Findings and Recommendations
Human stem
cell research holds enormous potential for contributing to our understanding of
fundamental human biology. Although it
is not possible to predict the outcomes from basic research, such studies will
offer the real possibility for treatments and ultimately for cures for many
diseases for which adequate therapies do not exist.
The benefits
to individuals and to society gained by the introduction of new drugs or
medical technologies are difficult to estimate. The introductions of antibiotics and vaccines, for example, have
dramatically increased life spans and improved the health of people all over
the world. Despite these and other
advances in the prevention and treatment of human diseases, devastating
illnesses such as heart disease, diabetes, cancer, and diseases of the nervous
system such as Alzheimer’s disease present continuing challenges to the health
and well-being of people everywhere.
The science leading to the development of techniques for culturing human
stem cells could lead to unprecedented treatments and even cures for these and
other diseases.
As with all
research, our ability even to contemplate
the possibilities offered by stem cell-derived therapies is a result of many
years of research. The science of stem
cells dates to the mid-1960s, and many papers have been published on the
isolation and laboratory manipulation of stem cells from animal models. While these models are imperfect, they are
accepted in the scientific community as good initial predictors of what occurs
in human beings.
There already
exists evidence from animal studies that stem cells can be made to
differentiate into cells of choice, and that these cells will act properly in
their transplanted environment. In
human beings, transplants of hematopoietic stem cells (the cells which
eventually produce blood) following treatments for cancer, for example, have
been done for years now. Further,
somewhat cruder experiments (e.g., the transplantation of fetal tissue into the
brains of Parkinson’s patients) indicate that the expectation that stem cell
therapies could provide robust treatments for many human diseases is a reasonable
one. It is only through controlled
scientific research that the true promise will be understood.
Innovative research and new
technologies derived from such research almost always raise ethical and policy
concerns. In biomedical research, these
issues include the ethical conduct of basic and clinical research as well as
the equitable distribution of new therapies.
These issues are relevant to discussions
about stem cell research and its eventual applications; however, they are part
of a constellation of ethical and policy concerns associated with all advances
in biomedical research. Guidelines or
policies for the use of human biological materials have been issued at many
levels, from internal review boards to the National Bioethics Advisory
Commission, which recently released a detailed report on the use of such
materials. Existing policies cover all
aspects of research, from the use of cell lines in laboratories, to human
subjects protections, that will surface in the consideration of stem cell
research.
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It
is essential that there be a public that is educated and informed about the
ethical and policy issues raised by stem cell research and its applications. Informed public discussion of these issues
should be based on an understanding of the science associated with stem cell
research, and it should involve a broad cross-section of society.
It is essential for citizens to
participate in a full and informed manner in public policy deliberations about
the development and application of new technologies that are likely to have
significant social impact. The
understanding of the science is particularly important for discussing ethical
and policy issues. Ideally, scientists
should communicate the results of their research in ways that will be readily
understandable to a diverse audience, and participate in public discussions
related to stem cell research.
The ethical and policy issues
raised by stem cell research are not unique, but this research has received a
significant amount of public attention and there is much to gain by open
reflection on the implications of this sensitive area of research. Congressional hearings, public meetings by
government agencies, and media coverage have pushed stem cell research issues
into a spotlight. There should be
continued support for the open manner that has allowed all those interested to
observe or participate in these processes and for a sustained dialogue among
scientists, policy makers, ethicists, theologians, and the public to consider
issues that emerge with the advancement of stem cell research.
The appearance
of new technology can evoke apprehension and engender uncertainty among
segments of the population about its uses.
Where these concerns are related to issues having important ethical and
social implications, certain levels of oversight are appropriate. But it is
important to create new oversight mechanisms or regulatory burdens only when
there are compelling reasons for doing so.
Federal
funding would automatically trigger a set of oversight mechanisms now in place
to ensure that the conduct of biomedical research is consistent with broad
social values and legal requirements.
