Sequential valproic acid/all-trans retinoic acid treatment reprograms differentiation in refractory and high-risk acute myeloid leukemia

Cancer Research

Volumn 66, Issue 17, 2006, Pages 8903-8911

  Cimino, Giuseppe ^a   Lo Coco, Francesco ^b   Fenu, Susanna ^a   Travaglini, Lorena ^a,c   Finolezzi, Erica ^a   Mancini, Marco ^a   Nanni, Mauro ^a   Careddu, Angela ^a   Fazi, Francesco ^a,c   Padula, Fabrizio ^a   Fiorini, Roberto ^a   Spiriti, Maria Antonietta Aloe ^a   Petti, Maria Concetta ^a   Venditti, Adriano ^b   Amadori, Sergio ^b   Mandelli, Franco ^a   Pelicci, Pier Giuseppe ^d   Nervi, Clara ^a,c  

^a SAPIENZA UNIVERSITY OF ROME   (Italy)

^b UNIVERSITY OF ROME TOR VERGATA   (Italy)

^c San Raffaele Biomedical Park Foundation   (Italy)

^d EUROPEAN INSTITUTE OF ONCOLOGY   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

RETINOIC ACID; VALPROIC ACID;

ACETYLATION; ACUTE GRANULOCYTIC LEUKEMIA; ADULT; AGED; ARTICLE; CELL DIFFERENTIATION; CELL PROLIFERATION; CLINICAL ARTICLE; DRUG BLOOD LEVEL; DRUG EFFECT; FEMALE; FLUORESCENCE IN SITU HYBRIDIZATION; GENE EXPRESSION; GENETIC TRANSCRIPTION; HUMAN; LIVER TOXICITY; MALE; PRIORITY JOURNAL; SIDE EFFECT;

ANTINEOPLASTIC AGENTS; BONE MARROW CELLS; CELL DIFFERENTIATION; ENZYME INHIBITORS; HUMANS; IN SITU HYBRIDIZATION, FLUORESCENCE; KARYOTYPING; LEUKEMIA, MYELOID, ACUTE; TREATMENT OUTCOME; TRETINOIN; VALPROIC ACID;

EID: 33748997409     PISSN: 00085472     EISSN: None     Source Type: Journal    
DOI: 10.1158/0008-5472.CAN-05-2726     Document Type: Article

References (44)

