A once untreatable and devastating brain disease may finally have met its match.
In what scientists are calling a “spectacular” development, Huntington’s disease long considered one of the cruellest genetic conditions has been successfully treated for the first time, according to breakthrough findings from a UK-led trial.
The condition, often likened to a brutal combination of dementia, Parkinson’s, and motor neurone disease, gradually destroys brain cells and tears through generations of families. But now, there’s real hope on the horizon.
Huntington’s disease successfully treated for first time
A pioneering form of gene therapy has shown remarkable results in early trials, slowing the disease’s progression by a staggering 75%.
“We never in our wildest dreams would have expected a 75% slowing of clinical progression,” said Professor Sarah Tabrizi, Director of the UCL Huntington’s Disease Centre.
To put it simply, what would normally be a year’s worth of decline now takes four. That means decades more quality life – a previously unimaginable future for those diagnosed.
“It does allow me to think my life could be that much longer,” shared Jack May-Davis, a 30-year-old who carries the Huntington’s gene.
Jack, like his late father and grandmother, has always lived with the looming certainty of developing the disease. But now, the narrative is shifting – and hope is replacing fear.
The Science Behind the Miracle
The treatment, developed by biotechnology firm uniQure, is delivered via 12 to 18 hours of intricate brain surgery, during which a genetically modified virus is infused deep into two key brain regions.
Once inside, the virus acts as a microscopic delivery agent, inserting a new DNA sequence that instructs brain cells to silence the faulty gene responsible for producing the toxic huntingtin protein.
This transforms the affected neurons into mini-factories, producing molecules that block the disease at its genetic source. The result? Dramatically reduced levels of the mutant protein and, crucially, preserved brain cells.
Why This Matters So Much
This isn’t just another medical trial. It’s a lifeline for thousands who’ve watched loved ones fade, knowing they were next. Now, for the first time, they have something they never dared imagine – time.
Around 75,000 people in the UK, Europe, and the US currently live with Huntington’s disease, and hundreds of thousands more carry the mutation. Until now, there was no treatment to stop or even slow it.
Patients in the trial experienced improved mobility, independence, and even returned to work – things that were previously impossible as the disease advanced.
Emotional Breakthrough
The research team, many of whom have dedicated their lives to the disease, were visibly emotional upon seeing the data.
“There was every chance that we would never see a result like this. To be living in a world where this is possible. It’s breathtaking,” said Prof Ed Wild, a consultant neurologist at UCLH.
Early Days, Big Hopes
While the results are being celebrated, the treatment isn’t yet widely available. It’s also not cheap – and due to the complexity of the surgery, access may initially be limited.
“It will be expensive for sure,” admits Prof Wild.
However, in the UK, the NHS already covers similarly costly gene therapies, such as the £2.6 million-per-patient treatment for haemophilia B. That offers hope that access may eventually be widened.
uniQure is planning to apply for US regulatory approval in early 2026, with UK and European discussions expected to follow.
This may only be the beginning. Researchers are now looking at ways to use the same therapy preventatively, before symptoms ever appear.
Prof Tabrizi is already working with individuals who carry the gene but haven’t yet developed symptoms – so-called “stage zero” patients.
“This is the beginning. We are overjoyed for the patients and families,” she says.
The first-ever prevention trial could soon be underway – and may open the door to stopping Huntington’s before it starts.
Huntington’s has long been one of the most feared diagnoses – a genetic curse passed down with ruthless certainty. But with this landmark development, science has taken a bold leap forward. For the first time, families can dare to plan for the future – and look forward to life, not just survival.
