PKC412 inhibits in vitro growth of neoplastic human mast cells expressing the D816V-mutated variant of KIT: Comparison with AMN107, imatinib, and cladribine (2CdA) and evaluation of cooperative drug effects

Blood

Volumn 107, Issue 2, 2006, Pages 752-759

  Gleixner, Karoline V ^b   Mayerhofer, Matthias ^b   Aichberger, Karl J ^b   Derdak, Sophia ^b   Sonneck, Karoline ^b   Böhm, Alexandra ^b   Gruze, Alexander ^b   Samorapoompichit, Puchit ^b   Manley, Paul W ^b   Fabbro, Doriano ^b   Pickl, Winfried F ^b   Sillaber, Christian ^b   Valent, Peter ^a  

^a MEDICAL UNIVERSITY OF VIENNA   (Austria)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA INTERFERON; CLADRIBINE; DOXYCYCLINE; IMATINIB; MIDOSTAURIN; NILOTINIB; PROTEIN TYROSINE KINASE; STEM CELL FACTOR;

ADULT; ANIMAL CELL; APOPTOSIS; ARTICLE; CANCER INHIBITION; CONTROLLED STUDY; DOWN REGULATION; DRUG EFFECT; DRUG POTENCY; DRUG POTENTIATION; DRUG SCREENING; DRUG TARGETING; ENZYME ACTIVITY; FEMALE; GENE MUTATION; GENETIC VARIABILITY; HUMAN; HUMAN CELL; IC 50; MAST CELL LEUKEMIA; MOUSE; NONHUMAN; OBSERVATION; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN INDUCTION; PROTEIN TARGETING; WILD TYPE;

EID: 30444446680     PISSN: 00064971     EISSN: 00064971     Source Type: Journal    
DOI: 10.1182/blood-2005-07-3022     Document Type: Article

References (54)

