Abstract
The blood brain barrier (BBB) is an obstacle for the delivery of potential molecular therapies for neurodegenerative diseases such as Parkinson's disease (PD), Alzheimer's disease (AD), and amyotrophic lateral sclerosis (ALS). Although there has been a proliferation of potential disease modifying therapies for these progressive conditions, strategies to deliver these large agents remain limited. High intensity MRI guided focused ultrasound has already been FDA approved to lesion brain targets to treat movement disorders, while lower intensity pulsed ultrasound coupled with microbubbles commonly used as contrast agents can create transient safe opening of the BBB. Pre-clinical studies have successfully delivered growth factors, antibodies, genes, viral vectors, and nanoparticles in rodent models of AD and PD. Recent small clinical trials support the safety and feasibility of this strategy in these vulnerable patients. Further study is needed to establish safety as MRI guided BBB opening is used to enhance the delivery of newly developed molecular therapies.
Highlights
Reviewed by: Fedor Panov, Mount Sinai Health System, United States Bhavya Shah, University of Texas Southwestern Medical Center, United States
High intensity MRI guided focused ultrasound has already been FDA approved to lesion brain targets to treat movement disorders, while lower intensity pulsed ultrasound coupled with microbubbles commonly used as contrast agents can create transient safe opening of the blood brain barrier (BBB)
This is true for the growing pharmacopeia of molecular therapies including growth factors, enzymes, monoclonal antibodies, and genetic material, all too large to cross the specialized endothelia that compose the blood-brain barrier (BBB)
Summary
Reviewed by: Fedor Panov, Mount Sinai Health System, United States Bhavya Shah, University of Texas Southwestern Medical Center, United States. Studies by Hynynen, McDannold, and colleagues [9,10,11,12] initially demonstrated that FUS applied during the circulation of microbubble suspensions (FDA-approved ultrasound contrast agents) can create a transient and safe disruption of the BBB, which can be targeted to a specific brain region using MRI.
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