By UCI Health News

CIRM award will support first-of-its-kind clinical trial in humans

This award will support a first-in-human safety and tolerability study of an embryonic stem cell-derived neural stem cell product for Huntington’s disease, a milestone for patients who currently have no available therapies that alter the course of this devastating disorder.

The study will focus on the safety of the cell product, which, it is hoped, can protect brain cells, repair damaged brain circuits and slow or prevent disease progression in humans. This will be the initial test of embryonic stem cell-derived cells in people with Huntington’s disease (HD); previous trials used cells from fetal cells or tissue. The clinical trial is expected to begin in mid-2026. This novel cell therapy has been tested extensively in preclinical safety and efficacy studies and shows promise to advance treatment for HD.

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