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Phase 1 ALS trial is first to test antisense treatment of neurodegenerative disease
Date:4/3/2013

The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis (ALS) blocking production of a mutant protein that causes an inherited form of the progressive neurodegererative disease may be a first step towards a new era in the treatment of such disorders. Investigators from Massachusetts General Hospital (MGH) and Washington University School of Medicine report that infusion of an antisense oligonucleotide against SOD1, the first gene to be associated with familial ALS, had no serious adverse effects and the drug was successfully distributed thoughout the central nervous system.

"This therapy directly targets the cause of this form of ALS a mutation in SOD1, which was originally discovered here at the MGH by my mentor Robert Brown," says Merit Cudkowicz, MD, chief of Neurology at MGH and senior author of the report in Lancet Neurology, which has been released online. "It's very exciting that we have reached a stage when we can start clinical trials against this type of ALS."

ALS causes the death of motor neurons in the brain and spinal cord, stopping transmission of neural signals to nerve fibers and leading to weakness, paralysis and usually death from respiratory failure. Only 10 percent of ALS cases are inherited, and mutations in SOD1 which produce an aberrant, toxic form of the protein account for about 20 percent of familial cases. Although that first SOD1 mutation was identified 20 years ago by the team lead by Brown who is now professor and chief of Neurology at the University of Massachusetts Medical School a technology that directly addresses such mutations became available only recently.

The current study, the first author of which is Timothy Miller, MD, PhD, of Washington University, used what are called antisense oligonucleotides small, single-stranded DNA or RNA molecules that prevent production of a protein by binding to its messenger RNA. While antisense medications hav
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Contact: Mike Morrison
mdmorrison@partners.org
617-724-6425
Massachusetts General Hospital
Source:Eurekalert

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