Abstract
Despite the generally held notion that embryonal cancers are simpler from a genetic standpoint than adult neoplasms, these cancers show multiple chromosomal rearrangements, suggesting a complicated series of acquired alterations during malignant evolution. This is best exemplified in neuroblastoma, an enigmatic and highly heterogeneous pediatric neoplasm that accounts for 15% of pediatric cancer mortality and remains an important clinical problem in which tumor genomics correlate with disease phenotype (George et al. 2007). Neuroblastoma represents a spectrum of diseases, and distinct patient subsets exist based on tumor biological features (Maris 2005). Low-risk neuroblastomas are likely characterized by mitotic dysfunction leading to a hyperdiploid modal karyotype with whole chromosome gains, but few, if any, structural cytogenetic rearrangements. These tumors may regress spontaneously, differentiate, or respond completely to modest doses of chemotherapy. In contrast, aggressive neuroblastomas are characterized by complex segmental chromosomal aberrations. Two main categories of unfavorable neuroblastomas exist, with a well-defined subset (approximately 40%) defined by deletion of the short arm of chromosome 1 and MYCN amplification. On the other hand, the majority of aggressive neuroblastomas show multiple other chromosomal rearrangements, most frequently involving chromosome 11q (Attiyeh et al. 2005).
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Alimonti A, Satta F, Pavese I, Burattini E, Zoffoli V, Vecchione A. 2003. Prevention of irinotecan plus 5-fluorouracil/leucovorin-induced diarrhoea by oral administration of neomycin plus bacitracin in first-line treatment of advanced colorectal cancer. Ann Oncol 14(5):805–6.
Allenspach EJ, Maillard I, Aster JC, Pear WS. 2002. Notch signaling in cancer. Cancer Biol Ther 1(5):466–76.
Alvarado CS, London WB, Look AT, Brodeur GM, Altmiller DH, Thorner PS, Joshi VV, Rowe ST, Nash MB, Smith EI, et al. 2000. Natural history and biology of stage a neuroblastoma: a Pediatric Oncology Group Study. J Pediatr Hematol Oncol 22(3):197–205.
Anderson CP, Reynolds CP. 2002. Synergistic cytotoxicity of buthionine sulfoximine (BSO) and intensive melphalan (L-PAM) for neuroblastoma cell lines established at relapse after myeloablative therapy. Bone Marrow Transplant 30(3):135–40.
Attiyeh EF, London WB, Mosse YP, Wang Q, Winter C, Khazi D, McGrady PW, Seeger RC, Look AT, Shimada H, et al. 2005. Chromosome 1p and 11q deletions and outcome in neuroblastoma. N Engl J Med 353(21):2243–53.
Azar C, Scavarda NJ, Reynolds CP, Brodeur GM. 1990. Multiple defects of the nerve growth factor receptor in human neuroblastomas. Cell Growth Differ 1:421–428.
Bagatell R, Gore L, Egorin MJ, Ho R, Heller G, Boucher N, Zuhowski EG, Whitlock JA, Hunger SP, Narendran A, et al. 2007. Phase I pharmacokinetic and pharmacodynamic study of 17-N-allylamino-17-demethoxygeldanamycin in pediatric patients with recurrent or refractory solid tumors: a pediatric oncology experimental therapeutics investigators consortium study. Clin Cancer Res 13(6):1783–8.
Baker DL, Reddy UR, Pleasure D, Thorpe CL, Evans AE, Cohen PS, Ross AH. 1989. Analysis of nerve growth factor receptor expression in human neuroblastoma and neuroepithelioma cell lines. Cancer Res 49:4142–6.
Banerjee D. 2001. Genasense (Genta Inc). Curr Opin Investig Drugs 2(4):574–80.
Barker E, Mueller BM, Handgretinger R, Herter M, Yu AL, Reisfeld RA. 1991. Effect of a chimeric anti-ganglioside GD2 antibody on cell-mediated lysis of human neuroblastoma cells. Cancer Res 51(1):144–9.
Beheshti B, Braude I, Marrano P, Thorner P, Zielenska M, Squire JA. 2003. Chromosomal localization of DNA amplifications in neuroblastoma tumors using cDNA microarray comparative genomic hybridization. Neoplasia 5(1):53–62.
Beppu K, Jaboine J, Merchant MS, Mackall CL, Thiele CJ. 2004. Effect of imatinib mesylate on neuroblastoma tumorigenesis and vascular endothelial growth factor expression. J Natl Cancer Inst 96(1):46–55.
Berthold F, Boos J, Burdach S, Erttmann R, Henze G, Hermann J, Klingebiel T, Kremens B, Schilling FH, Schrappe M, et al. 2005. Myeloablative megatherapy with autologous stem-cell rescue versus oral maintenance chemotherapy as consolidation treatment in patients with high-risk neuroblastoma: a randomised controlled trial. Lancet Oncol 6(9):649–58.
Berthold F, Treuner J, Brandeis WE, et al. 1982. Neuroblastoma study NBL-79 of the German Society for Pediatric Oncology; report after 2 years. Klin Padiatr 194:262–9.
