In what scientists are calling a revolutionary medical breakthrough, doctors have successfully treated Huntington’s disease for the very first time. The treatment, which uses advanced gene therapy, has shown remarkable results in slowing down the progression of this cruel and devastating condition.
What Is Huntington’s Disease?
Huntington’s disease is a rare, inherited condition that:
Kills brain cells over time, leading to loss of control over movement, memory, and behaviour.
Combines symptoms similar to dementia, Parkinson’s disease, and motor neurone disease.
Usually starts showing symptoms in people in their 30s or 40s.
Is always fatal within 15–20 years of the first symptoms appearing.
The disease is caused by a faulty gene called the huntingtin gene, which creates a toxic protein that damages neurons. If one parent carries the gene, their child has a 50% chance of inheriting it.
The Breakthrough Treatment
For the first time, researchers have been able to slow Huntington’s disease significantly using a one-time gene therapy.
The treatment is given through 12–18 hours of delicate brain surgery.
Doctors deliver a modified virus carrying a special DNA sequence deep into the brain using real-time MRI guidance.
This DNA then instructs brain cells to produce a small molecule called microRNA, which disables the faulty gene instructions.
As a result, the toxic protein levels drop, protecting brain cells and slowing the disease.
Results From the Trial
The trial, involving 29 patients, produced results that exceeded expectations:
The disease’s progression was slowed by 75%.
A decline that would normally happen in one year now takes four years.
Patients regained quality of life – one medically retired patient has returned to work, while others are still walking when doctors predicted they would need wheelchairs.
Levels of neurofilaments (a marker of dying brain cells) dropped instead of rising.
Professor Sarah Tabrizi from University College London called the results “spectacular” and said the treatment could give patients decades of good-quality life.
A Human Story – Families Living With Huntington’s
For families living with Huntington’s, the breakthrough feels nothing short of miraculous.
Jack May-Davis, 30, carries the faulty gene. His father, Fred, developed symptoms in his 30s and died in 2016 at the age of 54 after years of decline.
Jack feared the same fate, but this new therapy has given him fresh hope for a brighter, longer future.
He described the treatment as “absolutely incredible” and said he feels “overwhelmed” by the possibility of living a normal life.
Why This Matters
Huntington’s disease devastates families because it often strikes people at the prime of their lives, leaving children to watch parents suffer while knowing they themselves may carry the gene.
This treatment is a major leap forward because:
It offers a single-dose therapy that may last a lifetime.
It is the first time any treatment has slowed the disease so dramatically.
It opens doors for prevention trials – treating people who carry the gene before symptoms ever appear.
Challenges Ahead – Cost and Accessibility
While the results are historic, challenges remain:
The treatment requires highly complex neurosurgery that will not be widely available at first.
Gene therapies are extremely expensive – in the UK, the NHS already funds a £2.6m-per-patient treatment for haemophilia B, showing that high prices are possible to support if long-term benefits are proven.
Experts warn it may take years before widespread access is possible.
Despite these hurdles, researchers are optimistic. Professor Ed Wild of UCLH said:
“There was every chance we would never see a result like this. To witness such a breathtaking effect is overwhelming.”
What Happens Next?
Biotech company uniQure, which developed the therapy, plans to apply for a licence in the United States in 2026.
Conversations with regulators in the UK and Europe are also set to begin.
Researchers are already planning the first prevention trial, targeting people who carry the Huntington’s gene but have not yet developed symptoms.
Key Takeaways
Huntington’s disease has been successfully treated for the first time using gene therapy.
The therapy slowed the disease’s progression by 75%, giving patients far more years of quality life.
This marks a historic step in medicine, offering hope to tens of thousands of families worldwide.
Cost and surgical complexity remain major barriers, but the future looks far brighter than ever before.
Sources
University College London – Huntington’s Disease Research Centre
Statements from researchers at UCLH and uniQure
This could become one of the biggest medical breakthroughs of the decade, transforming the way Huntington’s disease – once seen as untreatable – is managed.
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