Tejal Somvanshi
A new gene therapy called AMT-130 has successfully slowed the progression of Huntington’s disease by 75% in clinical trials, marking the first time any treatment has effectively slowed this devastating neurological condition.
Developed by uniQure, the one-time treatment was tested in 29 patients who received brain surgery to deliver the therapy directly to affected areas. After 36 months, patients given the high dose of AMT-130 showed significantly less disease progression compared to a matched control group.
“This result changes everything,” said Professor Ed Wild from University College London’s Huntington’s Disease Centre. “On the basis of these results it seems likely AMT-130 will be the first licensed treatment to slow Huntington’s disease, which is truly world-changing stuff.”
Huntington’s disease causes a progressive breakdown of nerve cells in the brain, affecting movement, thinking, and emotions. It’s inherited – if one parent has the disease, their children have a 50% chance of developing it too. Symptoms typically start between ages 30-50 and worsen over time.
The treatment works by reducing levels of a toxic protein in the brain. It combines gene therapy with gene silencing technology, delivered through about 8–10 hours of delicate brain surgery. Surgeons use real-time MRI scanning to guide the treatment into two brain regions – the caudate nucleus and the putamen.
Once inside brain cells, the treatment creates genetic material that blocks instructions for making the harmful huntingtin protein. This appears to save brain cells from dying – a key marker of cell death in spinal fluid actually decreased rather than increasing as would be expected in untreated patients.
For patients, this could mean meaningful preservation of everyday abilities. One trial participant who had been medically retired was able to return to work. Others who would have needed wheelchairs by now are still walking.
“My patients in the trial are stable over time in a way I’m not used to seeing in Huntington’s disease,” Professor Wild noted. Jack May-Davis, 30, who carries the Huntington’s gene and lost his father to the disease, called the results “astonishing.”
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“When I started participating in trials I never thought something would be developed in a timeframe that might actually be useful for me,” he said. “This feels like a huge moment that will mean so much to families who carry the Huntington’s gene.”
Professor Sarah Tabrizi, lead scientific adviser on the trial, emphasized what this means for patients: “For patients, AMT-130 has the potential to preserve daily function, keep them in work longer, and meaningfully slow disease progression.” The slowing effect means that decline that would normally happen in one year would instead take four years with treatment, potentially giving patients decades more quality time.
The treatment was considered safe, though some patients developed inflammation from the virus that caused headaches and confusion, which either resolved naturally or needed steroid treatment.
Looking forward, uniQure plans to apply for approval in the United States in early 2026, with conversations about approval in the UK and Europe starting next year. However, challenges remain for widespread access. The treatment requires complex neurosurgery and is expected to be expensive, though specific pricing hasn’t been announced.
Professor Tabrizi remains optimistic, saying this gene therapy “is the beginning” that will open doors for more therapies reaching more people. She’s already working on prevention trials with people who carry the gene but don’t yet show symptoms, hoping to delay or even stop the disease before it starts.
For the approximately 75,000 people with Huntington’s disease in the UK, US, and Europe – and the hundreds of thousands more who carry the mutation – this breakthrough offers the first real hope against a disease that has previously had no effective treatment.
