Activity of the tyrosine kinase inhibitor PKC412 in a patient with mast cell leukemia with the D816V KIT mutation

Blood

Volumn 106, Issue 8, 2005, Pages 2865-2870

  Gotlib, Jason ^a   Berubé, Caroline ^b   Growney, Joseph D ^b   Chen, Ching Cheng ^b   George, Tracy I ^b   Williams, Christopher ^b   Kajiguchi, Tomohiro ^b   Ruan, Jia ^b   Lilleberg, Stan L ^b   Durocher, Jeffrey A ^b   Lichy, Jack H ^b   Wang, Yanfeng ^b   Cohen, Pamela S ^b   Arber, Daniel A ^b   Heinrich, Michael C ^b   Neckers, Len ^b   Galli, Stephen J ^b   Gilliland, D Gary ^b   Coutré, Steven E ^b  

^a STANFORD CANCER INSTITUTE   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

FUROSEMIDE; MIDOSTAURIN; PROTEIN TYROSINE KINASE INHIBITOR; SPIRONOLACTONE; TYROSINE KINASE RECEPTOR;

ADULT; ARTICLE; BLOOD CELL; CASE REPORT; CELL GROWTH; CLINICAL TRIAL; DRUG ACTIVITY; DRUG BLOOD LEVEL; DRUG EFFICACY; FEMALE; GENE; GENE FREQUENCY; GENE KIT; GENE MUTATION; HISTAMINE BLOOD LEVEL; HUMAN; HYPERBILIRUBINEMIA; IC 50; LIVER DISEASE; LIVER FUNCTION; MAST CELL LEUKEMIA; MYELODYSPLASTIC SYNDROME; MYELOPROLIFERATIVE DISORDER; NAUSEA; PHASE 1 CLINICAL TRIAL; PHOSPHORYLATION; PRIORITY JOURNAL; STATISTICAL SIGNIFICANCE; VOMITING;

EID: 27144506215     PISSN: 00064971     EISSN: 00064971     Source Type: Journal    
DOI: 10.1182/blood-2005-04-1568     Document Type: Article

References (29)

