Towards a turning point of neuroblastoma therapy

Cancer Letters

Volumn 326, Issue 2, 2012, Pages 128-134

  Tonini, Gian Paolo ^a   Nakagawara, Akira ^b   Berthold, Frank ^c  

^a UNIVERSITY OF GENOA   (Italy)

^b CHIBA UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^c UNIVERSITY OF COLOGNE   (Germany)

Author keywords

ACGH; ALK; High risk neuroblastoma; MYCN; Neuroblastoma; Personalized therapy

Indexed keywords

CRIZOTINIB; MAMMALIAN TARGET OF RAPAMYCIN; TUMOR MARKER;

ALK GENE; ASIA; CANCER CHEMOTHERAPY; CANCER IMMUNOTHERAPY; CANCER RADIOTHERAPY; CANCER RISK; CANCER THERAPY; DRUG EFFICACY; GENE AMPLIFICATION; GENE MUTATION; GENE TARGETING; GENETIC ASSOCIATION; GENETIC RISK; GENOME ANALYSIS; HEALTH CARE DISPARITY; HUMAN; MUTATOR GENE; MYCN GENE; NEUROBLASTOMA; NEUROSURGERY; ONCOGENE; PERSONALIZED MEDICINE; PRIORITY JOURNAL; SHORT SURVEY; STRUCTURAL CHROMOSOME ABERRATION;

EID: 84866878107     PISSN: 03043835     EISSN: 18727980     Source Type: Journal    
DOI: 10.1016/j.canlet.2012.08.017     Document Type: Review

References (60)

