First human trial using stem cells for Huntington's disease begins
First human trial using stem cells for Huntington's disease begins
Updated at: June 26, 2026 at 09:15 AM
In June 2026, the medical community hit a major milestone with the launch of the REGEN4HD clinical trial.
This marks the world’s first human study testing an embryonic stem cell-derived therapy for Huntington’s disease.
Huntington’s is a progressive, inherited neurodegenerative disorder that currently lacks a cure.
The trial focuses on an experimental therapy called hNSC-01, which consists of neural stem cells derived from human embryos.
Unlike gene therapies that aim to turn off toxic proteins, this regenerative approach involves implanting cells directly into the striatum—the region of the brain most affected by the disease.
While the primary goal of this phase is to assess safety and tolerability, preclinical models have already shown promising results in improving motor function and restoring brain signaling.
This trial offers new hope for addressing an unmet medical need in neurodegenerative care.
