Improved Delivery Methods for Gene Therapy and Cell Transplantation in Parkinson's Disease.

Authors: Larson PS

A number of cell transplantation and gene therapy trials have been performed over the last three decades in an effort to restore function in Parkinson's disease. Much has been learned about optimizing delivery methods for these therapeutics. This is particularly true in gene therapy, which has predominated the clinical trial landscape in recent years; however, cell transplantation for Parkinson's disease is currently undergoing a renaissance. Innovations such as cannula design, iMRI-guided surgery and an evolution in delivery strategy has radically changed the way investigators approach clinical trial design. Future therapeutic strategies may employ newer delivery methods such as chronically implanted infusion devices and focal opening of the blood brain barrier with focused ultrasound.

Introduction

Purpose other (review)

Outcomes and Safety

Summary of Outcomes This review summarizes the evolution of delivery methods for gene therapy and cell transplantation in Parkinson's disease, including stereotactic frame-based blind infusions, convection-enhanced delivery (CED), and real-time iMRI-guided infusions with gadoteridol co-infusion. Key advances include stepped cannula designs to prevent reflux, progressive infusion strategies to optimize target coverage, and use of Vd/Vi ratio (volume of distribution/volume of infusion, typically 2-3:1) to predict infusion volume. Focused ultrasound for blood-brain barrier opening is mentioned as a potential future noninvasive delivery strategy.

Brain Region

Targeted brain region(s) Not specified

Ultrasound Parameters

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