Pharmacodynamic markers and clinical results from the phase 2 study of the SMAC mimetic birinapant in women with relapsed platinum-resistant or -refractory epithelial ovarian cancer

Cancer

Volumn 122, Issue 4, 2016, Pages 588-597

  Noonan, Anne M ^a,d   Bunch, Kristen P ^a,b   Chen, Jin Qiu ^a   Herrmann, Michelle A ^a   Lee, Jung Min ^a   Kohn, Elise C ^a   O'Sullivan, Ciara C ^a   Jordan, Elizabeth ^a   Houston, Nicole ^a   Takebe, Naoko ^a   Kinders, Robert J ^c   Cao, Liang ^a   Peer, Cody J ^a   Figg, W Douglas ^a   Annunziata, Christina M ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

^b WALTER REED ARMY MEDICAL CENTER   (United States)

^c NATIONAL CANCER INSTITUTE   (United States)

^d OHIO STATE UNIVERSITY   (United States)

Author keywords

birinapant; inhibitors of apoptosis proteins (IAP); ovarian cancer; pharmacodynamics; second mitochondria derived activator of caspases (SMAC) mimetic

Indexed keywords

BIRINAPANT; CASPASE 3; HISTONE H2AX; INHIBITOR OF APOPTOSIS PROTEIN 1; PLATINUM COMPLEX; PROCASPASE 3; ANTINEOPLASTIC AGENT; BIRC2 PROTEIN, HUMAN; DIABLO PROTEIN, HUMAN; DIPEPTIDE; INDOLE DERIVATIVE; INHIBITOR OF APOPTOSIS PROTEIN; MITOCHONDRIAL PROTEIN; PLATINUM DERIVATIVE; SIGNAL PEPTIDE; UBIQUITIN PROTEIN LIGASE;

ADULT; AGED; ANEMIA; ARTICLE; B LYMPHOCYTE; BACKACHE; CANCER RECURRENCE; CLINICAL ARTICLE; CONSTIPATION; DEHYDRATION; DEPRESSION; DIARRHEA; DOWN REGULATION; DRUG RESPONSE; DRUG TOLERABILITY; ENZYME LOCALIZATION; EYE DISEASE; FACIAL NERVE DISEASE; FATIGUE; FEMALE; HEADACHE; HISTOPATHOLOGY; HUMAN; HUMAN CELL; HUMAN TISSUE; HYPERCALCEMIA; HYPOALBUMINEMIA; INTESTINE OBSTRUCTION; LYMPHOCYTOPENIA; MIDDLE AGED; MULTIPLE CYCLE TREATMENT; NATURAL KILLER CELL; OVARY CARCINOMA; PAIN; PERIPHERAL BLOOD MONONUCLEAR CELL; PHARMACODYNAMICS; PHASE 2 CLINICAL TRIAL; PLEURA EFFUSION; PRIORITY JOURNAL; PROGRESSION FREE SURVIVAL; SIDE EFFECT; SKIN PAIN; T LYMPHOCYTE; THROMBOEMBOLISM; TUMOR BIOPSY; UPPER RESPIRATORY TRACT INFECTION; URINARY TRACT INFECTION; URINE RETENTION; VOMITING; ADENOCARCINOMA, CLEAR CELL; CARCINOMA, ENDOMETRIOID; CHEMICALLY INDUCED; CLINICAL TRIAL; DISEASE FREE SURVIVAL; DRUG RESISTANCE; LYMPHOCYTE COUNT; METABOLISM; MONONUCLEAR CELL; NEOPLASMS, CYSTIC, MUCINOUS, AND SEROUS; NEOPLASMS, GLANDULAR AND EPITHELIAL; OVARIAN NEOPLASMS; TREATMENT FAILURE; TREATMENT OUTCOME;

ADENOCARCINOMA, CLEAR CELL; AGED; ANTINEOPLASTIC AGENTS; B-LYMPHOCYTES; CARCINOMA, ENDOMETRIOID; CASPASE 3; DIPEPTIDES; DISEASE-FREE SURVIVAL; DRUG RESISTANCE, NEOPLASM; FEMALE; HUMANS; INDOLES; INHIBITOR OF APOPTOSIS PROTEINS; INTRACELLULAR SIGNALING PEPTIDES AND PROTEINS; KILLER CELLS, NATURAL; LEUKOCYTES, MONONUCLEAR; LYMPHOCYTE COUNT; LYMPHOPENIA; MIDDLE AGED; MITOCHONDRIAL PROTEINS; NEOPLASMS, CYSTIC, MUCINOUS, AND SEROUS; NEOPLASMS, GLANDULAR AND EPITHELIAL; OVARIAN NEOPLASMS; PLATINUM COMPOUNDS; T-LYMPHOCYTES; TREATMENT FAILURE; TREATMENT OUTCOME; UBIQUITIN-PROTEIN LIGASES;

