Abstract #1064

Enhanced Delivery and Imaging of Neurotherapeutics Via US, MRI, SPECT

Michael Valdez¹, Eriko Yoshimaru¹, Pier Ingram², John Totenhagen¹, Aaron Forbes³, Stephen K. Moore¹, Paul Helquist⁴, Terry O. Matsunaga⁵, Russell Witte¹, Lars R. Furenlid⁶, Zhonglin Liu², Robert P. Erickson⁷, Theodore Trouard¹

¹Biomedical Engineering, University of Arizona, Tucson, AZ, United States; ²Radiology, University of Arizona, Tucson, AZ, United States; ³Chemistry & Biochemistry, University of Notre Dame, South Bend, NC, United States; ⁴Chemistry & Biochemistry, University of Notre Dame, Notre Dame, IN, United States; ⁵Radiology, University of Arizona, Tuscon, AZ, United States; ⁶Radiology and Optical Sciences, University of Arizona, Tucson, AZ, United States; ⁷Pediatrics, University of Arizona, Tucson, AZ, United States

Recent and novel techniques that use focused ultrasound (FUS) with microbubble agents have been developed that reversibly open up the BBB and have been demonstrated in animal models, including mice. BBB opening is verifiable with MRI using gadolinium contrast agents, but this does not provide information about delivery of the actual drug to the brain. Our studies address this by combining FUS-mediated BBB opening with high-resolution single-photon computed tomography of ¹²³I-radiolabeled beta-cyclodextrin (BCD) in Niemann-Pick type C disease (NPCD) model mice. When delivered to the brain, BCD is a promising treatment for NPCD, which is genetic, fatal, and affects children.