XIAP antisense therapy with AEG 35156 in acute myeloid leukemia

Expert Opinion on Investigational Drugs

Volumn 22, Issue 5, 2013, Pages 663-670

  Katragadda, Lakshmikanth ^a   Carter, Bing Z ^a   Borthakur, Gautam ^a  

^a UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

Author keywords

AEG 35156; AML; Antisense oligonucleotide; XIAP

Indexed keywords

AEG 35156; CYTARABINE; IDARUBICIN;

ABDOMINAL PAIN; ACUTE GRANULOCYTIC LEUKEMIA; BREAST CANCER; CANCER COMBINATION CHEMOTHERAPY; CENTRAL NERVOUS SYSTEM LEUKEMIA; CONTINUOUS INFUSION; DIARRHEA; DRUG ACCUMULATION; DRUG BLOOD LEVEL; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG ELIMINATION; DRUG HALF LIFE; DRUG MANUFACTURE; DRUG MEGADOSE; DRUG METABOLISM; DRUG SAFETY; DRUG TOLERABILITY; FATIGUE; HUMAN; HYPOPHOSPHATEMIA; LUNG EMBOLISM; LUNG NON SMALL CELL CANCER; LYMPHOCYTOPENIA; NAUSEA; NONHODGKIN LYMPHOMA; NONHUMAN; PANCREAS CANCER; PERIPHERAL NEUROPATHY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); REMISSION; REVIEW; SIDE EFFECT; THROMBOCYTOPENIA; VOMITING;

ANIMALS; ANTINEOPLASTIC AGENTS; HUMANS; LEUKEMIA, MYELOID, ACUTE; OLIGONUCLEOTIDES; OLIGONUCLEOTIDES, ANTISENSE; X-LINKED INHIBITOR OF APOPTOSIS PROTEIN;

EID: 84876207847     PISSN: 13543784     EISSN: 17447658     Source Type: Journal    
DOI: 10.1517/13543784.2013.789498     Document Type: Review

References (61)

