Reduction of human embryonal rhabdomyosarcoma tumor growth by inhibition of the hedgehog signaling pathway

Genes and Cancer

Volumn 1, Issue 9, 2010, Pages 941-951

  Tostar, Ulrica ^a   Toftgård, Rune ^a   Zaphiropoulos, Peter G ^a   Shimokawa, Takashi ^a  

^a KAROLINSKA INSTITUTE   (Sweden)

Author keywords

Embryonal rhabdomyosarcoma; GANT; GLI; Hedgehog signaling

Indexed keywords

CYCLOPAMINE; GANT 61; HEDGEHOG INTERACTING PROTEIN; PROTEIN INHIBITOR; PROTEIN PATCHED 1; SMALL INTERFERING RNA; SONIC HEDGEHOG PROTEIN; TRANSCRIPTION FACTOR; TRANSCRIPTION FACTOR GLI1; TRANSCRIPTION FACTOR GLI2; TRANSCRIPTION FACTOR GLI3; TRANSCRIPTION FACTOR SMO; UNCLASSIFIED DRUG;

APOPTOSIS; ARTICLE; CANCER CELL CULTURE; CANCER INHIBITION; CELL PROLIFERATION; CONTROLLED STUDY; DRUG EFFICACY; EMBRYONAL RHABDOMYOSARCOMA; GENE EXPRESSION REGULATION; GENE MUTATION; GENE TARGETING; GLI1 GENE; GROWTH INHIBITION; HHIP GENE; HUMAN; HUMAN CELL; HUMAN TISSUE; IN VIVO STUDY; NECROSIS; PRIORITY JOURNAL; PTCH1 GENE; SFRP1 GENE; SIGNAL TRANSDUCTION; SINGLE DRUG DOSE; TRANSCRIPTION INITIATION; TUMOR SUPPRESSOR GENE; UPREGULATION;

ERINACEIDAE;

EID: 79952820588     PISSN: 19476019     EISSN: 19476027     Source Type: Journal    
DOI: 10.1177/1947601910385449     Document Type: Article

References (36)

