Seminars in Oncology
Volume 36, Issue 4 , Pages 358-371 , August 2009

Other Targetable Sarcomas

  • Veridiana Pires de Camargo

      Affiliations

    • Sarcoma Program, Memorial Sloan-Kettering Cancer Center, New York, NY
    • These authors contributed equally to this work.
    • ,
  • Matt van de Rijn

      Affiliations

    • Department of Pathology, Stanford University Medical Center, Stanford, CA
    • These authors contributed equally to this work.
    • ,
  • Enrique de Alava

      Affiliations

    • Molecular Pathology Program, Centro de Investigación del Cáncer–IBMCC, University of Salamanca–CSIC, Salamanca, Spain
    • These authors contributed equally to this work.
    • ,
  • Juan Madoz-Gúrpide

      Affiliations

    • Molecular Pathology Program, Centro de Investigación del Cáncer–IBMCC, University of Salamanca–CSIC, Salamanca, Spain
    • These authors contributed equally to this work.
    • ,
  • Silvana Pilotti

      Affiliations

    • Laboratory of Experimental Molecular Pathology, Department of Pathology, Fondazione IRCCS, Istituto Nazionale Tumori, Milan, Italy
    • These authors contributed equally to this work.
    • ,
  • Margaret von Mehren

      Affiliations

    • Sarcoma Oncology, Fox Chase Cancer Center, Philadelphia, PA
    • These authors contributed equally to this work.
    • ,
  • Florence Pedeutour

      Affiliations

    • Laboratoire de Génétique des Tumeurs Solides, Faculté de Médecine, Centre Hospitalier Universitaire de Nice et CNRS UMRS 6543, Nice, France
    • These authors contributed equally to this work.
    • ,
  • Robert G. Maki

      Affiliations

    • Sarcoma Program, Memorial Sloan-Kettering Cancer Center, New York, NY
    • These authors contributed equally to this work.
    • ,
  • Piotr Rutkowski

      Affiliations

    • Maria Sklodowska-Curie Memorial Cancer Center and Institute of Oncology, Department of Soft Tissue/Bone Sarcoma and Melanoma, Warsaw, Poland
    • These authors contributed equally to this work.
    • ,
  • David M. Thomas

      Affiliations

    • Research Division, Peter MacCallum Cancer Centre, East Melbourne, Victoria, Australia
    • These authors contributed equally to this work.
    • Corresponding Author InformationAddress correspondence to David M. Thomas, FRACP, PhD, Research Division, Peter MacCallum Cancer Centre, St Andrew's Place, East Melbourne, Victoria 3002, Australia

