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Stem Cells Provide New Tool for Studying Disease and Identifying ALS Drugs
Results of two studies appearing in Nature Neuroscience (May 2007) demonstrate that embryonic stem cells may provide a new tool for studying disease mechanisms and for identifying drugs to slow ALS, also known as Lou Gehrig’s disease. The studies were performed by Harvard and Columbia University researchers who are participating in an ongoing collaboration with the Project A.L.S./Jenifer Estess Laboratory for Stem Cell Research, the world’s first and only privately funded laboratory focused exclusively on stem cells and ALS.
Led by Tom Maniatis of Harvard University and Kevin Eggan of the Harvard Stem Cell
Institute, and Serge Przedborski and Hynek Wichterle of Columbia University Medical
Center, the two studies demonstrate that embryonic stem cells can be used to create an in
vitro model of ALS, a fatal neurodegenerative disease that selectively destroys motor
neurons, the nerve cells responsible for all voluntary movement.
For many years, scientists have been trying to resolve whether motor neurons die in ALS
because of a problem within the neuron itself—or within cells that surround the neuron. The new studies report successful use of embryonic stem cell-based models for ALS that
may help scientists to answer this question. With this in vitro model, the Harvard and
Columbia studies report that a set of glial cells (glue-like supporting cells in the spinal
cord) called astrocytes exert a toxic effect on motor neurons when they express an ALS
disease gene.
The discovery that ALS gene expression causes astrocytes to produce toxic mediators
provides a new insight into the origins of the cellular damage associated with ALS, as
well as a potential biomarker for early diagnosis of the disease. In the longer term, the
new findings could also provide new targets for therapies aimed at slowing motor neuron
degeneration in ALS.
"The remarkable interest, curiosity and open-mindedness for new and promising lines of
research is emblematic of Project A.L.S.,” added Serge Przedborski, Page and William
Black Professor of Neurology, at Columbia.
“Project A.L.S. is honored and excited to support cutting-edge stem cell studies. We
created the Jenifer Estess Laboratory so that scientists could collaborate intensively on
understanding and treating ALS. Perhaps these cell culture models will help us to
identify the first effective drugs for the disease,” said Valerie Estess, of Project A.L.S.
In 1999, Project A.L.S. began to ask whether stem cells could be helpful in understanding
and treating ALS. Project A.L.S. researchers and clinicians have worked with a range of
stem cell lines and strategies in experiments at Johns Hopkins University, Harvard
University, the Salk Institute, Memorial Sloan-Kettering Cancer Institute, Dalhousie
University, and Columbia University.
In 2002, a Project A.L.S-funded study led by Hynek Wichterle and Thomas Jessell of
Columbia University showed that stem cells could be directed to become functional
motor neurons, the very cells destroyed in ALS. In 2003, a Project A.L.S.-funded team
from Johns Hopkins showed that rats paralyzed with an ALS like syndrome regain
significant motor function after receiving stem cells into cerebral spinal fluid.
In addition to Project ALS support, the new research was funded by grants from the
National Institute of Neurological Disorders and Stroke, the U.S. Department of Defense,
The Stowers Medical Institute and Harvard Stem Cell Institute, the Muscular Dystrophy
Association, the ALS Association, the Parkinson’s Disease Foundation, and the Bernard
and Anne Spitzer Fund.
Founded in 1998, Project A.L.S. is a non-profit, 501(3) whose mission is to find and fund
the first effective treatments and, ultimately, a cure for ALS, a uniformly fatal
neurodegenerative disease. Project A.L.S. drives collaborative research programs in
genetics, drug discovery, stem cells, and disease pathways.
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