UC Irvine Secures $12M CIRM Grant to Launch Phase 1/2 Stem Cell Trial for Huntington’s

Leslie M. Thompson, Donald Bren Professor of psychiatry and human behavior and of neurobiology and behavior at the University of California, Irvine, has received a $12-million grant from the California Institute for Regenerative Medicine to test a novel stem cell therapy for Huntington’s disease.

“This trial is an exciting step for regenerative medicine and its potential to change the course of Huntington’s disease,” said Thompson, in a statement. “It provides additional hope to patients and families who currently have very few options.”

The therapy, known as hNSC-01, uses neural stem cells that can protect existing brain cells from dying, replace lost cells, rebuild impaired brain circuits, release helpful proteins such as brain-derived neurotrophic factor that are low in patients with Huntington’s disease, and reduce harmful protein accumulations that damage brain cells. The treatment has been tested in preclinical safety and efficacy studies and shows potential to advance treatment for Huntington’s disease. The clinical trial is expected to begin in mid-2026.

Huntington’s disease is a genetic disorder that slowly destroys brain cells, typically beginning between the ages of 35 and 50 and worsening over 10 to 20 years. Symptoms include involuntary movements, difficulty thinking and planning daily tasks, and mood changes such as depression.

The UC Irvine-led trial will enroll 21 individuals with early-stage Huntington’s disease, with 12 participants in a Phase 1B dose-escalation group and nine in a Phase 2A expansion group. The cells will be surgically delivered into the brain, and participants will be closely monitored for safety and early signs of potential benefit.

In California, hospital costs associated with Huntington’s disease range from $3 million to $25 million annually. If successful, this therapy could allow people with Huntington’s disease to live independently for longer and reduce long-term care costs.

Information for this article was sourced from UC Irvine News.