A UK team has developed a genetic therapy that has slowed the disease progression by 75% in monitored patients for three years. The results remain preliminary, but offer a tangible hope.
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The team of researchers has announced promising results in a clinical study on Huntington’s disease, a hereditary neurodegenerative condition so far not treated. Experimental therapy could open the way to the first effective treatments for affected patients.
Innovation of the research team
The study was conducted by a group of neurologists, neurosurgeons and geneticists in the United Kingdom, who developed a method of intracerebral genetic therapy. Using a modified viral vector, the researchers delivered a fragment of therapeutic DNA directly in the key regions of the brain-the caudate nucleus and Putamen. The surgical procedure, assisted by real -time MRI imaging, lasts 12 to 18 hours and represents a technological premiere.
Of the 46 initially enrolled patients, 12 were completely monitored for 36 months. Compared to an external control group, the progression of the disease was slowed by about 75%. The team of researchers emphasizes that patients had a significantly slower evolution of motor and cognitive symptoms.
The following steps
Deserts many specialists from the scientific and health community appreciate the effort, they point out that the results come from a small, open and without domestic group. Before the therapy becomes widely available, phase III studies are required, with a much larger number of patients and rigorous methodology.
