Evaluation of alternative in vivo drug screening methodology: A single mouse analysis

Cancer Research

Volumn 76, Issue 19, 2016, Pages 5798-5809

  Murphy, Brendan ^a   Yin, Han ^a   Maris, John M ^b   Kolb, E Anders ^c   Gorlick, Richard ^d   Reynolds, C Patrick ^e   Kang, Min H ^f   Keir, Stephen T ^g   Kurmasheva, Raushan T ^h   Dvorchik, Igor ^a   Wu, Jianrong ⁱ   Billups, Catherine A ⁱ   Boateng, Nana ⁱ   Smith, Malcolm A ^j   Lock, Richard B ^k   Houghton, Peter J ^a  

^a NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c NEMOURS ALFRED I DUPONT HOSPITAL FOR CHILDREN   (United States)

^d CHILDREN S HOSPITAL AT MONTEFIORE   (United States)

^e TEXAS TECH UNIVERSITY HEALTH SCIENCES CENTER   (United States)

^f TEXAS TECH UNIVERSITY HEALTH SCIENCES CENTER SCHOOL OF MEDICINE   (United States)

^g DUKE UNIVERSITY MEDICAL CENTER   (United States)

^h UNIVERSITY OF TEXAS HEALTH SCIENCE CENTER AT SAN ANTONIO   (United States)

ⁱ ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^j NATIONAL CANCER INSTITUTE   (United States)

^k CHILDREN S CANCER INSTITUTE AUSTRALIA FOR MEDICAL RESEARCH   (Australia)

more..

Author keywords

[No Author keywords available]

Indexed keywords

1 [4 [(5 CYCLOPROPYL 1H PYRAZOL 3 YL)AMINO]PYRROLO[2,1 F][1,2,4]TRIAZIN 2 YL] N (6 FLUORO 3 PYRIDINYL) 2 METHYL 2 PYRROLIDINECARBOXAMIDE; 4 [2 (4 AMINO 1,2,5 OXADIAZOL 3 YL) 1 ETHYL 7 (3 PIPERIDINYLMETHOXY) 1H IMIDAZO[4,5 C]PYRIDIN 4 YL] 2 METHYL 3 BUTYN 2 OL; 5 CHLORO N2 [1 (5 FLUORO 2 PYRIMIDINYL)ETHYL] N4 (5 METHYL 1H PYRAZOL 3 YL) 2,4 PYRIMIDINEDIAMINE; ALVESPIMYCIN; ARSENIC TRIOXIDE; AZD 8055; BORTEZOMIB; CEDIRANIB; CHLORMETHINE; CIXUTUMUMAB; CYTARABINE; DASATINIB; DEHYDRODIDEMNIN B; DINACICLIB; LAPATINIB; LENALIDOMIDE; MAPATUMUMAB; N (6,7 DIHYDRO 6 OXO 5H DIBENZ[B,D]AZEPIN 7 YL) 2,2 DIMETHYL N' (2,2,3,3,3 PENTAFLUOROPROPYL)PROPANEDIAMIDE; N [1 CYCLOHEXYL 2 [2 [4 (4 FLUOROBENZOYL) 2 THIAZOLYL] 1 PYRROLIDINYL] 2 OXOETHYL] 2 (METHYLAMINO)PROPANAMIDE; NAVITOCLAX; ONALESPIB; PAZOPANIB; PEVONEDISTAT; QUISINOSTAT; RAPAMYCIN; SERDEMETAN; SORAFENIB; SUNITINIB; VORINOSTAT;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DISEASE COURSE; DRUG ACTIVITY; DRUG RESEARCH; DRUG RESPONSE; DRUG SCREENING; EXPERIMENTAL DESIGN; FEMALE; GENOTYPE; HUMAN; HUMAN CELL; IN VIVO STUDY; MOLECULARLY TARGETED THERAPY; MOUSE; NONHUMAN; PRECLINICAL STUDY; PREDICTIVE VALUE; PRIORITY JOURNAL; RETROSPECTIVE STUDY; TUMOR VOLUME; ANIMAL; NEOPLASMS; PRECURSOR CELL LYMPHOBLASTIC LEUKEMIA-LYMPHOMA; PROCEDURES; TUMOR CELL LINE;

