Monitoring molecular-specific pharmacodynamics of rapamycin in vivo with inducible Gal4→Fluc transgenic reporter mice

Molecular Cancer Therapeutics

Volumn 9, Issue 10, 2010, Pages 2752-2760

  Pan, Mei Hsiu ^a   Lin, Jeffrey ^a   Prior, Julie L ^a   Piwnica Worms, David ^a  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

MAMMALIAN TARGET OF RAPAMYCIN; RAP PROTEIN; RAPAMYCIN; TACROLIMUS;

ANIMAL EXPERIMENT; ARTICLE; BIOLUMINESCENCE; CONCENTRATION RESPONSE; CONTROLLED STUDY; DRUG MECHANISM; GENETIC TRANSCRIPTION; GENOMICS; HUMAN; HUMAN CELL; IN VIVO STUDY; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN FOLDING; PROTEIN PROTEIN INTERACTION; PROTEOMICS;

ANIMALS; BLOTTING, WESTERN; GENES, REPORTER; HELA CELLS; HUMANS; MICE; MICE, TRANSGENIC; SIROLIMUS; TWO-HYBRID SYSTEM TECHNIQUES;

EID: 77958023388     PISSN: 15357163     EISSN: 15388514     Source Type: Journal    
DOI: 10.1158/1535-7163.MCT-10-0265     Document Type: Article

References (54)

