Scientists have slowed the progression of Huntington’s disease for the first time with a “groundbreaking” new treatment.
Experts from University College London (UCL) said the finding could “change everything” for patients with the condition, which gets worse over time and has no cure.
The disease affects movement, thinking and mood.
The study tested a new gene therapy, AMT-130, which is delivered by brain surgery.
The early stage clinical trials among 29 patients concluded that those who were given a high dose of the treatment experienced 75 per cent less disease progression after 36 months, according to uniQure, a gene therapy company based in the Netherlands and the US.
A single dose is is expected to last for a person’s life.
“This result changes everything,” said principal investigator Professor Ed Wild, from the UCL Huntington’s Disease Centre.
“On the basis of these results it seems likely AMT-130 will be the first licensed treatment to slow Huntington’s disease, which is truly world-changing stuff.
“If that happens, we need to work hard to make it available to everyone who needs it, while working no less diligently to add more effective treatments to the list.
“Trial results come through in numbers and graphs, but behind each datapoint is an incredible patient who volunteered to undergo major neurosurgery to be treated with the first gene therapy we’ve ever tested in Huntington’s disease. That is an extraordinary act of bravery for the benefit of humanity.
“My patients in the trial are stable over time in a way I’m not used to seeing in Huntington’s disease – and one of them is my only medically retired Huntington’s disease patient who has been able to go back to work.”The lead scientific adviser on the trial, Professor Sarah Tabrizi from UCL Huntington’s Disease Centre, said: “I am thrilled that this study of AMT-130 showed statistically significant effects on disease progression at 36 months.
“These groundbreaking data are the most convincing evidence in the field to date and underscore the disease-modifying effect in Huntington’s disease, where an urgent need persists.
“For patients, AMT-130 has the potential to preserve daily function, keep them in work longer, and meaningfully slow disease progression.”
