Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood—Brain Barrier: Proceedings of a Workshop


Note: Proceedings contain the opinion of the presenters, but do NOT reflect the conclusions of the Health and Medicine Division or the National Academies. Learn more about the differences between Reports and Proceedings.

The blood-brain barrier (BBB) presents a special challenge to the development of therapeutics for many central nervous system (CNS) disorders. Far from acting simply as a physical barrier, the BBB is a complex dynamic system involving several cell types, passive and active transport mechanisms, and adaptive function to control the exchange of substances between the blood and the CNS. Few therapeutic agents readily traverse the BBB to reach the brain or spinal cord, including most small molecule drugs and the vast majority of large molecules such as proteins.

Recent methodological developments and innovative drug delivery approaches may greatly increase the armamentarium of potential drugs for treating psychiatric and neurological disorders. The Forum on Neuroscience and Nervous Systems Disorders of the National Academies of Sciences, Engineering, and Medicine convened a public workshop on September 8, 2017, bringing together experts from academia, government, the biotechnology and pharmaceutical sector, disease-focused organizations, and other interested stakeholders to explore current advances in traversing the BBB to deliver therapeutics for nervous system disorders and identify potential opportunities for moving the field forward. The workshop rapporteurs have prepared this proceedings as a factual summation of the session discussions.