A pioneering gene therapy has successfully slowed the progression of Huntington’s disease by 75% in patients after three years, marking the first effective treatment for the devastating genetic disorder, researchers announced on Tuesday.
Huntington’s, caused by a single faulty gene, gradually destroys brain cells, leading to dementia, uncontrolled movements, and paralysis, often proving fatal within a decade of diagnosis. Symptoms typically emerge in a person’s 30s or 40s, including mood swings and depression, with later stages bringing severe disability. Until now, treatments have only managed symptoms, offering no cure.
“We now have a treatment for one of the world’s more terrible diseases. This is absolutely huge. I’m really overjoyed,” said Prof. Sarah Tabrizi, director of University College London’s Huntington’s Disease Centre, who led the trial, The Guardian reported.
The therapy, developed by uniQure, involves a single 12- to 20-hour surgical procedure where a modified virus delivers DNA into the brain to block the production of a toxic huntingtin protein. Early results from 29 patients in the UK and the U.S. showed a significant slowdown in disease progression, measured by motor function, cognition, and daily life skills. Brain scans also revealed lower levels of neurofilaments — a marker of cell death — suggesting neurons are being preserved.
Robyn Perry, a 34-year-old Liverpool support worker who tested positive for the gene at 20 while caring for her father with Huntington’s, called the news a turning point.
“We’ve fought for so long for a cure. For this day to come – it means absolutely everything for us and our families,” she said, her voice reflecting years of struggle as she now experiences mild symptoms herself.
Jack May-Davis, a 30-year-old barrister’s clerk with the gene, echoed the sentiment, saying the breakthrough left him “overwhelmed” and offered a “brighter” future after watching his father’s decline, BBC reported.
The treatment’s impact could extend life quality significantly, Tabrizi noted, per The Guardian.
People would be able to work and live independently for significantly longer with treatment, she said, hinting at potential prevention if administered before symptoms appear.
This has spurred hope, with Tabrizi predicting more will seek genetic testing.
“Now I think many more people will come forward for the genetic test because there’s a treatment,” she added.
The trial’s success has researchers optimistic.
“We never in our wildest dreams would have expected a 75% slowing of clinical progression,” Tabrizi told BBC News, describing the results as “spectacular.”
Prof. Ed Wild, a consultant neurologist at UCLH, was moved by the findings.
“This is the result we’ve been waiting for … the magnitude of the effect is breathtaking,” he said, per BBC, admitting he felt “a bit teary” thinking of how the treatment could impact affected families.
Walid Abi-Saab, uniQure’s chief medical officer, called the results “incredibly exciting,” with plans to seek U.S. approval in early 2026 and launch the drug later that year, followed by discussions with UK and European regulators.
However, the therapy’s complexity and cost may limit access.
“It will be expensive for sure,” Wild cautioned, though he noted long-term benefits could justify the price. With 6,000 to 10,000 affected individuals and another 20,000 carriers in the UK alone, the treatment could transform lives if scaled effectively.
Tabrizi is planning to do a prevention trial with a group of young people who have the gene but have no symptoms of Huntington’s disease, to see whether the therapy can significantly delay the onset of symptoms or even stop them altogether.