Regenerative Medicine in the 21 Century: Managing Uncertainty at the Global … · 2019. 12. 20. · Regenerative Medicine in the 21st Century: Managing Uncertainty at the Global

Regenerative Medicine in the 21st Century: Managing Uncertainty at the Global Level

Presenter Abstracts

Standardizing Cell Banking Practices for Research and Clinical Applications Derek Hei, Ph.D., (Waisman Clinical Biomanufacturing Facility) The successful utilization of pluripotent stem cells in research, commercial, and therapeutic applications will require the development of cell culture processes capable of consistently producing cells that meet strict Quality Control requirements. Pluripotent stem cells pose several unique and significant manufacturing challenges including control of differentiation, genetic stability, and potential tumorigenicity, properties that may be significantly influenced by cell line characteristics. High quality, well characterized cell banks will therefore be essential for supporting development of stem cell technologies from research through to commercial applications. In order to support the growing field of human ES cell research, NIH provided funding to WiCell Research Institute to establish the National Stem Cell Bank (NSCB). The NSCB provided support to human embryonic stem cell (hESC) researchers by banking and distributing NIH-approved hESC lines and developing standardized methods for cell culture, cryopreservation and characterization. Cells distributed by the NSCB underwent extensive testing including characterization studies thereby providing detailed information on genetic stability, gene expression, and differentiation potential of the cell lines. The information gathered from these studies, including culture and testing protocols, is readily available to the public through the NSCB website (www.nationalstemcellbank.org). To further support translational research, the Waisman Clinical Biomanufacturing Facility (WCBF, www.gmpbiomanufacturing.org) at the University of Wisconsin has worked with WiCell to produce hESC cell banks under current Good Manufacturing Practice (cGMP) guidelines for future human clinical trials. This presentation will provide a brief overview of the NSCB and WCBF programs and participation in international efforts to establish banking standards through the International Stem Cell Banking Initiative (ISCBI). UK Stem Cell Bank: Developments & Challenges for International Banking Professor Andrew Webster (SATSU, University of York) This paper examines recent developments in the UK Stem Cell Bank in respect to its role within the UK stem cell community, including scientific, clinical, corporate and regulatory

sectors, and the wider contexts of trans-institutional banking as well as the commercialisation of the bank’s activities. It explores its role in the International Stem Cell Initiative and Forum and examines the ways in which technical and regulatory standards have been developed, arguing that these have had to contend with a range of science-based uncertainties that will shape the process of clinical application and corporate scale-up. Disbursements from a Stem Cell Bank: Some Governance Issues Pilar N. Ossorio (University of Wisconsin-Madison Law School) European RM firms and their Strategic Approaches Dr Michael Morrison (SATSU, University of York) Europe constitutes both an important market and a significant industry sector for commercial regenerative medicine (RM). As part of the REMEDiE project, a ‘universe’ of European companies working in the RM field has been mapped out. This data can be used to assess the positioning of European regenerative medicine firms on key strategic issues such as the source of cells for products (non-stem cell vs. stem cell; adult/foetal or embryonic stem cells), selection of therapeutic targets, and autologous vs. allogenic cell products. The data also allows for an analysis of the number and type of regenerative medicine products on the market and in development at European firms and an evaluation of the level of uptake and investment by the commercial sector in emergent technologies including induced pluripotent stem (iPS) cells and cell-based toxicity and target screening in conventional drug development. At a broader level, the European RM sector can be characterised with regards to some of the major promissory trajectories associated with regenerative medicine, notably the transition from a tissue engineering model to a more globalised, translation-orientated regenerative medicine (see Mason, 2007) and the promise of regenerative medicine products to address the problems of an ageing population. KEYNOTE ADDRESS: Policy and Politics in US Stem Cell Research Funding Alta Charo (Law and Medical History & Bioethics, University of Wisconsin-Madison) The history of US funding policies on embryo research and hES cell research in particular is explained more by the politics surrounding abortion rights; research with human subjects; church-state relations; and the demographic shifts in the political parties than it is by the research topics themselves. Nonetheless, this history has resulted in a tortured path littered with special reports and special ethics rules and guidelines, many of which are reflecting in today’s more flexible funding policies. This presentation will briefly trace this history, noting in particular the overlapping and occasionally conflicting rules and guidelines for both research practice and research funding. It will include a presentation on the final National Academies' ethics guidelines for human embryonic stem cell research, released 26 May 2010 to address ethical issues regarding gamete, embryo and somatic cell donation; creation of embryos for research by either IVF or SCNT; cautions and restrictions on experiments involving human-nonhuman chimeras, standards for biobanking, and options for institutional oversight. The final guidelines specifically integrate the new NIH funding rules, and reference the rules and guidelines from international societies and other national bodies. Governing Clinical Trials with Stem Cells in the United States, Europe, and China: Towards a Global Regulatory Framework Herbert Gottweis, Christian Haddad (University of Vienna) & Haidan Chen (Zhejiang University) During the last years, stem cell technologies and the public and political debates around

