In a groundbreaking advancement, researchers have reported that a new gene therapy could significantly slow the progression of Huntington’s disease, a hereditary brain disorder that leads to loss of motor control and cognitive decline.

Traditionally, treatment for Huntington’s has focused on managing symptoms rather than altering disease progression. However, a recent clinical trial has demonstrated that the experimental therapy AMT-130 may reduce disease progression by up to 75% compared to patients who did not receive the treatment.

Developed by uniQure, AMT-130 is a one-time gene therapy delivered via a surgical injection into specific brain regions. The study, led by University College London (UCL) researchers, followed 29 patients with Huntington’s disease in a phase I/II trial. Of these, 12 participants received a higher dose of the therapy and showed significant improvement after three years, including reduced levels of a spinal fluid protein linked to nerve damage.

According to Professor Anne Rosser, Clinical Neurosciences expert at Cardiff University and principal investigator for the UK arm of the study, these findings represent “a highly significant step suggesting Huntington’s disease may be modifiable.” She noted that while results are preliminary and not yet peer-reviewed, they offer proof-of-concept that the disease’s progression can potentially be slowed.

Still, researchers caution that more extensive studies are needed. The trial’s limited sample size, ongoing surgeries in the U.S., and lack of published peer-reviewed data mean that conclusions remain early-stage. Furthermore, the therapy’s delivery through a lengthy neurosurgical procedure presents practical challenges that future studies must address.

Despite these limitations, experts see enormous potential. Dr. Kan Cao, Professor at the University of Maryland, commented that this marks “the first durable, disease-modifying effect in humans” for Huntington’s disease. If validated in larger trials, the therapy could transform Huntington’s from an “untreatable degenerative condition” into one where slowing or halting progression becomes clinically possible.

The drug developer uniQure plans to seek accelerated FDA approval in early 2026, followed by applications in the UK and Europe.

Original Source: Medical News Today – “Gene therapy slows Huntington’s disease for first time, clinical trial shows”