Targeted therapy in bone and soft tissue sarcoma in children and adolescents

Current Oncology Reports

Volumn 14, Issue 2, 2012, Pages 197-205

  Thompson, Patrick A ^a   Chintagumpala, Murali ^a  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

Author keywords

Bone sarcoma; Pediatrics; Soft tissue sarcoma; Targeted therapy

Indexed keywords

ALISERTIB; ANTINEOPLASTIC AGENT; ARSENIC TRIOXIDE; BEVACIZUMAB; CEDIRANIB; CIXUTUMUMAB; CYCLOPHOSPHAMIDE; EPIDERMAL GROWTH FACTOR RECEPTOR 2; EZRIN; MITHRAMYCIN; MITOGEN ACTIVATED PROTEIN KINASE; NAVELBINE; NY ESO 1 ANTIGEN; PHOSPHATIDYLINOSITOL 3 KINASE; PLATELET DERIVED GROWTH FACTOR RECEPTOR; PROTEIN KINASE B; RAF PROTEIN; RAS PROTEIN; RNA BINDING PROTEIN EWS; SOMATOMEDIN C RECEPTOR; SORAFENIB; STEM CELL FACTOR RECEPTOR; SUNITINIB; TEMSIROLIMUS; TRANSCRIPTION FACTOR FLI 1; UNCLASSIFIED DRUG; VASCULOTROPIN RECEPTOR; YK 4 2709; ANGIOGENESIS INHIBITOR; PROTEIN KINASE INHIBITOR; VASCULOTROPIN;

ADOLESCENT; ALVEOLAR SOFT PART SARCOMA; ARTICLE; CANCER PROGNOSIS; CANCER RADIOTHERAPY; CANCER RECURRENCE; CANCER RESEARCH; CHILD; CHILDHOOD CANCER; DISEASE FREE SURVIVAL; DRUG TARGETING; EWING SARCOMA; HUMAN; LYMPHOCYTE TRANSFER; METASTASIS POTENTIAL; MULTIMODALITY CANCER THERAPY; NONHUMAN; ORTHOPEDIC SURGERY; OSTEOSARCOMA; OVERALL SURVIVAL; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); RANDOMIZED CONTROLLED TRIAL (TOPIC); RHABDOMYOSARCOMA; SOFT TISSUE SARCOMA; SYNOVIAL SARCOMA; BONE TUMOR; CLINICAL TRIAL (TOPIC); DRUG ANTAGONISM; METHODOLOGY; MOLECULARLY TARGETED THERAPY; PRESCHOOL CHILD; PROGNOSIS; REVIEW; SARCOMA; SOFT TISSUE TUMOR;

ADOLESCENT; ANGIOGENESIS INHIBITORS; ANTINEOPLASTIC AGENTS; BONE NEOPLASMS; CHILD; CHILD, PRESCHOOL; CLINICAL TRIALS AS TOPIC; HUMANS; MOLECULAR TARGETED THERAPY; PROGNOSIS; PROTEIN KINASE INHIBITORS; SARCOMA; SOFT TISSUE NEOPLASMS; VASCULAR ENDOTHELIAL GROWTH FACTORS;

EID: 84860873205     PISSN: 15233790     EISSN: 15346269     Source Type: Journal    
DOI: 10.1007/s11912-012-0223-2     Document Type: Article

References (85)

