Testing of the topoisomerase 1 inhibitor Genz-644282 by the pediatric preclinical testing program

Pediatric Blood and Cancer

Volumn 58, Issue 2, 2012, Pages 200-209

  Houghton, Peter J ^a   Lock, Richard ^b   Carol, Hernan ^b   Morton, Christopher L ^c   Gorlick, Richard ^d   Anders Kolb, E ^e   Keir, Stephen T ^f   Reynolds, C Patrick ^g   Kang, Min H ^g   Maris, John M ^h   Billups, Catherine A ^b   Zhang, Mindy X ⁱ   Madden, Stephen L ⁱ   Teicher, Beverly A ^j   Smith, Malcolm A ^j  

^a NATIONWIDE CHILDREN S HOSPITAL   (United States)

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

^c ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^d CHILDREN S HOSPITAL AT MONTEFIORE   (United States)

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

^f DUKE UNIVERSITY MEDICAL CENTER   (United States)

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

^h UNIVERSITY OF PENNSYLVANIA   (United States)

ⁱ GENZYME CORPORATION   (United States)

^j NATIONAL CANCER INSTITUTE   (United States)

more..

Author keywords

Developmental therapeutics; Genz 644282; Preclinical testing

Indexed keywords

DNA TOPOISOMERASE; DNA TOPOISOMERASE INHIBITOR; GENZ 644282; MESSENGER RNA; TOPOTECAN; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANTINEOPLASTIC ACTIVITY; ARTICLE; CANCER CELL CULTURE; CONTROLLED STUDY; DOSE RESPONSE; DRUG CYTOTOXICITY; DRUG EXPOSURE; DRUG POTENCY; EVENT FREE SURVIVAL; FEMALE; HUMAN; HUMAN CELL; IC 50; IN VITRO STUDY; IN VIVO STUDY; MAXIMUM TOLERATED DOSE; MOUSE; MULTIPLE CYCLE TREATMENT; NONHUMAN; PRIORITY JOURNAL; SOLID TUMOR; TUMOR MODEL;

ANIMALS; CELL PROLIFERATION; CHILD; DISEASE-FREE SURVIVAL; DNA TOPOISOMERASES, TYPE I; DRUG EVALUATION, PRECLINICAL; DRUG RESISTANCE, NEOPLASM; FEMALE; GENE EXPRESSION PROFILING; HUMANS; MICE; MICE, INBRED BALB C; MICE, INBRED NOD; MICE, SCID; NAPHTHYRIDINES; NEOPLASMS, EXPERIMENTAL; SURVIVAL RATE; TOPOISOMERASE I INHIBITORS; TOPOTECAN; TUMOR BURDEN; TUMOR CELLS, CULTURED; XENOGRAFT MODEL ANTITUMOR ASSAYS;

EID: 83455188335     PISSN: 15455009     EISSN: 15455017     Source Type: Journal    
DOI: 10.1002/pbc.23016     Document Type: Article

References (37)