Bilke S, Chen QR, Whiteford CC, Khan J. 2005. Detection of low level genomic alterations by comparative genomic hybridization based on cDNA micro-arrays. Bioinformatics 21(7):1138–45.
Bolesta E, Kowalczyk A, Wierzbicki A, Rotkiewicz P, Bambach B, Tsao CY, Horwacik I, Kolinski A, Rokita H, Brecher M, et al. 2005. DNA vaccine expressing the mimotope of GD2 ganglioside induces protective GD2 cross-reactive antibody responses. Cancer Res 65(8):3410–8.
Borgstrom P, Hassan M, Wassberg E, Refai E, Jonsson C, Larsson SA, Jacobsson H, Kogner P. 1999. The somatostatin analogue octreotide inhibits neuroblastoma growth in vivo. Pediatr Res 46(3):328–32.
Brignole C, Marimpietri D, Pastorino F, Nico B, Di Paolo D, Cioni M, Piccardi F, Cilli M, Pezzolo A, Corrias MV, et al. 2006. Effect of bortezomib on human neuroblastoma cell growth, apoptosis, and angiogenesis. J Natl Cancer Inst 98(16):1142–57.
Brodeur GM. 2003. Neuroblastoma: biological insights into a clinical enigma. Nat Rev Cancer 3(3):203–16.
Brodeur GM, Nakagawara A, Yamashiro DJ, Ikegaki N, Liu X-G, Lee CP, Evans AE. 1996. Expression of TrkA, TrkB, and TrkC in human neuroblastomas. J Neurooncol 12:37–41.
Carr J, Bell E, Pearson AD, Kees UR, Beris H, Lunec J, Tweddle DA. 2006. Increased frequency of aberrations in the p53/MDM2/p14(ARF) pathway in neuroblastoma cell lines established at relapse. Cancer Res 66(4):2138–45.
Chen QR, Bilke S, Wei JS, Whiteford CC, Cenacchi N, Krasnoselsky AL, Greer BT, Son CG, Westermann F, Berthold F, et al. 2004. cDNA array-CGH profiling identifies genomic alterations specific to stage and MYCN-amplification in neuroblastoma. BMC Genomics 5(1):70.
Chenn A, Walsh CA. 2002. Regulation of cerebral cortical size by control of cell cycle exit in neural precursors. Science 297(5580):365–9.
Cheung IY, Cheung NK. 2001. Quantitation of marrow disease in neuroblastoma by real-time reverse transcription-PCR. Clin Cancer Res 7(6):1698–705.
Cheung NK, Kushner BH, Yeh SDJ, Larson SM. 1998. 3F8 monoclonal antibody treatment of patients with stage 4 neuroblastoma: a phase II study. Int J Oncol 12(6):1299–306.
Cheung NK, Landmeier B, Neely J, Nelson AD, Abramowsky C, Ellery S, Adams RB, Miraldi F. 1986. Complete tumor ablation with iodine 131-radiolabeled disialoganglioside GD2-specific monoclonal antibody against human neuroblastoma xenografted in nude mice. J Natl Cancer Inst 77(3):739–45.
Cheung NK, Modak S. 2002. Oral (1 → 3), (1 → 4)-beta-d-glucan synergizes with antiganglioside GD2 monoclonal antibody 3F8 in the therapy of neuroblastoma. Clin Cancer Res 8(5):1217–23.
Chlenski A, Liu S, Crawford SE, Volpert OV, DeVries GH, Evangelista A, Yang Q, Salwen HR, Farrer R, Bray J, et al. 2002. SPARC is a key Schwannian-derived inhibitor controlling neuroblastoma tumor angiogenesis. Cancer Res 62(24):7357–63.
Cho SY, Adamson PC, Hagey AE, Widemann BC, Maris JM, Fox E, Medina D, Cui Y, Gordon GB, Balis FM. 2004. Phase I trial and pharmacokinetic (PK) study of ABT-751, an orally bioavailable tubulin binding agent, in pediatric patients with refractory solid tumors. J Clin Oncol ASCO Annual Meeting Proceedings 22(14S):2080
Coffey DC, Kutko MC, Glick RD, Butler LM, Heller G, Rifkind RA, Marks PA, Richon VM, La Quaglia MP. 2001. The histone deacetylase inhibitor, CBHA, inhibits growth of human neuroblastoma xenografts in vivo, alone and synergistically with all-trans retinoic acid. Cancer Res 61(9):3591–4.
Cohn SL, Look AT, Joshi VV, Holbrook T, Salwen H, Chagnovich D, Chesler L, Rowe ST, Valentine MB, Komuro H, et al. 1995. Lack of correlation of N-myc gene amplification with prognosis in localized neuroblastoma: a Pediatric Oncology Group Study. Cancer Res 55:721–726.
Cooper R, Khakoo Y, Matthay KK, Lukens JN, Seeger RC, Stram DO, Gerbing RB, Nakagawa A, Shimada H. 2001. Opsoclonus-myoclonus-ataxia syndrome in neuroblastoma: histopathologic features – a report from the Children’s Cancer Group. Med Pediatr Oncol 36(6):623–9.
D’Assoro AB, Lingle WL, Salisbury JL. 2002. Centrosome amplification and the development of cancer. Oncogene 21(40):6146–53.