1. Shimada H., Umehara S., Monobe Y., Hachitanda Y., Nakagawa A., Goto S., Gerbing R.B., Stram D.O., Lukens J.N., Matthay K.K. International neuroblastoma pathology classification for prognostic evaluation of patients with peripheral neuroblastic tumors: a report from the Children's Cancer Group. Cancer 2001, 92:2451-2461.
2. Goto S., Umehara S., Gerbing R.B., Stram D.O., Brodeur G.M., Seeger R.C., Lukens J.N., Matthay K.K., Shimada H. Histopathology (International Neuroblastoma Pathology Classification) and MYCN status in patients with peripheral neuroblastic tumors: a report from the Children's Cancer Group. Cancer 2001, 92:2699-2708.
3. Cohn S.L., Pearson A.D., London W.B., Monclair T., Ambros P.F., Brodeur G.M., Faldum A., Hero B., Iehara T., Machin D., Mosseri V., Simon T., Garaventa A., Castel V., Matthay K.K., Force I.T. The International Neuroblastoma Risk Group (INRG) classification system: an INRG Task Force report. J. Clin. Oncol. 2009, 27:289-297.
4. Coco S., Theissen J., Scaruffi P., Stigliani S., Moretti S., Oberthuer A., Valdora F., Fischer M., Gallo F., Hero B., Bonassi S., Berthold F., Tonini G.P. Age-dependent accumulation of genomic aberrations and deregulation of cell cycle and telomerase genes in metastatic neuroblastoma. Int. J. Cancer 2012, 131:1591-1600.
5. Coco S., Defferrari R., Scaruffi P., Cavazzana A., Di Cristofano C., Longo L., Mazzocco K., Perri P., Gambini C., Moretti S., Bonassi S., Tonini G.P. Genome analysis and gene expression profiling of neuroblastoma and ganglioneuroblastoma reveal differences between neuroblastic and Schwannian stromal cells. J. Pathol. 2005, 207:346-357.
6. Maris J.M. Recent advances in neuroblastoma. N. Engl. J. Med. 2010, 362:2202-2211.
7. Del Grosso F., Coco S., Scaruffi P., Stigliani S., Valdora F., Benelli R., Salvi S., Boccardo S., Truini M., Croce M., Ferrini S., Longo L., Tonini G.P. Role of CXCL13-CXCR5 crosstALK between malignant neuroblastoma cells and Schwannian stromal cells in neuroblastic tumors. Mol. Cancer Res. 2011, 9:815-823.
8. Ambros I.M., Zellner A., Roald B., Amann G., Ladenstein R., Printz D., Gadner H., Ambros P.F. Role of ploidy, chromosome 1p, and Schwann cells in the maturation of neuroblastoma. N. Engl. J. Med. 1996, 334:1505-1511.
9. Brodeur G.M., Nakagawara A., Yamashiro D.J., Ikegaki N., Liu X.G., Azar C.G., Lee C.P., Evans A.E. Expression of TrkA, TrkB and TrkC in human neuroblastomas. J. Neurooncol. 1997, 31:49-55.
10. Longo L., Panza E., Schena F., Seri M., Devoto M., Romeo G., Bini C., Pappalardo G., Tonini G.P., Perri P. Genetic predisposition to familial neuroblastoma: identification of two novel genomic regions at 2p and 12p. Hum. Hered. 2007, 63:205-211.
11. Mosse Y.P., Laudenslager M., Longo L., Cole K.A., Wood A., Attiyeh E.F., Laquaglia M.J., Sennett R., Lynch J.E., Perri P., Laureys G., Speleman F., Kim C., Hou C., Hakonarson H., Torkamani A., Schork N.J., Brodeur G.M., Tonini G.P., Rappaport E., Devoto M., Maris J.M. Identification of ALK as a major familial neuroblastoma predisposition gene. Nature 2008, 455:930-935.
12. Tonini G.P., Longo L., Coco S., Perri P. Familial neuroblastoma: a complex heritable disease. Cancer Lett. 2003, 197:41-45.
13. Longo L., Tonini G.P., Ceccherini I., Perri P. Oligogenic inheritance in neuroblastoma. Cancer Lett. 2005, 228:65-69.
