26 September 2025
The Advanced Neurotherapies Centre performed the gene therapy surgery as part of a global clinical trial for a new Huntington’s disease treatment.
Our director, Professor Liam Gray, performed the surgeries here in Cardiff, making us along with the team at Cardiff and Vale University Health Board, the only centre in the UK that can perform the surgeries for this trial.
Clinicians, researchers, and theatre teams in Cardiff also supported patient assessment, surgical delivery and post-operative follow-up.
Early results from the uniQure AMT-130 gene therapy clinical trial for Huntington’s has shown a 75% slowing of symptoms in the patients delivered the gene therapy.
Previously considered untreatable, and passed down through families, Huntington’s disease is genetic disorder caused by a mutation in the Huntingtin gene which causes the gradual and progressive breakdown of neurons in the brain.
The results of the clinical trial so far indicate that, if treated, patients with Huntington’s may have decades more healthy life or may never develop symptoms at all.
Professor Gray said “This is a landmark result for patients with this terrible disease and their families. We are delighted to have worked with Prof Rosser, here in Cardiff, and Profs Tabrizi and Wild in London to operate on their patients, delivering the gene therapy directly into the brain. Cardiff is the only UK centre – and only one of a few centres worldwide – performing these gene therapy surgeries.”
Speaking during an interview with BBC Radio Wales, Professor Liam Gray reflect further on the hopeful results of the trial, adding: “Nothing has ever modified this disease before and the magnitude of the modifications we are seeing is very significant.”
The gene therapy is not yet licensed, but there is widespread positivity about the results and hope for its future availability.
During the surgery, which takes between 12 and 14 hours to complete, a route is carefully planned into the brain using an MRI scanner.
The patient is put under general anaesthetic and, while still inside the MRI scanner, surgeons create small openings in the skull and thread fine catheters into the target areas.
The gene therapy is then infused at a controlled pace through convection-enhanced delivery to distribute the therapy through the tissue. After surgery, patients are monitored closely in hospital and then return for follow-up visits and scans.
Professor Gray concluded: “We are thrilled to have played a vital part in this ground-breaking trial. We wish to acknowledge the courage of our patients undergoing these experimental clinical trials, the work of uniQure, and the fantastic clinical and research teams here in Cardiff making these therapies a reality.”
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