UC Irvine Launches Groundbreaking Clinical Trial for Huntington’s Disease

The University of California, Irvine (UC Irvine) has secured a grant of $11,999,933 from the California Institute for Regenerative Medicine to initiate a pioneering clinical trial of a novel neural stem cell therapy aimed at treating Huntington’s disease. This trial, led by Leslie M. Thompson, a distinguished professor of psychiatry, neurobiology, and human behavior, represents a significant advancement in the search for effective treatments for this debilitating genetic disorder.

The clinical trial will be the first to evaluate the safety and tolerability of an embryonic stem cell-derived neural stem cell therapy, known as hNSC-01, in human patients. This innovative approach aims to protect and repair damaged brain cells in individuals suffering from Huntington’s disease, which currently has no therapies that can alter its progression. The study is scheduled to commence in mid-2026, focusing on the safety of the cell product and its potential to restore brain function.

Huntington’s disease is a hereditary condition that typically manifests between the ages of 35 and 50, leading to a gradual decline in cognitive and motor functions over a span of 10 to 20 years. Patients experience a range of symptoms, including involuntary movements, cognitive difficulties, and emotional changes. The new therapy aims to address these challenges by utilizing neural stem cells that can protect existing brain cells, replace those lost due to the disease, and rebuild impaired brain circuits.

The clinical trial will involve 21 participants diagnosed with early-stage Huntington’s disease, divided into two groups: 12 in a Phase 1B dose-escalation group and 9 in a Phase 2A expansion group. The delivery of the stem cells will be performed surgically, and participants will be closely monitored for safety and preliminary signs of therapeutic benefit.

The economic burden of Huntington’s disease is substantial. In California, hospital costs for patients can range from $3 million to $25 million annually. If successful, the hNSC-01 therapy could not only improve the quality of life for patients, allowing them to maintain independence for longer periods, but could also significantly decrease long-term care costs, providing relief to families and caregivers.

“This trial is an exciting step for regenerative medicine and its potential to change the course of Huntington’s disease,” said Thompson, who has dedicated over 30 years to researching Huntington’s disease. She expressed hope that the trial would bring additional options to patients and families facing limited treatment choices. Thompson acknowledged the collaborative effort behind the trial, highlighting contributions from Dr. Jack Reidling and Yuna Muyshondt at UC Irvine, as well as the UC Davis GMP facility involved in producing the cell product.

Thompson has previously received $17.6 million in funding from the California Institute for Regenerative Medicine to advance both fundamental and translational research related to Huntington’s disease. Her prior work, including a $6 million grant awarded in 2019 for developing stem cell-based therapies, has paved the way for this current clinical trial, which has cleared an investigational new drug application with the FDA.

In addition to her work on the clinical trial, Thompson has been awarded $2 million in October 2023 to investigate why Huntington’s disease selectively affects certain brain cells while sparing others. Her laboratory employs a range of techniques, including patient-derived stem cells and advanced bioinformatics, to explore the underlying mechanisms of the disease.

This initiative is part of the California Institute for Regenerative Medicine’s Clinical Trial Stage Projects, designed to accelerate promising stem cell therapies from late-stage preclinical development through clinical trials, addressing significant unmet medical needs. The trial at UC Irvine marks a crucial step towards potentially transformative therapies for Huntington’s disease, offering hope to countless patients and their families.