Pilot trial of K562/GM-CSF whole-cell vaccination in MDS patients

References

• Le DT, Uram J, Wang NH, et al. PD-1 blockade in tumors with mismatch-repair deficiency. N Engl J Med. 2015;372:2509–2520.
   PubMed Web of Science ®Google Scholar
• Ansell SM, Lesokhin AM, Borrello I, et al. PD-1 blockade with nivolumab in relapsed or refractory Hodgkin lymphoma. N Engl J Med. 2015;372:311–319.
   PubMed Web of Science ®Google Scholar
• Twyman-Saint Victor C, Rech A, Maity J, et al. Radiation and dual checkpoint blockade activate non-redundant immune mechanisms in cancer. Nature. 2015;520:373–377.
   PubMed Web of Science ®Google Scholar
• van den Eertwegh AJ, Versluis MJ, van den Berg HP, et al. Combined immunotherapy with granulocyte-macrophage colony-stimulating factor-transduced allogeneic prostate cancer cells and ipilimumab in patients with metastatic castration-resistant prostate cancer: a phase 1 dose-escalation trial. Lancet Oncol. 2012;13:509–517.
   PubMed Web of Science ®Google Scholar
• Lipson EJ, Sharfman WH, Chen S, et al. Safety and immunologic correlates of melanoma GVAX, a GM-CSF secreting allogeneic melanoma cell vaccine administered in the adjuvant setting. J Transl Med. 2015;13:214.
   PubMed Web of Science ®Google Scholar
• Le DT, Wang-Gillam A, Picozzi V, et al. Safety and survival with GVAX pancreas prime and listeria monocytogenes-expressing mesothelin (CRS-207) boost vaccines for metastatic pancreatic cancer. J Clin Oncol. 2015;33:1325–1333.,
   PubMed Web of Science ®Google Scholar
• Tsujikawa T, Kumar S, Borkar RN, et al. Quantitative multiplex immunohistochemistry reveals myeloid-inflamed tumor-immune complexity associated with poor prognosis. Cell Rep. 2017;19:203–217.
   PubMed Web of Science ®Google Scholar
• Lozzio CB, Lozzio BB. Human chronic myelogenous leukemia cell-line with positive Philadelphia chromosome. Blood. 1975;45:321–334.
   PubMed Web of Science ®Google Scholar
• Yang L, Han Y, Suarez Saiz F, et al. A tumor suppressor and oncogene: the WT1 story. Leukemia. 2007;21:868–876.
   PubMed Web of Science ®Google Scholar
• Rosenfeld C, Cheever MA, Gaiger A. WT1 in acute leukemia, chronic myelogenous leukemia and myelodysplastic syndrome: therapeutic potential of WT1 targeted therapies. Leukemia. 2003;17:1301–1312.
   PubMed Web of Science ®Google Scholar
• Tamaki H, Ogawa H, Ohyashiki K, et al. The Wilms' tumor gene WT1 is a good marker for diagnosis of disease progression of myelodysplastic syndromes. Leukemia. 1999;13:393–399.
   PubMed Web of Science ®Google Scholar
• Casalegno-Garduño R, Schmitt A, Spitschak A, et al. Immune responses to WT1 in patients with AML or MDS after chemotherapy and allogeneic stem cell transplantation. Int J Cancer. 2016;138:1792–1801.
   PubMed Web of Science ®Google Scholar
• Dranoff G, Jaffee E, Lazenby A, et al. Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long-lasting anti-tumor immunity. Proc Natl Acad Sci USA. 1993;90:3539–3543.
   PubMed Web of Science ®Google Scholar
• Smith BD, Kasamon YL, Kowalski J, et al. K562/GM-CSF immunotherapy reduces tumor burden in chronic myeloid leukemia patients with residual disease on imatinib mesylate. Clin Cancer Res. 2010;16:338–347.
   PubMed Web of Science ®Google Scholar
• Greenberg P, Tuechler L, Schanz H, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120:2454–2465.
   PubMed Web of Science ®Google Scholar
• Matzinger P. The JAM test. A simple assay for DNA fragmentation and cell death. J Immunol Methods. 1991;145:185–192.
   PubMed Web of Science ®Google Scholar
• Carlson CS, Emerson RO, Sherwood AM, et al. Using synthetic templates to design an unbiased multiplex PCR assay. Nat Comms. 2013;4:2680.
   PubMed Web of Science ®Google Scholar
• Cheson BD, Greenberg PL, Bennett JM, et al. Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood. 2006;108:419–425.
   PubMed Web of Science ®Google Scholar
• Bystry RS, Aluvihare V, Welch KA, et al. B cells and professional APCs recruit regulatory T cells via CCL4. Nat Immunol. 2001;2:1126–1132.
   PubMed Web of Science ®Google Scholar
• Neuber B, Herth I, Tolliver C, et al. Lenalidomide enhances antigen-specific activity and decreases CD45RA expression of T cells from patients with multiple myeloma. J Immunol. 2011;187:1047–1056.
   PubMed Web of Science ®Google Scholar
• McDaniel JM, Zou JX, Fulp W, et al. Reversal of T-cell tolerance in myelodysplastic syndrome through lenalidomide immune modulation. Leukemia. 2012;26:1425–1429.
   PubMed Web of Science ®Google Scholar
• Rezvani K, Yong ASM, Mielke S, et al. Leukemia-associated antigen-specific T-cell responses following combined PR1 and WT1 peptide vaccination in patients with myeloid malignancies. Blood. 2008;111:236–242.
