You are here

Back to top

Pluripotent Stem Cells in Translation: Preclinical Considerations

NIH Natcher Auditorium
Rockville, MD
July 10 – July 11, 2012


Day 1 - Tuesday, July 10, 2012


8:00 AM–8:30 AM

  • Registration

8:30 AM–9:20 AM

  • Session 1: Introduction 
    Moderator for Day 1 – Dr. Pamela Robey (5 min.)
  • NIH Welcome (10 min.) 
    Walter Koroshetz, Ph.D., Deputy Director, National Institute of Neurological Disorders and Stroke (NINDS), NIH
  • FDA Welcome and Summary of First Workshop (10 min.) 
    Celia Witten, M.D., Ph.D., Director, Office of Cellular, Tissue and Gene Therapies (OCTGT), CBER/FDA  
  • The Product Development Timeline: Aligning Manufacturing, Preclinical Testing, and Clinical Trials (20+5 min.) 
    Mahendra Rao, M.D., Ph.D., Director, NIH Center for Regenerative Medicine, NIH

9:20 AM–10:30 AM

  • Session 2: Turning Experimental Ideas into Therapeutic Products
  • Changing Your Mindset: The Promise and the Therapeutic Challenges (30+10 min.) 
    Pete Coffey, B.Sc., D.Phil., Director, London Project to Cure Blindness and Professor of Cellular Therapy and Visual Sciences, Institute of Ophthalmology, London, UK
  • Understanding FDA Expectations for Preclinical Studies (20+10 min.) 
    Patrick Au, Ph.D., CBER/FDA

10:30 AM–11:00 AM

  • Break

11:00 AM–12:30 PM

  • Session 3: Model/Species Choice for Preclinical Testing
    • General Considerations in Animal Species/Model Selection
      • Alice Tarantal, Ph.D., UC Davis – DO NOT VIDEOCAST (20+10 min.)
      • Robert Deans, Ph.D., Executive Vice President,Regenerative Medicine, Athersys (20+10 min.)
    • The Effects of Disease/Injury in Proof-of-Concept (POC) Evaluation - the Microenvironment and Stage of Disease (20+10 min.)
      • Dale Greiner, Ph.D., University of Massachusetts Medical School

12:30 PM–1:30 PM

  • Lunch on your own

1:30 PM–3:30 PM

  • The Value of Identifying Mechanism of Action (MOA) from POC Studies
    • Rajesh Ranganathan, Ph.D., Director, Office of Translational Research, NINDS, NIH (20+10 min)
    • Gary Steinberg, M.D., Ph.D., Chairman, Department of Neurosurgery, Stanford School of Medicine; Co-Director, Stanford Stroke Center; Director, Stanford Institute for Neuro-Innovation and Translational Neurosciences (20+10 min.)
  • Session 4: Cell Delivery and Tracking
    • Tracking Cell Distribution and Fate (20+10 min.) 
      Joe Frank, M.D., M.S., Director, Imaging Sciences Training Program and Senior Investigator, Clinical Center, NIH


    • Assessing the Effectiveness and Safety of the Cell Delivery Route and System (20+10 min.)
      Emerson Perin M.D., Director, Clinical Research for CardiovascularMedicine; Medical Director, Stem Cell Center, Texas Heart Institute

3:30 PM–4:00 PM

  • Afternoon Break

4:00 PM–4:30 PM

  • Panel Discussion (30 min.) 
    What are some of the challenges in conducting preclinical POC studies for pluripotent stem cell-derived products and how can these challenges be addressed? 
    Moderator – Mahendra Rao


DAY 2 - Wednesday, July 11, 2012

7:45 AM–8:15 AM

  • Day 2 Registration

8:15 AM–8:20 AM

  • Welcome Back
  • Moderator for Day 2 Dr. Naomi Kleitman (5 min.)

8:20 AM–10:15 AM

  • Session 5: Safety and Preclinical Study Design


    1. Best Practices for Preclinical Study Design (15+10 min.) 
      Wei Liang, Ph.D., CBER/FDA


    2. Effects of Disease in Pivotal Safety Evaluation (20+10 min.) 
      Clive Svendsen, Ph.D., Director, Cedars Sinai Regenerative Medicine Institute
    3. Achieving Cell Engraftment/Survival in Preclinical Safety Testing (20+10 min.) 
      Casey Case, Ph.D., Vice President, Research, SanBio


    4. Testing for Tumorigenicity and Ectopic Tissue Formation (20+10 min)
      Joy Cavagnaro, Ph.D., DBAT, RAC, President, Access Bio

10:15 AM–10:45 AM

  • Break

10:45 AM–12:15 PM

  • Session 6 : Case Studies: Lessons Learned 
    Investigator Experiences with Transitioning from Concept to Clinical Trial

    1. Industry Experience: (20 min.)
      Eva Feldman, M.D., Ph.D, Univ. of Michigan
    2. Industry Experience: (20 min.)
      Ed Mickunas, M.A., Vice President of Regulatory Affairs, Advanced Cell Technology
    3. Academic Experience: (20 min.)
      Josh Hare, M.D., Chief, Division of Cardiology and Director, Interdisciplinary Stem Cell Institute (ISCI) at University of Miami Miller School of Medicine
  • Panel Discussion on Lessons Learned in Bridging to Clinical (30 min.) 
    Panel Moderator Dr. Mercedes Serabian

12:15 PM–1:15 PM

  • Lunch on your own

1:15 PM–2:30 PM

  • Session 7: Combination Products
    1. Regulation of Combination Products (FDA) (10+5 min.) 
      Steven Oh, Ph.D., CBER/FDA
    2. Testing the Effectiveness and Safety of a Cell/Device Combination Product (20+10 min.) 
      Eugene Brandon, Ph.D., Director, Strategic Relations and ProjectManagement, ViaCyte
    3. Driving Cell Fate with Adjunct Factors (20+10 min.) 
      Arnold Kreigstein, M.D., Ph.D., Director, Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research, UCSF

2:30 PM–2:50 PM

  • Afternoon Break

2:50 PM–4:20 PM

  • Session 8: Emerging Areas
    1. The 3R's/In Vitro Testing (20+10 min.) 
      Dawn R. Applegate, Ph.D. President and CEO, RegeneMed
    2. Humanized Mouse Models for Immunogenicity Testing (20+10 min.) 
      Leonard Schultz, Ph.D., The Jackson Laboratory
    3. Large animal models (e.g., transgenic pigs) (20+10 min.) 
      R. Michael Roberts, Ph.D., Curator’s Professor of Animal Science and Biochemistry, University of Missouri-Columbia

4:20 PM–4:40 PM

  • Wrap-up and Summary of Meeting (20min.)
    Pete Coffey

4:40 PM

  • Adjourn