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Review Article

The potential impact of tumour biology on improved clinical practice for medulloblastoma: progress towards biologically driven clinical trials

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Pages 364-375 | Received 08 Jun 2009, Accepted 16 Jun 2009, Published online: 15 Sep 2009

References

  • Hart M N, Earle K M. Primitive neuroectodermal tumors of the brain in children. Cancer 1973; 32(4)890–897
  • Pfister S, Remke M, Toedt G, et al. Supratentorial primitive neuroectodermal tumors of the central nervous system frequently harbor deletions of the CDKN2A locus and other genomic aberrations distinct from medulloblastomas. Genes Chromosomes Cancer 2007; 46(9)839–851
  • Pomeroy S L, Tamayo P, Gaasenbeek M, et al. Prediction of central nervous system embryonal tumour outcome based on gene expression. Nature 2002; 415(6870)436–442
  • McNeil D E, Cote T R, Clegg L, et al. Incidence and trends in pediatric malignancies medulloblastoma/primitive neuroectodermal tumor: a SEER update. Surveillance Epidemiology and End Results. Med Pediatr Oncol 2002; 39(3)190–194
  • Bailey C C, Gnekow A, Wellek S, Jones M, Round C, Brown J, Phillips A, Neidhardt M K. Prospective randomised trial of chemotherapy given before radiotherapy in childhood medulloblastoma. International Society of Paediatric Oncology (SIOP) and the (German) Society of Paediatric Oncology (GPO): SIOP II. Med Pediatr Oncol 1995; 25(3)166–178
  • Grill J, Sainte-Rose C, Jouvet A, et al. Treatment of medulloblastoma with postoperative chemotherapy alone: an SFOP prospective trial in young children. Lancet Oncol 2005; 6(8)573–580
  • Zeltzer P M, Boyett J M, Finlay J L, et al. Metastasis stage, adjuvant treatment, and residual tumor are prognostic factors for medulloblastoma in children: conclusions from the Children's Cancer Group 921 randomized phase III study. J Clin Oncol 1999; 17(3)832–845
  • Albright A L, Wisoff J H, Zeltzer P M, et al. Effects of medulloblastoma resections on outcome in children: a report from the Children's Cancer Group. Neurosurgery 1996; 38(2)265–271
  • Landberg T G, Lindgren M L, Cavallin-Stahl E K, et al. Improvements in the radiotherapy of medulloblastoma, 1946–1975. Cancer 1980; 45(4)670–678
  • Bouffet E, Bernard J L, Frappaz D, et al. M4 protocol for cerebellar medulloblastoma: supratentorial radiotherapy may not be avoided. Int J Radiat Oncol Biol Phys 1992; 24(1)79–85
  • Paulino A C, Narayana A, Mohideen M N, et al. Posterior fossa boost in medulloblastoma: an analysis of dose to surrounding structures using 3-dimensional (conformal) radiotherapy. Int J Radiat Oncol Biol Phys 2000; 46(2)281–286
  • Taylor R E, Bailey C C, Robinson K, et al. Results of a randomized study of preradiation chemotherapy versus radiotherapy alone for nonmetastatic medulloblastoma: The International Society of Paediatric Oncology/United Kingdom Children's Cancer Study Group PNET-3 Study. J Clin Oncol 2003; 21(8)1581–1591
  • Packer R J, Gajjar A, Vezina G, et al. Phase III study of craniospinal radiation therapy followed by adjuvant chemotherapy for newly diagnosed average-risk medulloblastoma. J Clin Oncol 2006; 24(25)4202–4208
  • Taylor R E, Bailey C C, Robinson K J, et al. Outcome for patients with metastatic (M2-3) medulloblastoma treated with SIOP/UKCCSG PNET-3 chemotherapy. Eur J Cancer 2005; 41(5)727–734
  • Tarbell N, Kun L, Freidman H, et al. High stage medulloblastoma: results from pediatric oncology group study 9031 (abstract). Med Pediat Oncol 2002; 39(4)227
  • Gajjar A, Chintagumpala M, Ashley D, et al. Risk-adapted craniospinal radiotherapy followed by high-dose chemotherapy and stem-cell rescue in children with newly diagnosed medulloblastoma (St Jude Medulloblastoma-96): long-term results from a prospective, multicentre trial. Lancet Oncol 2006; 7(10)813–820
