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.