Boston – Results from eleven independent ALS (Lou Gehrig's Disease) research studies are giving hope to the ALS community – showing for the first time that the disease may be treatable by targeting new mechanisms revealed by neural stem cell-based studies. A decade of research conducted at multiple institutions, shows that 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. A summary of the findings from all eleven studies publishes online December 19 in Science Translational Medicine.
"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 Dr. Teng, who is one of the principal investigators of Project ALS' consortium project. "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."
The research studies were conducted at Brigham and Women's Hospital, Boston Children's Hospital, Sanford-Burnham Medical Research Institute, UMass Medical School, Johns Hopkins University, SUNY-Syracuse, Columbia University and VA Boston Healthcare System.
The research studies were primarily funded by Project ALS, the California Institute for Regenerative Medicine, the National Institute of Neurological Disorders and Stroke the Christopher Reeve Foundation/American Paralysis Association, the Sanford Children's Research Center, the A-T Children's Project and the Zinberg Foundation. Additional support to Dr. Teng was provided by a VA Biomedical Research and Development Grant, a VA Rehabilitation Research and Development Grant, and an NIH R21 grant.
Science Translational Medicine