References 

  1. Hirota S, Isozaki K, Moriyama Y, et al. Gain-of-function mutations of c-kit in human gastrointestinal stromal tumors. Science. 1998;279:577–580
  2. Grosso F, Jones RL, Demetri GD, et al. Efficacy of trabectedin (ecteinascidin-743) in advanced pretreated myxoid liposarcomas: a retrospective study. Lancet Oncol. 2007;8:595–602
  3. Schöffski P, Casali PG, Taron M, et al. DNA repair functionality modulates the clinical outcome of patients with advanced sarcoma treated with trabectedin (ET-743) [abstract]. J Clin Oncol. 2006;24:9522
  4. Espinosa I, Lee CH, Kim MK, et al. A novel monoclonal antibody against DOG1 is a sensitive and specific marker for gastrointestinal stromal tumors. Am J Surg Pathol. 2008;32:210–218
  5. Liegl B, Hornick JL, Corless CL, et al. Monoclonal antibody DOG1.1 shows higher sensitivity than KIT in the diagnosis of gastrointestinal stromal tumors, including unusual subtypes. Am J Surg Pathol. 2009;33:437–446
  6. West RB, Corless CL, Chen X, et al. The novel marker, DOG1, is expressed ubiquitously in gastrointestinal stromal tumors irrespective of KIT or PDGFRA mutation status. Am J Pathol. 2004;165:107–113
  7. Terry J, Saito T, Subramanian S, et al. TLE1 as a diagnostic immunohistochemical marker for synovial sarcoma emerging from gene expression profiling studies. Am J Surg Pathol. 2007;31:240–246
  8. Dhanasekaran SM, Dash A, Yu J, et al. Molecular profiling of human prostate tissues: insights into gene expression patterns of prostate development during puberty. FASEB J. 2005;19:243–245
  9. Higgins JP, Kaygusuz G, Wang L, et al. Placental S100 (S100P) and GATA3: markers for transitional epithelium and urothelial carcinoma discovered by complementary DNA microarray. Am J Surg Pathol. 2007;31:673–680
  10. Yao R, Lopez-Beltran A, Maclennan GT, et al. Expression of S100 protein family members in the pathogenesis of bladder tumors. Anticancer Res. 2007;27:3051–3058
  11. Dabbs DJ, Chivukula M, Carter G, et al. Basal phenotype of ductal carcinoma in situ: recognition and immunohistologic profile. Mod Pathol. 2006;19:1506–1511
  12. West RB, Rubin BP, Miller MA, et al. A landscape effect in tenosynovial giant-cell tumor from activation of CSF1 expression by a translocation in a minority of tumor cells. Proc Natl Acad Sci U S A. 2006;103:690–695
  13. Blay JY, El Sayadi H, Thiesse P, et al. Complete response to imatinib in relapsing pigmented villonodular synovitis/tenosynovial giant cell tumor (PVNS/TGCT). Ann Oncol. 2008;19:821–822
  14. Tamimi RM, Brugge JS, Freedman ML, et al. Circulating colony stimulating factor-1 and breast cancer risk. Cancer Res. 2008;68:18–21
  15. Kovacheva VP, Davison JM, Mellott TJ, et al. Raising gestational choline intake alters gene expression in DMBA-evoked mammary tumors and prolongs survival. FASEB J. 2009;23:1054–1063
  16. Beck AH, Espinosa I, Gilks CB, et al. The fibromatosis signature defines a robust stromal response in breast carcinoma. Lab Invest. 2008;88:591–601
  17. Cheang MC, van de Rijn M, Nielsen TO. Gene expression profiling of breast cancer. Annu Rev Pathol. 2008;3:67–97
  18. West RB, Nuyten DS, Subramanian S, et al. Determination of stromal signatures in breast carcinoma. PLoS Biol. 2005;3:e187
  19. Lee CH, Espinosa I, Vrijaldenhoven S, et al. Prognostic significance of macrophage infiltration in leiomyosarcomas. Clin Cancer Res. 2008;14:1423–1430
  20. Gygi SP, Rochon Y, Franza BR, et al. Correlation between protein and mRNA abundance in yeast. Mol Cell Biol. 1999;19:1720–1730
  21. Lu P, Vogel C, Wang R, et al. Absolute protein expression profiling estimates the relative contributions of transcriptional and translational regulation. Nat Biotechnol. 2007;25:117–124
  22. Aebersold R, Mann M. Mass spectrometry-based proteomics. Nature. 2003;422:198–207
  23. Yates JR, Gilchrist A, Howell KE, et al. Proteomics of organelles and large cellular structures. Nat Rev Mol Cell Biol. 2005;6:702–714
