Vaccines: a promising therapy for myelodysplastic syndrome.
Dendritic cells
Immunotherapy
Myelodysplastic neoplasms
Vaccines
Journal
Journal of hematology & oncology
ISSN: 1756-8722
Titre abrégé: J Hematol Oncol
Pays: England
ID NLM: 101468937
Informations de publication
Date de publication:
08 Jan 2024
08 Jan 2024
Historique:
received:
06
09
2023
accepted:
23
12
2023
medline:
9
1
2024
pubmed:
9
1
2024
entrez:
8
1
2024
Statut:
epublish
Résumé
Myelodysplastic neoplasms (MDS) define clonal hematopoietic malignancies characterized by heterogeneous mutational and clinical spectra typically seen in the elderly. Curative treatment entails allogeneic hematopoietic stem cell transplant, which is often not a feasible option due to older age and significant comorbidities. Immunotherapy has the cytotoxic capacity to elicit tumor-specific killing with long-term immunological memory. While a number of platforms have emerged, therapeutic vaccination presents as an appealing strategy for MDS given its promising safety profile and amenability for commercialization. Several preclinical and clinical trials have investigated the efficacy of vaccines in MDS; these include peptide vaccines targeting tumor antigens, whole cell-based vaccines and dendritic cell-based vaccines. These therapeutic vaccines have shown acceptable safety profiles, but consistent clinical responses remain elusive despite robust immunological reactions. Combining vaccines with immunotherapeutic agents holds promise and requires further investigation. Herein, we highlight therapeutic vaccine trials while reviewing challenges and future directions of successful vaccination strategies in MDS.
Identifiants
pubmed: 38191498
doi: 10.1186/s13045-023-01523-4
pii: 10.1186/s13045-023-01523-4
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
4Subventions
Organisme : FDA HHS
ID : R01FD007268
Pays : United States
Informations de copyright
© 2024. The Author(s).
Références
Ansprenger C, Amberger DC, Schmetzer HM. Potential of immunotherapies in the mediation of antileukemic responses for patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)—with a focus on Dendritic cells of leukemic origin (DCleu). Clin Immunol. 2020;217: 108467. https://doi.org/10.1016/j.clim.2020.108467 .
doi: 10.1016/j.clim.2020.108467
pubmed: 32464186
Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120(12):2454–65. https://doi.org/10.1182/blood-2012-03-420489 .
doi: 10.1182/blood-2012-03-420489
pmcid: 4425443
Garcia-Manero G, Daver NG, Xu J, et al. Magrolimab + azacitidine versus azacitidine + placebo in untreated higher risk (HR) myelodysplastic syndrome (MDS): The phase 3, randomized, ENHANCE study. J Clin Oncol. 2021;39(15_suppl):TPS7055. https://doi.org/10.1200/JCO.2021.39.15_suppl.TPS7055 .
doi: 10.1200/JCO.2021.39.15_suppl.TPS7055
Ogawa S. Genetics of MDS. Blood. 2019;133(10):1049–59. https://doi.org/10.1182/blood-2018-10-844621 .
doi: 10.1182/blood-2018-10-844621
pubmed: 30670442
pmcid: 6587668
Peng X, Zhu X, Di T, et al. The yin-yang of immunity: Immune dysregulation in myelodysplastic syndrome with different risk stratification. Front Immunol. 2022;13.
Bewersdorf JP, Xie Z, Bejar R, et al. Current landscape of translational and clinical research in myelodysplastic syndromes/neoplasms (MDS). In: Proceedings from the 1st International Workshop on MDS (iwMDS) Of the International Consortium for MDS (icMDS). Blood Rev. Published online March 11, 2023, p 101072. https://doi.org/10.1016/j.blre.2023.101072
Tinsley-Vance SM, Davis M, Ajayi O. Role of luspatercept in the management of lower-risk myelodysplastic syndromes. J Adv Pract Oncol. 2023;14(1):82–7. https://doi.org/10.6004/jadpro.2023.14.1.8 .
doi: 10.6004/jadpro.2023.14.1.8
pubmed: 36741213
pmcid: 9894202
Linder K, Lulla P. Myelodysplastic syndrome and immunotherapy novel to next in-line treatments. Hum Vaccines Immunother. 2021;17(8):2602–16. https://doi.org/10.1080/21645515.2021.1898307 .
doi: 10.1080/21645515.2021.1898307
Vaddepally RK, Kharel P, Pandey R, Garje R, Chandra AB. Review of indications of FDA-approved immune checkpoint inhibitors per NCCN guidelines with the level of evidence. Cancers. 2020;12(3):738. https://doi.org/10.3390/cancers12030738 .
