Parkinson's gene therapy improves motor function

Parkinson's gene therapy improves motor function
Parkinson's gene therapy improves motor function

HealthDay News -- A novel triple gene therapy for Parkinson's disease is safe and improves motor function, results of a phase I clinical trial suggest.

The therapy, ProSavin, is a lentiviral vector-based gene therapy that encodes three enzymes that the brain needs for dopamine production.

"ProSavin was safe and well tolerated in patients with advanced Parkinson's disease," Stéphane Palfi, MD, from Groupe Henri-Mondor Albert-Chenevier in Créteil, France, and colleagues reported in The Lancet.

They assessed the safety and efficacy of various doses of ProSavin after bilateral injection into the putamen of 15 patients with Parkinson's disease.

There was significant improvement in motor responses in all patients after both six (mean score 38 vs. 26, P=0.0001) and 12 months (38 vs. 27, P=0.0001), as assessed by the Unified Parkinson's Disease Rating Scale part III (off medication) scores.

During 12 months of follow-up there were 54 drug-related adverse events, all of which were mild or moderate, most commonly increased on-medication dyskinesias and on-off phenomena.

"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 concluded.

The study was funded by Oxford BioMedica, the manufacturer of ProSavin; several authors are current or former employees of or consultants to Oxford BioMedica.


References

  1. Palfi S et al. Lancet. 2014; doi:10.1016/S0140-6736(13)61939-X.
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