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Understanding Molecular Secrets of ALS

Scientists at Cedars-Sinai may be inching closer to understanding how amyotrophic lateral sclerosis (ALS) damages nerve cells.

Scientists may be inching closer to understanding how amyotrophic lateral sclerosis (ALS) damages nerve cells.

In the largest project of its kind, the Answer ALS Research Program headquartered its stem cell creation at Cedars-Sinai and is collecting blood samples from more than 1,000 patients. The research aims to genetically reprogram patient blood cells into powerful stem cells that can then be used to recreate the brain and spinal cord motor neurons that die with the disease. Investigators can then analyze the molecular signatures of these cells and pinpoint how ALS kills neurons.

“Others are not doing this kind of work at this scale,” says Clive Svendsen, PhD, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, and the Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine. “The volume and number of cell lines we’re creating have never been produced anywhere in the world for a single disease.”

Answer ALS released its first batch of open-source information in May 2020, spanning genomic, molecular and clinical data.