UC Irvine Receives $12 Million Grant for First-in-Human Huntington's Disease Stem Cell Trial
Key Insights
UC Irvine (search) researcher Leslie Thompson received nearly $12 million from the California Institute for Regenerative Medicine (search) to conduct the first clinical trial of embryonic stem cell-derived neural stem cells for Huntington's disease (search).
The Phase 1B/2A trial will test hNSC-01 (search) therapy in 21 early-stage Huntington's patients, marking the first use of embryonic stem cell-derived cells for this devastating genetic disorder.
The novel therapy demonstrated promising preclinical results, showing ability to protect brain cells, rebuild circuits, and improve movement in animal studies.
The University of California, Irvine has received an $11,999,933 grant from the California Institute for Regenerative Medicine (search) (CIRM) to conduct the first clinical trial of embryonic stem cell-derived neural stem cell therapy for Huntington's disease (search). The award will support a groundbreaking first-in-human safety and tolerability study of a novel cell product for patients who currently have no available therapies that alter the course of this devastating genetic disorder.
Novel Stem Cell Therapy Shows Promise
The therapy being tested, designated hNSC-01 (search), utilizes neural stem cells derived from embryonic stem cells that demonstrate multiple therapeutic mechanisms. In preclinical studies, these cells showed the ability to protect existing brain cells from dying, replace lost cells, rebuild impaired brain circuits, release helpful proteins such as BDNF that are low in Huntington's disease (search) patients, and reduce harmful protein accumulations that damage brain cells.
Animal studies demonstrated that the cells improved movement, restored brain function, and were shown to be safe over long periods. This will be the initial test of embryonic stem cell-derived cells in people with Huntington's disease (search), as previous trials used cells from fetal cells or tissue.
Clinical Trial Design and Timeline
The clinical trial at UC Irvine (search) will enroll 21 people with early-stage Huntington's disease (search), structured as a Phase 1B dose-escalation group with 12 participants and a Phase 2A expansion group with nine participants. The cells will be surgically delivered into the brain, and subjects will be closely monitored for safety as well as preliminary signs of potential benefit. The clinical trial is expected to begin in mid-2026.
Principal investigator Leslie M. Thompson, Donald Bren Professor of psychiatry and human behavior as well as neurobiology and behavior at UC Irvine (search), will lead the study through the UC Irvine Alpha Clinic. "This trial is an exciting step for regenerative medicine and its potential to change the course of Huntington's disease (search)," Thompson said. "It provides additional hope to patients and families who currently have very few options."
Addressing Significant Medical Need
Huntington's disease (search) 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 disease places a huge burden on patients, families and caregivers, often lasting decades.
In California alone, hospital costs for Huntington's disease (search) patients can 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 significantly reduce long-term care costs, easing the strain on families.
Extensive Research Foundation
Thompson has studied Huntington's disease (search) for more than three decades and has received $17.6 million in CIRM funding to advance both fundamental science and translational avenues. In 2019, she was awarded $6 million to develop stem cell-based therapies for Huntington's disease, an effort that has since cleared an investigational new drug application with the FDA, paving the way for the current award that funds the early-phase clinical trial in humans.
The award represents the culmination of a tremendous effort from a team of investigators, including Dr. Jack Reidling and Yuna Muyshondt at UC Irvine (search) and the UC Davis GMP facility that produced the cell product. Thompson also co-directs UC Irvine's Sue & Bill Gross Stem Cell Research Center.
This award is part of CIRM's Clinical Trial Stage Projects, which aim to accelerate promising stem cell therapies from late-stage preclinical development through clinical trials to address unmet medical needs.