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Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
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B

Workshop Agenda

Transgenic and Chimeric Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models—A Workshop

October 4, 2018
Keck Center of the National Academies
500 Fifth Street, NW, Washington, DC

Background:

The translational disconnect from preclinical studies with predominantly rodent animal models to human clinical trials remains a key challenge associated with lagging development of therapies for brain disorders. Since 2012, the Forum on Neuroscience and Nervous System Disorders has hosted a series of workshops examining different aspects of this challenge, including maximizing the translation of effective therapies from animal models to clinical practice and exploring the evidence needed to bring compounds that appear to be safe into human efficacy trials. While no animal model will fully recapitulate human nervous system disorders, nonhuman primates (NHPs)—such as marmosets and macaques—have shown promise in their ability to serve as models for complex brain disorders, given the phylogenetic proximity and genetic similarity to humans, similarity of neuroanatomical organization (e.g., a well-developed prefrontal cortex) and associated cognitive and behavioral functions, social cognition, and the ability to study developmental phenotypes and prodromal disease states.

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
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Workshop Objectives:

This 1-day public workshop will bring together experts and key stakeholders from academia, government, industry, and nonprofit organizations to examine the scientific opportunities and challenges, as well as bioethical considerations, of genetically engineered nonhuman primate models for neuroscience research.

Invited presentations and discussions will be designed to:

  • Discuss the state of the science of transgenic and chimeric neuroscience research and emerging models for nervous system disorders, and explore the potential usefulness of such models to enhance understanding of behavior and higher cortical function and advance therapeutic development.
  • Examine current tools and technologies used in rodent models (e.g., transgenesis, chimera, adeno-associated viruses [AAVs], gene therapy, etc.) and explore how they would need to be modified for use in other animal models, such as nonhuman primates.
  • Consider bioethical principles and issues related to genetic engineering of animal models for nervous system disorders, and discuss potential metrics for determining the models’ readiness for nonhuman primate research.
  • Discuss policies and infrastructure needed to advance research in this domain including, for example, training, recruitment of early career scientists, and the potential development of specialized research centers and international collaborations.
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
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DAY ONE: October 4, 2018

8:30 a.m. Welcome and Overview of Workshop

FRANCES JENSEN, Perelman School of Medicine, University of Pennsylvania (Chair)

Session I: Emerging Transgenic and Chimeric Nonhuman Primate Models for Neuroscience Research and Therapeutic Development for Nervous System Disorders

Objective:

  • Discuss the state of the science of transgenic and chimeric nonhuman primate models for nervous system disorders and explore the potential usefulness of such models to enhance understanding of behavior and higher cortical function and in translational science to advance therapeutic development.
8:45 a.m. Session Overview

SARAH CADDICK, Thalamic (Moderator)

8:55 a.m. Speakers

GUOPING FENG, Massachusetts Institute of Technology

HIDEYUKI OKANO, Keio University School of Medicine; RIKEN Brain Science Institute

YOLAND SMITH, Yerkes National Primate Research Center; Emory University

ANGELA ROBERTS, University of Cambridge

9:55 a.m. Discussion
Discussant: WILLIAM NEWSOME, Stanford University
10:30 a.m. BREAK
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×

Session II: Technology, Research Methodology, and Assessment Tools for Transgenic and Chimeric Nonhuman Primate Models

Objectives:

  • Examine how current tools and technologies developed in rodent models (e.g., transgenesis, chimera, AAVs, gene therapy, in vitro fertilization, etc.) through the BRAIN Initiative and elsewhere might be modified for use in nonhuman primates.
  • Consider potential logistical and feasibility issues unique to nonhuman primate models (e.g., cost).
10:45 a.m. Session Overview

ROBERT WURTZ, National Eye Institute (Scientist Emeritus) (Moderator)

11:00 a.m. Speakers

MU-MING POO, Chinese Academy of Sciences

BEN DEVERMAN, Broad Institute of Massachusetts Institute of Technology and Harvard University

JEAN BENNETT, University of Pennsylvania

KAREN PARKER, Stanford University

12:00 p.m. Discussion
  • Why and how do you make that leap from rodents to nonhuman primates technically?
  • What are the logistical and feasibility issues in using genetic and chimeric technologies in nonhuman primate models for neuroscience research (e.g., cost)?
  • What tools and technologies are currently being used or are needed to create these models?
  • What measures and assessment tools are needed (i.e., behavioral assessments)?

