For the first time, Huntington’s disease has been successfully treated with a new gene therapy, AMT-130, which slows the disease by 75 percent, targeting the root cause and giving patients better quality of life.
For the first time, scientists have found a way to dramatically slow Huntington’s disease, an inherited brain disorder that has long been considered untreatable. A groundbreaking gene therapy called AMT-130 has shown promising results in clinical trials, slowing disease progression by up to 75 percent. Huntington’s disease slowly damages nerve cells in the brain, leading to problems with movement, memory, and independence. Until now, patients could only rely on treatments that managed symptoms, not the disease itself. But new research is showing that things may be changing. With gene therapy, patients could experience a much slower decline and enjoy more years of good quality life, giving families a reason to feel hopeful about the future.
What have researchers found?
Huntington’s disease (HD) is often described as a combination of dementia, Parkinson’s, and motor neurone disease, as it affects memory, movement, and mental health. It is genetic, meaning it runs through families, and those with one affected parent have a 50 percent chance of inheriting it. The UK team leading part of the trial reported that gene therapy slowed patients’ decline by an astonishing 75 percent. To put that into perspective, a year’s worth of expected deterioration could now stretch over four years. For families used to watching loved ones fade quickly, this could mean decades of better living. Professor Sarah Tabrizi, director of the University College London Huntington’s Disease Centre, called the results “spectacular” and beyond what researchers imagined.
Why has Huntington’s disease been hard to treat?
Huntington’s is caused by a defective gene that produces a toxic protein called huntingtin, which slowly kills brain cells. Symptoms usually appear in mid-adulthood and worsen over 10 to 20 years, leaving patients needing full-time care. Until now, treatments could only ease symptoms such as involuntary movements or mood changes, but nothing slowed the disease itself. That’s why this breakthrough feels so significant, it directly targets the root cause, not just the visible effects.
How does AMT-130 gene therapy work?
The treatment of this brain disease involves a one-time brain surgery lasting 12 to 18 hours. Guided by real-time MRI, surgeons deliver a harmless virus carrying new genetic instructions into two brain regions, the caudate nucleus and putamen. These instructions tell brain cells to reduce production of the toxic huntingtin protein. By lowering this protein, the therapy slows or possibly halts brain cell damage. Unlike previous drugs that only managed symptoms, AMT-130 aims to change the disease’s trajectory itself, a true first in Huntington’s treatment.
What does this mean for patients and families?
For those affected, the potential impact is life-changing. Instead of relentless decline, a single treatment could preserve independence for years, even decades. Families who once faced the certainty of rapid deterioration now have a reason to hope for stability. While the treatment is expected to be very expensive, experts believe its long-lasting effects make it worthwhile. For the first time, Huntington’s patients and their loved ones can imagine a more manageable future.
