Mutations of FLT3/ITD confer resistance to multiple tyrosine kinase inhibitors (original) (raw)
Small D, Levenstein M, Kim E, Carrow C, Amin S, Rockwell P et al. STK-1, the human homolog of Flk-2/Flt-3, is selectively expressed in CD34+ human bone marrow cells and is involved in the proliferation of early progenitor/stem cells. Proc Natl Acad Sci USA 1994; 91: 459–463. ArticleCASPubMedPubMed Central Google Scholar
Rosnet O, Schiff C, Pebusque MJ, Marchetto S, Tonnelle C, Toiron Y et al. Human FLT3/FLK2 gene: cDNA cloning and expression in hematopoietic cells. Blood 1993; 82: 1110–1119. CASPubMed Google Scholar
Lavagna-Sevenier C, Marchetto S, Birnbaum D, Rosnet O . FLT3 signaling in hematopoietic cells involves CBL, SHC and an unknown P115 as prominent tyrosine-phosphorylated substrates. Leukemia 1998; 12: 301–310. ArticleCASPubMed Google Scholar
Rosnet O, Buhring HJ, deLapeyriere O, Beslu N, Lavagna C, Marchetto S et al. Expression and signal transduction of the FLT3 tyrosine kinase receptor. Acta Haematol 1996; 95: 218–223. ArticleCASPubMed Google Scholar
Lyman SD, James L, Vanden Bos T, de Vries P, Brasel K, Gliniak B et al. Molecular cloning of a ligand for the flt3/flk-2 tyrosine kinase receptor: a proliferative factor for primitive hematopoietic cells. Cell 1993; 75: 1157–1167. ArticleCASPubMed Google Scholar
Zhang S, Broxmeyer HE . Flt3 ligand induces tyrosine phosphorylation of gab1 and gab2 and their association with shp-2, grb2, and PI3 kinase. Biochem Biophys Res Commun 2000; 277: 195–199. ArticleCASPubMed Google Scholar
Brown P, Levis M, Shurtleff S, Campana D, Downing J, Small D . FLT3 inhibition selectively kills childhood acute lymphoblastic leukemia cells with high levels of FLT3 expression. Blood 2005; 105: 812–820. ArticleCASPubMed Google Scholar
Armstrong SA, Staunton JE, Silverman LB, Pieters R, den Boer ML, Minden MD et al. MLL translocations specify a distinct gene expression profile that distinguishes a unique leukemia. Nat Genet 2002; 30: 41–47. ArticleCASPubMed Google Scholar
Zheng R, Levis M, Piloto O, Brown P, Baldwin BR, Gorin NC et al. FLT3 ligand causes autocrine signaling in acute myeloid leukemia cells. Blood 2004; 103: 267–274. ArticleCASPubMed Google Scholar
Nakao M, Yokota S, Iwai T, Kaneko H, Horiike S, Kashima K et al. Internal tandem duplication of the flt3 gene found in acute myeloid leukemia. Leukemia 1996; 10: 1911–1918. CASPubMed Google Scholar
Abu-Duhier FM, Goodeve AC, Wilson GA, Care RS, Peake IR, Reilly JT . Identification of novel FLT-3 Asp835 mutations in adult acute myeloid leukaemia. Br J Haematol 2001; 113: 983–988. ArticleCASPubMed Google Scholar
Yamamoto Y, Kiyoi H, Nakano Y, Suzuki R, Kodera Y, Miyawaki S et al. Activating mutation of D835 within the activation loop of FLT3 in human hematologic malignancies. Blood 2001; 97: 2434–2439. ArticleCASPubMed Google Scholar
Griffith J, Black J, Faerman C, Swenson L, Wynn M, Lu F et al. The structural basis for autoinhibition of FLT3 by the juxtamembrane domain. Mol Cell 2004; 13: 169–178. ArticleCASPubMed Google Scholar
Smith CC, Wang Q, Chin CS, Salerno S, Damon LE, Levis MJ et al. Validation of ITD mutations in FLT3 as a therapeutic target in human acute myeloid leukaemia. Nature 2012; 485: 260–263. ArticleCASPubMedPubMed Central Google Scholar
Alvarado Y, Kantarjian H, Ravandi F, Luthra R, Borthakur G, Manero GG et al. FLT3 inhibitor treatment in FLT3-mutated AML is associated with development of secondary FLT3-TKD mutations. Blood 2011; 118: 1493. Google Scholar
Zhang W, Konopleva M, Jacamo RO, Borthakur G, Chen W, Cortes JE et al. Acquired point mutations of TKD are responsible for sorafenib resistance in FLT3-ITD mutant AML. Blood 2011, 118.
