Identification of benzodiazepine Ro5-3335 as an inhibitor of CBF leukemia through quantitative high throughput screen against RUNX1-CBFβ interaction

Proceedings of the National Academy of Sciences of the United States of America

Volumn 109, Issue 36, 2012, Pages 14592-14597

  Cunningham, Lea ^a   Finckbeiner, Steven ^a   Hyde, R Katherine ^a   Southall, Noel ^b   Marugan, Juan ^b   Yedavalli, Venkat R K ^c   Dehdashti, SeameenJean ^b   Reinhold, William C ^d   Alemu, Lemlem ^a   Zhao, Ling ^a   Yeh, Jing RueyJoanna ^e   Sood, Raman ^a,f   Pommier, Yves ^d   Austin, Christopher P ^b   Jeang, Kuan Teh ^c   Zheng, Wei ^b   Liu, Paul ^a  

^a NATIONAL INSTITUTES OF HEALTH   (United States)

^b NATIONAL INSTITUTES OF HEALTH   (United States)

^c NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^d NATIONAL CANCER INSTITUTE   (United States)

^e MASSACHUSETTS GENERAL HOSPITAL   (United States)

^f NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

7 CHLORO 1,3 DIHYDRO 5 (2 PYRROLYL) 2H 1,4 BENZODIAZEPIN 2 ONE; [7 CHLORO 5 (1H PYRROL 2 YL) 3H BENZO[E][1,4]DIAZEPIN 2 YL]METHYLAMINE; CORE BINDING FACTOR BETA; CYTARABINE; TRANSCRIPTION FACTOR RUNX1;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CBF LEUKEMIA; CELL VIABILITY; CONTROLLED STUDY; DRUG BLOOD LEVEL; EMBRYO; FLUORESCENCE RESONANCE ENERGY TRANSFER; HEMATOPOIESIS; HIGH THROUGHPUT SCREENING; HUMAN; HUMAN CELL; LEUKEMIA; LEUKEMIA CELL LINE; MOUSE; NONHUMAN; PHENOTYPE; PRELEUKEMIA; PRIORITY JOURNAL; PROTEIN PROTEIN INTERACTION; REPORTER GENE; TRANSACTIVATION; TRANSCRIPTION REGULATION; ZEBRA FISH;

AMINO ACID SEQUENCE; ANIMALS; BENZODIAZEPINES; BLOTTING, WESTERN; CORE BINDING FACTOR ALPHA 2 SUBUNIT; CORE BINDING FACTOR BETA SUBUNIT; ELECTROPHORETIC MOBILITY SHIFT ASSAY; FLOW CYTOMETRY; FLUORESCENCE RESONANCE ENERGY TRANSFER; GENETIC VECTORS; HEMATOPOIESIS; HIGH-THROUGHPUT SCREENING ASSAYS; HISTOLOGICAL TECHNIQUES; HUMANS; IMMUNOPRECIPITATION; JURKAT CELLS; LEUKEMIA, MYELOID, ACUTE; MICE; MOLECULAR SEQUENCE DATA; PRECURSOR CELL LYMPHOBLASTIC LEUKEMIA-LYMPHOMA; PROTEIN INTERACTION MAPPING; SURFACE PLASMON RESONANCE; TRANSCRIPTIONAL ACTIVATION; ZEBRAFISH;

DANIO RERIO;

EID: 84865960664     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1200037109     Document Type: Article

References (41)

