Prime Highlights:
- Scientists have successfully slowed Huntington’s disease for the first time using a new gene therapy, AMT-130.
- Patients in early trials showed significantly slower disease progression, giving hope for a better quality of life.
Key Facts:
- AMT-130 is delivered through brain surgery, and a single dose is expected to last a patient’s lifetime.
- Early-stage trials with 29 patients showed that those receiving a high dose experienced 75% less disease progression over 36 months.
Key Background:
Scientists have made a breakthrough with the first treatment that can slow Huntington’s disease, a condition with no current cure. Experts from University College London (UCL) say this could be life-changing for patients, as the disease affects movement, thinking, and mood.
The new therapy, called AMT-130, is a form of gene therapy delivered through brain surgery. Early-stage clinical trials involving 29 patients revealed that those who received a high dose of AMT-130 experienced 75% less disease progression over 36 months, according to uniQure, a gene therapy company operating in the Netherlands and the US. Remarkably, a single dose of this therapy is expected to provide long-term benefits, potentially lasting a patient’s lifetime.
Professor Ed Wild, the principal investigator at UCL’s Huntington’s Disease Centre, described the results as “truly world-changing.” He emphasized that patients in the trial showed unprecedented stability over time, with some even returning to work. “Behind each data point is an incredible patient who underwent major neurosurgery to receive the first gene therapy tested for Huntington’s disease. Their bravery is extraordinary,” he said.
Professor Sarah Tabrizi, lead scientific adviser on the trial, highlighted the significance of the results. “This study of AMT-130 demonstrated statistically significant effects on disease progression at 36 months. These data are the most convincing evidence to date of a disease-modifying effect in Huntington’s disease,” she said. She added that the treatment could help patients maintain daily functions, remain in work longer, and meaningfully slow the progression of the disease.
AMT-130 is a giant move in the research of Huntington’s disease. It can delay the disease, and not only manage symptoms. Researchers are trying to make it accessible to everyone with a disease and continue to find more treatment options.
This gives patients and their families hope for a future with slower disease progression and a better life.
