FINAL REPORT OF THE NATIONAL ACADEMIES’ HUMAN EMBRYONIC STEM CELL RESEARCH ADVISORY COMMITTEE AND 2010 AMENDMENTS TO THE NATIONAL ACADEMIES’ GUIDELINES FOR HUMAN EMBRYONIC STEM CELL RESEARCH

INTRODUCTION

The 2005 National Academies’ Guidelines for Human Embryonic Stem Cell Research laid out standards for responsible and ethical conduct in a controversial field of research that largely lacked federal funding or oversight. Those guidelines helped this important field of research to develop within a framework of defensible, self-imposed rules. The result was greater public confidence in the quality of the work. As certain states (California, Connecticut, Massachusetts, New York, Maryland, and others) have moved to regulate or fund this research, they have used the National Academies’ Guidelines as a template on which to build their own state regulations. The international voluntary standards written by the International Society for Stem Cell Research (ISSCR) also tracked closely the National Academies’ Guidelines.

Since their release, the National Academies’ Guidelines have been adopted wholly or in large part by most major research institutions in the United States. This response included the creation of new Embryonic Stem Cell Research Oversight (ESCRO) committees, use of detailed guidance on informing gamete and embryo donors, and substantive limitations on the range of materials that would be used and how those experiments would be conducted. To assist the research community, the National Academies’ Human Embryonic Stem Cell Research Advisory Committee has conducted regional and other outreach meetings to help investigators and ESCRO committee members to interpret and implement the Guidelines. The Advi-



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INTRODUCTION The 2005 National Academies’ Guidelines for Human Embryonic Stem Cell Research laid out standards for responsible and ethical conduct in a controversial field of research that largely lacked federal funding or over- sight. Those guidelines helped this important field of research to develop within a framework of defensible, self-imposed rules. The result was greater public confidence in the quality of the work. As certain states (California, Connecticut, Massachusetts, New York, Maryland, and others) have moved to regulate or fund this research, they have used the National Academies’ Guidelines as a template on which to build their own state regulations. The international voluntary standards written by the International Society for Stem Cell Research (ISSCR) also tracked closely the National Academies’ Guidelines. Since their release, the National Academies’ Guidelines have been ad- opted wholly or in large part by most major research institutions in the United States. This response included the creation of new Embryonic Stem Cell Research Oversight (ESCRO) committees, use of detailed guidance on informing gamete and embryo donors, and substantive limitations on the range of materials that would be used and how those experiments would be conducted. To assist the research community, the National Academies’ Human Embryonic Stem Cell Research Advisory Committee has conducted regional and other outreach meetings to help investigators and ESCRO committee members to interpret and implement the Guidelines. The Advi- 1