While basic laboratory research with personally non-identifiable stem
cells does not pose special ethical or oversight challenges, an elaborate
system of review is in place for research involving human subjects, ranging
from procurement issues to the conduct of clinical trials. The Federal Common Rule governing human
subjects research provides for local and federal agency review of research
proposals in such circumstances, weighing risks against benefits and requiring
involved and voluntary consent. The
Food and Drug Administration (FDA) has the authority to regulate the development
and use of human stem cells that will be used as biological products, drugs, or
medical devices to diagnose, treat or cure a disease or underlying condition.
Further, states should adopt the Federal Government’s
Model Program for the Certification of
Embryo Laboratories.
Complementing these
regulatory mechanisms are the National Bioethics Advisory Commission (NBAC),
which has demonstrated its legitimate claim to respect for its efforts as a
national body to promote public input into social policy related to advances in
biomedical research, and the Recombinant DNA Advisory Committee (RAC), which
currently has a mandate to review the ethical and policy issues associated with
gene therapy and could be authorized to change its mission to broaden its
purview. These federal bodies should
work with interested stakeholders in the conduct of stem cell
research - professional organizations, patient disease groups, religious
communities, the Congress, funding agencies and private foundations, industry,
and others - so that the public can be assured that appropriate safeguards are in
place as this research evolves.
Thus, at the
present time, no new regulatory mechanisms are needed to ensure responsible
social and professional control of stem cell research in the United States.
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Federal
funding for stem cell research is necessary in order to promote investment in
this promising line of research, to encourage sound public policy, and to
foster public confidence in the conduct of such research.
Realizing the
potential health benefits of stem cell technology will require a large and
sustained investment in research. The
federal government is the only realistic source for such an infusion of
funds. For those who are challenged
daily by serious diseases that could in the future be relieved by therapies
gained through stem cell research, public funding holds the greatest promise
for sooner rather than later research results that can be transferred from the
bench to the bedside. Without the stimulus
of public funding, new treatments could be substantially delayed.
The commitment of federal funds
also offers a basis for public review, approval, and monitoring through well
established oversight mechanisms that will promote the public’s interest in
ensuring that stem cell research is conducted in a way that is both
scientifically rigorous and ethically proper.
Additionally, public funding contributes to sound social policy by
increasing the probability that the results of stem cell research will reflect
broad social priorities that are unlikely to be considered if the research is
carried out in the private sector alone.
There are segments of American
society that disagree on moral grounds with using public monies to support
certain types of stem cell research.
However, public policy in a pluralistic society cannot resolve all the
differences that arise in national debates on sensitive social issues. In the context of stem cell research, this
leads to three practical conclusions.
One is a willingness to permit individuals, whether they are researchers
or embryo or fetal tissue donors, to act in conformity with their own moral
views on these matters. A second is the
commitment to public involvement in research support when this research is
related to the promotion and protection of public health, including the acquisition of new molecular and
cellular insights into basic human developmental biology. A third is respect for opposing views,
especially those based on religious grounds, to the extent that this is
consistent with the protection and promotion of public health and safety.
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Public
and private research on human stem cells derived from all sources (embryonic,
fetal, and adult) should be conducted in order to contribute to the rapidly
advancing and changing scientific understanding of the potential of human stem
cells from these various sources.
There are
three primary sources of stem cells, each with different characteristics as to
how many different developmental paths they can follow and how much they can
contribute to our understanding of a functioning organism. Embryonic stem cells (ES cells), derived
from a very early embryo, and embryonic germ cells (EG cells), collected from
fetal tissue at a somewhat later stage of development, have particular promise
for a wide range of therapeutic applications because, according to our present
knowledge, they are capable of giving rise to virtually any cell type. Research on these primordial cells will also
provide a unique opportunity to study human cell biology.
Adult stem cells, obtained from
mature tissues, differentiate into a narrower range of cell types. As a result, many cells of medical interest
cannot currently be obtained from adult-derived stem cells. It is also less feasible to develop
large-scale cultures from adult stem cells. However, it is important to note that, at this time, it is only
adult human stem cells that are well-enough understood that they can be
reliably differentiated into specific tissue types, and that have proceeded to
clinical trials.