ANIMALS; CELL LINE, TUMOR; HUMANS; MICE; NEOPLASMS; PRECURSOR CELL LYMPHOBLASTIC LEUKEMIA-LYMPHOMA; RETROSPECTIVE STUDIES; XENOGRAFT MODEL ANTITUMOR ASSAYS;

EID: 84989875414     PISSN: 00085472     EISSN: 15387445     Source Type: Journal    
DOI: 10.1158/0008-5472.CAN-16-0122     Document Type: Article

References (48)

1. Goldin A, Venditti JM. Progress report on the screening program at the Division of Cancer Treatment, National Cancer Institute. Cancer Treat Rev 1980;7:167-76.
2. Goldin A, Venditti JM. The new NCI screen and its implications for clinical evaluation. Recent Results Cancer Res 1980;70:5-20.
3. Goldin A, Venditti JM. A prospective screening program: current screening and its status. Recent Results Cancer Res 1981;76:176-91.
4. Sausville EA, Burger AM. Contributions of human tumor xenografts to anticancer drug development. Cancer Res 2006;66:3351-4.
5. Gould SE, Junttila MR, de Sauvage FJ. Translational value of mouse models in oncology drug development. Nat Med 2015;21:431-9.
6. Williams SA, Anderson WC, Santaguida MT, Dylla SJ. Patient-derived xenografts, the cancer stem cell paradigm, and cancer pathobiology in the 21st century. Lab Invest 2013;93:970-82.
7. Hidalgo M, Amant F, Biankin AV, Budinska E, Byrne AT, Caldas C, et al. Patient-derived xenograft models: an emerging platform for translational cancer research. Cancer Discov 2014;4:998-1013.
8. Pickard RG, Cobb LM, Steel GG. The growth kinetics of xenografts of human colorectal tumours in immune deprived mice. Br J Cancer 1975;31:36-45.
9. Houghton JA, Taylor DM. Maintenance of biological and biochemical characteristics of human colorectal tumours during serial passage in immune-deprived mice. Br J Cancer 1978;37:199-212.
10. Houghton JA, Houghton PJ, Webber BL. Growth and characterization of childhood rhabdomyosarcomas as xenografts. J Natl Cancer Inst 1982; 68:437-43.
11. Gillet JP, Calcagno AM, Varma S, Marino M, Green LJ, Vora MI, et al. Redefining the relevance of established cancer cell lines to the study of mechanisms of clinical anti-cancer drug resistance. ProcNatl Acad SciUS A 2011;108:18708-13.
12. Hausser HJ, Brenner RE. Phenotypic instability of Saos-2 cells in long-term culture. Biochem Biophys Res Commun 2005;333: 216-22.
13. Kwak EL, Bang YJ, Camidge DR, Shaw AT, Solomon B, Maki RG, et al. Anaplastic lymphoma kinase inhibition in non-small cell lung cancer. N Engl J Med 2010;363:1693-703.
14. Lynch TJ, Bell DW, Sordella R, Gurubhagavatula S, Okimoto RA, Brannigan BW, et al. Activating mutations in the epidermal growth factor receptor underlying responsiveness of non-small cell lung cancer to gefitinib. N Engl J Med 2004;350:2129-39.
15. Paez JG, Janne PA, Lee JC, Tracy S, Greulich H, Gabriel S, et al. EGFR mutations in lung cancer: correlation with clinical response to gefitinib therapy. Science 2004;304:1497-500.
16. Mack SC, Witt H, Wang X, Milde T, Yao Y, Bertrand KC, et al. Emerging insights into the ependymoma epigenome. Brain Pathol 2013;23:206-9.
17. Nagasawa DT, Trang A, Choy W, Spasic M, Yew A, Zarinkhou G, et al. Genetic expression profiles of adult and pediatric ependymomas: molecular pathways, prognostic indicators, and therapeutic targets. Clin Neurol Neurosurg 2013;115:388-99.