1. Wright ME, Han DK, Hockenbery DM. Caspase-3 and inhibitor of apoptosis protein(s) interactions in Saccharomyces cerevisiae and mammalian cells. FEBS Lett 2000;481(1):13-18
2. Eckelman BP, Salvesen GS, Scott FL. Human inhibitor of apoptosis proteins: why XIAP is the black sheep of the family. EMBO Rep 2006;7(10):988-94
3. Salvesen GS, Duckett CS. IAP proteins: blocking the road to death's door. Nat Rev Mol Cell Biol 2002;3(6):401-10
4. Notarbartolo M, Cervello M, Poma P, et al. Expression of the IAPs in multidrug resistant tumor cells. Oncol Rep 2004;11(1):133-6
5. Nakagawa Y, Hasegawa M, Kurata M, et al. Expression of IAP-family proteins in adult acute mixed lineage leukemia (AMLL). Am J Hematol 2005;78(3):173-80
6. Tamm I, Kornblau SM, Segall H, et al. Expression and prognostic significance of IAP-family genes in human cancers and myeloid leukemias. Clin Cancer Res 2000;6(5):1796-803
7. Tamm I, Richter S, Oltersdorf D, et al. High expression levels of x-linked inhibitor of apoptosis protein and survivin correlate with poor overall survival in childhood de novo acute myeloid leukemia. Clin Cancer Res 2004;10(11):3737-44
8. Yamamoto K, Abe S, Nakagawa Y, et al. Expression of IAP family proteins in myelodysplastic syndromes transforming to overt leukemia. Leuk Res 2004;28(11):1203-11
9. Sung KW, Choi J, Hwang YK, et al. Overexpression of X-linked inhibitor of apoptosis protein (XIAP) is an independent unfavorable prognostic factor in childhood de novo acute myeloid leukemia. J Korean Med Sci 2009;24(4):605-13
10. Holcik M, Yeh C, Korneluk RG, Chow T. Translational upregulation of X-linked inhibitor of apoptosis (XIAP) increases resistance to radiation induced cell death. Oncogene 2000;19(36):4174-7
11. Li Y, Jian Z, Xia K, et al. XIAP is related to the chemoresistance and inhibited its expression by RNA interference sensitize pancreatic carcinoma cells to chemotherapeutics. Pancreas 2006;32(3):288-96
12. Kaufmann SH, Vaux DL. Alterations in the apoptotic machinery and their potential role in anticancer drug resistance. Oncogene 2003;22(47):7414-30
13. Carter BZ, Kornblau SM, Tsao T, et al. Caspase-independent cell death in AML: caspase inhibition in vitro with pan-caspase inhibitors or in vivo by XIAP or Survivin does not affect cell survival or prognosis. Blood 2003;102(12):4179-86
14. Ferreira CG, van der Valk P, Span SW, et al. Expression of X-linked inhibitor of apoptosis as a novel prognostic marker in radically resected non-small cell lung cancer patients. Clin Cancer Res 2001;7(8):2468-74
15. Liu SS, Tsang BK, Cheung AN, et al. Anti-apoptotic proteins, apoptotic and proliferative parameters and their prognostic significance in cervical carcinoma. Eur J Cancer 2001;37(9):1104-10
16. Hussain AR, Uddin S, Ahmed M, et al. Prognostic significance of XIAP expression in DLBCL and effect of its inhibition on AKT signalling. J Pathol 2010;222(2):180-90
17. Mizutani Y, Nakanishi H, Li YN, et al. Overexpression of XIAP expression in renal cell carcinoma predicts a worse prognosis. Int J Oncol 2007;30(4):919-25
18. Xiang G, Wen X, Wang H, et al. Expression of X-linked inhibitor of apoptosis protein in human colorectal cancer and its correlation with prognosis. J Surg Oncol 2009;100(8):708-12
19. Schimmer AD, Dalili S, Batey RA, Riedl SJ. Targeting XIAP for the treatment of malignancy. Cell Death Differ 2006;13(2):179-88
20. Lacasse EC. Pulling the plug on a cancer cell by eliminating XIAP with AEG35156. Cancer Lett 2012; Epub ahead of print
21. Smolewski P, Robak T. Inhibitors of apoptosis proteins (IAPs) as potential molecular targets for therapy of hematological malignancies. Curr Mol Med 2011;11(8):633-49
22. Cheung HH, Lynn Kelly N, Liston P, Korneluk RG. Involvement of caspase-2 and caspase-9 in endoplasmic reticulum stress-induced apoptosis: a role for the IAPs. Exp Cell Res 2006;312(12):2347-57
23. Lu M, Lin SC, Huang Y, et al. XIAP induces NF-kappaB activation via the BIR1/TAB1 interaction and BIR1 dimerization. Mol Cell 2007;26(5):689-702
24. Dohi T, Okada K, Xia F, et al. An IAP-IAP complex inhibits apoptosis. J Biol Chem 2004;279(33):34087-90
25. Du C, Fang M, Li Y, et al. Smac, a mitochondrial protein that promotes cytochrome c-dependent caspase activation by eliminating IAP inhibition. Cell 2000;102(1):33-42
26. Hegde R, Srinivasula SM, Datta P, et al. The polypeptide chain-releasing factor GSPT1/eRF3 is proteolytically processed into an IAP-binding protein. J Biol Chem 2003;278(40):38699-706
27. Suzuki Y, Imai Y, Nakayama H, et al. A serine protease, HtrA2, is released from the mitochondria and interacts with XIAP, inducing cell death. Mol Cell 2001;8(3):613-21
28. Verhagen AM, Ekert PG, Pakusch M, et al. Identification of DIABLO, a mammalian protein that promotes apoptosis by binding to and antagonizing IAP proteins. Cell 2000;102(1):43-53
29. Lacasse EC, Kandimalla ER, Winocour P, et al. Application of XIAP antisense to cancer and other proliferative disorders: development of AEG35156/GEM640. Ann N Y Acad Sci 2005;1058:215-34
30. Schimmer AD, Welsh K, Pinilla C, et al. Small-molecule antagonists of apoptosis suppressor XIAP exhibit broad antitumor activity. Cancer Cell 2004;5(1):25-35
31. Sasaki H, Sheng Y, Kotsuji F, Tsang BK. Down-regulation of X-linked inhibitor of apoptosis protein induces apoptosis in chemoresistant human ovarian cancer cells. Cancer Res 2000;60(20):5659-66
32. Amantana A, London CA, Iversen PL, Devi GR. X-linked inhibitor of apoptosis protein inhibition induces apoptosis and enhances chemotherapy sensitivity in human prostate cancer cells. Mol Cancer Ther 2004;3(6):699-707
