Developing predictive assays: The phenotypic screening "rule of 3"

Science Translational Medicine

Volumn 7, Issue 293, 2015, Pages

  Vincent, Fabien ^a   Loria, Paula ^a   Pregel, Marko ^a   Stanton, Robert ^a   Kitching, Linda ^b   Nocka, Karl ^a   Doyonnas, Regis ^a   Steppan, Claire ^a   Gilbert, Adam ^a   Schroeter, Thomas ^a   Peakman, Marie Claire ^a  

^a PFIZER INC   (United States)

^b PFIZER GLOBAL RESEARCH AND DEVELOPMENT   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANGIOTENSIN 1 RECEPTOR; APOLIPOPROTEIN E; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; G PROTEIN COUPLED RECEPTOR; HYDROGEN PEROXIDE; RNA;

ARTICLE; CELL LINE; CHROMOSOME DELETION; CHROMOSOME REARRANGEMENT; CYSTIC FIBROSIS; DRUG MECHANISM; DYSAUTONOMIA; EPIGENETICS; GENE MUTATION; HUMAN; MONOCYTE; PHENOTYPIC ASSAY; PLURIPOTENT STEM CELL; PREDICTION; PRIMARY CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; SCREENING TEST; SIGNAL TRANSDUCTION; TUMOR MICROENVIRONMENT; X CHROMOSOME; BIOASSAY; PHENOTYPE; PRECLINICAL STUDY; PROCEDURES;

BIOLOGICAL ASSAY; DRUG EVALUATION, PRECLINICAL; ENDPOINT DETERMINATION; HUMANS; PHENOTYPE;

EID: 84937622522     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.aab1201     Document Type: Article

References (36)

