Gene Therapy Shows Promise in Slowing Huntington’s Disease Progression
Groundbreaking results from a clinical trial indicate a new gene therapy, AMT-130, significantly slows the progression of Huntington’s disease. Researchers reported that 12 patients receiving the highest dose of AMT-130 experienced 75% less disease progression over 36 months compared to individuals with Huntington’s who did not receive the treatment.
AMT-130 functions by permanently introducing new, functional DNA into a patient’s cells. Professor Wild, of the university College London (UCL) Huntington’s Disease Center, hailed the findings as possibly “world-changing,” suggesting AMT-130 coudl be the first licensed treatment to slow the disease. He noted patients in the trial demonstrated an unusual level of stability, with one individual even returning to work after medical retirement.
The therapy’s potential impact extends to maintaining independence; Professor Wild stated some trial participants who were predicted to require wheelchairs are still able to walk. Researchers hope to eventually administer AMT-130 to individuals in the earliest stages of the disease – even those with no symptoms – with the ultimate goal of preventing symptom onset altogether.
Professor Sarah Tabrizi, also from UCL, highlighted the therapy’s potential to “preserve daily function” and allow individuals to remain in the workforce longer.
Jack May-Davis, a participant from sussex who discovered he carried the Huntington’s gene at age 19 and has lost family members to the disease, expressed his excitement, stating he ”never thought something would be developed in a time frame that might actually be useful” for him.
Experts believe these findings represent a critically important advancement in gene therapy, with potential implications for other neurodegenerative disorders. Professor Mike Hanna,director of the UCL Queen Square Institute of Neurology,noted the results “point to a new chapter” in the field. Cath Staney, chief executive of the huntington’s Disease association, described the 75% slowing of disease progression as “astonishing” and a source of “much-needed optimism” for the Huntington’s community.
