Leslie Thompson gets $6 million CIRM grant to advance Huntington’s disease treatments

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Leslie Thompson of the Sue & Bill Gross Stem Cell Research Center and UCI MIND has been awarded $6 million by the California Institute for Regenerative Medicine to continue her CIRM-supported efforts to create stem cell treatments for Huntington’s disease. The funding will allow the Thompson lab to conduct the late-stage testing needed to apply to the U.S. Food and Drug Administration for permission to start a clinical trial in people. The therapy involves transplanting stem cells that have been turned into neural stem cells and shown to improve the function of brain cells in HD models. The goal is to slow down progression of the inherited, incurable and fatal neurodegenerative disorder. With this current award, Thompson, a Chancellor’s Professor of psychiatry & human behavior and neurobiology & behavior, has received nearly $12 million in CIRM grants since 2008 to produce stem cell lines from individuals carrying the Huntington’s genetic mutation and – together with an HD consortium – use them to investigate what goes wrong in HD. The neural stem cells are also used to support areas of the brain susceptible to HD.