Forum on Regenerative Medicine: 2018 and 2019 Program Activities (2020)

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Forum on REGENERATIVE MEDICINE 2018 and 2019 Program Activities 

ABOUT THE FORUM The National Academies of Sciences, Engineering, and Medicine offers a variety of activities to address the science policy needs of federal agencies and others who are in need of credible, independent advice, or are seeking a neutral setting for consideration of challenging issues. Forums are convening activities that provide both a mechanism and a venue for interested parties from academia, industry, government, and other stakeholder groups to meet and discuss issues of mutual interest and concern in a neutral setting. The primary purpose of Forums is to foster dialogue across sectors and institutions, and to illuminate issues, but not necessarily to resolve them. The goal of these activities is to develop a mutual understanding of the relevant issues, and to provide a mechanism that fosters collaboration among stakeholders in addressing these issues. Forums are self-governing, i.e., the membership identifies the topics that it wishes to address, and with assistance from staff, develops meeting agendas and identifies workshop topics. As a result, the topics are likely to span a broad range of issues in research, policy and practice. The National Academies of Sciences, Engineering, and Medicine’s Forum on Regenerative Medicine (“the Forum”) was established in 2016 and provides a convening mechanism for interested parties from academia, industry, government, patient and provider organizations, regulatory bodies, foundations, societies, associations, and others. Members meet and discuss sensitive and difficult issues in a neutral setting in order to engage in dialogue and discussions that address the challenges facing the application of, and the opportunities for, regenerative medicine to improve health through the development of effective new therapies. The Forum identifies existing and potential barriers to scientific and therapeutic advances; discusses opportunities to assist in facilitating more effective partnerships among key stakeholders; examines the impact that current policies have on the discovery, development, and translation of regenerative medicine therapies; considers the unique challenges of identifying, validating, and bringing regenerative medicine applications to market; and explores the ethical, legal, and social issues posed by regenerative medicine advances. Over the past three years, the Forum has identified important questions and challenges in the field of regenerative medicine that have led the group to explore a broad array of issues ranging from the unique challenges of manufacturing regenerative medicine products, to the complex needs of people who are exploring regenerative medicine as a treatment, to the difficulties with regulating a rapidly evolving field. By bringing together experts and leveraging a wide range of perspectives and knowledge, Forum members have collaborated to identify strategic opportunities to advance the field of regenerative medicine through hosting public workshops, publishing perspective papers and workshop proceedings, and developing resources for the broader regenerative medicine community to support the advancement of high quality science, efficient and effective therapies, and the safety and health of patients. nationalacademies.org/RegenMedForum 

Working Groups and Areas of Interest During 2018-2019, Forum members organized into two working groups and continued to explore issues in a number of other areas. WORKING GROUPS Regenerative Engineering and Gene Therapy Systems Thinking The Forum’s working group on regenerative engineering and The Forum’s working group on systems thinking is interested gene therapy is exploring barriers impeding the successful in advancing the field of regenerative medicine by exploring translation of regenerative and gene-based therapies and new technologies and theoretical models that may improve forward thinking approaches aimed at improving clinical product characterization, manufacturing processes, and outcomes. The working group hosted a workshop in October patient stratification, resulting in greater clinical success 2018 exploring sources of variability related to regenerative of new therapies. The group is interested in examining engineering products and in November 2019, the group opportunities for developing systems-based analytical hosted a workshop focused on designing gene-based methods that can advance the mechanistic understanding clinical trials. The working group is currently interested in of regenerative medicine products and help to overcome exploring solutions to challenges related to the immune challenges with manufacturing and patient outcomes. response to regenerative therapies. The group intends to disseminate information about the value of deep integration of knowledge between materials science, stem cell science, engineering, developmental biology, immunology, computational, and clinical communities. ADDITIONAL AREAS OF INTEREST Workforce Issues Regulatory and Legislative Affairs There is interest among Forum members in understanding Forum members are interested in exploring regulatory what skillsets industry currently needs and what will be challenges including the development of standards and needed in the future to create and manufacture regenerative potential roles for various regulatory bodies involved in medicines. enforcing regulations related to unproven therapies. Communication Cost and Access to Therapies The Forum members are exploring how patients can become Forum members are exploring barriers for patients in more involved in the research and development process accessing regenerative therapies and trying to understand of new therapies and how experts in the field can better the various factors that may contribute to cost and access communicate about regenerative medicine with the public. issues. 2018 and 2019 Program Activities | 1 

