Phenotypic screens as a renewed approach for drug discovery

Drug Discovery Today

Volumn 18, Issue 21-22, 2013, Pages 1067-1073

  Zheng, Wei ^a   Thorne, Natasha ^a   McKew, John C ^a  

^a NATIONAL INSTITUTES OF HEALTH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

NEW DRUG; RECOMBINANT DNA;

DRUG APPROVAL; DRUG DEVELOPMENT; DRUG IDENTIFICATION; DRUG INDUSTRY; DRUG REPOSITIONING; DRUG SCREENING; FOOD AND DRUG ADMINISTRATION; HIGH THROUGHPUT SCREENING; NONHUMAN; RECOMBINANT DNA TECHNOLOGY; REVIEW; STRUCTURE ACTIVITY RELATION;

ANIMALS; DRUG APPROVAL; DRUG DESIGN; DRUG DISCOVERY; DRUG INDUSTRY; HUMANS; MOLECULAR TARGETED THERAPY; PHENOTYPE; UNITED STATES;

EID: 84886953565     PISSN: 13596446     EISSN: 18785832     Source Type: Journal    
DOI: 10.1016/j.drudis.2013.07.001     Document Type: Review

References (82)

1. I.H. Segel Enzyme Kinetics; Behavior and Analysis of Rapid Equilibrium and Steady-state Enzyme Systems 1975 Wiley
2. J.C. Foreman, and T. Johansen Textbook of Receptor Pharmacology 1996 CRC Press
3. K.H. Lundstrom, and M.L. Chiu G Protein-coupled Receptors in Drug Discovery 2006 Taylor & Francis
4. W.R. Leifert G Protein-coupled Receptors in Drug Discovery 2009 Humana Press
5. J. Drews Genomic sciences and the medicine of tomorrow Nat. Biotechnol. 14 1996 1516 1518
6. A.L. Hopkins, and C.R. Groom The druggable genome Nat. Rev. Drug Discov. 1 2002 727 730
7. P. Imming Drugs, their targets and the nature and number of drug targets Nat. Rev. Drug Discov. 5 2006 821 834
8. M. Rask-Andersen Trends in the exploitation of novel drug targets Nat. Rev. Drug Discov. 10 2011 579 590
9. D.J. Diller The synergy between combinatorial chemistry and high-throughput screening Curr. Opin. Drug Discov. Dev. 11 2008 346 355
10. D.A. Pereira, and J.A. Williams Origin and evolution of high throughput screening Br. J. Pharmacol. 152 2007 53 61
11. T. Takenaka Classical vs reverse pharmacology in drug discovery BJU Int. 88 Suppl 2 2001 7 10 discussion 49-50
12. F. Darvas Recent advances in chemical genomics Curr. Med. Chem. 11 2004 3119 3145
13. A. Vogt, and J.S. Lazo Chemical complementation: a definitive phenotypic strategy for identifying small molecule inhibitors of elusive cellular targets Pharmacol. Ther. 107 2005 212 221
14. D.C. Swinney, and J. Anthony How were new medicines discovered? Nat. Rev. Drug Discov. 10 2011 507 519
15. J. Arrowsmith Trial watch: phase III and submission failures: 2007-2010 Nat. Rev. Drug Discov. 10 2011 87
16. J. Arrowsmith Trial watch: Phase II failures: 2008-2010 Nat. Rev. Drug Discov. 10 2011 328 329
17. J.P. Overington How many drug targets are there? Nat. Rev. Drug Discov. 5 2006 993 996
18. A.S. Pina An historical overview of drug discovery Methods Mol. Biol. 572 2009 3 12
19. H. Yuan Use of reprogrammed cells to identify therapy for respiratory papillomatosis N. Engl. J. Med. 367 2012 1220 1227
20. C.Z. Chen A homogenous luminescence assay reveals novel inhibitors for giardia lamblia carbamate kinase Curr. Chem. Genom. 6 2012 93 102
21. M. Xu delta-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders J. Biol. Chem. 287 2012 39349 39360
22. E. Gregori-Puigjane Identifying mechanism-of-action targets for drugs and probes Proc Natl Acad Sci U S A 109 2012 11178 11183
23. J.R. Vane, and R.M. Botting The mechanism of action of aspirin Thromb. Res. 110 2003 255 258
24. W.J. Elliott, and C.V. Ram Calcium channel blockers J. Clin. Hypertens. 13 2011 687 689
25. D.J. Triggle Calcium antagonists. History and perspective Stroke 21 1990 IV49 IV58
26. D.J. Triggle Calcium channel antagonists: clinical uses - past, present and future Biochem. Pharmacol. 74 2007 1 9
27. A. Fleckenstein History of calcium antagonists Circ. Res. 52 1983 I3 I16
28. T. Tanabe Primary structure of the receptor for calcium channel blockers from skeletal muscle Nature 328 1987 313 318
