The ALS Association

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Brian Frederick
The ALS Association



ALS Association Funded Study Finds Helping the Motor Neurons in the Brain Also Helps Those in the Spinal Cord

Washington, D.C. (November 12, 2014) — In work supported by The ALS Association, researchers have found evidence that motor neurons in the brain may offer an unexpected new target for therapy. The study was published in the Journal of Neuroscience.

ALS (amyotrophic lateral sclerosis), also known 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. 

While the motor neurons that extend from the brain into the spinal cord (so-called “upper” motor neurons) have been known to be involved in the disease, previous work in mouse models has focused primarily on “lower” motor neurons, those that extend from the spinal cord to the muscle. Recent research on upper motor neurons by other Association-funded scientists, including Hande Ozdinler, Ph.D., of Northwestern University, has led to progress in better understanding this important and unique group of neurons, and their contributions to the ALS disease process.

In the new study, Association-funded lead researcher Gretchen Thomsen, Ph.D., along with principal investigator Clive Svendsen, Ph.D., examined the effect of the disease-causing mutant SOD1 protein in a rat model of ALS. They found that reducing the level of mutant SOD1 in upper motor neurons delayed disease onset, prolonged survival, and enhanced the survival of lower motor neurons, even though the lower motor neurons continued to have harmful levels of the protein. The results suggest that the survival of lower motor neurons depends in part on upper motor neurons and that therapies targeting upper motor neurons may be especially helpful.

“These results point toward the motor neurons in the brain as a significant target for therapy development,” said Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The Association. “They also point out the importance of studying multiple disease models, each of which may provide unique insights into the causes and treatment of ALS.”

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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