them have increasingly moved from basic laboratory research to clinical and experimental settings. The field of autologous SC treatments delivered to patients in hospitals is gaining importance. A small number of innovative companies try to move commercial stem cell products into clinical trials. At the same time, a growing number of dubious clinics offers stem cell therapies for a broad variety of health conditions. This move from basic research to clinical translation and application poses challenges for regulators all over the world. In this paper, we investigate the emerging regulatory frameworks and regimes governing the clinical dimensions of regenerative medicine and stem cell therapy. We especially focus on institutional aspects and the articulation and deployment of risk discourses in the regulatory politics of three geographical and political areas: Europe, China, and the US. We argue that despite the fact that regenerative medicine governance brings about new transnational regimes of convergence in policy and practice, significant differences in the institutional arrangements and regulatory practices persist that can be explained by national idiosyncrasies of regulatory cultures and their relation to risk and innovation. Beating the Evidence and Regulatory Game: Medical Tourism and The Direct-to-Consumer Marketing of Stem Cell Therapies Ubaka Ogbogu, (Consortium on Law & Values in Health, Environment & Life Sciences, University of Minnesota) Despite the promise of stem cell research for regenerative medicine, existing evidence indicates that stem cell therapies are not ready for routine access outside of clinical trials. However, ongoing media reports reveal that numerous clinics outside North America are providing fee-based stem cell therapies to patients from the United States and Canada to treat a broad spectrum of disease conditions. The primary mode of contact with potential patients appears to be by direct-to-consumer advertising through the Internet. For example, EmCell (www.emcell.com), based in Ukraine, claims to utilize fetal and embryonic stem cells to treat a long list of conditions ranging from aging to Alzheimer's disease. In this presentation, I will discuss the results of a content analysis of websites offering stem cell therapies, including data on treatments offered, access conditions, therapeutic claims and evidence, indications, and portrayal of risks and benefits of therapy. To assess the evidentiary basis for therapies offered, I will also present the results of a review of published clinical evidence on existing stem cell therapies. The presentation will conclude with recommendations for future policy action. State Strategies and Multi-Level Governance: The Politics of Innovation in Regenerative Medicine Professor Brian Salter & Stuart Hogarth (Global Biopolitics Research Group, Kings College, London) State support for the RM innovation process can be characterised as forming a triangle of interacting policy domains: science, society and the market, reflecting three overlapping policy priorities: the development of a viable R&D platform; the creation of market conditions favourable to commercialisation, and the formation of regulatory regimes which ensure public trust by addressing ethical concerns and issues of risk and safety. Within Europe this policy agenda is addressed by actors in a complex multi-level governance framework involving the European Union, Member States and regional governments. This presentation will explore how the development of RM in Europe is shaped by interactions between these different governance levels, analysing the capacity of the different institutional actors to make effective policy interventions and their relationship with counterparts in the broader global polity. Comparison will be made with multi-level governance within the US, focusing on the respective roles of the federal government and state authorities.

Informed Consent & Human Embryonic Stem Cell Research Robert Streiffer (Philosophy & Medical History & Bioethics, University of Wisconsin-Madison) The standards of consent for ethically permissible research using human embryonic stem cells are often thought to be quite high. In contrast, the standards of consent for ethically permissible research using already existing, anonymized human biological materials are often thought to be quite low. However, in many cases human embryonic stem cell lines just are already existing, anonymized human biological materials. The ethical and policy questions arising from these competing standards will be discussed, and problems with informed consent for existing human embryonic stem cell lines will be reviewed with an eye towards improving consent processes as the field moves forward. KEYNOTE ADDRESS: Oöcyte Procurement for Stem Cell Research: Public Subsidy and the Origins of the Oöcyte Market Catherine Waldby (University of Sydney) While embryo procurement for stem cell research is now a fairly well regulated and ethically standardized area, oöcyte procurement has proved to be a much more volatile affair. Many laboratories have abandoned SCNT research programs because of the difficulties involved in procuring oöcytes, and debate rages around the world as to the appropriate ethical model. In Britain and the USA two programs are currently in train which use public funds to subsidize oöcyte procurement for SCNT research; the egg sharing pilot program in Newcastle-on-Tyne, funded by the Medical Research Council, which gives women who donate research oöcytes a £1,500 rebate on their IVF fees, and the program recently announced in the State of New York, which mandates a $10,000 fee for research oöcytes, funded through public research grants. While phase one clinical trial subjects are routinely paid significant fees, this is the first time that significant public funds have been used to procure research tissues directly from donors. This paper will investigate the historical lineages of this state of affairs. It will consider the origins of oöcyte procurement in 20th century industrialized agriculture, and the development of the unregulated reproductive oöcyte market in the United States in the 1980s. In particular it will consider the contractualisation and monetization of reproductive oöcyte procurement, and the path-dependant constraints these practices place on current attempts to recruit research donors.