AADC Gene Therapy for Parkinson’s Disease

UPMC and the University of Pittsburgh are conducting a new gene therapy study for individuals who have been diagnosed with Parkinson's disease for at least five years and have had varying responses to their current medication.

The purpose of this gene therapy study is to test the safety and tolerability of the transfer of a gene, AADC (aromatic amino acid decarboxylase) into the brains of people with moderately advanced Parkinson’s disease. In this study, the AADC gene will be delivered to a specific area of the brain and it may increase the ability of certain brain cells to make dopamine. The study will also assess the effect of the treatment on clinical tests, such as mobility.

AADC is an enzyme in the brain that converts levodopa into dopamine which the brain can use to improve Parkinson’s symptoms. This study will test whether or not this treatment will tell the brain cells to make more AADC, which may increase dopamine levels in your brain when you take levodopa. This may provide improvement in the movement areas in the brain and may help symptoms of PD.

Individuals who have been diagnosed and received treatment for Parkinson’s disease for at least five years and are between the ages of 40 and 70 may be eligible to participate.

Participants for this study will participate in 16 visits over a three-year period with the bulk of the study visits occurring in the first year after the surgery. Study participants will receive the study drug through surgical delivery into the brain and then undergo Parkinson’s disease specific tests and assessments.

Participants will receive study procedures at no charge. The study will pay for meals and all parking fees for study visits.

Individuals interested in participating in this study, or learning more information, should contact the study coordinator, Patricia Porter, at 412-648-8983.

[Watch KDKA-TV report on gene therapy study below featuring R. Mark Richardson, MD, PhD, lead local researcher.]