Elsevier

Mayo Clinic Proceedings

Volume 78, Issue 8, August 2003, Pages 993-1003
Mayo Clinic Proceedings

Special Article: Stem Cell Research
An Overview of Stem Cell Research and Regulatory Issues

https://doi.org/10.4065/78.8.993Get rights and content

Stem cells are noted for their ability to self-renew and differentiate into a variety of cell types. Some stem cells, described as totipotent cells, have tremendous capacity to self-renew and differentiate. Embryonic stem cells have pluripotent capacity, able to form tissues of all 3 germ layers but unable to form an entire live being. Research with embryonic stem cells has enabled investigators to make substantial gains in developmental biology, therapeutic tissue engineering, and reproductive cloning. However, with these remarkable opportunities many ethical challenges arise, which are largely based on concerns for safety, efficacy, resource allocation, and methods of harvesting stem cells. Discussing the moral and legal status of the human embryo is critical to the debate on stem cell ethics. Religious perspectives and political events leading to regulation of stem cell research are presented and discussed, with special attention directed toward the use of embryonic stem cells for therapeutic and reproductive cloning. Adult stem cells were previously thought to have a restricted capacity to differentiate; however, several reports have described their plasticity potential. Furthermore, there have been close ties between the behavior of stem cells and cancer cells. True eradication of cancer will require a deeper understanding of stem cell biology. This article was written to inform medical scientists and practicing clinicians across the spectrum of medical education about the research and regulatory issues affecting the future of stem cell therapy.

Section snippets

EMBRYONIC STEM CELLS

Some stem cells have a greater capacity of self-renewal and multilineage differentiation than others (Table 1). At the time of conception, the fertilized egg (zygote) contains dividing cells (blastomeres) that form an embryo and placenta (Figure 1). These blastomeres are totipotent; they have the potential to form an entire living organism. After about 4 days, these totipotent cells begin to specialize and form into a hollow ball, the blastocyst, containing an outer shell (trophoectoderm) and a

ADULT STEM CELLS

It is generally accepted that each organ of our body is in balance between degradation and repair. The liver that we were born with is not the same liver that we have when we die. Throughout life, toxic insults wound our organs, bringing about the question of what keeps the balance between destruction and construction. In adults, stem cells have been found in many tissues, such as liver, bone marrow, pancreas, and brain, maintaining this homeostasis. Moreover, some of these adult stem cells,

LIVER

One of the first discoveries in end-organ repair and cell plasticity occurred in the liver. When hepatocytes are prevented from proliferating in response to liver damage, hepatic oval cells come to the rescue and produce more hepatocytes and bile duct cells. Interestingly, these oval cells, viewed as resident liver progenitor cells, share similar protein production with the HSC (namely, CD34, c-kit, Thy-1, and flt-3 receptor). This led Petersen et al37 to discover, through transplantation

PANCREAS

The liver and pancreas have close ties, going back to development in which both emerge from the same general area of the ventral foregut endoderm.65 The pancreatic progenitor cell, located within the ductal epithelia of the pancreas, has the capacity to differentiate into endocrine islets of Langerhans, including the glucagon-producing $aL cells, the insulin-producing ß cells, the pancreatic polypeptide-producing $gM cells, and the somatostatin-producing $dL cells.66 Given the close

KIDNEY

Unlike the liver and pancreas, the kidney progenitor cell has yet to be discovered. During development, the human kidney results from collision of the ureteric bud (lower urinary tract) and the metanephric mesenchyme (upper urinary tract). Precursors for the collecting tubules reside within the ureteric bud, whereas precursors for the rest of the kidney come from the metanephric mesenchyme. After development, in situations of injury, the kidney undergoes repair, with myofibroblast cells

HEART

Adult cardiac muscle regeneration is modest and largely restricted to viable myocardium.73 Several investigators have sought to enhance the heart regeneration challenge by injecting different cell sources into damaged myocardium and observing for engraftment. Cell sources such as cell lines, skeletal muscle myoblasts, and bone marrow cells have been used.74, 75 Interestingly, these cellular cardiomyoplasty approaches have identified the bone marrow as one of the richest founts of cardiac

BRAIN

Animal studies of the brain have found that bone marrow can differentiate into neurons and glial cells.84, 85, 86 Whether these transplanted cells are functional and impact cognitive function has yet to be determined; however, environmental influences such as ischemia, seizure, learning, and exercise can substantially stimulate hippocampal neuropoiesis.87, 88, 89, 90 In humans studies, we54 and others53 have found that bone marrow can make brain cells. Female patients undergoing therapeutic

