September 18, 2019
The Division of Movement Disorders at the Departments of Neurology and Neurosurgery at Hackensack Meridian Health University Medical Center have been selected as one of multiple medical centers in the United States to participate in the RESTORE-1 Phase 2 Clinical Study of VY-AADC, an investigational gene therapy for Parkinson’s disease. The randomized, double-blinded, placebo-surgery controlled trial, RESTORE-1, will evaluate the safety and efficacy of the surgically delivered VY-AADC for people with advancing Parkinson’s disease.
The hospital is recruiting participants ages 40-75, male and female, who have been diagnosed with Parkinson’s disease for four years or more and who have been experiencing unpredictable motor functions despite oral medications. The study will involve two months of pre-treatment assessment and 12 months of follow-up visits.
Each eligible study participant will undergo a surgical procedure while asleep. Prior to the procedure, study participants will be randomly assigned to one of two treatment groups: either the experimental gene therapy group or the placebo group. The study is double-blinded, so neither the patient nor their neurologist will know which group they are in.
Parkinson’s disease is characterized by a loss of dopamine and its function. Dopamine is a chemical “messenger” that is produced in the brain and is involved in the control of movement. Dopamine is made in the brain when the enzyme AADC (Aromatic l-amino acid decarboxylase) converts the chemical levodopa to dopamine. Levodopa, AADC, and dopamine are each present at normal levels in healthy people. As Parkinson’s disease worsens, there is less AADC enzyme in parts of the brain where it is needed to convert levodopa to dopamine. When this happens, patients’ motor function may worsen with a less predictable response to medications.
VY-AADC is an investigational gene therapy designed to put the AADC enzyme into brain cells where it can convert levodopa to dopamine. To do this, the AADC gene is delivered inside a transporter called “adeno-associated viral vector” (AAV). The investigational one-time therapy is delivered directly to the putamen, a part of the brain that helps control motor function, during a neurosurgical procedure monitored with real-time MRI imaging.