A phase 1 and pharmacodynamic study of depsipeptide (FK228) in chronic lymphocytic leukemia and acute myeloid leukemia

Blood

Volumn 105, Issue 3, 2005, Pages 959-967

  Byrd, John C ^a   Marcucci, Guido ^b   Parthun, Mark R ^b   Xiao, Jim J ^b   Klisovic, Rebecca B ^b   Moran, Mollie ^b   Lin, Thomas S ^b   Liu, Shujun ^b   Sklenar, Amy R ^b   Davis, Melanie E ^b   Lucas, David M ^b   Fischer, Beth ^b   Shank, Roshini ^b   Tejaswi, Sooraj L ^b   Binkley, Philip ^b   Wright, John ^b   Chan, Kenneth K ^b   Grever, Michael R ^b  

^a OHIO STATE UNIVERSITY   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

DEPSIPEPTIDE; FR 901228; HISTONE DEACETYLASE; HISTONE H3; HISTONE H4; PROTEIN P21; TROPONIN;

ABDOMINAL PAIN; ACETYLATION; ACUTE GRANULOCYTIC LEUKEMIA; ACUTE KIDNEY FAILURE; ADULT; AGED; AGEUSIA; ANEMIA; ANOREXIA; ANTIGEN EXPRESSION; ANTINEOPLASTIC ACTIVITY; APOPTOSIS; AREA UNDER THE CURVE; ARTHRALGIA; ARTICLE; ATAXIA; CARDIOTOXICITY; CHILL; CHRONIC LYMPHATIC LEUKEMIA; CLINICAL ARTICLE; CONSTIPATION; CONTROLLED STUDY; COUGHING; DEHYDRATION; DIAPHORESIS; DIARRHEA; DISEASE COURSE; DIZZINESS; DRUG CLEARANCE; DRUG DOSE REGIMEN; DRUG EFFICACY; DYSPEPSIA; DYSPNEA; EDEMA; ELECTROLYTE DISTURBANCE; EPISTAXIS; FATIGUE; FEMALE; FEVER; HEADACHE; HEART EJECTION FRACTION; HEART LEFT BUNDLE BRANCH BLOCK; HUMAN; HUMAN CELL; HYPERGLYCEMIA; HYPERKALEMIA; HYPERPHOSPHATEMIA; HYPERURICEMIA; HYPOCALCEMIA; HYPONATREMIA; HYPOTENSION; HYPOXIA; ILEUS; INFECTION; INSOMNIA; LEUKOCYTE COUNT; MALE; MOOD DISORDER; MYALGIA; NAUSEA; NEUTROPENIA; PETECHIA; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; PRURITUS; RASH; RIGOR; SINUS TACHYCARDIA; ST SEGMENT DEPRESSION; ST SEGMENT ELEVATION; STOMATITIS; THORAX PAIN; THROMBOCYTOPENIA; TREATMENT OUTCOME; TUMOR CELL DESTRUCTION; TUMOR LYSIS SYNDROME; VOMITING; WEIGHT REDUCTION;

EID: 19944432566     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood-2004-05-1693     Document Type: Article

References (58)