14. Schleiermacher G., Janoueix-Lerosey I., Ribeiro A., Klijanienko J., Couturier J., Pierron G., Mosseri V., Valent A., Auger N., Plantaz D., Rubie H., Valteau-Couanet D., Bourdeaut F., Combaret V., Bergeron C., Michon J., Delattre O. Accumulation of segmental alterations determines progression in neuroblastoma. J. Clin. Oncol. 2010, 28:3122-3130.
15. Schleiermacher G., Michon J., Ribeiro A., Pierron G., Mosseri V., Rubie H., Munzer C., Benard J., Auger N., Combaret V., Janoueix-Lerosey I., Pearson A., Tweddle D.A., Bown N., Gerrard M., Wheeler K., Noguera R., Villamon E., Canete A., Castel V., Marques B., de Lacerda A., Tonini G.P., Mazzocco K., Defferrari R., de Bernardi B., di Cataldo A., van Roy N., Brichard B., Ladenstein R., Ambros I., Ambros P., Beiske K., Delattre O., Couturier J. Segmental chromosomal alterations lead to a higher risk of relapse in infants with MYCN-non-amplified localised unresectable/disseminated neuroblastoma (a SIOPEN collaborative study). Brit. J. Cancer 2011, 105:1940-1948.
16. Tonini G.P. Neuroblastoma: the result of multistep transformation?. Stem Cells 1993, 11:276-282.
17. Maris J.M., Hogarty M.D., Bagatell R., Cohn S.L. Neuroblastoma. Lancet 2007, 369:2106-2120.
18. Schwab M., Alitalo K., Klempnauer K.H., Varmus H.E., Bishop J.M., Gilbert F., Brodeur G., Goldstein M., Trent J. Amplified DNA with limited homology to myc cellular oncogene is shared by human neuroblastoma cell lines and a neuroblastoma tumour. Nature 1983, 305:245-248.
19. Brodeur G.M., Seeger R.C., Schwab M., Varmus H.E., Bishop J.M. Amplification of N-myc in untreated human neuroblastomas correlates with advanced disease stage. Science 1984, 224:1121-1124.
20. Schwab M. Amplification of N-myc as a prognostic marker for patients with neuroblastoma. Semin. Cancer Biol. 1993, 4:13-18.
21. Schwab M., Westermann F., Hero B., Berthold F. Neuroblastoma: biology and molecular and chromosomal pathology. Lancet Oncol. 2003, 4:472-480.
22. Vermeulen J., De Preter K., Naranjo A., Vercruysse L., Van Roy N., Hellemans J., Swerts K., Bravo S., Scaruffi P., Tonini G.P., De Bernardi B., Noguera R., Piqueras M., Canete A., Castel V., Janoueix-Lerosey I., Delattre O., Schleiermacher G., Michon J., Combaret V., Fischer M., Oberthuer A., Ambros P.F., Beiske K., Benard J., Marques B., Rubie H., Kohler J., Potschger U., Ladenstein R., Hogarty M.D., McGrady P., London W.B., Laureys G., Speleman F., Vandesompele J. Predicting outcomes for children with neuroblastoma using a multigene-expression signature: a retrospective SIOPEN/COG/GPOH study. Lancet Oncol. 2009, 10:663-671.
23. Oberthuer A., Hero B., Berthold F., Juraeva D., Faldum A., Kahlert Y., Asgharzadeh S., Seeger R., Scaruffi P., Tonini G.P., Janoueix-Lerosey I., Delattre O., Schleiermacher G., Vandesompele J., Vermeulen J., Speleman F., Noguera R., Piqueras M., Benard J., Valent A., Avigad S., Yaniv I., Weber A., Christiansen H., Grundy R.G., Schardt K., Schwab M., Eils R., Warnat P., Kaderali L., Simon T., Decarolis B., Theissen J., Westermann F., Brors B., Fischer M. Prognostic impact of gene expression-based classification for neuroblastoma. J. Clin. Oncol. 2010, 28:3506-3515.
24. Thiele C.J., Reynolds C.P., Israel M.A. Decreased expression of N-myc precedes retinoic acid-induced morphological differentiation of human neuroblastoma. Nature 1985, 313:404-406.
25. Seeger R.C., Brodeur G.M., Sather H., Dalton A., Siegel S.E., Wong K.Y., Hammond D. Association of multiple copies of the N-myc oncogene with rapid progression of neuroblastomas. N. Engl. J. Med. 1985, 313:1111-1116.
