Gene therapy slows Huntington’s disease progression by up to 75% in trial
Early results from a small study suggest therapy AMT-130 may substantially slow disease progression; full findings not yet published
A gene-therapy trial for Huntington’s disease slowed disease progression by as much as 75% over a three-year follow-up in a group of 29 patients, researchers said. The participants underwent a 12- to 18-hour neurosurgical procedure to receive the treatment.
The therapy, AMT-130, is developed by uniQure and uses a harmless viral vector to deliver DNA to brain cells. It prompts production of microRNA that lowers levels of mutant huntingtin protein. The infusion is delivered directly to the brain under MRI guidance and takes about 12 to 18 hours.
Over three years, the trial showed an average 75% slowing of clinical progression compared with the expected course without treatment. Biomarkers also suggested slowed neurodegeneration; neurofilament levels in spinal fluid, which rise as brain cells die, were lower at three years than at baseline.
Experts described the results as spectacular. Dr. Sarah Tabrizi, director of the University College London Huntington's Disease Centre, said, "We never in our wildest dreams would have expected a 75 per cent slowing of clinical progression." Prof. Ed Wild, a consultant neurologist at University College Hospital London, added, "This is the result we've been waiting for."
Huntington’s disease is a genetic disorder affecting the central nervous system and causing involuntary movements, speech difficulties, and memory loss. On average, patients live 10 to 20 years after diagnosis. The NHS says there is currently no way to stop the disease from getting worse.
The condition affects about 8,500 adults in the United Kingdom and around 30,000 in the United States.
AMT-130 combines gene therapy and gene-silencing technology. It uses an engineered viral vector to deliver DNA that prompts brain cells to produce microRNA, which reduces levels of mutant huntingtin.
Results were released by uniQure in a statement and have not yet been published in full in a peer-reviewed journal.
Experts caution that the results come from a small, early-stage trial and must be confirmed in larger studies. If confirmed, the therapy could represent a major advance, though challenges remain in safety, manufacturing, and long-term durability.