For the first time, doctors have successfully treated Huntington’s disease – a cruel, inherited condition that destroys the brain and robs people of their independence, often in the prime of life. The results of a landmark gene therapy trial, described as “spectacular” by researchers, have given hope to thousands of families living with the devastating illness.
A devastating disease slowed by 75%
Huntington’s is caused by a faulty gene that turns a vital brain protein into a killer of neurons. The illness usually strikes in a patient’s 30s or 40s, progressing relentlessly until death within 15 to 20 years. Symptoms combine those seen in dementia, Parkinson’s disease and motor neurone disease, leaving families with little treatment beyond palliative care.
Now, results from a trial involving 29 patients suggest the decline can be slowed by as much as 75%. In practical terms, this means a year’s worth of deterioration could instead stretch across four years.
“We never in our wildest dreams would have expected a 75% slowing of clinical progression,” said Professor Sarah Tabrizi, director of the Huntington’s Disease Centre at University College London. “It’s spectacular.”
‘Absolutely incredible’ for families
For families who have lived with the shadow of Huntington’s, the breakthrough offers a sense of relief once thought impossible.
Jack May-Davis, 30, carries the faulty gene. He watched his father, Fred, deteriorate rapidly after symptoms began in his late 30s. Fred eventually needed 24-hour care before dying at 54. Jack’s grandmother, Joyce, also had the disease.
“It was really awful watching my dad’s decline,” Jack said. “I always knew I was destined to share the same fate.”
Now, he says the new treatment leaves him “overwhelmed” and hopeful: “It does allow me to think my life could be that much longer. The future suddenly seems a little brighter.”
How the treatment works
The therapy is a complex blend of cutting-edge genetics and brain surgery. During a 12- to 18-hour procedure, surgeons use real-time MRI scans to guide a microcatheter deep into two regions of the brain – the caudate nucleus and putamen – which are heavily affected by Huntington’s.
A modified, harmless virus is then delivered into brain cells. This virus acts as a “microscopic postman,” carrying a specially designed DNA sequence. Once inside, it instructs the neurons to produce fragments of genetic material, known as microRNA.
These fragments intercept the faulty instructions that normally cause the production of mutant huntingtin protein. As a result, levels of the toxic protein fall, slowing the death of brain cells.
Laboratory tests confirmed this effect: levels of neurofilaments in spinal fluid – a key marker of cell death – fell instead of rising as expected.
Signs of hope in daily life
The impact on patients’ lives has been profound. One individual who had taken early medical retirement has returned to work. Others, who were expected to be using wheelchairs by now, are still walking independently.
While the therapy is not a cure, it significantly changes the outlook for those affected. For many, it represents the first real glimmer of hope since the gene responsible was identified in 1993.
Professor Ed Wild, consultant neurologist at UCLH, admitted he was “a bit teary” thinking of the trial’s impact. “There was every chance we would never see a result like this,” he said. “The actual magnitude of the effect is breathtaking.”
Challenges ahead: cost and complexity
Despite the breakthrough, significant challenges remain. The treatment involves highly specialised surgery and is likely to be expensive. Gene therapies already on the market can cost millions per patient.
“This will be expensive for sure,” Prof Wild admitted. “But if it lasts for life, as we expect it might, then it could still be affordable compared with decades of care.”
In the UK, the NHS already funds £2.6m-per-patient therapy for haemophilia B. Advocates believe Huntington’s patients deserve the same opportunity.
What’s next for the therapy
The trial was conducted by Dutch biotech company uniQure, which plans to apply for a US licence in early 2026. Discussions with European and UK regulators are expected to begin next year. If approved, the therapy could be available to patients later this decade.
Importantly, researchers are already planning the next phase: a prevention trial. By treating people who carry the gene but have not yet developed symptoms – known as stage zero Huntington’s – scientists hope it may be possible to delay or even prevent the disease entirely.
“This is the beginning,” Prof Tabrizi said. “We want to open the gates for therapies that can reach more people.”
The emotional toll of Huntington’s
For families, the diagnosis of Huntington’s can feel like a life sentence. If one parent carries the gene, each child has a 50% chance of inheriting it. Many live for years knowing they are at risk but unable to change their fate.
Mrs Saleem, whose husband carried the gene, described the breakthrough as “a miracle we never thought we’d see in our lifetime”. Others in the community echoed similar feelings of relief, describing the trial as the start of “a new era”.
Why this matters beyond Huntington’s
Experts believe the success of this gene therapy could have far wider implications. The approach of delivering DNA into brain cells could potentially be adapted to tackle other neurodegenerative conditions, including some forms of Alzheimer’s and Parkinson’s.
“This is not just about Huntington’s,” Prof Wild said. “It shows what is possible in tackling diseases we once thought untreatable.”
A cautious optimism
The full results have yet to be peer-reviewed, and larger trials will be needed. But the early signs are overwhelmingly positive.
Dr Walid Abi-Saab, chief medical officer at uniQure, said the findings had “the potential to fundamentally transform” Huntington’s.
For now, families are celebrating a step forward that only a decade ago seemed unimaginable. “We’ve lived with nothing but fear,” Jack May-Davis said. “Now we can finally start to live with hope.”