Dam V, Morgan BT, Mazanek P, Hogarty MD. 2006. Mutations in PIK3CA are infrequent in neuroblastoma. BMC Cancer 6:177.
Dickson PV, Hamner JB, Sims TL, Fraga CH, Ng CY, Rajasekeran S, Hagedorn NL, McCarville MB, Stewart CF, Davidoff AM. 2007. Bevacizumab-induced transient remodeling of the vasculature in neuroblastoma xenografts results in improved delivery and efficacy of systemically administered chemotherapy. Clin Cancer Res 13(13):3942–50.
DuBois SG, Messina J, Maris JM, Huberty J, Glidden DV, Veatch J, Charron M, Hawkins R, Matthay KK. 2004. Hematologic toxicity of high-dose iodine-131-metaiodobenzylguanidine therapy for advanced neuroblastoma. J Clin Oncol 22(12):2452–60.
Eggert A, Ikegaki N, Kwiatkowski J, Zhao H, Brodeur GM, Himelstein BP. 2000. High-level expression of angiogenic factors is associated with advanced tumor stage in human neuroblastomas. Clin Cancer Res 6(5):1900–8.
Erdreich-Epstein A, Shimada H, Groshen S, Liu M, Metelitsa LS, Kim KS, Stins MF, Seeger RC, Durden DL. 2000. Integrins alpha(v)beta3 and alpha(v)beta5 are expressed by endothelium of high-risk neuroblastoma and their inhibition is associated with increased endogenous ceramide. Cancer Res 60(3):712–21.
Evans AE, Kisselbach KD, Liu X, Eggert A, Ikegaki N, Camoratto AM, Dionne C, Brodeur GM. 2001. Effect of CEP-751 (KT-6587) on neuroblastoma xenografts expressing TrkB. Med Pediatr Oncol 36(1):181–4.
Evans AE, Kisselbach KD, Yamashiro DJ, Ikegaki N, Camoratto AM, Dionne CA, Brodeur GM. 1999. Antitumor activity of CEP-751 (KT-6587) on human neuroblastoma and medulloblastoma xenografts. Clin Cancer Res 5(11):3594–602.
Evans AE, Silber JH, Shpilsky A, D’Angio GJ. 1996. Successful management of low-stage neuroblastoma without adjuvant therapies: a comparison of two decades, 1972 through 1981 and 1982 through 1992, in a single institution. J Clin Oncol 14(9):2504–10.
Fotsis T, Breit S, Lutz W, Rossler J, Hatzi E, Schwab M, Schweigerer L. 1999. Down-regulation of endothelial cell growth inhibitors by enhanced MYCN oncogene expression in human neuroblastoma cells. Eur J Biochem 263(3):757–64.
Fox E, Maris JM, Widemann BC, Meek K, Goodwin A, Goodspeed W, Kromplewski M, Fouts ME, Medina D, Cho SY, et al. 2006. A phase 1 study of ABT-751, an orally bioavailable tubulin inhibitor, administered daily for 7 days every 21 days in pediatric patients with solid tumors. Clin Cancer Res 12(16):4882–7.
Frantz CN, London WB, Diller L, Seeger R, Sawyer K. 2004. Recurrent neuroblastoma: Randomized treatment with topotecan + cyclophosphamide (T + C) vs. topotecan alone(T). A POG/CCG Intergroup Study. J Clin Oncol ASCO Annual Meeting Proceedings 22(14S):8512
Fukasawa K. 2005. Centrosome amplification, chromosome instability and cancer development. Cancer Lett 230(1):6–19.
Fukasawa K. 2007. Oncogenes and tumour suppressors take on centrosomes. Nat Rev Cancer 7(12):911–24.
Garaventa A, Bellagamba O, Lo Piccolo MS, Milanaccio C, Lanino E, Bertolazzi L, Villavecchia GP, Cabria M, Scopinaro G, Claudiani F, et al. 1999. 131I-metaiodobenzylguanidine (131I-MIBG) therapy for residual neuroblastoma: a mono-institutional experience with 43 patients. Br J Cancer 81(8):1378–84.
Garaventa A, Luksch R, Lo Piccolo MS, Cavadini E, Montaldo PG, Pizzitola MR, Boni L, Ponzoni M, Decensi A, De Bernardi B, et al. 2003. Phase I trial and pharmacokinetics of fenretinide in children with neuroblastoma. Clin Cancer Res 9(6):2032–9.
George R, Attiyeh E, Li S, Moreau L, Neuberg D, Li C, Fox EA, Meyerson M, Diller L, Fontina P, et al. 2007. Genome-wide analysis of neuroblastomas using high-density single nucleotide polymorphism arrays. PLoS One 2:e255.
Giles RH, van Es JH, Clevers H. 2003. Caught up in a Wnt storm: Wnt signaling in cancer. Biochim Biophys Acta 1653(1):1–24.
Goldsmith KC, Hogarty MD. 2005. Targeting programmed cell death pathways with experimental therapeutics: opportunities in high-risk neuroblastoma. Cancer Lett 228(1–2):133–41.
