First Patient Dosed in Neural Stem Cell Therapy for Huntington’s Trial at UCI Health
The embryonic-derived cell therapy is implanted directly into the brain, and this early trial hopes to establish safety and baseline efficacy.
Key Points
- UCI Health has treated the first patient in a Phase 1b/2a clinical trial testing hNSC-01, an embryonic stem cell-derived neural stem cell therapy for Huntington’s disease.
- The 21-patient study is evaluating safety, with 12 participants in a dose-escalation Phase 1b group and nine in a Phase 2a expansion group.
- According to the researchers, the first patient received the intervention in May and has not reported any serious adverse events to date.
First patient treated in Huntington’s study
UCI Health has treated the first patient in what it describes as the world’s first in-human clinical trial of an embryonic stem cell-derived neural stem cell therapy for Huntington’s disease. The Phase 1b/2a study is testing the safety of hNSC-01 in people with early-stage disease.
The patient was dosed in May, and the second is scheduled to receive treatment in July.
The trial as a whole is expected to enroll 21 patients ages 18 to 65, including 12 participants in a Phase 1b dose-escalation group and nine in a Phase 2a expansion group.
“The first patient intervention went very well. To date, they haven’t reported any serious adverse events,” said UCI Health neurologist Dr. Ravi Rajmohan, the clinical trial’s principal investigator. “This trial may help us move one step closer to a future with available treatments that could potentially slow the progression of Huntington’s disease.”
What is hNSC-01?
hNSC-01 uses pluripotent neural stem cells derived from embryonic stem cells manufactured through the UC Davis GMP facility. The company and researchers mention that in animal studies, the cells were shown to:
- Protect existing brain cells
- Replace lost cells
- Rebuild impaired brain circuits
- Release helpful proteins such as brain-derived neurotrophic factor (BDNF)
- Reduce harmful protein accumulations that damage brain cells
With good long-term safety.
It’s delivered during a roughly six-hour surgical procedure under general anesthesia. The cells are implanted directly into the striatum, a deep brain region affected in Huntington’s disease, using a stereotactic navigation and delivery system in an MRI suite.
The study is the result of years of basic and translational research at UC Irvine led by Leslie M. Thompson, Ph.D., and funded by the California Institute for Regenerative Medicine. The trial is supported by a $12 million CIRM grant and coordinated through the UC Irvine Alpha Clinic.
Huntington’s disease is a fatal genetic disorder that gradually destroys brain cells, typically beginning between ages 35 and 50 and progressing over 10 to 30 years. Symptoms can include involuntary movements, difficulty with thinking and planning, and mood changes such as depression. The researchers say that if the therapy is successful, it could help extend independent living and reduce the burden on families and caregivers.
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