Meeting Agenda

Using Stem Cells for Biological and Therapeutics Discovery in Mental Illness

April 24, 2012 – April 25, 2012
Bethesda, Maryland

Day 1: Tuesday April 24, 2012

8:00 AM – Breakfast

8:30 AM – Introduction

• Thomas R. Insel, M.D. (NIMH)
  Welcome
• David M. Panchision, Ph.D. (NIMH)
  Cell Reprogramming Overview and Meeting Goals
• Jeffrey S. Nye, M.D., Ph.D. (Janssen / Johnson & Johnson)
  Promise and Perils of Reprogrammed Cells in Neuropsychiatric Disorders, Therapeutics Discovery

9:10 AM – Patient Selection Criteria: Clinical Phenotyping, Genetics
Chaired by Jeffrey S. Nye, M.D., Ph.D.

• Russell L. Margolis, M.D. (Johns Hopkins University School of Medicine)
  Schizophrenia phenotype, genotype, and drug response: implications for human cell line studies
• John Kelsoe, M.D. (University of California, San Diego)
  Genomics and pharmacogenomics of Bipolar Disorder: Strategies for cellular models
• Joachim F. Hallmayer, M.D. (Stanford University)
  Genetics and clinical phenotyping of Autism Spectrum Disorders
• Group-wide discussion including panel members:
  Andrew McIntosh, M.D. (University of Edinburgh)
  Flora M. Vaccarino, M.D. (Yale University)
  Judith L. Rapoport, M.D. (NIMH)
  Joachim F. Hallmayer, M.D.

10:30 AM – Break and Poster Viewing

10:50 AM – Reprogramming and Validation Strategies
Chaired by Jeffrey S. Nye, M.D., Ph.D.

• Ludovic Vallier, Ph.D. (Wellcome Trust Sanger Institute; Cambridge University)
  Large scale derivation of human induced pluripotent stem cells: Old challenges and new opportunities
• Paul J. Tesar, Ph.D. (Case Western Reserve University)
  Interrogating the transcriptome and chromatin landscape of distinct cell fates from pluripotent cells
• Jeanne Loring, Ph.D. (Scripps Institute, La Jolla)
  Epigenetic stability in iPSCs, global phenotyping and the stem cell matrix database
• Emile F. Nuwaysir, Ph.D. (Cellular Dynamics, Inc)
  Industrial-scale pipeline for high-throughput reprogramming and large volume production of differentiated cells
• Nan Yang, Ph.D. (Stanford University)
  Making neurons from fibroblasts: Direct reprogramming to neural lineages
• Group-wide discussion including panel members:
  Kevin Eggan, Ph.D. (Harvard University)
  Mike Sheldon, Ph.D. (Rutgers University)
  Mahendra Rao, Ph.D. (NIH Center for Regenerative Medicine)
  Nan Yang, Ph.D.

12:50 PM – Lunch and Main Poster Session (titles below)

• Stewart A. Anderson, M.D. (Weill Cornell Medical College)
  Tools for the study of gene-gene and gene-environment interactions in neuropsychiatric disease
• Ronald Hart, Ph.D. (Rutgers University)
  Neurogenesis using iPSC derived from cell repository specimens having specific risk-associated mutations
• John Kelsoe, M.D.
  Pharmacogenomic Studies of Bipolar Disorder
• Pascal Laeng, Ph.D. (Galenea Corp.)
  Optimization of neuronal cultures derived from hiPSCs for high throughput assays of synaptic function
• Jeanne Loring, Ph.D.
  MicroRNA regulation of hESC proliferation
• Andrew McIntosh, M.D.
  Modeling psychosis in families with rare private mutations
• Jennifer Moore, Ph.D. (Rutgers University)
  Small-Scale, Footprint-Free Generation of Human iPSC from Cell Repository Specimens to Study Neuropsychiatric and Addiction Disorders
• Kristopher Nazor, (Scripps Institute, La Jolla)
  DNA methylation in human PSCs

1:50 PM – Assays 1: Getting the “Right” Cell for the Job – Lessons from Neurological Disorders
Chaired by Stephen J. Haggarty, Ph.D. (Harvard, Broad Institute)

