Emerging Gene Therapy for Parkinson’s Disease Creates New Brain Circuits

 

A recent study found that gene therapy in development for treatment of patients with Parkinson's disease creates new circuits in the brain associated with improved movement. 

In the study published in Science Translational Medicine,  patients with Parkinson’s disease w were randomly assigned to receive gene therapy (AAV2-GAD) (n = 15) or sham surgery (n = 20). Patients had metabolic PET scans before surgery and then 6 and 12 months after surgery. 

Patients who received gene therapy started to form new brain connections that matured by the end of the 12-month study. Appearance of these new circuits will be used as a treatment biomarker in an upcoming phase 3 clinical trial for this new intervention. 

This research revealed that the AAV2-GAD therapy’s mechanism of action is different from other treatments. “Current Parkinson's disease therapies act on the abnormal disease network in the brain and often stop working as the body builds a tolerance,” said David Eidelberg, MD, who led the study. “What we observed with AAV2-GAD therapy is quite the opposite. We found that AAV2-GAD leads to the formation of new neural pathways in the brain, connecting the subthalamic nucleus to other motor regions, thereby improving motor symptoms for as long as 12 months."

These findings will help the scientific and clinical communities by progressing AAV2-GAD into clinical trials while providing a better understanding about Parkinson's disease treatment response and effectiveness. 

 

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