Huntington’s disease treatment slows progression by 75%, study finds

Gene therapy slowed Huntington’s disease progression by 75 per cent in a recent trial, in what researchers describe as the first effective treatment for the condition.

The therapy means that decline expected in one year would instead take four years, potentially giving patients decades of better quality life, researchers said.

Researchers from University College London Huntington’s Disease Centre reported results from the trial of 29 patients, who received a one-off gene therapy during 12 to 18 hours of brain surgery.

Professor Sarah Tabrizi, director of the UCL centre, described the results as “spectacular.”

She said: “We never in our wildest dreams would have expected a 75 per cent slowing of clinical progression.”

The treatment uses a modified virus to deliver specially designed DNA deep into two brain regions – the caudate nucleus and putamen – with real-time MRI guiding a microcatheter during surgery.

Once inside brain cells, the DNA produces microRNA – small genetic fragments that intercept and disable instructions for building the toxic huntingtin protein that causes the disease.

This permanently lowers levels of the harmful protein.

Three years after surgery, patients showed an average 75 per cent slowing of disease progression, measured through cognition, motor function and daily living abilities.

Some returned to work after early medical retirement, while others kept walking when wheelchairs were expected.

Brain cell death markers in spinal fluid, which would normally have risen by a third over that time, were actually lower than at the trial’s start.

Professor Ed Wild is consultant neurologist at the National Hospital for Neurology and Neurosurgery at UCLH.

Wild said: “There was every chance that we would never see a result like this, so to be living in a world where we know this is not only possible, but the actual magnitude of the effect is breathtaking, it’s very difficult to fully encapsulate the emotion.”

He said he was “a bit teary” thinking about the impact on families and believes the therapy “should last for life” as brain cells are not replaced like blood, bone or skin cells.

UniQure, the company behind the therapy, says it will apply for a licence in the US in the first quarter of 2026, with launch expected later that year.

Discussions with regulators in the UK and Europe are due to start next year.