doi: 10.3390/cancers12030738
pubmed: 32245016
pmcid: 7140028
Lin MJ, Svensson-Arvelund J, Lubitz GS, et al. Cancer vaccines: the next immunotherapy frontier. Nat Cancer. 2022;3(8):911–26. https://doi.org/10.1038/s43018-022-00418-6 .
doi: 10.1038/s43018-022-00418-6
Cheever MA, Higano CS. PROVENGE (Sipuleucel-T) in prostate cancer: the first FDA-approved therapeutic cancer vaccine. Clin Cancer Res. 2011;17(11):3520–6. https://doi.org/10.1158/1078-0432.CCR-10-3126 .
doi: 10.1158/1078-0432.CCR-10-3126
pubmed: 21471425
Moderna and Merck Announce mRNA-4157/V940, an Investigational Personalized mRNA Cancer Vaccine, in Combination with KEYTRUDA(R) (pembrolizumab), Met Primary Efficacy Endpoint in Phase 2b KEYNOTE-942 Trial. Accessed 14 Oct 2023. https://investors.modernatx.com/news/news-details/2022/Moderna-and-Merck-Announce-mRNA-4157V940-an-Investigational-Personalized-mRNA-Cancer-Vaccine-in-Combination-with-KEYTRUDAR-pembrolizumab-Met-Primary-Efficacy-Endpoint-in-Phase-2b-KEYNOTE-942-Trial/default.aspx
Rojas LA, Sethna Z, Soares KC, et al. Personalized RNA neoantigen vaccines stimulate T cells in pancreatic cancer. Nature. 2023;618(7963):144–50. https://doi.org/10.1038/s41586-023-06063-y .
doi: 10.1038/s41586-023-06063-y
pubmed: 37165196
pmcid: 10171177
Avigan D, Rosenblatt J. Vaccine therapy in hematologic malignancies. Blood. 2018;131(24):2640–50. https://doi.org/10.1182/blood-2017-11-785873 .
doi: 10.1182/blood-2017-11-785873
Liu W, Teodorescu P, Halene S, Ghiaur G. The coming of age of preclinical models of MDS. Front Oncol. 2022;12.
Kennedy AL, Shimamura A. Genetic predisposition to MDS: clinical features and clonal evolution. Blood. 2019;133(10):1071–85. https://doi.org/10.1182/blood-2018-10-844662 .
doi: 10.1182/blood-2018-10-844662
pubmed: 30670445
pmcid: 6405331
M.D. Anderson Cancer Center. Phase 2 Study of Proteinase 3 PR1 Peptide Vaccine in Myelodysplastic Syndrome (MDS). clinicaltrials.gov; 2012. Accessed 17 June 2023. https://clinicaltrials.gov/ct2/show/NCT00893997 .
Ambinder AJ, DeZern AE. Navigating the contested borders between myelodysplastic syndrome and acute myeloid leukemia. Front Oncol. 2022;12:1033534. https://doi.org/10.3389/fonc.2022.1033534 .
doi: 10.3389/fonc.2022.1033534
pubmed: 36387170
pmcid: 9650616
Pfeilstöcker M, Tuechler H, Sanz G, et al. Time-dependent changes in mortality and transformation risk in MDS. Blood. 2016;128(7):902–10. https://doi.org/10.1182/blood-2016-02-700054 .
doi: 10.1182/blood-2016-02-700054
pubmed: 27335276
pmcid: 5161006
Bejar R. What biologic factors predict for transformation to AML? Best Pract Res Clin Haematol. 2018;31(4):341–5. https://doi.org/10.1016/j.beha.2018.10.002 .
doi: 10.1016/j.beha.2018.10.002
pubmed: 30466744
Rautenberg C, Germing U, Pechtel S, et al. Prognostic impact of peripheral blood WT1-mRNA expression in patients with MDS. Blood Cancer J. 2019;9(11):86. https://doi.org/10.1038/s41408-019-0248-y .
doi: 10.1038/s41408-019-0248-y
pubmed: 31719523
pmcid: 6851368
Gejman RS, Chang AY, Jones HF, et al. Rejection of immunogenic tumor clones is limited by clonal fraction. Elife. 2018;7:e41090. https://doi.org/10.7554/eLife.41090 .
doi: 10.7554/eLife.41090
pmcid: 6269121
Stephens AJ, Burgess-Brown NA, Jiang S. Beyond just peptide antigens: the complex world of peptide-based cancer vaccines. Front Immunol. 2021. https://doi.org/10.3389/fimmu.2021.696791 .
doi: 10.3389/fimmu.2021.696791
pubmed: 34659195
pmcid: 8515132
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(1):236–42. https://doi.org/10.1182/blood-2007-08-108241 .
