A phase I trial of vorinostat and bortezomib in children with refractory or recurrent solid tumors: A Children's Oncology Group phase I consortium study (ADVL0916)

Pediatric Blood and Cancer

Volumn 60, Issue 3, 2013, Pages 390-395

  Muscal, Jodi A ^a   Thompson, Patrick A ^a   Horton, Terzah M ^a   Ingle, Ashish M ^b   Ahern, Charlotte H ^a   McGovern, Renee M ^c   Reid, Joel M ^c   Ames, Matthew M ^c   Espinoza Delgado, Igor ^d   Weigel, Brenda J ^e   Blaney, Susan M ^a  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

^b CHILDREN S ONCOLOGY GROUP   (United States)

^c MAYO CLINIC   (United States)

^d NATIONAL CANCER INSTITUTE   (United States)

^e UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

Author keywords

Bortezomib; Children's Oncology Group; Pediatric cancer; Phase I trial; Solid tumors; Vorinostat

Indexed keywords

ANALGESIC AGENT; BORTEZOMIB; IMMUNOGLOBULIN ENHANCER BINDING PROTEIN; VORINOSTAT;

ABDOMINAL PAIN; ADOLESCENT; ADULT; ANALGESIA; ANEMIA; ANOREXIA; ARTICLE; BACKACHE; CANCER COMBINATION CHEMOTHERAPY; CHILD; CLINICAL ARTICLE; COUGHING; DEHYDRATION; DIARRHEA; DRUG DISPOSITION; DRUG DISTRIBUTION; DRUG DOSE ESCALATION; DRUG ELIMINATION; DRUG FEVER; DRUG INDUCED HEADACHE; DRUG WITHDRAWAL; DYSPNEA; FATIGUE; FEMALE; HUMAN; INFANT; LEUKOPENIA; LYMPHOCYTOPENIA; MALE; MAXIMUM TOLERATED DOSE; MULTIPLE CYCLE TREATMENT; NAUSEA; NEUTROPENIA; PERIPHERAL BLOOD MONONUCLEAR CELL; PHASE 1 CLINICAL TRIAL; PRESCHOOL CHILD; PRIORITY JOURNAL; PSYCHOSIS; SCHOOL CHILD; SENSORY NEUROPATHY; SOLID TUMOR; THROMBOCYTOPENIA; TINNITUS; TREATMENT RESPONSE; TUMOR RECURRENCE; VOMITING;

ADOLESCENT; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; BORONIC ACIDS; CHILD; CHILD, PRESCHOOL; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; HUMANS; HYDROXAMIC ACIDS; INFANT; MALE; MAXIMUM TOLERATED DOSE; NEOPLASMS; PYRAZINES; YOUNG ADULT;

EID: 84872470330     PISSN: 15455009     EISSN: 15455017     Source Type: Journal    
DOI: 10.1002/pbc.24271     Document Type: Article

References (43)

