Amyotrophic Lateral Sclerosis (ALS)

ISIS-SOD1-Rx: So Far, So Good

Early results of a phase 1 clinical trial of ISIS-SOD1-Rx, an antisense oligonucleotide drug for SOD1-related amyotrophic lateral sclerosis (ALS), show the drug appears to be safe and well tolerated at the lowest dosage level, based on a group of six patients who received 12-hour infusions of it into their spinal fluid.

ALS TDI Webinar Discusses Current Research

The state of the science in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease), current lines of research, efforts to establish a TDP43 research mouse colony, and a primer on protein-based therapies were the topics of a webcast on April 7, 2011, presented by ALS Therapy Development Institute (ALS TDI) CEO and Chief Scientific Officer Steve Perrin, and colleagues.

ALS Research Briefs from 2011 AAN Meeting

Neuralstem's trial of spinal cord injections of stem cells is on track

MDA Surveying Families about ‘Cost of Illness’

Most people affected by neuromuscular disease know all too well that their condition is costly. MDA wants to find out just how costly — and then translate those figures into terms that will speak to policy makers.

Scholarships, Grants Available to People with Disabilities

(Update 9/14/11: The 2011 scholarship winners have been selected. To view a list of winners visit www.deshae.org/cmms/awards/2011/scholars.pdf. Applications for quality of life grants continue to be accepted and are awarded on an ongoing basis.)

Phase 3 Trial of Dexpramipexole Launched in ALS

Update 8/31/11: Enrollment screening for this study has closed to new prospective participants.

MDA Awards Grant to Glialogix to Test Potential ALS Treatment

MDA is funding research into a potential treatment for amyotrophic lateral sclerosis (ALS) that inhibits excessive glutamate signaling in the brain and spinal cord.

ALS Research Briefs

TDP43 has unexpectedly extensive role

Scientists have found that the protein TDP43 normally influences the synthesis of more than 1,500 other proteins, including itself. Mutations in the gene for TDP43 are a known cause of ALS, and TDP43 is depleted in the nucleus and enriched in the cytoplasm (main cell compartment) in ALS-affected nerve cells, even when no TDP43 mutations exist. Understanding TDP43's many functions is likely to improve understanding of ALS.

For more, see:

MDA Awards Grant to Test Heat Shock Protein in ALS Mice

UPDATE (April 9, 2012): MDA and ALS Biopharma agreed to terminate this project in April 2012, when ALS Biopharma determined that it was unable to manufacture its HSP70-based compound in a pure enough form to meet study standards.  The ability to reliably produce the compound in sufficient quantity and quality for testing purposes was one of a number of milestones that had to be met in order for the project to receive full funding.

MDA Conference Brings Together Researchers, Clinicians, Industry

Moving therapeutic strategies from the laboratory to clinical trials and ultimately to the market as treatments was the theme of the MDA National Scientific Conference held March 13-16, 2011, in Las Vegas.

Some 300 people attended the conference, the first in a planned series of such MDA-sponsored meetings that will emphasize new research and current medical care. The majority of presenters and many of the audience members were current or former MDA research grantees or physicians at MDA-supported clinics.

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