A new gene therapy, AMT-130, has delivered promising results for Huntington’s disease, according to a phase I/II study supported by NIHR.
Key Findings
- Slowed disease progression: High-dose patients showed 75% less progression over 36 months compared to a historical cohort from Enroll-HD.
- Motor and cognitive benefits: Participants experienced slower degeneration in movement, thinking, and daily functioning.
- Safety: AMT-130 was generally well-tolerated, with a manageable safety profile.
Study Details
- Conducted at NIHR UCLH Clinical Research Facility and other UK sites.
- Lead neurosurgeries by Professor Liam Gray, Advanced Neurotherapies Centre, Cardiff.
- Scientific lead: Professor Sarah Tabrizi, UCLH, supported by the Biomedical Research Centre.
- 29 patients completed up to 36 months; 12 received high-dose therapy.
Why It Matters
Huntington’s disease is a fatal genetic disorder affecting movement, thinking, and behaviour, with 8,000 people living with it in the UK. Until now, no treatments could meaningfully slow disease progression.
Professor Tabrizi called the results “the most convincing in the field to date”, while Professor Edward Wild described it as “the dawn of a new age for families impacted by Huntington’s disease.”
AMT-130 is the first gene therapy tested in humans for Huntington’s and represents a potentially disease-modifying treatment, offering hope to patients and families after decades of research.
