Groundbreaking Real-Time MRI Enhances Nervous System Gene Therapy

Researchers at The Ohio State University Gene Therapy Institute and The Ohio State University Wexner Medical Center have introduced a groundbreaking approach to deliver gene therapy to the nervous system. The method involves real-time magnetic resonance imaging (MRI), offering crucial insights into optimizing the effectiveness, safety, and efficiency of gene therapy in treating neurodegenerative, metabolic, and enzyme deficiency diseases.

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Intraparenchymal Delivery in Clinical Trials

In several “first-in-human” clinical trials worldwide, including initiatives led by Ohio State’s Dr. Krzysztof S. Bankiewicz, direct intraparenchymal delivery of gene therapy has been employed. This method involves delivering therapy directly into the parenchyma, a tissue type with essential functions in the nervous system.

Dr. Bankiewicz, Chief Scientific Officer of the Gene Therapy Institute, anticipates broader adoption of real-time MRI in gene therapy delivery over the next 3 to 5 years. As more sites develop the necessary infrastructure and expertise, the innovation will play a pivotal role in enhancing safety, understanding distribution parameters, and gaining insights into efficacy in various trials and clinical care.

Clinical Impact and Therapeutic Understanding

The use of image-guided direct intraparenchymal gene therapy has shown promise in various neurologic disorders, including Alzheimer’s, Huntington’s, and Parkinson’s diseases. Dr. Asad S. Akhter, a neurosurgery resident at Ohio State and the study’s first author, emphasizes that this innovation not only improves biologic and clinical understanding but also leads to optimal clinical care for patients undergoing direct intraparenchymal nervous system gene therapy.

Dr. Bankiewicz has led studies involving children treated for AADC-deficiency, a neurotransmitter disorder. Pre-infusion symptoms, such as developmental delay and autonomic dysfunction, saw significant improvement post-infusion, showcasing the potential of this gene therapy approach. Real-time MRI allows for accurate cannula placement and optimized target structure perfusion, offering a comprehensive understanding that can lead to future refinements in delivery parameters.

Author

Sinead Huang