ABSTRACT
Support is requested for a Keystone Symposia conference entitled Drug Delivery to the Brain: Challenges and
Progress, organized by Drs. Robert Thorne and Reina Bendayan. The conference will be held in
Breckenridge, Colorado from January 26-39, 2023.
This Keystone Symposia conference is designed to address the urgent scientific need for innovative, new
approaches to central nervous system (CNS) drug delivery. Indeed, the current shortage of disease-modifying
treatments for Alzheimer’s, Parkinson’s and other CNS diseases represent among the most significant unmet
health needs of our time. Our ability to effectively tap into the vast potential that protein, oligonucleotide, and
gene therapies have for treating CNS disorders has been sharply limited by the typically insufficient brain
exposure that results after their systemic or central administration. Even many orally administered small
molecules suffer from brain exposure limitations. Among the primary reasons for these limitations are the
physical and biochemical barriers that exist at key CNS interfaces, including the blood-brain barrier at the level
of the cerebrovasculature, other specialized barriers between the blood and cerebrospinal fluid, and further
obstacles hindering drug exchange between cerebrospinal fluid and CNS tissue. CNS drug delivery research
lies at the crossroads between many different fields, including physiology, pharmacology, pharmaceutical
science, neuroscience, neurosurgery, engineering, genetics, and vascular biology, among others. Surprisingly
few conferences to date have focused exclusively on the multidisciplinary challenges associated with CNS
drug delivery. The goals of this symposium are (i) to bring together international experts and junior
investigators from multiple research fields for the purpose of exchanging new ideas and brainstorming novel
solutions to existing CNS drug delivery challenges and (ii) to highlight new methods and perspectives with the
potential to change how CNS drug delivery research studies are performed and, ultimately, to transform the
field. Sessions are to include a number of short talks chosen to integrate late-breaking developments and new
research directions from the field.