1. Carol H, Houghton PJ, Morton CL, et al. Initial testing of topotecan by the pediatric preclinical testing program. Pediatr Blood Cancer 2010; 54: 707-715.
2. Houghton PJ, Cheshire PJ, Hallman JD II, et al. Efficacy of topoisomerase I inhibitors, topotecan and irinotecan, administered at low dose levels in protracted schedules to mice bearing xenografts of human tumors. Cancer Chemother Pharmacol 1995; 36: 393-403.
3. Houghton PJ, Cheshire PJ, Myers L, et al. Evaluation of 9-dimethylaminomethyl-10-hydroxycamptothecin against xenografts derived from adult and childhood solid tumors. Cancer Chemother Pharmacol 1992; 31: 229-239.
4. Thompson J, Zamboni WC, Cheshire PJ, et al. Efficacy of systemic administration of irinotecan against neuroblastoma xenografts. Clin Cancer Res 1997; 3: 423-431.
5. Vassal G, Boland I, Santos A, et al. Potent therapeutic activity of irinotecan (CPT-11) and its schedule dependency in medulloblastoma xenografts in nude mice. Int J Cancer 1997; 73: 156-163.
6. Vassal G, Couanet D, Stockdale E, et al. Phase II trial of irinotecan in children with relapsed or refractory rhabdomyosarcoma: A joint study of the French Society of Pediatric Oncology and the United Kingdom Children's Cancer Study Group. J Clin Oncol 2007; 25: 356-361.
7. Santana VM, Furman WL, Billups CA, et al. Improved response in high-risk neuroblastoma with protracted topotecan administration using a pharmacokinetically guided dosing approach. J Clin Oncol 2005; 23: 4039-4047.
8. Furman WL, Stewart CF, Poquette CA, et al. Direct translation of a protracted irinotecan schedule from a xenograft model to a phase I trial in children. J Clin Oncol 1999; 17: 1815-1824.
9. Hsiang YH, Lihou MG, Liu LF. Arrest of replication forks by drug-stabilized topoisomerase I-DNA cleavable complexes as a mechanism of cell killing by camptothecin. Cancer Res 1989; 49: 5077-5082.
10. Koster DA, Crut A, Shuman S, et al. Cellular strategies for regulating DNA supercoiling: A single-molecule perspective. Cell 2010; 142: 519-530.
11. Koster DA, Palle K, Bot ES, et al. Antitumour drugs impede DNA uncoiling by topoisomerase I. Nature 2007; 448: 213-217.
12. Tubergen DG, Stewart CF, Pratt CB, et al. Phase I trial and pharmacokinetic (PK) and pharmacodynamics (PD) study of topotecan using a five-day course in children with refractory solid tumors: A pediatric oncology group study. J Pediatr Hematol Oncol 1996; 18: 352-361.
13. Furman WL, Crews KR, Billups C, et al. Cefixime allows greater dose escalation of oral irinotecan: A phase I study in pediatric patients with refractory solid tumors. J Clin Oncol 2006; 24: 563-570.
14. Takimoto CH, Kieffer LV, Arbuck SG. DNA topoisomerase I inhibitors. Cancer Chemother Biol Resp Modif 1997; 17: 80-113.
15. Takimoto CH, Arbuck SG. Clinical status and optimal use of topotecan. Oncology (Williston Park) 1997; 11: 1635-1646; discussion 1649-1651, 1655-1637.
16. Creemers GJ, Wanders J, Gamucci T, et al. Topotecan in colorectal cancer: A phase II study of the EORTC early clinical trials group. Ann Oncol 1995; 6: 844-846.
17. Li TK, Houghton PJ, Desai SD, et al. Characterization of ARC-111 as a novel topoisomerase I-targeting anticancer drug. Cancer Res 2003; 63: 8400-8407.
18. Kurtzberg LS, Battle T, Rouleau C, et al. Bone marrow and tumor cell colony-forming units and human tumor xenograft efficacy of noncamptothecin and camptothecin topoisomerase I inhibitors. Mol Cancer Ther 2008; 7: 3212-3222.
19. Frgala T, Kalous O, Proffitt RT, et al. A fluorescence microplate cytotoxicity assay with a 4-log dynamic range that identifies synergistic drug combinations. Mol Cancer Ther 2007; 6: 886-897.
20. Houghton PJ, Morton CL, Kolb EA, et al. Initial testing (stage 1) of the proteasome inhibitor bortezomib by the pediatric preclinical testing program. Pediatr Blood Cancer 2008; 50: 37-45.
21. Sebaugh JL. Guidelines for accurate EC50/IC50 estimation. Pharmaceut Statist 2010; DOI: 10.1002/pst.426.
22. Friedman HS, Colvin OM, Skapek SX, et al. Experimental chemotherapy of human medulloblastoma cell lines and transplantable xenografts with bifunctional alkylating agents. Cancer Res 1988; 48: 4189-4195.
23. Graham C, Tucker C, Creech J, et al. Evaluation of the antitumor efficacy, pharmacokinetics, and pharmacodynamics of the histone deacetylase inhibitor depsipeptide in childhood cancer models in vivo. Clin Cancer Res 2006; 12: 223-234.
24. Peterson JK, Tucker C, Favours E, et al. In vivo evaluation of ixabepilone (BMS 247550), a novel epothilone B derivative, against pediatric cancer models. Clin Cancer Res 2005; 11: 6950-6958.
25. Liem NL, Papa RA, Milross CG, et al. Characterization of childhood acute lymphoblastic leukemia xenograft models for the preclinical evaluation of new therapies. Blood 2004; 103: 3905-3914.
26. Houghton PJ, Morton CL, Tucker C, et al. The Pediatric Preclinical Testing Program: Description of models and early testing results. Pediatr Blood Cancer 2007; 49: 928-940.
27. Plowman JCR, Alley M, Sausville E, Schepartz S. US-NCI testing procedures. In: Feibig HH, editor. Relevance of tumor models for anticancer drug development. Basel: Karger; 1999. pp. 121-135.
28. Irizarry RA, Hobbs B, Collin F, et al. Exploration, normalization, and summaries of high density oligonucleotide array probe level data. Biostatistics 2003; 4: 249-264.
29. Bolstad BM, Irizarry RA, Astrand M, et al. A comparison of normalization methods for high density oligonucleotide array data based on variance and bias. Bioinformatics 2003; 19: 185-193.
30. West M, Blanchette C, Dressman H, et al. Predicting the clinical status of human breast cancer by using gene expression profiles. Proc Natl Acad Sci USA 2001; 98: 11462-11467.
31. Bild AH, Yao G, Chang JT, et al. Oncogenic pathway signatures in human cancers as a guide to targeted therapies. Nature 2006; 439: 353-357.
32. Neale G, Su X, Morton CL, et al. Molecular characterization of the pediatric preclinical testing panel. Clin Cancer Res 2008; 14: 4572-4583.
33. Peterson JK, Houghton PJ. Integrating pharmacology and in vivo cancer models in preclinical and clinical drug development. Eur J Cancer 2004; 40: 837-844.
34. Masubuchi N, May RD, Atsumi R. A predictive model of human myelotoxicity using five camptothecin derivatives and the in vitro colony-forming unit granulocyte/macrophage assay. Clin Cancer Res 2004; 10: 6722-6731.
35. Erickson-Miller CL, May RD, Tomaszewski J, et al. Differential toxicity of camptothecin, topotecan and 9-aminocamptothecin to human, canine, and murine myeloid progenitors (CFU-GM) in vitro. Cancer Chemother Pharmacol 1997; 39: 467-472.
36. Kurtzberg L, Yao M, Rouleau C, et al. Genz-644282 a novel non-camptothecin topoisomerase I inhibitor for cancer treatment. Mol Cancer Ther Volume 8 2009; Suppl (AACR-NCI-EORTC Molecular Targets Conference):Abstr #C217.
37. Kurtzberg LS, Roth S, Krumbholz R, et al. Genz-644282, a novel non-camptothecin topoisomerase I inhibitor for cancer treatment. Clin Cancer Res 2011. [Epub ahead of print].