BLOOD VESSELS

The close ties between blood and blood vessels have always been suspected given the recognition of a hemangioblast in the developing embryo. However, whether an adult hemangioblast worked double-duty, replenishing blood and repairing vessels, was debatable until 2002, when Grant et al38 formally proved that the adult HSC can function as a hemangioblast. Furthermore, these investigators were the first to show functional plasticity, with active perfusion of donor-derived vessels. Follow-up

STEM CELLS AND CANCER

In addition to their role in responding to physiologic repair, stem cells are implicated in certain cancers. This conviction is still being deliberated; however, lines of evidence seem compelling. Cancer cells have the ability to selfrenew much like stem cells. In human acute myelogenous leukemia studies, Bonnet and Dick99 have shown that the acute myelogenous leukemia stem cell could be purified from the CD34+CD38 fraction—the same fraction as the HSC. Solid tumors like lung cancer also have

Conventional Indication for HSC Transplantation

Mr S is a 36-year-old accountant who was in otherwise excellent health until increasing fatigue and left upper quadrant abdominal discomfort brought him to his family practitioner. Noting lethargy and splenomegaly, the physician ordered a complete blood cell count, results of which were an elevated white blood cell count of 81,000/$mUL (81.0 × 109/L), no basophilia, a hematocrit of 23%, and a platelet count of 12,000/$mUL (12.0 × 109/L). Bone marrow evaluation showed hypercellularity with

ETHICAL AND REGULATORY ISSUES IN STEM CELL RESEARCH

New discoveries in stem cell biology will soon bring revolutionary changes in the way physicians approach degenerative diseases, wound repair, autoimmune conditions, cancer, and reproductive medicine. Stem cells are self-renewing cells capable of producing many different cell types. Adult stem cells do well in repairing their organ of origin but have limited capabilities in self-renewal and distant organ repair under normal physiologic conditions. The degree of plasticity potential of the adult

CONCERNS REGARDING THE USE OF STEM CELLS

Certainly, stem cells are not the first human discovery to stretch the boundaries of medical knowledge and create waves of ethical debate. Since ancient times, society has admonished man for approaching these boundaries, eg, the Greek myth of Icarus who did not heed his father's command; he reveled in the “unnatural” sensation of flight and then plummeted to his death after the sun melted his wings. This Greek myth embodies our apprehensions about meddling with nature. Galileo Galilei, who

MORAL AND LEGAL STATUS OF THE HUMAN EMBRYO

Many religious perspectives consider the human fetus to constitute an individualized human entity. However, there is substantial debate regarding at which specific stage dignity is conferred in development (conception, primitive streak development, implantation, “quickening,” or birth).106, 107, 108, 109, 110, 111, 112 Recently, a less specific “developmental view” of moral status surfaced, meriting moral rights to the individual as consciousness and relationships develop.113, 114

Taking into

THERAPEUTIC AND REPRODUCTIVE CLONING

The embryonic stem cell also has tremendous potential in producing replicate tissues and genetically identical offspring. Cloning is accomplished by removing the chromosomes of an oocyte, inserting the nucleus of a donor adult (somatic) cell, and then stimulating the hybrid cell to divide as if it were a totipotent zygote. The cell is then prepared to produce any tissue of the body (therapeutic cloning) or if implanted in a female uterus to produce offspring with the same genetic material as

REFERENCES (122)

  • DF Sun et al.

    Possible involvement of myofibroblasts in cellular recovery of uranyl acetate-induced acute renal failure in rats

    Am J Pathol

    (2000)
  • NN Malouf et al.

    Adult-derived stem cells from the liver become myocytes in the heart in vivo

    Am J Pathol

    (2001)
  • C Stamm et al.

    Autologous bone-marrow stem-cell transplantation for myocardial regeneration

    Lancet

    (2003)
  • A Capela et al.

    LeX/ssea-1 is expressed by adult mouse CNS stem cells, identifying them as nonependymal

    Neuron

    (2002)
  • Q Shi et al.

    Evidence for circulating bone marrow-derived endothelial cells

    Blood

    (1998)
  • RD Owen

    Immunogenetic consequences of vascular anastomoses between bovine twins

    Science

    (1945)
  • JE Till et al.

    A direct measurement of the radiation sensitivity of normal mouse bone marrow cells

    Radiat Res

    (1961)
  • AJ Becker et al.

    Cytological demonstration of the clonal nature of spleen colonies derived from transplanted mouse marrow cells

    Nature

    (1963)
  • L Siminovitch et al.