1. Reilly JT. Class III receptor tyrosine kinases: role in leukaemogenesis. Br J Haematol. 2002;116:744-757.
2. Deininger MW, Druker BJ. Specific targeted therapy of chronic myelogenous leukemia with imatinib. Pharmacol Rev. 2003;55:401-423.
3. Pardanani A, Tefferi A. Imatinib targets other than bcr/abl and their clinical relevance in myeloid disorders. Blood. 2004;104:1931-1939.
4. Shah NP, Tran C, Lee FY, Chen P, Norris D, Sawyers CL. Overriding imatinib resistance with a novel ABL kinase inhibitor. Science. 2004;305: 399-401.
5. Fabbro D, Ruetz S, Bodis S, et al. PKC412 - a protein kinase inhibitor with a broad therapeutic potential. Anticancer Drug Des. 2000;15:17-28.
6. Lennert K, Parwaresch MR. Mast cells and mast cell neoplasia: a review. Histopathology. 1979;3: 349-365.
7. Metcalfe DD. Classification and diagnosis of mastocytosis: current status. J Invest Dermatol. 1991; 96:2S-4S.
8. Valent P. Biology, classification and treatment of human mastocytosis. Wien Klin Wochenschr. 1996;108:385-397.
9. Valent P, Akin C, Sperr WR, et al. Diagnosis and treatment of systemic mastocytosis: state of the art. Br J Haematol. 2003;122:695-717.
10. Akin C, Metcalfe DD. Systemic mastocytosis. Annu Rev Med. 2004;55:419-432.
11. Tefferi A, Pardanani A. Clinical, genetic, and therapeutic insights into systemic mast cell disease. Curr Opin Hematol. 2004;11:58-64.
12. Valent P, Ghannadan M, Akin C, et al. On the way to targeted therapy of mast cell neoplasms: identification of molecular targets in neoplastic mast cells and evaluation of arising treatment concepts. Eur J Clin Invest. 2004;34:41-52.
13. Nagata H, Worobec AS, Oh CK, et al. Identification of a point mutation in the catalytic domain of the proto-oncogene c-kit in peripheral blood mononuclear cells of patients who have mastocytosis with an associated hematologic disorder. Proc Natl Acad Sci U S A. 1995;92:10560-10564.
14. Longley BJ, Tyrrell L, Lu SZ, et al. Somatic c-kit activating mutation in urticaria pigmentosa and aggressive mastocytosis: establishment of clonality in a human mast cell neoplasm. Nat Genet. 1996;12:312-314.
15. Longley BJ, Metcalfe DD, Tharp M, et al. Activating and dominant inactivating c-kit catalytic domain mutations in distinct forms of human mastocytosis. Proc Natl Acad Sci U S A. 1999;96:1609-1614.
16. Fritsche-Polanz R, Jordan JH, Feix A, et al. Mutation analysis of C-KIT in patients with myelodys-plastic syndromes without mastocytosis and cases of systemic mastocytosis. Br J Haematol. 2001;113:357-364.
17. Feger F, Ribadeau Dumas A, Leriche L, Valent P, Arock M. Kit and c-kit mutations in mastocytosis: a short overview with special reference to novel molecular and diagnostic concepts. Int Arch Allergy Immunol. 2002;127:110-114.
18. Furitsu T, Tsujimura T, Tono T, et al. Identification of mutations in the coding sequence of the proto-oncogene c-kit in a human mast cell leukemia cell line causing ligand-independent activation of the c-kit product. J Clin Invest. 1993;92:1736-1744.
19. Tefferi A, Pardanani A. Systemic mastocytosis: current concepts and treatment advances. Curr Hematol Rep. 2004;3:197-202.
20. Akin C, Brockow K, D'Ambrosio C, et al. Effects of tyrosine kinase inhibitor STI571 on human mast cells bearing wild-type or mutated forms of c-kit. Exp Hematol. 2003;31:686-692.
21. Ma Y, Zeng S, Metcalfe DD, et al. The c-KIT mutation causing human mastocytosis is resistant to STI571 and other KIT kinase inhibitors; kinases with enzymatic site mutations show different inhibitor sensitivity profiles than wild-type kinases and those with regulatory type mutations. Blood. 2002;99:1741-1744.
22. Frost MJ, Ferrao PT, Hughes TP, Ashman LK. Juxtamembrane mutant V560GKit is more sensitive to imatinib (STI571) compared with wild-type c-kit whereas the kinase domain mutant D816VKit is resistant. Mol Cancer Ther. 2002;1: 1115-1124.
23. Akin C, Fumo G, Yavuz AS, Lipsky PE, Neckers L, Metcalfe DD. A novel form of mastocytosis associated with a transmembrane c-kit mutation and response to imatinib. Blood. 2004;103:3222-3225.
24. Pardanani A, Ketterling RP, Brockman SR, et al. CHIC2 deletion, a surrogate for FIP1L1-PDGFRA fusion, occurs in systemic mastocytosis associated with eosinophilia and predicts response to imatinib mesylate therapy. Blood. 2003;102:3093-3096.
25. Pardanani A, Elliott M, Reeder T, et al. Imatinib for systemic mast-cell disease. Lancet. 2003;362: 535-536.
26. Pardanani A. Systemic mastocytosis: bone marrow pathology, classification, and current therapies. Acta Haematol. 2005;114:41-51.
27. Weisberg E, Manley PW, Breitenstein W, et al. Characterization of AMN107, a selective inhibitor of native and mutant Bcr-Abl. Cancer Cell. 2005; 7:129-141.
28. Butterfield JH, Weiler D, Dewald G, Gleich GJ. Establishment of an immature mast cell line from a patient with mast cell leukemia. Leuk Res. 1988;12:345-355.
