UC Irvine Secures $11.9 Million for Pioneering Huntington’s Trial

The University of California, Irvine (UC Irvine) has received a significant grant of $11,999,933 from the California Institute for Regenerative Medicine to conduct an innovative clinical trial targeting Huntington’s disease. This funding will support a groundbreaking study on a novel neural stem cell therapy, marking a crucial advancement for patients suffering from this debilitating genetic disorder.

Led by Leslie M. Thompson, the Donald Bren Professor of Psychiatry and Human Behavior, as well as Neurobiology and Behavior, the trial will be the first to test embryonic stem cell-derived neural stem cells in human subjects with Huntington’s disease. The primary focus of the study will be to assess the safety and tolerability of the hNSC-01 therapy, which aims to protect brain cells, repair damaged circuits, and potentially slow or halt disease progression.

The clinical trial is anticipated to commence in mid-2026, enrolling 21 participants diagnosed with early-stage Huntington’s disease. The structure includes a Phase 1B dose-escalation group with 12 participants and a Phase 2A expansion group featuring nine individuals. Cells will be surgically delivered into the brain, with participants monitored closely for any safety concerns and early indications of therapeutic benefits.

Huntington’s disease is a progressive genetic disorder that typically manifests between the ages of 35 and 50, with symptoms intensifying over a period of 10 to 20 years. Key symptoms include involuntary movements, cognitive difficulties, and mood changes such as depression. The economic burden of Huntington’s disease is substantial, with annual hospital costs in California ranging from $3 million to $25 million per patient.

The hNSC-01 therapy has shown promise in preclinical studies, demonstrating its ability to protect existing brain cells, replace lost cells, rebuild impaired circuits, and release beneficial proteins that are typically low in Huntington’s patients. In animal models, the therapy has led to improved movement and restored brain function, while also proving to be safe over prolonged periods.

“This trial is an exciting step for regenerative medicine and its potential to change the course of Huntington’s disease,” stated Leslie M. Thompson, the principal investigator. “It provides additional hope to patients and families who currently have very few options.” Thompson’s research spans over three decades, and she has previously received $17.6 million in funding from the California Institute for Regenerative Medicine to advance both fundamental and translational research in this area.

In 2019, Thompson was awarded $6 million to develop stem cell-based therapies for Huntington’s disease, an initiative that has successfully cleared an investigational new drug application with the FDA. This paved the way for the current funding that will facilitate the early-phase clinical trial through the UC Irvine Alpha Clinic.

In addition to her work on Huntington’s disease, Thompson recently secured $2 million in funding to explore why Huntington’s disease disproportionately affects certain brain cells while sparing others. Her laboratory employs advanced techniques, including patient-derived stem cells and bioinformatics, to investigate disease mechanisms and identify new therapeutic pathways.

The grant is part of the California Institute for Regenerative Medicine’s Clinical Trial Stage Projects, which aim to expedite promising stem cell therapies from late-stage preclinical development through clinical trials to meet unmet medical needs. As the trial moves forward, it holds the potential to transform the lives of individuals living with Huntington’s disease, offering hope for improved independence and reduced long-term care costs.