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