20 Sep UW-Madison receives $7.2 million grant to develop ALS stem cell therapy
Madison, Wis. – A team of University of Wisconsin-Madison researchers will explore the potential of stem cells and natural growth factors to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, after receiving a $7.2 million grant from the National Institutes of Health.
The five-year grant will fund research aimed at finding new therapies for treating the debilitating and nearly always fatal condition, which is caused by the withering of motor neurons – the brain cells that control the body’s muscles.
There are no proven effective treatments for ALS, which afflicts an estimated 30,000 people in the United States. Most patients die within three to five years of diagnosis.
UW-Madison neuroscientist Clive Svendsen, a stem cell researcher at the Waisman Center, will direct the project along with fellow university neuroscientists Su-Chun Zhang, a professor of anatomy in the School of Medicine and Public Health, and Gordon S. Mitchell, a professor in the School of Veterinary Medicine.
According to Svendsen, the new grant is important because it addresses unexplored issues in regenerative medicine, an emerging field in which diseased or damaged tissues and cells are replaced or regenerated.
On their nerves
The grant will support a combined cell-based approach to treating ALS. The Wisconsin program will use both embryonic and fetal stem cells and will explore the possibility of stimulating healthy nerve cells to release growth factors and other chemicals to protect motor neurons. It also will focus on three strategies for promoting healthy motor neurons and connecting new and rescued motor neurons to the muscles they control.
The approach will be tested in concert in a rat model for ALS. Previous work at UW-Madison has shown that neural cells derived from fetal tissue and engineered to release a key growth factor known as GDNF, a chemical that promotes cell health, protected motor neurons in rats with ALS.
However, the rescued nerve cells did not reattach to the muscles they control.
The new study may prove there are synergistic effects between the two types of cells that protect and augment motor neurons in the animal model, and promote connections with muscles.
Zhang has successfully derived motor neurons from embryonic stem cells.
“We’re putting them right into the rat model and assessing their effects,” Svendsen explained in a release. “Motor neurons don’t survive very well in transplants, and the hope is the cells in combination with GDNF release may promote a better result.”
Also planned are studies to address the failure of the respiratory system in ALS, which is the ultimate cause of death for patients with the disease.
Work by Mitchell will include inducing hypoxia, a condition where tissues are deprived of oxygen, to prompt the release of growth factors that have neuroprotective qualities on the respiratory motor neurons in the lungs.
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