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Guidelines for Human Embryonic Stem Cell Research 2 sory Committee also updated the Guidelines in 2007 and 2008 to reflect the lessons learned by scientists and administrators around the country and to reflect changes in the science of stem cell research. Finally, the Advisory Committee organized or participated in several public workshops on key areas of concern, such as the medical risks of oocyte donation and the next steps toward translating bench science to clinical trials. The inauguration of President Barack Obama in January 2009 led to a marked shift in federal policies on stem cell research. On March 9, President Obama issued Executive Order (EO) 13505, “Removing Barriers to Respon- sible Scientific Research Involving Human Stem Cells.” (Federal Register Volume 74, Number 46, pp. 10667-10668). President Obama’s EO stated that the “Secretary of Health and Human Services, through the Director of NIH [National Institutes of Health], may support and conduct responsible, scientifically worthy human stem cell research, including human embryonic stem cell research, to the extent permitted by law.” While leaving untouched the “Dickey-Wicker” amendment,1 which can only be changed by Congress and which effectively prohibits the use of federal funds to derive new hu- man embryonic stem (hES) cell lines, the EO did rescind prior Executive branch policy. Specifically, the EO rescinded the previous policy that had restricted federal funding for hES cell research to in vitro work on lines derived before an earlier EO issued by President George W. Bush, by stating “The Presidential statement of August 9, 2001, limiting Federal funding for research involving human embryonic stem cells, shall have no further effect as a statement of governmental policy.” The EO issued by President Obama also called upon NIH to review its own existing guidance as well as other widely recognized guidelines on human stem cell research, including provisions establishing appropriate safeguards, and to develop and issue new NIH guidance for such research that is consistent with the EO’s call to support “responsible, scientifically worthy” stem cell research. Without the restrictions placed upon it by the previous administration, the NIH announced that it would begin a broader 1 The so-called “Dickey Wicker” amendment has been included in the annual federal ap- propriation for government-funded activities and has been interpreted to prevent the creation of new human embryonic stem cell lines using federal funds. For example, Section 509 of the Omnibus Appropriations Act 2009, enacted as Public Law 111-8) says: None of the funds made available in this Act may be used for—(1) the creation of a human em- bryo or embryos for research purposes; or (2) research in which a human embryo or embryos are destroyed, discarded, or knowingly subjected to risk of injury or death greater than that allowed for research on fetuses in utero under 45 CFE 46.204(b) and section 498(b) of the Public Health Service Act (42 U.S.C. 289g(b)).

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2010 Amendments 3 program of funding extramural hES cell research according to its own new guidelines on eligibility for funding. The NIH Guidelines on Human Stem Cell Research were issued on July 7, 2009 (Appendix A). They establish mechanisms to determine the eligi- bility of hES cell lines for federal research funding based on the principles that (1) responsible research with hES cells has the potential to improve our understanding of human health and illness and discover new ways to prevent and/or treat illness; and (2) individuals donating embryos for research pur- poses should do so freely, with voluntary and informed consent. Many of the provisions defining informed consent in the NIH guidelines closely resemble those of the National Academies, ISSCR, and others that predate the new NIH requirements. Thus, the NIH guidelines address both the evaluation of lines already in existence, derived under a variety of rules and guidelines, as well as lines yet to be derived. NIH has established a Working Group of the Advisory Committee to the Director of NIH to determine which hES cell lines were derived under conditions that meet the requirements of the NIH guidelines.2 It should be noted that the NIH guidelines prohibit the use of federal funding for research using hES cell lines derived from any source other than excess in vitro fertilization (IVF) embryos created for reproductive purposes. Thus research on lines that may, in the future, be derived by somatic cell nuclear transfer (SCNT), parthenogenesis, or from IVF embryos created spe- cifically for research purposes is not currently eligible for federal funding. As a consequence, they would not be subject to the NIH guidelines, including its standards for ensuring voluntary, informed consent for donated materials. The NIH has also established a new Registry of hES cell lines eligible for NIH funding, containing those lines that its Working Group deems to conform with the requirements of the guidelines.3 The NIH approved the first list of hES cell lines for NIH funding on December 2, 2009, a second set on December 14, 2009, and additional lines in the first half of 2010 and indicated that it anticipated a continuing flow of approved hES cell lines to be listed on the NIH Registry. Use of those lines with federal funding will henceforth be governed by the NIH guidelines. This letter report sets out an updated version of the National Academies’ Guidelines, one that takes into account the new, expanded role of the NIH in overseeing hES cell research. It also identifies those avenues of continu- ing National Academies’ involvement deemed most valuable by the research community and other significant stakeholders. 2 See for information about the Working Group. 3 The Registry is available at .

