Contact:
Carrie Munk
The ALS Association
(571) 319-3047
cmunk@alsa-national.org

 

FOR IMMEDIATE RELEASE

Cell-to-Cell Spread of Misfolded ALS Protein Seen in Cell Model

Washington, D.C. (February 25, 2014) — Scientists have demonstrated a potential new mechanism for the spread of ALS disease pathology in cell models, whose results may point to possible new therapeutic strategies. The study was published in the journal Proceedings of the American Academy of Sciences.

ALS (amyotrophic lateral sclerosis), often referred to as Lou Gehrig’s Disease, 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 superoxide dismutase 1 (SOD1) are a cause of about 20 percent of inherited ALS. Misfolded mutant SOD1 protein is believed to contribute to the disease process in those carrying the mutation, but the role of misfolded non-mutant protein in the disease is unclear. The researchers in this study, led by Neil Cashman, M.D., of the University of British Columbia, Vancouver, Canada, showed that both misfolded mutant protein and misfolded normal protein can be released by one cell and picked up by another cell. The uptake of misfolded protein can lead to misfolding of normal protein in the cell that takes it in, propagating the misfolding process from cell to cell. That cell-to-cell transmission could be reduced by antibodies against the SOD1 protein.

“These results are intriguing and potentially important in understanding the ALS disease process,” said Lucie Bruijn, Ph.D., MBA, Chief Scientist for The Association. “If the spread from cell to cell seen in this cell model also occurs in people with ALS, it could help explain the progression of the disease after it begins and point to blocking that spread as a new and important treatment strategy, including for individuals with non-SOD1-related ALS. However, more work will need to be done to determine whether misfolded SOD1 does in fact move from cell to cell in humans and whether this process contributes to the pattern of disease progression we see. Answering these important questions takes on new urgency with the publication of this study.”

Indeed, one approach to therapy would be to treat with antibodies against misfolded proteins. Several groups have shown that this strategy has benefit in the SOD1 mouse model of ALS, and investigators Janice Robertson, Ph.D., University of Toronto, Toronto, Canada and
Joan Coates, University of Missouri, Mo., are testing this approach in a canine model of ALS in a study funded by The ALS Association.

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 www.alsa.org.

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