Robert J. Nikolai
BME PhD Proposal Presentation

Date: 2025-04-17
Time: 12:15PM
Location / Meeting Link: EBB Krone Children's Healthcare of Atlanta Seminar Room

Committee Members:
Dr. Stanislav Y. Emelianov (Advisor); Dr. Nicolas M. Boulis; Dr. Brooks Lindsey; Dr. Costas D. Arvanitis; F. Levent Degertekin


Title: Laser Activated Perfluorocarbon Nanodroplets for Intrathecal Drug Delivery to the Spinal Cord

Abstract:
The intrathecal space offers a direct and minimally invasive route for delivering therapeutics to the spinal cord, but the intrathecal-spinal barrier (ISB) limits the transport of large-molecule drugs, such as adeno-associated viruses (AAV), used in gene therapy. This research proposes investigating the use of optically responsive lipid-shell perfluorocarbon nanodroplets (PFCnDs) to transiently open the ISB and enable intrathecal drug delivery to the spinal cord and an intrathecal fiberoptic catheter to deliver the energy required to activate the nanodroplets in vivo. Three specific aims will be addressed: (1) demonstrating that laser-activated PFCnDs can enhance AAV delivery in vitro, (2) developing and validating a fiberoptic catheter for PFCnD delivery and activation, and (3) evaluating the system's ability to open the ISB and improve drug penetration in vivo using rodent models. This approach leverages NIR dye-loaded PFCnDs for biosafe optical droplet vaporization (ODV) and a fiberoptic catheter for precise delivery and activation. Successful proof-of-concept results could enable future non-invasive therapies for spinal cord diseases, overcoming size constraints for therapeutic agents and advancing treatment modalities.