EID: 84959227515     PISSN: 0008543X     EISSN: 10970142     Source Type: Journal    
DOI: 10.1002/cncr.29783     Document Type: Article

References (35)

1. Howlader N, Noone AM, Krapcho M, et al, eds. SEER Cancer Statistics Review, 1975-2012. Bethesda, MD: National Cancer Institute; 2015.
2. Cain K, Bratton SB, Langlais C, et al., Apaf-1 oligomerizes into biologically active approximately 700-kDa and inactive approximately 1.4-MDa apoptosome complexes. J Biol Chem. 2000; 275: 6067-6070.
3. Liu JR, Opipari AW, Tan L, et al., Dysfunctional apoptosome activation in ovarian cancer: implications for chemoresistance. Cancer Res. 2002; 62: 924-931.
4. Antoon JW, Lai R, Struckhoff AP, et al., Altered death receptor signaling promotes epithelial-to-mesenchymal transition and acquired chemoresistance. Sci Rep. 2012; 2: 539.
5. Deveraux QL, Reed JC,. IAP family proteins-suppressors of apoptosis. Genes Dev. 1999; 13: 239-252.
6. Sui L, Dong Y, Ohno M, Watanabe Y, Sugimoto K, Tokuda M,. Survivin expression and its correlation with cell proliferation and prognosis in epithelial ovarian tumors. Int J Oncol. 2002; 21: 315-320.
7. Du C, Fang M, Li Y, Li L, Wang X,. Smac, a mitochondrial protein that promotes cytochrome c-dependent caspase activation by eliminating IAP inhibition. Cell. 2000; 102: 33-42.
8. Wu G, Chai J, Suber TL, et al., Structural basis of IAP recognition by Smac/DIABLO. Nature. 2000; 408: 1008-1012.
9. Gatti L, De Cesare M, Ciusani E, et al., Antitumor activity of a novel homodimeric SMAC mimetic in ovarian carcinoma. Mol Pharm. 2014; 11: 283-293.
10. Krepler C, Chunduru SK, Halloran MB, et al., The novel SMAC mimetic birinapant exhibits potent activity against human melanoma cells. Clin Cancer Res. 2013; 19: 1784-1794.
11. Scavullo C, Servida F, Lecis D, et al., Single-agent Smac-mimetic compounds induce apoptosis in B chronic lymphocytic leukaemia (B-CLL). Leuk Res. 2013; 37: 809-815.
12. Benetatos CA, Mitsuuchi Y, Burns JM, et al., Birinapant (TL32711), a bivalent SMAC mimetic, targets TRAF2-associated cIAPs, abrogates TNF-induced NF-κB activation, and is active in patient-derived xenograft models. Mol Cancer Ther. 2014; 13: 867-879.
13. Rothe M, Pan MG, Henzel WJ, Ayres TM, Goeddel DV,. The TNFR2-TRAF signaling complex contains two novel proteins related to baculoviral inhibitor of apoptosis proteins. Cell. 1995; 83: 1243-1252.
14. Hernandez L, Hsu SC, Davidson B, Birrer MJ, Kohn EC, Annunziata CM,. Activation of NF-κB signaling by inhibitor of NF-κB kinase β increases aggressiveness of ovarian cancer. Cancer Res. 2010; 70: 4005-4014.
15. De Jaco P, Asselain B, Orlandi C, Fridman WH, Teillaud JL,. Evaluation of circulating tumor necrosis factor-α in patients with gynecological malignancies. Int J Cancer. 1991; 48: 375-378.
16. Piura B, Medina L, Rabinovich A, Dyomin V, Levy RS, Huleihel M,. Distinct expression and localization of TNF system in ovarian carcinoma tissues: possible involvement of TNF-α in morphological changes of ovarian cancerous cells. Anticancer Res. 2014; 34: 745-752.
17. Baker SJ, Reddy EP,. Transducers of life and death: TNF receptor superfamily and associated proteins. Oncogene. 1996; 12: 1-9.
