The application of human embryonic stem cell technologies to drug discovery

Drug Discovery Today

Volumn 12, Issue 17-18, 2007, Pages 688-699

  Sartipy, Peter ^a   Björquist, Petter ^a   Strehl, Raimund ^a   Hyllner, Johan ^a  

^a Cellartis AB   (Sweden)

Author keywords

[No Author keywords available]

Indexed keywords

MOLECULAR MARKER; NEW DRUG;

ACCURACY; BIOMEDICINE; CELL DIFFERENTIATION; CELL ISOLATION; CELL LINE; CELL STRUCTURE; CELL TYPE; COST EFFECTIVENESS ANALYSIS; DRUG INDUSTRY; DRUG MANUFACTURE; DRUG RESEARCH; DRUG SAFETY; EMBRYONIC STEM CELL; ETHICS; GENE EXPRESSION; GENETIC ENGINEERING; HEART MUSCLE CELL; HUMAN; IN VITRO STUDY; LIVER CELL; MOLECULAR MECHANICS; NONHUMAN; PLURIPOTENT STEM CELL; REGENERATIVE MEDICINE; RESEARCH; REVIEW; RISK ASSESSMENT; TECHNOLOGY;

BIOMEDICAL RESEARCH; CELL CULTURE TECHNIQUES; CELL DIFFERENTIATION; DRUG DESIGN; DRUG EVALUATION, PRECLINICAL; EMBRYONIC STEM CELLS; HEPATOCYTES; HUMANS; MYOCYTES, CARDIAC; PHARMACOLOGY; PLURIPOTENT STEM CELLS; TOXICITY TESTS;

EID: 34548386096     PISSN: 13596446     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.drudis.2007.07.005     Document Type: Review

References (116)