Gonzalez S, Naranjo A, Serrano LM, Chang WC, Wright CL, Jensen MC. 2004. Genetic engineering of cytolytic T lymphocytes for adoptive T-cell therapy of neuroblastoma. J Gene Med 6(6):704–11.
Ho R, Eggert A, Hishiki T, Minturn JE, Ikegaki N, Foster P, Camoratto AM, Evans AE, Brodeur GM. 2002. Resistance to chemotherapy mediated by TrkB in neuroblastomas. Cancer Res 62(22):6462–6.
Ho R, Minturn JE, Hishiki T, Zhao H, Wang Q, Cnaan A, Maris J, Evans AE, Brodeur GM. 2005. Proliferation of human neuroblastomas mediated by the epidermal growth factor receptor. Cancer Res 65(21):9868–75.
Houghton PJ, Morton CL, Tucker C, Payne D, Favours E, Cole C, Gorlick R, Kolb EA, Zhang W, Lock R, et al. 2007. The pediatric preclinical testing program: description of models and early testing results. Pediatr Blood Cancer 49(7):928–40.
Howard JP, Maris JM, Kersun LS, Huberty JP, Cheng SC, Hawkins RA, Matthay KK. 2005. Tumor response and toxicity with multiple infusions of high dose (131)I-MIBG for refractory neuroblastoma. Pediatr Blood Cancer 44(3):232–9.
Huang D, Rutkowski JL, Brodeur GM, Chou PM, Kwiatkowski JL, Babbo A, Cohn SL. 2000. Schwann cell-conditioned medium inhibits angiogenesis. Cancer Res 60(21):5966–71.
Jaboin J, Kim CJ, Kaplan DR, Thiele CJ. 2002a. Brain-derived neurotrophic factor activation of TrkB protects neuroblastoma cells from chemotherapy-induced apoptosis via phosphatidylinositol 3′-kinase pathway. Cancer Res 62(22):6756–63.
Jaboin J, Wild J, Hamidi H, Khanna C, Kim CJ, Robey R, Bates SE, Thiele CJ. 2002b. MS-27-275, an inhibitor of histone deacetylase, has marked in vitro and in vivo antitumor activity against pediatric solid tumors. Cancer Res 62(21):6108–15.
Kang TI, Brophy P, Hickeson M, Heyman S, Evans AE, Charron M, Maris JM. 2003. Targeted radiotherapy with submyeloablative doses of 131I-MIBG is effective for disease palliation in highly refractory neuroblastoma. J Pediatr Hematol Oncol 25(10):769–73.
Katzenstein HM, Rademaker AW, Senger C, Salwen HR, Nguyen NN, Thorner PS, Litsas L, Cohn SL. 1999. Effectiveness of the angiogenesis inhibitor TNP-470 in reducing the growth of human neuroblastoma in nude mice inversely correlates with tumor burden. Clin Cancer Res 5(12):4273–8.
Keshelava N, Davicioni E, Wan Z, Ji L, Sposto R, Triche TJ, Reynolds CP. 2007. Histone deacetylase 1 gene expression and sensitization of multidrug-resistant neuroblastoma cell lines to cytotoxic agents by depsipeptide. J Natl Cancer Inst 99(14):1107–19.
Keshelava N, Zuo JJ, Chen P, Waidyaratne SN, Luna MC, Gomer CJ, Triche TJ, Reynolds CP. 2001. Loss of p53 function confers high-level multidrug resistance in neuroblastoma cell lines. Cancer Res 61(16):6185–93.
King DM, Albertini MR, Schalch H, Hank JA, Gan J, Surfus J, Mahvi D, Schiller JH, Warner T, Kim K, et al. 2004. Phase I clinical trial of the immunocytokine EMD 273063 in melanoma patients. J Clin Oncol 22(22):4463–73.
Kogner P, Barbany G, Dominici C, Castello MA, Raschella G, Persson H. 1993. Coexpression of messenger RNA for TRK protooncogene and low affinity nerve growth factor receptor in neuroblastoma with favorable prognosis. Cancer Res 53:2044–50.
Kushner BH, Cheung NK. 1989. GM-CSF enhances 3F8 monoclonal antibody-dependent cellular cytotoxicity against human melanoma and neuroblastoma. Blood 73(7):1936–41.
Kushner BH, Cheung NK, LaQuaglia MP, Ambros PF, Ambros IM, Bonilla MA, Gerald WL, Ladanyi M, Gilbert F, Rosenfield NS, et al. 1996a. Survival from locally invasive or widespread neuroblastoma without cytotoxic therapy. J Clin Oncol 14(2):373–81.
Kushner BH, Cheung NK, LaQuaglia MP, Ambros PF, Ambros IM, Bonilla MA, Ladanyi M, Gerald WL. 1996b. International neuroblastoma staging system stage 1 neuroblastoma: a prospective study and literature review. J Clin Oncol 14(7):2174–80.
Kushner BH, Kramer K, Cheung NK. 2001. Phase II trial of the anti-GD2 monoclonal antibody 3F8 and granulocyte-macrophage colony-stimulating factor for neuroblastoma. J Clin Oncol 19:4189–94.
Langler A, Christaras A, Abshagen K, Krauth K, Hero B, Berthold F. 2002. Topotecan in the treatment of refractory neuroblastoma and other malignant tumors in childhood – a phase-II-study. Klin Padiatr 214(4):153–6.
