Executive Summary

Stem cell research offers unprecedented opportunities for developing new medical therapies for debilitating diseases and a new way to explore fundamental questions of biology. Stem cells are unspecialized cells that can self-renew indefinitely and also differentiate into more mature cells with specialized functions. Research on human embryonic stem cells, however, is controversial, given the diverse views held in our society about the moral and legal status of the early embryo. The controversy has encouraged provocative and conflicting claims both inside and outside the scientific community about the biology and biomedical potential of both adult and embryonic stem cells.

The National Research Council and Institute of Medicine formed the Committee on the Biological and Biomedical Applications of Stem Cell Research to address the potential of stem cell research. The committee organized a workshop that was held on June 22, 2001. At the workshop, the committee heard from many leading scientists who are engaged in stem cell research and from philosophers, ethicists, and legal scholars. (Audio files of the speakers’ presentations are available until December 31, 2002, at the workshop Web site, www.nationalacademies.org/stemcells.)

The participants discussed the science of stem cells and a variety of ethical and other arguments relevant to public policy as it applies to stem cells. The committee considered the



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Stem Cells and the Future of Regenerative Medicine Executive Summary Stem cell research offers unprecedented opportunities for developing new medical therapies for debilitating diseases and a new way to explore fundamental questions of biology. Stem cells are unspecialized cells that can self-renew indefinitely and also differentiate into more mature cells with specialized functions. Research on human embryonic stem cells, however, is controversial, given the diverse views held in our society about the moral and legal status of the early embryo. The controversy has encouraged provocative and conflicting claims both inside and outside the scientific community about the biology and biomedical potential of both adult and embryonic stem cells. The National Research Council and Institute of Medicine formed the Committee on the Biological and Biomedical Applications of Stem Cell Research to address the potential of stem cell research. The committee organized a workshop that was held on June 22, 2001. At the workshop, the committee heard from many leading scientists who are engaged in stem cell research and from philosophers, ethicists, and legal scholars. (Audio files of the speakers’ presentations are available until December 31, 2002, at the workshop Web site, www.nationalacademies.org/stemcells.) The participants discussed the science of stem cells and a variety of ethical and other arguments relevant to public policy as it applies to stem cells. The committee considered the

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Stem Cells and the Future of Regenerative Medicine information presented, explored the literature on its own, and contemplated the substance and importance of the preliminary data from recent stem cell experiments. The committee’s deliberations on the issues led to the following conclusions and recommendations. Experiments in mice and other animals are necessary, but not sufficient, for realizing the potential of stem cells to develop tissue-replacement therapies that will restore lost function in damaged organs. Because of the substantial biological differences between nonhuman animal and human development and between animal and human stem cells, studies with human stem cells are essential to make progress in the development of treatments for human disease, and this research should continue. There are important biological differences between adult and embryonic stem cells and among adult stem cells found in different types of tissue. The implications of these biological differences for therapeutic uses are not yet clear, and additional data are needed on all stem cell types. Adult stem cells from bone marrow have so far provided most of the examples of successful therapies for replacement of diseased or destroyed cells. Despite the enthusiasm generated by recent reports, the potential of adult stem cells to differentiate fully into other cell types (such as brain, nerve, pancreas cells) is still poorly understood and remains to be clarified. In contrast, studies of human embryonic stem cells have shown that they can develop into multiple tissue types and exhibit long-term self-renewal in culture, features that have not yet been demonstrated with many human adult stem cells. The application of stem cell research to therapies for human disease will require much more knowledge about the biological properties of all types of stem cells. Although stem cell research is on the cutting edge of biological science today, it is still in its infancy. Studies of both embryonic and adult human stem cells will be required to most efficiently advance the scientific and therapeutic potential of regenerative medicine. Moreover, research on embryonic stem cells will be important to inform research on

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Stem Cells and the Future of Regenerative Medicine adult stem cells, and vice versa. Research on both adult and embryonic human stem cells should be pursued. Over time, all cell lines in tissue culture change, typically accumulating harmful genetic mutations. There is no reason to expect stem cell lines to behave differently. In addition, most existing stem cell lines have been cultured in the presence of non-human cells or serum that could lead to potential human health risks. Consequently, while there is much that can be learned using existing stem cell lines if they are made widely available for research, such concerns necessitate continued monitoring of these cells as well as the development of new stem cell lines in the future. High-quality, publicly funded research is the wellspring of medical breakthroughs. Although private, for-profit research plays a critical role in translating the fruits of basic research into medical advances that are broadly available to the public, stem cell research is far from the point of providing therapeutic products. Without public funding of basic research on stem cells, progress toward medical therapies is likely to be hindered. In addition, public funding offers greater opportunities for regulatory oversight and public scrutiny of stem cell research. Stem cell research that is publicly funded and conducted under established standards of open scientific exchange, peer review, and public oversight offers the most efficient and responsible means of fulfilling the promise of stem cells to meet the need for regenerative medical therapies. Conflicting ethical perspectives surround the use of embryonic stem cells in medical research, particularly where the moral and legal status of human embryos is concerned. The use of embryonic stem cells is not the first biomedical research activity to raise ethical and social issues among the public. Restrictions and guidelines for the conduct of controversial research have been developed to address such concerns in other instances. For example, when recombinant-DNA techniques raised questions and were subject to intense debate and public scrutiny, a national advisory body, the Recombinant DNA Advisory Committee, was established at the National Institutes of Health (NIH) to ensure that

