Phase i trial of a bivalent gangliosides vaccine in combination with β-glucan for high-risk neuroblastoma in second or later remission

Clinical Cancer Research

Volumn 20, Issue 5, 2014, Pages 1375-1382

  Kushner, Brian H ^a   Cheung, Irene Y ^a   Modak, Shakeel ^a   Kramer, Kim ^a   Ragupathi, Govind ^a   Cheung, Nai Kong V ^a  

^a MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLUCAN; CANCER VACCINE; GANGLIOSIDE GD2; GANGLIOSIDE GD3; HEMOCYANIN; IMMUNOLOGICAL ADJUVANT; IRINOTECAN; LACTONE; OPT 821; TEMOZOLOMIDE; TUMOR ANTIGEN; UNCLASSIFIED DRUG;

ANTIBODY RESPONSE; ARTICLE; CANCER IMMUNOTHERAPY; CANCER RADIOTHERAPY; CANCER RECURRENCE; CANCER REGRESSION; CHILD; CLINICAL ARTICLE; CLINICAL PROTOCOL; DOSE RESPONSE; DRUG CONJUGATION; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG STABILITY; DRUG TOLERABILITY; HIGH RISK PATIENT; HUMAN; IMMUNE RESPONSE; IMMUNOSTIMULATION; MAXIMUM TOLERATED DOSE; MINIMAL RESIDUAL DISEASE; MULTIPLE CYCLE TREATMENT; NEUROBLASTOMA; PHASE 1 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; RECURRENCE FREE SURVIVAL;

ADOLESCENT; ANTIGENS, NEOPLASM; BETA-GLUCANS; CANCER VACCINES; CHILD; CHILD, PRESCHOOL; FEMALE; GANGLIOSIDES; HUMANS; IMMUNOTHERAPY; MALE; NEOPLASM RECURRENCE, LOCAL; NEOPLASM STAGING; NEOPLASM, RESIDUAL; NEUROBLASTOMA; RADIOTHERAPY; REMISSION INDUCTION; TREATMENT OUTCOME;

EID: 84895814045     PISSN: 10780432     EISSN: 15573265     Source Type: Journal    
DOI: 10.1158/1078-0432.CCR-13-1012     Document Type: Article

References (48)

