Pharmaceutical targeting of the brain

István A. Krizbai, Nyúl-Tóth, Hans Christian Bauer, Attila E. Farkas, Andreas Traweger, János Haskó, Hannelore Bauer, I. Wilhelm

Research output: Contribution to journalReview article

10 Citations (Scopus)


Besides being indispensable for the protection and nutrition of the central nervous system (CNS), blood-brain barrier (BBB)-forming cerebral endothelial cells (CECs) have a major role in hampering drugs to reach therapeutically relevant concentrations in the brain. In this respect, the most important defense systems of CECs are tight junctions (TJs) sealing the paracellular way of transport, efflux pumps (ABC transporters) and metabolic enzymes. Here we review current strategies aiming at overcoming the BBB with the purpose of effectively delivering drugs to the CNS. Besides chemical modification of drug candidates to improve CNS availability, the main strategies include: bypassing the BBB (intracranial or nasal routes), reversible opening of TJs (using hyperosmotic mannitol, ultrasounds, peptides and other physical methods or chemical agents), vector-mediated drug delivery systems (nanocarriers, exploitation of receptor-and carrier-mediated transport) and inhibition of efflux transporters. We discuss the main advantages, disadvantages and clinical relevance of each strategy. Special emphasis will be given to the description of the chemical characteristics of nanoparticles (lipidic, polymeric, inorganic, etc.) and the main strategies of targeting them to the CNS.

Original languageEnglish
Pages (from-to)5442-5462
Number of pages21
JournalCurrent Pharmaceutical Design
Issue number35
Publication statusPublished - Oct 1 2016


  • ABC transporter
  • Blood-brain barrier (BBB)
  • Carrier-mediated transport (CMT)
  • Central nervous system (CNS)
  • Drug delivery
  • Nanoparticle
  • Receptor-mediated transcytosis (RMT)
  • Tight junction (TJ)

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery

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  • Cite this

    Krizbai, I. A., Nyúl-Tóth, Bauer, H. C., Farkas, A. E., Traweger, A., Haskó, J., Bauer, H., & Wilhelm, I. (2016). Pharmaceutical targeting of the brain. Current Pharmaceutical Design, 22(35), 5442-5462.