Crenolanib inhibits the drug-resistant PDGFRA D842V mutation associated with imatinib-resistant gastrointestinal stromal tumors

Clinical Cancer Research

Volumn 18, Issue 16, 2012, Pages 4375-4384

  Heinrich, Michael C ^a   Griffith, Diana ^a   McKinley, Arin ^a   Patterson, Janice ^a   Presnell, Ajia ^a   Ramachandran, Abhijit ^b   Debiec Rychter, Maria ^c  

^a OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^b AROG Pharmaceuticals LLC   (United States)

^c UNIVERSITY OF LEUVEN   (Belgium)

Author keywords

[No Author keywords available]

Indexed keywords

CRENOLANIB; IMATINIB; PLATELET DERIVED GROWTH FACTOR ALPHA KINASE; PLATELET DERIVED GROWTH FACTOR ALPHA RECEPTOR; PLATELET DERIVED GROWTH FACTOR BETA RECEPTOR; PROTEIN KINASE; UNCLASSIFIED DRUG;

ANIMAL CELL; ANTIPROLIFERATIVE ACTIVITY; ARTICLE; BINDING AFFINITY; CANCER CELL CULTURE; CANCER GROWTH; CONCENTRATION RESPONSE; CONTROLLED STUDY; DRUG BINDING; DRUG INHIBITION; DRUG POTENCY; ENZYME ACTIVITY; ENZYME INHIBITION; GASTROINTESTINAL STROMAL TUMOR; GENE; GENE MUTATION; GENETIC ASSOCIATION; HUMAN; HUMAN CELL; HUMAN TISSUE; IC 50; IN VITRO STUDY; NONHUMAN; PDGRFA D842V GENE; PRIORITY JOURNAL; PROTEIN EXPRESSION;

ANIMALS; ANTINEOPLASTIC AGENTS; BENZIMIDAZOLES; CELL LINE, TUMOR; CHO CELLS; CRICETINAE; DRUG RESISTANCE, NEOPLASM; ENZYME ACTIVATION; GASTROINTESTINAL STROMAL TUMORS; HUMANS; INHIBITORY CONCENTRATION 50; MUTATION; PIPERAZINES; PROTEIN KINASE INHIBITORS; PYRIMIDINES; RECEPTOR, PLATELET-DERIVED GROWTH FACTOR ALPHA;

EID: 84865074356     PISSN: 10780432     EISSN: 15573265     Source Type: Journal    
DOI: 10.1158/1078-0432.CCR-12-0625     Document Type: Article

References (34)

