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Novel ALS drug slows symptom progression, reduces mortality in phase 2 trial
Date:11/20/2011

Treatment with dexpramipexole a novel drug believed to prevent dysfunction of mitochondria, the subcellular structures that provide most of a cell's energy appears to slow symptom progression in the neurodegenerative disease amyotrophic lateral sclerosis (ALS). Promising results of a phase 2 trial of dexpramipexole are receiving advance online publication in Nature Medicine. Some preliminary results of the study were presented at the 2009 International Symposium on ALS/MND and the 2010 American Academy of Neurology annual meeting.

"Today there are only two FDA-approved drugs used to treat ALS riluzole, which extends life about 10 percent, and Nuedexta, which treats the emotional instability that characterizes ALS and other neurological disorders," says Merit Cudkowicz, MD, director of the Massachusetts General Hospital (MGH) Neurology Clinical Trials Unit and ALS Center, lead author of the study. "We need more therapies to slow, halt and ultimately reverse the course of disease and also therapies to treat the symptoms."

Also known as Lou Gehrig's disease, ALS is a progressive neurodegenerative disease affecting motor neurons in the brain and spinal cord. Death of these nerve cells stops the transmission of neural impulses to muscle fibers, leading to weakness, paralysis and usually death from respiratory failure. Mitochondrial dysfunction is among several factors believed to underlie nerve cell death. Initially developed by Knopp Biosciences of Pittsburgh, dexpramipexole appears to protect neurons from mitochondrial dysfunction.

Several investigators from Knopp, which sponsored the study reported in the current article, collaborated with Cudkowicz and the Northeast ALS Consortium on the trial. Co-founded and co-directed by Cudkowicz, the Northeast ALS Consortium includes over 100 clinical sites throughout North America that work collaboratively on clinical trials and has established the infrastructure necessarily to qui
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Contact: Mike Morrison
mdmorrison@partners.org
617-724-6425
Massachusetts General Hospital
Source:Eurekalert

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