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Guidelines for Human Embryonic Stem Cell Research 4 THE 2010 NATIONAL ACADEMIES’ GUIDELINES Overall, there are three areas in which non-NIH guidelines will continue to be the source of guidance for hES cell research. • irst, because the continuing effect of the Dickey-Wicker amendment F means that derivation of hES cell lines cannot be supported by federal funds, such derivations will need continuing oversight outside the NIH guidelines. And since the acceptability of the cell lines for use in NIH-funded research hinges on the underlying conditions of non- federally funded derivation, the NIH guidelines implicitly overlap many of the National Academies’ Guidelines on derivation. • econd, only hES cell lines derived from excess IVF embryos initially S produced for reproductive purposes are currently eligible for NIH funding. Therefore, hES cell lines derived from other sources (e.g., from embryos produced by IVF for research purposes or by nuclear transfer or other methods) will not be eligible for NIH funding and not subject to the NIH guidelines; this work will continue to need oversight under other guidelines. • hird, because the NIH guidelines only briefly address limits on the T research uses to which embryonic stem cell lines may be put, other guidelines will continue to be useful for a wider range of experiments with chimeras than those currently identified by NIH. To avoid complications, contradictions, and confusion, this Advisory Committee has developed an updated version of the National Academies’ Guidelines that recognizes the new and increased influence of the NIH guidelines, and which incorporates references to the NIH guidelines as ap- propriate in the text of the National Academies’ Guidelines. Where there is complete overlap, the Advisory Committee recommends that the NIH guidelines supersede its own. Where there are gaps or limitations in the NIH guidelines, the Advisory Committee recommends continued adoption of its own Guidelines. The Advisory Committee also notes some areas in which there is tension between NIH, National Academies, and other guidelines or state funding rules, and identifies those for which some variation from National Acad- emies’ Guidelines is to be expected. The first concerns the issue of egg donation. Since the issuance of the 2008 Amendments to the National Academies’ Guidelines, the Ethics Com-

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2010 Amendments 5 mittee of the State of New York’s Empire State Stem Cell Board adopted a resolution allowing New York State-funded stem cell researchers to compen- sate women who donate their oocytes directly and solely to research for the time, risk and burden involved in donating.4 Amounts of compensation are to be comparable to those received by women in New York State for similar donations for reproductive purposes. Compensation may not be based upon number or quality of eggs, but should cover only time and burden. While this Advisory Committee acknowledges that the circumstances surrounding the issue of compensation to oocyte donors continues to evolve, it chose not to change the National Academies’ Guidelines. Therefore, the Advisory Com- mittee leaves intact the wording of Section 3.4(b), recognizing that states and other entities may choose to set their own policies, as New York has done. Second, the Advisory Committee notes that the requirement in the Na- tional Academies’ Guidelines for consent of all gamete donors (see Section 3.3) is not reflected in the new NIH guidelines. Further, a number of states and research institutions have declined to adopt this rule, given the lack of clear legal need for such consent from anonymous donors. The Advisory Committee also notes that the Food and Drug Administration’s (FDA’s) recent tissue transplant rules require screening of gamete donors except in cases involving sexually intimate partners. This suggests that stem cell lines made with donor (i.e., screened) gametes may be marginally safer for tissue transplants and may be more useable for FDA-regulated trials and therapies. The Advisory Committee recognizes that this requirement may be widely overlooked, and that the issue will be relevant only for a small percentage of derivations. Nonetheless, the Advisory Committee still believes that the practice of obtaining informed consent from all gamete donors, as well as other relevant parties (e.g., intended parents), should continue to be followed because it is the most cautious and respectful standard for donation. The combination of the new NIH guidelines and those National Acad- emies’ Guidelines remaining in effect will continue to represent a comprehen- sive and responsible approach as this research advances into the future. THE FUTURE ROLE OF THE NATIONAL ACADEMIES IN STEM CELL RESEARCH OVERSIGHT In addition to reviewing the National Academies’ Guidelines, the Advi- sory Committee also considered the future role of the National Academies 4 The resolution is available at