1. Dimasi J.A. Risks in new drug development: approval success rates for investigational drugs. Clin. Pharmacol. Ther. 69 (2001) 297-307
2. Pearson H. The bitterest pill. Nature 444 (2006) 532-533
3. Kola I., and Landis J. Can the pharmaceutical industry reduce attrition rates?. Nat. Rev. Drug Discov. 3 (2004) 711-715
4. Friedrich Ben-Nun I., and Benvenisty N. Human embryonic stem cells as a cellular model for human disorders. Mol. Cell Endocrinol. 252 (2006) 154-159
5. Vats A., et al. Stem cells. Lancet 366 (2005) 592-602
6. Scadden D.T. The stem-cell niche as an entity of action. Nature 441 (2006) 1075-1079
7. Ding S., and Schultz P.G. A role for chemistry in stem cell biology. Nat. Biotechnol. 22 (2004) 833-840
8. Cai J., et al. In search of "stemness". Exp. Hematol. 32 (2004) 585-598
9. Jiang Y., et al. Pluripotency of mesenchymal stem cells derived from adult marrow. Nature 418 (2002) 41-49
10. Broxmeyer H.E., et al. High-efficiency recovery of functional hematopoietic progenitor and stem cells from human cord blood cryopreserved for 15 years. Proc. Natl. Acad. Sci. U. S. A. 100 (2003) 645-650
11. McNeish J. Embryonic stem cells in drug discovery. Nat. Rev. Drug Discov. 3 (2004) 70-80
12. Thomson J.A., et al. Embryonic stem cell lines derived from human blastocysts. Science 282 (1998) 1145-1147
13. Zambrowicz B.P., and Sands A.T. Knockouts model the 100 best-selling drugs - will they model the next 100?. Nat. Rev. Drug Discov. 2 (2003) 38-51
14. Guhr A., et al. Current state of human embryonic stem cell research: an overview of cell lines and their use in experimental work. Stem Cells 24 (2006) 2187-2191
15. Martin G.R. Isolation of a pluripotent cell line from early mouse embryos cultured in medium conditioned by teratocarcinoma stem cells. Proc. Natl. Acad. Sci. U. S. A. 78 (1981) 7634-7638
16. Evans M.J., and Kaufman M.H. Establishment in culture of pluripotential cells from mouse embryos. Nature 292 (1981) 154-156
17. Bongso A., et al. Isolation and culture of inner cell mass cells from human blastocysts. Hum. Reprod. 9 (1994) 2110-2117
18. Thomson J.A., et al. Isolation of a primate embryonic stem cell line. Proc. Natl. Acad. Sci. U. S. A. 92 (1995) 7844-7848
19. Heins N., et al. Derivation, characterization, and differentiation of human embryonic stem cells. Stem Cells 22 (2004) 367-376
20. Amit M., and Itskovitz-Eldor J. Derivation and spontaneous differentiation of human embryonic stem cells. J. Anat. 200 Pt 3 (2002) 225-232
21. Reubinoff B.E., et al. Embryonic stem cell lines from human blastocysts: somatic differentiation in vitro. Nat. Biotechnol. 18 (2000) 399-404
22. Stojkovic M., et al. Derivation of human embryonic stem cells from day-8 blastocysts recovered after three-step in vitro culture. Stem Cells 22 (2004) 790-797
23. Baharvand H., et al. Establishment and in vitro differentiation of a new embryonic stem cell line from human blastocyst. Differentiation 72 (2004) 224-229
24. Kim H.S., et al. Methods for derivation of human embryonic stem cells. Stem Cells 23 (2005) 1228-1233
25. Xu R.H., et al. BMP4 initiates human embryonic stem cell differentiation to trophoblast. Nat. Biotechnol. 20 (2002) 1261-1264
26. Draper J.S., et al. Culture and characterization of human embryonic stem cells. Stem. Cells Dev. 13 (2004) 325-336
27. Pesce M., and Scholer H.R. Oct-4: gatekeeper in the beginnings of mammalian development. Stem Cells 19 (2001) 271-278
28. Chambers I., et al. Functional expression cloning of Nanog, a pluripotency sustaining factor in embryonic stem cells. Cell 113 (2003) 643-655
29. Bhattacharya B., et al. Comparison of the gene expression profile of undifferentiated human embryonic stem cell lines and differentiating embryoid bodies. BMC Dev. Biol. 5 (2005) 22
30. Heins N., et al. Clonal derivation and characterization of human embryonic stem cell lines. J. Biotechnol. 122 (2006) 511-520
31. Pera M.F., and Trounson A.O. Human embryonic stem cells: prospects for development. Development 131 (2004) 5515-5525
32. Lim J.W., and Bodnar A. Proteome analysis of conditioned medium from mouse embryonic fibroblast feeder layers which support the growth of human embryonic stem cells. Proteomics 2 (2002) 1187-1203
33. Xu C., et al. Feeder-free growth of undifferentiated human embryonic stem cells. Nat. Biotechnol. 19 (2001) 971-974