1. 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. Vol. 2. Geneva, Switzerland: WHO; 2001:291-302.
2. 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.
3. Galli SJ, Zsebo KM, Geissler EN. The kit ligand, stem cell factor. Adv Immunol 1994;55:1-96.
4. Nagata H, Worobec AS, Oh CK, et al. Identification of a point mutation in the catalytic domain of the protooncogene c-kit in peripheral blood mononuclear cells of patients who have mastocytosis with an associated hematologic disorder. Proc Natl Acad Sci U SA. 1995;92:10560-10564.
5. Longley BJ, Tyrrell L, Lu Sz, et al. Somatic c-KIT activating mutation in uticaria pigmentosa and aggressive mastocytosis: establishment of clonality in a human mast cell neoplasm. Nat Genet. 1996;12:312-314.
6. Furitsu T, Tsujimura T, Tono T, et al. Identification of mutations in the coding sequence of the protooncogene c-kit in a human mast cell leukemia cell line causing ligand-independent activation of c-kit product. J Clin Invest. 1993;92:1736-1744.
7. Casassus P, Caillat-Vigneron N, Martin A, et al. Treatment of adult systemic mastocytosis with interferon-alfa: results of a multicentre phase II trial on 20 patients. Br J Haematol. 2002;119:1090-1097.
8. Hauswirth AW, Simonitsch-Klupp I, Uffmann M, et al. Response to therapy with interferon-alpha-2b and prednisolone in aggressive systemic mastocytosis: report of five cases and review of literature. Leuk Res. 2003;28:249-257.
9. Kluin-Nelemans HC, Oldhoff JM, Van Doormaal JJ, et al. Cladribine therapy for systemic mastocytosis. Blood. 2003;102:4270-4276.
10. Akin C, Brockow K, D'Ambrosio C, et al. Effects of tyrosine kinase inhibitor STI571 on human mast cells bearing wild-type or mutated c-kit. Exp Hematol. 2003;31:686-692.
11. 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.
12. Pardanani A, Elliott M, Reeder T, et al. Imatinib for systemic mast-cell disease. Lancet. 2003;362:535-536.
13. Propper DJ, McDonald AC, Man A, et al. Phase I and pharmacokinetic study of PKC412, an inhibitor of protein kinase C. J Clin Oncol. 2001;19:1485-1492.
14. Andersson J, Sjogren H, Meis-Kindblom JM, Stenman G, Aman P, Kindblom LG. The complexity of KIT gene mutations and chromosome rearrangements and their clinical correlation in gastrointestinal stromal (pacemaker cell) tumors. Am J Pathol. 2002;160:15-22.
15. Lilleberg SL, Durocher J, Sanders C, Walters K, Culver K. High sensitivity scanning of colorectal tumors and matched plasma DNA for mutations in APC, TP53, K-RAS, and BRAF genes with a novel DHPLC fluorescence detection platform. Ann N Y Acad Sci. 2004;1022:250-256.
16. Qiu P, Shandilya H, D'Alessio JM, O'Connor K, Durocher J, Gerard GF. Mutation detection using Surveyor nuclease. Biotech. 2004;6:702-707.
17. Fumo G, Akin C, Metcalfe D, Neckers L. 17-Allylamino-17- demethoxygeldanamycin (17-AAG) is effective in down-regulating mutated, constitutively activated KIT protein in human mast cells. Blood. 2004;103:1078-1084.
18. Cancer Therapy Evaluation Program: Common terminology criteria for adverse events, version 3.0. Bethesda, MD: National Cancer Institute, December 2003.
19. 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.
20. Casteran N, De Sepulveda P, Beslu N, et al. Signal transduction by several KIT juxtamembrane domain mutations. Oncogene. 2003;22:4710-4722.
21. Ning ZQ, Li J, Arceci RJ. Signal transducer and activator of transcription 3 activation is required for Asp(816) mutant c-Kit-mediated cytokine-independent survival and proliferation in human leukemia cells. Blood. 2001;97:3559-3567.
22. Weisberg E, Boulton C, Kelly LM, et al. Inhibition of mutant FLT3 receptors in leukemia cells by the small molecule tyrosine kinase inhibitor PKC412. Cancer Cell 2002;1:433-443.
23. Fabbro D, Ruetz S, Bodis S, et al. PKC412 - a protein kinase inhibitor with a broad therapeutic potential. Anticancer Drug Res. 2000;15:17-28.
24. Chen J, DeAngelo DJ, Kutok JL, et al. PKC412 inhibits the zinc finger 198-fibroblast growth factor receptor 1 fusion tyrosine kinase and is active in the treatment of stem cell myeloproliferative disorder. Proc Natl Acad Sci U S A. 2004;101:14479-14484.
25. Stone RM, DeAngelo DJ, Klimek V, et al. Patients with acute myeloid leukemia and an activating mutation in FLT3 respond to a small-molecule FLT3 tyrosine kinase inhibitor, PKC412. Blood. 2005;105:54-60.
26. Cools J, Stover EH, Boulton CL, et al. PKC412 overcomes resistance to imatinib in a murine model of FIP1L1-PDGFRalpha-induced myeloproliferative disease. Cancer Cell. 2003;3:459-469.
27. Valent P, Akin C, Sperr WR, et al. Aggressive systemic mastocytosis and related mast cell disorders: current treatment options and proposed response criteria. Leuk Res. 2003;27:635-641.
28. Sperr WR, Stehberger B, Wimazal F, et al. Serum tryptase measurements in patients with myelodysplastic syndromes. Leuk Lymphoma. 2002;43:1097-1105.
29. Gilliland DG. FLT3 inhibitors in the treatment of AML. Clin Adv Hematol Oncol. 2004;2:708-710.