What the early results show
News from a European biotech company on a new gene therapy has sparked guarded optimism in the Huntington’s disease community. The approach, delivered during brain surgery, targets the genetic error that drives the condition. In a small, preliminary trial involving roughly 30 participants, the highest dose of the treatment appeared to slow disease progression by about 75% over three years for those who received it at that dose. The trial size is limited and the therapy did not cure Huntington’s disease, but the reduction in pace of decline represents the most encouraging signal in years.
University College London researchers, who helped lead parts of the project, described the findings as a potential turning point. Ed Wild, the principal investigator at UCL’s Huntington’s disease centre, told CBC’s As It Happens that the results generate “unbridled optimism” and that this is the best news the Huntington’s community has seen to date.
Why this matters for people living with Huntington’s disease
Huntington’s disease is a hereditary neurodegenerative disorder that gradually erodes movement, memory and behavior. For many, the disease accelerates during what should be productive years, eventually shortening life expectancy. Slowing progression, even without a cure, could meaningfully improve quality of life—allowing people to stay in work longer, maintain independence longer, and preserve cognitive function for more time.
Quality-of-life implications
Dr. Sarah Tabrizi, director of UCL’s Huntington’s Disease Centre, emphasized that even a slower disease trajectory could translate into tangible daily benefits. “People will be able to stay in work longer, function longer, and maintain their independence,” she noted during a company conference call.
Approach and what we know about AMT-130
The therapy, named AMT-130, uses a modified viral vector delivered directly to the brain during surgery to lower levels of the mutant huntingtin protein. The goals are to reduce the abnormal protein that disrupts neuron function, potentially slowing the disease’s advance. The operation is lengthy—carried out with real-time MRI guidance—and targets two brain regions thought to be central to Huntington’s pathology. While the prospect of bypassing a fatal disease is compelling, the therapy is not a cure, and the company stresses that the data are early.
In context, these gene therapies can command high price tags; previous single-dose efforts have cost around $2 million. The current sponsor says it plans to discuss the data with U.S. regulators this year as it prepares for a 2026 FDA submission for marketing approval, assuming continued positive signals.
Important caveats and questions
Analysts caution that the results come from a small study and the data have not yet been publicly released or peer-reviewed. Jonathan Kimmelman, a bioethics professor at McGill University, warned that early press releases can present an exuberant and selective view of the findings. He noted that the results appear to slow progression rather than halt or reverse it.
Other experts, such as Rachel Harding of the University of Toronto, welcomed the development but highlighted unresolved questions: how long the effect lasts, whether benefits extend to all patients or only those eligible for brain surgery, and how dosing, timing, and long-term safety will be managed. The trial’s limited sample size—12 patients at the highest dose followed for 36 months—means long-term data are essential before broad conclusions can be drawn.
What happens next
Beyond the immediate scientific questions, regulatory steps loom large. The company has scheduled meetings with the FDA to share interim data and plan for a formal 2026 submission. Independent confirmation through peer review and additional, larger trials will be critical to determine whether AMT-130 becomes a standard option for Huntington’s disease, and for which patient groups it may be appropriate.
Context and cautious optimism
In the wake of uncertain times for Huntington’s research, the current results offer a glimmer of hope without guaranteeing a cure. If subsequent data corroborate these findings, the approach could become part of a multi-pronged strategy to manage the disease—complementing symptomatic therapies and other disease-modifying approaches under investigation.
