Pitt Shield

Focused ultrasound-mediated drug delivery through the blood-brain barrier.

Authors: Burgess A, Shah K, Hough O, Hynynen K

Despite recent advances in blood-brain barrier (BBB) research, it remains a significant hurdle for the pharmaceutical treatment of brain diseases. Focused ultrasound (FUS) is one method to transiently increase permeability of the BBB to promote drug delivery to specific brain regions. An introduction to the BBB and a brief overview of the methods, which can be used to circumvent the BBB to promote drug delivery, is provided. In particular, we discuss the advantages and limitations of FUS technology and the efficacy of FUS-mediated drug delivery in models of disease. MRI for targeting and evaluating FUS treatments, combined with administration of microbubbles, allows for transient, reproducible BBB opening. The integration of a real-time acoustic feedback controller has improved treatment safety. Successful clinical translation of FUS has the potential to transform the treatment of brain disease worldwide without requiring the development of new pharmaceutical agents.

Introduction

Purpose Drug delivery with BBB opening
Study Objective To review focused ultrasound as a method to transiently increase blood–brain barrier permeability to enhance drug delivery, discussing its advantages, limitations, efficacy in disease models, and potential for clinical translation.
Animal model / Human subject rodents and Mice
Disease model Alzheimer's disease, Parkinson's disease, Amytrophic Lateral Sclerosis (ALS) and Multiple Sclerosis
MRI or image guidance method MRI guidance (with real-time acoustic feedback controller)
Targeted brain region(s) Hippocampus
Target coordinates not provided
Cargo name and characteristics not provided
Route of administration intrvenous and intransanal

Outcomes and Safety

Summary of Outcomes Focused ultrasound combined with systemically administered microbubbles and MRI targeting produces transient, reproducible opening of the blood–brain barrier to enhance region-specific drug delivery, and the addition of real-time acoustic feedback controllers improves treatment safety.
Duration of biological effect 1 hr
Safety-related matter The paper notes that integration of a real-time acoustic feedback controller has improved treatment safety during focused ultrasound-mediated BBB opening; no specific adverse effects are reported.

Brain Region

Ultrasound Parameters

Ultrasound instrument not provided
FUS Frequency 0.2-1.5 MHz and MHz
FUS Intensity Not provided
FUS Pressure not provided
FUS Mode Not provided
Pulse duration 100ms
Duration of a single FUS session not provided
Focal Characteristics Focal depth: None; Focal length: None; Aperture size: None
Treatment frequency not provided

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