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