Cools J, Mentens N, Furet P, Fabbro D, Clark JJ, Griffin JD et al. Prediction of resistance to small molecule FLT3 inhibitors: implications for molecularly targeted therapy of acute leukemia. Cancer Res 2004; 64: 6385–6389. ArticleCASPubMed Google Scholar
von Bubnoff N, Engh RA, Aberg E, Sanger J, Peschel C, Duyster J . FMS-like tyrosine kinase 3-internal tandem duplication tyrosine kinase inhibitors display a nonoverlapping profile of resistance mutations in vitro. Cancer Res 2009; 69: 3032–3041. ArticleCASPubMed Google Scholar
Bagrintseva K, Schwab R, Kohl TM, Schnittger S, Eichenlaub S, Ellwart JW et al. Mutations in the tyrosine kinase domain of FLT3 define a new molecular mechanism of acquired drug resistance to PTK inhibitors in FLT3-ITD-transformed hematopoietic cells. Blood 2004; 103: 2266–2275. ArticleCASPubMed Google Scholar
Zhou J, Bi C, Janakakumara JV, Liu SC, Chng WJ, Tay KG et al. Enhanced activation of STAT pathways and overexpression of survivin confer resistance to FLT3 inhibitors and could be therapeutic targets in AML. Blood 2009; 113: 4052–4062. ArticleCASPubMed Google Scholar
Stolzel F, Steudel C, Oelschlagel U, Mohr B, Koch S, Ehninger G et al. Mechanisms of resistance against PKC412 in resistant FLT3-ITD positive human acute myeloid leukemia cells. Ann Hematol89: 653–662. ArticlePubMed Google Scholar
Prescott H, Kantarjian H, Cortes J, Ravandi F . Emerging FMS-like tyrosine kinase 3 inhibitors for the treatment of acute myelogenous leukemia. Expert Opin Emerg Drugs 2011; 16: 407–423. ArticleCASPubMedPubMed Central Google Scholar
Tse KF, Allebach J, Levis M, Smith BD, Bohmer FD, Small D . Inhibition of the transforming activity of FLT3 internal tandem duplication mutants from AML patients by a tyrosine kinase inhibitor. Leukemia 2002; 16: 2027–2036. ArticleCASPubMed Google Scholar
Azam M, Latek RR, Daley GQ . Mechanisms of autoinhibition and STI-571/imatinib resistance revealed by mutagenesis of BCR-ABL. Cell 2003; 112: 831–843. ArticleCASPubMed Google Scholar
Levis M, Allebach J, Tse KF, Zheng R, Baldwin BR, Smith BD et al. A FLT3-targeted tyrosine kinase inhibitor is cytotoxic to leukemia cells in vitro and in vivo. Blood 2002; 99: 3885–3891. ArticleCASPubMed Google Scholar
Tse KF, Mukherjee G, Small D . Constitutive activation of FLT3 stimulates multiple intracellular signal transducers and results in transformation. Leukemia 2000; 14: 1766–1776. ArticleCASPubMed Google Scholar
Zhu X, Kim JL, Newcomb JR, Rose PE, Stover DR, Toledo LM et al. Structural analysis of the lymphocyte-specific kinase Lck in complex with non-selective and Src family selective kinase inhibitors. Structure 1999; 7: 651–661. ArticleCASPubMed Google Scholar
Wood ER, Truesdale AT, McDonald OB, Yuan D, Hassell D, Dickerson SH et al. A unique structure for epidermal growth factor receptor bound to GW572016 (Lapatinib): relationships among protein conformation, inhibitor off-rate, and receptor activity in tumor cells. Cancer Res 2004; 64: 6652–6659. ArticleCASPubMed Google Scholar
Simard JR, Grutter C, Pawar V, Aust B, Wolf A, Rabiller M et al. High-throughput screening to identify inhibitors which stabilize inactive kinase conformations in p38alpha. J Am Chem Soc 2009; 131: 18478–18488. ArticleCASPubMed Google Scholar
Grebien F, Hantschel O, Wojcik J, Kaupe I, Kovacic B, Wyrzucki AM et al. Targeting the SH2-kinase interface in Bcr-Abl inhibits leukemogenesis. Cell 2011; 147: 306–319. ArticleCASPubMedPubMed Central Google Scholar
Breitenbuecher F, Markova B, Kasper S, Carius B, Stauder T, Bohmer FD et al. A novel molecular mechanism of primary resistance to FLT3-kinase inhibitors in AML. Blood 2009; 113: 4063–4073. ArticleCASPubMed Google Scholar
el-Shami K, Stone RM, Smith BD . FLT3 inhibitors in acute myeloid leukemia. Expert Rev Hematol 2008; 1: 153–160. ArticleCASPubMed Google Scholar
Knapper S, Mills KI, Gilkes AF, Austin SJ, Walsh V, Burnett AK . The effects of lestaurtinib (CEP701) and PKC412 on primary AML blasts: the induction of cytotoxicity varies with dependence on FLT3 signaling in both FLT3-mutated and wild-type cases. Blood 2006; 108: 3494–3503. ArticleCASPubMed Google Scholar
