Therapeutic Development in the Absence of Predictive Animal Models of Nervous System Disorders Proceedings of a Workshop (2017) / Chapter Skim
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Appendix B: Workshop Agenda
Appendix B: Workshop Agenda
Pages 67-76
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From page 67... ...
In 2012, the Forum on Neuroscience and Nervous System Disorders hosted a public workshop on Improving the Utility and Translation of Animal Models for Nervous System Disorders to discuss potential opportunities for maximizing the translation of effective therapies from animal models to clinical practice. During the workshop, several participants emphasized the utility of animal models for investigating basic neural processes, but their limitations for fully recapitulating nervous system disorders, and predicting therapeutic efficacy in human clinical trials.
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From page 68... ...
Meeting Objectives: • Explore the utility of novel approaches to the process of target validation and biomarker development including human genet ics, stem cell technologies, including use of iPS cells and human brain organoids, experimental human biology such as molecular imaging and neurophysiology, and computational modeling. • Discuss future technological developments that would facilitate bringing compounds that appear to be safe into human dose find ing and efficacy trials, even if an animal model of the human disease is not achievable.
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From page 69... ...
Day One: September 12, 2016 1:00 p.m. Opening Remarks and Review of Previous Neuroscience Forum Workshops STEVEN HYMAN, Workshop Chair Director, Stanley Center for Psychiatric Research Broad Institute of Massachusetts Institute of Technology and Harvard University Distinguished Service Professor Professor of Stem Cell Biology and Regenerative Biology Harvard University 1:15 p.m.
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• Discuss the role of bidirectional translational endpoints and the relationship between preclinical endophenotypes and clinical outcome measures. o What are the clinical questions that could drive preclinical research?
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BREAK 3:45 p.m. Stem Cells and Organoids LEE RUBIN Professor, Department of Stem Cell and Regenerative Biology Harvard University Director of Translational Medicine, Harvard Stem Cell Institute STEVE FINKBEINER Associate Director and Senior Investigator Gladstone Institute of Neurological Disease 4:20 p.m.
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From page 72... ...
Adjourn Day One Day Two: September 13, 2016 8:30 a.m. Day Two Opening Remarks STEVEN HYMAN, Workshop Chair SESSION II: PRIVATE-SECTOR THRESHOLDS FOR INVESTMENT IN NEUROSCIENCE CLINICAL TRIALS Session Objectives: • Discuss the decision-making process within the private sector for pro ceeding with drug development approaches in situations that lack pre dictive animal models of disease.
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Understanding the Role of Public−Private Partnerships to De-Risk the Development Process and to Facilitate Data Sharing JOHN MICHAEL SAUER Executive Director of the Predictive Safety Testing Consortium Critical Path Institute Adjunct Research Professor, Department of Pharmacology University of Arizona College of Medicine 9:55 a.m. Discussion Among Speakers and Workshop Participants FRANK YOCCA, Discussant Senior Vice President of CNS Research and Development BioXcel Corporation
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• Discuss the unique challenges for trials in vulnerable populations. • Discuss the regulatory landscape and the evidence needed for regulatory agencies to consider trials in humans in the absence of predictive animal models of disease.
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Discussion Among Speakers and Workshop Participants LUCIE BRUIJN, Discussant Chief Scientist The ALS Association 12:15 p.m. LUNCH Regulatory Considerations • What evidence is needed to conduct efficacy trials in humans?
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Discussion Among Speakers and Workshop Participants 2:45 p.m. BREAK SESSION IV: MOVING FORWARD Session Objectives: • Highlight workshop key themes.
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