Three-Year Outcomes of Gene Therapy for Parkinson Disease

Gene therapy with an enzyme that converts levodopa to dopamine showed promise in a phase 1b study.

Aromatic L-amino acid decarboxylase (AADC) is an enzyme that converts levodopa to dopamine. As Parkinson disease (PD) progresses, loss of nigrostriatal cells that produce AADC leads to need for increasing levodopa doses and additional treatments for motor fluctuations and dyskinesias. Researchers now report 36-month safety and clinical outcomes from a phase 1b, open-label, dose-escalation trial of NBIb-1817 (VY-AADC01), an investigational (not FDA-approved) gene therapy for PD. The therapy was delivered bilaterally by intraoperative MRI-guided putaminal infusions to three groups of 5 patients each, receiving different doses: ≤7.5x10¹¹ vector genomes (vg); ≤1.5x10¹² vg; or ≤4.7x10¹² vg.

No serious, treatment-related adverse events were report…

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)