34. Cowan C.A., et al. Derivation of embryonic stem-cell lines from human blastocysts. N. Engl. J. Med. 350 (2004) 1353-1356
35. Richards M., et al. Human feeders support prolonged undifferentiated growth of human inner cell masses and embryonic stem cells. Nat. Biotechnol. 20 (2002) 933-936
36. Ellerstrom C., et al. Derivation of a xeno-free human embryonic stem cell line. Stem. Cells 24 (2006) 2170-2176
37. Watanabe K., et al. A ROCK inhibitor permits survival of dissociated human embryonic stem cells. Nat. Biotechnol. 25 (2007) 681-686
38. Mitalipova M.M., et al. Preserving the genetic integrity of human embryonic stem cells. Nat. Biotechnol. 23 (2005) 19-20
39. Sato N., et al. Maintenance of pluripotency in human and mouse embryonic stem cells through activation of Wnt signaling by a pharmacological GSK-3-specific inhibitor. Nat. Med. 10 (2004) 55-63
40. Amit M., et al. Human feeder layers for human embryonic stem cells. Biol. Reprod. 68 (2003) 2150-2156
41. Wang G., et al. Noggin and bFGF cooperate to maintain the pluripotency of human embryonic stem cells in the absence of feeder layers. Biochem. Biophys. Res. Commun. 330 (2005) 934-942
42. Levenstein M.E., et al. Basic fibroblast growth factor support of human embryonic stem cell self-renewal. Stem Cells 24 (2006) 568-574
43. Yao S., et al. Long-term self-renewal and directed differentiation of human embryonic stem cells in chemically defined conditions. Proc. Natl. Acad. Sci. U. S. A. 103 (2006) 6907-6912
44. Ludwig T.E., et al. Feeder-independent culture of human embryonic stem cells. Nat. Methods 3 (2006) 637-646
45. Andrews P.W., et al. The International Stem Cell Initiative: toward benchmarks for human embryonic stem cell research. Nat. Biotechnol. 23 (2005) 795-797
46. Thomson H. Bioprocessing of embryonic stem cells for drug discovery. Trends Biotechnol. 25 (2007) 224-230
47. Terstegge S., et al. Automated maintenance of embryonic stem cell cultures. Biotechnol. Bioeng. 96 (2007) 195-201
48. Loring J.F., and Rao M.S. Establishing standards for the characterization of human embryonic stem cell lines. Stem Cells 24 (2006) 145-150
49. Zeng X., et al. An in vitro model of human dopaminergic neurons derived from embryonic stem cells: MPP+ toxicity and GDNF neuroprotection. Neuropsychopharmacology 31 (2006) 2708-2715
50. Pellizzer C., et al. Developmental toxicity testing from animal towards embryonic stem cells. Altex 22 (2005) 47-57
51. Nussler A., et al. Present status and perspectives of cell-based therapies for liver diseases. J. Hepatol. 45 (2006) 144-159
52. Rodriguez-Antona C., et al. Cytochrome P450 expression in human hepatocytes and hepatoma cell lines: molecular mechanisms that determine lower expression in cultured cells. Xenobiotica 32 (2002) 505-520
53. Wilkening S., et al. Comparison of primary human hepatocytes and hepatoma cell line Hepg2 with regard to their biotransformation properties. Drug Metab Dispos. 31 (2003) 1035-1042
54. Zaret K.S. Regulatory phases of early liver development: paradigms of organogenesis. Nat. Rev. Genet. 3 (2002) 499-512
55. Suzuki A., et al. Clonal identification and characterization of self-renewing pluripotent stem cells in the developing liver. J. Cell Biol. 156 (2002) 173-184
56. Schwartz R.E., et al. Multipotent adult progenitor cells from bone marrow differentiate into functional hepatocyte-like cells. J. Clin. Invest 109 (2002) 1291-1302
57. Hamazaki T., et al. Hepatic maturation in differentiating embryonic stem cells in vitro. FEBS Lett. 497 (2001) 15-19
58. Soto-Gutierrez A., et al. Reversal of mouse hepatic failure using an implanted liver-assist device containing ES cell-derived hepatocytes. Nat. Biotechnol. 24 (2006) 1412-1419
59. Gouon-Evans V., et al. BMP-4 is required for hepatic specification of mouse embryonic stem cell-derived definitive endoderm. Nat. Biotechnol. 24 (2006) 1402-1411
60. Schuldiner M., et al. Effects of eight growth factors on the differentiation of cells derived from human embryonic stem cells. Proc. Natl. Acad. Sci. U. S. A. 97 (2000) 11307-11312
61. Rambhatla L., et al. Generation of hepatocyte-like cells from human embryonic stem cells. Cell Transpl. 12 (2003) 1-11
62. Lavon N., et al. Differentiation and isolation of hepatic-like cells from human embryonic stem cells. Differentiation 72 (2004) 230-238