1. Johnstone RW, Licht JD. Histone deacetylase inhibitors in cancer therapy: is transcription the primary target? Cancer Cell 2003;4:13-8.
2. Egger G, Liang G, Aparicio A, Jones PA. Epigenetics in human disease and prospects for epigenetic therapy. Nature 2004;429:457-63.
3. Minucci S, Nervi C, Coco FL, Pelicci PG. Histone deacetylases: a common molecular target for differentiation treatment of acute myeloid leukemias? Oncogene 2001;20:3110-5.
4. Jaenisch R, Bird A. Epigenetic regulation of gene expression: how the genome integrates intrinsic and environmental signals. Nat Genet 2003;33 Suppl:245-54.
5. Grewal SI, Moazed D. Heterochromatin and epigenetic control of gene expression. Science 2003;301:798-802.
6. Grignani F, De Matteis S, Nervi C, et al. Fusion proteins of the retinoic acid receptor-α recruit histone deacetylase in promyelocytic leukaemia. Nature 1998; 391:815-8.
7. Guidez F, Ivins S, Zhu J, Soderstrom M, Waxman S, Zelent A. Reduced retinoic acid-sensitivities of nuclear receptor corepressor binding to PML- and PLZF-RARα underlie molecular pathogenesis and treatment of acute promyelocytic leukemia. Blood 1998;91:2634-42.
8. He LZ, Guidez F, Tribioli C, et al. Distinct interactions of PML-RARα and PLZF-RARα with co-repressors determine differential responses to RA in APL. Nat Genet 1998; 18:126-35.
9. Lin RJ, Nagy L, Inoue S, et al. Role of the histone deacetylase complex in acute promyelocytic leukaemia. Nature 1998;391:811-4.
10. Melnick A, Licht JD. Deconstructing a disease: RARα, its fusion partners, and their roles in the pathogenesis of acute promyelocytic leukemia. Blood 1999;93:3167-215.
11. Lin RJ, Evans RM. Acquisition of oncogenic potential by RAR chimeras in acute promyelocytic leukemia through formation of homodimers. Mol Cell 2000;5: 821-30.
12. Minucci S, Maccarana M, Cioce M, et al. Oligomerization of RAR and AML1 transcription factors as a novel mechanism of oncogenic activation. Mol Cell 2000;5:811-20.
13. Di Croce L, Raker VA, Corsaro M, et al. Methyltransferase recruitment and DNA hypermethylation of target promoters by an oncogenic transcription factor. Science 2002;295:1079-82.
14. Tallman MS, Nabhan C, Feusner JH, Rowe JM. Acute promyelocytic leukemia: evolving therapeutic strategies. Blood 2002;99:759-67.
15. Sanz MA, Tallman MS, Lo-Coco F. Tricks of the trade for the appropriate management of newly diagnosed acute promyelocytic leukemia. Blood 2005;105:3019-25.
16. Tenen DG, Hromas R, Licht JD, Zhang DE. Transcription factors, normal myeloid development, and leukemia. Blood 1997;90:489-519.
17. Zhu J, Heyworth CM, Glasow A, et al. Lineage restriction of the RARα gene expression in myeloid differentiation. Blood 2001;98:2563-7.
18. Glasow A, Prodromou N, Xu K, Von Lindern M, Zelent A. Retinoids and myelomonocytic growth factors cooperatively activate RARA and induce human myeloid leukemia cell differentiation via MAP kinase pathways. Blood 2005;105:341-9.
19. Ferrara FF, Fazi F, Bianchini A, et al. Histone deacetylase targeted treatment restores retinoic acid signaling and differentiation in acute myeloid leukemia. Cancer Res 2001;61:2-7.
20. Gottlicher M, Minucci S, Zhu P, et al. Valproic acid defines a novel class of HDAC inhibitors inducing differentiation of transformed cells. EMBO J 2001;20: 6969-78.
21. Marks PA, Richon VM, Miller T, Kelly WK. Histone deacetylase inhibitors. Adv Cancer Res 2004;91:137-68.
22. Nervi C, Borello U, Fazi F, Buffa V, Pelicci PG, Cossu G. Inhibition of histone deacetylase activity by trichostatin A modulates gene expression during mouse embryogenesis without apparent toxicity. Cancer Res 2001;61:1247-9.
23. Nebbioso A, Clarke N, Voltz E, et al. Tumor-selective action of HDAC inhibitors involves TRAIL induction in acute myeloid leukemia ceus. Nat Med 2005;11:77-84.
24. Insinga A, Monestiroli S, Ronzoni S, et al. Inhibitors of histone deacetylases induce tumor-selective apoptosis through activation of the death receptor pathway. Nat Med 2005;11:71-6.
25. Raffoux E, Chaibi P, Dombret H, Degos L. Valproic acid and all-trans retinoic acid for the treatment of elderly patients with acute myeloid leukemia. Haematologica 2005;90:986-8.
26. Kuendgen A, Strupp C, Aivado M, et al. Treatment of myelodysplastic syndromes with valproic acid alone or in combination with all-trans retinoic acid. Blood 2004; 104:1266-9.
27. Kuendgen A, Knipp S, Fox F, et al. Results of a phase 2 study of valproic acid alone or in combination with all-trans retinoic acid in 75 patients with myelodysplastic syndrome and relapsed or refractory acute myeloid leukemia. Ann Hematol 2005;84 Suppl 13:61-6.
28. Bennett JM, Catovsky D, Daniel MT, et al. Proposed revised criteria for the classification of acute myeloid leukemia. A report of the French-American-British Cooperative Group. Ann Intern Med 1985;103:620-5.
29. Vardiman JW, Harris NL, Brunning RD. The World Health Organization (WHO) classification of the myeloid neoplasms. Blood 2002;100:2292-302.
30. Mitelman F. An international system for human cytogenetic nomenclature. Basel (Switzerland): Karger; 1995.
31. Mancini M, Nanni M, Cedrone M, et al. Combined cytogenetic, FISH and molecular analysis in acute promyelocytic leukemia at diagnosis and in complete remission. Br J Haematol 1995;91:878-84.
32. Cheson BD, Bennett JM, Kopecky KJ, et al. Revised recommendations of the International Working Group for Diagnosis, Standardization of Response Criteria, Treatment Outcomes, and Reporting Standards for Therapeutic Trials in Acute Myeloid Leukemia. J Clin Oncol 2003;21:4642-9.
33. Cheson BD, Bennett JM, Kantarjian H, et al. Report of an International Working Group to standardize response criteria for myelodysplastic syndromes. Blood 2000;96:3671-4.
34. Zschunke F, Salmassi A, Kreipe H, Buck F, Parwaresch MR, Radzun HJ. cDNA cloning and characterization of human monocyte/macrophage serine esterase-1. Blood 1991;78:506-12.
35. Fazi F, Travaglini L, Carotti D, et al. Retinoic acid targets DNA-methyltransferases and histone deacetylases during APL blast differentiation in vitro and in vivo. Oncogene 2005;24:1820-30.
36. Racanicchi S, Maccherani C, Liberatore C, et al. Targeting fusion protein/corepressor contact restores differentiation response in leukemia cells. EMBO J 2005; 24:1232-42.
37. Chambon P. A decade of molecular biology of retinoic acid receptors. FASEB J 1996;10:940-54.
38. Gore SD, Weng LJ, Figg WD, et al. Impact of prolonged infusions of the putative differentiating agent sodium phenylbutyrate on myelodysplastic syndromes and acute myeloid leukemia. Clin Cancer Res 2002;8: 963-70.
39. Whitman SP, Liu S, Vukosavljevic T, et al. The MLL partial tandem duplication: evidence for recessive gain-of-function in acute myeloid leukemia identifies a novel patient subgroup for molecular targeted therapy. Blood 2005;106:345-52.
40. 1 arrest and apoptosis in myeloid leukemia cells: structure-function analysis. Leukemia 1999;13: 1243-53.
41. Warrell RP, Jr., Frankel SR, Miller WH, Jr., et al. Differentiation therapy of acute promyelocytic leukemia with tretinoin (all-trans-retinoic acid). N Engl J Med 1991;324:1385-93.
42. Diverio D, Lo Coco F, D'Adamo F, et al. Identification of DNA rearrangements at the retinoic acid receptor-α (RARα) locus in all patients with acute promyelocytic leukemia (APL) and mapping of APL breakpoints within the RARα second intron. Blood 1992;79:1-5.
43. Castaigne S, Chomienne C, Daniel MT, et al. All-trans retinoic acid as a differentiation therapy for acute promyelocytic leukemia. I. Clinical results. Blood 1990; 76:1704-9.
44. Schlenk RF, Frohling S, Hartmann F, et al. Phase III study of all-trans retinoic acid in previously untreated patients 61 years or older with acute myeloid leukemia. Leukemia 2004;18:1798-803.