UCL
The study was led by Ed Wild and Sarah Tabrizi, researchers at Huntington’s Disease Center at University College London Hospital
There is still no cure for this fatal condition, but a team of researchers has been able to delay the progression of Huntington’s disease through direct brain injection.
A complex genetic procedure offers new hope in the fight against one of the Most Cruel Diseases in the World.
A team of UK scientists announced Wednesday that it was able, for the first time, to slow the progression of Huntington’s diseasea fatal neural condition, thanks to an innovative genetic therapy.
Some of the patients who participated in early clinical trials at University College London (UCL) observed 75% reduction at the rate of disease progression after three years.
The study tested a new genetic therapy, AMT-130administered through an injection directly into the brain. This therapy works by introducing permanently new functional DNA in the patient’s cellsexplain the investigators.
“This result changes everything,” he said Ed Wildmain researcher at Huntington’s Disease Center at University College London Hospital, in.
“Based on these results, it seems likely that the AMT-130 will be the first authorized treatment Able to slow Huntington’s disease, which is truly revolutionary, ”he added.
29 patients participated in the test, and the 12 receiving the highest dose recorded the highest softening of the disease.
Currently there is no cure for Huntington’s disease, also known as Huntington or Huntington’s evila fatal neurodegenerative disease caused by a single genetic mutation.
Huntington’s disease is a “genetic thief”explains: usually comes at the height of life, and Combines the horrors of dementia, Parkinson and motor neuron disease In a single brutal package.
The disease relentlessly kills brain cells, leading to a catastrophic decline in the ability to think, move and function. For carriers of the defective gene, the future has always been a terrifying certainty. If one of the parents has Huntington, Your child has 50% probabilities to inherit the same fate.
Nearly 8,000 people in the UK They live with this condition, which affects movement, reasoning and humor. The prevalence of the disease is estimated at about 14 in 100,000 people in populations of European origin, which suggests that Portugal will have at least 1400 cases.
Wild explains that the patients who participated in the rehearsal are now stable “in a way that I was not used to seeing in Huntington’s disease. One of them is my only patient with Huntington’s disease who was medically reformed and who was able to return to work. ”
Mike Hannadirector of UCL Queen Square Institute of Neurology, greeted the results as “a new chapter In the development of genetic therapy for Huntington’s disease, with clear relevance ”for other neurodegenerative diseases.