33. Carter BZ, Milella M, Tsao T, et al. Regulation and targeting of antiapoptotic XIAP in acute myeloid leukemia. Leukemia 2003;17(11):2081-9
34. Hu Y, Cherton-Horvat G, Dragowska V, et al. Antisense oligonucleotides targeting XIAP induce apoptosis and enhance chemotherapeutic activity against human lung cancer cells in vitro and in vivo. Clin Cancer Res 2003;9(7):2826-36
35. Carter BZ, Gronda M, Wang Z, et al. Small-molecule XIAP inhibitors derepress downstream effector caspases and induce apoptosis of acute myeloid leukemia cells. Blood 2005;105(10):4043-50
36. Fakler M, Loeder S, Vogler M, et al. Small molecule XIAP inhibitors cooperate with TRAIL to induce apoptosis in childhood acute leukemia cells and overcome Bcl-2-mediated resistance. Blood 2009;113(8):1710-22
37. Loeder S, Drensek A, Jeremias I, et al. Small molecule XIAP inhibitors sensitize childhood acute leukemia cells for CD95-induced apoptosis. Int J Cancer 2010;126(9):2216-28
38. Jones D. The long march of antisense. Nat Rev Drug Discov 2011;10(6):401-2
39. Agrawal S, Jiang Z, Zhao Q, et al. Mixed-backbone oligonucleotides as second generation antisense oligonucleotides: in vitro and in vivo studies. Proc Nat Acad Sci USA 1997;94(6):2620-5
40. Farman CA, Kornbrust DJ. Oligodeoxynucleotide studies in primates: antisense and immune stimulatory indications. Toxicol Pathol 2003;31(Suppl):119-22
41. LaCasse EC, Cherton-Horvat GG, Hewitt KE, et al. Preclinical characterization of AEG35156/GEM 640, a second-generation antisense oligonucleotide targeting X-linked inhibitor of apoptosis. Clin Cancer Res 2006;12(17):5231-41
42. Dean E, Jodrell D, Connolly K, et al. Phase I trial of AEG35156 administered as a 7-day and 3-day continuous intravenous infusion in patients with advanced refractory cancer. J Clin Oncol 2009;27(10):1660-6
43. Schimmer AD, Estey EH, Borthakur G, et al. Phase I/II trial of AEG35156 X-linked inhibitor of apoptosis protein antisense oligonucleotide combined with idarubicin and cytarabine in patients with relapsed or primary refractory acute myeloid leukemia. J Clin Oncol 2009;27(28):4741-6
44. Carter BZ, Mak DH, Morris SJ, et al. XIAP antisense oligonucleotide (AEG35156) achieves target knockdown and induces apoptosis preferentially in CD34+38-cells in a phase 1/2 study of patients with relapsed/refractory AML. Apoptosis 2011;16(1):67-74
45. Shaw TJ, Lacasse EC, Durkin JP, Vanderhyden BC. Downregulation of XIAP expression in ovarian cancer cells induces cell death in vitro and in vivo. Int J Cancer J 2008;122(6):1430-4
46. McManus DC, Lefebvre CA, Cherton-Horvat G, et al. Loss of XIAP protein expression by RNAi and antisense approaches sensitizes cancer cells to functionally diverse chemotherapeutics. Oncogene 2004;23(49):8105-17
47. Holt SV, Brookes KE, Dive C, Makin GW. Down-regulation of XIAP by AEG35156 in paediatric tumour cells induces apoptosis and sensitises cells to cytotoxic agents. Oncol Rep 2011;25(4):1177-81
48. Schimmer AD, Herr W, Hanel M, et al. Addition of AEG35156 XIAP antisense oligonucleotide in reinduction chemotherapy does not improve remission rates in patients with primary refractory acute myeloid leukemia in a randomized phase II study. Clin Lymphoma Myeloma Leuk 2011;11(5):433-8
49. Mahadevan D, Chalasani P, Rensvold D, et al. Phase I Trial of AEG35156 an Antisense Oligonucleotide to XIAP Plus Gemcitabine in Patients With Metastatic Pancreatic Ductal Adenocarcinoma. Am J Clin Oncol 2012; Epub ahead of print
50. Brandwein JM, Gupta V, Schuh AC, et al. Predictors of response to reinduction chemotherapy for patients with acute myeloid leukemia who do not achieve complete remission with frontline induction chemotherapy. Am J Hematol 2008;83(1):54-8
51. Hiddemann W, Kreutzmann H, Straif K, et al. High-dose cytosine arabinoside and mitoxantrone: a highly effective regimen in refractory acute myeloid leukemia. Blood 1987;69(3):744-9
52. Larson SM, Campbell NP, Huo D, et al. High dose cytarabine and mitoxantrone: an effective induction regimen for high-risk acute myeloid leukemia (AML). Leukemia Lymphoma 2012;53(3):445-50
53. Harlin H, Reffey SB, Duckett CS, et al. Characterization of XIAP-deficient mice. Mol Cell Biol 2001;21(10):3604-8
54. Lee AS, Zee BC, Lai M, et al. Randomized phase II study of the x-linked inhibitor of apoptosis (XIAP) antisense AEG35156 in combination with sorafenib in patients with advanced hepatocellular carcinoma. J Clin Oncol 2012;30(Suppl):abstract 4105
55. Carter BZ, Mak DH, Schober WD, et al. Simultaneous activation of p53 and inhibition of XIAP enhance the activation of apoptosis signaling pathways in AML. Blood 2010;115(2):306-14
56. Ricotta DN, Frishman W. Mipomersen: a safe and effective antisense therapy adjunct to statins in patients with hypercholesterolemia. Cardiol Rev 2012;20(2):90-5
57. Servida F, Lecis D, Scavullo C, et al. Novel second mitochondria-derived activator of caspases (Smac) mimetic compounds sensitize human leukemic cell lines to conventional chemotherapeutic drug-induced and death receptor-mediated apoptosis. Invest New Drugs 2011;29(6):1264-75
58. Chen DJ, Huerta S. Smac mimetics as new cancer therapeutics. Anticancer Drugs 2009;20(8):646-58
59. Weisberg E, Kung AL, Wright RD, et al. Potentiation of antileukemic therapies by Smac mimetic, LBW242: effects on mutant FLT3-expressing cells. Mol Cancer Ther 2007;6(7):1951-61
60. Fulda S, Vucic D. Targeting IAP proteins for therapeutic intervention in cancer. Nat Rev Drug Discov 2012;11(2):109-24
61. Bai L, Chen W, Wang X, et al. IKKbeta-mediated nuclear factor-kappaB activation attenuates smac mimetic-induced apoptosis in cancer cells. Mol Cancer Ther 2009;8(6):1636-45