Lode HN, Xiang R, Duncan SR, Theofilopoulos AN, Gillies SD, Reisfeld RA. 1999. Tumor-targeted IL-2 amplifies T cell-mediated immune response induced by gene therapy with single-chain IL-12. Proc Natl Acad Sci USA 96(15):8591–6.
Maris JM. 2005. The biologic basis for neuroblastoma heterogeneity and risk stratification. Curr Opin Pediatr 17(1):7–13.
Maris JM, Courtright J, Houghton PJ, Morton CL, Gorlick R, Kolb EA, Lock R, Tajbakhsh M, Reynolds CP, Keir ST, et al. 2008. Initial testing of the VEGFR inhibitor AZD2171 by the pediatric preclinical testing program. Pediatr Blood Cancer 50(3):581–7.
Marks PA, Richon VM, Breslow R, Rifkind RA. 2001. Histone deacetylase inhibitors as new cancer drugs. Curr Opin Oncol 13(6):477–83.
Marshall JL, Kindler H, Deeken J, Bhargava P, Vogelzang NJ, Rizvi N, Luhtala T, Boylan S, Dordal M, Robertson P, et al. 2005. Phase I trial of orally administered CEP-701, a novel neurotrophin receptor-linked tyrosine kinase inhibitor. Invest New Drugs 23(1):31–7.
Matthay KK, DeSantes K, Hasegawa B, Huberty J, Hattner RS, Ablin A, Reynolds CP, Seeger RC, Weinberg VK, Price D. 1998a. Phase I dose escalation of 131I-metaiodobenzylguanidine with autologous bone marrow support in refractory neuroblastoma. J Clin Oncol 16(1):229–36.
Matthay KK, Perez C, Seeger RC, Brodeur GM, Shimada H, Atkinson JB, Black CT, Gerbing R, Haase GM, Stram DO, et al. 1998b. Successful treatment of stage III neuroblastoma based on prospective biologic staging: a Children’s Cancer Group Study. J Clin Oncol 16(4):1256–64.
Matthay KK, Sather HN, Seeger RC, Haase GM, Hammond GD. 1989. Excellent outcome of stage II neuroblastoma is independent of residual disease and radiation therapy. J Clin Oncol 7(2):236–44.
Matthay KK, Tan JC, Villablanca JG, Yanik GA, Veatch J, Franc B, Twomey E, Horn B, Reynolds CP, Groshen S, et al. 2006. Phase I dose escalation of iodine-131-metaiodobenzylguanidine with myeloablative chemotherapy and autologous stem-cell transplantation in refractory neuroblastoma: a new approaches to Neuroblastoma Therapy Consortium Study. J Clin Oncol 24(3):500–6.
Matthay KK, Villablanca JG, Seeger RC, Stram DO, Harris RE, Ramsay NK, Swift P, Shimada H, Black CT, Brodeur GM, et al. 1999. Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. Children’s Cancer Group. N Engl J Med 341(16):1165–73.
Matthay KK, Yanik G, Messina J, Quach A, Huberty J, Cheng SC, Veatch J, Goldsby R, Brophy P, Kersun LS, et al. 2007. Phase II study on the effect of disease sites, age, and prior therapy on response to iodine-131-metaiodobenzylguanidine therapy in refractory neuroblastoma. J Clin Oncol 25(9):1054–60.
Maurer BJ, Melton L, Billups C, Cabot MC, Reynolds CP. 2000. Synergistic cytotoxicity in solid tumor cell lines between N-(4-hydroxyphenyl)retinamide and modulators of ceramide metabolism. J Natl Cancer Inst 92(23):1897–909.
Maurer BJ, Metelitsa LS, Seeger RC, Cabot MC, Reynolds CP. 1999. Increase of ceramide and induction of mixed apoptosis/necrosis by N-(4-hydroxyphenyl)-retinamide in neuroblastoma cell lines [see comments]. J Natl Cancer Inst 91(13):1138–46.
Meitar D, Crawford SE, Rademaker AW, Cohn SL. 1996. Tumor angiogenesis correlates with metastatic disease, N-Myc amplification, and poor outcome in human neuroblastoma. J Clin Oncol 14(2):405–14.
Miller BS, Yee D. 2005. Type I insulin-like growth factor receptor as a therapeutic target in cancer. Cancer Res 65(22):10123–7.
Morowitz MJ, Barr R, Wang Q, King R, Rhodin N, Pawel B, Zhao H, Erickson SA, Sheppard GS, Wang J, et al. 2005. Methionine aminopeptidase 2 inhibition is an effective treatment strategy for neuroblastoma in preclinical models. Clin Cancer Res 11(7):2680–5.
Mosse YP, Greshock J, Margolin A, Naylor T, Cole K, Khazi D, Hii G, Winter C, Shahzad S, Asziz MU, et al. 2005a. High-resolution detection and mapping of genomic DNA alterations in neuroblastoma. Genes Chromosomes Cancer 43(4):390–403.
Mosse YP, Greshock J, Weber BL, Maris JM. 2005b. Measurement and relevance of neuroblastoma DNA copy number changes in the post-genome era. Cancer Lett 228(1–2):83–90.