1. Ries L, Smith M, Guerney J, et al. Cancer incidence and survival among children and adolescents: United States SEER program 1975-1995. NIH Pub. No. 99-4649. Bethesda, MD: National Cancer Institute; 1999.
2. Merlino G, Helman LJ. Rhabdomyosarcoma: working out the pathways. Oncogene. 1999;18:5340-8.
3. Parham D, Barr F. Embryonal rhabdomyosarcoma. In: Fletcher CDM, Unni KK, Mertens F, editors. World Health Organization Classification of Tumours. Pathology and genetics: tumours of soft tissue and bone. Lyon: IARC Press; 2002. p. 146-9.
4. Parham D, Barr F. Alveolar rhabdomyosarcoma. In: Fletcher CDM, Unni KK, Mertens F, editors. World Health Organization Classification of Tumours. Pathology and genetics: tumours of soft tissue and bone. Lyon: IARC Press; 2002. p. 150-2.
5. Zhan S, Shapiro DN, Helman LJ. Activation of an imprinted allele of the insulin-like growth factor II gene implicated in rhabdomyosarcoma. J Clin Invest. 1994;94:445-8.
6. Varjosalo M, Taipale J. Hedgehog: functions and mechanisms. Genes Dev. 2008;22:2454-72.
7. Teglund S, Toftgård R. Hedgehog beyond medulloblastoma and basal cell carcinoma. Biochim Biophys Acta. 2010;1805:181-208.
8. Chen JK, Taipale J, Cooper MK, Beachy PA. Inhibition of hedgehog signaling by direct binding of cyclopamine to smoothened. Genes Dev. 2002;16:2743-8.
9. Von Hoff DD, LoRusso PM, Rudin CM, et al. Inhibition of the hedgehog pathway in advanced basal-cell carcinoma. N Engl J Med.2009;361:1164-72.
10. Scales SJ, de Sauvage FJ. Mechanisms of hedgehog pathway activation in cancer and implications for therapy. Trends Pharmacol Sci.2009;30:303-12.
11. Lauth M, Bergström A, Shimokawa T, Toftgård R. Inhibition of GLImediated transcription and tumor cell growth by small-molecule antagonists. Proc Natl Acad Sci U S A. 2007;104:8455-60.
12. Tostar U, Malm CJ, Meis-Kindblom JM, Kindblom LG, Toftgård R, Unden AB. Deregulation of the hedgehog signalling pathway: a possible role for the PTCH and SUFU genes in human rhabdomyoma and rhabdomyosarcoma development. J Pathol. 2006;208:17-25.
13. Bridge JA, Liu J, Weibolt V, et al. Novel genomic imbalances in embryonal rhabdomyosarcoma revealed by comparative genomic hybridization and fluorescence in situ hybridization: an intergroup rhabdomyosarcoma study. Genes Chromosomes Cancer. 2000;27:337-44.
14. Hahn H, Wojnowski L, Zimmer AM, Hall J, Miller G, Zimmer A. Rhabdomyosarcomas and radiation hypersensitivity in a mouse model of Gorlin syndrome. Nat Med. 1998;4:619-22.
15. Lee Y, Kawagoe R, Sasai K, et al. Loss of suppressor-of-fused function promotes tumorigenesis. Oncogene. 2007;26:6442-7.
16. Svärd J, Rozell B, Toftgård R, Teglund S. Tumor suppressor gene cooperativity in compound Patched1 and suppressor of fused heterozygous mutant mice. Mol Carcinog. 2009;48:408-19.
17. Mao J, Ligon KL, Rakhlin EY, et al. A novel somatic mouse model to survey tumorigenic potential applied to the hedgehog pathway. Cancer Res. 2006;66:10171-8.
18. Gorlin RJ. Nevoid basal-cell carcinoma syndrome. Medicine (Baltimore).1987;66:98-113.
19. Hagedorn M, Javerzat S, Gilges D, et al. Accessing key steps of human tumor progression in vivo by using an avian embryo model. Proc Natl Acad Sci U S A. 2005;102:1643-8.
20. Wang B, Fallon JF, Beachy PA. Hedgehog-regulated processing of Gli3 produces an anterior/posterior repressor gradient in the developing vertebrate limb. Cell. 2000;100:423-34.
21. Tsanev R, Tiigimagi P, Michelson P, Metsis M, Osterlund T, Kogerman P. Identification of the gene transcription repressor domain of Gli3. FEBS Lett. 2009;583:224-8.
22. Bridge JA, Liu J, Qualman SJ, et al. Genomic gains and losses are similar in genetic and histologic subsets of rhabdomyosarcoma, whereas amplification predominates in embryonal with anaplasia and alveolar subtypes. Genes Chromosomes Cancer. 2002;33:310-21.
23. Ecke I, Rosenberger A, Obenauer S, et al. Cyclopamine treatment of full-blown Hh/Ptch-associated RMS partially inhibits Hh/Ptch signaling, but not tumor growth. Mol Carcinog. 2008;47:361-72.
24. Zwerner JP, Joo J, Warner KL, et al. The EWS/FLI1 oncogenic transcription factor deregulates GLI1. Oncogene. 2008;27:3282-91.
25. Beauchamp E, Bulut G, Abaan O, et al. GLI1 is a direct transcriptional target of EWS-FLI1 oncoprotein. J Biol Chem. 2009;284:9074-82.
26. Joo J, Christensen L, Warner K, et al. GLI1 is a central mediator of EWS/FLI1 signaling in Ewing tumors. PLoS One. 2009;4:e7608.
27. Sasaki H, Nishizaki Y, Hui C, Nakafuku M, Kondoh H. Regulation of Gli2 and Gli3 activities by an amino-terminal repression domain: implication of Gli2 and Gli3 as primary mediators of Shh signaling. Development. 1999;126:3915-24.
28. Nolan-Stevaux O, Lau J, Truitt ML, et al. GLI1 is regulated through smoothened-independent mechanisms in neoplastic pancreatic ducts and mediates PDAC cell survival and transformation. Genes Dev.2009;23:24-36.
29. Humke EW, Dorn KV, Milenkovic L, Scott MP, Rohatgi R. The output of hedgehog signaling is controlled by the dynamic association between suppressor of Fused and the Gli proteins. Genes Dev. 2010;24:670-82.
30. Kimura H, Ng JM, Curran T. Transient inhibition of the hedgehog pathway in young mice causes permanent defects in bone structure. Cancer Cell. 2008;13:249-60.
31. Park HL, Bai C, Platt KA, et al. Mouse Gli1 mutants are viable but have defects in SHH signaling in combination with a Gli2 mutation. Development. 2000;127:1593-605.
32. Roberts I, Gordon A, Wang R, Pritchard-Jones K, Shipley J, Coleman N. Molecular cytogenetic analysis consistently identifies translocations involving chromosomes 1, 2 and 15 in five embryonal rhabdomyosarcoma cell lines and a PAX-FOXO1A fusion gene negative alveolar rhabdomyosarcoma cell line. Cytogenet Cell Genet.2001;95:134-42.
33. Clayton J, Pincott JR, van den Berghe JA, Kemshead JT. Comparative studies between a new human rhabdomyosarcoma cell line, JR-1 and its tumour of origin. Br J Cancer. 1986;54:83-90.
34. De Giovanni C, Nanni P, Nicoletti G, et al. Metastatic ability and differentiative properties of a new cell line of human embryonal rhabdomyosarcoma (CCA). Anticancer Res. 1989;9:1943-9.
35. Brooks PC, Montgomery AM, Rosenfeld M, et al. Integrin alpha v beta 3 antagonists promote tumor regression by inducing apoptosis of angiogenic blood vessels. Cell. 1994;79:1157-64.
36. Shimokawa T, Svard J, Heby-Henricson K, Teglund S, Toftgard R, Zaphiropoulos PG. Distinct roles of first exon variants of the tumor-suppressor Patched1 in hedgehog signaling. Oncogene. 2007;26: 4889-96.