1. Abraham RT, Eng CH. Mammalian target of rapamycin as a therapeutic target in oncology. Expert Opin Ther Targets 2008;12:209-22.
2. Choi J, Chen J, Schreiber SL, Clardy J. Structure of the FKBP12-rapamycin complex interacting with the binding domain of human FRAP. Science 1996;273:239-42.
3. Edwards SR, Wandless TJ. The rapamycin-binding domain of the protein kinase mammalian target of rapamycin is a destabilizing domain. J Biol Chem 2007;282:13395-401.
4. Groth CG, Backman L, Morales JM, et al. Sirolimus European Renal Transplant Study Group. Sirolimus (rapamycin)-based therapy in human renal transplantation: similar efficacy and different toxicity compared with cyclosporine. Transplantation 1999;67:1036-42.
5. Sessa C, Tosi D, Vigano L, et al. Phase Ib study of weekly mammalian target of rapamycin inhibitor ridaforolimus (AP23573; MK-8669) with weekly paclitaxel. Ann Oncol 2010;21:1315-22.
6. Galanis E, Buckner JC, Maurer MJ, et al. Phase II trial of temsirolimus (CCI-779) in recurrent glioblastoma multiforme: a North Central Cancer Treatment Group study. J Clin Oncol 2005;23:5294-304.
7. Hudes G, Carducci M, Tomczak P, et al. Temsirolimus, interferon α, or both for advanced renal-cell carcinoma. N Engl J Med 2007;356:2271-81.
8. Kamada Y, Funakoshi T, Shintani T, Nagano K, Ohsumi M, Ohsumi Y. Tor-mediated induction of autophagy via an Apg1 protein kinase complex. J Cell Biol 2000;150:1507-13.
9. Zaragoza D, Ghavidel A, Heitman J, Schultz MC. Rapamycin induces the G0 program of transcriptional repression in yeast by interfering with the TOR signaling pathway. Mol Cell Biol 1998;18:4463-70.
10. Graziani EI. Recent advances in the chemistry, biosynthesis and pharmacology of rapamycin analogs. Nat Prod Rep 2009;26:602-9.
11. Hedley DW, Chow S, Goolsby C, Shankey TV. Pharmacodynamic monitoring of molecular-targeted agents in the peripheral blood of leukemia patients using flow cytometry. Toxicol Pathol 2008;36:133-9.
12. Liebes L, Potmesil M, Kim T, et al. Pharmacodynamics of topoisomerase I inhibition: western blot determination of topoisomerase I and cleavable complex in patients with upper gastrointestinal malignancies treated with topotecan. Clin Cancer Res 1998;4:545-57.
13. Baselga J, Albanell J, Ruiz A, et al. Phase II and tumor pharmacodynamic study of gefitinib in patients with advanced breast cancer. J Clin Oncol 2005;23:5323-33.
14. Sarker D, Workman P. Pharmacodynamic biomarkers for molecular cancer therapeutics. Adv Cancer Res 2007;96:213-68.
15. Chautard E, Thierry-Mieg N, Ricard-Blum S. Interaction networks: from protein functions to drug discovery. A review. Pathol Biol (Paris) 2009;57:324-33.
16. Ferrer-Alcon M, Arteta D, Guerrero MJ, Fernandez-Orth D, Simon L, Martinez A. The use of gene array technology and proteomics in the search of new targets of diseases for therapeutics. Toxicol Lett 2009;186:45-51.
17. Gross S, Piwnica-Worms D. Molecular imaging strategies for drug discovery and development. Curr Opin Chem Biol 2006;10:334-42.
18. Stell A, Belcredito S, Ramachandran B, et al. Multimodality imaging: novel pharmacological applications of reporter systems. Q J Nucl Med Mol Imaging 2007;51:127-38.
19. US Code of Federal Regulations; vol 21, pp. 500-60.
20. Pichler A, Prior JL, Luker GD, Piwnica-Worms D. Generation of a highly inducible Gal4→Fluc universal reporter mouse for in vivo bioluminescence imaging. Proc Natl Acad Sci U S A 2008;105:15932-7.
21. Berman HM, Westbrook J, Feng Z, et al. The Protein Data Bank. Nucleic Acids Res 2000;28:235-42.
22. Odagaki Y, Clardy J. Structural basis for peptidomimicry by the effector element of rapamycin. J Am Chem Soc 1997;119:10253.
23. Jonker HR, Wechselberger RW, Boelens R, Folkers GE, Kaptein R. Structural properties of the promiscuous VP16 activation domain. Biochemistry 2005;44:827-39.
24. Wilson KP, Yamashita MM, Sintchak MD, et al. Comparative X-ray structures of the major binding protein for the immunosuppressant FK506 (tacrolimus) in unliganded form and in complex with FK506 and rapamycin. Acta Crystallogr D Biol Crystallogr 1995;51:511-21.
25. Hong M, Fitzgerald MX, Harper S, Luo C, Speicher DW, Marmorstein R. Structural basis for dimerization in DNA recognition by Gal4. Structure 2008;16:1019-26.
26. Bayle JH, Grimley JS, Stankunas K, Gestwicki JE, Wandless TJ, Crabtree GR. Rapamycin analogs with differential binding specificity permit orthogonal control of protein activity. Chem Biol 2006;13:99-107.
27. Gross S, Piwnica-Worms D. Real-time imaging of ligand-induced IKK activation in intact cells and in living mice. Nat Methods 2005;2:607-14.
28. Cheng Y, Prusoff W. Relationship between inhibition constant (ki) and the concentration of the inhibitor which causes 50% inhibition (I50) of an enzymatic reaction. Biochem Pharmacol 1973;22:3099-108.