Murray JL, Cunningham JE, Brewer H, Mujoo K, Zukiwski AA, Podoloff DA, Kasi LP, Bhadkamkar V, Fritsche HA, Benjamin RS, et al. 1994. Phase I trial of murine monoclonal antibody 14G2a administered by prolonged intravenous infusion in patients with neuroectodermal tumors. J Clin Oncol 12(1):184–93.
Nakagawara A, Arima M, Azar CG, Scavarda NJ, Brodeur GM. 1992. Inverse relationship between trk expression and N-myc amplification in human neuroblastomas. Cancer Res 52:1364–1368.
Nakagawara A, Arima-Nakagawara M, Scavarda NJ, Azar CG, Cantor AB, Brodeur GM. 1993. Association between high levels of expression of the TRK gene and favorable outcome in human neuroblastoma. N Engl J Med 328(12):847–54.
Nakagawara A, Azar CG, Scavarda NJ, Brodeur GM. 1994. Expression and function of TRK-B and BDNF in human neuroblastomas. Mol Cell Biol 14:759–67.
Neal ZC, Yang JC, Rakhmilevich AL, Buhtoiarov IN, Lum HE, Imboden M, Hank JA, Lode HN, Reisfeld RA, Gillies SD, et al. 2004. Enhanced activity of hu14.18-IL2 immunocytokine against murine NXS2 neuroblastoma when combined with interleukin 2 therapy. Clin Cancer Res 10(14):4839–47.
Nishihira H, Toyoda Y, Tanaka Y, Ijiri R, Aida N, Takeuchi M, Ohnuma K, Kigasawa H, Kato K, Nishi T. 2000. Natural course of neuroblastoma detected by mass screening: s 5-year prospective study at a single institution. J Clin Oncol 18(16):3012–7.
O’Dorisio MS, Hauger M, Cecalupo AJ. 1994. Somatostatin receptors in neuroblastoma: diagnostic and therapeutic implications. Semin Oncol 21(5 Suppl 13):33–7.
Osenga KL, Hank JA, Albertini MR, Gan J, Sternberg AG, Eickhoff J, Seeger RC, Matthay KK, Reynolds CP, Twist C, et al. 2006. A phase I clinical trial of the hu14.18-IL2 (EMD 273063) as a treatment for children with refractory or recurrent neuroblastoma and melanoma: a study of the Children’s Oncology Group. Clin Cancer Res 12(6):1750–9.
Oue T, Inoue M, Yoneda A, Kubota A, Okuyama H, Kawahara H, Nishikawa M, Nakayama M, Kawa K. 2005. Profile of neuroblastoma detected by mass screening, resected after observation without treatment: results of the Wait and See pilot study. J Pediatr Surg 40(2):359–63.
Ozkaynak MF, Sondel PM, Krailo MD, Gan J, Javorsky B, Reisfeld RA, Matthay KK, Reaman GH, Seeger RC. 2000. Phase I study of chimeric human/murine anti-ganglioside G(D2) monoclonal antibody (ch14.18) with granulocyte-macrophage colony-stimulating factor in children with neuroblastoma immediately after hematopoietic stem-cell transplantation: a Children’s Cancer Group Study. J Clin Oncol 18(24):4077–85.
Perez CA, Matthay KK, Atkinson JB, Seeger RC, Shimada H, Haase GM, Stram DO, Gerbing RB, Lukens JN. 2000. Biologic variables in the outcome of stages I and II neuroblastoma treated with surgery as primary therapy: a Children’s Cancer Group Study. J Clin Oncol 18(1):18–26.
Reya T, Clevers H. 2005. Wnt signalling in stem cells and cancer. Nature 434(7035):843–50.
Reynolds CP. 2004. Detection and treatment of minimal residual disease in high-risk neuroblastoma. Pediatr Transplant 8(Suppl 5):56–66.
Reynolds CP, Matthay KK, Villablanca JG, Maurer BJ. 2003. Retinoid therapy of high-risk neuroblastoma. Cancer Lett 197(1–2):185–92.
Reynolds CP, Maurer BJ, Kolesnick RN. 2004. Ceramide synthesis and metabolism as a target for cancer therapy. Cancer Lett 206(2):169–80.
Rodriguez-Galindo C, Crews KR, Stewart CF, Furman W, Panetta JC, Daw NC, Cain A, Tan M, Houghton PH, Santana VM. 2006. Phase I study of the combination of topotecan and irinotecan in children with refractory solid tumors. Cancer Chemother Pharmacol 57:15–24.
Rousseau RF, Haight AE, Hirschmann-Jax C, Yvon ES, Rill DR, Mei Z, Smith SC, Inman S, Cooper K, Alcoser P, et al. 2003. Local and systemic effects of an allogeneic tumor cell vaccine combining transgenic human lymphotactin with interleukin-2 in patients with advanced or refractory neuroblastoma. Blood 101(5):1718–26.
Ryden M, Sehgal R, Dominici C, Schilling FH, Ibanez CF, Kogner P. 1996. Expression of mRNA for the neurotrophin receptor trkC in neuroblastomas with favourable tumour stage and good prognosis. Br J Cancer 74(5):773–9.