26. De Bernardi B., Mosseri V., Rubie H., Castel V., Foot A., Ladenstein R., Laureys G., Beck-Popovic M., de Lacerda A.F., Pearson A.D., De Kraker J., Ambros P.F., de Rycke Y., Conte M., Bruzzi P., Michon J., Group S.E.N. Treatment of localised resectable neuroblastoma. Results of the LNESG1 study by the SIOP Europe Neuroblastoma Group. Brit. J. Cancer 2008, 99:1027-1033.
27. Rubie H., De Bernardi B., Gerrard M., Canete A., Ladenstein R., Couturier J., Ambros P., Munzer C., Pearson A.D., Garaventa A., Brock P., Castel V., Valteau-Couanet D., Holmes K., Di Cataldo A., Brichard B., Mosseri V., Marquez C., Plantaz D., Boni L., Michon J. Excellent outcome with reduced treatment in infants with nonmetastatic and unresectable neuroblastoma without MYCN amplification: results of the prospective INES 99.1. J. Clin. Oncol. 2011, 29:449-455.
28. Capasso M., Devoto M., Hou C., Asgharzadeh S., Glessner J.T., Attiyeh E.F., Mosse Y.P., Kim C., Diskin S.J., Cole K.A., Bosse K., Diamond M., Laudenslager M., Winter C., Bradfield J.P., Scott R.H., Jagannathan J., Garris M., McConville C., London W.B., Seeger R.C., Grant S.F., Li H., Rahman N., Rappaport E., Hakonarson H., Maris J.M. Common variations in BARD1 influence susceptibility to high-risk neuroblastoma. Nat. Genet. 2009, 41:718-723.
29. Boehm T., Foroni L., Kaneko Y., Perutz M.F., Rabbitts T.H. The rhombotin family of cysteine-rich LIM-domain oncogenes: distinct members are involved in T-cell translocations to human chromosomes 11p15 and 11p13. Proc. Natl. Acad. Sci. USA 1991, 88:4367-4371.
30. Wang K., Diskin S.J., Zhang H., Attiyeh E.F., Winter C., Hou C., Schnepp R.W., Diamond M., Bosse K., Mayes P.A., Glessner J., Kim C., Frackelton E., Garris M., Wang Q., Glaberson W., Chiavacci R., Nguyen L., Jagannathan J., Saeki N., Sasaki H., Grant S.F., Iolascon A., Mosse Y.P., Cole K.A., Li H., Devoto M., McGrady P.W., London W.B., Capasso M., Rahman N., Hakonarson H., Maris J.M. Integrative genomics identifies LMO1 as a neuroblastoma oncogene. Nature 2011, 469:216-220.
31. Maris J.M., Kyemba S.M., Rebbeck T.R., White P.S., Sulman E.P., Jensen S.J., Allen C., Biegel J.A., Yanofsky R.A., Feldman G.L., Brodeur G.M. Familial predisposition to neuroblastoma does not map to chromosome band 1p36. Cancer Res. 1996, 56:3421-3425.
32. Lo Cunsolo C., Iolascon A., Cavazzana A., Cusano R., Strigini P., Mazzocco K., Giordani L., Massimo L., De Bernardi B., Conte M., Tonini G.P. Neuroblastoma in two siblings supports the role of 1p36 deletion in tumor development. Cancer Genet. Cytogenet. 1999, 109:126-130.
33. Janoueix-Lerosey I., Schleiermacher G., Michels E., Mosseri V., Ribeiro A., Lequin D., Vermeulen J., Couturier J., Peuchmaur M., Valent A., Plantaz D., Rubie H., Valteau-Couanet D., Thomas C., Combaret V., Rousseau R., Eggert A., Michon J., Speleman F., Delattre O. Overall genomic pattern is a predictor of outcome in neuroblastoma. J. Clin. Oncol. 2009, 27:1026-1033.
34. Schilling F.H., Spix C., Berthold F., Erttmann R., Fehse N., Hero B., Klein G., Sander J., Schwarz K., Treuner J., Zorn U., Michaelis J. Neuroblastoma screening at 1 year of age. N. Engl. J. Med. 2002, 346:1047-1053.
35. Brodeur G.M., Pritchard J., Berthold F., Carlsen N.L., Castel V., Castelberry R.P., De Bernardi B., Evans A.E., Favrot M., Hedborg F., et al. Revisions of the international criteria for neuroblastoma diagnosis, staging, and response to treatment. J. Clin. Oncol. 1993, 11:1466-1477.