1. Ito Y (2008) RUNX genes in development and cancer: Regulation of viral gene expression and the discovery of RUNX family genes. Adv Cancer Res 99:33-76.
2. Speck NA, Gilliland DG (2002) Core-binding factors in haematopoiesis and leukaemia. Nat Rev Cancer 2:502-513. (Pubitemid 37328932)
3. Liu P, et al. (1993) Fusion between transcription factor CBF beta/PEBP2 beta and a myosin heavy chain in acute myeloid leukemia. Science 261:1041-1044. (Pubitemid 23300863)
4. Liu PP, Hajra A, Wijmenga C, Collins FS (1995) Molecular pathogenesis of the chromosome 16 inversion in the M4Eo subtype of acute myeloid leukemia. Blood 85:2289-2302.
5. Erickson P, et al. (1992) Identification of breakpoints in t(8;21) acute myelogenous leukemia and isolation of a fusion transcript, AML1/ETO, with similarity to Drosophila segmentation gene, runt. Blood 80:1825-1831.
6. Golub TR, et al. (1995) Fusion of the TEL gene on 12p13 to the AML1 gene on 21q22 in acute lymphoblastic leukemia. Proc Natl Acad Sci USA 92:4917-4921.
7. Look AT (1997) Oncogenic transcription factors in the human acute leukemias. Science 278:1059-1064.
8. Loh ML, et al. (2006) Prospective analysis of TEL/AML1-positive patients treated on Dana-Farber Cancer Institute Consortium Protocol 95-01. Blood 107:4508-4513. (Pubitemid 43801379)
9. Appelbaum FR, et al. (2006) The clinical spectrum of adult acute myeloid leukaemia associated with core binding factor translocations. Br J Haematol 135:165-173. (Pubitemid 44401613)
10. von Neuhoff C, et al. (2010) Prognostic impact of specific chromosomal aberrations in a large group of pediatric patients with acute myeloid leukemia treated uniformly according to trial AML-BFM 98. J Clin Oncol 28:2682-2689.
11. Roudaia L, et al. (2009) CBFbeta is critical for AML1-ETO and TEL-AML1 activity. Blood 113:3070-3079.
12. Shigesada K, van de Sluis B, Liu PP (2004) Mechanism of leukemogenesis by the inv (16) chimeric gene CBFB/PEBP2B-MHY11. Oncogene 23:4297-4307. (Pubitemid 38758595)
13. Hyde RK, Liu PP (2010) RUNX1 repression-independent mechanisms of leukemogenesis by fusion genes CBFB-MYH11 and AML1-ETO (RUNX1-RUNX1T1). J Cell Biochem 110:1039-1045.
14. Eglen RM, et al. (2008) The use of AlphaScreen technology in HTS: Current status. Curr Chem Genomics 1:2-10.
15. Degorce F, et al. (2009) HTRF: A technology tailored for drug discovery - a review of theoretical aspects and recent applications. Curr Chem Genomics 3:22-32.
16. Okuda T, van Deursen J, Hiebert SW, Grosveld G, Downing JR (1996) AML1, the target of multiple chromosomal translocations in human leukemia, is essential for normal fetal liver hematopoiesis. Cell 84:321-330. (Pubitemid 26042836)
17. Wang Q, et al. (1996) The CBFbeta subunit is essential for CBFalpha2 (AML1) function in vivo. Cell 87:697-708. (Pubitemid 26386943)
18. Sood R, et al. (2010) Development of multilineage adult hematopoiesis in the zebrafish with a runx1 truncation mutation. Blood 115:2806-2809.
19. Tang YY, et al. (2000) Energetic and functional contribution of residues in the core binding factor beta (CBFbeta) subunit to heterodimerization with CBFalpha. J Biol Chem 275:39579-39588.
20. Lin HF, et al. (2005) Analysis of thrombocyte development in CD41-GFP transgenic zebrafish. Blood 106:3803-3810.
21. Langenau DM, et al. (2004) In vivo tracking of T cell development, ablation, and engraftment in transgenic zebrafish. Proc Natl Acad Sci USA 101:7369-7374. (Pubitemid 38638039)
