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Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
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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

Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×
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

Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×
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

Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×
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
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×

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.
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×

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
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×
Page 89
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×
Page 90
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×
Page 91
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×
Page 92
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×
Page 93
Suggested Citation:"Appendix A: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25371.
×
Page 94
Next: Appendix B: Speaker Biographical Sketches »
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The emerging multidisciplinary field of regenerative engineering is devoted to the repair, regeneration, and replacement of damaged tissues or organs in the body. To accomplish this it uses a combination of principles and technologies from disciplines such as advanced materials science, developmental and stem cell biology, immunology, physics, and clinical translation. The term "regenerative engineering" reflects a new understanding of the use of tissue engineering for regeneration and also the growing number of research and product development efforts that incorporate elements from a variety of fields. Because regenerative engineered therapies rely on live cells and scaffolds, there are inherent challenges in quality control arising from variability in source and final products. Furthermore, each patient recipient, tissue donor, and product application is unique, meaning that the field faces complexities in the development of safe and effective new products and therapies which are not faced by developers of more conventional therapies. Understanding the many sources of variability can help reduce this variability and ensure consistent results.

The Forum on Regenerative Medicine hosted a public workshop on October 18, 2018, in Washington, DC, to explore the various factors that must be taken into account in order to develop successful regenerative engineering products. Invited speakers and participants discussed factors and sources of variability in the development and clinical application of regenerative engineering products, characteristics of high-quality products, and how different clinical needs, models, and contexts can inform the development of a product to improve patient outcomes. This publication summarizes the presentation and discussion of the workshop.

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