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Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
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B

Workshop Agenda

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

September 8, 2017
Keck Center of the National Academies
500 Fifth Street, NW | Washington, DC

Background:

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 therapeutic agents 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

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
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BBB to deliver therapeutics for nervous system disorders and to identify potential opportunities for moving the field forward.

Workshop Objectives:

  • Provide an overview of current knowledge on the role of the BBB biology and delivery mechanisms examining gaps in our current knowledge that future research may address.
  • Discuss BBB passive and active mechanisms that challenge development and delivery of effective therapeutic interventions to CNS targets.
  • Highlight current data and innovative approaches for delivery of therapeutics across the BBB 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 BBB—from the preclinical to the clinical phase—with an emphasis on risks, levers, and potential collaborative efforts among sectors.

September 8, 2017, Room 100

8:00 a.m. Welcome and Overview of Workshop

HUSSEINI MANJI, Janssen Research & Development, LLC (Co-Chair)

DANICA STANIMIROVIC, National Research Council of Canada (Co-Chair)

Session Objectives:

  • Provide background information about BBB biology, including its function in health and disease states and active and passive mechanisms challenging delivery of therapeutics to the CNS.
  • Review different mechanisms and modes for traversing the BBB for the purpose of therapeutic delivery to the CNS.
  • Highlight gaps in our understanding of BBB biology and transport mechanisms for delivery of therapeutics to the brain.
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
×
8:10 a.m. Introduction

HUSSEINI MANJI, Janssen Research & Development, LLC (Moderator)

8:20 a.m. BBB Structure, Function, and Pathology

BERISLAV ZLOKOVIC, University of Southern California

8:35 a.m. Modes of Traversing and Overcoming the BBB

WILLIAM PARDRIDGE, University of California, Los Angeles

8:50 a.m. Discussion
9:05 a.m. Break

Session Objectives:

  • Describe current understanding of modalities for traversing the BBB.
  • Survey innovative technologies—including Trojan horse approaches, physical targeting and disruption, nanoparticles, and ultrasound—for delivery of therapeutics to the CNS.
  • Discuss desirable characteristics for development of new technologies for traversing the BBB.
9:20 a.m. Session Overview

ERIC SCHAEFFER, Janssen Research & Development, LLC (Moderator)

9:30 a.m. Presentations

FRANK WALSH, Ossianix

VIVIANA GRADINARU, California Institute of Technology

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
×

ROBERT THORNE, University of Wisconsin–Madison

ALEXANDER KABANOV, University of North Carolina at Chapel Hill

CHOI-FONG CHO, Brigham and Women’s Hospital

10:30 a.m. Discussion
11:00 a.m. Break

Session Objectives:

  • Discuss the translation—from late preclinical work to clinical trials—of delivery strategies for traversing the BBB, including delivery of synthetic molecules, biologics, and gene therapy.
  • Describe the limitations of current methods for traversing the BBB and identify research and other potential next steps that would move the field forward.
11:15 a.m. Session Overview

DANICA STANIMIROVIC, National Research Council of Canada (Co-Moderator)

E. ANTONIO CHIOCCA, Harvard Medical School (Co-Moderator)

11:25 a.m. Presentations

BALU CHAKRAVARTHY, National Research Council of Canada

ALEXANDRA GOLBY, Brigham and Women’s Hospital

RUBEN BOADO, ArmaGen

DAHAVALKUMAR SHAH, State University of New York at Buffalo

12:25 p.m. Discussion
12:55 p.m. Lunch
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
×

Session Objectives:

  • Discuss approaches, tools, and lessons learned from other regulatory domains that may advance the development and translation of novel methods to traverse the BBB.
  • Identify specific barriers and opportunities in the regulatory domain related to the development and application of methods for traversing the BBB.
  • Explore issues related to critical attributes and potency assays; safety, including immunogenicity and CNS toxicity; and animal models, including appropriate species selection.
  • Explore best practices and strategies to facilitate regulatory consideration of novel technologies for traversing the BBB.
1:40 p.m. Session Overview

FRANCESCA BOSETTI, National Institute of Neurological Disorders and Stroke (Moderator)

1:50 p.m. Panel Remarks

DOUGLAS HUNT, ArmaGen

VIKRAM PATEL, Food and Drug Administration

2:20 p.m. Discussion
2:40 p.m. Break

Session Objectives:

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
×
  • Identify specific barriers and opportunities for increased coordinating among ongoing efforts in academia, the private sector, and federal agencies.
  • Brainstorm potential collaborative projects that could be submitted through current or planned mechanisms.
  • Explore novel mechanisms for catalyzing innovative technologies for traversing the BBB through new public–private partnerships and consortia, including discussion of potential practical next steps.
2:55 p.m. Session Overview

SARAH H. LISANBY, National Institute of Mental Health (MODERATOR)

3:05 p.m. Reflecting on the Workshop: Challenges and Emerging Opportunities for Development of Innovative Methods to Traverse the BBB

ERIC SCHAEFFER, Session 1 Moderator

E. ANTONIO CHIOCCA, Session 2 Co-Moderator

FRANCESCA BOSETTI, Panel 1 Moderator

3:30 p.m. Panel Remarks

BERND STOWASSER, Sanofi

ANDREW WELCHMAN, Wellcome Trust

EDMUND TALLEY, National Institute for Neurological Disorders and Stroke/BRAIN Initiative

KATJA BROSE, Chan Zuckerberg Initiative

GRANT CAMPANY, XPRIZE Foundation

5:00 p.m. Discussion
5:30 p.m. Adjourn Workshop
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
×
Page 45
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
×
Page 46
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
×
Page 47
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
×
Page 48
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
×
Page 49
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2018. Enabling Novel Treatments for Nervous System Disorders by Improving Methods for Traversing the Blood–Brain Barrier: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25044.
×
Page 50
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Despite substantial advances in developing treatments for the serious illnesses that affect people worldwide, there remains a tremendous unmet need in the treatment of complex neurologic diseases, including neuropsychiatric and neurodegenerative disorders. Chief among the challenges that have hindered the development of therapeutics for central nervous system (CNS) disorders is the blood–brain barrier (BBB). The Forum on Neuroscience and Nervous System Disorders of the National Academies of Sciences, Engineering, and Medicine convened a workshop to explore the challenges associated with the BBB that have thus far stymied development of CNS drugs, examine new technologies that could address these challenges, and highlight potential opportunities for moving the field forward. This publication summarizes the presentations and discussions from the workshop.

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