The ALS Association

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Carrie Munk
The ALS Association
(571) 319-3047



Critical Protein Measurement Means Progress Toward Antisense Trial in ALS

Washington, D.C. (June 15, 2015) — Researchers funded by The ALS Association have for first time determined a critical parameter called the half-life for a disease-causing protein. This information will be crucial in the design and interpretation of therapeutic trials to reduce the levels of the protein.

ALS (amyotrophic lateral sclerosis) is a progressive neurodegenerative disease that affects neurons (nerve cells) in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. There is no cure and no life-prolonging treatments for the disease. 

Mutations in the gene for the protein, called superoxide dismutase 1 (SOD1), are the second most common cause of inherited forms of ALS. A treatment approach called antisense therapy has recently emerged as a highly promising strategy to reducing the amount of mutant SOD1 protein in people with this form of the disease. In order to proceed, however, researchers must know how quickly the protein is broken down in the body. The rate of breakdown is called the half-life (more specifically, the half-life is the time it takes for one half of any quantity of the protein to break down). The research was led by Timothy Miller, M.D., Ph.D., and Randall Bateman, M.D., and conducted by Matthew Crisp, all of Washington University in Saint Louis, MO.

The team found the half-life of the normal protein in rat cerebrospinal fluid (CSF) was 16 days, and in human CSF about 25 days, a relatively slow turnover rate in the nervous system. The half-life of the mutant protein in rats was slightly faster. The half-life of the mutant protein in humans will be determined in future studies.

“This finding is a necessary step in the development of antisense therapy,” said Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The ALS Association. “These data will be used to help decide when and how often we will need to measure remaining mutant SOD1, in order to judge the effectiveness of antisense treatment. In that light, this study represents true progress toward a trial.”

About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front.  By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.  For more information about The ALS Association, visit our website at

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