The ALS Association

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Carrie Munk
The ALS Association



Choice of Stem Cells Can Improve Therapeutic Potential

Washington, D.C. ((June 27, 2016)— Choosing stem cells with specific desirable properties can improve their therapeutic potential, according to a new research study supported by The ALS Association. The study, led by Stefania Corti, M.D., Ph.D., Assistant Professor of Neurology at the University of Milan, and her colleagues, was published in the journal Human Molecular Genetics on June 6, 2016.

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which leads to total paralysis and death, usually within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.

Stem cells are cells that are capable of developing into multiple different types of cells, including neurons and cells that nourish and protect them. Subpopulations of stem cells differ from one another in several ways, including their ability to migrate and to promote production of growth factors that aid neurons. To test the potential of one such subpopulation, Dr. Corti isolated stem cells with both migratory and growth factor-production abilities. She mixed them with motor neurons that were cultured with ALS-causing astrocytes, and found that the motor neurons survived longer and grew better as a result. When the stem cells were injected into a SOD1 mouse model of ALS, they migrated and supported neurons in the spinal cord, reducing signs of inflammation and promoting growth, and demonstrated some survival benefit in the mice.

“This important work increases our understanding of the potential of stem cells for therapy in ALS,” said Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The ALS Association. “As we continue to develop this therapy, it may be very useful to focus on those cells with the migration and growth-promoting properties demonstrated here, but understand that there are still many hurdles to overcome to take this to clinical trials. So far, efficacy in the clinic is unclear.”

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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