2018 and 2019 Meetings FEBRUARY 26-27, 2018 Forum members convened leaders in the field of sports medicine to explore how stem and cell therapies are being used in this area and how information about therapeutic options is being communicated to professional athletes. The group also highlighted case studies in the field JULY 23-24, 2018 of regenerative engineering as well as cell and gene Forum members met with stakeholders to discuss issues therapies to learn about challenges and successes in of cost and reimbursement related to gene therapies and moving products through the translational pathway. explored the potential role of the Forum in the space. The group also learned about current gene therapy clinical tri- als and discussed lessons learned in due diligence in product development efforts and how these can be translated to the field of regenerative medicine. OCTOBER 18, 2018 workshop OCTOBER 19, 2018 Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products Forum members met with experts in the field to discuss advancing stem cell therapies for cardiac failure and a novel academic program in regenerative biology and tissue engineering. FEBRUARY 26-27, 2019 The meeting featured information-gathering sessions on several topics including the role of the immune system in the response to regenerative therapies, workforce development challenges, and advertising and marketing of stem cell therapies. JULY 17-18, 2019 Forum members convened stakeholders from the National Library of Medicine, Research!America, and the Califor- nia State Medical Board Stem Cell Task Force to better understand what types of stem cell therapies are currently available for patients. The meeting also included a strategic planning exercise to help the Forum define priorities for the next three years. NOVEMBER 13, 2019 workshop NOVEMBER 14, 2019 Exploring Novel Clinical Trial Designs for Gene-Based Therapies Forum members discussed challenges and opportunities for cell manufacturing technologies, potential opportunities for improving gene-based clinical trials, and advertising policies around stem cells. 2 | Forum on Regenerative Medicine 

Public Workshops OCTOBER 18, 2018 To further explore the various factors that contribute to successful regenerative engineering products, an ad hoc committee planed a one-day public workshop Exploring Sources of in Washington, DC. Invited speakers and participants discussed factors and Variability Related to the sources of variability in the development and clinical application of regenerative Clinical Translation of engineering products, characteristics of high-quality products, and how different Regenerative Engineering clinical needs, models, and contexts can inform the development of a product. Products Learn more about this workshop here NOVEMBER 13, 2019 This workshop explored the design complexities and ethical issues associated with clinical trials for gene therapies. Workshop speakers highlighted Exploring Novel Clinical the importance of natural history datasets, developing clinical endpoints, Trial Designs for opportunities for effectively communicating with patients about gene-based Gene-Based Therapies clinical trials, and issues with long-term clinical management for patients who participate in gene-based clinical trials. Workshop participants and speakers Learn more about this workshop here discussed strategies for supporting the clinical development of safe and effective gene-based therapies and potential next steps to move the field forward. In 2019, Google released a new advertising policy to describe a category of “speculative and experimental medical treatments.” Google’s decision to Influencing Policy explore their advertising policy was informed by their participation in the Forum’s February 2019 meeting and subsequently through individual follow up discussions with several members. PUBLICATIONS NAM PERSPECTIVE Exploring Sources of Reducing Risks and Variability Related to the Delays in the Translation Clinical Translation of of Cell and Gene Therapy Innovations Regenerative Engineering into Regulated Products: Products: Proceedings of a Discussion Paper (2019) Workshop (2019) Read paper here View report here Perspectives, published by the National Academy of Medicine (NAM), are individually authored by Roundtable and Forum members and outside experts in health and health care. The views expressed in these papers are those of the authors and not necessarily of the authors’ organizations or of the NAM. Perspectives are intended to help inform and stimulate discussion. They have not been subjected to the review procedures of and are not reports of the NAM or the National Academies of Sciences, Engineering, and Medicine. 2018 and 2019 Program Activities | 3 

