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AAAS/ICS Report on
Stem Cell Research available online.
 Click here to download.
Produced by the American Association for the Advancement
of Science and Institute for Civil Society, November 1999
· 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.
· This research raises ethical and policy concerns,
but these are not unique to stem cell research.
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.
· 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.
· Existing federal regulatory and professional control
mechanisms, combined with informed public dialogue, provide a sufficient
framework for oversight of human 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.
· 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.
· 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.
· 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.
· Embryonic stem cells should be obtained from embryos
remaining from infertility procedures after the embryo's progenitors
have made a decision that they do not wish to preserve them. This
decision should be explicitly renewed prior to securing the progenitors'
consent to use the embryos in ES cell research.
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.
· Persons considering donating their excess embryos
for research purposes should be afforded the highest standards of
protection for the informed consent and voluntariness of their decision.
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.
· Where appropriate, guidelines that can attract
professional and public support for conducting stem cell research
should be developed.
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.
· 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.
· Special efforts should be made to promote equitable
access to the benefits of stem cell research.
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.
· Intellectual property regimes for stem cell research
should set conditions that do not restrict basic research or encumber
future product development.
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.
· The formation of company-based, independent ethics
advisory boards should be encouraged in the private sector.
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.
The complete report of the AAAS/ICS study is available
here in PDF format. We anticipate the availability
of a print version in the near future.
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