doi: 10.1182/blood-2007-08-108241
pubmed: 17875804
pmcid: 2200809
Gessler M, Poustka A, Cavenee W, Neve RL, Orkin SH, Bruns GAP. Homozygous deletion in Wilms tumours of a zinc-finger gene identified by chromosome jumping. Nature. 1990;343(6260):774–8. https://doi.org/10.1038/343774a0 .
doi: 10.1038/343774a0
pubmed: 2154702
Bergmann L, Miething C, Maurer U, et al. High levels of Wilms’ tumor gene (wt1) mRNA in acute myeloid leukemias are associated with a worse long-term outcome. Blood. 1997;90(3):1217–25.
doi: 10.1182/blood.V90.3.1217
pubmed: 9242555
Cilloni D, Gottardi E, Messa F, et al. Significant correlation between the degree of WT1 expression and the International Prognostic Scoring System Score in patients with myelodysplastic syndromes. J Clin Oncol Off J Am Soc Clin Oncol. 2003;21(10):1988–95. https://doi.org/10.1200/JCO.2003.10.503 .
doi: 10.1200/JCO.2003.10.503
Brayer J, Lancet JE, Powers J, et al. WT1 vaccination in AML and MDS: a pilot trial with synthetic analog peptides. Am J Hematol. 2015;90(7):602–7. https://doi.org/10.1002/ajh.24014 .
doi: 10.1002/ajh.24014
pmcid: 4617516
Rezvani K, Yong ASM, Mielke S, et al. Repeated PR1 and WT1 peptide vaccination in Montanide-adjuvant fails to induce sustained high-avidity, epitope-specific CD8+ T cells in myeloid malignancies. Haematologica. 2011;96(3):432–40. https://doi.org/10.3324/haematol.2010.031674 .
doi: 10.3324/haematol.2010.031674
pubmed: 21134985
Ueda Y, Ogura M, Miyakoshi S, et al. Phase 1/2 study of the WT1 peptide cancer vaccine WT4869 in patients with myelodysplastic syndrome. Cancer Sci. 2017;108(12):2445–53. https://doi.org/10.1111/cas.13409 .
doi: 10.1111/cas.13409
pubmed: 28949050
pmcid: 5715294
Ueda Y, Usuki K, Fujita J, et al. Phase 1/2 study evaluating the safety and efficacy of DSP-7888 dosing emulsion in myelodysplastic syndromes. Cancer Sci. 2022;113(4):1377–92. https://doi.org/10.1111/cas.15245 .
doi: 10.1111/cas.15245
pubmed: 34932235
pmcid: 8990724
Di Stasi A, Jimenez AM, Minagawa K, Al-Obaidi M, Rezvani K. Review of the results of WT1 peptide vaccination strategies for myelodysplastic syndromes and acute myeloid leukemia from nine different studies. Front Immunol. 2015;6.
Keilholz U, Letsch A, Busse A, et al. A clinical and immunologic phase 2 trial of Wilms tumor gene product 1 (WT1) peptide vaccination in patients with AML and MDS. Blood. 2009;113(26):6541–8. https://doi.org/10.1182/blood-2009-02-202598 .
doi: 10.1182/blood-2009-02-202598
pubmed: 19389880
Molldrem J, Dermime S, Parker K, et al. Targeted T-cell therapy for human leukemia: cytotoxic T lymphocytes specific for a peptide derived from proteinase 3 preferentially lyse human myeloid leukemia cells. Blood. 1996;88(7):2450–7. https://doi.org/10.1182/blood.V88.7.2450.bloodjournal8872450 .
doi: 10.1182/blood.V88.7.2450.bloodjournal8872450
pubmed: 8839835
Molldrem JJ, Lee PP, Kant S, et al. Chronic myelogenous leukemia shapes host immunity by selective deletion of high-avidity leukemia-specific T cells. J Clin Investig. 2003;111(5):639–47. https://doi.org/10.1172/JCI16398 .
doi: 10.1172/JCI16398
pubmed: 12618518
pmcid: 151894
Qazilbash MH, Wieder E, Thall PF, et al. PR1 peptide vaccine induces specific immunity with clinical responses in myeloid malignancies. Leukemia. 2017;31(3):697–704. https://doi.org/10.1038/leu.2016.254 .
doi: 10.1038/leu.2016.254
pubmed: 27654852
Thomas R, Al-Khadairi G, Roelands J, et al. NY-ESO-1 based immunotherapy of cancer: current perspectives. Front Immunol. 2018;9:947. https://doi.org/10.3389/fimmu.2018.00947 .
