Advancements in ultrasound-mediated drug delivery for central nervous system disorders

Expert Opin Drug Deliv. 2025 Jan;22(1):15-30. doi: 10.1080/17425247.2024.2438188. Epub 2024 Dec 5.

Abstract

Introduction: Central nervous system (CNS) disorders present major therapeutic challenges due to the presence of the blood - brain barrier (BBB) and disease heterogeneity. The BBB impedes most therapeutic agents, which restricts conventional treatments. Focused ultrasound (FUS) -assisted delivery offers a novel solution by temporarily disrupting the BBB and thereby enhancing drug delivery to the CNS.

Areas covered: This review outlines the fundamental principles of FUS-assisted drug delivery technology, with an emphasis on its role in enhancing the spatial precision of therapeutic interventions and its molecular effects on the cellular composition of the BBB. Recent promising clinical studies are surveyed, and a comparative analysis of current US-assisted delivery system is provided. Additionally, the latest advancements and challenges of this technology are discussed.

Expert opinion: FUS-mediated drug delivery shows promise, but the clinical translation of research findings is challenging. Key issues include safety, dosage optimization, and balancing efficacy with the risk of tissue damage. Continued research is crucial to address these challenges and bridge the gap between preclinical and clinical applications, and could transform treatments of CNS disorders.

Keywords: Focus ultrasound; blood brain barrier opening; central nervous system; drug delivery; neurological disorders; ultrasound-responsive particles.

Publication types

  • Review

MeSH terms

  • Animals
  • Blood-Brain Barrier* / metabolism
  • Central Nervous System Agents* / administration & dosage
  • Central Nervous System Agents* / pharmacokinetics
  • Central Nervous System Diseases* / drug therapy
  • Drug Delivery Systems*
  • Humans
  • Ultrasonography / methods

Substances

  • Central Nervous System Agents