29. Chen J, Zheng XF, Brown EJ, Schreiber SL. Identification of an 11-kDa FKBP12-rapamycin-binding domain within the 289-kDa FKBP12-rapamycin-associated protein and characterization of a critical serine residue. Proc Natl Acad Sci U S A 1995;92:4947-51.
30. Lai JH, Tan TH. CD28 signaling causes a sustained down-regulation of I κ B α which can be prevented by the immunosuppressant rapamycin. J Biol Chem 1994;269:30077-80.
31. Serruys PW, Kutryk MJ, Ong AT. Coronary-artery stents. N Engl J Med 2006;354:483-95.
32. Poon M, Marx SO, Gallo R, Badimon JJ, Taubman MB, Marks AR. Rapamycin inhibits vascular smooth muscle cell migration. J Clin Invest 1996;98:2277-83.
33. Motzer RJ, Escudier B, Oudard S, et al. Efficacy of everolimus in advanced renal cell carcinoma: a double-blind, randomised, placebo-controlled phase III trial. Lancet 2008;372:449-56.
34. Atkins MB, Yasothan U, Kirkpatrick P. Everolimus. Nat Rev Drug Discov 2009;8:535-6.
35. Lyons WE, George EB, Dawson TM, Steiner JP, Snyder SH. Immunosuppressant FK506 promotes neurite outgrowth in cultures of PC12 cells and sensory ganglia. Proc Natl Acad Sci U S A 1994;91:3191-5.
36. Ruan B, Pong K, Jow F, et al. Binding of rapamycin analogs to calcium channels and FKBP52 contributes to their neuroprotective activities. Proc Natl Acad Sci U S A 2008;105:33-8.
37. Banaszynski LA, Sellmyer MA, Contag CH, Wandless TJ, Thorne SH. Chemical control of protein stability and function in living mice. Nat Med 2008;14:1123-7.
38. Jimeno A, Rudek MA, Kulesza P, et al. Pharmacodynamic-guided modified continuous reassessment method-based, dose-finding study of rapamycin in adult patients with solid tumors. J Clin Oncol 2008;26:4172-9.
39. Johnson BE, Jackman D, Janne PA. Rationale for a phase I trial of erlotinib and the mammalian target of rapamycin inhibitor everolimus (RAD001) for patients with relapsed non small cell lung cancer. Clin Cancer Res 2007;13:s4628-31.
40. Muller-Steinhardt M, Wortmeier K, Fricke L, Ebel B, Hartel C. The pharmacodynamic effect of sirolimus: individual variation of cytokine mRNA expression profiles in human whole blood samples. Immunobiology 2009;214:17-26.
41. Tanaka C, O'Reilly T, Kovarik JM, et al. Identifying optimal biologic doses of everolimus (RAD001) in patients with cancer based on the modeling of preclinical and clinical pharmacokinetic and pharmacodynamic data. J Clin Oncol 2008;26:1596-602.
42. Luker KE, Smith MC, Luker GD, Gammon ST, Piwnica-Worms H, Piwnica-Worms D. Kinetics of regulated protein-protein interactions revealed with firefly luciferase complementation imaging in cells and living animals. Proc Natl Acad Sci U S A 2004;101:12288-93.
43. Galarneau A, Primeau M, Trudeau L-E, Michnick S. b-Lactamase protein fragment complementation assays as in vivo and in vitro sensors of protein-protein interactions. Nat Biotechnol 2002;20:619-22.
44. Remy I, Michnick S. Clonal selection and in vivo quantitation of protein interactions with protein-fragment complementation assays. Proc Natl Acad Sci U S A 1999;96:5394-9.
45. Shama S, Meyuhas O. The translational cis-regulatory element of mammalian ribosomal protein mRNAs is recognized by the plant translational apparatus. Eur J Biochem 1996;236:383-8.
46. Hara K, Yonezawa K, Weng QP, Kozlowski MT, Belham C, Avruch J. Amino acid sufficiency and mTOR regulate p70 S6 kinase and eIF-4E BP1 through a common effector mechanism. J Biol Chem 1998;273:14484-94.
47. Moser BA, Dennis PB, Pullen N, et al. Dual requirement for a newly identified phosphorylation site in p70s6k. Mol Cell Biol 1997;17:5648-55.
48. Turck F, Kozma SC, Thomas G, Nagy F. A heat-sensitive Arabidopsis thaliana kinase substitutes for human p70s6k function in vivo. Mol Cell Biol 1998;18:2038-44.
49. Cruz MC, Goldstein AL, Blankenship J, et al. Rapamycin and less immunosuppressive analogs are toxic to Candida albicans and Cryptococcus neoformans via FKBP12-dependent inhibition of TOR. Antimicrob Agents Chemother 2001;45:3162-70.
50. Vidalain PO, Boxem M, Ge H, Li S, Vidal M. Increasing specificity in high-throughput yeast two-hybrid experiments. Methods 2004;32:363-70.
51. Figeys D. Mapping the human protein interactome. Cell Res 2008;18:716-24.
52. Iyer M, Wu L, Carey M, Wang Y, Smallwood A, Gambhir S. Two-step transcriptional amplification as a method for imaging reporter gene expression using weak promoters. Proc Natl Acad Sci U S A 2001;98:14595-600.
53. Griggs DW, Johnston M. Promoter elements determining weak expression of the GAL4 regulatory gene of Saccharomyces cerevisiae. Mol Cell Biol 1993;13:4999-5009.
54. Villalobos V, Naik S, Piwnica-Worms D. Current state of imaging protein-protein interactions in vivo with genetically encoded reporters. Annu Rev Biomed Eng 2007;9:321-49.