For the first time, gene therapy has appeared that truly slows the progression of Huntington’s disease, a severe inherited brain disorder long considered incurable.
The treatment, developed by uniQure and tested at University College London, is based on microRNAs that reduce the activity of the mutant HTT gene. In clinical trials, 29 patients received a single dose of AMT-130 through a surgical procedure using a viral carrier.
Three years of follow-up produced striking results: in patients who received the high dose, the therapy slowed disease progression by 75%, and neuronal damage decreased. Side effects were minimal and temporary. Among the positive outcomes, patients were able to remain active for longer: one participant even returned to work.
Huntington’s disease is inherited with a 50% probability and severely affects the brain. Today, about 250,000 people worldwide have a real chance for the first time. This is a breakthrough not only for the treatment of Huntington’s disease, but also for neurogenetics as a whole.