1. Corless CL, Heinrich MC. Molecular pathobiology of gastrointestinal stromal sarcomas. Annu Rev Pathol 2008;3:557-86.
2. Corless CL, Barnett CM, Heinrich MC. Gastrointestinal stromal tumours: origin and molecular oncology. Nat Rev Cancer 2011;11:865-78.
3. Gastrointestinal Stromal Tumor Meta-Analysis Group (MetaGIST). Comparison of two doses of imatinib for the treatment of unresectable or metastatic gastrointestinal stromal tumors: a meta-analysis of 1,640 patients. J Clin Oncol 2010;28:1247-53.
4. Blanke CD, Demetri GD, von MM, Heinrich MC, Eisenberg B, Fletcher JA, et al. Long-term results from a randomized phase II trial of standard-versus higher-dose imatinib mesylate for patients with unresectable or metastatic gastrointestinal stromal tumors expressing KIT. J Clin Oncol 2008;26:620-5.
5. Demetri GD, Van Oosterom AT, Garrett CR, Blackstein ME, Shah MH, Verweij J, et al. Efficacy and safety of sunitinib in patients with advanced gastrointestinal stromal tumour after failure of imatinib: a randomised controlled trial. Lancet 2006;368:1329-38.
6. Heinrich MC, Owzar K, Corless CL, Hollis D, Borden EC, Fletcher CD, et al. Correlation of kinase genotype and clinical outcome in the North American Intergroup Phase III Trial of imatinib mesylate for treatment of advanced gastrointestinal stromal tumor: CALGB 150105 Study by Cancer and Leukemia Group B and Southwest Oncology Group. J Clin Oncol 2008;26:5360-7.
7. Heinrich MC, Maki RG, Corless CL, Antonescu CR, Harlow A, Griffith D, et al. Primary and secondary kinase genotypes correlate with the biological and clinical activity of sunitinib in imatinib-resistant gastrointestinal stromal tumor. J Clin Oncol 2008;26:5352-9.
8. Heinrich MC, Corless CL, Demetri GD, Blanke CD, von Mehren M, Joensuu H, et al. Kinase mutations and imatinib response in patients with metastatic gastrointestinal stromal tumor. J Clin Oncol 2003;21:4342-9.
9. Debiec-Rychter M, Dumez H, Judson I, Wasag B, Verweij J, Brown M, et al. Use of c-KIT/PDGFRA mutational analysis to predict the clinical response to imatinib in patientswith advanced gastrointestinal stromal tumours entered on phase I and II studies of the EORTC Soft Tissue and Bone Sarcoma Group. Eur J Cancer 2004;40:689-95.
10. Debiec-Rychter M, Sciot R, Le CA, Schlemmer M, Hohenberger P, Van Oosterom AT, et al. KIT mutations and dose selection for imatinib in patients with advanced gastrointestinal stromal tumours. Eur J Cancer 2006;42:1093-103.
11. Hirota S, Ohashi A, Nishida T, Isozaki K, Kinoshita K, Shinomura Y, et al. Gain-of-function mutations of platelet-derived growth factor receptor alpha gene in gastrointestinal stromal tumors. Gastroenterology 2003;125:660-7.
12. Debiec-Rychter M, Cools J, Dumez H, Sciot R, Stul M, Mentens N, et al. Mechanisms of resistance to imatinib mesylate in gastrointestinal stromal tumors and activity of the PKC412 inhibitor against imatinib-resistant mutants. Gastroenterology 2005;128:270-9.
13. Corless CL, Schroeder A, Griffith D, Town A, McGreevey L, Harrell P, et al. PDGFRA mutations in gastrointestinal stromal tumors: frequency, spectrum and in vitro sensitivity to imatinib. J Clin Oncol 2005;23:5357-64.
14. Biron P, Cassier PA, Fumagalli E, Blesius M, Debiec-Rychter M, Adenis A, et al. Outcome of patients (pts) with PDGFRA D842V mutant gastrointestinal stromal tumor (GIST) treated with imatinib (IM) for advanced disease. J Clin Oncol 2010;28:15s.
15. Heinrich MC, Corless CL, Duensing A, McGreevey L, Chen CJ, Joseph N, et al. PDGFRA activating mutations in gastrointestinal stromal tumors. Science 2003;299:708-10.
16. Lewis NL, Lewis LD, Eder JP, Reddy NJ, Guo F, Pierce KJ, et al. Phase I study of the safety, tolerability, and pharmacokinetics of oral CP-868,596, a highly specific platelet-derived growth factor receptor tyrosine kinase inhibitor in patients with advanced cancers. J Clin Oncol 2009;27:5262-9.
