CIRM awards UC Irvine neuroscientist another $2 million for Huntington's disease research
Goal is to determine why the fatal condition destroys some brain cells but spares others
Irvine, Calif., Oct. 8, 2025 — A UC Irvine neuroscientist has been awarded more than $2 million by California’s stem cell agency to probe one of medicine’s most baffling mysteries: why Huntington’s disease devastates some brain cells while sparing others.
Leslie M. Thompson, the Donald Bren Professor of Psychiatry & Human Behavior and Neurobiology & Behavior, will receive $2,056,195 from the California Institute for Regenerative Medicine. Her team plans to use stem cell-based models to map how the defective Huntington’s protein interacts with other proteins and RNA across different cell types. This approach takes advantage of the unique ability of stem cells to differentiate into multiple cell types.
The hope, Thompson says, is to explain the selective vulnerability of neurons to the disorder and, ultimately, to identify new therapeutic targets.
Huntington’s disease is a fatal, inherited condition marked by progressive loss of motor control, psychiatric symptoms and cognitive decline. It stems from a single gene mutation discovered in 1993 – a project in which Thompson played a key role – but the disorder’s biology remains poorly understood.
“The disability, loss of independence and medical costs associated with Huntington’s disease and other neurodegenerative disorders have a profound impact on patients, families and society,” Thompson said. “There have been exciting new advances in the treatment of HD showing that it’s feasible to modify the course of the disease, and this project has the potential to better understand the disease and provide a new approach that can also lead to insights into other brain diseases.”
She has studied Huntington’s for more than two decades and has received $17.6 million in CIRM funding to advance both fundamental science and translational avenues. In 2019, Thompson was awarded $6 million to develop stem cell-based therapies for Huntington’s, an effort that has since cleared an investigational new drug application with the U.S. Food and Drug Administration, paving the way for an early-phase clinical trial through the UC Irvine Alpha Clinic.
Her laboratory integrates patient-derived stem cells, proteomics and bioinformatics to probe disease mechanisms, while also pursuing new avenues such as induced pluripotent stem cells to understand why certain brain cells are more vulnerable than others to Huntington’s disease. She also co-directs UC Irvine’s Sue & Bill Gross Stem Cell Research Center and Institute for Precision Health.
The new award comes through CIRM’s Discovery Program, which supports early-stage regenerative research. Beyond potential therapies, the agency says the program could generate technologies and jobs that contribute to California’s biotechnology sector.
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