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Guidelines for Human Embryonic Stem Cell Research 6 in helping to guide responsible conduct in this field. The Advisory Com- mittee dedicated most of its August 7, 2009, meeting to hear input from stakeholders from the stem cell research community and from those who have experience with the implementation of the National Academies’ Guide- lines; a list of these individuals participating in the meeting may be found in Appendix B. One area of considerable discussion was the future of ESCRO commit- tees, as most institutions that have been following the National Academies’ or other non-federal guidelines since 2005 have established such commit- tees. Most participants in the August 7 meeting thought that ESCRO/SCRO committees5 play valuable roles and function in such a way that their elimi- nation could leave gaps not filled by other oversight bodies (e.g., Institutional Review Boards, Institutional Animal Care and Use Committees, Institutional Biosafety Committees). It was stated that ESCRO committees could continue to be useful in maintaining deeper expertise on stem cell research than is nec- essarily provided by these other oversight bodies. ESCRO committees could also be helpful in assisting research institutions in monitoring developments in the field of stem cell research. In light of these comments, the Advisory Committee agrees that the continued use of ESCRO committees is useful, especially in circumstances where new hES cells are being derived. Even for research with existing cell lines funded by NIH—and therefore subject to NIH guidelines and the NIH hES cell registry—ESCRO committees could also help institutions by providing needed expertise and training for the members of their other committees. The stakeholders at the August 2009 meeting also discussed whether the National Academies should continue to play a role by maintaining an activity, such as a roundtable, that would allow periodic meetings to discuss knowledge and policy gaps, new problems, and contentious issues. It was suggested that, in the future, the uses of stem cells, as opposed to derivation of new lines, are likely to provide a larger share of any controversy or con- cern surrounding stem cell research. Stakeholders at the meeting suggested that the National Academies are viewed as providing a neutral setting for discussions that can help guide research institutions to make appropriate decisions about research, particularly in areas that are outside the bounds of NIH funding. Several guests stated that research using chimeras represents one such area of potential concern, but that other issues (e.g., stem cell- derived gametes) are also likely to emerge that may provoke controversy. Other topics identified as being potentially important in the future for stem 5 Other guidelines called for the establishment of Stem Cell Research Oversight (SCRO) committees whose mandate was not limited to embryonic stem cell research.

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2010 Amendments 7 cell research guidance included the relative merits of hES cells vs. induced pluripotent stem cells and clinical trials and translational research. Some of these topics may have little to do with the Guidelines themselves, but might make excellent topics for future workshops or studies. In light of these discussions, the Advisory Committee decided that: • he Human Embryonic Stem Cell Research Advisory Committee T should prepare this brief final report communicating to the stem cell research community those elements of the National Academies’ Guidelines that should remain in effect and under what conditions. • ollowing the completion of this task, the Advisory Committee F should disband. The Advisory Committee also discussed the feedback from stakeholders on future mechanisms for discussion of stem cell issues. Although govern- ment agencies such as the NIH, professional societies such as the ISSCR, consortia such as the Interstate Alliance on Stem Cell Research,6 and meet- ings organized by many different organizations and institutions provide opportunities for discussion, there does not seem to be an ongoing neutral forum for productive discussion of stem cell issues. Participants at the com- mittee’s August 2009 meeting mentioned that the National Academies and the Advisory Committee had served this important convening function over the last several years, and there was a need for a similar continuing activity. Perhaps most needed is a forum that could bring together key stakehold- ers—including federal, state, academic, patient, and industry organizations and institutions— for periodic meetings that would address topics of shared interest and concern to the broader stem cell research, regenerative medicine, and policy communities. 2010 AMENDMENTS TO THE NATIONAL ACADEMIES’ GUIDELINES FOR HUMAN EMBRYONIC STEM CELL RESEARCH Finally, the Advisory Committee presents here an amended version of the National Academies’ Guidelines (Appendix C) delineating those sections of the Guidelines that are superseded by the NIH rules for federally funded research. 6 The Interstate Alliance (IASCR) is a voluntary body of states and affiliate countries and organizations interested in increasing opportunities for interstate collaboration on stem cell research. See for more information.

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