29. M. Van Heek In vivo metabolism-based discovery of a potent cholesterol absorption inhibitor, SCH58235, in the rat and rhesus monkey through the identification of the active metabolites of SCH48461 J. Pharmacol. Exp. Ther. 283 1997 157 163
30. S.B. Rosenblum Discovery of 1-(4-fluorophenyl)-(3R)-[3-(4-fluorophenyl)- (3S)-hydroxypropyl]-(4S)-(4 -hydroxyphenyl)-2-azetidinone (SCH 58235): a designed, potent, orally active inhibitor of cholesterol absorption J. Med. Chem. 41 1998 973 980
31. E.A. Nutescu, and N.L. Shapiro Ezetimibe: a selective cholesterol absorption inhibitor Pharmacotherapy 23 2003 1463 1474
32. S.W. Altmann Niemann-Pick C1 Like 1 protein is critical for intestinal cholesterol absorption Science 303 2004 1201 1204
33. J. Giacomotto, and L. Segalat High-throughput screening and small animal models, where are we? Br. J. Pharmacol. 160 2010 204 216
34. E. Braungart Caenorhabditis elegans MPP□ model of Parkinson's disease for high-throughput drug screenings Neuro-degen. Dis. 1 2004 175 183
35. S. Camus Identification of phosphorylase kinase as a novel therapeutic target through high-throughput screening for anti-angiogenesis compounds in zebrafish Oncogene 31 2012 4333 4342
36. S. Ridges Zebrafish screen identifies novel compound with selective toxicity against leukemia Blood 119 2012 5621 5631
37. U.B. Pandey, and C.D. Nichols Human disease models in Drosophila melanogaster and the role of the fly in therapeutic drug discovery Pharmacol. Rev. 63 2011 411 436
38. M.L. Tomlinson Chemical genetics and drug discovery in Xenopus Methods Mol. Biol. 917 2012 155 166
39. S.N. Rampersad Multiple applications of Alamar Blue as an indicator of metabolic function and cellular health in cell viability bioassays Sensors 12 2012 12347 12360
40. T. Mosmann Rapid colorimetric assay for cellular growth and survival: application to proliferation and cytotoxicity assays J. Immunol. Methods 65 1983 55 63
41. C. Riccardi, and I. Nicoletti Analysis of apoptosis by propidium iodide staining and flow cytometry Nat. Protoc. 1 2006 1458 1461
42. M.H. Cho A bioluminescent cytotoxicity assay for assessment of membrane integrity using a proteolytic biomarker Toxicol. In Vitro 22 2008 1099 1106
43. S.A. Titus High-throughput multiplexed quantitation of protein aggregation and cytotoxicity in a Huntington's disease model Curr. Chem. Genom. 6 2012 79 86
44. M. Xia Compound cytotoxicity profiling using quantitative high-throughput screening Environ. Health Perspect. 116 2008 284 291
45. E. Michelini Cell-based assays: fuelling drug discovery Anal. Bioanal. Chem. 398 2010 227 238
46. E.C. Butcher Systems biology in drug discovery Nat. Biotechnol. 22 2004 1253 1259
47. J. Inglese High-throughput screening assays for the identification of chemical probes Nat. Chem. Biol. 3 2007 466 479
48. W.F. An, and N. Tolliday Cell-based assays for high-throughput screening Mol. Biotechnol. 45 2010 180 186
49. B.B. Aggarwal Targeting cell signaling pathways for drug discovery: an old lock needs a new key J. Cell. Biochem. 102 2007 580 592
50. C.T. Aiken A cell-based screen for drugs to treat Huntington's disease Neurobiol. Dis. 16 2004 546 555
51. A.I. Rosenbaum Chemical screen to reduce sterol accumulation in Niemann-Pick C disease cells identifies novel lysosomal acid lipase inhibitors Biochim. Biophys. Acta 1791 2009 1155 1165
52. F.C. Ramaekers, and F.T. Bosman The cytoskeleton and disease J. Pathol. 204 2004 351 354
53. J.M. Zock Applications of high content screening in life science research Comb. Chem. High Throughput Screen. 12 2009 870 876
54. F. Zanella High content screening: seeing is believing Trends Biotechnol. 28 2010 237 245
55. A. Barabasz The use of high-content screening for the discovery and characterization of compounds that modulate mitotic index and cell cycle progression by differing mechanisms of action Assay Drug Dev. Technol. 4 2006 153 163
56. F. Gasparri Cell-cycle inhibitor profiling by high-content analysis Adv. Exp. Med. Biol. 604 2007 137 148
57. J. Bacart The BRET technology and its application to screening assays Biotechnol. J. 3 2008 311 324