1. Jemal A, Thomas A, Murray T, Thun M. Cancer statistics, 2002. CA Cancer J Clin. 2002;52:23-47.
2. Estey EH. Therapeutic options for acute myelogenous leukemia. Cancer. 2001;92:1059-1073.
3. Mayer RJ, Davis RB, Schiffer CA, et al. Intensive postremission chemotherapy in adults with acute myeloid leukemia. Cancer and Leukemia Group B. N Engl J Med. 1994;331:896-903.
4. Stone RM. The difficult problem of acute myeloid leukemia in the older adult. CA Cancer J Clin. 2002;52:363-371.
5. Byrd JC, Mrozek K, Dodge RK, et al. Pretreatment cytogenetic abnormalities are predictive of induction success, cumulative incidence of relapse, and overall survival in adult patients with de novo acute myeloid leukemia: results from Cancer and Leukemia Group B (CALGB 8461). Blood. 2002;100:4325-4336.
6. Smith SM, Le Beau MM, Huo D, et al. Clinical-cytogenetic associations in 306 patients with therapy-related myelodysplasia and myeloid leukemia: the University of Chicago series. Blood. 2003;102:43-52.
7. Rai KR, Sawitsky A, Cronkite EP, Chanana AD, Levy RN, Pasternack BS. Clinical staging of chronic lymphocytic leukemia. Blood. 1975;46:219-234.
8. Grever MR, Kopecky KJ, Coltman CA, et al. Fludarabine monophosphate: a potentially useful agent in chronic lymphocytic leukemia. Nouv Rev Fr Hematol. 1988;30:457-459.
9. Keating MJ, Kantarjian H, Talpaz M, et al. Fludarabine: a new agent with major activity against chronic lymphocytic leukemia. Blood. 1989;74:19-25.
10. Leporrier M, Chevret S, Cazin B, et al. Randomized comparison of fludarabine, CAP, and ChOP in 938 previously untreated stage B and C chronic lymphocytic leukemia patients. Blood. 2001;98:2319-2325.
11. Johnson S, Smith AG, Loffler H, et al. Multicentre prospective randomised trial of fludarabine versus cyclophosphamide, doxorubicin, and prednisone (CAP) for treatment of advanced-stage chronic lymphocytic leukaemia. The French Cooperative Group on CLL. Lancet. 1996;347:1432-1438.
12. Rai KR, Peterson BL, Appelbaum FR, et al. Fludarabine compared with chlorambucil as primary therapy for chronic lymphocytic leukemia. N Engl J Med. 2000;343:1750-1757.
13. O'Brien SM, Kantarjian H, Thomas DA, et al. Rituximab dose-escalation trial in chronic lymphocytic leukemia. J Clin Oncol. 2001;19:2165-2170.
14. Byrd JC, Peterson BL, Morrison VA, et al. Randomized phase 2 study of fludarabine with concurrent versus sequential treatment with rituximab in symptomatic, untreated patients with B-cell chronic lymphocytic leukemia: results from Cancer and Leukemia Group B 9712 (CALGB 9712). Blood. 2003;101:6-14.
15. Keating MJ, O'Brien S, Lerner S, et al. Combination chemo-antibody therapy with fludarabine (F), cyclophosphamide (C) and rituximab (R) achieves a high CR rate in previously untreated chronic lymphocytic leukemia (CLL)[abstract]. Blood. 2000;96:514a.
16. Byrd JC, Murphy T, Howard RS, et al. Rituximab using a thrice weekly dosing schedule in B-cell chronic lymphocytic leukemia and small lymphocytic lymphoma demonstrates clinical activity and acceptable toxicity. J Clin Oncol. 2001;19:2153-2164.
17. Rush LJ, Dai Z, Smiraglia DJ, et al. Novel methylation targets in de novo acute myeloid leukemia with prevalence of chromosome 11 loci. Blood. 2001;97:3226-3233.
18. Rush LJ, Raval A, Funchain P, et al. Epigenetic profiling in chronic lymphocytic leukemia reveals novel methylation targets. Cancer Res. 2004;64:2424-2433.
19. Guo SX, Taki T, Ohnishi H, et al. Hypermethylation of p16 and p15 genes and RB protein expression in acute leukemia. Leuk Res. 2000;24:39-46.
20. Voso MT, Scardocci A, Guidi F, et al. Aberrant methylation of DAP-kinase in therapy-related acute myeloid leukemia and myelodysplastic syndromes. Blood. 2004;103:698-700.