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Stem Cells and the Future of Regenerative Medicine the research met the highest scientific and ethical standards. If the federal government chooses to fund research on human embryonic stem cells, a similar national advisory group composed of exceptional researchers, ethicists, and other stakeholders should be established at NIH to oversee it. Such a group should ensure that proposals to work on human embryonic stem cells are scientifically justified and should scrutinize such proposals for compliance with federally mandated ethical guidelines. Regenerative medicine is likely to involve the implantation of new tissue in patients with damaged or diseased organs. A substantial obstacle to the success of transplantation of any cells, including stem cells and their derivatives, is the immune-mediated rejection of foreign tissue by the recipient’s body. In current stem cell transplantation procedures with bone marrow and blood, success can hinge on obtaining a close match between donor and recipient tissues and on the use of immunosuppressive drugs, which often have severe and life-threatening side effects. To ensure that stem cell-based therapies can be broadly applicable for many conditions and individuals, new means to overcome the problem of tissue rejection must be found. Although ethically controversial, somatic cell nuclear transfer, a technique that produces a lineage of stem cells that are genetically identical to the donor, promises such an advantage. Other options for this purpose include genetic manipulation of the stem cells and the development of a very large bank of embryonic stem cell lines. In conjunction with research on stem cell biology and the development of stem cell therapies, research on approaches that prevent immune rejection of stem cells and stem cell-derived tissues should be actively pursued. The committee is aware of and respectful of the wide array of social, political, legal, ethical, and economic issues that must be considered in policy-making in a democracy. And it is impressed by the commitment of all parties in this debate to life and health, regardless of the different conclusions they draw. The committee hopes that this report, by clarifying what is known about the scientific potential of stem cells and how that potential can best be realized, will be a useful contribution to the

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Stem Cells and the Future of Regenerative Medicine debate and to the enhancement of treatments for disabling human diseases and injuries. On August 9, 2001, when President Bush announced a new federal policy permitting limited use of human embryonic stem cells for research, this report was already in review. Because this report presents the committee’s interpretation of the state of the science of stem cells independent of any specific policy, only minor modifications to refer to the new policy have been made in the report. RECOMMENDATIONS Studies with human stem cells are essential to make progress in the development of treatments for human disease, and this research should continue. Although stem cell research is on the cutting edge of biological science today, it is still in its infancy. Studies of both embryonic and adult human stem cells will be required to most efficiently advance the scientific and therapeutic potential of regenerative medicine. Research on both adult and embryonic human stem cells should be pursued. While there is much that can be learned using existing stem cell lines if they are made widely available for research, concerns about changing genetic and biological properties of these stem cell lines necessitate continued monitoring as well as the development of new stem cell lines in the future. Human stem cell research that is publicly funded and conducted under established standards of open scientific exchange, peer review, and public oversight offers the most efficient and responsible means to fulfill the promise of stem cells to meet the need for regenerative medical therapies. If the federal government chooses to fund human stem cell research, proposals to work on human embryonic stem cells should be required to justify the decision on scientific grounds and should be strictly scrutinized for compliance with existing and future federally mandated ethical guidelines. A national advisory group composed of exceptional researchers, ethicists, and other stakeholders should be established at the National Institutes of Health (NIH) to oversee research on human embryonic stem cells. The group should include leading experts in the most current scientific knowledge relevant to stem cell research who can evaluate the technical merit of any proposed research on human embryonic stem cells. Other roles for the group could include evaluation of potential risks to research subjects and ensuring compliance with all legal requirements and ethical standards. In conjunction with research on stem cell biology and the development of potential stem cell therapies, research on approaches that prevent immune rejection of stem cells and stem cell-derived tissues should be actively pursued. These scientific efforts include the use of a number of techniques to manipulate the genetic makeup of stem cells, including somatic cell nuclear transfer.

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