Saylors RL, 3rd, Stine KC, Sullivan J, Kepner JL, Wall DA, Bernstein ML, Harris MB, Hayashi R, Vietti TJ. 2001. Cyclophosphamide plus topotecan in children with recurrent or refractory solid tumors: a Pediatric Oncology Group phase II study. J Clin Oncol 19(15):3463–9.
Schmidt ML, Lal A, Seeger RC, Maris JM, Shimada H, O’Leary M, Gerbing RB, Matthay KK. 2005. Favorable prognosis for patients 12 to 18 months of age with stage 4 nonamplified MYCN neuroblastoma: a Children’s Cancer Group Study. J Clin Oncol 23(27):6474–80.
Schmidt ML, Lukens JN, Seeger RC, Brodeur GM, Shimada H, Gerbing RB, Stram DO, Perez C, Haase GM, Matthay KK. 2000. Biologic factors determine prognosis in infants with stage IV neuroblastoma: a prospective Children’s Cancer Group Study. J Clin Oncol 18(6):1260–8.
Schramm A, Schulte JH, Klein-Hitpass L, Havers W, Sieverts H, Berwanger B, Christiansen H, Warnat P, Brors B, Eils J, et al. 2005. Prediction of clinical outcome and biological characterization of neuroblastoma by expression profiling. Oncogene 24(53):7902–12.
Sears R, Nuckolls F, Haura E, Taya Y, Tamai K, Nevins JR. 2000. Multiple Ras-dependent phosphorylation pathways regulate Myc protein stability. Genes Dev 14(19):2501–14.
Shah NP, Tran C, Lee FY, Chen P, Norris D, Sawyers CL. 2004. Overriding imatinib resistance with a novel ABL kinase inhibitor. Science 305(5682):399–401.
Shusterman S, Grupp SA, Barr R, Carpentieri D, Zhao H, Maris JM. 2001. The angiogenesis inhibitor TNP-470 effectively inhibits human neuroblastoma xenograft growth, especially in the setting of subclinical disease. Clin Cancer Res 7(4):977–84.
Siapati KE, Barker S, Kinnon C, Michalski A, Anderson R, Brickell P, Thrasher AJ, Hart SL. 2003. Improved antitumour immunity in murine neuroblastoma using a combination of IL-2 and IL-12. Br J Cancer 88(10):1641–8.
Sidell N. 1982. Retinoic acid-induced growth inhibition and morphologic differentiation of human neuroblastoma cells in vitro. J Natl Cancer Inst 68(4):589–96.
Slack C, Overton PM, Tuxworth RI, Chia W. 2007. Asymmetric localisation of Miranda and its cargo proteins during neuroblast division requires the anaphase-promoting complex/cyclosome. Development 134(21):3781–7.
Sohara Y, Shimada H, Scadeng M, Pollack H, Yamada S, Ye W, Reynolds CP, DeClerck YA. 2003. Lytic bone lesions in human neuroblastoma xenograft involve osteoclast recruitment and are inhibited by bisphosphonate. Cancer Res 63(12):3026–31.
Suita S, Zaizen Y, Sera Y, Takamatsu H, Mizote H, Ohgami H, Kurosaki N, Ueda K, Tasaka H, Miyazaki S, et al. 1996. Mass screening for neuroblastoma: quo vadis? A 9-year experience from the Pediatric Oncology Study Group of the Kyushu area in Japan. J Pediatr Surg 31(4):555–8.
Suzuki T, Bogenmann E, Shimada H, Stram D, Seeger RC. 1993. Lack of high-affinity nerve growth factor receptors in aggressive neuroblastomas. J Natl Cancer Inst 85:377–384.
Taipale J, Beachy PA. 2001. The Hedgehog and Wnt signalling pathways in cancer. Nature 411(6835):349–54.
Tanaka T, Sugimoto T, Sawada T. 1998. Prognostic discrimination among neuroblastomas according to Ha-ras/Trk A gene expression. Cancer 83:1626–33.
Teitz T, Wei T, Valentine MB, Vanin EF, Grenet J, Valentine VA, Behm FG, Look AT, Lahti JM, Kidd VJ. 2000. Caspase 8 is deleted or silenced preferentially in childhood neuroblastomas with amplification of MYCN. Nat Med 6:529–35.
Tepmongkol S, Heyman S. 1999. 131I MIBG therapy in neuroblastoma: mechanisms, rationale, and current status. Med Pediatr Oncol 32(6):427–31; discussion 432.
Thompson PM, Maris JM, Hogarty MD, Seeger RC, Reynolds CP, Brodeur GM, White PS. 2001. Homozygous deletion of CDKN2A (p16INK4a/p14ARF) but not within 1p36 or at other tumor suppressor loci in neuroblastoma. Cancer Res 61(2):679–86.
Tweddle DA, Malcolm AJ, Bown N, Pearson AD, Lunec J. 2001. Evidence for the development of p53 mutations after cytotoxic therapy in a neuroblastoma cell line. Cancer Res 61(1):8–13.
