![\"\"](\"https:\/\/www.newsbeep.com\/ca\/wp-content\/uploads\/2025\/10\/Co-founded-of-the-UCL-Huntingtons-Disease-Centre-Professors-Tabrizi-and-Wild-released.jpg\")
Co-founders of the UCL Huntington\u2019s Disease Centre \u2013 Professors Tabrizi and Wild<\/p>\n Co-founders of the UCL Huntington\u2019s Disease Centre \u2013 Professors Tabrizi and Wild<\/p>\n
There is no cure for Huntington\u2019s disease, a devastating brain disorder that causes severe motor loss, dementia, and eventual death.<\/p>\n
But, a clinical trial delivered positive results this week, finding that patients receiving a new treatment for 36 months experienced 75% less progression of the disease overall (compared to patients who did not receive the treatment).<\/p>\n
This is the first time a drug trial achieved a significant slowing of Huntington\u2019s progression, according to uniQurea, a gene therapy company based in the Netherlands and US.<\/p>\n
The new gene therapy, AMT-130, was performed on 29 patients including 12 who were given a high dose. Those given a high dosage of AMT-130 experienced 75% less disease progression, as measured by a standard rating scale that incorporates motor, cognitive, and functional measures.<\/p>\n
There was also a statistically significant benefit as measured by other scales of disease progression, including motor and cognitive function.<\/p>\n
MORE GENE BREAKTHROUGHS: Type 1 Diabetic Produces His Own Insulin After Gene-Edited Cell Transplant<\/a><\/p>\n The researchers also measured participants\u2019 levels of neurofilament light protein (NfL), a protein that is released into the spinal fluid when neurons are injured\u2014a useful marker for neuronal damage. The scientists found that NfL levels in the spinal fluid were lower in people treated with the drug than they had been at the start of the trial, even though NfL levels would be expected to increase by 20-30% over three years. They say this suggests the course of the disease has been slowed.<\/p>\n The trial, conducted by scientists at University College London (UCL) with funding from uniQure, also found that \u201cAMT-130 is generally well-tolerated by study participants and has a manageable safety profile\u201d.<\/p>\n \u201cl am thrilled,\u201d said Professor Sarah Tabrizi of UCL\u2019s Huntington\u2019s Disease Research Centre and lead scientific advisor on the trial. \u201cFor patients, AMT-130 has the potential to preserve daily function, keep them in work longer, and meaningfully slow disease progression.\u201d<\/p>\n It is expected that a single dose of AMT-130 would last for a person\u2019s whole life.<\/p>\n THE MIRACLE OF SIGHT: 100 Times Improvement in Sight Seen After Gene Therapy Trial for Disease That Deteriorates Vision in Childhood<\/a><\/p>\n UniQure plans to submit an application to the US Food and Drug Administration early next year requesting accelerated approval to market the drug, with applications in the UK and Europe to follow.<\/p>\n \u201cThis result changes everything,\u201d said Professor Ed Wild, principal investigator of the UCL Huntington\u2019s Disease Centre trial site at UCL.<\/p>\n \u201cMy patients in the trial are stable over time in a way I\u2019m not used to seeing in Huntington\u2019s disease \u2013 and one of them is my only medically-retired Huntington\u2019s disease patient who has been able to go back to work.\u201d<\/p>\n