• Kevin Eggan, Ph.D.
  iPSCs from Amyotophic Lateral Sclerosis and Syndrome Lesch-Nyhan
• Kazuhiro Aiba, Ph.D. (Kyoto University)
  Site-directed integration of transgenes and human PSC-derived neurodegenerative disease models
• Yasuyuki Asai, Ph.D. (ReproCELL, Inc.)
  Automated cellular assays for neuronal cell death
• Lorenz P. Studer, M.D. (Memorial Sloan Kettering Cancer Center)
  From iPSC model to novel candidate therapeutic compounds – the case study of familial dysautonomia
• Group-wide discussion including panel members:
  Paul J. Tesar, Ph.D.
  Russell L. Margolis, M.D.
  Evan Y. Snyder M.D., Ph.D. (Sanford Burnham Institute)
  Lorenz P. Studer, M.D.

3:10 PM – Break and Poster Viewing

3:30 PM – Assays 2: Cells to Systems – Developmental & Pathophysiological Relevance of Assays
Chaired by Stephen J. Haggarty, Ph.D.

• Brian Tobe, M.D., Ph.D. (Sanford Burnham Institute)
  Merging proteomics with hiPSC-derived neurons for discovery of lithium targets in bipolar disorder
• Stewart A. Anderson, M.D.
  Derivation and study of cerebral cortical GABAergic interneurons from human PSCs
• Flora M. Vaccarino, M.D.
  Three dimensional in vitro differentiation assays derived from iPSCs
• Guo-li Ming, Ph.D. (Johns Hopkins University School of Medicine)
  In vitro and transplantation studies of Schizophrenia patient-specific iPSCs
• Group-wide discussion including panel members:
  Vikas Duvvuri, M.D., Ph.D. (University of California, San Diego)
  Jack Price, Ph.D. (King's College, London)
  Stewart A. Anderson, M.D.
  Guo-li Ming, Ph.D.

5:20 PM – Break and Poster Viewing

Events at Terrace Level

5:30 PM – Cash Bar Reception / Social

6:45 PM – Working Dinner for Invited Participants (Sponsored by One Mind for Research)
Topic: Steps toward the Formation of an International Collaboration

• Brief Remarks by Jeffrey S. Nye, M.D., Ph.D.
• Brief Remarks by Gen. Peter W. Chiarelli (One Mind for Research)
• Discussion

Day 2: Wednesday April 25, 2012

8:00 AM – Breakfast

8:30 AM – Assays 3: Molecular Discovery, Target Identification, Screening
Chaired by Kalpana M. Merchant, Ph.D. (Eli Lilly and Company)

• Herbert M. Lachman, M.D. (Albert Einstein College of Medicine)
  Transcriptome analysis of neurons derived from iPSCs
• Sergiu Pasca, M.D. (Stanford University)
  Revealing cellular endophenotypes and potential drugs for Timothy syndrome using iPSCs
• Pascal Laeng, Ph.D.
  Optimization of neuronal cultures derived from human iPSCs for high throughput assays of synaptic function
• Stephen J. Haggarty, Ph.D.
  Targeting neuroplasticity using human iPSC-derived neurons
• Group-wide discussion including panel members:
  Ludovic Vallier, Ph.D.
  Herbert M. Lachman, M.D.
  Stephen J. Haggarty, Ph.D.
  David Collier, Ph.D. (Eli Lilly & Company)
  William Z. Potter, M.D., Ph.D. (NIMH)

10:10 AM – Break and Poster Take-Down

10:30 AM – Coordinating Resources and Future Efforts
 Chaired by Kalpana M. Merchant, Ph.D.

• Mike Sheldon, Ph.D. (Rutgers University)
  The NIMH Center for Collaborative Genetic Studies of Mental Disorders: Stem Cell Resource
• Mahendra Rao, Ph.D.
  NIH Center for Regenerative Medicine
• Yong Yao, Ph.D. (NIMH)
  NIH resources for target identification and screening
• Group-wide discussion including panel members:
  Jay Tischfield, Ph.D. (Rutgers University)
  Jeanne Loring, Ph.D.
  John Kelsoe, M.D.
  Sarah J. Caddick, Ph.D. (Gatsby Charitable Foundation, UK)
  Linda S. Brady, Ph.D. (NIMH)

12:00 Noon – Meeting Adjourned