doi: 10.3389/fimmu.2018.00947
pubmed: 29770138
pmcid: 5941317
De Smet C, De Backer O, Faraoni I, Lurquin C, Brasseur F, Boon T. The activation of human gene MAGE-1 in tumor cells is correlated with genome-wide demethylation. Proc Natl Acad Sci USA. 1996;93(14):7149–53. https://doi.org/10.1073/pnas.93.14.7149 .
doi: 10.1073/pnas.93.14.7149
pmcid: 38951
Almstedt M, Blagitko-Dorfs N, Duque-Afonso J, et al. The DNA demethylating agent 5-aza-2’-deoxycytidine induces expression of NY-ESO-1 and other cancer/testis antigens in myeloid leukemia cells. Leuk Res. 2010;34(7):899–905. https://doi.org/10.1016/j.leukres.2010.02.004 .
doi: 10.1016/j.leukres.2010.02.004
pubmed: 20381863
Satie AP, Meyts ERD, Spagnoli GC, et al. The cancer-testis gene, NY-ESO-1, Is expressed in normal fetal and adult testes and in spermatocytic seminomas and testicular carcinoma in situ. Lab Invest. 2002;82(6):775–80. https://doi.org/10.1097/01.LAB.0000017169.26718.5F .
doi: 10.1097/01.LAB.0000017169.26718.5F
pubmed: 12065688
Srivastava P, Matsuzaki J, Paluch BE, et al. NY-ESO-1 vaccination in combination with decitabine for patients with MDS induces CD4+ and CD8+ T-cell responses. Blood. 2015;126(23):2873. https://doi.org/10.1182/blood.V126.23.2873.2873 .
doi: 10.1182/blood.V126.23.2873.2873
Griffiths EA, Srivastava P, Matsuzaki J, et al. NY-ESO-1 vaccination in combination with decitabine induces antigen-specific T-lymphocyte responses in patients with myelodysplastic syndrome. Clin Cancer Res. 2018;24(5):1019–29. https://doi.org/10.1158/1078-0432.CCR-17-1792 .
doi: 10.1158/1078-0432.CCR-17-1792
pubmed: 28947565
Karbach J, Gnjatic S, Bender A, et al. Tumor-reactive CD8+ T-cell responses after vaccination with NY-ESO-1 peptide, CpG 7909 and Montanide ISA-51: association with survival. Int J Cancer. 2010;126(4):909–18. https://doi.org/10.1002/ijc.24850 .
doi: 10.1002/ijc.24850
pubmed: 19728336
Gnjatic S, Nishikawa H, Jungbluth AA, et al. NY-ESO-1: review of an immunogenic tumor antigen. Adv Cancer Res. 2006;95:1–30. https://doi.org/10.1016/S0065-230X(06)95001-5 .
doi: 10.1016/S0065-230X(06)95001-5
pubmed: 16860654
Roswell Park Cancer Institute. A phase I study of DEC205mAb-NY-ESO-1 fusion protein (CDX-1401) given with adjuvant PoIylCLC in conjunction with 5-Aza-2’deoxycytidine (decitabine) in patients with MDS or low blast count AML. clinicaltrials.gov; 2022. Accessed 2 June 2023. https://clinicaltrials.gov/ct2/show/study/NCT01834248 .
Holmberg-Thydén S, Dufva IH, Ortved Gang A, et al. Therapeutic cancer vaccination targeting shared tumor associated antigens in combination with azacitidine for high risk myelodysplastic syndrome—a phase I clinical trial. Blood. 2020;136:23–4. https://doi.org/10.1182/blood-2020-142806 .
doi: 10.1182/blood-2020-142806
Hinneh JA, Gillis JL, Moore NL, Butler LM, Centenera MM. The role of RHAMM in cancer: Exposing novel therapeutic vulnerabilities. Front Oncol. 2022;12.
Schmitt M, Schmitt A, Rojewski MT, et al. RHAMM-R3 peptide vaccination in patients with acute myeloid leukemia, myelodysplastic syndrome, and multiple myeloma elicits immunologic and clinical responses. Blood. 2008;111(3):1357–65. https://doi.org/10.1182/blood-2007-07-099366 .
doi: 10.1182/blood-2007-07-099366
pubmed: 17978170
Greiner J, Schmitt A, Giannopoulos K, et al. High-dose RHAMM-R3 peptide vaccination for patients with acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Haematologica. 2010;95(7):1191–7. https://doi.org/10.3324/haematol.2009.014704 .
doi: 10.3324/haematol.2009.014704
pubmed: 20081055
pmcid: 2895045
Snauwaert S, Vanhee S, Goetgeluk G, et al. RHAMM/HMMR (CD168) is not an ideal target antigen for immunotherapy of acute myeloid leukemia. Haematologica. 2012;97(10):1539–47. https://doi.org/10.3324/haematol.2012.065581 .