Siendones E, Barbarroja N, Torres LA, Buendia P, Velasco F, Dorado G et al. Inhibition of Flt3-activating mutations does not prevent constitutive activation of ERK/Akt/STAT pathways in some AML cells: a possible cause for the limited effectiveness of monotherapy with small-molecule inhibitors. Hematol Oncol 2007; 25: 30–37. ArticleCASPubMed Google Scholar
Kurzrock R, Talpaz M . The molecular pathology of chronic myelogenous leukaemia. Br J Haematol 1991; 79 (Suppl 1): 34–37. ArticlePubMed Google Scholar
Druker BJ . Current treatment approaches for chronic myelogenous leukemia. Cancer J. 2001; 7 (Suppl 1): S14–S18. PubMed Google Scholar
Druker BJ, Talpaz M, Resta DJ, Peng B, Buchdunger E, Ford JM et al. Efficacy and safety of a specific inhibitor of the BCR-ABL tyrosine kinase in chronic myeloid leukemia. N Engl J Med 2001; 344: 1031–1037. ArticleCASPubMed Google Scholar
O′Brien SG, Guilhot F, Larson RA, Grathman I, Baccarani M, Cervantes F et al. Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med 2003; 348: 994–1004. ArticlePubMed Google Scholar
Apperley JF, Part I . mechanisms of resistance to imatinib in chronic myeloid leukaemia. Lancet Oncol 2007; 8: 1018–1029. ArticleCASPubMed Google Scholar
O′Hare T, Deininger MW, Eide CA, Clackson T, Druker BJ . Targeting the BCR-ABL signaling pathway in therapy-resistant Philadelphia chromosome-positive leukemia. Clin Cancer Res 2011; 17: 212–221. ArticlePubMed Google Scholar
Soverini S, Hochhaus A, Nicolini FE, Gruber F, Lange T, Saglio g et al. BCR-ABL kinase domain mutation analysis in chronic myeloid leukemia patients treated with tyrosine kinase inhibitors: recommendations from an expert panel on behalf of European LeukemiaNet. Blood 2011; 118: 1208–1215. ArticleCASPubMed Google Scholar
Clark JJ, Cools J, Curley DP, Yu JC, Lokker NA, Giese NA et al. Variable sensitivity of FLT3 activation loop mutations to the small molecule tyrosine kinase inhibitor MLN518. Blood 2004; 104: 2867–2872. ArticleCASPubMed Google Scholar
Liu Y, Gray NS . Rational design of inhibitors that bind to inactive kinase conformations. Nat Chem Biol 2006; 2: 358–364. ArticleCASPubMed Google Scholar
Cowan-Jacob SW, Fendrich G, Floersheimer A, Furet P, Liebetanz J, Rummel G et al. Structural biology contributions to the discovery of drugs to treat chronic myelogenous leukaemia. Acta Crystallogr D Biol Crystallogr 2007; 63: 80–93. ArticleCASPubMed Google Scholar
Weisberg E, Roesel J, Furet P, Bold G, Imbach P, Florsheimer J et al. Antileukemic effects of novel first- and second-generation FLT3 inhibitors: structure-affinity comparison. Genes Cancer 2010; 1: 1021–1032. ArticleCASPubMedPubMed Central Google Scholar
von Bubnoff N, Rummelt C, Menzel H, Sigl M, Peschel C, Duyster J . Identification of a secondary FLT3/A848P mutation in a patient with FLT3-ITD-positive blast phase CMML and response to sunitinib and sorafenib. Leukemia 2010; 24: 1523–1525. ArticleCASPubMed Google Scholar
Kindler T, Breitenbuecher F, Kasper S, Estey E, Giles F, Feldman E et al. Identification of a novel activating mutation (Y842C) within the activation loop of FLT3 in patients with acute myeloid leukemia (AML). Blood 2005; 105: 335–340. ArticleCASPubMed Google Scholar
Jiang J, Paez JG, Lee JC, Bo R, Stone RM, DeAngelo DJ et al. Identifying and characterizing a novel activating mutation of the FLT3 tyrosine kinase in AML. Blood 2004; 104: 1855–1858. ArticleCASPubMed Google Scholar
Mony U, Jawad M, Seedhouse C, Russell N, Pallis M . Resistance to FLT3 inhibition in an in vitro model of primary AML cells with a stem cell phenotype in a defined microenvironment. Leukemia 2008; 22: 1395–1401. ArticleCASPubMed Google Scholar
Sato T, Yang X, Knapper S, White P, Smith BD, Galkin S et al. FLT3 ligand impedes the efficacy of FLT3 inhibitors in vitro and in vivo. Blood 2011; 117: 3286–3293. ArticleCASPubMedPubMed Central Google Scholar
Yoshimoto G, Miyamoto T, Jabbarzadeh-Tabrizi S, Iino T, Rocnik JL, Kikushige Y et al. FLT3-ITD up-regulates MCL-1 to promote survival of stem cells in acute myeloid leukemia via FLT3-ITD-specific STAT5 activation. Blood 2009; 114: 5034–5043. ArticleCASPubMedPubMed Central Google Scholar
Heidel F, Solem FK, Breitenbuecher F, Lipka DB, Kasper S, Thiede MH et al. Clinical resistance to the kinase inhibitor PKC412 in acute myeloid leukemia by mutation of Asn-676 in the FLT3 tyrosine kinase domain. Blood 2006; 107: 293–300. ArticleCASPubMed Google Scholar