1. Adams J, Palombella VJ, Sausville EA, et al. Proteasome inhibitors: A novel class of potent and effective antitumor agents. Cancer Res 1999; 59: 2615-2622.
2. Jackson G, Einsele H, Moreau P, et al. Bortezomib, a novel proteasome inhibitor, in the treatment of hematologic malignancies. Cancer Treat Rev 2005; 31: 591-602.
3. Zavrski I, Kleeberg L, Kaiser M, et al. Proteasome as an emerging therapeutic target in cancer. Curr Pharm Des 2007; 13: 471-485.
4. Nawrocki ST, Carew JS, Dunner K, Jr., et al. Bortezomib inhibits PKR-like endoplasmic reticulum (ER) kinase and induces apoptosis via ER stress in human pancreatic cancer cells. Cancer Res 2005; 65: 11510-11519.
5. Houghton PJ, Morton CL, Kolb EA, et al. Initial testing (stage 1) of the proteasome inhibitor bortezomib by the Pediatric Preclinical Testing Program. Pediatr Blood Cancer 2008; 50: 37-45.
6. Blaney SM, Bernstein M, Neville K, et al. Phase I study of the proteasome inhibitor bortezomib in pediatric patients with refractory solid tumors: A Children's Oncology Group Study (ADVL 0015). J Clin Oncol 2004; 22: 4804-4809.
7. Horton TM, Pati D, Plon SE, et al. A phase 1 study of the proteasome inhibitor bortezomib in pediatric patients with refractory leukemia: A Children's Oncology Group Study. Clin Cancer Res 2007; 13: 1516-1522.
8. Marks P, Rifkind RA, Richon VM, et al. Histone deacetylases and cancer: Causes and therapies. Nat Rev Cancer 2001; 1: 194-202.
9. Marks PA, Richon VM, Miller T, et al. Histone deacetylase inhibitors. Adv Cancer Res 2004; 91: 137-168.
10. Bachmann PS, Piazza RG, Janes ME, et al. Epigenetic silencing of BIM in glucocorticoid poor-responsive pediatric acute lymphoblastic leukemia, and its reversal by histone deacetylase inhibition. Blood 2010; 116: 3013-3022.
11. Leclerc GJ, Mou C, Leclerc GM, et al. Histone deacetylase inhibitors induce FGPS mRNA expression and intracellular accumulation of long-chain methotrexate polyglutamates in childhood acute lymphoblastic leukemia: Implications for combination therapy. Leukemia 2010; 24: 552-562.
12. Einsiedel HG, Kawan L, Eckert C, et al. Histone deacetylase inhibitors have antitumor activity in two NOD/SCID mouse models of B-cell precursor childhood acute lymphoblastic leukemia. Leukemia 2006; 20: 1435-1436.
13. De Los Santos M, Zambrano A, Aranda A. Combined effects of retinoic acid and histone deacetylase inhibitors on human neuroblastoma SH-SY5Y cells. Mol Cancer Ther 2007; 6: 1425-1432.
14. Glick RD, Swendeman SL, Coffey DC, et al. Hybrid polar histone deacetylase inhibitor induces apoptosis and CD95/CD95 ligand expression in human neuroblastoma. Cancer Res 1999; 59: 4392-4399.
15. Milde T, Kleber S, Korshunov A, et al. A novel human high-risk ependymoma stem cell model reveals the differentiation-inducing potential of the histone deacetylase inhibitor vorinostat. Acta Neuropathol 2011; 122: 637-650.
16. Spiller SE, Ravanpay AC, Hahn AW, et al. Suberoylanilide hydroxamic acid is effective in preclinical studies of medulloblastoma. J Neurooncol 2006; 79: 259-270.
17. Fouladi M, Park JR, Stewart CF, et al. Pediatric phase I trial and pharmacokinetic study of vorinostat: A Children's Oncology Group Phase I Consortium Report. J Clin Oncol 2010; 28: 3623-3629.
18. Emanuele S, Lauricella M, Carlisi D, et al. SAHA induces apoptosis in hepatoma cells and synergistically interacts with the proteasome inhibitor bortezomib. Apoptosis 2007; 12: 1327-1338.
19. Pei XY, Dai Y, Grant S. Synergistic induction of oxidative injury and apoptosis in human multiple myeloma cells by the proteasome inhibitor bortezomib and histone deacetylase inhibitors. Clin Cancer Res 2004; 10: 3839-3852.
20. Yu C, Rahmani M, Conrad D, et al. The proteasome inhibitor bortezomib interacts synergistically with histone deacetylase inhibitors to induce apoptosis in Bcr/Abl+ cells sensitive and resistant to STI571. Blood 2003; 102: 3765-3774.
21. Heider U, von Metzler I, Kaiser M, et al. Synergistic interaction of the histone deacetylase inhibitor SAHA with the proteasome inhibitor bortezomib in mantle cell lymphoma. Eur J Haematol 2008; 80: 133-142.
22. Nawrocki ST, Carew JS, Pino MS, et al. Aggresome disruption: A novel strategy to enhance bortezomib-induced apoptosis in pancreatic cancer cells. Cancer Res 2006; 66: 3773-3781.