doi: 10.3324/haematol.2012.065581
pubmed: 22532518
pmcid: 3487554
Cohen KA, Liu TF, Cline JM, Wagner JD, Hall PD, Frankel AE. Safety evaluation of DT388IL3, a diphtheria toxin/interleukin 3 fusion protein, in the cynomolgus monkey. Cancer Immunol Immunother CII. 2005;54(8):799–806. https://doi.org/10.1007/s00262-004-0643-4 .
doi: 10.1007/s00262-004-0643-4
Frankel A, Weir M, Hall P, et al. Induction of remission in patients with acute myeloid leukemia without prolonged myelosuppression using diphtheria toxin-interleukin 3 fusion protein. J Clin Oncol. 2007;25:7068–7068. https://doi.org/10.1200/jco.2007.25.18_suppl.7068 .
doi: 10.1200/jco.2007.25.18_suppl.7068
Lane A. Phase 1 Study of SL-401 in Combination With Azacitidine and Venetoclax in Relapsed/Refractory Acute Myeloid Leukemia (AML) and in Treatment-Naive Subjects With AML Not Eligible for Standard Induction and in Subjects With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) or SL-401 in Combination With Azacitidine in Subjects With High-Risk Myelodysplastic Syndrome (MDS). clinicaltrials.gov; 2022. Accessed 2 June 2023. https://clinicaltrials.gov/ct2/show/NCT03113643 .
Klausen U, Holmberg S, Holmström MO, et al. Novel strategies for peptide-based vaccines in hematological malignancies. Front Immunol. 2018;9:2264. https://doi.org/10.3389/fimmu.2018.02264 .
doi: 10.3389/fimmu.2018.02264
pubmed: 30327655
pmcid: 6174926
Maslak PG, Dao T, Bernal Y, et al. Phase 2 trial of a multivalent WT1 peptide vaccine (galinpepimut-S) in acute myeloid leukemia. Blood Adv. 2018;2(3):224–34. https://doi.org/10.1182/bloodadvances.2017014175 .
doi: 10.1182/bloodadvances.2017014175
pubmed: 29386195
pmcid: 5812332
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(8):3539–43.
doi: 10.1073/pnas.90.8.3539
pubmed: 8097319
pmcid: 46336
Ho VT, Kim HT, Brock J, et al. GM-CSF secreting leukemia cell vaccination for MDS/AML after allogeneic HSCT: a randomized, double-blinded, phase 2 trial. Blood Adv. 2022;6(7):2183–94. https://doi.org/10.1182/bloodadvances.2021006255 .
doi: 10.1182/bloodadvances.2021006255
pubmed: 34807983
pmcid: 9006263
Robinson TM, Prince GT, Thoburn C, et al. Pilot trial of K562/GM-CSF whole-cell vaccination in MDS patients. Leuk Lymphoma. 2018;59(12):2801–11. https://doi.org/10.1080/10428194.2018.1443449 .
doi: 10.1080/10428194.2018.1443449
pubmed: 29616857
pmcid: 6352988
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(37):15825–30. https://doi.org/10.1073/pnas.0908358106 .
doi: 10.1073/pnas.0908358106
pubmed: 19717467
pmcid: 2747203
Biavati L, Huff CA, Ferguson A, et al. An allogeneic multiple myeloma GM-CSF-secreting vaccine with lenalidomide induces long-term immunity and durable clinical responses in patients in near complete remission. Clin Cancer Res Off J Am Assoc Cancer Res. 2021;27(24):6696–708. https://doi.org/10.1158/1078-0432.CCR-21-1916 .
doi: 10.1158/1078-0432.CCR-21-1916
Padron E, Komrokji RS, Lancet JE, et al. A phase I pilot study of bystander vaccine and lenalidomide immune augmentation in patients with myelodysplastic syndrome (MDS). Blood. 2010;116(21):2925. https://doi.org/10.1182/blood.V116.21.2925.2925 .
doi: 10.1182/blood.V116.21.2925.2925
Yu J, Sun H, Cao W, Song Y, Jiang Z. Research progress on dendritic cell vaccines in cancer immunotherapy. Exp Hematol Oncol. 2022;11(1):3. https://doi.org/10.1186/s40164-022-00257-2 .
doi: 10.1186/s40164-022-00257-2
pubmed: 35074008
pmcid: 8784280
Van Tendeloo VF, Van de Velde A, Van Driessche A, et al. Induction of complete and molecular remissions in acute myeloid leukemia by Wilms’ tumor 1 antigen-targeted dendritic cell vaccination. Proc Natl Acad Sci USA. 2010;107(31):13824–9. https://doi.org/10.1073/pnas.1008051107 .