1. PIzzo PA, Poplack DG. Principles and Practice of Pediatric Oncology. 5th ed. Philadelphia, PA: Lippincott Williams & Wilkins; 2006. p. 1780.
2. Taylor BS, Barretina J, Maki RG, et al., Advances in sarcoma genomics and new therapeutic targets. Nat Rev Cancer. 11 (8): p. 541-57. Excellent review article focusing on the molecular changes involved in the formation of sarcomas. The review emphasizes the genomic methods that will likely have a significant impact on the development of future therapeutic strategies.
3. Ries LAG, Smith MA, Gurney JG, et al. (eds.) Soft Tissue Sarcomas. Cancer Incidence and Survival Among Children and Adolescents: United States SEER Program 1975-1995. National Cancer Institute, SEER Progam. NIH Pub. No. 99-4649. Bethesda, MD, 1999.
4. Ries LAG, Smith MA, Gurney JG, et al. (eds.) Malignant bone tumors. Cancer Incidence and Survival Among Children and Adolescents: United States SEER Program 1975-1995. National Cancer Institute, SEER Progam. NIH Pub. No. 99-4649. Bethesda, MD, 1999.
5. Wachtel M and BW Schafer, Targets for cancer therapy in childhood sarcomas. Cancer Treat Rev. 36 (4): p. 318-27.
6. Ognjanovic S, Linabery AM, Charbonneau B, Ross JA. Trends inchildhood rhabdomyosarcoma incidence and survival in the UnitedStates, 1975-2005. Cancer. 2009;115 (18): 4218-26.
7. Barr FG. Molecular genetics and pathogenesis of rhabdomyosarcoma. J Pediatr Hematol Oncol. 1997;19 (6): 483-91. (Pubitemid 28099078)
8. Barr FG, Nauta LE, Hollows JC. Structural analysis of PAX3 genomic rearrangements in alveolar rhabdomyosarcoma. Cancer Genet Cytogenet. 1998;102 (1): 32-9. (Pubitemid 28129056)
9. Barr FG, Smith LM, Lynch JC, et al. Examination of gene fusion status in archival samples of alveolar rhabdomyosarcoma entered on the Intergroup Rhabdomyosarcoma Study-III trial: a report from the Children's Oncology Group. J Mol Diagn. 2006;8 (2): 202-8. (Pubitemid 43811243)
10. Barr FG, Qualman SJ, Macris MH, et al. Genetic heterogeneity in the alveolar rhabdomyosarcoma subset without typical gene fusions. Cancer Res. 2002;62 (16): 4704-10.
11. Davicioni E, Anderson MJ, Finckenstein FG, et al. Molecular classification of rhabdomyosarcoma-genotypic and phenotypic determinants of diagnosis: a report from the Children's Oncology Group. Am J Pathol. 2009;174 (2): 550-64.
12. Parham DM, Qualman SJ, Teot L, et al. Correlation between histology and PAX/FKHR fusion status in alveolar rhabdomyosarcoma: a report from the Children's Oncology Group. Am J Surg Pathol. 2007;31 (6): 895-901. (Pubitemid 47165143)
13. Sorensen PHB, Lynch JC, Qualman SJ, et al. PAX3-FKHR and PAX7-FKHR gene fusions are prognostic indicators in alveolar rhabdomyosarcoma: a report from the children's oncology group. J Clin Oncol. 2002;20 (11): 2672-9. (Pubitemid 34575640)
14. Lukasiewicz E, Miekus K, Kijowski J, et al. Inhibition of rhabdomyosarcoma's metastatic behavior through downregulation of MET receptor signaling. Folia Histochem Cytobiol. 2009;47 (3): 485-9.
15. Davis IJ, McFadden AW, Zhang Y, et al. Identification of the receptor tyrosine kinase c-Met and its ligand, hepatocyte growth factor, as therapeutic targets in clear cell sarcoma. Cancer Res. 2010;70 (2): 639-45.
16. Gao CF, Zhang YW, Su Y, et al. Therapeutic potential of hepatocyte growth factor/scatter factor neutralizing antibodies: inhibition of tumor growth in both autocrine and paracrine hepatocyte growth factor/scatter factor: c-Met-driven models of leiomyosarcoma. Mol Cancer Ther. 2009;8 (10): 2803-10.