  24. Ong SE, Mann M. Mass spectrometry-based proteomics turns quantitative. Nat Chem Biol. 2005;1:252–262
  25. Cox J, Mann M. Is proteomics the new genomics?. Cell. 2007;130:395–398
  26. Izbicka E, Campos D, Marty J, et al. Molecular determinants of differential sensitivity to docetaxel and paclitaxel in human pediatric cancer models. Anticancer Res. 2006;26:1983–1988
  27. Gonzalez I, Andreu EJ, Panizo A, et al. Imatinib inhibits proliferation of Ewing tumor cells mediated by the stem cell factor/KIT receptor pathway, and sensitizes cells to vincristine and doxorubicin-induced apoptosis. Clin Cancer Res. 2004;10:751–761
  28. Pollak MN, Schernhammer ES, Hankinson SE. Insulin-like growth factors and neoplasia. Nat Rev Cancer. 2004;4:505–518
  29. Mateo-Lozano S, Tirado OM, Notario V. Rapamycin induces the fusion-type independent downregulation of the EWS/FLI-1 proteins and inhibits Ewing's sarcoma cell proliferation. Oncogene. 2003;22:9282–9287
  30. Scotlandi K, Benini S, Sarti M, et al. Insulin-like growth factor I receptor-mediated circuit in Ewing's sarcoma/peripheral neuroectodermal tumor: a possible therapeutic target. Cancer Res. 1996;56:4570–4574
  31. Scotlandi K, Manara MC, Nicoletti G, et al. Antitumor activity of the insulin-like growth factor-I receptor kinase inhibitor NVP-AEW541 in musculoskeletal tumors. Cancer Res. 2005;65:3868–3876
  32. Martins AS, Mackintosh C, Martin DH, et al. Insulin-like growth factor I receptor pathway inhibition by ADW742, alone or in combination with imatinib, doxorubicin, or vincristine, is a novel therapeutic approach in Ewing tumor. Clin Cancer Res. 2006;12:3532–3540
  33. Martins AS, Ordonez JL, Garcia-Sanchez A, et al. A pivotal role for heat shock protein 90 in Ewing sarcoma resistance to anti-insulin-like growth factor 1 receptor treatment: in vitro and in vivo study. Cancer Res. 2008;68:6260–6270
  34. Kovar H, Aryee D, Zoubek A. The Ewing family of tumors and the search for the Achilles' heel. Curr Opin Oncol. 1999;11:275–284
  35. Cohen P. The regulation of protein function by multisite phosphorylation—a 25 year update. Trends Biochem Sci. 2000;25:596–601
  36. Armistead PM, Salganick J, Roh JS, et al. Expression of receptor tyrosine kinases and apoptotic molecules in rhabdomyosarcoma: correlation with overall survival in 105 patients. Cancer. 2007;110:2293–2303
  37. Casali PG, Messina A, Stacchiotti S, et al. Imatinib mesylate in chordoma. Cancer. 2004;101:2086–2097
  38. Lagonigro MS, Tamborini E, Negri T, et al. PDGFRalpha, PDGFRbeta and KIT expression/activation in conventional chondrosarcoma. J Pathol. 2006;208:615–623
  39. Li Y, Chang Q, Rubin BP, et al. Insulin receptor activation in solitary fibrous tumours. J Pathol. 2007;211:550–554
  40. Liegl B, Gully C, Reich O, et al. Expression of platelet-derived growth factor receptor in low-grade endometrial stromal sarcomas in the absence of activating mutations. Histopathology. 2007;50:448–452
  41. McArthur GA, Demetri GD, van Oosterom A, et al. Molecular and clinical analysis of locally advanced dermatofibrosarcoma protuberans treated with imatinib: Imatinib Target Exploration Consortium Study B2225. J Clin Oncol. 2005;23:866–873
  42. Rubin BP, Schuetze SM, Eary JF, et al. Molecular targeting of platelet-derived growth factor B by imatinib mesylate in a patient with metastatic dermatofibrosarcoma protuberans. J Clin Oncol. 2002;20:3586–3591
  43. Signoroni S, Frattini M, Negri T, et al. Cyclooxygenase-2 and platelet-derived growth factor receptors as potential targets in treating aggressive fibromatosis. Clin Cancer Res. 2007;13:5034–5040
  44. Tamborini E, Bonadiman L, Albertini V, et al. Re: potential use of imatinib in Ewing's sarcoma: evidence for in vitro and in vivo activity. [letter] J Natl Cancer Inst. 2003;95:1087–1088
  45. Tamborini E, Bonadiman L, Greco A, et al. Expression of ligand-activated KIT and platelet-derived growth factor receptor beta tyrosine kinase receptors in synovial sarcoma. Clin Cancer Res. 2004;10:938–943
