453
Views
2
CrossRef citations to date
0
Altmetric
Commentary

Targeted therapy in KITD816V-positive mastocytosis: waiting for proof-of-principle

&
Pages 360-362 | Published online: 08 Feb 2010

References

  • Horny HP, Metcalfe DD, Bennett JM, et al Mastocytosis. In: Swerdlow SH, Campo E, Harris NL, et al editors. WHO classification of tumours of haematopoietic and lymphoid tissues. Lyon: International Agency for Research on Cancer (IARC); 2008.
  • Lim KH, Tefferi A, Lasho TL, et al Systemic mastocytosis in 342 consecutive adults: survival studies and prognostic factors. Blood 2009;113:5727–5736.
  • Lim KH, Pardanani A, Butterfield JH, Li CY, Tefferi A. Cytoreductive therapy in 108 adults with systemic mastocytosis: outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxyadenosine. Am J Hematol 2009;84:790–794.
  • Pan J, Quintas-Cardama A, Manshouri T, Cortes J, Kantarjian H, Verstovsek S. Sensitivity of human cells bearing oncogenic mutant kit isoforms to the novel tyrosine kinase inhibitor INNO-406. Cancer Sci 2007;98:1223–1225.
  • 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.
  • 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.
  • Schittenhelm MM, Shiraga S, Schroeder A, et al Dasatinib (BMS-354825), a dual SRC/ABL kinase inhibitor, inhibits the kinase activity of wild-type, juxtamembrane, and activation loop mutant KIT isoforms associated with human malignancies. Cancer Res 2006;66:473–481.
  • Gleixner KV, Mayerhofer M, Aichberger KJ, et al 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 2006;107:752–759.
  • Verstovsek S, Akin C, Manshouri T, et al Effects of AMN107, a novel aminopyrimidine tyrosine kinase inhibitor, on human mast cells bearing wild-type or mutated codon 816 c-kit. Leuk Res 2006;30:1365–1370.
  • Corbin AS, Griswold IJ, La Rosee P, et al Sensitivity of oncogenic KIT mutants to the kinase inhibitors MLN518 and PD180970. Blood 2004;104:3754–3757.
  • Growney JD, Clark JJ, Adelsperger J, et al Activation mutations of human c-KIT resistant to imatinib mesylate are sensitive to the tyrosine kinase inhibitor PKC412. Blood 2005;106:721–724.
  • Pan J, Quintas-Cardama A, Kantarjian HM, et al EXEL-0862, a novel tyrosine kinase inhibitor, induces apoptosis in vitro and ex vivo in human mast cells expressing the KIT D816V mutation. Blood 2007;109:315–322.
  • Shah NP, Lee FY, Luo R, Jiang Y, Donker M, Akin C. Dasatinib (BMS-354825) inhibits KITD816V, an imatinib-resistant activating mutation that triggers neoplastic growth in most patients with systemic mastocytosis. Blood 2006;108:286–291.
  • Jin Y, Chen Q, Shi X, et al Activity of triptolide against human mast cells harboring the kinase domain mutant KIT. Cancer Sci 2009;100:1335–1343.
  • Kondo R, Gleixner KV, Mayerhofer M, et al Identification of heat shock protein 32 (Hsp32) as a novel survival factor and therapeutic target in neoplastic mast cells. Blood 2007;110:661–669.
  • Faderl S, Pal A, Bornmann W, et al Kit inhibitor APcK110 induces apoptosis and inhibits proliferation of acute myeloid leukemia cells. Cancer Res 2009;69:3910–3917.
  • Pardanani A, Elliott M, Reeder T, et al Imatinib for systemic mast-cell disease. Lancet 2003;362:535–536.
  • Droogendijk HJ, Kluin-Nelemans HJ, van Doormaal JJ, Oranje AP, van de Loosdrecht AA, van Daele PL. Imatinib mesylate in the treatment of systemic mastocytosis: a phase II trial. Cancer 2006;107:345–351.
  • Vega-Ruiz A, Cortes JE, Sever M, et al Phase II study of imatinib mesylate as therapy for patients with systemic mastocytosis. Leuk Res 2009;33:1481–1484.
  • 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.
  • Verstovsek S, Tefferi A, Cortes J, et al Phase II study of dasatinib in Philadelphia chromosome-negative acute and chronic myeloid diseases, including systemic mastocytosis. Clin Cancer Res 2008;14:3906–3915.
  • Purtill D, Cooney J, Sinniah R, et al Dasatinib therapy for systemic mastocytosis: four cases. Eur J Haematol 2008;80:456–458.
  • Zappulla JP, Dubreuil P, Desbois S, et al Mastocytosis in mice expressing human Kit receptor with the activating Asp816Val mutation. J Exp Med 2005;202:1635–1641.
  • Ferrao PT, Gonda TJ, Ashman LK. Constitutively active mutant D816VKit induces megakaryocyte and mast cell differentiation of early haemopoietic cells from murine foetal liver. Leuk Res 2003;27:547–555.
  • Gleixner KV, Mayerhofer M, Sonneck K, et al Synergistic growth-inhibitory effects of two tyrosine kinase inhibitors, dasatinib and PKC412, on neoplastic mast cells expressing the D816V-mutated oncogenic variant of KIT. Haematologica 2007;92:1451–1459.
  • Mayerhofer M, Gleixner KV, Hoelbl A, et al Unique effects of KIT D816V in BaF3 cells: induction of cluster formation, histamine synthesis, and early mast cell differentiation antigens. J Immunol 2008;180:5466–5476.
  • Chian R, Young S, Danilkovitch-Miagkova A, et al Phosphatidylinositol 3 kinase contributes to the transformation of hematopoietic cells by the D816V c-Kit mutant. Blood 2001;98:1365–1373.
  • Harir N, Boudot C, Friedbichler K, et al Oncogenic Kit controls neoplastic mast cell growth through a Stat5/PI3-kinase signaling cascade. Blood 2008;112:2463–2473.
  • Weiler SR, Mou S, DeBerry CS, et al JAK2 is associated with the c-kit proto-oncogene product and is phosphorylated in response to stem cell factor. Blood 1996;87:3688–3693.
  • Pardanani A. JAK2 inhibitor therapy in myeloproliferative disorders: rationale, preclinical studies and ongoing clinical trials. Leukemia 2008;22:23–30.
  • Pardanani A, Hood J, Lasho T, et al TG101209, a small molecule JAK2-selective kinase inhibitor potently inhibits myeloproliferative disorder-associated JAK2V617F and MPLW515L/K mutations. Leukemia 2007;21:1658–1668.
  • Lasho TL, Tefferi A, Hood JD, Verstovsek S, Gilliland DG, Pardanani A. TG101348, a JAK2-selective antagonist, inhibits primary hematopoietic cells derived from myeloproliferative disorder patients with JAK2V617F, MPLW515K or JAK2 exon 12 mutations as well as mutation negative patients. Leukemia 2008;22:1790–1792.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.