17. Yan D, Dong XD, Chen X, et al. MicroRNA-1/206 targets c-Met and inhibits rhabdomyosarcoma development. J Biol Chem. 2009;284 (43): 29596-604.
18. Petricoin 3rd EF, Espina V, Araujo RP, et al. Phosphoprotein pathway mapping: Akt/mammalian target of rapamycin activation is negatively associated with childhood rhabdomyosarcoma survival. Cancer Res. 2007;67 (7): 3431-40. (Pubitemid 46724884)
19. Casanova M and A Ferrari, Pharmacotherapy for pediatric softtissue sarcomas. Expert Opin Pharmacother. 12 (4): p. 517-31.
20. Kalebic T, Tsokos M, Helman LJ. In vivo treatment with antibody against IGF-1 receptor suppresses growth of human rhabdomyosarcoma and down-regulates p34cdc2. Cancer Res. 1994;54 (21): 5531-4.
21. Kalebic T, Blakesley V, Slade C, et al. Expression of a kinasedeficient IGF-I-R suppresses tumorigenicity of rhabdomyosarcoma cells constitutively expressing a wild type IGF-I-R. Int J Cancer. 1998;76 (2): 223-7. (Pubitemid 28180376)
22. Kolb EA, Gorlick R, Houghton PJ, et al. Initial testing (stage 1) of a monoclonal antibody (SCH 717454) against the IGF-1 receptor by the pediatric preclinical testing program. Pediatr Blood Cancer. 2008;50 (6): 1190-7. (Pubitemid 351555543)
23. Scotlandi K, ManaraMC, 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 (9): 3868-76. (Pubitemid 40616368)
24. Gordon IK, Ye F, Kent MS. Evaluation of the mammalian target ofrapamycin pathway and the effect of rapamycin on target expression and cellular proliferation in osteosarcoma cells from dogs. Am J Vet Res. 2008;69 (8): 1079-84.
25. Hosoi H, Dilling MB, Shikata T, et al. Rapamycin causes poorly reversible inhibition of mTOR and induces p53-independent apoptosis in human rhabdomyosarcoma cells. Cancer Res. 1999;59 (4): 886-94. (Pubitemid 29086744)
26. Houghton PJ, Morton CL, Kolb EA, et al. Initial testing (stage 1) of the mTOR inhibitor rapamycin by the pediatric preclinical testing program. Pediatr Blood Cancer. 2008;50 (4): 799-805. (Pubitemid 351354788)
27. Wan X, Shen N, Mendoza A, et al. CCI-779 inhibits rhabdomyosarcoma xenograft growth by an antiangiogenic mechanism linked to the targeting of mTOR/Hif-1alpha/VEGF signaling. Neoplasia. 2006;8 (5): 394-401. (Pubitemid 43830746)
28. Wan X, Harkavy B, Shen N, et al. Rapamycin induces feedback activation of Akt signaling through an IGF-1R-dependent mechanism. Oncogene. 2007;26 (13): 1932-40. (Pubitemid 46474636)
29. Thimmaiah KN, Easton J, Huang S, et al. Insulin-like growth factor I-mediated protection from rapamycin-induced apoptosis is independent of Ras-Erk1-Erk2 and phosphatidylinositol 3?-kinase- Akt signaling pathways. Cancer Res. 2003;63 (2): 364-74. (Pubitemid 36152494)
30. A Phase I Study of IMC-A12 in Combination With Temsirolimus in Pediatric Patients With Recurrent or Refractory Solid Tumors. 2011 [cited September 1, 2011]; Available from: http: //www.clinicaltrials.gov/ct2/show/NCT01182883.
31. IMC-A12 in Combination With Temsirolimus (CCI-779) in Patients With Advanced Cancers. 2011 [cited September 1, 2011]; Available from: http: //www.clinicaltrials.gov/ct2/show/NCT00678769.
32. Linden O, Greiff L, Wahlberg P, et al. Chemorefractory rhabdomyosarcoma treated with radiotherapy, bevacizumab, statins and surgery and maintenance with bevacizumab and chemotherapy. Onkologie. 2008;31 (7): 391-3. (Pubitemid 351967577)
33. Myers AL, Williams RF, Ng CY, et al., Bevacizumab-induced tumor vessel remodeling in rhabdomyosarcoma xenografts increases the effectiveness of adjuvant ionizing radiation. J Pediatr Surg. 45 (6): p. 1080-5.
