Parkinson's Gene Therapy Improves Motor Function
Phase 1/2 trial shows that the treatment is safe
FRIDAY, Jan. 10, 2014 (HealthDay News) -- A triple gene therapy for Parkinson's disease is safe and improves motor function, according to a study published online Jan. 10 in The Lancet.
Stéphane Palfi, M.D., from Groupe Henri-Mondor Albert-Chenevier in Créteil, France, and colleagues assessed the safety and efficacy of various doses of ProSavin after bilateral injection into the putamen of 15 patients with Parkinson's disease. ProSavin is a lentiviral vector-based gene therapy encoding three enzymes needed for dopamine production.
During 12 months of follow-up, the researchers recorded 54 drug-related adverse events, all of which were mild or moderate, most commonly increased on-medication dyskinesias and on-off phenomena. There was significant improvement in motor responses in all patients after both six and 12 months, as assessed by the Unified Parkinson's Disease Rating Scale part III (off medication) scores.
"In summary, the data from these early phase clinical trials provide preliminary evidence for the safety and potential clinical benefit of ProSavin as a long-term treatment for Parkinson's disease," Palfi and colleagues conclude.
The study was funded by Oxford BioMedica, the manufacturer of ProSavin; several authors are current or former employees of or consultants to Oxford BioMedica.
Abstract (http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(13)61939-X/abstract )Full Text (subscription or payment may be required) (http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(13)61939-X/fulltext )Editorial (subscription or payment may be required) (http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(13)62108-X/fulltext )