International
Breakthrough Gene Therapy Offers Hope in Huntington’s Disease Treatment
A groundbreaking gene therapy trial has achieved a 75% reduction in the progression of Huntington’s disease over three years, offering new hope for treatment. With an estimated 6,000 to 10,000 people affected in the UK, this breakthrough could transform the lives of many and encourage more individuals to seek genetic testing.
Huntington’s disease, a hereditary neurodegenerative disorder, has been successfully treated for the first time in a groundbreaking gene therapy trial. The disease, stemming from a single gene defect, leads to dementia, paralysis, and eventually death, with individuals having a 50% chance of inheriting it from an affected parent. In a trial involving 29 patients across the UK and US, the gene therapy demonstrated a remarkable 75% reduction in disease progression over three years, as measured by motor function, cognition, and daily life experiences. The therapy targets the toxic huntingtin protein produced by the mutant gene, using a modified virus to deliver a specially designed strand of DNA to neurons. The infusion process, which takes 12 to 20 hours and is performed via a micro-catheter into two brain regions, aims to instruct cells to block the production of this harmful protein.
An estimated 6,000 to 10,000 people in the UK are affected by Huntington’s disease, with another 20,000 likely to develop it due to being carriers of the faulty gene. As a result of this breakthrough, there is an expectation that more individuals will seek genetic testing, as previously the only available treatments addressed symptoms rather than slowing progression. The trial results indicate significant clinical benefits and a potential pathway for early intervention. With the company uniQure planning to seek drug approval in the US, this therapy holds the promise of improving quality of life and longevity for those diagnosed with Huntington’s disease.
SOURCE: THE GUARDIAN
