Noninvasive and targeted delivery of therapeutics to the brain using focused ultrasound

- Focused ultrasound allows noninvasive targeted opening of the blood-brain barrier.
- A variety of therapeutics have been delivered with positive functional outcomes.
- Safety profile is high when appropriate ultrasound parameters are used.
- Clinical trials using focused ultrasound to deliver drugs are underway.

The range of therapeutic treatment options for central nervous system (CNS) diseases is greatly limited by the blood-brain barrier (BBB). While a variety of strategies to circumvent the blood-brain barrier for drug delivery have been investigated, little clinical success has been achieved. Focused ultrasound (FUS) is a unique approach whereby the transcranial application of acoustic energy to targeted brain areas causes a noninvasive, safe, transient, and targeted opening of the BBB, providing an avenue for the delivery of therapeutic agents from the systemic circulation into the brain. There is a great need for viable treatment strategies for CNS diseases, and we believe that the preclinical success of this technique should encourage a rapid movement towards clinical testing. In this review, we address the versatile applications of FUS-mediated BBB opening, the safety profile of the technique, and the physical and biological mechanisms that drive this process.This article is part of the Special Issue entitled “Beyond small molecules for neurological disorders”.