Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood-Brain Barrier – A Workshop

When: September 8, 2017 (8:30 AM Eastern)

Topics Biomedical and Health Research, Substance Use and Mental Health, Diseases, Aging, Public Health, Quality and Patient Safety
Activity: Forum on Neuroscience and Nervous System Disorders
Board: Board on Health Sciences Policy




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. Several research groups are exploiting intrinsic BBB transport mechanisms, such as molecular Trojan horses, and exploring technologies, such as chemical modifications and physical disruption, to test delivery of therapeutics to the CNS. Such strategies may greatly increase the armamentarium of potential drugs for treating psychiatric and neurological disorders. This public workshop will bring together key experts from academia, government, the biotechnology and pharmaceutical sector, disease-focused organizations, and other interested stakeholders to explore current development of novel methods for traversing the BBB to deliver therapeutics for nervous system disorders and identify potential opportunities for moving the field forward.

Workshop Objectives:

  • Provide an overview of current knowledge on the role of the brain-blood barrier biology and delivery mechanisms examining gaps in our current knowledge that future research may address.
  • Discuss brain-blood barrier passive and active mechanisms that challenge development and delivery of effective therapeutic interventions to central nervous system targets.
  • Highlight current data and innovative approaches for delivery of therapeutics across the brain-blood barrier harnessing methods including chemical modifications, Trojan horse approaches, physical targeting and disruption, nanoparticles, ultrasound, and other technologies.
  • Explore potential opportunities for catalyzing development of novel treatments that cross the blood-brain barrier--from the preclinical to clinical phase--with an emphasis on risks, levers, and potential collaborative efforts among sectors.


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