63. Schwartz R.E., et al. Defined conditions for development of functional hepatic cells from human embryonic stem cells. Stem Cells Dev. 14 (2005) 643-655
64. Baharvand H., et al. Differentiation of human embryonic stem cells into hepatocytes in 2D and 3D culture systems in vitro. Int. J. Dev. Biol. 50 (2006) 645-652
65. Hay D.C., et al. Direct differentiation of human embryonic stem cells to hepatocyte-like cells exhibiting functional activities. Cloning Stem Cells 9 (2007) 51-62
66. Soto-Gutierrez A., et al. Differentiation of human embryonic stem cells to hepatocytes using deleted variant of HGF and poly-amino-urethane-coated nonwoven polytetrafluoroethylene fabric. Cell Transpl. 15 (2006) 335-341
67. Soderdahl T., et al. Glutathione transferases in hepatocyte-like cells derived from human embryonic stem cells. Toxicol. In Vitro 21 (2007) 929-937
68. D'Amour K.A., et al. Efficient differentiation of human embryonic stem cells to definitive endoderm. Nat. Biotechnol. 23 (2005) 1534-1541
69. Cai J., et al. Directed differentiation of human embryonic stem cells into functional hepatic cells. Hepatology 45 (2007) 1229-1239
70. Lazarou J., et al. Incidence of adverse drug reactions in hospitalized patients: a meta-analysis of prospective studies. JAMA 279 (1998) 1200-1205
71. Masimirembwa C.M., et al. In vitro high throughput screening of compounds for favorable metabolic properties in drug discovery. Comb. Chem. High Throughput Screen 4 (2001) 245-263
72. Cross D.M., and Bayliss M.K. A commentary on the use of hepatocytes in drug metabolism studies during drug discovery and development. Drug Metab Rev. 32 (2000) 219-240
73. Rendic S., and Di Carlo F.J. Human cytochrome P450 enzymes: a status report summarizing their reactions, substrates, inducers, and inhibitors. Drug Metab. Rev. 29 (1997) 413-580
74. Chandra P., and Brouwer K.L. The complexities of hepatic drug transport: current knowledge and emerging concepts. Pharm. Res. 21 (2004) 719-735
75. Kulkarni J.S., and Khanna A. Functional hepatocyte-like cells derived from mouse embryonic stem cells: a novel in vitro hepatotoxicity model for drug screening. Toxicol. In Vitro 20 (2006) 1014-1022
76. Olson E.N. A decade of discoveries in cardiac biology. Nat. Med. 10 (2004) 467-474
77. Doetschman T.C., et al. The in vitro development of blastocyst-derived embryonic stem cell lines: formation of visceral yolk sac, blood islands and myocardium. J. Embryol. Exp. Morphol. 87 (1985) 27-45
78. Goh G., et al. Molecular and phenotypic analyses of human embryonic stem cell-derived cardiomyocytes: opportunities and challenges for clinical translation. Thromb Haemost. 94 (2005) 728-737
79. Kehat I., et al. Human embryonic stem cells can differentiate into myocytes with structural and functional properties of cardiomyocytes. J. Clin. Invest 108 (2001) 407-414
80. Passier R., et al. Increased cardiomyocyte differentiation from human embryonic stem cells in serum-free cultures. Stem Cells 23 (2005) 772-780
81. Yoon B.S., et al. Enhanced differentiation of human embryonic stem cells into cardiomyocytes by combining hanging drop culture and 5-azacytidine treatment. Differentiation 74 (2006) 149-159
82. Snir M., et al. Assessment of the ultrastructural and proliferative properties of human embryonic stem cell-derived cardiomyocytes. Am. J. Physiol. Heart Circ. Physiol. 285 (2003) H2355-H2363
83. Norstrom A., et al. Molecular and pharmacological properties of human embryonic stem cell-derived cardiomyocytes. Exp. Biol. Med. (Maywood) 231 (2006) 1753-1762
84. Xu C., et al. Characterization and enrichment of cardiomyocytes derived from human embryonic stem cells. Circ. Res. 91 (2002) 501-508
85. Mummery C., et al. Differentiation of human embryonic stem cells to cardiomyocytes: role of coculture with visceral endoderm-like cells. Circulation 107 (2003) 2733-2740
86. He J.Q., et al. Human embryonic stem cells develop into multiple types of cardiac myocytes: action potential characterization. Circ. Res. 93 (2003) 32-39
87. Xue T., et al. Functional integration of electrically active cardiac derivatives from genetically engineered human embryonic stem cells with quiescent recipient ventricular cardiomyocytes: insights into the development of cell-based pacemakers. Circulation 111 (2005) 11-20
88. Poueymirou W.T., et al. F0 generation mice fully derived from gene-targeted embryonic stem cells allowing immediate phenotypic analyses. Nat. Biotechnol. 25 (2007) 91-99