34. Folkman J. Angiogenesis. Annu Rev Med. 2006;57: 1-18. (Pubitemid 43261975)
35. Balasubramanian L, Evens AM. Targeting angiogenesis for the treatment of sarcoma. Curr Opin Oncol. 2006;18 (4): 354-9. (Pubitemid 44297246)
36. Gee MF, Tsuchida R, Eichler-Jonsson C, et al. Vascular endothelial growth factor acts in an autocrine manner in rhabdomyosarcoma cell lines and can be inhibited with all-trans-retinoic acid. Oncogene. 2005;24 (54): 8025-37. (Pubitemid 41746746)
37. Onisto M, Slongo ML, Gregnanin L, et al. Expression and activity of vascular endothelial growth factor and metalloproteinases in alveolar and embryonal rhabdomyosarcoma cell lines. Int J Oncol. 2005;27 (3): 791-8.
38. Temozolomide, Cixutumumab, and Combination Chemotherapy in Treating Patients With Metastatic Rhabdomyosarcoma. 2011 [cited September 1, 2011]; Available from: http: //www.clinicaltrials.gov/ct2/show/NCT01055314.
39. Vinorelbine Ditartrate and Cyclophosphamide in Combination With Bevacizumab or Temsirolimus in Treating PatientsWith Recurrent or Refractory Rhabdomyosarcoma. 2011 [cited September 1, 2011]; Available from: http: //clinicaltrials.gov/ct/show/NCT01222715.
40. de Alava E, Gerald WL. Molecular biology of the Ewing's sarcoma/ primitive neuroectodermal tumor family. J Clin Oncol. 2000;18 (1): 204-13. (Pubitemid 30036354)
41. Jedlicka P, Ewing Sarcoma, an enigmatic malignancy of likely progenitor cell origin, driven by transcription factor oncogenic fusions. Int J Clin Exp Pathol. 3 (4): p. 338-47.
42. Ludwig JA. Ewing sarcoma: historical perspectives, current stateof- the-art, and opportunities for targeted therapy in the future. Curr Opin Oncol. 2008;20 (4): 412-8.
43. Subbiah Vand Anderson P, Targeted Therapy of Ewing's Sarcoma. Sarcoma. 2011: p. 686985. This comprehensive review describes recent and possible future targeted therapies in Ewing's sarcoma.
44. Cotterill SJ, Ahrens S, Paulussen M, et al. Prognostic factors in Ewing's tumor of bone: analysis of 975 patients from the European Intergroup Cooperative Ewing's Sarcoma Study Group. J Clin Oncol. 2000;18 (17): 3108-14.
45. Burdach S, Jurgens H. High-dose chemoradiotherapy (HDC) in the Ewing family of tumors (EFT). Crit Rev Oncol Hematol. 2002;41 (2): 169-89. (Pubitemid 34150128)
46. Arvand A, Denny CT. Biology of EWS/ETS fusions in Ewing's family tumors. Oncogene. 2001;20 (40): 5747-54. (Pubitemid 32928112)
47. Janknecht R. EWS-ETS oncoproteins: the linchpins of Ewing tumors. Gene. 2005;363: 1-14. (Pubitemid 41691887)
48. Kovar H. Context matters: the hen or egg problem in Ewing's sarcoma. Semin Cancer Biol. 2005;15 (3): 189-96.
49. Ladanyi M. EWS-FLI1 and Ewing's sarcoma: recent molecular data and new insights. Cancer Biol Ther. 2002;1 (4): 330-6.
50. Riggi N, Stamenkovic I. The Biology of Ewing sarcoma. Cancer Lett. 2007;254 (1): 1-10. (Pubitemid 47058800)
51. Erkizan HV, Kong Y, Merchant M, et al., A small molecule blocking oncogenic protein EWS-FLI1 interaction with RNA helicase A inhibits growth of Ewing's sarcoma. Nat Med, 2009. 15 (7): p. 750-6. This manuscript describes the identification of YK-4- 279, a small molecule that blocks the binding of RNA helicase A (RHA) to EWS-FLI1 and induces apoptosis in ESFT cells and reduces the growth of ESFT orthotopic xenografts.
52. Grohar PJ, Woldemichael GM, Griffin LB, et al., Identification of an inhibitor of the EWS-FLI1 oncogenic transcription factor by high-throughput screening. J Natl Cancer Inst. 103 (12): p. 962-78.
