Huntington’s disease is the most devastating genetic neurological disorder, and for the first time, it has been successfully treated, offering a new hope to families and patients. A defective huntingtin gene causes it and progressively damages brain cells, exhibiting the symptoms of dementia, motor neurone disease, and Parkinson’s disease. Symptoms typically appear in people in their 30s or 40s, with death usually occurring within 20 years. No prior treatment can slow down the progression.
A clinical trial led by Professor Sarah Tabrizi at University College London has shown that a novel type of gene therapy can reduce disease progression by approximately 75% over 3 years. This means that a decline expected in one year now takes 4 years. Researchers acknowledged that the results were remarkable, with several patients recovering independently, including one who returned to work after being medically retired.
The treatment involves a single dose of gene therapy administered in the brain during a 12- to 18-hour period of neurosurgery. A modified virus carrying a DNA sequence is infused in the caudate nucleus and putamen using real-time MRI guidance. The virus triggers the release of microRNA, which silences the mutant messenger RNA, lowering toxic huntingtin protein levels and protecting neurons. This decreases the level of harmful proteins and protects neurons. Biomarker analysis confirmed decreased neuronal death, as indicated by a decrease in neurofilament levels in spinal fluid, rather than an increase.
The trial involved 29 patients and was conducted by the biotech company UniQure. It reported manageable side effects such as headaches, inflammation, and confusion, with no severe safety concerns. Prof Ed Wild described the lifelong impact of the therapy as “breathtaking.”
For families, the emotional impact has been profound. Participant Jack May-Davis, a 30-year-old participant whose father and grandmother died from Huntington’s, said the therapy “made my future seem brighter,” highlighting the transformative potential of this breakthrough.
Looking ahead, uniQure plans to seek U.S. regulatory approval in 2026, followed by submissions in Europe and the UK. While the procedure is complex and highly expensive, it is limited by the complexity of surgery, as seen with other gene therapies funded by the NHS. In the UK, the NHS funds a £2.6 million-per-patient gene therapy for haemophilia B.
Prof Tabrizi is preparing prevention trials in young, gene-positive individuals without symptoms, aiming to delay or prevent disease onset entirely. Researchers believe the trial not only offers hope for Huntington’s but could open the door to similar approaches for other neurodegenerative conditions.
About 75,000 people are affected in the UK, the US, and Europe. This breakthrough marks the first significant medical advancement against Huntington’s disease. It is a new era of hope for patients and families alike.
Reference: James Gallagher. Huntington’s disease successfully treated for first time. BBC News. Published September 24, 2025. Accessed September 25, 2025. Huntington’s disease successfully treated for first time