89. Shah R.R. The significance of QT interval in drug development. Br. J. Clin. Pharmacol. 54 (2002) 188-202
90. Meyer T., et al. Micro-electrode arrays in cardiac safety pharmacology: a novel tool to study QT interval prolongation. Drug Saf. 27 (2004) 763-772
91. Reppel M., et al. The electrocardiogram of human embryonic stem cell-derived cardiomyocytes. J. Electrocardiol. 38 4 Suppl (2005) 166-170
92. Muzikant A.L., and Penland R.C. Models for profiling the potential QT prolongation risk of drugs. Curr. Opin. Drug Discov. Dev. 5 (2002) 127-135
93. Schuster D., et al. Why drugs fail--a study on side effects in new chemical entities. Curr. Pharm. Des. 11 (2005) 3545-3559
94. Wang Z.Z., et al. Endothelial cells derived from human embryonic stem cells form durable blood vessels in vivo. Nat. Biotechnol. 25 (2007) 317-318
95. Huang H., et al. Differentiation of human embryonic stem cells into smooth muscle cells in adherent monolayer culture. Biochem. Biophys. Res. Commun. 351 (2006) 321-327
96. D'Amour K.A., et al. Production of pancreatic hormone-expressing endocrine cells from human embryonic stem cells. Nat. Biotechnol. 24 (2006) 1392-1401
97. Green H., et al. Marker succession during the development of keratinocytes from cultured human embryonic stem cells. Proc. Natl. Acad. Sci. U. S. A. 100 (2003) 15625-15630
98. Nistor G.I., et al. Human embryonic stem cells differentiate into oligodendrocytes in high purity and myelinate after spinal cord transplantation. Glia 49 (2005) 385-396
99. Zhang S.C., et al. In vitro differentiation of transplantable neural precursors from human embryonic stem cells. Nat. Biotechnol. 19 (2001) 1129-1133
100. Nat R., et al. Neurogenic neuroepithelial and radial glial cells generated from six human embryonic stem cell lines in serum-free suspension and adherent cultures. Glia 55 (2007) 385-399
101. Kee K., et al. Bone morphogenetic proteins induce germ cell differentiation from human embryonic stem cells. Stem Cells Dev. 15 (2006) 831-837
102. Xiong C., et al. Derivation of adipocytes from human embryonic stem cells. Stem Cells Dev. 14 (2005) 671-675
103. Vats A., et al. Chondrogenic differentiation of human embryonic stem cells: the effect of the micro-environment. Tissue Eng. 12 (2006) 1687-1697
104. Bielby R.C., et al. In vitro differentiation and in vivo mineralization of osteogenic cells derived from human embryonic stem cells. Tissue Eng. 10 (2004) 1518-1525
105. Woll P.S., et al. Human embryonic stem cell-derived NK cells acquire functional receptors and cytolytic activity. J. Immunol. 175 (2005) 5095-5103
106. Galic Z., et al. T lineage differentiation from human embryonic stem cells. Proc. Natl. Acad. Sci. U. S. A. 103 (2006) 11742-11747
107. Slukvin I.I., et al. Directed differentiation of human embryonic stem cells into functional dendritic cells through the myeloid pathway. J. Immunol. 176 (2006) 2924-2932
108. Gaur M., et al. Megakaryocytes derived from human embryonic stem cells: a genetically tractable system to study megakaryocytopoiesis and integrin function. J. Thromb. Haemost. 4 (2006) 436-442
109. Chang K.H., et al. Definitive-like erythroid cells derived from human embryonic stem cells coexpress high levels of embryonic and fetal globins with little or no adult globin. Blood 108 (2006) 1515-1523
110. Anderson J.S., et al. Derivation of normal macrophages from human embryonic stem (hES) cells for applications in HIV gene therapy. Retrovirology 3 (2006) 24
111. Fang D., et al. Defining the conditions for the generation of melanocytes from human embryonic stem cells. Stem Cells 24 (2006) 1668-1677
112. Lamba D.A., et al. Efficient generation of retinal progenitor cells from human embryonic stem cells. Proc. Natl. Acad. Sci. U. S. A. 103 (2006) 12769-12774
113. Lim U.M., et al. Derivation of motor neurons from three clonal human embryonic stem cell lines. Curr. Neurovasc. Res. 3 (2006) 281-288
114. Samadikuchaksaraei A., et al. Derivation of distal airway epithelium from human embryonic stem cells. Tissue Eng. 12 (2006) 867-875
115. Taylor R.A., et al. Formation of human prostate tissue from embryonic stem cells. Nat. Methods 3 (2006) 179-181
116. Wang D., et al. A pure population of lung alveolar epithelial type II cells derived from human embryonic stem cells. Proc. Natl. Acad. Sci. U. S. A. 104 (2007) 4449-4454