Forum Membership CO-CHAIRS: Michael May, Ph.D. Rachel Salzman, D.V.M. Tim Coetzee, Ph.D. Centre for Commercialization of American Society of Gene and Cell National Multiple Sclerosis Society Regenerative Medicine Therapy Katherine Tsokas, J.D. Richard McFarland, Ph.D., M.D. Ivonne Schulman, M.D. Johnson & Johnson Advanced Regenerative National Institute of Diabetes and Manufacturing Institute Digestive and Kidney Diseases James C. Beck, Ph.D. (NIDDK) Parkinson’s Foundation Jack Mosher, Ph.D. International Society for Stem Jay Siegel, M.D. Sangeeta N. Bhatia, M.D., Ph.D. Cell Research Retired, Johnson & Johnson Massachusetts Institute of Technology David Owens, Ph.D. Lana Skirboll, Ph.D., M.S. National Institute of Neurological Sanofi Philip John (P.J.) Brooks, Ph.D. Disorders and Stroke (NINDS) National Center for Advancing Martha Somerman, D.D.S., Ph.D. Translational Sciences (NCATS) Amy Patterson, M.D. National Institute of Dental and National Heart, Lung, and Blood Craniofacial Research (NIDCR) George Q. Daley, M.D., Ph.D. Institute (NHLBI) Harvard Medical School; Boston Michael Steinmetz, Ph.D. Children’s Hospital & Dana-Farber Duanqing Pei, Ph.D. Naitonal Eye Institute (NEI) Cancer Institute Chinese Academy of Sciences Lisa Strovink, M.B.A. Brian Fiske, Ph.D. Thomas Petersen, M.D., Ph.D. The New York Stem Cell Foundation The Michael J. Fox Foundation for United Therapeutics Corporation Sohel Talib, Ph.D. Parkinson’s Research Anne Plant, Ph.D. California Institute for Regenerative Larry Goldstein, Ph.D. National Institute of Standards and Medicine Sanford Consortium for Regenerative Technology Philip Vanek, Ph.D. Medicine; UCSD School of Medicine Kimberlee Potter, Ph.D. GE Healthcare Candance Kerr, M.S., Ph.D. Department of Veterans Affairs Daniel Weiss, M.D., Ph.D. National Institute on Aging (NIA) David Rampulla, Ph.D. International Society for Cellular Robert S. Langer, Sc.D. National Institute of Biomedical Therapy Massachusetts Institute of Imaging and Bioengineering Michael Werner, J.D. Technology Derek Robertson, M.B.A., J.D. Alliance for Regenerative Medicine Cato T. Laurencin, M.D., Ph.D. Maryland Sickle Cell Disease Association Celia M. Witten, Ph.D., M.D. The Connecticut Convergence U.S. Food and Drug Administration Institute for Translation in Kelly Rose, Ph.D. Regenerative Engineering; The Claudia Zylberberg, Ph.D. Burroughs Wellcome Fund University of Connecticut Akron Biotech Krishnendu Roy, Ph.D. Terry Magnuson, Ph.D. Georgia Institute of Technology University of North Carolina, Chapel Hill Krishanu Saha, Ph.D. University of Wisconsin-Madison (AS OF MARCH 2020) 4 | Forum on Regenerative Medicine 

Forum Sponsors Advanced Regenerative Manufacturing Institute National Institutes of Health Akron Biotech National Center for Advancing Translational Sciences National Eye Institute Alliance for Regenerative Medicine National Heart, Lung, and Blood Institute American Society of Gene & Cell Therapy National Institute of Biomedical Imaging and Burroughs Wellcome Fund Bioengineering National Institute of Dental and Craniofacial Research California Institute for Regenerative Medicine National Institute of Diabetes and Digestive and Kidney Centre for Commercialization of Regenerative Medicine Diseases U.S. Department of Veterans Affairs National Institute of Neurological Disorders and Stroke National Institute on Aging U.S. Food and Drug Administration The New York Stem Cell Foundation GE Healthcare Parkinson’s Foundation International Society for Cellular Therapy Sanofi International Society for Stem Cell Research United Therapeutics Corporation Johnson & Johnson The Michael J. Fox Foundation for Parkinson’s Research National Institute of Standards and Technology (AS OF MARCH 2020) Patient Advocacy Groups Foundations, Associations, Societies 9% 22% 44% 26% Government Industry May not equal 100% due to rounding 2018 and 2019 Program Activities | 5 

FORUM STAFF Sarah H. Beachy, Ph.D. Siobhan Addie, Ph.D. Senior Program Officer and Program Officer Forum Director Michael Berrios, M.A. Meredith Hackmann Senior Program Assistant Associate Program Officer (until March 2020) Kelly Choi Senior Program Assistant (from March 2020) BOARD ON HEALTH SCIENCES POLICY STAFF Andrew Pope, Ph.D. Bridget Borel Senior Board Director Program Coordinator ABOUT THE The National Academy of Sciences, National Academy of Engineering, and NATIONAL National Academy of Medicine work together as the National Academies ACADEMIES of Sciences, Engineering, and Medicine (“the Academies”) to provide independent, objective analysis and advice to the nation and conduct other activities to solve complex problems and inform public policy decisions. The Academies also encourage education and research, recognize outstanding contributions to knowledge, and increase public understanding in matters of science, engineering, and medicine. The Health and Medicine Division (HMD), formerly known as the program unit of the Institute of Medicine, is a division of the Academies. HMD’s aim is to help those in government and the private sector make informed health decisions by providing evidence upon which they can rely. Each year, more than 3,000 individuals volunteer their time, knowledge, and expertise to advance the nation’s health through the work of HMD. Many of the studies that HMD undertakes are requested by federal agencies and independent organizations; others begin as specific mandates from Congress. While our expert, consensus committees are vital to our advisory role, HMD also convenes a series of forums, roundtables, and standing committees, as well as other activities, to facilitate discussion; discovery; and critical, cross-disciplinary thinking.