehalose was associated with slower disease progression and longer life span in mice with a disease mimicking amyotrophic lateral sclerosis (ALS), an MDA-supported research team has reported.

Trehalose is a compound composed of two glucose (sugar) molecules.

ALS — NeuRx Diaphragm Pacing System (DPS)

The Muscular Dystrophy Association has committed $750,000 to help support a phase 2 clinical trial assessing the ability of the NeuRx Diaphragm Pacing System (DPS) to improve respiratory function and quality of life in people with amyotrophic lateral sclerosis (ALS).

Multicenter Trial to Test Safety of Mexiletine in Sporadic ALS

The Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is actively seeking participants for a phase 2 clinical trial that will evaluate the safety and tolerability of mexiletine in people with the sporadic form of amyotrophic lateral sclerosis (ALS).

Approximately 90 to 95 percent of ALS is sporadic, in which there is no known family history of the disease.

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.