  • Gandola L, Massimino M, Cefalo G, et al. Hyperfractionated accelerated radiotherapy in the Milan strategy for metastatic medulloblastoma. J Clin Oncol 2009; 27(4)566–571
  • Jakacki R, Burger P, Zhou T, et al. Outcome for metastatic (M+) medulloblastoma (MB) treated with carboplatin during craniospinal radiotherapy (CSRT) followed by cyclophosphamide (CPM) and vincristine (VCR): Preliminary results of COG 99701. Proc Amer Soc Clin Oncol 2007; 25: 2017
  • Rutkowski S, Bode U, Deinlein F, et al. Treatment of early childhood medulloblastoma by postoperative chemotherapy alone. N Engl J Med 2005; 352(10)978–986
  • Louis D N, Ohgaki H, Wiestler O D, et al. The 2007 WHO classification of tumours of the central nervous system. Acta Neuropathol 2007; 114(2)97–109
  • McManamy C S, Pears J, Weston C L, et al. Nodule formation and desmoplasia in medulloblastomas-defining the nodular/desmoplastic variant and its biological behavior. Brain Pathol 2007; 17(2)151–164
  • Eberhart C G, Kepner J L, Goldthwaite P T, et al. Histopathologic grading of medulloblastomas: a Pediatric Oncology Group study. Cancer 2002; 94(2)552–560
  • Cogen P H, Daneshvar L, Metzger A K, et al. Involvement of multiple chromosome 17p loci in medulloblastoma tumorigenesis. Am J Hum Genet 1992; 50(3)584–589
  • Huang H, Mahler-Araujo B M, Sankila A, et al. APC mutations in sporadic medulloblastomas. Am J Pathol 2000; 156(2)433–437
  • Koch A, Waha A, Tonn J C, et al. Somatic mutations of WNT/wingless signaling pathway components in primitive neuroectodermal tumors. Int J Cancer 2001; 93(3)445–449
  • Malkin D, Li F P, Strong L C, Fraumeni J F, Jr., Nelson C E, Kim D H, Kassel J, Gryka M A, Bischoff F Z, Tainsky M A, et al. Germ line p53 mutations in a familial syndrome of breast cancer, sarcomas, and other neoplasms. Science 1990; 250(4985)1233–1238
  • Hahn H, Wicking C, Zaphiropoulous P G, et al. Mutations of the human homolog of Drosophila patched in the nevoid basal cell carcinoma syndrome. Cell 1996; 85(6)841–851
  • Hamilton S R, Liu B, Parsons R E, et al. The molecular basis of Turcot's syndrome. N Engl J Med 1995; 332(13)839–847
  • Raffel C, Jenkins R B, Frederick L, et al. Sporadic medulloblastomas contain PTCH mutations. Cancer Res 1997; 57(5)842–845
  • Pietsch T, Waha A, Koch A, et al. Medulloblastomas of the desmoplastic variant carry mutations of the human homologue of Drosophila patched. Cancer Res 1997; 57(11)2085–2088
  • Wolter M, Reifenberger J, Sommer C, et al. Mutations in the human homologue of the Drosophila segment polarity gene patched (PTCH) in sporadic basal cell carcinomas of the skin and primitive neuroectodermal tumors of the central nervous system. Cancer Res 1997; 57(13)2581–2585
  • Vorechovsky I, Tingby O, Hartman M, et al. Somatic mutations in the human homologue of Drosophila patched in primitive neuroectodermal tumours. Oncogene 1997; 15(3)361–366
  • Kleihues P, Schauble B, zur Hausen A, et al. Tumors associated with p53 germline mutations: a synopsis of 91 families. Am J Pathol 1997; 150(1)1–13
  • Johnson R L, Rothman A L, Xie J, et al. Human homolog of patched, a candidate gene for the basal cell nevus syndrome. Science 1996; 272(5268)1668–1671
  • Evans D G, Farndon P A, Burnell L D, et al. The incidence of Gorlin syndrome in 173 consecutive cases of medulloblastoma. Br J Cancer 1991; 64(5)959–961
  • Paraf F, Jothy S, Van Meir E G. Brain tumor-polyposis syndrome: two genetic diseases?. J Clin Oncol 1997; 15(7)2744–2758
  • Gilbertson R J, Ellison D W. The origins of medulloblastoma subtypes. Annu Rev Pathol 2008; 3: 341–365
  • Marino S. Medulloblastoma: developmental mechanisms out of control. Trends Mol Med 2005; 11(1)17–22
  • Taipale J, Beachy P A. The Hedgehog and Wnt signalling pathways in cancer. Nature 2001; 411(6835)349–354
  • Clevers H. Axin and hepatocellular carcinomas. Nat Genet 2000; 24(3)206–208
  • Morin P J. beta-catenin signaling and cancer. Bioessays 1999; 21(12)1021–1030
  • Zurawel R H, Chiappa S A, Allen C, et al. Sporadic medulloblastomas contain oncogenic beta-catenin mutations. Cancer Res 1998; 58(5)896–899
