Drug targeting to the brain

Annu Rev Pharmacol Toxicol. 2007:47:323-55. doi: 10.1146/annurev.pharmtox.47.120505.105237.


The central nervous system (CNS) is a sanctuary site and is protected by various barriers. These regulate brain homeostasis and the transport of endogenous and exogenous compounds by controlling their selective and specific uptake, efflux, and metabolism in the brain. Unfortunately, potential drugs for the treatment of most brain diseases are therefore often not able to cross these barriers. As a result, various drug delivery and targeting strategies are currently being developed to enhance the transport and distribution of drugs into the brain. Here we discuss briefly the biology and physiology of the blood-brain barrier (BBB) and the blood-cerebro-spinal-fluid barrier (BCSFB), and, in more detail, the possibilities for delivering large-molecular-weight drugs by local and global delivery and by viral and receptor-mediated nonviral drug delivery to the (human) brain.

Publication types

  • Review

MeSH terms

  • Biological Transport / physiology*
  • Blood-Brain Barrier / drug effects*
  • Blood-Brain Barrier / physiology*
  • Brain / metabolism
  • Central Nervous System Diseases / drug therapy
  • Drug Carriers / pharmacokinetics*
  • Drug Delivery Systems*
  • Genetic Vectors
  • Humans
  • Macromolecular Substances / pharmacokinetics*
  • Pharmaceutical Preparations
  • Tissue Distribution
  • Viruses


  • Drug Carriers
  • Macromolecular Substances
  • Pharmaceutical Preparations