doi: 10.1073/pnas.1008051107
pmcid: 2922237
Palma M, Hansson L, Choudhury A, et al. Vaccination with dendritic cells loaded with tumor apoptotic bodies (Apo-DC) in patients with chronic lymphocytic leukemia: effects of various adjuvants and definition of immune response criteria. Cancer Immunol Immunother. 2011;61(6):865.
doi: 10.1007/s00262-011-1149-5
pubmed: 22086161
Litzinger MT, Foon KA, Tsang KY, Schlom J, Palena C. Comparative analysis of MVA-CD40L and MVA-TRICOM vectors for enhancing the immunogenicity of chronic lymphocytic leukemia (CLL) cells. Leuk Res. 2010;34(10):1351–7. https://doi.org/10.1016/j.leukres.2009.12.013 .
doi: 10.1016/j.leukres.2009.12.013
pubmed: 20122733
pmcid: 2891581
Weinstock M, Rosenblatt J, Avigan D. Dendritic cell therapies for hematologic malignancies. Mol Ther Methods Clin Dev. 2017;5:66–75. https://doi.org/10.1016/j.omtm.2017.03.004 .
doi: 10.1016/j.omtm.2017.03.004
pmcid: 5415319
Davison GM, Novitzky N, Abdulla R. Monocyte derived dendritic cells have reduced expression of co-stimulatory molecules but are able to stimulate autologous T-cells in patients with MDS. Hematol Oncol Stem Cell Ther. 2013;6(2):49–57. https://doi.org/10.1016/j.hemonc.2013.05.001 .
doi: 10.1016/j.hemonc.2013.05.001
pubmed: 23714180
Durable responses and survival in high risk AML and MDS patients treated with an allogeneic leukemia-derived dendritic cell vaccine. Elsevier enhanced reader. https://doi.org/10.1182/blood-2019-127881 .
Mendus. An International, Multicentre, Open-Label Study To Evaluate The Efficacy and Safety of Two Different Vaccination Regimens of Immunotherapy With Allogeneic Dendritic Cells, DCP-001, in Patients With Acute Myeloid Leukaemia That Are In Remission With Persistent MRD. clinicaltrials.gov; 2022. Accessed 13 April 2023. https://clinicaltrials.gov/ct2/show/NCT03697707 .
Li Z, Cai H, Li Z, et al. A tumor cell membrane-coated self-amplified nanosystem as a nanovaccine to boost the therapeutic effect of anti-PD-L1 antibody. Bioact Mater. 2023;21:299–312. https://doi.org/10.1016/j.bioactmat.2022.08.028 .
doi: 10.1016/j.bioactmat.2022.08.028
pubmed: 36157245
Johnson DT, Zhou J, Kroll AV, et al. Acute myeloid leukemia cell membrane-coated nanoparticles for cancer vaccination immunotherapy. Leukemia. 2022;36(4):994–1005. https://doi.org/10.1038/s41375-021-01432-w .
doi: 10.1038/s41375-021-01432-w
pubmed: 34845316
Comont T, Treiner E, Vergez F. From immune dysregulations to therapeutic perspectives in myelodysplastic syndromes: a review. Diagnostics. 2021;11(11):1982. https://doi.org/10.3390/diagnostics11111982 .
doi: 10.3390/diagnostics11111982
pubmed: 34829329
pmcid: 8620222
Komrokji RS, Mailloux AW, Chen DT, et al. A phase II multicenter rabbit anti-thymocyte globulin trial in patients with myelodysplastic syndromes identifying a novel model for response prediction. Haematologica. 2014;99(7):1176–83. https://doi.org/10.3324/haematol.2012.083345 .
doi: 10.3324/haematol.2012.083345
pubmed: 24488560
pmcid: 4077078
Haider M, Al Ali N, Padron E, et al. Immunosuppressive therapy: exploring an underutilized treatment option for myelodysplastic syndrome. Clin Lymphoma Myeloma Leuk. 2016;16(Suppl):S44-48. https://doi.org/10.1016/j.clml.2016.02.017 .
doi: 10.1016/j.clml.2016.02.017
pubmed: 27521323
Kittang AO, Kordasti S, Sand KE, et al. Expansion of myeloid derived suppressor cells correlates with number of T regulatory cells and disease progression in myelodysplastic syndrome. OncoImmunology. 2016;5(2): e1062208. https://doi.org/10.1080/2162402X.2015.1062208 .
doi: 10.1080/2162402X.2015.1062208
pubmed: 27057428
Bullock K, Richmond A. Suppressing MDSC recruitment to the tumor microenvironment by antagonizing CXCR2 to enhance the efficacy of immunotherapy. Cancers. 2021;13(24):6293. https://doi.org/10.3390/cancers13246293 .
doi: 10.3390/cancers13246293
pubmed: 34944914
pmcid: 8699249
SX-682 treatment in subjects with myelodysplastic syndrome who had disease progression or are intolerant to prior therapy—full text view—ClinicalTrials.gov. Accessed 25 June 2023. https://clinicaltrials.gov/ct2/show/NCT04245397 .
