A New Gene Therapy for Parkinson Disease

ProSavin showed promise in a phase 1/2 trial, but safety and efficacy questions remain open.

Parkinson disease (PD) is a progressive neurodegenerative syndrome that affects motor and nonmotor abilities. This manufacturer-funded study addressed the safety, tolerability, and efficacy of bilateral intrastriatal delivery of ProSavin, a lentiviral vector–based gene therapy intended to enhance continuous dopamine production in the brain. The study was a phase 1/2 open-label trial with an open-label follow-up for safety. Fifteen patients with mild-to-moderate PD and a well-documented response to dopamine therapy received low-dose (1·9×10⁷ transducing units [TU]; 3 patients), mid-dose (4·0×10⁷ TU; 6 patients), or high dose (1×10⁸ TU; 6 patients) ProSavin.

The patients were followed for 12 months, during which 54 adverse events potentially r…

Reviewing Author

Michael S. Okun, MD

Disclosures

Consultant/Advisory BoardParkinson’s Foundation; Tourette Association of America

Speaker’s BureauMedscape/WebMD CME, Movement Disorders Society CME, American Academy of Neurology CME

RoyaltiesBooks4Patients; Demos; Cambridge; Taylor and Francis; Robert Rose; Public Affairs

Grant/Research SupportNational Institutes of Health; Parkinson’s Foundation; Tourette Association of America; University of Florida Foundation

Editorial BoardsTremor and Other Hyperkinetic Movements; JAMA Neurology

Leadership Positions in Professional SocietiesParkinson’s Foundation (Medical Advisor)