UC Irvine Receives CIRM Grant for Huntington’s Disease Clinical Trial

Irvine, Calif. – Leslie M. Thompson, Donald Bren Professor of psychiatry and human behavior and also neurobiology and behavior at the University of California, Irvine, has received an $11,999,933 grant from the California Institute for Regenerative Medicine for an unprecedented clinical trial of a novel neural stem cell therapy for Huntington’s disease.

This award will support a first-in-human safety and tolerability study of an embryonic stem cell-derived neural stem cell product for Huntington’s disease, a milestone for patients who currently have no available therapies that alter the course of this devastating disorder.

The study will focus on the safety of the cell product, which researchers hope can protect brain cells, repair damaged brain circuits, and slow or prevent disease progression in humans. This will be the initial test of embryonic stem cell-derived cells in people with Huntington’s disease (HD); previous trials used cells from fetal cells or tissue. The clinical trial is expected to begin in mid-2026. This novel cell therapy has undergone extensive preclinical safety and efficacy studies and shows promise to advance treatment for HD.

Huntington’s disease is a genetic disorder that gradually destroys brain cells, usually starting 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 therapy being tested, called 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 BDNF that are low in HD patients, and reduce harmful protein accumulations that damage brain cells. Animal studies have demonstrated these outcomes, showing improved movement, restored brain function, and long-term safety.

The clinical trial at UC Irvine will enroll 21 people 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 subjects will be closely monitored for safety and also preliminary signs of potential benefit.

Huntington’s disease places a huge burden on