22. Kira T, Hashimoto K, Baba M, Okamoto T, Shigeta S (1996) 2-Glycineamide-5-chlorophenyl 2-pyrryl ketone, a non-benzodiazepin Tat antagonist, is effective against acute and chronic HIV-1 infections in vitro. Antiviral Res 32:55-62. (Pubitemid 26321141)
23. Hsu MC, et al. (1991) Inhibition of HIV replication in acute and chronic infections in vitro by a Tat antagonist. Science 254:1799-1802.
24. Haubrich RH, et al. (1995) A randomized trial of the activity and safety of Ro 24-7429 (Tat antagonist) versus nucleoside for human immunodeficiency virus infection. The AIDS Clinical Trials Group 213 Team. J Infect Dis 172:1246-1252.
25. Jin H, et al. (2009) Definitive hematopoietic stem/progenitor cells manifest distinct differentiation output in the zebrafish VDA and PBI. Development 136:647-654.
26. Rhoades KL, et al. (1996) Synergistic up-regulation of the myeloid-specific promoter for the macrophage colony-stimulating factor receptor by AML1 and the t(8;21) fusion protein may contribute to leukemogenesis. Proc Natl Acad Sci USA 93:11895-11900. (Pubitemid 26347220)
27. Kamikubo Y, et al. (2010) Accelerated leukemogenesis by truncated CBF beta-SMMHC defective in high-affinity binding with RUNX1. Cancer Cell 17:455-468.
28. Holbeck SL, Collins JM, Doroshow JH (2010) Analysis of Food and Drug Administration-approved anticancer agents in the NCI60 panel of human tumor cell lines. Mol Cancer Ther 9:1451-1460.
29. Reinhold WC, et al. (2010) Exon array analyses across the NCI-60 reveal potential regulation of TOP1 by transcription pausing at guanosine quartets in the first intron. Cancer Res 70:2191-2203.
30. Zoppoli G, et al. (2011) Coordinated regulation of mitochondrial topoisomerase IB with mitochondrial nuclear encoded genes and MYC. Nucleic Acids Res 39:6620-6632.
31. Yeh JR, et al. (2008) AML1-ETO reprograms hematopoietic cell fate by downregulating scl expression. Development 135:401-410.
32. Castilla LH, et al. (1996) Failure of embryonic hematopoiesis and lethal hemorrhages in mouse embryos heterozygous for a knocked-in leukemia gene CBFB-MYH11. Cell 87:687-696. (Pubitemid 26386942)
33. Castilla LH, et al. (1999) The fusion gene Cbfb-MYH11 blocks myeloid differentiation and predisposes mice to acute myelomonocytic leukaemia. Nat Genet 23:144-146. (Pubitemid 29455384)
34. Kuo YH, et al. (2006) Cbf beta-SMMHC induces distinct abnormal myeloid progenitors able to develop acute myeloid leukemia. Cancer Cell 9:57-68.
35. Shahabuddin M, Volsky B, Hsu MC, Volsky DJ (1992) Restoration of cell surface CD4 expression in human immunodeficiency virus type 1-infected cells by treatment with a Tat antagonist. J Virol 66:6802-6805.
36. Jäger S, et al. (2012) Vif hijacks CBF-β to degrade APOBEC3G and promote HIV-1 infection. Nature 481:371-375.
37. Zhang W, Du J, Evans SL, Yu Y, Yu XF (2012) T-cell differentiation factor CBF-β regulates HIV-1 Vif-mediated evasion of host restriction. Nature 481:376-379.
38. Yanagisawa K, Horiuchi T, Fujita S (1991) Establishment and characterization of a new human leukemia cell line derived from M4E0. Blood 78:451-457.
39. Yedavalli VS, Jeang KT (2011) Matrin 3 is a co-factor for HIV-1 Rev in regulating post-transcriptional viral gene expression. Retrovirology 8:61.
40. Gmeiner WH, Reinhold WC, Pommier Y (2010) Genome-wide mRNA and microRNA profiling of the NCI 60 cell-line screen and comparison of FdUMP[10] with fluorouracil, floxuridine, and topoisomerase 1 poisons. Mol Cancer Ther 9:3105-3114.
41. Zuber J, et al. (2009) Mouse models of human AML accurately predict chemotherapy response. Genes Dev 23:877-889.