58. D. Auld, and N. Thorne Molecular sensors for transcriptional and post-transcriptional assays H. Fu, Chemical Genomics 2012 Cambridge University Press 173 197
59. R. Eglen, and T. Reisine Primary cells and stem cells in drug discovery: emerging tools for high-throughput screening Assay Drug Dev. Technol. 9 2011 108 124
60. A.D. Ebert, and C.N. Svendsen Human stem cells and drug screening: opportunities and challenges Nat. Rev. Drug Discov. 9 2010 367 372
61. G.L. Boulting A functionally characterized test set of human induced pluripotent stem cells Nat. Biotechnol. 29 2011 279 286
62. A. Mackay-Sim Patient-derived stem cells: pathways to drug discovery for brain diseases Front. Cell. Neurosci. 7 2013 29
63. G. Lee Large-scale screening using familial dysautonomia induced pluripotent stem cells identifies compounds that rescue IKBKAP expression Nat. Biotechnol. 30 2012 1244 1248
64. W.N. Zhao A high-throughput screen for Wnt/beta-catenin signaling pathway modulators in human iPSC-derived neural progenitors J. Biomol. Screen. 17 2012 1252 1263
65. D. McLaren Automated large-scale culture and medium-throughput chemical screen for modulators of proliferation and viability of human induced pluripotent stem cell-derived neuroepithelial-like stem cells J. Biomol. Screen. 18 2013 258 268
66. J. McNeish High-throughput screening in embryonic stem cell-derived neurons identifies potentiators of alpha-amino-3-hydroxyl-5-methyl-4- isoxazolepropionate-type glutamate receptors J. Biol. Chem. 285 2010 17209 17217
67. C.W. Scott Human induced pluripotent stem cells and their use in drug discovery for toxicity testing Toxicol. Lett. 219 2013 49 58
68. D. Rajamohan Current status of drug screening and disease modelling in human pluripotent stem cells BioEssays 35 2013 281 298
69. R. Huang The NCGC pharmaceutical collection: a comprehensive resource of clinically approved drugs enabling repurposing and chemical genomics Sci. Transl. Med. 3 2011 80ps16
70. S.C. Miller Identification of known drugs that act as inhibitors of NF-kappaB signaling and their mechanism of action Biochem. Pharmacol. 79 2010 1272 1280
71. M. Shen Identification of Therapeutic Candidates for Chronic Lymphocytic Leukemia from a Library of Approved Drugs 2013 submitted for publication
72. M. Xia Identification of repurposed small molecule drugs for chordoma therapy Cancer Biology and Therapy 14 2013 563-564
73. N. Nilubol Four clinically utilized drugs were identified and validated for treatment of adrenocortical cancer using quantitative high-throughput screening J. Transl. Med. 10 2012 198
74. L. Zhang Quantitative high-throughput drug screening identifies novel classes of drugs with anticancer activity in thyroid cancer cells: opportunities for repurposing J. Clin. Endocrinol. Metab. 97 2012 E319 E328
75. S. Kang CaV1.3-selective L-type calcium channel antagonists as potential new therapeutics for Parkinson's disease Nat. Commun. 3 2012 1146
76. N. Singh A safe lithium mimetic for bipolar disorder Nat. Commun. 4 2013 1332
77. A.G. Reaume Drug repurposing through nonhypothesis driven phenotypic screening Drug Discov. Today. Ther. Strat. 8 2011 85 88
78. T.T. Ashburn, and K.B. Thor Drug repositioning: identifying and developing new uses for existing drugs Nat. Rev. Drug Discov. 3 2004 673 683
79. S. Neumann Small-molecule agonists for the thyrotropin receptor stimulate thyroid function in human thyrocytes and mice Proc. Natl. Acad. Sci. U. S. A. 106 2009 12471 12476
80. T. Neuberger Use of primary cultures of human bronchial epithelial cells isolated from cystic fibrosis patients for the pre-clinical testing of CFTR modulators Methods Mol. Biol. 741 2011 39 54
81. M.L. Fulcher Novel human bronchial epithelial cell lines for cystic fibrosis research Am. J. Physiol. Lung Cell. Mol. Physiol. 296 2009 L82 L91
82. J. Xiao Discovery, synthesis, and biological evaluation of novel SMN protein modulators J. Med. Chem. 54 2011 6215 6233