Preclinical Evaluation of Transaxial Intramutaminal Trajectory for Enhanced Distribution of Grafted Cells in Parkinson’s Disease Summary

Introduction

This comprehensive study, conducted by Marina E. Emborg and colleagues, published on July 26, 2024, investigates a novel surgical technique for administering human-induced pluripotent stem cell-derived dopaminergic neuroprogenitor cells (DANPCs) into the putamen of nonhuman primates. This approach aims to refine treatments for Parkinson's disease by enhancing the precision and reducing the invasiveness of cell transplantation procedures.

Methods and Findings

The study utilized a transaxial surgical approach to deliver cells across the putamen nucleus, a method believed to minimize surgical risks such as hemorrhage and infection by reducing the number of needle tracks needed. Intraoperative MRI was used to guide the injections, ensuring accurate placement of cells within the target area. The experiment involved nine immunosuppressed, unlesioned adult cynomolgus macaques, which received either cell infusions or a vehicle solution. The procedure's outcomes were promising, with all animals surviving to their scheduled endpoints without significant neurological deficits. Some complications, such as mild brain swelling and transient reduced vision, were reported but were effectively managed with adjustments to surgical techniques.

Conclusions and Implications for Clinical Practice

The successful application of this novel technique in a preclinical setting suggests its potential for clinical use. The study provides a foundation for further research into the clinical translation of this approach, potentially offering a more effective and safer method for cell-based therapy in Parkinson's disease patients. The findings advocate for the initiation of clinical trials to explore the practicality and efficacy of this method in human subjects.