By Dwaipayan Roy | Sep 24, 2025, 08:00 PM
**A Major Breakthrough in Huntington’s Disease Treatment**
In a groundbreaking medical advancement, doctors have successfully treated Huntington’s disease for the first time. This hereditary condition causes severe brain cell damage and has been compared to a combination of dementia, Parkinson’s disease, and motor neuron disease.
The innovative treatment involves gene therapy administered during delicate brain surgery lasting between 12 to 18 hours.
**Impact: Decades of Good Quality Life Expected**
Patients undergoing this new treatment can expect decades of “good quality life.” According to Professor Sarah Tabrizi from University College London (UCL), the therapy has shown promising results by slowing the disease’s progression by 75%. This means that a decline that would normally occur in one year could take four years post-treatment.
Typically, the first symptoms of Huntington’s appear in a person’s 30s or 40s, and the disease can be fatal within two decades.
**How the Treatment Works**
The treatment involves injecting a modified virus deep into the brain using advanced genetic medicine that combines gene therapy and gene silencing technologies. The virus carries a specially designed sequence of DNA, which is delivered inside brain cells to permanently reduce levels of a toxic protein responsible for the disease.
This procedure requires between 12 to 18 hours of neurosurgery, performed under real-time MRI scanning guidance to ensure precision and safety.
**Clinical Trial Outcomes: Promising Results**
The clinical trial included 29 patients and demonstrated an average 75% slowing of the disease three years after surgery. Additionally, data indicated that the treatment preserves brain cells, with lower levels of neurofilaments in spinal fluid compared to baseline.
Professor Ed Wild from UCL described this development as “the result we’ve been waiting for,” expressing deep emotions about its potential impact on affected families.
**Safety and Side Effects**
While the treatment was deemed safe overall, some patients experienced inflammation caused by the virus, leading to headaches and confusion. These symptoms either resolved on their own or were managed with steroid treatment.
Professor Wild expects the effects of the therapy to last a lifetime since brain cells are not replaced by the body in the same way as blood, bone, or skin cells.
**Accessibility and Future Prospects**
Huntington’s disease affects approximately 75,000 people across the UK, US, and Europe, with hundreds of thousands more carrying the gene mutation.
Due to the complex surgery and cost involved, this gene therapy will not be accessible to all patients. However, Professor Tabrizi believes this marks “the beginning” and will pave the way for therapies that can reach a broader population.
She is already working with young individuals who carry the Huntington’s gene but have not yet shown symptoms—referred to as stage zero Huntington’s—and plans to initiate prevention trials in the near future.
—
*This landmark treatment provides new hope for patients and families affected by Huntington’s disease and signals a significant step forward in the fight against neurodegenerative disorders.*
https://www.newsbytesapp.com/news/science/groundbreaking-treatment-for-huntington-s-disease-achieved/story