Kotsianidis I, Bouchliou I, Nakou E, et al. Kinetics, function and bone marrow trafficking of CD4+CD25+FOXP3+ regulatory T cells in myelodysplastic syndromes (MDS). Leukemia. 2009;23(3):510–8. https://doi.org/10.1038/leu.2008.333 .
doi: 10.1038/leu.2008.333
pubmed: 19020538
Kerkhoff N, Bontkes HJ, Westers TM, de Gruijl TD, Kordasti S, van de Loosdrecht AA. Dendritic cells in myelodysplastic syndromes: from pathogenesis to immunotherapy. Immunotherapy. 2013;5(6):621–37. https://doi.org/10.2217/imt.13.51 .
doi: 10.2217/imt.13.51
pubmed: 23725285
Ma L, Delforge M, van Duppen V, et al. Circulating myeloid and lymphoid precursor dendritic cells are clonally involved in myelodysplastic syndromes. Leukemia. 2004;18(9):1451–6. https://doi.org/10.1038/sj.leu.2403430 .
doi: 10.1038/sj.leu.2403430
van Leeuwen-Kerkhoff N, Westers TM, Poddighe PJ, et al. Reduced frequencies and functional impairment of dendritic cell subsets and non-classical monocytes in myelodysplastic syndromes. Haematologica. 2021;107(3):655–67. https://doi.org/10.3324/haematol.2020.268136 .
doi: 10.3324/haematol.2020.268136
pmcid: 8883570
Abaza Y, Zeidan AM. Immune checkpoint inhibition in acute myeloid leukemia and myelodysplastic syndromes. Cells. 2022;11(14):2249. https://doi.org/10.3390/cells11142249 .
doi: 10.3390/cells11142249
pubmed: 35883692
pmcid: 9318025
Yang X, Ma L, Zhang X, Huang L, Wei J. Targeting PD-1/PD-L1 pathway in myelodysplastic syndromes and acute myeloid leukemia. Exp Hematol Oncol. 2022;11(1):11. https://doi.org/10.1186/s40164-022-00263-4 .
doi: 10.1186/s40164-022-00263-4
pubmed: 35236415
pmcid: 8889667
Łuksza M, Riaz N, Makarov V, et al. A neoantigen fitness model predicts tumour response to checkpoint blockade immunotherapy. Nature. 2017;551(7681):517–20. https://doi.org/10.1038/nature24473 .
doi: 10.1038/nature24473
pmcid: 6137806
Alexandrov LB, Nik-Zainal S, Wedge DC, et al. Signatures of mutational processes in human cancer. Nature. 2013;500(7463):415–21. https://doi.org/10.1038/nature12477 .
doi: 10.1038/nature12477
pubmed: 23945592
pmcid: 3776390
Saini SK, Holmberg-Thydén S, Bjerregaard AM, et al. Neoantigen reactive T cells correlate with the low mutational burden in hematological malignancies. Leukemia. 2022;36(11):2734–8. https://doi.org/10.1038/s41375-022-01705-y .
doi: 10.1038/s41375-022-01705-y
pmcid: 9613475
Attermann AS, Bjerregaard AM, Saini SK, Grønbæk K, Hadrup SR. Human endogenous retroviruses and their implication for immunotherapeutics of cancer. Ann Oncol. 2018;29(11):2183–91. https://doi.org/10.1093/annonc/mdy413 .
doi: 10.1093/annonc/mdy413
pubmed: 30239576
Chao MP, Weissman IL, Majeti R. The CD47-SIRPα pathway in cancer immune evasion and potential therapeutic implications. Curr Opin Immunol. 2012;24(2):225–32. https://doi.org/10.1016/j.coi.2012.01.010 .
doi: 10.1016/j.coi.2012.01.010
pubmed: 22310103
pmcid: 3319521
Garcia-Manero G, Erba HP, Sanikommu SR, et al. Evorpacept (ALX148), a CD47-blocking myeloid checkpoint inhibitor, in combination with azacitidine: a phase 1/2 study in patients with myelodysplastic syndrome (ASPEN-02). Blood. 2021;138(Supplement 1):2601. https://doi.org/10.1182/blood-2021-146547 .
