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NIH-FDA Workshop: Pluripotent Stem Cells in Translation: Preclinical Considerations

July 10-11, 2012 
Natcher Auditorium at NIH


Day 1 - July 10, 2012

8:00 - 8:30 a.m.

Moderator for Day 1
Pamela G. Robey, Ph.D., Chief, Craniofacial and Skeletal Diseases Branch, 
National Institute of Dental and Craniofacial Research (NIDCR), NIH 
8:30 - 9:20 a.m.
Session 1: Introduction

NIH Welcome
Walter J. Koroshetz, M.D., Deputy Director, National Institute of Neurological Disorders and Stroke (NINDS), NIH

FDA Welcome and Summary of First Workshop
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
Mahendra Rao, M.D., Ph.D., Director, NIH Center for Regenerative Medicine, NIH
9:20 - 10:30 a.m.
Session 2: Turning Experimental Ideas into Therapeutic Products

Changing Your Mindset: The Promise and the Therapeutic Challenges
Pete Coffey, 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
Patrick Au, Ph.D., CBER/FDA
10:30 - 11:00 a.m.
Morning Break
11:00 - 12:30 p.m.
Session 3: Model/Species Choice for Preclinical Testing

General Considerations in Animal Species/Model Selection
Alice Tarantal, Ph.D., Professor and Vice-Chair for Research, Department of Pediatrics and Associate Director, UC Davis Stem Cell Program

Robert Deans, Ph.D., Executive Vice President, Regenerative Medicine, Athersys

The Effects of Disease/Injury in Proof-of-Concept (POC) Evaluation - the Microenvironment and Stage of Disease
Dale Greiner, Ph.D., University of Massachusetts Medical School
12:30 - 1:30 p.m.
Lunch on your own
1:30 - 3:30 p.m.
The Value of Identifying Mechanism of Action (MOA) from POC Studies
Rajesh Raganathan, Ph.D., Director, Office of Translational Research, NINDS, NIH

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

Session 4: Cell Delivery and Tracking

Tracking Cell Distribution and Fate
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
Emerson Perin M.D., Director, Clinical Research for Cardiovascular Medicine; Medical Director, Stem Cell Center, Texas Heart Institute
3:30 - 4:00 p.m.
Afternoon Break
4:00 - 4:30 p.m.
Panel Discussion: 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 - July 11, 2012

7:45 - 8:15 a.m.
8:15 - 8:20 a.m.
Welcome Back

Moderator for Day 2
Naomi Kleitman, Ph.D., Program Director, NINDS, NIH
8:20 - 10:15 a.m.
Session 5: Safety and Preclinical Study Design

Best Practices for Preclinical Study Design
Wei Liang, Ph.D., CBER/FDA

Effects of Disease in Pivotal Safety Evaluation
Clive Svendsen, Ph.D., Director, Cedars Sinai Regenerative Medicine Institute

Achieving Cell Engraftment/Survival in Preclinical Safety Testing
Casey Case, Ph.D., Vice President of Research, SanBio

Testing for Tumorigenicity and Ectopic Tissue Formation
Joy Cavagnaro, Ph.D., DABT, RAC, President, Access Bio
10:15 - 10:45 a.m.
10:45 - 12:15 p.m.
Session 6: Case Studies: Lessons Learned

Investigator Experiences with Transitioning from Concept to Clinical Trial

Industry Experience:
Eva L. Feldman, M.D., Ph.D., F.A.A.N., Director, A. Alfred Taubman Medical Research Institute; Director, Program for Neurology Research and Discovery and Director of Research, ALS Clinic, University of Michigan Health System

Industry Experience:
Ed Mickunas, M.A., Vice President of Regulatory Affairs, Advanced Cell Technology

Academic Experience:
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
Panel Moderator: Mercedes Serabian, M.S., DABT, CBER/FDA
12:15 - 1:15 p.m.
Lunch on your own
1:15 - 2:30 p.m.
Session 7: Combination Products

Regulation of Combination Products (FDA)
Steven Oh, Ph.D., CBER/FDA

Testing the Effectiveness and Safety of a Cell/Device Combination Product
Eugene Brandon, Ph.D., Director, Strategic Relations and Project Management, ViaCyte

Driving Cell Fate with Adjunct Factors
Arnold Kreigstein, M.D., Ph.D., Director, Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research, UCSF
2:30 - 2:50 p.m.
Afternoon Break
2:50 - 4:20 p.m.
Session 8: Emerging Areas

The 3R’s/In Vitro Testing
Dawn R. Applegate, Ph.D. President and CEO, RegeneMed

Humanized Mouse Models for Immunogenicity Testing
Leonard Schultz, Ph.D., The Jackson Laboratory

Large animal models (e.g., transgenic pigs)
R. Michael Roberts, Ph.D., Curator’s Professor of Animal Science and Biochemistry, University of Missouri-Columbia
4:20 - 4:40 p.m.
Wrap-up and Summary of Meeting
Pete Coffey
4:40 p.m.

Last Modified October 4, 2012