36. Monclair T., Brodeur G.M., Ambros P.F., Brisse H.J., Cecchetto G., Holmes K., Kaneko M., London W.B., Matthay K.K., Nuchtern J.G., von Schweinitz D., Simon T., Cohn S.L., Pearson A.D., Force I.T. The International Neuroblastoma Risk Group (INRG) staging system: an INRG Task Force report. J. Clin. Oncol. 2009, 27:298-303.
37. Hero B., Simon T., Spitz R., Ernestus K., Gnekow A.K., Scheel-Walter H.G., Schwabe D., Schilling F.H., Benz-Bohm G., Berthold F. Localized infant neuroblastomas often show spontaneous regression: results of the prospective trials NB95-S and NB97. J. Clin. Oncol. 2008, 26:1504-1510.
38. Matthay K.K., Reynolds C.P., Seeger R.C., Shimada H., Adkins E.S., Haas-Kogan D., Gerbing R.B., London W.B., Villablanca J.G. Long-term results for children with high-risk neuroblastoma treated on a randomized trial of myeloablative therapy followed by 13-cis-retinoic acid: a children's oncology group study. J. Clin. Oncol. 2009, 27:1007-1013.
39. Moroz V., Machin D., Faldum A., Hero B., Iehara T., Mosseri V., Ladenstein R., De Bernardi B., Rubie H., Berthold F., Matthay K.K., Monclair T., Ambros P.F., Pearson A.D., Cohn S.L., London W.B. Changes over three decades in outcome and the prognostic influence of age-at-diagnosis in young patients with neuroblastoma: a report from the International Neuroblastoma Risk Group Project. Eur. J. Cancer 2010, 47:561-571.
40. Hobbie W.L., Moshang T., Carlson C.A., Goldmuntz E., Sacks N., Goldfarb S.B., Grupp S.A., Ginsberg J.P. Late effects in survivors of tandem peripheral blood stem cell transplant for high-risk neuroblastoma. Pediatr. Blood Cancer 2008, 51:679-683.
41. Yalcin B., Kremer L.C., Caron H.N., van Dalen E.C. High-dose chemotherapy and autologous haematopoietic stem cell rescue for children with high-risk neuroblastoma. Cochrane Database Syst. Rev. 2010, 12:CD006301.
42. Yu A.L., Gilman A.L., Ozkaynak M.F., London W.B., Kreissman S.G., Chen H.X., Smith M., Anderson B., Villablanca J.G., Matthay K.K., Shimada H., Grupp S.A., Seeger R., Reynolds C.P., Buxton A., Reisfeld R.A., Gillies S.D., Cohn S.L., Maris J.M., Sondel P.M. Children's Oncology Group, Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N. Engl. J. Med. 2010, 363:1324-1334.
43. Simon T., Hero B., Faldum A., Handgretinger R., Schrappe M., Klingebiel T., Berthold F. Long term outcome of high-risk neuroblastoma patients after immunotherapy with antibody ch14.18 or oral metronomic chemotherapy. BMC Cancer 2011, 11:21.
44. Carpenter E.L., Mosse Y.P. Targeting ALK in neuroblastoma-preclinical and clinical advancements. Nat. Rev. Clin. Oncol. 2012, 9:391-399.
45. Matthay K.K., George R.E., Yu A.L. Promising therapeutic targets in neuroblastoma. Clin. Cancer Res. 2012, 18:2740-2753.
46. Kwak E.L., Bang Y.J., Camidge D.R., Shaw A.T., Solomon B., Maki R.G., Ou S.H., Dezube B.J., Janne P.A., Costa D.B., Varella-Garcia M., Kim W.H., Lynch T.J., Fidias P., Stubbs H., Engelman J.A., Sequist L.V., Tan W., Gandhi L., Mino-Kenudson M., Wei G.C., Shreeve S.M., Ratain M.J., Settleman J., Christensen J.G., Haber D.A., Wilner K., Salgia R., Shapiro G.I., Clark J.W., Iafrate A.J. Anaplastic lymphoma kinase inhibition in non-small-cell lung cancer. N. Engl. J. Med. 2010, 363:1693-1703.
47. Bresler S.C., Wood A.C., Haglund E.A., Courtright J., Belcastro L.T., Plegaria J.S., Cole K., Toporovskaya Y., Zhao H., Carpenter E.L., Christensen J.G., Maris J.M., Lemmon M.A., Mosse Y.P. Differential inhibitor sensitivity of anaplastic lymphoma kinase variants found in neuroblastoma. Sci. Trans. Med. 2011, 3:108ra114.