  • Eberhart C G, Tihan T, Burger P C. Nuclear localization and mutation of beta-catenin in medulloblastomas. J Neuropathol Exp Neurol 2000; 59(4)333–337
  • Dahmen R P, Koch A, Denkhaus D, et al. Deletions of AXIN1, a component of the WNT/wingless pathway, in sporadic medulloblastomas. Cancer Res 2001; 61(19)7039–7043
  • Baeza N, Masuoka J, Kleihues P, et al. AXIN1 mutations but not deletions in cerebellar medulloblastomas. Oncogene 2003; 22(4)632–636
  • Ellison D W, Onilude O E, Lindsey J C, et al. beta-Catenin status predicts a favorable outcome in childhood medulloblastoma: the United Kingdom Children's Cancer Study Group Brain Tumour Committee. J Clin Oncol 2005; 23(31)7951–7957
  • Clifford S C, Lusher M E, Lindsey J C, et al. Wnt/Wingless pathway activation and chromosome 6 loss characterize a distinct molecular sub-group of medulloblastomas associated with a favorable prognosis. Cell Cycle 2006; 5(22)2666–2670
  • Thompson M C, Fuller C, Hogg T L, et al. Genomics identifies medulloblastoma subgroups that are enriched for specific genetic alterations. J Clin Oncol 2006; 24(12)1924–1931
  • Kool M, Koster J, Bunt J, et al. Integrated genomics identifies five medulloblastoma subtypes with distinct genetic profiles, pathway signatures and clinicopathological features. PLoS ONE 2008; 3(8)e3088
  • Wechsler-Reya R, Scott M P. The developmental biology of brain tumors. Annu Rev Neurosci 2001; 24: 385–428
  • Stone D M, Murone M, Luoh S, et al. Characterization of the human suppressor of fused, a negative regulator of the zinc-finger transcription factor Gli. J Cell Sci 1999; 112(Pt 23)4437–4448
  • Koch A, Waha A, Hartmann W, et al. No evidence for mutations or altered expression of the Suppressor of Fused gene (SUFU) in primitive neuroectodermal tumours. Neuropathol Appl Neurobiol 2004; 30(5)532–539
  • Reifenberger J, Wolter M, Weber R G, et al. Missense mutations in SMOH in sporadic basal cell carcinomas of the skin and primitive neuroectodermal tumors of the central nervous system. Cancer Res 1998; 58(9)1798–1803
  • Taylor M D, Liu L, Raffel C, et al. Mutations in SUFU predispose to medulloblastoma. Nat Genet 2002; 31(3)306–310
  • Wetmore C, Eberhart D E, Curran T. The normal patched allele is expressed in medulloblastomas from mice with heterozygous germ-line mutation of patched. Cancer Res 2000; 60(8)2239–2246
  • Goodrich L V, Milenkovic L, Higgins K M, et al. Altered neural cell fates and medulloblastoma in mouse patched mutants. Science 1997; 277(5329)1109–1113
  • Lee Y, Kawagoe R, Sasai K, et al. Loss of suppressor-of-fused function promotes tumorigenesis. Oncogene 2007; 26(44)6442–6447
  • Hallahan A R, Pritchard J I, Hansen S, et al. The SmoA1 mouse model reveals that notch signaling is critical for the growth and survival of sonic hedgehog-induced medulloblastomas. Cancer Res 2004; 64(21)7794–7800
  • Schofield D, West D C, Anthony D C, et al. Correlation of loss of heterozygosity at chromosome 9q with histological subtype in medulloblastomas. Am J Pathol 1995; 146(2)472–480
  • Nicholson J C, Ross F M, Kohler J A, et al. Comparative genomic hybridization and histological variation in primitive neuroectodermal tumours. Br J Cancer 1999; 80(9)1322–1331
  • Broderick D K, Di C, Parrett T J, et al. Mutations of PIK3CA in anaplastic oligodendrogliomas, high-grade astrocytomas, and medulloblastomas. Cancer Res 2004; 64(15)5048–5050
  • Gilbertson R J, Clifford S C. PDGFRB is overexpressed in metastatic medulloblastoma. Nat Genet 2003; 35(3)197–198
  • Gilbertson R J, Langdon J A, Hollander A, et al. Mutational analysis of PDGFR-RAS/MAPK pathway activation in childhood medulloblastoma. Eur J Cancer 2006; 42(5)646–649
  • Grotzer M A, Janss A J, Fung K, et al. TrkC expression predicts good clinical outcome in primitive neuroectodermal brain tumors. J Clin Oncol 2000; 18(5)1027–1035