1. J. W. Scannell, A. Blanckley, H. Boldon, B. Warrington, Diagnosing the decline in pharmaceutical R&D efficiency. Nat. Rev. Drug Discov. 11, 191-200 (2012).
2. M. Allison, Reinventing clinical trials. Nat. Biotechnol. 30, 41-49 (2012).
3. F. Sams-Dodd, Is poor research the cause of the declining productivity of the pharmaceutical industry? An industry in need of a paradigm shift. Drug Discov. Today 18, 211-217 (2013).
4. D. C. Swinney, J. Anthony, How were new medicines discovered? Nat. Rev. Drug Discov. 10, 507-519 (2011).
5. F. Prinz, T. Schlange, K. Asadullah, Believe it or not: How much can we rely on published data on potential drug targets? Nat. Rev. Drug Discov. 10, 712 (2011).
6. C. G. Begley, L. M. Ellis, Drug development: Raise standards for preclinical cancer research. Nature 483, 531-533 (2012).
7. J. Eder, R. Sedrani, C. Wiesmann, The discovery of first-in-class drugs: Origins and evolution. Nat. Rev. Drug Discov. 13, 577-587 (2014).
8. J. A. Lee, E. L. Berg, Neoclassic drug discovery: The case for lead generation using phenotypic and functional approaches. J. Biomol. Screen. 18, 1143-1155 (2013).
9. D. N. Wilson, F. Schluenzen, J. M. Harms, A. L. Starosta, S. R. Connell, P. Fucini, The oxazolidinone antibiotics perturb the ribosomal peptidyl-transferase center and effect tRNA positioning. Proc. Natl. Acad. Sci. U.S.A. 105, 13339-13344 (2008).
10. D. Choi, J. P. Stables, H. Kohn, Synthesis and anticonvulsant activities of N-benzyl-2-acetamidopropionamide derivatives. J. Med. Chem. 39, 1907-1916 (1996).
11. A. C. Errington, T. Stöhr, C. Heers, G. Lees, The investigational anticonvulsant lacosamide selectively enhances slow inactivation of voltage-gated sodium channels. Mol. Pharmacol. 73, 157-169 (2008).
12. U. S. Eggert, The why and how of phenotypic small-molecule screens. Nat. Chem. Biol. 9, 206-209 (2013).
13. D. C. Swinney, The value of translational biomarkers to phenotypic assays. Front. Pharmacol. 5, 171 (2014).
14. J. A. Thomson, J. Itskovitz-Eldor, S. S. Shapiro, M. A. Waknitz, J. J. Swiergiel, V. S. Marshall, J. M. Jones, Embryonic stem cell lines derived from human blastocysts. Science 282, 1145-1147 (1998).
15. T. R. Cech, J. A. Steitz, The noncoding RNA revolution-trashing old rules to forge new ones. Cell 157, 77-94 (2014).
16. P. S. Eriksson, E. Perfilieva, T. Björk-Eriksson, A. M. Alborn, C. Nordborg, D. A. Peterson, F. H. Gage, Neurogenesis in the adult human hippocampus. Nat. Med. 4, 1313-1317 (1998).
17. H. Seely, Pieces of Intelligence: The Existential Poetry of Donald H. Rumsfeld (Free Press 2010), pp. 128.
18. S. J. Engle, F. Vincent, Small molecule screening in human induced pluripotent stem cell-derived terminal cell types. J. Biol. Chem. 289, 4562-4570 (2014).
19. M. D. Odero, N. J. Zeleznik-Le, V. Chinwalla, J. D. Rowley, Cytogenetic and molecular analysis of the acute monocytic leukemia cell line THP-1 with an MLL-AF9 translocation. Genes Chromosomes Cancer 29, 333-338 (2000).
20. T. P. Kenakin, A Pharmacology Primer (Academic Press, Burlington, MA, ed. 2, 2006).
21. M. Bellot, S. Galandrin, C. Boularan, H. J. Matthies, F. Despas, C. Denis, J. Javitch, S. Mazères, S. J. Sanni, V. Pons, M. H. Seguelas, J. L. Hansen, A. Pathak, A. Galli, J. M. Sénard, C. Galés, Dual agonist occupancy of AT1-R-α2C-AR heterodimers results in atypical Gs-PKA signaling. Nat. Chem. Biol. 11, 271-279 (2015).
22. N. Pedemonte, V. Tomati, E. Sondo, L. J. Galietta, Infuence of cell background on pharmacological rescue of mutant CFTR. Am. J. Physiol. Cell Physiol. 298, C866-C874 (2010).
23. M. A. Ashlock, E. R. Olson, Therapeutics development for cystic fibrosis: A successful model for a multisystem genetic disease. Annu. Rev. Med. 62, 107-125 (2011).
24. F. Van Goor, S. Hadida, P. D. Grootenhuis, B. Burton, J. H. Stack, K. S. Straley, C. J. Decker, M. Miller, J. McCartney, E. R. Olson, J. J. Wine, R. A. Frizzell, M. Ashlock, P. A. Negulescu, Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc. Natl. Acad. Sci. U.S.A. 108, 18843-18848 (2011).