48. Doebele R.C., Pilling A.B., Aisner D.L., Kutateladze T.G., Le A.T., Weickhardt A.J., Kondo K.L., Linderman D.J., Heasley L.E., Franklin W.A., Varella-Garcia M., Camidge D.R. Mechanisms of resistance to crizotinib in patients with ALK gene rearranged non-small cell lung cancer. Clin. Cancer Res. 2012, 18:1472-1482.
49. Schulte J.H., Bachmann H.S., Brockmeyer B., Depreter K., Oberthur A., Ackermann S., Kahlert Y., Pajtler K., Theissen J., Westermann F., Vandesompele J., Speleman F., Berthold F., Eggert A., Brors B., Hero B., Schramm A., Fischer M. High ALK receptor tyrosine kinase expression supersedes ALK mutation as a determining factor of an unfavorable phenotype in primary neuroblastoma. Clin. Cancer Res. 2011, 17:5082-5092.
50. Camoratto A.M., Jani J.P., Angeles T.S., Maroney A.C., Sanders C.Y., Murakata C., Neff N.T., Vaught J.L., Isaacs J.T., Dionne C.A. CEP-751 inhibits TRK receptor tyrosine kinase activity in vitro exhibits anti-tumor activity. Int. J. Cancer 1997, 72:673-679.
51. Nakagawara A., Azar C.G., Scavarda N.J., Brodeur G.M. Expression and function of TRK-B and BDNF in human neuroblastomas. Mol. Cell. Biol. 1994, 14:759-767.
52. Fulda S. The PI3K/Akt/mTOR pathway as therapeutic target in neuroblastoma. Curr. Cancer Drug Targets 2009, 9:729-737.
53. Sun W., Modak S. Emerging treatment options for the treatment of neuroblastoma: potential role of perifosine. Onco Targets Ther. 2012, 5:21-29.
54. Maris J.M., Morton C.L., Gorlick R., Kolb E.A., Lock R., Carol H., Keir S.T., Reynolds C.P., Kang M.H., Wu J., Smith M.A., Houghton P.J. Initial testing of the aurora kinase A inhibitor MLN8237 by the Pediatric Preclinical Testing Program (PPTP). Pediatr. Blood Cancer 2010, 55:26-34.
55. Houghton P.J., Adamson P.C., Blaney S., Fine H.A., Gorlick R., Haber M., Helman L., Hirschfeld S., Hollingshead M.G., Israel M.A., Lock R.B., Maris J.M., Merlino G., Patterson W., Reynolds C.P., Shannon K., Yu A., Yu J., Smith M.A. Testing of new agents in childhood cancer preclinical models: meeting summary. Clin. Cancer Res. 2002, 8:3646-3657.
56. Houghton P.J., Morton C.L., Tucker C., Payne D., Favours E., Cole C., Gorlick R., Kolb E.A., Zhang W., Lock R., Carol H., Tajbakhsh M., Reynolds C.P., Maris J.M., Courtright J., Keir S.T., Friedman H.S., Stopford C., Zeidner J., Wu J., Liu T., Billups C.A., Khan J., Ansher S., Zhang J., Smith M.A. The pediatric preclinical testing program: description of models and early testing results. Pediatr. Blood Cancer 2007, 49:928-940.
57. Carol H., Morton C.L., Gorlick R., Kolb E.A., Keir S.T., Reynolds C.P., Kang M.H., Maris J.M., Billups C., Smith M.A., Houghton P.J., Lock R.B. Initial testing (stage 1) of the Akt inhibitor GSK690693 by the pediatric preclinical testing program. Pediatr. Blood Cancer 2010, 55:1329-1337.
58. Houghton P.J., Gorlick R., Kolb E.A., Lock R., Carol H., Morton C.L., Keir S.T., Reynolds C.P., Kang M.H., Phelps D., Maris J.M., Billups C., Smith M.A. Initial testing (stage 1) of the mTOR kinase inhibitor AZD8055 by the pediatric preclinical testing program. Pediatr. Blood Cancer 2012, 58:191-199.
59. Kolb E.A., Gorlick R., Lock R., Carol H., Morton C.L., Keir S.T., Reynolds C.P., Kang M.H., Maris J.M., Billups C., Smith M.A., Houghton P.J. Initial testing (stage 1) of the IGF-1 receptor inhibitor BMS-754807 by the pediatric preclinical testing program. Pediatr. Blood Cancer 2011, 56:595-603.
60. Couzin-Frankel J. Personalized medicine. Pushing the envelope in neuroblastoma therapy. Science 2011, 333:1569-1571.