   PubMed Web of Science ®Google Scholar
• Qi Q, Liu Y, Cheng Y, et al. Diversity and clonal selection in the human T-cell repertoire, Proc. Natl Acad Sci USA. 2014;111:13139–13144.
   PubMed Web of Science ®Google Scholar
• Day D, Hansen AR. Immune-related adverse events associated with immune checkpoint inhibitors. BioDrugs. 2016;30:571–584.
   PubMed Web of Science ®Google Scholar
• Bourke JM, O'Sullivan M, Khattak MA. Management of adverse events related to new cancer immunotherapy (immune checkpoint inhibitors). Med J Aust. 2016;205:418–424.
   PubMed Web of Science ®Google Scholar
• Sallman DA, Davila ML. Is disease-specific immunotherapy a potential reality for MDS? Clin Lymphoma Myeloma Leuk. 2017;17:S26–S30.
   Web of Science ®Google Scholar
• Burkhardt U, Hainz E, Stevenson UK, et al. Autologous CLL cell vaccination early after transplant induces leukemia-specific T cells. J Clin Invest. 2013;123:3756–3765.
   PubMed Web of Science ®Google Scholar
• Ho VT, Kim HT, Bavli N, et al. Vaccination with autologous myeloblasts admixed with GM-K562 cells in patients with advanced MDS or AML after allogeneic HSCT. Blood Adv. 2017;1:2269–2279.
   PubMed Web of Science ®Google Scholar
• Eksioglu EA, Mahmood SS, Chang M, et al. GM-CSF promotes differentiation of human dendritic cells and T lymphocytes toward a predominantly type 1 proinflammatory response. Exp Hematol. 2007;35:1163–1171.
   PubMed Web of Science ®Google Scholar
• Butterfield LH, Zhao F, Lee S, et al. Immune correlates of GM-CSF and melanoma peptide vaccination in a randomized trial for the adjuvant therapy of resected high-risk melanoma (E4697). Clin Cancer Res. 2017;23:5034–5043.
   PubMed Web of Science ®Google Scholar
• Laheru D, Lutz E, Burke J, et al. Allogeneic granulocyte macrophage colony-stimulating factor-secreting tumor immunotherapy alone or in sequence with cyclophosphamide for metastatic pancreatic cancer: a pilot study of safety, feasibility, and immune activation. Clin Cancer Res. 2008;14:1455–1463.
   PubMed Web of Science ®Google Scholar
• Ho VT, Vanneman M, Kim H, et al. Biologic activity of irradiated, autologous, GM-CSF-secreting leukemia cell vaccines early after allogeneic stem cell transplantation. Proc Natl Acad Sci USA. 2009;106:15825–15830.
   PubMed Web of Science ®Google Scholar
• Zandberg DP, Huang T-Y, Ke X, et al. Treatment and outcomes for chronic myelomonocytic leukemia compared to myelodysplastic syndromes in older adults. Haematologica. 2013;98:584–590.
   PubMed Web of Science ®Google Scholar
• Kitagawa M, Saito I, Kuwata T, et al. Overexpression of tumor necrosis factor (TNF)-alpha and interferon (IFN)-gamma by bone marrow cells from patients with myelodysplastic syndromes. Leukemia. 1997;11:2049–2054.
   PubMed Web of Science ®Google Scholar
• Kondo A, Yamashita T, Tamura H, et al. Interferon-gamma and tumor necrosis factor-alpha induce an immunoinhibitory molecule, B7-H1, via nuclear factor-kappaB activation in blasts in myelodysplastic syndromes. Blood. 2010;116:1124–1131.
   PubMed Web of Science ®Google Scholar
• Robert L, Tsoi J, Wang X, et al. CTLA4 blockade broadens the peripheral T-cell receptor repertoire. Clin Cancer Res. 2014;20:2424–2432.
   PubMed Web of Science ®Google Scholar
• Postow MA, Manuel M, Wong P, et al. Peripheral T cell receptor diversity is associated with clinical outcomes following ipilimumab treatment in metastatic melanoma. J Immunother Cancer. 2015;3:23.
   PubMed Web of Science ®Google Scholar
• Brusic A, Hainz U, Wadleigh M, et al. Detecting T-cell reactivity to whole cell vaccines: proof of concept analysis of T-cell response to K562 cell antigens in CML patients. OncoImmunology. 2012;1:1095–1103.
   PubMed Web of Science ®Google Scholar
• Yuan J, Gnjatic S, Li H, et al. CTLA-4 blockade enhances polyfunctional NY-ESO-1 specific T cell responses in metastatic melanoma patients with clinical benefit. Proc Natl Acad Sci USA. 2008;105:20410–20415.
   PubMed Web of Science ®Google Scholar
• Attig S, Hennenlotter J, Pawelec G, et al. Simultaneous infiltration of polyfunctional effector and suppressor T cells into renal cell carcinomas. Cancer Res. 2009;69:8412–8419.
   PubMed Web of Science ®Google Scholar
• Zamai L, Mariani AR, Zauli G, et al. Kinetics of in vitro natural killer activity against K562 cells as detected by flow cytometry. Cytometry. 1998;32:280–285.
   PubMedGoogle Scholar
• Zheng L, Edil BH, Soares KC, et al. A safety and feasibility study of an allogeneic colon cancer cell vaccine administered with a granulocyte-macrophage colony stimulating factor-producing bystander cell line in patients with metastatic colorectal cancer. Ann Surg Oncol. 2014;21:3931–3937.
   PubMed Web of Science ®Google Scholar
• Navada SC, Steinmann J, Lübbert M, et al. Clinical development of demethylating agents in hematology. J Clin Invest. 2014;124:40–46.
   PubMed Web of Science ®Google Scholar