53. Olmos D, Martins AS, Jones RL, et al., Targeting the Insulin-Like Growth Factor 1 Receptor in Ewing's Sarcoma: Reality and Expectations. Sarcoma. 2011: p. 402508.
54. Toretsky JA, Steinberg SM, Thakar M, et al. Insulin-like growth factor type 1 (IGF-1) and IGF binding protein-3 in patients with Ewing sarcoma family of tumors. Cancer. 2001;92 (11): 2941-7.
55. Yee D, Favoni RE, Lebovic GS, et al. Insulin-like growth factor I expression by tumors of neuroectodermal origin with the t (11;22) chromosomal translocation. A potential autocrine growth factor. J Clin Invest. 1990;86 (6): 1806-14.
56. Strammiello R, Benini S, Manara MC, et al. Impact of IGF-I/IGFIR circuit on the angiogenetic properties of Ewing's sarcoma cells. Horm Metab Res. 2003;35 (11-12): 675-84. (Pubitemid 38058171)
57. Iwasa T, Okamoto I, Suzuki M, et al. Inhibition of insulin-like growth factor 1 receptor by CP-751, 871 radiosensitizes non-small cell lung cancer cells. Clin Cancer Res. 2009;15 (16): 5117-25.
58. Maris JM, Morton CL, Gorlick R, et al., Initial testing of the aurora kinase A inhibitor MLN8237 by the Pediatric Preclinical Testing Program (PPTP). Pediatr Blood Cancer. 55 (1): p. 26-34.
59. Beauchamp EM, Ringer L, Bulut G, et al., Arsenic trioxide inhibits human cancer cell growth and tumor development in mice by blocking Hedgehog/GLI pathway. J Clin Invest. 121 (1): p. 148-60.
60. MLN8237 in Treating Young Patients With Recurrent or Refractory Solid Tumors or Leukemia. 2011 [cited September 1, 2011]; Available from: http: //clinicaltrials.gov/ct2/show/NCT01154816.
61. Fraumeni Jr JF. Stature and malignant tumors of bone in childhood and adolescence. Cancer. 1967;20 (6): 967-73.
62. Kager L, Zoubek A, Potschger U, et al. Primary metastatic osteosarcoma: presentation and outcome of patients treated on neoadjuvant Cooperative Osteosarcoma Study Group protocols. J Clin Oncol. 2003;21 (10): 2011-8. (Pubitemid 46606391)
63. Meyers PA, Gorlick R. Osteosarcoma. Pediatr Clin North Am. 1997;44 (4): 973-89. (Pubitemid 27368674)
64. Geller DS and Gorlick R, HER-2 targeted treatment of osteosarcoma: the challenges of developing targeted therapy and prognostic factors for rare malignancies. Expert Opin Pharmacother. 11 (1): p. 51-61.
65. Grignani G, Palmerini E, Dileo P, et al., A phase II trial of sorafenib in relapsed and unresectable high-grade osteosarcoma after failure of standard multimodal therapy: an Italian Sarcoma Group study. Ann Oncol.
66. Kubo T, Piperdi S, Rosenblum J, et al. Platelet-derived growth factor receptor as a prognostic marker and a therapeutic target for imatinib mesylate therapy in osteosarcoma. Cancer. 2008;112 (10): 2119-29. (Pubitemid 351628620)
67. McGary EC, Weber K, Mills L, et al. Inhibition of platelet-derived growth factor-mediated proliferation of osteosarcoma cells by the novel tyrosine kinase inhibitor STI571. Clin Cancer Res. 2002;8 (11): 3584-91. (Pubitemid 35340737)
68. Sulzbacher I, Traxler M, Mosberger I, et al. Platelet-derived growth factor-AA and -alpha receptor expression suggests an autocrine and/or paracrine loop in osteosarcoma. Mod Pathol. 2000;13 (6): 632-7. (Pubitemid 30436799)
69. Manara MC, Nicoletti G, Zambelli D, et al., NVP-BEZ235 as a new therapeutic option for sarcomas. Clin Cancer Res. 16 (2): p. 530-40.
70. Abdeen A, Chou AJ, Healey JH, et al. Correlation between clinical outcome and growth factor pathway expression in osteogenic sarcoma. Cancer. 2009;115 (22): 5243-50.