18. Varfolomeev E, Goncharov T, Fedorova AV, et al., c-IAP1 and c-IAP2 are critical mediators of tumor necrosis factor alpha (TNFα)-induced NF-κB activation. J Biol Chem. 2008; 283: 24295-24299.
19. Bertrand MJ, Milutinovic S, Dickson KM, et al., cIAP1 and cIAP2 facilitate cancer cell survival by functioning as E3 ligases that promote RIP1 ubiquitination. Mol Cell. 2008; 30: 689-700.
20. Eisenhauer EA, Therasse P, Bogaerts J, et al., New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer. 2009; 45: 228-247.
21. Chen JQ, Heldman MR, Herrmann MA, et al., Absolute quantitation of endogenous proteins with precision and accuracy using a capillary Western system. Anal Biochem. 2013; 442: 97-103.
22. Chen JQ, Lee JH, Herrmann MA, et al., Capillary isoelectric-focusing immunoassays to study dynamic oncoprotein phosphorylation and drug response to targeted therapies in non-small cell lung cancer. Mol Cancer Ther. 2013; 12: 2601-2613.
23. Rose PG, Tian C, Bookman MA,. Assessment of tumor response as a surrogate endpoint of survival in recurrent/platinum-resistant ovarian carcinoma: a Gynecologic Oncology Group study. Gynecol Oncol. 2010; 117: 324-329.
24. Banerjee A, Tsiatis AA,. Adaptive two-stage designs in phase II clinical trials. Stat Med. 2006; 25: 3382-3395.
25. Amaravadi RK, Schilder RJ, Martin LP, et al., A Phase 1 Study of the SMAC-Mimetic Birinapant in Adults with Refractory Solid Tumors or Lymphoma. Mol Cancer Ther. 2015. In press.
26. Fan AC, Deb-Basu D, Orban MW, et al., Nanofluidic proteomic assay for serial analysis of oncoprotein activation in clinical specimens. Nat Med. 2009; 15: 566-571.
27. Wang CY, Mayo MW, Baldwin AS Jr., TNF- and cancer therapy-induced apoptosis: potentiation by inhibition of NF-κB. Science. 1996; 274: 784-787.
28. Hurwitz HI, Smith DC, Pitot HC, et al., Safety, pharmacokinetics, and pharmacodynamic properties of oral DEBIO1143 (AT-406) in patients with advanced cancer: results of a first-in-man study. Cancer Chemother Pharmacol. 2015; 75: 851-859.
29. Sun H, Nikolovska-Coleska Z, Lu J, et al., Design, synthesis, and characterization of a potent, nonpeptide, cell-permeable, bivalent Smac mimetic that concurrently targets both the BIR2 and BIR3 domains in XIAP. J Am Chem Soc. 2007; 129: 15279-15294.
30. Sun H, Nikolovska-Coleska Z, Yang CY, et al., Design of small-molecule peptidic and nonpeptidic Smac mimetics. Acc Chem Res. 2008; 41: 1264-1277.
31. Frey NV, Luger S, Mangan J, et al., A phase I study using single agent birinapant in patients with relapsed myelodysplastic syndrome and acute myelogenous leukemia. Blood. 2014; 124: 3758.
32. Sikic BI, Eckhardt SG, Gallant G, et al., Safety, pharmacokinetics (PK), and pharmacodynamics (PD) of HGS1029, an inhibitor of apoptosis protein (IAP) inhibitor, in patients (Pts) with advanced solid tumors: results of a phase I study [abstract]. J Clin Oncol. 2012; 29 (suppl): 3008.
33. Eytan DF, Snow GE, Carlson SG, Schiltz S, Chen Z, Van Waes C,. Combination effects of SMAC mimetic birinapant with TNFα, TRAIL, and docetaxel in preclinical models of HNSCC. Laryngoscope. 2015; 125: E118-E124.
34. Fulda S,. Smac mimetics as IAP antagonists. Semin Cell Dev Biol. 2015; 39: 132-138.
35. Tian A, Wilson GS, Lie S, et al., Synergistic effects of IAP inhibitor LCL161 and paclitaxel on hepatocellular carcinoma cells. Cancer Lett. 2014; 351: 232-241.