18. Hovestadt V, Jones DT, Picelli S, Wang W, Kool M, Northcott PA, et al. Decoding the regulatory landscape of medulloblastoma using DNA methylation sequencing. Nature 2014;510:537-41.
19. Hovestadt V, Remke M, Kool M, Pietsch T, Northcott PA, Fischer R, et al. Robust molecular subgrouping and copy-number profiling of medulloblastoma from small amounts of archival tumour material using highdensity DNA methylation arrays. Acta Neuropathol 2013;125:913-6.
20. Schleiermacher G, Javanmardi N, Bernard V, Leroy Q, Cappo J, Rio Frio T, et al. Emergence of new ALK mutations at relapse of neuroblastoma. J Clin Oncol 2014;32:2727-34.
21. Kushner BH, Modak S, Kramer K, LaQuaglia MP, Yataghene K, Basu EM, et al. Striking dichotomy in outcome of MYCN-amplified neuroblastoma in the contemporary era. Cancer 2014;120:2050-9.
22. Maris JM. Recent advances in neuroblastoma. N Engl J Med 2010; 362:2202-11.
23. Parham DM, Barr FG. Classification of rhabdomyosarcoma and its molecular basis. Adv Anat Pathol 2013;20:387-97.
24. Abraham J, Nunez-Alvarez Y, Hettmer S, Carrio E, Chen HI, Nishijo K, et al. Lineage of origin in rhabdomyosarcoma informs pharmacological response. Genes Dev 2014;28:1578-91.
25. Khanna C, Fan TM, Gorlick R, Helman LJ, Kleinerman ES, Adamson PC, et al. Toward a drug development path that targets metastatic progression in osteosarcoma. Clin Cancer Res 2014;20:4200-9.
26. Shern JF, Chen L, Chmielecki J, Wei JS, Patidar R, Rosenberg M, et al. Comprehensive genomic analysis of rhabdomyosarcoma reveals a landscape of alterations affecting a common genetic axis in fusion-positive and fusion-negative tumors. Cancer Discov 2014;4:216-31.
27. Mullighan CG. Genomic characterization of childhood acute lymphoblastic leukemia. Semin Hematol 2013;50:314-24.
28. Gao H, Korn JM, Ferretti S, Monahan JE, Wang Y, Singh M, et al. Highthroughput screening using patient-derived tumor xenografts to predict clinical trial drug response. Nat Med 2015;21:1318-25.
29. Kolb EA, Gorlick R, Houghton PJ, Morton CL, Neale G, Keir ST, et al. Initial testing (stage 1) of AZD6244 (ARRY-142886) by the Pediatric Preclinical Testing Program. Pediatr Blood Cancer 2010;55:668-77.
30. Smith MA, Hampton OA, Reynolds CP, Kang MH, Maris JM, Gorlick R, et al. Initial testing (stage 1) of the PARP inhibitorBMN673 by the pediatric preclinical testing program: PALB2 mutation predicts exceptional in vivo response to BMN 673. Pediatr Blood Cancer 2015;62:91-8.
31. Smith MA, Reynolds CP, Kang MH, Kolb EA, Gorlick R, Carol H, et al. Synergistic activity of PARP inhibition by talazoparib (BMN 673) with temozolomide in pediatric cancer models in the pediatric preclinical testing program. Clin Cancer Res 2015;21:819-32.
32. Kolb EA, Gorlick R, Houghton PJ, Morton CL, Lock R, Carol H, et al. Initial testing (stage 1) of a monoclonal antibody (SCH 717454) against the IGF- 1 receptor by the pediatric preclinical testing program. Pediatr Blood Cancer 2008;50:1190-7.
33. Peterson JK, Houghton PJ. Integrating pharmacology and in vivo cancer models in preclinical and clinical drug development. Eur J Cancer 2004; 40:837-44.
34. Houghton PJ, Morton CL, Tucker C, Payne D, Favours E, Cole C, et al. The pediatric preclinical testing program: description of models and early testing results. Pediatr Blood Cancer 2007;49:928-40.
