Magnetic resonance-guided focused ultrasound: potential for peptide and protein delivery across the blood-brain barrier (#29)
Significant progress has been made in the development of novel therapeutic agents for treatment of neurological disorders. However, the effective delivery of these agents across the blood-brain barrier (BBB) remains a challenge. The AL002 clinical trial, “Assessment of Safety and Efficacy of Exablate Blood-Brain Barrier Disruption for the Treatment of Patients with Probable Alzheimer’s Disease (ClinicalTrials.gov Identifier NCT03671889),” is directed at the use of magnetic resonance-guided focused ultrasound (MRgFUS) technology in early Alzheimer’s disease (AD) patients to demonstrate (a) reversible and safe blood-brain barrier opening, (b) spatially precise blood-brain barrier opening, and (c) improved delivery of gadolinium-based contrast agents. Patients who meet the specific study criteria receive three FUS treatments, two weeks apart, and are followed for five years after the final procedure. At present there are four sites that, combined, have treated a total of 22 patients under AL002: West Virginia University Rockefeller Neuroscience Center, Weill Cornell Medicine, The Ohio State University, and Florida Atlantic University/Delray Medical Center. FUS treatments were well tolerated through reversible and repeated BBB opening in multiple brain locations. Gadobutrol was effectively delivered during treatment. Parallel studies found that MRgFUS could improve delivery of therapeutic antibodies to the brain. In conjunction with AL002, blood-based biomarkers are being developed that can be applied for analysis of AD treatments (such as aducanumab and lecanemab) used in conjunction with MRgFUS. This presentation will provide an update on the effectiveness of MRgFUS and progress in identification of a blood-based biomarker panel.