UC Irvine Awarded $12 Million for First Human Trial of Stem Cell Therapy in Huntington’s Disease

Leslie M. Thompson, professor of psychiatry and human behavior at UC Irvine, has received a $12 million grant from the California Institute for Regenerative Medicine to support a first-in-human clinical trial of a novel neural stem cell therapy for Huntington’s disease (HD).

The award will fund an initial safety and tolerability study using an embryonic stem cell-derived neural stem cell product in patients with HD.

HD is a genetic disorder that progressively destroys brain cells, typically beginning between ages 35 and 50, and worsening over one to two decades. Current treatment options do not alter the course of this disorder.

This will be the first clinical trial in humans of embryonic stem cell-derived cells for HD (previous trials have used fetal tissue-derived cells), and they’re expected to begin in mid-2026.

The investigational therapy, known as hNSC-01, uses neural stem cells designed to:

• Protect existing brain cells from degeneration
• Replace lost or damaged brain cells
• Rebuild impaired brain circuits
• Release proteins such as BDNF (brain-derived neurotrophic factor), which are deficient in HD patients
• Reduce harmful protein accumulations associated with neuronal damage

These effects have been demonstrated in animal studies where the therapy reportedly improved movement, restored brain function, and showed long-term safety.

The UC Irvine clinical trial plans to enroll 21 individuals with early-stage Huntington’s disease:

• Twelve participants will be included in a Phase 1B dose-escalation cohort.
• Nine participants will join a Phase 2A expansion group.

Cells will be delivered surgically into the brain. Participants will be monitored closely for safety outcomes as well as preliminary evidence of benefit.

Want to keep up on regenerative medicine? Get the weekly newsletter here.

Similar stories

Encellin and Breakthrough T1D Planning Phase 1 of Immune-Protected Cell Therapy for Type 1 DiabetesSep 23, 2026Webinar: Scaling iPSC Therapies Without Scaling VariabilityOn-DemandAspen Neuroscience Publishes Preclinical Data on Parkinson’s iPSC-Derived Cell TherapySep 21, 2026Made Scientific Acquires Resilience’s 85K-Square-Foot Cell Therapy FacilitySep 21, 2026

Editor’s Picks

Century Therapeutics Reports Sustained Glucose Control With iPSC-Derived Islets in Diabetic Mice, IND SoonOct 20, 2026Umbilical Cord Mesenchymal Stem Cell Injections Lower Pain Scores More Than Hyaluronate in 50-Patient Severe Knee Osteoarthritis TrialOct 18, 2026Stempeutics Nabs US Patent for Osteoarthritis Treatment Combining Bone Marrow Stem Cells and Hyaluronic AcidOct 18, 2026Novadip Secures €10.4M for Pivotal Phase 3 Bone Repair Cell Therapy TrialSep 21, 2026