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HSCI researchers identify another potential ALS treatment avenue
Date:8/6/2014

Cambridge, MA, Aug 6 - A series of studies begun by Harvard Stem Cell Institute (HSCI) scientists eight years ago has lead to a report published today that may be a major step forward in the quest to develop real treatments for amyotrophic lateral sclerosis, ALS, or Lou Gehrig's disease.

The findings by Harvard professor of Stem Cell and Regenerative Biology (HSCRB) Kevin Eggan and colleagues also has produced functionally identical results in human motor neurons in a laboratory dish and in a mouse model of the disease, demonstrating that the modeling of human disease with customized stem cells in the laboratory could someday relatively soon eliminate some of the need for animal testing.

The new study, published today in Science Translational Medicine, suggest that compounds already in clinical trials for other purposes may be promising candidate therapeutics for ALS. The Harvard authors found that genetically intervening in the pathway these drugs act on increased survival time of an ALS animal model 5-10 percent, and while that is a long way from curing the universally fatal neurodegenerative disease, "any ALS patient would be excited about this extended life span," said Eggan, who pioneered the disease in a dish concept.

Sophie De Boer, a graduate student in Eggan's lab, is the first author on the Science Translational Medicine paper.

This latest finding is expected to push towards clinical studies the second major ALS discovery from Eggan's lab in less than a year. The HSCI stem cell biologist, and his neuroscience and neurology collaborators at Massachusetts General Hospital and Boston Children's Hospital, are preparing for a phase I clinical trial of a medication already approved for epilepsy which Eggan and colleagues discovered quiets disease related electrical excitability in the motor neurons effected in ALS.

In a paper in 2007, Eggan and colleagues demonstrated that glial cells, background cells
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Contact: B. D. Colen
617-495-7821
Harvard University
Source:Eurekalert  

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