A new gene therapy has slowed the progression of Huntingdon’s disease by as much as 75%.
Known as AMT-130, it is delivered via brain surgery, and a single dose is expected to last someone’s whole life.
“This result changes everything,” said principal investigator Professor Ed Wild.
Thirty-year-old Jack May-Davis, who took part in trials, said the results were “astonishing”, leaving him “lost for words”.
Huntington’s is a neurodegenerative disease which gets worse over time and has no cure, affecting thinking, movement and mood. About 8,000 people in the UK suffer from it.
While the disease typically lasts for about 20 years, loss of function can begin quite quickly.
In a trial, 12 patients were given the highest dose of AMT-130. Researchers reported they experienced 75% less disease progression after 36 months compared to a group of people with Huntington’s who were not given the treatment.
AMT-130 works by permanently introducing new functional DNA into a person’s cells, researchers said.
“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,” said Prof Wild, from the University College London (UCL) Huntington’s Disease Centre.
“My patients in the trial are stable over time in a way I’m not used to seeing in Huntington’s disease.”
One of them, he added, was medically retired but has been able to return to work.
‘Absolutely breathtaking’ findings
Prof Wild told Sky News presenter Jayne Secker that the 75% slowing was “absolutely breathtaking… that could be years or decades of top quality life or even complete disease freedom if we treat early enough”.
He added: “There are undoubtedly patients in this trial who would have been in wheelchairs by now, who are still able to walk and are independent.
“And if we can bring this therapy to as many people as possible, the impact on families who are struggling with this condition for themselves and their…
