This clinical study, co-conducted since 2008 by Professor Stéphane Palfi, head of the neurosurgery department at the Henri-Mondor hospital in Créteil and the Addenbrookes hospital in Cambridge (United Kingdom) has demonstrated the effectiveness of the Prosavin gene therapy on movement control in patients with Parkinson’s disease. It therefore gives hope to the 5 million people in the world and 120,000 people in France affected by this neurodegenerative disease.
“We have undertaken clinical trials with 12 months follow-up at two study sites in France and the UK. We evaluated the safety and efficacy of ProSavin gene therapy after injecting a horse virus into the brains of 15 patients with Parkinson’s disease. This virus harmless to humans belonging to the lentivirus family. It has been emptied of its content and ‘filled’ with the three genes (AADC, TH, CH1) essential for the manufacture of dopamine,” explains Professor Palfi. This essential neurotransmitter for movement control is no longer produced in patients suffering from this neurodegenerative disease. Scientists wanted through this therapy to restore a continuous level of dopamine in patients.
Encouraging results
Thanks to this injection, the 15 patients again secreted dopamine continuously and were able to improve the control of their movements.
“We tested three dose levels, the strongest proved to be the most effective”, explains Professor Palfi. But he recalls all the same that beyond 4 years, the motor progress decreased due to the evolution of the disease: “the motor symptoms of the disease were improved up to 12 months after the administration of the treatment in all patients, even up to 4 years in the first to have been operated on”. And, unfortunately, if this treatment acts on the motor disorders, it has no effect on the hallucinations and character changes and on the evolution of the disease.
These encouraging results need to be confirmed by further trials and studies. The team of researchers hope “to be able to treat patients whose disease is not too advanced and to extend the benefits of drug treatment beyond 10 years to 3 to 5 years today. »