71. Pignochino Y, Grignani G, Cavalloni G, et al. Sorafenib blocks tumour growth, angiogenesis and metastatic potential in preclinical models of osteosarcoma through a mechanism potentially involving the inhibition of ERK1/2, MCL-1 and ezrin pathways. Mol Cancer. 2009;8: 118.
72. Bulut G, Hong SH, Chen K, et al., Small molecule inhibitors of ezrin inhibit the invasive phenotype of osteosarcoma cells. Oncogene. Jun 27. doi: 10.1038/onc.2011.245. [Epub ahead of print]. High ezrin expression is associated with lung metastasis and poor survival in osteosarcoma. This manuscript describes the identification of small-molecule inhibitors of ezrin which inhibit lung metastases and demonstrate that this novel target may have a future role in preventing tumor metastasis.
73. Wan X, Kim SY, Guenther LM, et al. Beta4 integrin promotes osteosarcoma metastasis and interacts with ezrin. Oncogene. 2009;28 (38): 3401-11.
74. Khanna C, Wan X, Bose S, et al. The membrane-cytoskeleton linker ezrin is necessary for osteosarcoma metastasis. Nat Med. 2004;10 (2): 182-6. (Pubitemid 38524889)
75. Khanna C, Khan J, Nguyen P, et al. Metastasis-associated differences in gene expression in a murine model of osteosarcoma. Cancer Res. 2001;61 (9): 3750-9. (Pubitemid 32694989)
76. Ahmed N, Salsman VS, Yvon E, et al., Immunotherapy for osteosarcoma: genetic modification of T cells overcomes low levels of tumor antigen expression. Mol Ther, 2009. 17 (10): p. 1779-87. This manuscript demonstrates that the adoptive transfer of HER2- specific T cells can cause regression of established osteosarcoma xenografts in locoregional as well as metastatic mouse models.
77. Scotlandi K, Manara MC, Hattinger CM, et al. Prognostic and therapeutic relevance of HER2 expression in osteosarcoma and Ewing's sarcoma. Eur J Cancer. 2005;41 (9): 1349-61. (Pubitemid 40813013)
78. Sorafenib in Relapsed High Grade Osteosarcoma. 2011 [cited September 1, 2011]; Available from: http: //www.clinicaltrials.gov/ct2/show/NCT00889057.
79. A Study of Bevacizumab in Combination With Chemotherapy for Treatment of Osteosarcoma. 2011 [cited September 1, 2011]; Available from: http: //www.clinicaltrials.gov/ct2/show/NCT00667342.
80. Her2 Chimeric Antigen Receptor Expressing T Cells in Advanced Sarcoma. 2011 [cited September 1, 2011]; Available from: http: // www.clinicaltrials.gov/ ct2/show/NCT00902044.
81. Stacchiotti S, Negri T, Zaffaroni N, et al., Sunitinib in advanced alveolar soft part sarcoma: evidence of a direct antitumor effect. Ann Oncol. 22 (7): p. 1682-90.
82. Stacchiotti S, Tamborini E, Marrari A, et al. Response to sunitinib malate in advanced alveolar soft part sarcoma. Clin Cancer Res. 2009;15 (3): 1096-104.
83. Robbins PF, Morgan RA, Feldman SA, et al., Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1. J Clin Oncol. 29 (7): p. 917-24. This manuscript describes a clinical trial carried out to evaluate the ability of adoptively transferred autologous T cells transduced with a T-cell receptor directed against NY-ESO-1 to mediate tumor regression in patients with metastatic melanoma and synovial cell sarcoma. Objective clinical responses were observed in four of six patients with synovial cell sarcoma.
84. A Pilot Study of Genetically Engineered NY-ESO-1 Specific (c259) T Cells in HLA-A2+ Patients With Synovial Sarcoma. 2011 [cited September 1, 2011]; Available from: http: //www.clinicaltrials.gov/ct2/show/NCT01343043.
85. Sunitinib or Cediranib for Alveolar Soft Part Sarcoma. 2011 [cited September 1, 2011.]; Available from: http: //www.clinicaltrials.gov/ct2/show/ NCT01391962.