Breakthrough Gene Therapy Offers Hope in Huntington’s Disease Treatment
For the first time, doctors say they have successfully slowed the devastating progression of Huntington’s disease, one of the cruellest hereditary brain conditions.
The breakthrough therapy has been shown to slow the disease by 75%, offering patients the possibility of decades of improved quality of life.
What is Huntington’s Disease?
Huntington’s is an inherited disorder that destroys brain cells, causing symptoms that resemble dementia, Parkinson’s, and motor neurone disease. Symptoms often appear in a person’s 30s or 40s, with the disease usually fatal within 20 years.
If one parent carries the faulty huntingtin gene, there is a 50% chance their children will inherit the mutation. This defective gene produces a toxic protein that gradually kills neurons in the brain.
The Breakthrough Treatment
The new therapy, developed by uniQure and trialled at University College London, is a form of gene therapy delivered directly into the brain. The procedure takes 12–18 hours and involves using real-time MRI scans to infuse a specially engineered virus into targeted brain regions.
Once inside, the therapy effectively “reprograms” brain cells to disable the mutant huntingtin protein, reducing its harmful effects and protecting neurons.
According to trial data, the treatment:
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Slowed disease progression by an average of 75% over three years.
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Reduced markers of brain cell death.
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Allowed patients to maintain mobility and independence longer than expected.
One patient who had medically retired even returned to work, while others remained mobile when they were expected to need wheelchairs.
Emotional Response From Researchers
Professor Sarah Tabrizi, Director of the UCL Huntington’s Disease Centre, described the results as “spectacular”.
“We never in our wildest dreams expected a 75% slowing of clinical progression,” she told BBC News.
Her colleague, Professor Ed Wild, a neurologist at UCLH, admitted he was moved to tears:
“The magnitude of this effect is breathtaking. To see hope for families living with this cruel disease is overwhelming.”
Families Living With Huntington’s
For families like that of Jack May-Davis, the news is life-changing. Jack, 30, carries the gene just like his father and grandmother before him. He watched his father’s decline until his death in 2016 at age 54.
Now, Jack says the breakthrough leaves him “overwhelmed”:
“It does allow me to think my life could be that much longer.”
Challenges Ahead
While the results bring huge hope, the treatment is highly complex and costly. Gene therapies are among the most expensive medical treatments in the world.
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In the UK, the NHS already funds a £2.6m-per-patient gene therapy for haemophilia B.
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UniQure plans to apply for a US licence in 2026, with European approvals to follow.
Side effects, including temporary brain inflammation in some patients, were reported but treated successfully. Experts believe the therapy could last a lifetime because brain cells are not replaced like other cells in the body.
A Turning Point in Genetic Medicine
This trial is considered just the beginning. Researchers are already preparing prevention studies on people who carry the gene but have not yet shown symptoms, aiming to delay or even stop Huntington’s before it starts.
Professor Tabrizi paid tribute to the volunteers who participated:
“They are truly brave. I am overjoyed for the patients and families. This is the beginning of a new era.”