Gene therapy trial seeks to stimulate brain’s production of dopamine enzyme in Parkinson’s patients

Hackensack University Medical Center is one of just a few U.S. medical centers participating in the trial.

The Division of Movement Disorders at the Departments of Neurology and Neurosurgery at Hackensack Meridian Health University Medical Center (HUMC) is one of a few US medical centers participating in the RESTORE-1 Phase 2 Clinical Study of VY-AADC. This investigational gene therapy trial is for Parkinson’s patients whose response to medications has been unpredictable or increasingly less beneficial.

The ongoing trial surgically places a one-time AADC gene into a specific part of the brain where it can convert levodopa into dopamine. The goal of the procedure is to program the brain to produce more dopamine via the AADC enzyme.

Dr. Hooman Azmi performed the first such surgery on October 7, 2019. Dr. Azmi co-leads the clinical trial with Dr. Naomi Lubarr.