35. Morton CL, Papa RA, Lock RB, Houghton PJ. Preclinical chemotherapeutic tumor models of common childhood cancers: solid tumors, acute lymphoblastic leukemia, and disseminated neuroblastoma. Curr Protoc Pharmacol 2007;Chapter 14:Unit148.
36. Friedman HS, Colvin OM, Skapek SX, Ludeman SM, Elion GB, Schold SC Jr, et al. Experimental chemotherapy of human medulloblastoma cell lines and transplantable xenografts with bifunctional alkylating agents. Cancer Res 1988;48:4189-95.
37. Liem NL, Papa RA, Milross CG, Schmid MA, Tajbakhsh M, Choi S, et al. Characterization of childhood acute lymphoblastic leukemia xenograft models for the preclinical evaluation of new therapies. Blood 2004; 103:3905-14.
38. Whiteford CC, Bilke S, Greer BT, Chen Q, Braunschweig TA, Cenacchi N, et al. Credentialing preclinical pediatric xenograft models using gene expression and tissue microarray analysis. Cancer Res 2007;67:32-40.
39. Neale G, Su X, Morton CL, Phelps D, Gorlick R, Lock RB, et al. Molecular characterization of the pediatric preclinical testing panel. Clin Cancer Res 2008;14:4572-83.
40. Kolb EA, Gorlick R, Houghton PJ, Morton CL, Lock RB, Tajbakhsh M, et al. Initial testing of dasatinib by the pediatric preclinical testing program. Pediatr Blood Cancer 2008;50:1198-206.
41. Maris JM, Courtright J, Houghton PJ, Morton CL, Kolb EA, Lock R, et al. Initial testing (stage 1) of sunitinib by the pediatric preclinical testing program. Pediatr Blood Cancer 2008;51:42-8.
42. Carol H, Reynolds CP, Kang MH, Keir ST, Maris JM, Gorlick R, et al. Initial testing of the MDM2 inhibitor RG7112 by the pediatric preclinical testing program. Pediatr Blood Cancer 2013;60:633-41.
43. Richmond J, Carol H, Evans K, High L, Mendomo A, Robbins A, et al. Effective targeting of the P53-MDM2 axis in preclinical models of infant MLL-rearranged acute lymphoblastic leukemia. Clin Cancer Res 2015;21: 1395-405.
44. Moradi Manesh D, El-Hoss J, Evans K, Richmond J, Toscan CE, Bracken LS, et al. AKR1C3 is a biomarker of sensitivity to PR-104 in preclinical models of T-cell acute lymphoblastic leukemia. Blood 2015;126:1193-202.
45. Houghton PJ, Morton CL, Gorlick R, Kolb EA, Keir ST, Reynolds CP, et al. Initial testing of a monoclonal antibody (IMC-A12) against IGF-1R by the pediatric preclinical testing program. Pediatr Blood Cancer 2010;54:921-6.
46. Pappo AS, Vassal G, Crowley JJ, Bolejack V, Hogendoorn PC, Chugh R, et al. A phase 2 trial of R1507, a monoclonal antibody to the insulin-like growth factor-1 receptor (IGF-1R), in patients with recurrent or refractory rhabdomyosarcoma, osteosarcoma, synovial sarcoma, and other soft tissue sarcomas: results of a Sarcoma Alliance for Research Through Collaboration study. Cancer 2014;120:2448-56.
47. Taylor JGt, Cheuk AT, Tsang PS, Chung JY, Song YK, Desai K, et al. Identification of FGFR4-activating mutations in human rhabdomyosarcomas that promote metastasis in xenotransplanted models. J Clin Invest 2009;119:3395-407.
48. Keir ST, Maris JM, Reynolds CP, Kang MH, Kolb EA, Gorlick R, et al. Initial testing (stage 1) of temozolomide by the pediatric preclinical testing program. Pediatr Blood Cancer 2013;60:783-90.