For the first time, researchers have been able to slow the course of Huntington’s disease with a gene therapy that tackles the condition at its genetic root. The therapy, called AMT-130, was tested in a clinical trial led by University College London in collaboration with the biotech company uniQure. According to the BBC, patients who received the therapy showed about a 75 per cent reduction in the pace of their disease progression over three years, a result scientists describe as a watershed moment in neurodegenerative research.
Huntington’s is caused by a mutation in a single gene that leads to the production of a toxic protein called huntingtin. Over time, this protein kills brain cells, leading to uncontrolled movements, cognitive decline and psychiatric symptoms. Until now, there has been no treatment capable of slowing or halting that process. The new therapy delivers a strand of microRNA directly into parts of the brain most affected by the disease, silencing the faulty gene and reducing levels of the harmful protein.
How the trial worked
The therapy was administered through a delicate surgical procedure that lasted up to 20 hours, in which neurosurgeons infused the treatment into the striatum and cortex, two key regions that degenerate in Huntington’s. Early results show that treated patients not only fared better in motor and cognitive tests, but also had lower levels of neurofilament light chain, a biomarker of neuronal damage. Reporting in The Guardian noted that these reductions in damage markers strongly suggest the therapy is slowing the underlying disease process rather than just masking symptoms.
The trial involved 29 patients across the UK and US, split into different dosage groups. Those who received the higher dose of AMT-130 saw the clearest benefit, though even the lower-dose patients showed encouraging trends. Importantly, no major safety concerns were observed, with side effects limited to manageable post-surgical complications. As the Washington Post pointed out in its coverage, the durability of the effect, persisting over several years, makes this the first Huntington’s therapy to demonstrate long-term benefit rather than short-lived improvements.
What comes next
For families affected by Huntington’s, the news is nothing short of transformative. Many live with the knowledge that they or their children will inherit a disease that is both fatal and untreatable. The trial’s success offers a glimpse of a future where that inevitability may be challenged. It also validates the broader field of gene therapy, showing that highly targeted interventions can slow neurodegeneration in humans, a goal once thought out of reach.
There are still hurdles. The procedure requires complex neurosurgery, which is costly and not without risk. Questions remain about when the therapy should ideally be given: before symptoms appear, early in progression, or later when damage is already evident. Researchers will need larger studies to confirm effectiveness and to refine dosing. And then comes the challenge of access. As Investors’ Business Daily noted, uniQure’s stock soared nearly 250 per cent after the results were announced, reflecting both excitement and the enormous commercial stakes involved. That raises questions about affordability and how quickly public health systems like the NHS could adopt such a treatment.
Even with those caveats, experts say the breakthrough sets a precedent for tackling other brain diseases with similar strategies. If Huntington’s, a condition with a single known genetic trigger, can be slowed, it may pave the way for therapies targeting the complex genetics of Alzheimer’s, Parkinson’s or motor neurone disease.
For the first time in decades, people living with Huntington’s can imagine a future where diagnosis doesn’t equal decline. The science is still young, but the principle has been proven: it is possible to intervene in the genetic machinery of a neurodegenerative disease and change its course.