    The distribution of colony-forming cells among spleen colonies

    J Cell Comp Physiol

    (1963)
  • AM Wu et al.

    Cytological evidence for a relationship between normal hemotopoietic colony-forming cells and cells of the lymphoid system

    J Exp Med

    (1968)
  • JA Thomson et al.

    Embryonic stem cell lines derived from human blastocysts [published correction appears in Science. 1998;282:1827]

    Science

    (1998)
  • JK Henderson et al.

    Preimplantation human embryos and embryonic stem cells show comparable expression of stage-specific embryonic antigens

    Stem Cells

    (2002)
  • MJ Shamblott et al.

    Derivation of pluripotent stem cells from cultured human primordial germ cells [published correction appears in Proc Natl Acad Sci U S A. 1999; 96;1162]

    Proc Natl Acad Sci U S A

    (1998)
  • MJ Shamblott et al.

    Human embryonic germ cell derivatives express a broad range of developmentally-distinct markers and proliferate extensively in vitro

    Proc Natl Acad Sci U S A

    (2001)
  • RH Xu et al.

    BMP4 initiates human embryonic stem cell differentiation to trophoblast

    Nat Biotechnol

    (2002)
  • C Xu et al.

    Characterization and enrichment of cardiomyocytes derived from human embryonic stem cells

    Circ Res

    (2002)
  • S Assady et al.

    Insulin production by human embryonic stem cells

    Diabetes

    (2001)
  • S Levenberg et al.

    Endothelial cells derived from human embryonic stem cells

    Proc Natl Acad Sci U S A

    (2002)
  • R Briggs et al.

    Transplantation of living nuclei from blastula cells into enucleated frogs' eggs

    Proc Natl Acad Sci U S A

    (1952)
  • CL Keefer et al.

    Bovine inner cell mass cells as donor nuclei in the production of nuclear transfer embryos and calves

    Biol Reprod

    (1994)
  • DN Wells et al.

    Production of cloned lambs from an established embryonic cell line: a comparison between in vivo- and in vitro-matured cytoplasts

    Biol Reprod

    (1997)
  • T Wakayama et al.

    Mice cloned from embryonic stem cells

    Proc Natl Acad Sci U S A

    (1999)
  • Scientists clone their first mule: horses could be next to join the clone parade of the animals. MSNBC News Web site

  • KH Campbell et al.

    Sheep cloned by nuclear transfer from a cultured cell line

    Nature

    (1996)
  • I Wilmut et al.

    Viable offspring derived from fetal and adult mammalian cells [published correction appears in Nature. 1997;386:200]

    Nature

    (1997)
  • JB Cibelli et al.

    Cloned transgenic calves produced from nonquiescent fetal fibroblasts

    Science

    (1998)
  • A Baguisi et al.

    Production of goats by somatic cell nuclear transfer

    Nat Biotechnol

    (1999)
  • CL Keefer et al.

    Production of cloned goats after nuclear transfer using adult somatic cells

    Biol Reprod

    (2002)
  • T Wakayama et al.

    Full-term development of mice from enucleated oocytes injected with cumulus cell nuclei

    Nature

    (1998)
  • J Betthauser et al.

    Production of cloned pigs from in vitro systems

    Nat Biotechnol

    (2000)
  • IA Polejaeva et al.

    Cloned pigs produced by nuclear transfer from adult somatic cells

    Nature

    (2000)
  • A Onishi et al.

    Pig cloning by microinjection of fetal fibroblast nuclei

    Science

    (2000)
  • PA De Sousa et al.

    Somatic cell nuclear transfer in the pig: control of pronuclear formation and integration with improved methods for activation and maintenance of pregnancy

    Biol Reprod

    (2002)
  • JR Hill et al.

    Evidence for placental abnormality as the major cause of mortality in first-trimester somatic cell cloned bovine fetuses

    Biol Reprod

    (2000)
  • PA De Sousa et al.

    Evaluation of gestational deficiencies in cloned sheep fetuses and placentae

    Biol Reprod

    (2001)
  • N Ogonuki et al.

    Early death of mice cloned from somatic cells

    Nat Genet

    (2002)
  • KL Tamashiro et al.

    Postnatal growth and behavioral development of mice cloned from adult cumulus cells

    Biol Reprod

    (2000)
  • RP Lanza et al.

    Extension of cell life-span and telomere length in animals cloned from senescent somatic cells

    Science

    (2000)
  • BE Petersen et al.

    Bone marrow as a potential source of hepatic oval cells

    Science

    (1999)
  • MB Grant et al.

    Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization

    Nat Med

    (2002)
  • Cited by (0)

    View full text