Because the study of human stem
cells is at an early stage of development, it is difficult to predict outcomes
and findings at this point in time. As
more research takes place, the full developmental potential of different kinds
of stem cells will become better understood.
In view of the moral concerns
surrounding the uses of embryonic and fetal tissue voiced by a segment of the
American population, strengthening federally and privately funded research into
alternative sources and/or methods for the derivation of stem cells, including
further initiatives on adult stem cells, should be encouraged. Human
stem cell research can be conducted in a fully ethical manner, but it is true
that the extraction of embryonic stem cells from the inner mass of blastocysts
raises ethical questions for those who consider the intentional loss of
embryonic life by intentional means to be morally wrong. Likewise, the derivation of embryonic germ
cells from the gonadal tissue of aborted fetuses is problematic for those who
oppose abortion. In contrast, adult
stem cell research is more broadly acceptable to the American population.
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Public
funding should be provided for embryonic stem cell and embryonic germ cell
research, but not at this time for activities involved in the isolation of
embryonic stem cells, about which there remains continuing debate. This approach will allow publicly-funded
researchers to move more quickly toward discoveries that will lead to
alleviating the suffering caused by human disease.
Although the derivation of human stem cells can be done in an ethical
manner, there is enough objection to the process of deriving stem cells to
consider recommending against its public funding. Further, for the foreseeable future there will be sufficient
material isolated by researchers not using public funding that this exclusion
will not have a negative impact on research.
There are many individuals who believe that any use of human embryos
other than for achieving a pregnancy is unethical, believing that the embryo is
a full human being from the earliest moments in the conception process. However, many religious traditions take a
“developmental” view of personhood, believing that the early embryo or fetus
only gradually becomes a full human being and thus may not be entitled to the
same moral protections as it will later; others hold that while the embryo
represents human life, that life may be taken for the sake of saving and
preserving other lives in the future.
The dialogue about these issues is ongoing in the United States, but
these concerns need not exclude publicly-funded research activities on cell
lines that have already been established.
The most
ethical source of human primordial stem cells is embryos produced for the process of in vitro fertilization
whose progenitors have decided not to implant them and have given full and
informed consent for the use of these embryos for research purposes. Two appropriate potential sources of
donation are embryos with poor quality that makes them inappropriate for
transfer and embryos remaining when couples have definitely completed their
family and do not wish to donate the excess embryos to others.
Informed
consent requires that the woman or couple, with substantial understanding and
without controlling influences, authorize the use of their spare embryos for
research purposes. Because assisted
reproduction can be a stressful process, informed consent should be secured in
two stages. The two-stage process would
also maintain a separation between personnel working with the woman or couple
who hope to get pregnant and personnel requesting embryos for stem cell
research.
At the
beginning of the process, personnel working with the woman or couple who hope
to become pregnant should ascertain their preferences as to the future of
embryos remaining after the assisted reproduction process. These options should include consent for
embryo donation to another couple, consent for donation for research, and
consent for destruction of the spare embryos.
Once a couple has definitely decided that it has completed its family,
then the couple should be approached a second time to secure an explicit
consent to use the embryos in ES cell research.
Securing
embryos for the purpose of harvesting stem
cells must proceed in a careful fashion for several reasons. These are to protect the interests of the
gamete donors, to reassure the public that important boundaries are not being
overstepped, to enable those who are ethically uncomfortable with elements of
this research to participate to the greatest extent possible, and to ensure the
highest quality of research and outcomes possible.
Consonant with
good research practice, policies on the procurement of embryos should include
at least the following points: (1) Women should not undergo extra cycles of
ovulation and retrieval in order to produce more “spare” embryos in the hope
that some of them might eventually be donated for research;
(2) Analogous
with our current practice for organ donation, there should be a solid “wall”
between personnel working with the woman or couple who hope to get pregnant,
and personnel requesting embryos for stem cell purposes; (3) Women and men, as
individuals or as couples, should not be paid to produce embryos, nor should
they receive reduced fees for their infertility procedures for doing so; and
(4) Consent of both gamete donors should be obtained.
