Design and pharmacology of a highly specific dual FMS and KIT kinase inhibitor

Proceedings of the National Academy of Sciences of the United States of America

Volumn 110, Issue 14, 2013, Pages 5689-5694

  Zhang, Chao ^a   Ibrahim, Prabha N ^a   Zhang, Jiazhong ^a   Burton, Elizabeth A ^a   Habets, Gaston ^a   Zhang, Ying ^a   Powell, Ben ^a   West, Brian L ^a   Matusow, Bernice ^a   Tsang, Garson ^a   Shellooe, Rafe ^a   Carias, Heidi ^a   Nguyen, Hoa ^a   Marimuthu, Adhirai ^a   Zhang, Kam Y J ^a   Oh, Angela ^a   Bremer, Ryan ^a   Hurt, Clarence R ^a   Artis, Dean R ^a   Wu, Guoxian ^a   Nespi, Marika ^a   Spevak, Wayne ^a   Lin, Paul ^a   Nolop, Keith ^a   Hirth, Peter ^a   Tesch, Greg H ^b   Bollag, Gideon ^a   more..

^a PLEXXIKON INC   (United States)

^b MONASH UNIVERSITY   (Australia)

Author keywords

Cancer bone metastasis; CSF1R; Rheumatoid arthritis; Scaffold based drug design

Indexed keywords

FMS KINASE INHIBITOR; INTERLEUKIN 6; KIT KINASE INHIBITOR; LIPOPOLYSACCHARIDE; PHOSPHOTRANSFERASE INHIBITOR; PLX 647; TUMOR NECROSIS FACTOR ALPHA; UNCLASSIFIED DRUG;

ACUTE KIDNEY INFLAMMATION; ALLODYNIA; ANIMAL CELL; ANIMAL CELL CULTURE; ANIMAL TISSUE; AREA UNDER THE CURVE; ARTHRITIS; ARTICLE; BONE PAIN; CELL ACTIVATION; CRYSTAL STRUCTURE; CYTOKINE RELEASE; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DESIGN; DRUG PROTEIN BINDING; DRUG SELECTIVITY; DRUG STRUCTURE; DRUG TARGETING; MACROPHAGE; MAST CELL; MOUSE; NEPHRITIS; NONHUMAN; OSTEOCLAST; OSTEOLYSIS; PASSIVE SKIN ANAPHYLAXIS; PRIORITY JOURNAL; RAT; UNILATERAL URETER OBSTRUCTION; URETER OBSTRUCTION; VIRUS ONCOGENE;

AMINOPYRIDINES; ANIMALS; CELL SURVIVAL; CHROMATOGRAPHY, AFFINITY; CRYSTALLIZATION; ESCHERICHIA COLI; HUMAN UMBILICAL VEIN ENDOTHELIAL CELLS; HUMANS; INDOLES; INFLAMMATION; MACROPHAGES; MAST CELLS; MODELS, MOLECULAR; MOLECULAR STRUCTURE; MUTATION, MISSENSE; NEOPLASM METASTASIS; ONCOGENE PROTEIN GP140(V-FMS); OSTEOCLASTS; PROTEIN CONFORMATION; PROTEIN KINASE INHIBITORS; PROTO-ONCOGENE PROTEINS C-KIT; PYRROLES; SF9 CELLS; SPODOPTERA;

FELIDAE;

EID: 84875862910     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1219457110     Document Type: Article

References (48)

