Researchers identify another potential ALS treatment avenue

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.

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