Trial Overview
UCI Health has initiated a phase 1b/2a clinical trial for Huntington's disease using hNSC-01 neural stem cells derived from embryonic stem cells. The first patient received the intervention in May, and a second is scheduled for July.
The Intervention
The therapy, hNSC-01, involves pluripotent neural stem cells that—in animal studies—have shown the ability to:
- Protect existing brain cells
- Replace lost cells
- Rebuild impaired circuits
- Release helpful proteins (e.g., BDNF)
- Reduce harmful protein accumulations
The cells were manufactured at the UC Davis GMP facility.
"The first patient intervention went very well. To date, they haven't reported any serious adverse events." — Dr. Ravi Rajmohan, principal investigator
Delivery Method
Stem cells are implanted during a six-hour surgical procedure under general anesthesia. Using an MRI scanner, cells are delivered directly into the striatum via a proprietary navigation and delivery platform.
Enrollment
The trial plans to enroll 21 early-stage Huntington's disease patients aged 18–65:
- 12 patients in a phase 1b dose-escalation group
- 9 patients in a phase 2a expansion group
Funding
The trial is funded by a $12 million grant from the California Institute for Regenerative Medicine (CIRM) and coordinated through UC Irvine's Alpha Clinic.
Statements
Leslie M. Thompson, Ph.D. , trial sponsor: "This clinical trial highlights the important role that an interdisciplinary academic and clinical team together with the HD families plays in advancing medicine."