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Transplanted Neural Stem Cells Slows ALS Onset And Progression In Mouse Models

Promising new research provides evidence that ALS, amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, may be treatable using neural . A consortium of researchers at multiple institutions, including the , Sanford-Burnham Medical Research Institute and Brigham and Women’s Hospital, have shown that neural , when transplanted into the spinal cord of a mouse model with familial ALS, slow disease onset and progression while improving motor function, breathing and survival time compared to untreated mice. A summary of the studies was published online in Science Translational Medicine.

ALS is a progressive, neurodegenerative disorder affecting the in the central nervous system. As die, the brain’s ability to send signals to the body’s muscles is compromised. This leads to loss of voluntary muscle movement, paralysis and eventually respiratory failure. The cause of most cases of ALS is not known.

Approximately 10 percent of cases are inherited. Though investigators at and elsewhere have identified several genes shown to cause inherited or familial ALS, almost 50 percent of these cases have an unknown genetic cause.

are the precursors of all brain cells. They can self-renew, making more , and differentiate, becoming or other brain cells. These cells can also rescue malfunctioning and help preserve and regenerate brain tissue. But they’ve never before been studied extensively in a good model of adult ALS.

In 11 independent studies, the group, headed by Dr. Evan Snyder of the Burnham Institute, transplanted neural stem cells into the spinal cord of a mouse model of ALS. The transplanted neural stem cells benefited the mice with ALS by preserving the health and function of the remaining nerve cells.

Specifically, the neural stem cells promoted the production of protective molecules that spared remaining nerve cells from destruction. They also reduced inflammation and suppressed the number of toxin-producing and disease-causing cells in the host’s spinal cord.

“It is striking that the stem cells improve motor neuron viability without generating new motor neurons. These findings encourage us to explore further the role of cell therapies in ALS,” said Robert Brown, MD, DPhil, a co-author on the study and chair of neurology at UMass Medical School. A leading expert in ALS, Dr. Brown led the team that discovered the first gene linked to familial ALS, a protein anti-oxidant known as superoxide dismutase, or SOD1 in 1993.


This research was funded by Project ALS, California Institute for Regenerative Medicine, the U.S. National Institutes of Health (National Institute of Neurological Disorders and Stroke grants R21NS053935, 1RC2NS070342-01, 1RC1NS068391-01, R01NS050557-05, U01NS05225-03), U.S. Department of Veterans Affairs, Christopher Reeve Foundation/American Paralysis Association, Project ALS, P2ALS, Sanford Children’s Health Research Center, Zinberg Foundation, ALS Therapy Alliance, ALS Association, Angel Fund, Al-Athel Foundation, Pierre L. deBourgknect ALS Research Foundation, and HeadNorth.

University of Massachusetts Medical School