25. G. Lee, C. N. Ramirez, H. Kim, N. Zeltner, B. Liu, C. Radu, B. Bhinder, Y. J. Kim, I. Y. Choi, B. Mukherjee-Clavin, H. Djaballah, L. Studer, Large-scale screening using familial dysautonomia induced pluripotent stem cells identifies compounds that rescue IKBKAP expression. Nat. Biotechnol. 30, 1244-1248 (2012).
26. T. Farfel-Becker, E. B. Vitner, A. H. Futerman, Animal models for Gaucher disease research. Dis. Model. Mech. 4, 746-752 (2011).
27. S. H. Choi, Y. H. Kim, M. Hebisch, C. Sliwinski, S. Lee, C. D'Avanzo, H. Chen, B. Hooli, C. Asselin, J. Mufat, J. B. Klee, C. Zhang, B. J. Wainger, M. Peitz, D. M. Kovacs, C. J. Woolf, S. L. Wagner, R. E. Tanzi, D. Y. Kim, A three-dimensional human neural cell culture model of Alzheimer's disease. Nature 515, 274-278 (2014).
28. D. Gosselin, V. M. Link, C. E. Romanoski, G. J. Fonseca, D. Z. Eichenfield, N. J. Spann, J. D. Stender, H. B. Chun, H. Garner, F. Geissmann, C. K. Glass, Environment drives selection and function of enhancers controlling tissue-specific macrophage identities. Cell 159, 1327-1340 (2014).
29. N. C. Kleinstreuer, J. Yang, E. L. Berg, T. B. Knudsen, A. M. Richard, M. T. Martin, D. M. Reif, R. S. Judson, M. Polokoff, D. J. Dix, R. J. Kavlock, K. A. Houck, Phenotypic screening of the ToxCast chemical library to classify toxic and therapeutic mechanisms. Nat. Biotechnol. 32, 583-591 (2014).
30. A. Nahirnyj, I. Livne-Bar, X. Guo, J. M. Sivak, ROS detoxification and proinflammatory cytokines are linked by p38 MAPK signaling in a model of mature astrocyte activation. PLOS ONE 8, e83049 (2013).
31. M. J. Wawer, K. Li, S. M. Gustafsdottir, V. Ljosa, N. E. Bodycombe, M. A. Marton, K. L. Sokolnicki, M. A. Bray, M. M. Kemp, E. Winchester, B. Taylor, G. B. Grant, C. S. Hon, J. R. Duvall, J. A. Wilson, J. A. Bittker, V. Dančík, R. Narayan, A. Subramanian, W. Winckler, T. R. Golub, A. E. Carpenter, A. F. Shamji, S. L. Schreiber, P. A. Clemons, Toward performance-diverse small-molecule libraries for cellbased phenotypic screening using multiplexed high-dimensional profiling. Proc. Natl. Acad. Sci. U.S.A. 111, 10911-10916 (2014).
32. J. P. Ioannidis, Biomarker failures. Clin. Chem. 59, 202-204 (2013).
33. J. Black, Nobel lecture in physiology or medicine-1988. Drugs from emasculated hormones: The principle of syntopic antagonism. In Vitro Cell. Dev. Biol. 25, 311-320 (1989).
34. M. Rottmann, C. McNamara, B. K. Yeung, M. C. Lee, B. Zou, B. Russell, P. Seitz, D. M. Ploufe, N. V. Dharia, J. Tan, S. B. Cohen, K. R. Spencer, G. E. González-Páez, S. B. Lakshminarayana, A. Goh, R. Suwanarusk, T. Jegla, E. K. Schmitt, H. P. Beck, R. Brun, F. Nosten, L. Renia, V. Dartois, T. H. Keller, D. A. Fidock, E. A. Winzeler, T. T. Diagana, Spiroindolones, a potent compound class for the treatment of malaria. Science 329, 1175-1180 (2010).
35. D. G. Perregaux, P. McNiff, R. Laliberte, N. Hawryluk, H. Peurano, E. Stam, J. Eggler, R. Griffiths, M. A. Dombroski, C. A. Gabel, Identification and characterization of a novel class of interleukin-1 post-translational processing inhibitors. J. Pharmacol. Exp. Ther. 299, 187-197 (2001).
36. A. M. Edwards, C. H. Arrowsmith, C. Bountra, M. E. Bunnage, M. Feldmann, J. C. Knight, D. D. Patel, P. Prinos, M. D. Taylor, M. Sundström, P. Barker, D. Barsyte, M. H. Bengtson, C. Bell, P. Bowness, K. M. Boycott, C. Buser-Doepner, C. L. Carpenter, A. J. Carr, K. Clark, A. M. Das, D. Dhanak, P. Dirks, J. Ellis, V. R. Fantin, C. Flores, E. A. Fon, D. E. Frail, O. Gileadi, R. C. O'Hagan, T. Howe, J. T. R. Isaac, N. Jabado, P.-J. Jakobsson, L. Klareskog, S. Knapp, W. H. Lee, E. Lima-Fernandes, I. E. Lundberg, J. Marshall, K. B. Massirer, A. E. MacKenzie, T. Maruyama, A. Mueller-Fahrnow, S. Muthuswamy, J. Nanchahal, C. O'Brien, U. Oppermann, N. Ostermann, K. Petrecca, B. G. Pollock, V. Poupon, R. K. Prinjha, S. H. Rosenberg, G. Rouleau, M. Skingle, A. S. Slutsky, G. A. M. Smith, D. Verhelle, H. Widmer, L. T. Young, SGC Open Source Target-Discovery Partnership, Preclinical target validation using patient-derived cells. Nat. Rev. Drug Discov. 14, 149-150 (2015). on June 25, 2015