17. Michael M, Vlahovic G, Khamly K, Pierce KJ, Guo F, Olszanski AJ. Phase Ib study of CP-868,596, a PDGFR inhibitor, combined with docetaxel with or without axitinib, a VEGFR inhibitor. Br J Cancer 2010;103:1554-61.
18. Fabian MA, Biggs WH III, Treiber DK, Atteridge CE, Azimioara MD, Benedetti MG, et al. A small molecule-kinase interaction map for clinical kinase inhibitors. Nat Biotechnol 2005;23:329-36.
19. Karaman MW, Herrgard S, Treiber DK, Gallant P, Atteridge CE, Campbell BT, et al. A quantitative analysis of kinase inhibitor selectivity. Nat Biotechnol 2008;26:127-32.
20. Taguchi T, Sonobe H, Toyonaga S, Yamasaki I, Shuin T, Takano A, et al. Conventional and molecular cytogenetic characterization of a new human cell line, GIST-T1, established from gastrointestinal stromal tumor. Lab Invest 2002;82:663-5.
21. Griffin JH, Leung J, Bruner RJ, Caligiuri MA, Briesewitz R. Discovery of a fusion kinase in EOL-1 cells and idiopathic hypereosinophilic syndrome. Proc Natl Acad Sci U S A 2003;100:7830-5.
22. Ramos AH, Dutt A, Mermel C, Perner S, Cho J, Lafargue CJ, et al. Amplification of chromosomal segment 4q12 in non-small cell lung cancer. Cancer Biol Ther 2009;8:2042-50.
23. Patricelli MP, Nomanbhoy TK, Wu J, Brown H, Zhou D, Zhang J, et al. In situ kinase profiling reveals functionally relevant properties of native kinases. Chem Biol 2011;18:699-710.
24. Lierman E, Michaux L, Beullens E, Pierre P, Marynen P, Cools J, et al. FIP1L1-PDGFRalpha D842V, a novel panresistant mutant, emerging after treatment of FIP1L1-PDGFRalpha T674I eosinophilic leukemia with single agent sorafenib. Leukemia 2009;23: 845-51.
25. Lierman E, Folens C, Stover EH, Mentens N, Van MH, Scheers W, et al. Sorafenib is a potent inhibitor of FIP1L1-PDGFRalpha and the imatinib-resistant FIP1L1-PDGFRalpha T674I mutant. Blood 2006;108:1374-6.
26. LoRusso PM, Eder JP. Therapeutic potential of novel selective-spectrum kinase inhibitors in oncology. Expert Opin Investig Drugs 2008;17:1013-28.
27. Zarghooni M, Bartels U, Lee E, Buczkowicz P, Morrison A, Huang A, et al. Whole-genome profiling of pediatric diffuse intrinsic pontine gliomas highlights platelet-derived growth factor receptor alpha and poly (ADP-ribose) polymerase as potential therapeutic targets. J Clin Oncol 2010;28:1337-44.
28. Martinho O, Longatto-Filho A, Lambros MB, Martins A, Pinheiro C, Silva A, et al. Expression, mutation and copy number analysis of platelet-derived growth factor receptor A (PDGFRA) and its ligand PDGFA in gliomas. Br J Cancer 2009;101:973-82.
29. Dewaele B, Wasag B, Cools J, Sciot R, Prenen H, Vandenberghe P, et al. Activity of dasatinib, a dual SRC/ABL kinase inhibitor, and IPI-504, a heat shock protein 90 inhibitor, against gastrointestinal stromal tumor-associated PDGFRAD842V mutation. Clin Cancer Res 2008;14:5749-58.
30. Zhang J, Yang PL, Gray NS. Targeting cancer with small molecule kinase inhibitors. Nat Rev Cancer 2009;9:28-39.
31. Gajiwala KS, Wu JC, Christensen J, Deshmukh GD, Diehl W, DiNitto JP, et al. KIT kinase mutants show unique mechanisms of drug resistance to imatinib and sunitinib in gastrointestinal stromal tumor patients. Proc Natl Acad Sci U S A 2009;106:1542-7.
32. Foster R, Griffith R, Ferrao P, Ashman L. Molecular basis of the constitutive activity and STI571 resistance of Asp816Val mutant KIT receptor tyrosine kinase. J Mol Graph Model 2004;23:139-52.
33. Appelmann I, Liersch R, Kessler T, Mesters RM, Berdel WE. Angiogenesis inhibition in cancer therapy: platelet-derived growth factor (PDGF) and vascular endothelial growth factor (VEGF) and their receptors: biological functions and role in malignancy. Recent Results Cancer Res 2010;180:51-81.
34. Tefferi A. Molecular drug targets in myeloproliferative neoplasms: mutant ABL1, JAK2, MPL, KIT, PDGFRA, PDGFRB and FGFR1. J Cell Mol Med 2009;13:215-37.