Regulation of ABC transporters at the blood-brain barrier: new targets for CNS therapy.

Worldwide, more than one billion people are affected by CNS disorders. Despite the huge demand for treatments, existing drugs have limited or no efficacy for some neurological diseases, including brain cancer and certain epilepsies. Furthermore, no effective therapies are available at all for some common disorders of the central nervous system (CNS) such as Alzheimer's disease. ATP-binding cassette (ABC) transporters at the blood-brain barrier (BBB) have become increasingly important in the treatment and pathogenesis of CNS disorders. Here we highlight a novel strategy--targeting signaling pathways that control ABC transporters at the BBB--to protect the brain, improve brain drug delivery, and reduce CNS pathology.