1. Kontzias A, Laurence A, Gadina M, O'Shea JJ (2012) Kinase inhibitors in the treatment of immune-mediated disease. F1000. Med-Rep 4:5.
2. Fleischmann R, et al.; ORAL Solo Investigators (2012) Placebo-controlled trial of tofacitinib monotherapy in rheumatoid arthritis. N Engl J Med 367(6):495-507.
3. Sherr CJ, et al. (1985) The c-fms proto-oncogene product is related to the receptor for the mononuclear phagocyte growth factor, CSF-1. Cell 41(3):665-676.
4. Stanley ER, Cifone M, Heard PM, Defendi V (1976) Factors regulating macrophage production and growth: Identity of colony-stimulating factor and macrophage growth factor. J Exp Med 143(3):631-647.
5. Lin H, et al. (2008) Discovery of a cytokine and its receptor by functional screening of the extracellular proteome. Science 320(5877):807-811.
6. Liu H, et al. (2012) The mechanism of shared but distinct CSF-1R signaling by the non-homologous cytokines IL-34 and CSF-1. Biochim Biophys Acta 1824(7):938-945.
7. Ma X, et al. (2012) Structural basis for the dual recognition of helical cytokines IL-34 and CSF-1 by CSF-1R. Structure 20(4):676-687.
8. Wei S, et al. (2010) Functional overlap but differential expression of CSF-1 and IL-34 in their CSF-1 receptor-mediated regulation ofmyeloid cells. J Leukoc Biol 88(3):495-505.
9. Chitu V, Stanley ER (2006) Colony-stimulating factor-1 in immunity and inflammation. Curr Opin Immunol 18(1):39-48.
10. Murray PJ, Wynn TA (2011) Protective and pathogenic functions of macrophage subsets. Nat Rev Immunol 11(11):723-737.
11. Ruffell B, Affara NI, Coussens LM (2012) Differential macrophage programming in the tumor microenvironment. Trends Immunol 33(3):119-126.
12. Ahn GO, et al. (2010) Inhibition of Mac-1 (CD11b/CD18) enhances tumor response to radiation by reducing myeloid cell recruitment. Proc Natl Acad Sci USA 107(18):8363-8368.
13. DeNardo DG, et al. (2011) Leukocyte complexity predicts breast cancer survival and functionally regulates response to chemotherapy. Cancer Discov 1(1):54-67.
14. El-Gamal MI, Anbar HS, Yoo KH, Oh CH (2012) FMS Kinase Inhibitors: Current Status and Future Prospects. Med Res Rev, 10.1002/med.21258.
15. Yarden Y, et al. (1987) Human proto-oncogene c-kit: A new cell surface receptor tyrosine kinase for an unidentified ligand. EMBO J 6(11):3341-3351.
16. Furitsu T, et al. (1993) Identification of mutations in the coding sequence of the proto-oncogene c-kit in a human mast cell leukemia cell line causing ligandindependent activation of c-kit product. J Clin Invest 92(4):1736-1744.
17. Hirota S, et al. (1998) Gain-of-function mutations of c-kit in human gastrointestinal stromal tumors. Science 279(5350):577-580.
18. Carvajal RD, et al. (2011) KIT as a therapeutic target in metastatic melanoma. JAMA 305(22):2327-2334.
19. Demetri GD, et al. (2002) Efficacy and safety of imatinib mesylate in advanced gastrointestinal stromal tumors. N Engl J Med 347(7):472-480.
20. Goodman VL, et al. (2007) Approval summary: Sunitinib for the treatment of imatinib refractory or intolerant gastrointestinal stromal tumors and advanced renal cell carcinoma. Clin Cancer Res 13(5):1367-1373.
21. Staser K, Yang FC, Clapp DW (2012) Pathogenesis of plexiform neurofibroma: Tumorstromal/ hematopoietic interactions in tumor progression. Annu Rev Pathol 7:469-495.
22. Chang DZ (2012) Mast cells in pancreatic ductal adenocarcinoma. OncoImmunology 1(5):754-755.
23. Paniagua RT, et al. (2010) c-Fms-mediated differentiation and priming of monocyte lineage cells play a central role in autoimmune arthritis. Arthritis Res Ther 12(1):R32.
24. Reber L, Da Silva CA, Frossard N (2006) Stem cell factor and its receptor c-Kit as targets for inflammatory diseases. Eur J Pharmacol 533(1-3):327-340.
25. Eklund KK, Joensuu H (2003) Treatment of rheumatoid arthritis with imatinib mesylate: Clinical improvement in three refractory cases. Ann Med 35(5):362-367.
