J Korean Med Assoc.  2011 May;54(5):454-461. 10.5124/jkma.2011.54.5.454.

Clinical application of human embryonic stem cells

• Chung HM

• KMID: 2190701
• DOI: http://doi.org/10.5124/jkma.2011.54.5.454

Abstract

Recent advances in stem cell biology, including the development of optimized cell type-specific culture systems, and the broader understandings of biochemical and molecular signals involved in cell self-renewal and differentiation have brought cell-based therapy closer to practical application. As of now, at least 250 adult stem cell therapies are being used or tested in clinical situations. Stem cells have two important properties that distinguish them from other types of cells; they can both proliferate without changing their phenotypes indefinitely, and they also can differentiate into one or more new kinds of cells depending on their culture conditions. Thus, stem cell therapy could be most effective for treating the diseases that are marked by the loss of cells. The typical examples are Parkinson's disease, Alzheimer's disease, diabetes, heart failure, blindness, spinal cord injury, and stroke. Additionally, stem cell derivatives can be used in drug discovery as well. In the last decade, various types of stem cells have been identified from preimplantation stage embryos, fetuses, placentas, and adult tissues. Moreover, it is now almost a common practice to produce induced pluripotent stem (iPS) cells from various adult somatic cells using only a few defined factors. Thus, it is feasible that patient-specific stem cells will be generated with less controversy in the near future. However, human embryonic stem (ES) cells firmly remain "the gold standard" because of their greatest potential to become any type of cell in the body. The vast knowledge obtained from human ES cell research in the past decade has made cell-based therapy more promising than ever. Even the recent establishment of iPS cell technology is the culmination of human ES cells research. In our laboratory, interesting human cardiovascular cells including endothelial precursor cells and beating myocardiac cells, artificial blood cells, and retinal pigment epithelial cells were successfully differentiated and their therapeutic potential was confirmed after cell transplantation into animal models. Thus, here, the current research status of human embryonic stem cell-based therapy will be introduced and the future directions of stem cell applications in clinical trials will be discussed.

Keyword

Human embryonic stem cells; Cell therapy product; Differentiation

MeSH Terms

Adult
Adult Stem Cells
Alzheimer Disease
Artificial Cells
Biology
Blindness
Blood Cells
Cell Transplantation
Drug Discovery
Embryonic Stem Cells
Embryonic Structures
Epithelial Cells
Fetus
Heart Failure
Humans
Induced Pluripotent Stem Cells
Models, Animal
Parkinson Disease
Phenotype
Placenta
Retinaldehyde
Spinal Cord Injuries
Stem Cells
Stroke
Transplants
Retinaldehyde

Figure

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  Figure 1 HLA typing of 27 CHA-human embryonic stem cell (hESC) lines vs. 6,740 donated cord bloods for simulation of stem cell transplantation.

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  Figure 2 Geron initiates clinical trial of human embryonic stem cell-based therapy (courtesy of Geron Inc.). IND, industrialization of new drug.

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  Figure 3 Generation of retinal pigment epithelial cells (RPEs) from human embryonic stem cells. WCB, working cell bank; hESC, human embryonic stem cell; EB, embryoid body.

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  Figure 4 Morphological and immunohistochemical characterizations of human embryonic stem cell (hESC) derived retinal pigment epithelial cells (RPEs). Upper panel: In vitro differentiated human RPEs from hESCs (×100). Lower panel: molecular marker analysis of RPE from hESCs. Immunos-taining with antibodies of human RPE cells, tight junction protein (ZO-1), microphthalmia associated transcription fator (MITF), Pax-6, and Bes-trophin (×200).

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  Figure 5 Human embryonic stem (hES) cell derived retinal pigment epithelial cells (RPEs) rescue visual function in dystrophic the Royal College of Surgeons rats. ERG, elect-roretinogram; INL, inner nuclear layer; ONL, outer nuclear layer.

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  Figure 6 Optima dose finding results in human embryonic stem cellsderived retinal pigment epithelial cells in dystrophic Royal College of Surgeons rats.

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