Scientists will test the effectiveness of transforming growth factor alpha infusions in mice who have a condition similar to the familial form of amytrophic lateral sclerosis (ALS).
Transgenic mice with a superoxide dismutase gene mutation have a loss of motor neurons, resulting in diminished motor function and a shorter life span.
Studies at other institutions suggest that infusing transforming growth factor alpha directly into the brains of rats with Parkinsonism regenerated dopamine cells within the basal ganglia. These new cells arose from the proliferation, migration and differentiation of stem cells in the rat's subventrical zone.
UW studies are under way to see if transforming growth factor alpha will stimulate the familial ALS mouse's stem cells to replace dying motor neurons and form supporting glial cells. In addition, scientists will see if transforming growth factor alpha protects motor neurons against oxidative damage, harm from neurotransmitters, and a shriveling form of cell death.
This strategy involves administering a drug that may perhaps encourage the recipients' own stem cells to divide and ultimately replace lost or damaged neural cells. The researchers emphasize that this technique does not involve the transplantation of embryonic or adult donor stem cells.
The study is funded by a grant from Project ALS, a philanthropy formed by members of the arts and entertainment industry. The research is also in collaboration with a local biotechnology company, Stem Cell Pharmaceuticals, Inc.
The co-principal investigators are Daniel Lazar and Michel Kliot, both from the UW Department of Neurological Surgery, Nancy Schuman from the UW Department of Biobehavioral Nursing and Health Systems, and Jim Fallon from the Department of Neurobiology at University of California Irvine.
