Cited by 33Nanoparticle-Based Drug DeliveryBarrier Structure and Function StudiesExtracellular vesicles in disease

Clinical translation of nanomedicines for brain diseases: Current challenges and future directions

Wang, Guoying, Wang, Ningyang, Xia, Xue, Ismail, Muhammad, Shi, Bingyang·2026·Journal of Controlled Release

Abstract

Since the approval of Doxorubicin Liposomal (Doxil®) by the U.S. Food and Drug Administration (FDA) in 1995, the field of nanomedicine has undergone substantial growth, transforming the landscape of drug delivery. Nanomedicine offers innovative approaches to enhance the pharmacokinetics and pharmacodynamics of conventional therapeutic agents. Notably, nanomedicine facilitates targeted and multifunctional drug delivery as well as enabling at-site controlled drug release, improving therapeutic precision and minimizing off-target effects. To date, over 50 nanomedicines have received regulatory approval for the treatment of various diseases, with more than 100 currently undergoing clinical trials. However, despite these great advancements, the application of nanomedicines in treating brain diseases remains limited. The blood-brain barrier (BBB) hurdle and the intricate complexity of the central nervous system (CNS) significantly hinder the development of brain-targeted nanotherapeutics. In this review, we will summarize the current challenges encountered in developing nanomedicines for brain diseases and explore potential strategies to overcome these barriers, aiming to accelerate their translation into clinical practice.

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Crossref · 10.1016/j.jconrel.2025.114426

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Authors
Wang, G., Wang, N., Xia, X., Ismail, M., & Shi, B.
Affiliations
  • Macquarie University (AU)
  • Henan University (CN)
  • University of Technology Sydney (AU)
Corresponding author
  • Ismail, Muhammad
  • Shi, Bingyang
Venue
Journal of Controlled Release
Publisher
Elsevier BV
Published
2026-01
Volume
389
Pages
114426
ISSN
0168-3659 (ISSN-L)
OpenAlex
W4416416234
Cited by
33
References
409
Citations by year
2026: 33
FWCI
1.92
Type
journal article
Language
en
Open access
closed
Locations
  • Journal of Controlled Release (publishedVersion)
  • PubMed (publishedVersion)
Funders
Cancer Institute NSW (2022/ECF1449)
Topics
  • Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
  • Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Extracellular vesicles in disease
Keywords
nanomedicine, brain disorders, blood-brain barrier, brain drug delivery, nanotherapeutics, clinical translation, central nervous system, controlled drug release, targeted drug delivery
MeSH terms
Animals, Blood-Brain Barrier, Brain Diseases, Humans, Drug Delivery Systems, Nanomedicine, Nanoparticles

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