  46. Tamborini E, Casieri P, Miselli F, et al. Analysis of potential receptor tyrosine kinase targets in intimal and mural sarcomas. J Pathol. 2007;212:227–235
  47. Tamborini E, Miselli F, Negri T, et al. Molecular and biochemical analyses of platelet-derived growth factor receptor (PDGFR) B, PDGFRA, and KIT receptors in chordomas. Clin Cancer Res. 2006;12:6920–6928
  48. Heinrich MC, Joensuu H, Demetri GD, et al. Phase II, open-label study evaluating the activity of imatinib in treating life-threatening malignancies known to be associated with imatinib-sensitive tyrosine kinases. Clin Cancer Res. 2008;14:2717–2725
  49. Maki RG, Awan RA, Dixon RH, et al. Differential sensitivity to imatinib of 2 patients with metastatic sarcoma arising from dermatofibrosarcoma protuberans. Int J Cancer. 2002;100:623–626
  50. Shaw RJ, Cantley LC. Ras, PI(3)K and mTOR signalling controls tumour cell growth. Nature. 2006;441:424–430
  51. Azizi AA, Haberler C, Czech T, et al. Vascular-endothelial-growth-factor (VEGF) expression and possible response to angiogenesis inhibitor bevacizumab in metastatic alveolar soft part sarcoma. Lancet Oncol. 2006;7:521–523
  52. Lazar AJ, Das P, Tuvin D, et al. Angiogenesis-promoting gene patterns in alveolar soft part sarcoma. Clin Cancer Res. 2007;13:7314–7321
  53. Tsuda M, Davis IJ, Argani P, et al. TFE3 fusions activate MET signaling by transcriptional up-regulation, defining another class of tumors as candidates for therapeutic MET inhibition. Cancer Res. 2007;67:919–929
  54. Sirvent N, Coindre JM, Maire G, et al. Detection of MDM2-CDK4 amplification by fluorescence in situ hybridization in 200 paraffin-embedded tumor samples: utility in diagnosing adipocytic lesions and comparison with immunohistochemistry and real-time PCR. Am J Surg Pathol. 2007;31:1476–1489
  55. Italiano A, Cardot N, Dupre F, et al. Gains and complex rearrangements of the 12q13-15 chromosomal region in ordinary lipomas: the “missing link” between lipomas and liposarcomas?. Int J Cancer. 2007;121:308–315
  56. Mandahl N, Akerman M, Aman P, et al. Duplication of chromosome segment 12q15-24 is associated with atypical lipomatous tumors: a report of the CHAMP collaborative study group. CHromosomes And MorPhology. Int J Cancer. 1996;67:632–635
  57. Mandahl N, Heim S, Johansson B, et al. Lipomas have characteristic structural chromosomal rearrangements of 12q13-q14. Int J Cancer. 1987;39:685–688
  58. Storlazzi CT, Mertens F, Domanski H, et al. Ring chromosomes and low-grade gene amplification in an atypical lipomatous tumor with minimal nuclear atypia. Int J Oncol. 2003;23:67–71
  59. Italiano A, Bianchini L, Keslair F, et al. HMGA2 is the partner of MDM2 in well-differentiated and dedifferentiated liposarcomas whereas CDK4 belongs to a distinct inconsistent amplicon. Int J Cancer. 2008;122:2233–2241
  60. Ambrosini G, Sambol EB, Carvajal D, et al. Mouse double minute antagonist Nutlin-3a enhances chemotherapy-induced apoptosis in cancer cells with mutant p53 by activating E2F1. Oncogene. 2007;26:3473–3481
  61. Merks RM, Perryn ED, Shirinifard A, et al. Contact-inhibited chemotaxis in de novo and sprouting blood-vessel growth. PLoS Comput Biol. 2008;4:e1000163
  62. Folkman J. Tumor angiogenesis: therapeutic implications. N Engl J Med. 1971;285:1182–1186
  63. Folkman J. Successful treatment of an angiogenic disease. N Engl J Med. 1989;320:1211–1212
  64. Ribatti D, Conconi MT, Nussdorfer GG. Nonclassic endogenous novel [corrected] regulators of angiogenesis. Pharmacol Rev. 2007;59:185–205
  65. Carmeliet P, Ferreira V, Breier G, et al. Abnormal blood vessel development and lethality in embryos lacking a single VEGF allele. Nature. 1996;380:435–439
  66. Tobe T, Ortega S, Luna JD, et al. Targeted disruption of the FGF2 gene does not prevent choroidal neovascularization in a murine model. Am J Pathol. 1998;153:1641–1646