Discussants: DAVID AMARAL, University of California, Davis

ROBERT DESIMONE, Massachusetts Institute of Technology

12:30 p.m. LUNCH
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×

Session III: Bioethical Considerations for Transgenic and Chimeric Nonhuman Primate Models in Neuroscience Research

Objectives:

  • Explore bioethical principles and issues related to the genetic engineering of nonhuman primate models or the creation of chimeric nonhuman primate models for neuroscience research.
  • Consider key questions that will necessitate nonhuman primate models for basic and translational research.
  • Discuss potential safeguards needed for transgenic and chimeric nonhuman primate models of nervous system disorders to ensure proper animal welfare.
1:30 p.m. Session Overview

HENRY T. GREELY, Stanford University (Moderator)

1:40 p.m. Speakers

MARGARET LANDI, GlaxoSmithKline

STEFAN TREUE, German Primate Center; Georg-August University

JEFFREY KAHN, Johns Hopkins University

2:25 p.m. Discussion
  • As NHPs deserve greater or different consideration than other nonhuman animal species used in research generally, should there be particular considerations about their use in transgenic and chimeric neuroscience research? How should that be reflected in which research is carried out, and in the care of NHPs in research settings?
  • What criteria must be met in order to justify the use of nonhuman primates in transgenic and chimeric neuroscience research, that is, type and importance of research questions; unique aspects of nonhuman primates; data from other research models; clinical testing that cannot be performed in human subjects, etc.?
  • What are the possibilities that transgenic and chimeric neuroscience research in NHPs could confer some qualitatively different aspect of cognition on the NHP? How could that be assessed? What
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×
  • would be the significance if that were to happen?

    Discussant: MARINA EMBORG, Wisconsin National Primate Research Center; University of Wisconsin–Madison
3:00 p.m. BREAK

Session IV: Moving Forward: Policy and Infrastructure Needs to Advance Research

Objectives:

  • Synthesize and discuss key highlights from the workshop presentations and discussions, including identifying next steps and promising areas for future action and research.
  • Discuss policies and infrastructure needed to advance research in this domain, including, for example, training, recruitment of early career scientists, and the potential development of specialized research centers and international collaborations.
  • Consider the roles of national primate research centers, governments, private philanthropy, and other key stakeholders to advance this research.
3:15 p.m. Session Overview

FRANCES JENSEN, Perelman School of Medicine, University of Pennsylvania (Chair)

3:25 p.m. Keynote

MU-MING POO, Chinese Academy of Sciences

3:40 p.m. Panel Discussion

JOHN MORRISON, California National Primate Research Center, University of California, Davis

HIDEYUKI OKANO, Keio University School of Medicine;

RIKEN Brain Science Institute

JOSHUA GORDON, National Institute of Mental Health

MARK FRASIER, The Michael J. Fox Foundation for Parkinson’s Research

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×

JOHN SPIRO, Simons Foundation Autism Research Initiative

LISA STANEK, Sanofi

4:25 p.m. Discussion
5:15 p.m. Closing Remarks

STEVEN HYMAN, Broad Institute of Massachusetts Institute of Technology and Harvard University

5:30 p.m. Adjourn Workshop
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×

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Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
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Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×
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Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×
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Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×
Page64
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×
Page65
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×
Page66
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
×
Page67
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Transgenic Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25362.
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To examine the promise, concerns, and challenges related to neuroscience research using genetically modified nonhuman primates, the National Academies of Sciences, Engineering, and Medicine hosted a public workshop on October 4, 2018, bringing together an international group of experts and stakeholders representing academia, industry, laboratory animal management, disease-focused foundations, and federal agencies. The workshop was designed to explore the current state and future promise of research using genetically modified nonhuman primate models of disease to understand the complex functions of the brain that control behavior, movement, and cognition in both health and disease states. This publication summarizes the presentations and discussions from the workshop.

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