26. Miyachi K, et al. (2003) Efficacy of imatinib mesylate (STI571) treatment for a patient with rheumatoid arthritis developing chronic myelogenous leukemia. Clin Rheumatol 22(4-5):329-332.
27. Card GL, et al. (2005) A family of phosphodiesterase inhibitors discovered by cocrystallography and scaffold-based drug design. Nat Biotechnol 23(2):201-207.
28. Tsai J, et al. (2008) Discovery of a selective inhibitor of oncogenic B-Raf kinase with potent antimelanoma activity. Proc Natl Acad Sci USA 105(8):3041-3046.
29. Artis DR, et al. (2009) Scaffold-based discovery of indeglitazar, a PPAR pan-active antidiabetic agent. Proc Natl Acad Sci USA 106(1):262-267.
30. Bollag G, et al. (2010) Clinical efficacy of a RAF inhibitor needs broad target blockade in BRAF-mutant melanoma. Nature 467(7315):596-599.
31. Bollag G, et al. (2012) Vemurafenib: The first drug approved for BRAF-mutant cancer. Nat Rev Drug Discov 11(11):873-886.
32. Kumar A, et al. (2005) Crystal structures of proto-oncogene kinase Pim1: A target of aberrant somatic hypermutations in diffuse large cell lymphoma. J Mol Biol 348(1):183-193.
33. Hubbard SR (2004) Juxtamembrane autoinhibition in receptor tyrosine kinases. Nat Rev Mol Cell Biol 5(6):464-471.
34. Daley GQ, Baltimore D (1988) Transformation of an interleukin 3-dependent hematopoietic cell line by the chronic myelogenous leukemia-specific P210bcr/abl protein. Proc Natl Acad Sci USA 85(23):9312-9316.
35. Lacey DL, et al. (1998) Osteoprotegerin ligand is a cytokine that regulates osteoclast differentiation and activation. Cell 93(2):165-176.
36. Yoshida H, et al. (1990) The murine mutation osteopetrosis is in the coding region of the macrophage colony stimulating factor gene. Nature 345(6274):442-444.
37. Minkin C (1982) Bone acid phosphatase: Tartrate-resistant acid phosphatase as a marker of osteoclast function. Calcif Tissue Int 34(3):285-290.
38. Nakao M, et al. (1996) Internal tandem duplication of the flt3 gene found in acute myeloid leukemia. Leukemia 10(12):1911-1918.
39. Wershil BK, Mekori YA, Murakami T, Galli SJ (1987) 125I-fibrin deposition in IgEdependent immediate hypersensitivity reactions in mouse skin. Demonstration of the role of mast cells using genetically mast cell-deficient mice locally reconstituted with cultured mast cells. J Immunol 139(8):2605-2614.
40. Le Meur Y, et al. (2002) Macrophage accumulation at a site of renal inflammation is dependent on the M-CSF/c-fms pathway. J Leukoc Biol 72(3):530-537.
41. Brand DD, Kang AH, Rosloniec EF (2004) The mouse model of collagen-induced arthritis. Methods Mol Med 102:295-312.
42. Medhurst SJ, et al. (2002) A rat model of bone cancer pain. Pain 96(1-2):129-140.
43. Ohno H, et al. (2006) A c-fms tyrosine kinase inhibitor, Ki20227, suppresses osteoclast differentiation and osteolytic bone destruction in a bone metastasis model. Mol Cancer Ther 5(11):2634-2643.
44. Ohno H, et al. (2008) The orally-active and selective c-Fms tyrosine kinase inhibitor Ki20227 inhibits disease progression in a collagen-induced arthritis mouse model. Eur J Immunol 38(1):283-291.
45. Cummings SR, et al.; FREEDOM Trial (2009) Denosumab for prevention of fractures in postmenopausal women with osteoporosis. N Engl J Med 361(8):756-765.
46. Suva LJ, Washam C, Nicholas RW, Griffin RJ (2011) Bone metastasis: Mechanisms and therapeutic opportunities. Nat Rev Endocrinol 7(4):208-218.
47. Dai XM, et al. (2002) Targeted disruption of the mouse colony-stimulating factor 1 receptor gene results in osteopetrosis, mononuclear phagocyte deficiency, increased primitive progenitor cell frequencies, and reproductive defects. Blood 99(1):111-120.
48. Galli SJ, Kitamura Y (1987) Genetically mast-cell-deficient W/Wv and Sl/Sld mice. Their value for the analysis of the roles of mast cells in biologic responses in vivo. Am J Pathol 127(1):191-198.