In a groundbreaking development, researchers in the UK have reported the first-ever successful treatment for Huntington’s disease, a hereditary neurodegenerative disorder previously deemed incurable. The experimental gene therapy, known as AMT-130, slowed disease progression by 75% over three years, offering renewed hope to patients and families affected by the condition.
What Is Huntington’s Disease?
Huntington’s disease is a progressive neurological disorder caused by a mutation in the HTT gene, leading to the production of a toxic protein that damages brain cells. Symptoms typically manifest in mid-adulthood and include motor dysfunction, cognitive decline, and psychiatric disturbances. The disease is fatal, with patients often succumbing within 15–20 years of symptom onset.
The Breakthrough Treatment
Led by Professor Sarah Tabrizi at University College London (UCL), the pioneering clinical trial utilised an innovative gene therapy approach. The treatment involves a one-time surgical procedure lasting up to 20 hours, during which a specially engineered, harmless virus is infused directly into the brain. This virus carries a DNA strand designed to deactivate the mutant huntingtin protein responsible for the disease.
The results have been nothing short of remarkable. According to a press release from UCL, patients who underwent the treatment experienced a 75% reduction in the expected progression of the disease over a three-year period. Instead of the expected rapid decline, these patients maintained neurological function, effectively gaining years of quality life.
Expert Insights
Professor Tabrizi expressed her astonishment at the results, stating, ‘We never in our wildest dreams would have expected a 75% slowing of clinical progression.’ She emphasised that this breakthrough could pave the way for earlier interventions, potentially preventing the onset of symptoms altogether.
Where to Access Huntington’s Disease Treatment in the UK
For individuals…
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