29. Sillaber C, Strobl H, Bevec D, et al. IL-4 regulates c-kit proto-oncogene product expression in human mast and myeloid progenitor cells. J Immunol. 1991;147:4224-4228.
30. Mayerhofer M, Aichberger KJ, Florian S, et al. c-kit D816V provides a strong signal for myelomastocytic differentiation and cluster formation in murine Ba/F3 cells [abstract]. Blood. 2004;104:141a.
31. Daley GQ, Baltimore D. Transformation of an interleukin 3-dependent hematopoietic cell line by the chronic myelogenous leukemia-specific P210bcr/abl protein. Proc Natl Acad Sci U S A. 1988;85:9312-9316.
32. Sillaber C, Gesbert F, Frank DA, Sattler M, Griffin JD. STAT5 activation contributes to growth and viability in Bcr/Abl-transformed cells. Blood. 2000;95:2118-2125.
33. Valent P, Horny H-P, Escribano L, et al. Diagnostic criteria and classification of mastocytosis: a consensus proposal. Conference Report of "Year 2000 Working Conference on Mastocytosis." Leuk Res. 2001;25:603-625.
34. Valent P, Horny H-P, Li CY, et al. Mastocytosis (mast cell disease). In: Jaffe ES, Harris NL, Stein H, Vardiman JW, eds. World Health Organization (WHO) Classification of Tumours: Pathology & Genetics: Tumours of Haematopoietic and Lymphoid Tissues. Lyon, France: IARC Press; 2001: 291-302.
35. Yavuz AS, Lipsky PE, Yavuz S, Metcalfe DD, Akin C. Evidence for the involvement of a hematopoietic progenitor cell in systemic mastocytosis from single-cell analysis of mutations in the c-kit gene. Blood. 2002;100:661-665.
36. Valent P, Akin C, Sperr WR, Horny HP, Metcalfe DD. Smouldering mastocytosis: a novel subtype of systemic mastocytosis with slow progression. Int Arch Allergy Immunol. 2002;127:137-139.
37. Broudy VC, Lin N, Zsebo KM, et al. Isolation and characterization of a monoclonal antibody that recognizes the human c-kit receptor. Blood. 1992; 79:338-346.
38. Bühring HJ, Ashman LK, Gattei V, et al. Stem-cell factor receptor (p145(c-kit)) summary report (CD117). In: Schlossmann SF, Boumsell L, Gilks W, et al, eds. Leucocyte Typing V: White Cell Differentiation Antigens. Vol 2. Oxford, United Kingdom: Oxford University Press; 1995:1882-1888.
39. Chou TC, Talalay P. Quantitative analysis of dose-effect relationships: the combined effects of multiple drugs or enzyme inhibitors. Adv Enzyme Regul. 1984;22:27-55.
40. Van Cruchten S, Van Den Broeck W. Morphological and biochemical aspects of apoptosis, oncosis and necrosis. Anat Histol Embryol. 2002;31: 214-223.
41. Schedle A, Samorapoompichit P, Füreder W, et al. Metal ion-induced toxic histamine release from human basophils and mast cells. J Biomed Mater Res. 1998;39:560-567.
42. Samorapoompichit P, Kiener HP, Schernthaner GH, et al. Detection of tryptase in cytoplasmic granules of basophils in patients with chronic myeloid leukemia and other myeloid neoplasms. Blood. 2001;98:2580-2583.
43. Gorczyca W, Gong J, Darzynkiewicz Z. Detection of strand breaks in individual apoptotic cells by the in situ terminal deoxynucleotidal transferase and nick translation assays. Cancer Res. 1993;53:1945-1951.
44. Walker PR, Carson C, Leblanc J, Sikorska M. Labeling DNA damage with terminal transferase: applicability, specificity, and limitations. Methods Mol Biol. 2002;203:3-19.
45. Escribano L, Diaz-Agustin B, Bellas C, et al. Utility of flow cytometric analysis of mast cells in the diagnosis and classification of adult mastocytosis. Leuk Res. 2001;25:563-570.
46. Valent P, Schernthaner GH, Sperr WR, et al. Variable expression of activation-linked surface antigens on human mast cells in health and disease. Immunol Rev. 2001;179:74-81.
47. Ghannadan M, Hauswirth AW, Schernthaner GH, et al. Detection of novel CD antigens on the surface of human mast cells and basophils. Int Arch Allergy Immunol. 2002;127:299-307.
48. Growney JD, Clark JJ, Adelsperger J, et al. Activation mutations of human c-KIT resistant to imatinib are sensitive to the tyrosine kinase inhibitor PKC412. Blood. 2005;106:721-724.
49. Corless CL, Fletcher JA, Heinrich MC. Biology of gastrointestinal stromal tumors. J Clin Oncol. 2004;22:3813-3825.
50. Cowan-Jacob SW, Guez V, Fendrich G, et al. Imatinib (STI571) resistance in chronic myelogenous leukemia: molecular basis of the underlying mechanisms and potential strategies for treatment. Mini Rev Med Chem. 2004;4:285-299.
51. Weisberg E, Griffin JD. Resistance to imatinib (Glivec): update on clinical mechanisms. Drug Resist Updat. 2003;6:231-238.
52. Gotlib J, Berube C, Growney JD, et al. Activity of the tyrosine kinase inhibitor PKC412 in a patient with mast cell leukemia with the D816V KIT mutation. Blood. 2005;106:2865-2870.
53. Droogendijk HJ, Kluin-Nelemans JC, van Daele PLA. Imatinib mesylate in the treatment of systemic mastocytosis: a phase I/II trial [abstract]. Blood. 2004;104:1516a.
54. Kluin-Nelemans HC, Oldhoff JM, Van Doormaal JJ, et al. Cladribine therapy for systemic mastocytosis. Blood. 2003;102:4270-4276.