  • Hernan R, Fasheh R, Calabrese C, et al. ERBB2 up-regulates S100A4 and several other prometastatic genes in medulloblastoma. Cancer Res 2003; 63(1)140–148
  • MacDonald T J, Brown K M, LaFleur B, et al. Expression profiling of medulloblastoma: PDGFRA and the RAS/MAPK pathway as therapeutic targets for metastatic disease. Nat Genet 2001; 29(2)143–152
  • Bigner S H, Mark J, Friedman H S, et al. Structural chromosomal abnormalities in human medulloblastoma. Cancer Genet Cytogenet 1988; 30(1)91–101
  • Lamont J M, McManamy C S, Pearson A D, et al. Combined histopathological and molecular cytogenetic stratification of medulloblastoma patients. Clin Cancer Res 2004; 10(16)5482–5493
  • Biegel J A, Rorke L B, Packer R J, et al. Isochromosome 17q in primitive neuroectodermal tumors of the central nervous system. Genes Chromosomes Cancer 1989; 1(2)139–147
  • Reardon D A, Michalkiewicz E, Boyett J M, et al. Extensive genomic abnormalities in childhood medulloblastoma by comparative genomic hybridization. Cancer Res 1997; 57(18)4042–4047
  • Gilbertson R, Wickramasinghe C, Hernan R, et al. Clinical and molecular stratification of disease risk in medulloblastoma. Br J Cancer 2001; 85(5)705–712
  • Avet-Loiseau H, Venuat A M, Terrier-Lacombe M J, et al. Comparative genomic hybridization detects many recurrent imbalances in central nervous system primitive neuroectodermal tumours in children. Br J Cancer 1999; 79(11–12)1843–1847
  • Eberhart C G, Kratz J E, Schuster A, et al. Comparative genomic hybridization detects an increased number of chromosomal alterations in large cell/anaplastic medulloblastomas. Brain Pathol 2002; 12(1)36–44
  • Aldosari N, Bigner S H, Burger P C, et al. MYCC and MYCN oncogene amplification in medulloblastoma. A fluorescence in situ hybridization study on paraffin sections from the Children's Oncology Group. Arch Pathol Lab Med 2002; 126(5)540–544
  • Boon K, Eberhart C G, Riggins G J. Genomic amplification of orthodenticle homologue 2 in medulloblastomas. Cancer Res 2005; 65(3)703–707
  • Di C, Liao S, Adamson D C, et al. Identification of OTX2 as a medulloblastoma oncogene whose product can be targeted by all-trans retinoic acid. Cancer Res 2005; 65(3)919–924
  • Fan X, Mikolaenko I, Elhassan I, et al. Notch1 and notch2 have opposite effects on embryonal brain tumor growth. Cancer Res 2004; 64(21)7787–7793
  • Fan X, Wang Y, Kratz J, et al. hTERT gene amplification and increased mRNA expression in central nervous system embryonal tumors. Am J Pathol 2003; 162(6)1763–1769
  • Ellison D W, Clifford S C, Gajjar A, et al. What's new in neuro-oncology? Recent advances in medulloblastoma. Eur J Paediatr Neurol 2003; 7(2)53–66
  • Rutkowski S, von Bueren A, von Hoff K, et al. Prognostic relevance of clinical and biological risk factors in childhood medulloblastoma: results of patients treated in the prospective multicenter trial HIT'91. Clin Cancer Res 2007; 13(9)2651–2657
  • Gajjar A, Hernan R, Kocak M, et al. Clinical, histopathologic, and molecular markers of prognosis: toward a new disease risk stratification system for medulloblastoma. J Clin Oncol 2004; 22(6)984–993
  • Berman D M, Karhadkar S S, Hallahan A R, et al. Medulloblastoma growth inhibition by hedgehog pathway blockade. Science 2002; 297(5586)1559–1561
  • Romer J T, Kimura H, Magdaleno S, et al. Suppression of the Shh pathway using a small molecule inhibitor eliminates medulloblastoma in Ptc1(+/−)p53(−/−) mice. Cancer Cell 2004; 6(3)229–240
  • Taipale J, Chen J K, Cooper M K, et al. Effects of oncogenic mutations in Smoothened and Patched can be reversed by cyclopamine. Nature 2000; 406(6799)1005–1009
  • van Es J H, Clevers H. Notch and Wnt inhibitors as potential new drugs for intestinal neoplastic disease. Trends Mol Med 2005; 11(11)496–502
  • Kimura H, Ng J M, Curran T. Transient inhibition of the Hedgehog pathway in young mice causes permanent defects in bone structure. Cancer Cell 2008; 13(3)249–260

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