Appendix A
Workshop Agenda
Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: A Workshop
October 18, 2018
National Academy of Sciences Building
Lecture Room
2101 Constitution Avenue, NW
Washington, DC 20418
| 8:30 a.m. | Opening Remarks |
|
JAY SIEGEL, Forum Co-Chair
Scientific Advisor Tycho Therapeutics, Inc. |
|
| 8:35 a.m. | Charge to Workshop Speakers and Participants |
|
MARTHA LUNDBERG, Workshop Co-Chair
Program Director, Division of Cardiovascular Sciences Advanced Technologies and Surgery Branch National Heart, Lung, and Blood Institute National Institutes of Health |
|
KATHY TSOKAS, Workshop Co-Chair
Regulatory Head of Regenerative Medicine and Advanced Therapy Johnson & Johnson |
|
| 8:45 a.m. | Stage Setting—The Impact of Variability on Regenerative Engineering Products |
|
GUILLERMO AMEER
Daniel Hale Williams Professor of Biomedical Engineering and Surgery Director, Center for Advanced Regenerative Engineering Northwestern University |
SESSION I: USING CASE STUDIES TO IDENTIFY THE SOURCES OF VARIABILITY ASSOCIATED WITH REGENERATIVE THERAPIES
Session Objective:
- To gain a better understanding of the sources of variability associated with regenerative engineering products through a series of case studies.
Session Moderator: Cato Laurencin, University Professor, Director, Institute for Regenerative Engineering, University of Connecticut
| 9:05 a.m. |
CASE STUDY 1: Variability in the Use of Mesenchymal Stem
Cells for Treating Cardiomyopathy IVONNE HERNANDEZ SCHULMAN Professor of Clinical Medicine University of Miami Miller School of Medicine |
| 9:20 a.m. |
CASE STUDY 2: Sources of Variability in Preclinical and Clinical Research on Stem Cell Therapies for Amyotrophic Lateral Sclerosis
CLIVE SVENDSEN Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine Cedars–Sinai Medical Center |
| 9:35 a.m. |
CASE STUDY 3: Variability in the Development of Cellular Therapies
DAVID STRONCEK Chief, Cell Processing Section Department of Transfusion Medicine National Institutes of Health Clinical Center |
| 9:50 a.m. | Panel Discussion with Speakers and Workshop Participants |
| 10:20 a.m. | Break |
SESSION II: CONSIDERING THE FACTORS THAT CONTRIBUTE TO PATIENT VARIABILITY AND APPROACHES TO ADDRESSING THOSE DIFFERENCES
Session Objectives:
- Discuss factors that contribute to patient variability such as a patient’s genetics, the severity of the condition, past treatments, the placebo effect, and the patient’s built environment/geography.
- Examine the feasibility of a precision medicine approach that would target the right patient with the right regenerative engineering therapy.
Session Moderator: Brian Fiske, Senior Vice President, Research Programs, The Michael J. Fox Foundation for Parkinson’s Research
| 10:35 a.m. |
JENNIFER ELISSEEFF
Morton Goldberg Professor Wilmer Eye Institute and Biomedical Engineering, Translational Tissue Engineering Center Johns Hopkins University |
| 10:50 a.m. |
JOSEPH WU
Director Stanford Cardiovascular Institute Simon H. Stertzer Professor of Cardiovascular Medicine and Radiology Stanford University School of Medicine |
| 11:05 a.m. | STEVE BADYLAK |
|
Professor of Surgery McGowan Institute for Regenerative Medicine University of Pittsburgh |
| 11:20 a.m. |
FLAGG FLANAGAN
Chief Executive Officer and Chairman of the Board DiscGenics |
| 11:35 a.m. | Panel Discussion with Speakers and Audience Members |
| 12:05 p.m. | Working Lunch |
SESSION III: THE IMPORTANCE OF ADDRESSING VARIABILITY IN DONOR TISSUES AND CELLS
Session Objectives:
- Consider the sources of variability among donor tissues and cells, such as the source (e.g., bone marrow, adipose, cord blood), dose, route of administration, and culture conditions, among other factors.
- Discuss methods to address the variability among source tissues and cells so that patients receive a consistent and effective product.
Session Moderator: Martha Lundberg, Program Director, Division of Cardiovascular Sciences, Advanced Technologies and Surgery Branch, National Heart, Lung, and Blood Institute
| 1:00 p.m. |
ANDREW FESNAK
Assistant Professor of Clinical Pathology and Laboratory Medicine University of Pennsylvania Perelman School of Medicine |
| 1:15 p.m. |
GEORGE MUSCHLER
Staff Member Department of Biomedical Engineering Cleveland Clinic |
| 1:30 p.m. |
ALLISON HUBEL
Professor of Mechanical Engineering University of Minnesota |
| 1:45 p.m. | Panel Discussion with Speakers and Audience Members |
| 2:15 p.m. | Break |
SESSION IV: THE IMPORTANCE OF ADDRESSING VARIABILITY AND MEETING QUALITY EXPECTATIONS IN THE MANUFACTURING SETTING
Session Objectives:
- Explore the translational research priorities for the maturing of the fields of tissue science and regenerative engineering.
- Describe advances in preservation technologies needed to sustain fragile cells and tissues under biologically optimized conditions for storage, shipment, and handling.
- Discuss metrics for reproducibility, robustness, and user-friendliness that will enable the broad distribution of products.
Session Moderator: Krish Roy, Robert A. Milton Endowed Chair and Director, Center for ImmunoEngineering, Georgia Institute of Technology
| 2:30 p.m. |
CARL BURKE
BioTherapeutics Development Johnson & Johnson |
| 2:45 p.m. |
MICHELE MYERS
Senior Director, Cell Process Development Cell and Gene Therapy Platform GlaxoSmithKline |
| 3:00 p.m. |
ERIK FINGER
Assistant Professor, Department of Surgery University of Minnesota |
| 3:15 p.m. | Panel Discussion with Speakers and Workshop Participants |
SESSION V: EXPLORING OBJECTIVE METRICS AND OUTCOMES FOR CLINICAL TRIALS AND THE REGULATORY APPROVAL PATHWAY
Session Objectives:
- Discuss ideas for objective metrics and reliable approaches to interpreting the outcomes of clinical trials of regenerative engineering therapies.
- Explore how variability in regenerative engineering products can affect the regulatory approval pathway.
Session Moderator: Kathy Tsokas, Regulatory Head of Regenerative Medicine and Advanced Therapy, Johnson & Johnson
| 3:45 p.m. |
KAREN CHRISTMAN
Scientific Co-Founder Ventrix |
| 4:00 p.m. |
PETER MARKS
Director Center for Biologics Evaluation and Research U.S. Food and Drug Administration |
| 4:15 p.m. | Panel Discussion with Speakers and Workshop Participants |
| 4:35 p.m. | Final Panel Discussion |
| CARL BURKE KAREN CHRISTMAN ALLISON HUBEL PETER MARKS CLIVE SVENDSEN |
|
| 5:00 p.m. | Final Remarks from Workshop Co-Chairs |
|
MARTHA LUNDBERG, Workshop Co-Chair
Program Director, Division of Cardiovascular Sciences Advanced Technologies and Surgery Branch National Heart, Lung, and Blood Institute National Institutes of Health |
|
|
KATHY TSOKAS, Workshop Co-Chair
Regulatory Head of Regenerative Medicine and Advanced Therapy Johnson & Johnson |
|
| 5:10 p.m. | Adjourn |
