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New model of ALS is based on human cells from autopsied tissue
Date:8/11/2011

By isolating cells from patients' spinal tissue within a few days after death, researchers funded by the National Institutes of Health have developed a new model of the paralyzing disease amyotrophic lateral sclerosis (ALS). They found that during the disease, cells called astrocytes become toxic to nerve cells a result previously found in animal models but not in humans. The new model could be used to investigate many more questions about ALS, also known as Lou Gehrig's disease.

ALS can run in families, but in the majority of cases, it is sporadic, with no known cause. The researchers derived astrocytes from patients who had succumbed to either type of ALS, and found that the cells secrete toxic factors that cause nerve cells to degenerate. A similar mechanism has been found in mouse models of ALS.

"The mouse models capture a type of familial ALS that accounts for only 2 percent of all cases. The field has begged for new disease models that can provide a clear window into sporadic ALS," said senior author Brian Kaspar, Ph.D., an investigator at the Nationwide Children's Research Institute (NCRI) in Columbus, Ohio. His collaborators were Jerry Mendell, M.D., director of NCRI's Center for Gene Therapy, and Arthur Burghes, Ph.D., a professor of molecular and cellular biochemistry at Ohio State University in Columbus. Drs. Kaspar and Mendell also hold faculty positions at Ohio State.

The research is reported in Nature Biotechnology and was funded in part by NIH's National Institute of Neurological Disorders and Stroke (NINDS), including a $1.7 million stimulus grant made possible by the American Recovery and Reinvestment Act.

ALS is characterized by the death of motor neurons, which are muscle-controlling nerve cells in the spinal cord. As these neurons die, the body's voluntary muscles weaken and waste away. Death within five years of diagnosis is common. The only approved treatment, riluzole, extends life exp
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Contact: Daniel Stimson
stimsond@ninds.nih.gov
301-496-5751
NIH/National Institute of Neurological Disorders and Stroke
Source:Eurekalert

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