ALS May Be Treatable

It's too late to treat Lou Gehrig, but he would probably still be batting .300 and playing every day for the New York Yankees if he had not been struck down by the disease that now bears his name. Researchers who are not in the US, or who are Red Sox fans, prefer the more scientific amyotrophic lateral sclerosis (ALS) term for Lou Gehrig's Disease and results from eleven independent ALS studies provide some hope for the afflicted community – because they reveal that the disease may be treatable by targeting new mechanisms revealed in neural stem cell-based studies. 

It's too late to treat Lou Gehrig, but he would probably still be batting .300 and playing every day for the New York Yankees if he had not been struck down by the disease that now bears his name.

Researchers who are not in the US, or who are Red Sox fans, prefer the more scientific amyotrophic lateral sclerosis (ALS) term for Lou Gehrig's Disease and results from eleven independent ALS studies provide some hope for the afflicted community – because they reveal that the disease may be treatable by targeting new mechanisms revealed in neural stem cell-based studies. 

When neural stem cells were transplanted into multi-levels of the spinal cord of a mouse model with familial ALS, disease onset and progression slowed, motor and breathing function improved and treated mice survived three to four times longer than untreated mice.  

"This significant research will help us better understand the mechanisms underlying motor neuron diseases," said Yang (Ted) Teng, PhD, MD, the study's co-lead author and director of the Spinal Cord Injury and Stem Cell Biology Research Laboratory in the Department of Neurosurgery at Brigham and Women's Hospital. "This work sheds new light on detrimental roles played by non-neuronal cells in triggering motor neuron death and these events should be targeted for developing more effective therapeutics to treat ALS."

The transplanted neural stem cells benefited the mice with ALS by boosting the health and function of their remaining nerve cells. The neural stem cells also reduced inflammation and suppressed the number of disease-causing cells in their spinal cords. The neural stem cells did not replace deteriorating nerve cells.

"This is not a cure for ALS," explained Teng, "but it shows the potential that mechanisms used by neural stem cells in our study have for improving an ALS patient's quality of life and length of life."

 A summary of all eleven studies is in Science Translational Medicine.

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