21. Fulop Z, Csernus B, Timar B, Szepesi A, Matolcsy A. Microsatellite instability and hMLH1 promoter hypermethylation in Richter's transformation of chronic lymphocytic leukemia. Leukemia. 2003;17:411-415.
22. Wang J, Hosnino T, Redner RL, Kajigaya S, Liu JM. ETO, fusion partner in t(8;21) acute myeloid leukemia, represses transcription by interaction with the human N-CoR/mSin3/HDAC1 complex. Proc Natl Acad Sci U S A. 1998;95:10860-10865.
23. 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
24. Moe-Behrens GH, Pandolfi PP. Targeting aberrant transcriptional repression in acute myeloid leukemia. Rev Clin Exp Hematol. 2003;7:139-159.
25. Zhang L, Freitas MA, Wickham J, et al. Differential expression of histone post-translational modifications in acute myeloid and chronic lymphocytic leukemia determined by high-pressure liquid chromatography and mass spectrometry. J Am Soc Mass Spectrom. 2004;15:77-86.
26. Cameron EE, Bachman KE, Myohanen S, Herman JG, Baylin SB. Synergy of demethylation and histone deacetylase inhibition in the re-expression of genes silenced in cancer. Nat Genet. 1999;21:103-107.
27. Zhu WG, Lakshmanan RR, Beal MD, Otterson GA. DNA methyltransferase inhibition enhances apoptosis induced by histone deacetylase inhibitors. Cancer Res. 2001;61:1327-1333.
28. Shaker S, Bernstein M, Momparler LF, Momparler RL. Preclinical evaluation of antineoplastic activity of inhibitors of DNA methylation (5-aza-2′-deoxycytidine) and histone deacetylation (trichostatin A, depsipeptide) in combination against myeloid leukemic cells. Leuk Res. 2003;27:437-444.
29. Klisovic MI, Maghraby EA, Parthun MR, et al. Depsipeptide (FR 901228) promotes histone acetylation, gene transcription, apoptosis and its activity is enhanced by DNA methyltransferase inhibitors in AML1/ETO-positive leukemic cells. Leukemia. 2003;17:350-358.
30. Shigematsu N, Ueda H, Takase S, Tanaka H, Yamamoto K, Tada T. FR901228, a novel antitumor bicyclic depsipeptide produced by Chromobacterium violaceum No. 968, II: Structure determination. J Antibiot (Tokyo). 1994;47:311-314.
31. Nakajima H, Kim YB, Terano H, Yoshida M, Horinouchi S. FR901228, a potent antitumor antibiotic, is a novel histone deacetylase inhibitor. Exp Cell Res. 1998;241:128-133.
32. Kosugi H, Ito M, Yamamoto Y, et al. In vivo effects of a histone deacetylase inhibitor, FK228, on human acute promyelocytic leukemia in NOD/Shiscid/scid mice. Jpn J Cancer Res. 2001;92:529-536.
33. Huang X, Kadonaga JT. Biochemical analysis of transcriptional repression by Drosophila histone deacetylase 1. J Biol Chem. 2001;276:12497-12500.
34. Furumai R, Matsuyama A, Kobashi N, et al. FK228 (depsipeptide) as a natural prodrug that inhibits class I histone deacetylases. Cancer Res. 2002;62:4916-4921.
35. Xiao JJ, Byrd J, Marcucci G, Grever M, Chan KK. Identification of thiols and glutathione conjugates of depsipeptide FK228 (FR901228), a novel histone protein deacetylase inhibitor, in the blood. Rapid Commun Mass Spectrom. 2003;17:757-766.
36. Byrd JC, Shinn C, Ravi R, et al. Depsipeptide (FR901228): a novel therapeutic agent with selective, in vitro activity against human B-cell chronic lymphocytic leukemia cells. Blood. 1999; 94:1401-1408.
37. Aron JL, Parthun MR, Marcucci G, et al. Depsipeptide (FR901228) induces histone acetylation and inhibition of histone deacetylase in chronic lymphocytic leukemia cells concurrent with activation of caspase 8-mediated apoptosis and down-regulation of c-FLIP protein. Blood. 2003; 102:652-658.
38. Klisovic DD, Katz SE, Effron D, et al. Depsipeptide (FR901228) inhibits proliferation and induces apoptosis in primary and metastatic human uveal melanoma cell lines. Invest Ophthalmol Vis Sci. 2003;44:2390-2398.
39. Sandor V, Bakke S, Robey RW, et al. Phase I trial of the histone deacetylase inhibitor, depsipeptide (FR901228, NSC 630176), in patients with refractory neoplasms. Clin Cancer Res. 2002;8:718-728.