Vandesompele J, Speleman F, Van Roy N, Laureys G, Brinskchmidt C, Christiansen H, Lampert F, Lastowska M, Bown N, Pearson A, et al. 2001. Multicentre analysis of patterns of DNA gains and losses in 204 neuroblastoma tumors: how many genetic subgroups are there? Med Pediatr Oncol 36(1):5–10.
Vassal G, Doz F, Frappaz D, Imadalou K, Sicard E, Santos A, O’Quigley J, Germa C, Risse ML, Mignard D, et al. 2003. A phase I study of irinotecan as a 3-week schedule in children with refractory or recurrent solid tumors. J Clin Oncol 21(20):3844–52.
Vitali R, Cesi V, Nicotra MR, McDowell HP, Donfrancesco A, Mannarino O, Natali PG, Raschella G, Dominici C. 2003. c-Kit is preferentially expressed in MYCN-amplified neuroblastoma and its effect on cell proliferation is inhibited in vitro by STI-571. Int J Cancer 106(2):147–52.
Wagner LM, Crews KR, Iacono LC, Houghton PJ, Fuller CE, McCarville MB, Goldsby RE, Albritton K, Stewart CF, Santana VM. 2004. Phase I trial of temozolomide and protracted irinotecan in pediatric patients with refractory solid tumors. Clin Cancer Res 10(3):840–8.
Wang Q, Diskin S, Rappaport E, Attiyeh E, Mosse Y, Shue D, Seiser E, Jagannathan J, Shusterman S, Bansal M, et al. 2006. Integrative genomics identifies distinct molecular classes of neuroblastoma and shows that multiple genes are targeted by regional alterations in DNA copy number. Cancer Res 66(12):6050–62.
Wiseman GA, Kvols LK. 1995. Therapy of neuroendocrine tumors with radiolabeled MIBG and somatostatin analogues. Semin Nucl Med 25(3):272–8.
Yaari S, Jacob-Hirsch J, Amariglio N, Haklai R, Rechavi G, Kloog Y. 2005. Disruption of cooperation between Ras and MycN in human neuroblastoma cells promotes growth arrest. Clin Cancer Res 11(12):4321–30.
Yamamoto K, Hanada R, Kikuchi A, Ichikawa M, Aihara T, Oguma E, Moritani T, Shimanuki Y, Tanimura M, Hayashi Y. 1998. Spontaneous regression of localized neuroblastoma detected by mass screening. J Clin Oncol 16(4):1265–9.
Yamashiro DJ, Nakagawara A, Ikegaki N, Liu XG, Brodeur GM. 1996. Expression of TrkC in favorable human neuroblastomas. Oncogene 12(1):37–41.
Yang Q, Tian Y, Liu S, Zeine R, Chlenski A, Salwen HR, Henkin J, Cohn SL. 2007. Thrombospondin-1 peptide ABT-510 combined with valproic acid is an effective antiangiogenesis strategy in neuroblastoma. Cancer Res 67(4):1716–24.
Yanik GA, Levine JE, Matthay KK, Sisson JC, Shulkin BL, Shapiro B, Hubers D, Spalding S, Braun T, Ferrara JL, et al. 2002. Pilot study of iodine-131-metaiodobenzylguanidine in combination with myeloablative chemotherapy and autologous stem-cell support for the treatment of neuroblastoma. J Clin Oncol 20(8):2142–9.
Yu A, Batova A, Strother D, Angelini P, Castleberry RP. 2004. Promising results of a pilot trial of a GD2 directed anti-idiotypic antibody as a vaccine for high risk neuroblastoma. Genoa, Italy. p 75.
Yu AL, Uttenreuther-Fischer MM, Huang CS, Tsui CC, Gillies SD, Reisfeld RA, Kung FH. 1998. Phase I trial of a human-mouse chimeric anti-disialoganglioside monoclonal antibody ch14.18 in patients with refractory neuroblastoma and osteosarcoma. J Clin Oncol 16(6):2169–80.
Zechner D, Fujita Y, Hulsken J, Muller T, Walther I, Taketo MM, Crenshaw EB, 3rd, Birchmeier W, Birchmeier C. 2003. Beta-catenin signals regulate cell growth and the balance between progenitor cell expansion and differentiation in the nervous system. Dev Biol 258(2):406–18.
Zhou H, Kuang J, Zhong L, Kuo WL, Gray JW, Sahin A, Brinkley BR, Sen S. 1998. Tumour amplified kinase STK15/BTAK induces centrosome amplification, aneuploidy and transformation. Nat Genet 20(2):189–93.
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2010 Springer Science+Business Media, LLC
About this chapter
Cite this chapter
Mossé, Y.P., Maris, J.M. (2010). Molecular Therapy for Neuroblastoma. In: Houghton, P., Arceci, R. (eds) Molecularly Targeted Therapy for Childhood Cancer. Springer, New York, NY. https://doi.org/10.1007/978-0-387-69062-9_17
Download citation
DOI: https://doi.org/10.1007/978-0-387-69062-9_17
Published:
Publisher Name: Springer, New York, NY
Print ISBN: 978-0-387-69060-5
Online ISBN: 978-0-387-69062-9
eBook Packages: Biomedical and Life SciencesBiomedical and Life Sciences (R0)