Huntington's disease that was successfully treated for the first time in the British Gene Therapy trial reports researchers breakthrough

Huntington’s disease, one of the most devastating inherited brain disorders, was successfully treated for the first time. A gene therapy trial that led from the UK showed dramatic results, delaying the progress of the disease with three-quarters after three years. The disease, transmitted by families, is caused by a single faulty gene. Once active, it gradually destroys brain cells, leading to mood changes, uncontrolled movements, dementia and, over time, death. Until now, there was no treatment that could delay it. “It’s absolutely big. I am very happy, ‘says Prof. Sarah Tabrizi, who directed the trial at University College London’s Huntington’s Disease Center. “We now have a treatment for one of the world’s more terrible illnesses,” she told The Guardian. How the therapy works, the treatment, known as AMT-130, is delivered by a long surgical procedure that lasts 12 to 20 hours. Doctors use a microcateter to slowly place a modified virus in two parts of the brain. Once inside, the virus carries a string of DNA that turns off the production of the toxic protein “hunt”, the cause of the disease. Patients only need one shot. But the complexity of the procedure means it will be expensive if it is rolled out widely, BBC reports. Results of the hearing The study, conducted by the Dutch Biotech Company Uniqure, followed 29 patients in the UK and the US. According to their study, three years after treatment, patients who received the high dose of therapy had a slowdown of 75% of the progression of disease compared to the control group. Motor skills, memory and daily function have all dropped less than expected. Markers in the brain also told the same story. The levels of neurophilaments, a sign of cell death, were significantly lower in those who gave the drug. “These findings reinforce our belief that AMT-1330 has the potential to fundamentally transform the treatment landscape for Huntington’s disease,” Uniqure’s medical officer Dr Walid Abi-Saab said in the press release issued by the company. What this means for families from about 6,000 to 10,000 people, living with Huntington’s disease in the UK, while at least 20,000 more carry the erroneous gene. Only a fraction is testing for this, because until now there has been no hope of delaying the disease. “Now I think many more people will come forward for the genetic test because there is a treatment,” Tabrizi told The Guardian. Uniqure said it intended to submit the results to US regulators in early 2026. Researchers and patient groups now describe the findings as the most convincing progress against an illness that has long been untreatable. Questions What is Huntington’s disease? It is a rare genetic brain disorder that causes movement, memory and mood problems, which eventually lead to dementia and death. What did the British trial display? The hearing found that a one -time gene therapy delayed the progression of disease by about 75% after three years. Who led the study? The trial was led by prof. Sarah Tabrizi, director of University College London Huntington’s Disease Center. What is the new treatment called? The therapy is known as AMT-130 and was developed by the Biotech Company Uniqure.