40. Marshall JL, Rizvi N, Kauh J, et al. A phase I trial of depsipeptide (FR901228) in patients with advanced cancer. J Exp Ther Oncol. 2002;2:325-332.
41. Piekarz RL, Robey R, Sandor V, et al. Inhibitor of histone deacetylation, depsipeptide (FR901228), in the treatment of peripheral and cutaneous T-cell lymphoma: a case report. Blood. 2001;98: 2865-2868.
42. Harris NL, Jaffe ES, Diebold J, et al. World Health Organization classification of neoplastic diseases of the hematopoietic and lymphoid tissues: report of the Clinical Advisory Committee meeting-Airlie House, Virginia, November 1997. J Clin Oncol. 1999;17:3835-3849.
43. Cheson BD, Bennett JM, Grever MR, et al. National Cancer Institute-sponsored Working Group guidelines for chronic lymphocytic leukemia: revised guidelines for diagnosis and treatment. Blood. 1996;87:4990-4997.
44. Cheson BD, Cassileth PA, Head DR, et al. Report of the National Cancer Institute-sponsored workshop on definitions of diagnosis and response in acute myeloid leukemia. J Clin Oncol. 1990;8: 813-819.
45. Li Z, Chan KK. A subnanogram APILC/MS/MS quantitation method for depsipeptide FR901228 and its preclinical pharmacokinetics. J Pharm Biomed Anal. 2000;22:33-44.
46. Bruner RJ, Marcucci G, Binkley P, et al. A phase I study to determine minimal effective pharmacologic dose of depsipeptide (FR901228) in selected hematologic malignancies [abstract]. Am Soc Clin Oncol. 2002;21:1059.
47. Sandor V, Senderowicz A, Mertins S, et al. p21-dependent g(1) arrest with downregulation of cyclin D1 and upregulation of cyclin E by the histone deacetylase inhibitor FR901228. Br J Cancer. 2000;83:817-825.
48. Kim JS, Lee S, Lee T, Lee YW, Trepel JB. Transcriptional activation of p21 (WAF1/CIP1) by apicidin, a novel histone deacetylase inhibitor. Biochem Biophys Res Commun. 2001;281:866-871.
49. Kobayashi H, Tan EM, Fleming SE. Acetylation of histones associated with the p21WAF1/CIP1 gene by butyrate is not sufficient for p21WAF1/CIP1 gene transcription in human colorectal adenocarcinoma cells. Int J Cancer. 2004;109:207-213.
50. Maeda T, Towatari M, Kosugi H, Saito H. Upregulation of costimulatory/adhesion molecules by histone deacetylase inhibitors in acute myeloid leukemia cells. Blood. 2000;96:3847-3856.
51. Lucas DM, Davis ME, Parthun MR, et al. The histone deacetylase inhibitor MS-275 induces caspase-dependent apoptosis in B-cell chronic lymphocytic leukemia cells. Leukemia. 2004;18: 1207-1214.
52. 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;8:963-970.
53. Gilbert J, Baker SD, Bowling MK, et al. A phase I dose escalation and bioavailability study of oral sodium phenylbutyrate in patients with refractory solid tumor malignancies. Clin Cancer Res. 2001; 7:2292-2300.
54. Prakash S, Foster BJ, Meyer M, et al. Chronic oral administration of CI-994: a phase 1 study. Invest New Drugs. 2001;19:1-11.
55. Hardy JR, Rees EA, Gwilliam B, Ling J, Broadley K, A'Hern R. A phase II study to establish the efficacy and toxicity of sodium valproate in patients with cancer-related neuropathic pain. J Pain Symptom Manage. 2001;21:204-209.
56. Messmann RA, Ullmann CD, Lahusen T, et al. Flavopiridol-related proinflammatory syndrome is associated with induction of interleukin-6. Clin Cancer Res. 2003;9:562-570.
57. Milhem M, Mahmud N, Lavelle D, et al. Modification of hematopoietic stem cell fate by 5-aza 2′deoxycytidine and trichostatin A. Blood. 2004; 103:4102-4110.
58. Piekarz RL, Robey RW, Zhan Z, et al. T-cell lymphoma as a model for the use of histone deacetylase inhibitors in cancer therapy: impact of depsipeptide on molecular markers, therapeutic targets, and mechanisms of resistance. Blood. 2004;103:4636-4643. AQ0: Right running head OK as supplied? If not, please edit to no longer than 50 characters, including spaces.