At present,
stem cell research raises no unique ethical or policy issues. As research advances issues may emerge that
challenge acceptable ethical practices and public policy. Hence, there should be opportunities for
public reconsideration of the need for guidelines specifically targeted to
human stem cell research. Such efforts
should be informed by the most current scientific evidence and should occur
through a process that encourages broad involvement by all sectors of society.
Almost two decades of
experience with the Recombinant DNA Advisory Committee’s (RAC) oversight of
recombinant DNA research suggest that the RAC could be an effective
institutional focal point within the federal government to facilitate the type
of public dialogue on stem cell research proposed here, and to coordinate
efforts to develop new guidelines, where needed. The RAC has a proven track record of providing an open forum for
sorting out complex ethical issues and of defusing conflict. Furthermore, it has acquired a degree of
legitimacy among scientists in both the public and private sectors, with its
widely accepted Points to Consider
in the design and conduct of gene therapy.
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In
order to allow persons who hold diverse moral positions on the status of the
early embryo to participate in stem cell research to the greatest degree
possible without compromising their principles, and also to foster sound
science, stem cells (and stem cell lines) should be identified with respect to
their original source.
Patients and researchers should
be able to avoid participating in stem cell use if the cells were derived in a
way that they would consider to be unethical.
As a matter of good scientific practice, records are routinely
maintained on the sources of biological materials. It is of utmost importance that documentation of the original
source of the stem cells can be made readily available to researchers and to
potential recipients of stem cell therapies.
The
therapeutic potential for treating and possibly curing many serious diseases
constitutes a major rationale for large-scale investments of public and private
resources in human stem cell research.
To justify funding stem cell research on the basis of its potential
benefits, particularly the use of public resources, however, requires some
assurance that people in need will have access to the therapies as they become
available.
Several factors make it
unlikely that there will be equitable access to the benefits of this
research. Unlike other western
democracies, the United States does not have a commitment to universal health
care. More than 44 million people lack health
insurance and therefore do not have reliable access even to basic health care. Others are underinsured. Moreover, if stem cell research were to
result in highly technological and expensive therapies, health insurers might
be reluctant to fund such treatments.
Overcoming these hurdles and
assuring equitable access to the benefits of stem cell research in this country
will be a politically and financially challenging task. It is therefore appropriate to begin
considering how to do so now in advance of the development of applications. The federal government should consider ways
to achieve equitable access to the benefits derived from stem cell research.
The U.S.
Patent and Trademark Office (PTO) has already stated that purified and isolated
stem cell products and research tools meet the criteria for patentable subject
matter. When research is funded by the
private sector, as is currently the case with stem cell research, and is patented,
it is a private matter whether and under what terms new intellectual property
is obtainable for research purposes or development. This is of particular concern because the private sector will not
invest resources in potential applications that they consider to lack
commercial value, but that may have considerable therapeutic promise.
Given the promise of stem cell
research, it is important to encourage the development of broadly beneficial
therapeutic products with widespread access.
This objective could be achieved in a variety of ways. Government investment in promising areas of
research would enable federal agencies and laboratories to hold patents and to exercise them in ways that enhance
development and contribute to the dissemination of this stem cell
technology. Congress or the PTO should define a strong research exemption that
would give third parties access to stem cell products and research tools for
research purposes without having to obtain permission from the patent
holder. Another possibility is to
require compulsory licensing under limited and clearly defined circumstances.
Private sector
research has played a crucial part in the advancement of research on stem
cells. The leadership exhibited by the
company that has sponsored all of the published human embryonic and germ cell
research to date in establishing an external Ethics Advisory Board to develop
guidelines for the ethical conduct of such research is laudable. While these private sector boards are not a
substitute for public oversight and guidance, they can be a positive influence
on the way that industry-funded stem cell research proceeds.
The
credibility and impact of such ethics advisory boards will be enhanced if they
review ethical issues at the start-up phase of the research, have
multidisciplinary membership, including representatives from the local
community, give minimum, if any, financial compensation for service, and share
their own findings and recommendations with other companies. The latter provision could be especially
helpful in developing a “case law” in the private sphere that would inform
public efforts to develop national guidelines.
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