1. Maris JM. Recent advances in neuroblastoma. N Engl J Med 2010;362:2202-11.
2. Kushner BH, Kramer K, Modak S, Cheung NK. Sensitivity of surveillance studies for detecting asymptomatic and unsuspected relapse of high-risk neuroblastoma. J Clin Oncol 2009;27:1041-6.
3. Kushner BH, Kramer K, Modak S, Cheung NK. Irinotecan plus temozolomide for relapsed or refractory neuroblastoma. J Clin Oncol 2006;24:5271-6.
4. Kramer K, Kushner BH, Modak S, Pandit-Taskar N, Smith-Jones P, Zanzonico P, et al. Compartmental intrathecal radioimmunotherapy: results for treatment for metastatic CNS neuroblastoma. J Neurooncol 2010;97:409-18.
5. Kushner BH, Kramer K, Modak S, Qin LX, Cheung NK. Differential impact of high-dose cyclophosphamide, topotecan, and vincristine in clinical subsets of patients with chemoresistant neuroblastoma. Cancer 2010;116:3054-60.
6. Kushner BH, Kramer K, Modak S, Cheung NK. High-dose carboplatinirinotecan-temozolomide: treatment option for neuroblastoma resistant to topotecan. Pediatr Blood Cancer 2011;56:403-8.
7. Kushner BH, Kramer K, Modak S, Yataghene K, Cheung NK. Highdose cyclophosphamide-irinotecan-vincristine for primary refractory neuroblastoma. Eur J Cancer 2011;47:84-9.
8. Kushner BH, Modak S, Kramer K, Basu EM, Roberts SS, Cheung NK. Ifosfamide, carboplatin, and etoposide for neuroblastoma: a highdose salvage regimen and review of the literature. Cancer 2013;119: 665-71.
9. Hamilton WB, Helling F, Lloyd KO, Livingston PO. Ganglioside expression on human malignant melanoma assessed by quantitative immune thin-layer chromatography. Int J Cancer 1993;53: 566-73.
10. Zhang S, Cordon-Cardo C, Zhang HS, Reuter VE, Adluri S, Hamilton WB, et al. Selection of tumor antigens as targets for immune attack using immunohistochemistry. I. Focus on gangliosides. Int J Cancer 1997;73:42-9.
11. Houghton AN, Mintzer D, Cordon-Cardo C, Welt S, Fliegel B, Vadhan S, et al. Mouse monoclonal IgG3 antibody detecting GD3 ganglioside: a phase I trial in patients with malignant melanoma. Proc Natl Acad Sci U S A 1985;82:1242-6.
12. Yu AL, Gilman AL, Ozkaynak MF, London WB, Kreissman SG, Chen HX, et al. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med 2010;363:1324-34.
13. Cheung NK, Cheung IY, Kushner BH, Ostrovnaya I, Chamberlain E, Kramer K, et al. Murine anti-GD2 monoclonal antibody 3F8 combined with granulocyte-macrophage colony-stimulating factor and 13-cisretinoic acid in high-risk patients with stage 4 neuroblastoma in first remission. J Clin Oncol 2012;30:3264-70.
14. Ritter G, Boosfeld E, Calves MJ, Oettgen HF, Old LJ, Livingston PO. Antibody response to immunization with purified GD3 ganglioside and GD3 derivatives (lactones, amide and gangliosidol) in the mouse. Immunobiology 1990;182:32-43.
15. Ritter G, Boosfeld E, Adluri R, Calves M, Oettgen HF, Old LJ, et al. Antibody response to immunization with ganglioside GD3 and GD3 congeners (lactones, amide and gangliosidol) in patients with malignant melanoma. Int J Cancer 1991;48:379-85.
16. Kensil CR, Patel U, Lennick M, Marciani D. Separation and characterization of saponins with adjuvant activity from Quillaja saponaria Molina cortex. J Immunol 1991;146:431-7.
17. Helling F, Shang A, Calves M, Zhang S, Ren S, Yu RK, et al. GD3 vaccines for melanoma: superior immunogenicity of keyhole limpet hemocyanin conjugate vaccines. Cancer Res 1994;54:197-203.
18. Ragupathi G, Meyers M, Adluri S, Howard L, Musselli C, Livingston PO. Induction of antibodies against GD3 ganglioside in melanoma patients by vaccination with GD3-lactone-KLH conjugate plus immunological adjuvant QS-21. Int J Cancer 2000;85:659-66.
19. Ragupathi G, Livingston PO, Hood C, Gathuru J, Krown SE, Chapman PB, et al. Consistent antibody response against ganglioside GD2 induced in patients with melanoma by a GD2 lactone-keyhole limpet hemocyanin conjugate vaccine plus immunological adjuvant QS-21. Clin Cancer Res 2003;9:5214-20.
20. Livingston PO, Adluri S, Helling F, Yao TJ, Kensil CR, Newman MJ, et al. Phase 1 trial of immunological adjuvant QS-21 with a GM2 ganglioside-keyhole limpet haemocyanin conjugate vaccine in patients with malignant melanoma. Vaccine 1994;12:1275-80.
21. Yan J, Vetvicka V, Xia Y, Coxon A, Carroll MC, Mayadas TN, et al. Betaglucan, a "specific" biologic response modifier that uses antibodies to target tumors for cytotoxic recognition by leukocyte complement receptor type 3 (CD11b/CD18). J Immunol 1999;163:3045-52.
22. Taylor PR, Brown GD, Reid DM, Willment JA, Martinez-Pomares L, Gordon S, et al. The b-glucan receptor, dectin-1, is predominantly expressed on the surface of cells of the monocyte/macrophage and neutrophil lineages. J Immunol 2002;169:3876-82.
23. Hong F, Yan J, Baran JT, Allendorf DJ, Hansen RD, Ostroff GR, et al. Mechanism by which orally administered b(1,3)-glucans function with anti-tumor monoclonal antibodies to mediate tumor regression and tumor-free survival. J Immunol 2004;173:797-806.