23. Drexler HC, Euler M. Synergistic apoptosis induction by proteasome and histone deacetylase inhibitors is dependent on protein synthesis. Apoptosis 2005; 10: 743-758.
24. Lin Z, Bazzaro M, Wang MC, et al. Combination of proteasome and HDAC inhibitors for uterine cervical cancer treatment. Clin Cancer Res 2009; 15: 570-577.
25. Pitts TM, Morrow M, Kaufman SA, et al. Vorinostat and bortezomib exert synergistic antiproliferative and proapoptotic effects in colon cancer cell models. Mol Cancer Ther 2009; 8: 342-349.
26. Fuchs O, Provaznikova D, Marinov I, et al. Antiproliferative and proapoptotic effects of proteasome inhibitors and their combination with histone deacetylase inhibitors on leukemia cells. Cardiovasc Hematol Disord Drug Targets 2009; 9: 62-77.
27. Mitsiades CS, Mitsiades NS, McMullan CJ, et al. Transcriptional signature of histone deacetylase inhibition in multiple myeloma: Biological and clinical implications. Proc Natl Acad Sci USA 2004; 101: 540-545.
28. Campbell RA, Sanchez E, Steinberg J, et al. Vorinostat enhances the antimyeloma effects of melphalan and bortezomib. Eur J Haematol 2010; 84: 201-211.
29. Simms-Waldrip T, Rodriguez-Gonzalez A, Lin T, et al. The aggresome pathway as a target for therapy in hematologic malignancies. Mol Genet Metab 2008; 94: 283-286.
30. Hideshima T, Bradner JE, Wong J, et al. Small-molecule inhibition of proteasome and aggresome function induces synergistic antitumor activity in multiple myeloma. Proc Natl Acad Sci USA 2005; 102: 8567-8572.
31. Weber D, Badros AZ, Jagannath S, et al. Vorinostat plus bortezomib for the treatment of relapsed/refractory multiple myeloma: Early clinical experience. Blood (ASH Annu Meet Abstr) 2008; 112: 871.
32. Badros A, Burger AM, Philip S, et al. Phase I study of vorinostat in combination with bortezomib for relapsed and refractory multiple myeloma. Clin Cancer Res 2009; 15: 5250-5257.
33. Schelman WR, Kolesar J, Schell K, et al. A phase I study of vorinostat in combination with bortezomib in refractory solid tumors. J Clin Oncol, 2007; ASCO Annual Meeting Proceedings Part I; 25: 3573.
34. Skolnik JM, Barrett JS, Jayaraman B, et al. Shortening the timeline of pediatric phase I trials: The rolling six design. J Clin Oncol 2008; 26: 190-195.
35. 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.
36. Parise RA, Holleran JL, Beumer JH, et al. A liquid chromatography-electrospray ionization tandem mass spectrometric assay for quantitation of the histone deacetylase inhibitor, vorinostat (suberoylanilide hydroxamicacid, SAHA), and its metabolites in human serum. J Chromatogr B Analyt Technol Biomed Life Sci 2006; 840: 108-115.
37. Argyriou AA, Iconomou G, Kalofonos HP. Bortezomib-induced peripheral neuropathy in multiple myeloma: A comprehensive review of the literature. Blood 2008; 112: 1593-1599.
38. Messinger Y, Gaynon P, Raetz E, et al. Phase I study of bortezomib combined with chemotherapy in children with relapsed childhood acute lymphoblastic leukemia (ALL): A report from the Therapeutic Advances in Childhood Leukemia (TACL) Consortium. Pediatr Blood Cancer 2010; 55: 254-259.
39. Weber D, Jagannath S, Sobecks R, et al. Combination of vorinostat plus bortezomib for the treatment of patients with multiple myeloma who have previously received bortezomib. Blood (ASH Annu Meet Abstr) 2008; 112: 3711.
40. Ninan J, Bailey H, Kolesar J, et al. A phase I study of vorinostat in combination with bortezomib in refractory solid tumors. J Clin Oncol 2009; 27: 2531A.
41. Voorhees P, Gasparetto C, Osman K, et al. Results of a phase I study of vorinostat in combination with pegylated liposomal doxorubicin and bortezomib in patients with relapsed/refractoy multiple myeloma. Blood (ASH Annu Meet Abstr) 2010; 116: 1955.
42. Jones MW, Zhang C, Oettel KR, et al. Vorinostat and bortezomib as third-line treatment in patients with advanced non-small cell lung cancer: A Wisconsin Oncology Network Phase II Study. J Clin Oncol 2011; 29: Abstr 7567.
43. Attia S, Mahoney MR, Okuno SH, et al. A phase II consortium trial of vorinostat and bortezomib for advanced soft tissue sarcomas. J Clin Oncol 2011; 29: Abstr 10075.