  67. Zhou M, Sutliff RL, Paul RJ, et al. Fibroblast growth factor 2 control of vascular tone. Nat Med. 1998;4:201–207
  68. Lindahl P, Johansson BR, Leveen P, et al. Pericyte loss and microaneurysm formation in PDGF-B-deficient mice. Science. 1997;277:242–245
  69. Urness LD, Sorensen LK, Li DY. Arteriovenous malformations in mice lacking activin receptor-like kinase-1. Nat Genet. 2000;26:328–331
  70. Whitehead KJ, Plummer NW, Adams JA, et al. Ccm1 is required for arterial morphogenesis: implications for the etiology of human cavernous malformations. Development. 2004;131:1437–1448
  71. Dezube BJ, Von Roenn JH, Holden-Wiltse J, et al. AIDS Clinical Trial Group No. 215 Team Fumagillin analog in the treatment of Kaposi's sarcoma: a phase I AIDS Clinical Trial Group study. J Clin Oncol. 1998;16:1444–1449
  72. Baker LH, Rowinsky EK, Mendelson D, et al. Randomized, phase II study of the thrombospondin-1-mimetic angiogenesis inhibitor ABT-510 in patients with advanced soft tissue sarcoma. J Clin Oncol. 2008;26:5583–5588
  73. D'Adamo DR, Anderson SE, Albritton K, et al. Phase II study of doxorubicin and bevacizumab for patients with metastatic soft-tissue sarcomas. J Clin Oncol. 2005;23:7135–7142
  74. Keohan ML, Morgan JA, D'Adamo DR, et al. Continuous daily dosing of sunitinib in patients with metastatic soft tissue sarcomas other than GIST: results of a phase II trial [abstract]. J Clin Oncol. 2008;26:10533
  75. Sleijfer S, Papai Z, Le Cesne A, et al. Phase II study of pazopanib (GW786034) in patients with relapsed or refractory soft tissue sarcoma: EORTC 62043. [abstract] J Clin Oncol. 2007;25:10031
  76. D'Adamo DR, Keohan M, Schuetze S, et al. Clinical results of a phase II study of sorafenib in patients (pts) with non-GIST sarcomas (CTEP study #7060). [abstract] J Clin Oncol. 2007;25:10001
  77. Fata F, O'Reilly E, Ilson D, et al. Paclitaxel in the treatment of patients with angiosarcoma of the scalp or face. Cancer. 1999;86:2034–2037
  78. Skubitz KM, Haddad PA. Paclitaxel and pegylated-liposomal doxorubicin are both active in angiosarcoma. Cancer. 2005;104:361–366
  79. Penel N, Bui BN, Bay JO, et al. Phase II trial of weekly paclitaxel for unresectable angiosarcoma: the ANGIOTAX study. J Clin Oncol. 2008;26:5269–5274
  80. Fury MG, Antonescu CR, Van Zee KJ, et al. A 14-year retrospective review of angiosarcoma: clinical characteristics, prognostic factors, and treatment outcomes with surgery and chemotherapy. Cancer J. 2005;11:241–247
  81. Weenink JJ, Groeneveld JO, de Fijter CW. Sirolimus monotherapy for Kaposi's sarcoma in an HIV-negative patient. Lancet Oncol. 2006;7:875–876
  82. Sodhi A, Chaisuparat R, Hu J, et al. The TSC2/mTOR pathway drives endothelial cell transformation induced by the Kaposi's sarcoma-associated herpesvirus G protein-coupled receptor. Cancer Cell. 2006;10:133–143
  83. Stacchiotti S, Tamborini E, Marrari A, Brich S, Arisi Rota S, Orsenigo M, et al. Response to sunitinib malate in advanced alveolar soft part sarcoma. Clin Cancer Res. 2009;15:1096–1104

 Enrique de Alava is supported by the European Commission (NoE EuroBoNet), Ministry of Science and Innovation of Spain-FEDER (PI052524, RD06/0020/0059). Silvana Pilotti is supported by Associazione Italiana per la Ricerca sul Cancro (AIRC). Robert G. Maki is supported by Program Project Grant No. P01-CA47179; Cycle for Survival. David M. Thomas is supported by the Victorian Cancer Agency Clinician Researcher fellowship, and Cancer Australia.

Disclosures Robert G. Maki: Research support (Pfizer); honoraria (Ziopharm); testimony (Roche)Margaret von Mehren: Research support (Novartis, Pfizer, Johnson & Johnson); consultation fees (Novartis, Pfizer, Medimmune)David Thomas: Research support (Novartis, Amgen, Pfizer)

PII: S0093-7754(09)00108-0

doi: 10.1053/j.seminoncol.2009.06.008

Seminars in Oncology
Volume 36, Issue 4 , Pages 358-371 , August 2009

Article Tools

* Image Usage & Resolution