24. Cheung NK, Modak S. Oral (1!3),(1!4)-b-D-glucan synergizes with antiganglioside GD2 monoclonal antibody 3F8 in the therapy of neuroblastoma. Clin Cancer Res 2002;8:1217-23.
25. Cheung NK, Modak S, Vickers A, Knuckles B. Orally administered beta-glucans enhance anti-tumor effects of monoclonal antibodies. Cancer Immunol Immunother 2002;51:557-64.
26. Brodeur GM, Pritchard J, Berthold F, Carlsen NL, Castel V, Castelberry RP, et al. Revisions of the international criteria for neuroblastoma diagnosis, staging, and response to treatment. J Clin Oncol 1993;11: 1466-77.
27. Cheung IY, Hsu K, Cheung NK. Activation of peripheral-blood granulocytes is strongly correlated with patient outcome after immunotherapy with anti-GD2 monoclonal antibody and granulocyte-macrophage colony-stimulating factor. J Clin Oncol 2012;30:426-432.
28. Ross GD. Role of the lectin domain of Mac-1/CR3 (CD11b/CD18) in regulating intercellular adhesion. Immunol Res 2002;25:219-27.
29. Cheung IY, Lo Piccolo MS, Kushner BH, Cheung NK. Early molecular response of marrow disease to biologic therapy is highly prognostic in neuroblastoma. J Clin Oncol 2003;21:3853-8.
30. Cheung IY, Feng Y, Gerald W, Cheung NK. Exploiting gene expression profiling to identify novel minimal residual disease markers of neuroblastoma. Clin Cancer Res 2008;14:7020-7.
31. London WB, Castel V, Monclair T, Ambros PF, Pearson AD, Cohn SL, et al.Clinical and biologic features predictive of survival after relapse of neuroblastoma: a report from the International Neuroblastoma Risk Group project. J Clin Oncol 2011;29:3286-92.
32. Simon T, Berthold F, Borkhardt A, Kremens B, De Carolis B, Hero B. Treatment and outcomes of patients with relapsed, high-risk neuroblastoma: results of German trials. Pediatr Blood Cancer 2011;56:578-83.
33. Cole KA, Maris JM. New strategies in refractory and recurrent neuroblastoma: translational opportunities to impact patient outcome. Clin Cancer Res 2012;18:2423-8.
34. Cheung IY, Feng Y, Cheung NK. Early negative minimal residual disease in bone marrow after immunotherapy is less predictive of late or non-marrow relapse among patients with high-risk stage 4 neuroblastoma. Pediatr Blood Cancer 2013;60:E32-E34.
35. Russell HV, Strother D, Mei Z, Rill D, Popek E, Biagi E, et al. A phase 1/2 study of autologous neuroblastoma tumor cells genetically modified to secrete IL-2 in patients with high-risk neuroblastoma. J Immunother 2008;31:812-9.
36. Rousseau RF, Haight AE, Hirschmann-Jax C, Yvon ES, Rill DR, Mei Z, et al. Local and systemic effects of an allogeneic tumor cell vaccine combining transgenic human lymphotactin with interleukin-2 in patients with advanced or refractory neuroblastoma. Blood 2003;101: 1718-26.
37. Louis CU, Brenner MK. Cellular immunotherapy for neuroblastoma: a review of current vaccine and adoptive T cell therapeutics. Curr Pharm Des 2009;15:424-9.
38. Geiger JD, Hutchinson RJ, Hohenkirk LF, McKenna EA, Yanik GA, Levine JE, et al. Vaccination of pediatric solid tumor patients with tumor lysate-pulsed dendritic cells can expand specific T cells and mediate tumor regression. Cancer Res 2001;61:8513-9.
39. Caruso DA, Orme LM, AmorGM,Neale AM, Radcliff FJ, Downie P, et al. Results of a phase I study utilizing monocyte-derived dendritic cells pulsed with tumorRNAin children with Stage 4 neuroblastoma. Cancer 2005;103:1280-91.
40. Coughlin CM, Fleming MD, Carroll RG, Pawel BR, Hogarty MD, Shan X, et al. Immunosurveillance and survivin-specific T-cell immunity in children with high-risk neuroblastoma. J Clin Oncol 2006;24:5725-34.
41. Nasi ML, Meyers M, Livingston PO, Houghton AN, Chapman PB. Antimelanoma effects of R24, a monoclonal antibody against GD3 ganglioside. Melanoma Res 1997;7:S155-62.
42. Zhang H, Zhang S, Cheung NK, Ragupathi G, Livingston PO. Antibodies against GD2 ganglioside can eradicate syngeneic cancer micrometastases. Cancer Res 1998;58:2844-9.
43. Kim SK, Wu X, Ragupathi G, Gathuru J, Koide F, Cheung NK, et al. Impact of minimal tumor burden on antibody response to vaccination. Cancer Immunol Immunother 2011;60:621-7.
44. Mellman I, Coukos G, Dranoff G. Cancer immunotherapy comes of age. Nature 2011;480:480-9.
45. Navid F, Armstrong M, Barfield RC. Immune therapies for neuroblastoma. Cancer Biol Ther 2009;8:874-82.
46. Jerne NK. The generative grammar of the immune system. EMBO 1985;4847-52.
47. Raychaudhuri S, Saeki Y, Chen JJ, Kohler H. Analysis of the idiotypic network in tumor immunity. J Immunol 1987;139:3902-10.
48. Cheung NK, Guo HF, Heller G, Cheung IY. Induction of Ab3 and Ab30 antibody was associated with long-term survival after anti-GD2 antibody therapy of stage 4 neuroblastoma. Clin Cancer Res 2000;6: 2653-60.