Amyotrophic Lateral Sclerosis (ALS)

MDA Commits $10.7 Million to Neuromuscular Disease Research

The Muscular Dystrophy Association has awarded 33 new grants totaling $10,684,481 to fund research projects focused on uncovering the causes of, and developing therapies for, neuromuscular disease.

MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical Advisory Committees, and the grants took effect Aug. 1.

ALS: New MDA Grants Focus on Multisystem Aspects of the Disease

Three new MDA grants totaling $891,156 have been awarded to research projects aimed at uncovering some of the many complex processes that underlie ALS.

MDA Funds Efficacy Trial of Diaphragm Pacing System in ALS

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).

'Repeats' in Normal C9ORF72 Gene Don't Affect ALS, FTD

Humans carry two copies of the C9ORF72 gene, each containing a stretch of DNA in which a sequence of DNA building blocks coded GGGGCC is repeated a number of times. (Each six-letter sequence is called a "repeat.")

When one of the two copies is mutated so that it contains a far greater than normal number of GGGGCC repeats (a repeat expansion mutation), it can cause some forms of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Phase 3 Trial of Ceftriaxone in ALS Stopped

On Aug. 8, 2012, the National Institute of Neurological Disorders and Stroke (NINDS) reported that it has stopped a phase 3 clinical trial of the antibiotic ceftriaxone in amyotrophic lateral sclerosis (ALS) because the study was "unlikely to reach the predetermined efficacy criteria."

MDA Launches '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 — Marc Weisskopf, Sc.D., Ph.D.

MDA awarded a research grant totaling $301,614 over three years to Marc Weisskopf, associate professor of environmental and occupational epidemiology in the departments of environmental health and epidemiology at Harvard School of Public Health in Boston. The funds will help support Weisskopf’s search for nongenetic risk factors for amyotrophic lateral sclerosis (ALS).

ALS — Christine Vande Velde, Ph.D.

MDA awarded a research grant totaling $358,242 over three years to Christine Vande Velde, research assistant professor in the department of medicine at the University of Montreal Hospital Research Center in Montreal, Quebec (Canada). The funds will help support Vande Velde’s study of the role of TDP43 and the stress granule mechanism in amyotrophic lateral sclerosis (ALS).

Another Gene Linked to Familial ALS

A genetic mutation in the gene for a protein called profilin 1 (PFN1) has been identified as a cause of familial amyotrophic lateral sclerosis (ALS), an MDA-supported team of researchers has reported.

Only about 5 percent of ALS is familial (where there is a history of ALS in more than one family member) with the other 95 percent occurring sporadically (without any family history of the disease).

ALS — Alex Parker, Ph.D.

MDA awarded a research grant totaling $231,300 over three years to Alex Parker, assistant professor in the department of pathology and cellular biology at the University of Montreal Hospital Research Center in Montreal, Quebec (Canada). The funds will help support Parker's study of cellular stress response in neurodegeneration associated with amyotrophic lateral sclerosis (ALS).

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