doi: 10.1182/blood-2021-146547
Asayama T, Tamura H, Ishibashi M, et al. Functional expression of Tim-3 on blasts and clinical impact of its ligand galectin-9 in myelodysplastic syndromes. Oncotarget. 2017;8(51):88904–17. https://doi.org/10.18632/oncotarget.21492 .
doi: 10.18632/oncotarget.21492
pmcid: 5687656
Brunner AM, Esteve J, Porkka K, et al. Efficacy and safety of sabatolimab (MBG453) in combination with hypomethylating agents (HMAs) in patients (Pts) with very high/high-risk myelodysplastic syndrome (vHR/HR-MDS) and acute myeloid leukemia (AML): final analysis from a phase Ib study. Blood. 2021;138(Supplement 1):244. https://doi.org/10.1182/blood-2021-146039 .
doi: 10.1182/blood-2021-146039
Gallazzi M, Ucciero MAM, Faraci DG, et al. New frontiers in monoclonal antibodies for the targeted therapy of acute myeloid leukemia and myelodysplastic syndromes. Int J Mol Sci. 2022;23(14):7542. https://doi.org/10.3390/ijms23147542 .
doi: 10.3390/ijms23147542
pubmed: 35886899
pmcid: 9320300
Testa U, Pelosi E, Castelli G. CD123 as a therapeutic target in the treatment of hematological malignancies. Cancers. 2019;11(9):1358. https://doi.org/10.3390/cancers11091358 .
doi: 10.3390/cancers11091358
pubmed: 31547472
pmcid: 6769702
Uckun FM, Lin TL, Mims AS, et al. A clinical phase 1B study of the CD3xCD123 bispecific antibody APVO436 in patients with relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome. Cancers. 2021;13(16):4113. https://doi.org/10.3390/cancers13164113 .
doi: 10.3390/cancers13164113
pubmed: 34439266
pmcid: 8394899
Swaminathan M, Cortes JE. Update on the role of gemtuzumab-ozogamicin in the treatment of acute myeloid leukemia. Ther Adv Hematol. 2023;14:20406207231154708. https://doi.org/10.1177/20406207231154708 .
doi: 10.1177/20406207231154708
pubmed: 36845850
pmcid: 9943952
Daver N, Kantarjian H, Ravandi F, et al. A phase II study of decitabine and gemtuzumab ozogamicin in newly diagnosed and relapsed acute myeloid leukemia and high-risk myelodysplastic syndrome. Leukemia. 2016;30(2):268–73. https://doi.org/10.1038/leu.2015.244 .
doi: 10.1038/leu.2015.244
pubmed: 26365212
Senapati J, Almanza EH, Kadia TM, et al. Updated results of CPX-351 in combination with gemtuzumab ozogamicin (GO) in relapsed refractory (R/R) acute myeloid leukemia (AML) and post-hypomethylating agent (Post-HMA) failure high-risk myelodysplastic syndrome (HR-MDS). Blood. 2022;140(Supplement 1):9050–3. https://doi.org/10.1182/blood-2022-171011 .
doi: 10.1182/blood-2022-171011
Warlick ED, Weisdorf DJ, Vallera DA, et al. GTB-3550 TriKE™ for the treatment of high-risk myelodysplastic syndromes (mds) and refractory/relapsed acute myeloid leukemia (AML) safely drives natural killer (NK) cell proliferation at initial dose cohorts. Blood. 2020;136:7–8. https://doi.org/10.1182/blood-2020-136398 .
doi: 10.1182/blood-2020-136398
Wei Y, Dimicoli S, Bueso-Ramos C, et al. Toll-like receptor alterations in myelodysplastic syndrome. Leukemia. 2013;27(9):1832–40. https://doi.org/10.1038/leu.2013.180 .
doi: 10.1038/leu.2013.180
pubmed: 23765228
pmcid: 4011663
Garcia-Manero G, Jabbour EJ, Konopleva MY, et al. A clinical study of tomaralimab (OPN-305), a toll-like receptor 2 (TLR-2) antibody, in heavily pre-treated transfusion dependent patients with lower risk myelodysplastic syndromes (MDS) that have received and failed on prior hypomethylating agent (HMA) therapy. Blood. 2018;132:798. https://doi.org/10.1182/blood-2018-99-119805 .
doi: 10.1182/blood-2018-99-119805
Tavakkoli M, Chung SS, Park CY. Do preclinical studies suggest that CD99 is a potential therapeutic target in acute myeloid leukemia and the myelodysplastic syndromes? Expert Opin Ther Targets. 2018;22(5):381–3. https://doi.org/10.1080/14728222.2018.1464140 .
doi: 10.1080/14728222.2018.1464140
pubmed: 29637789
Barreyro L, Will B, Bartholdy B, et al. Overexpression of IL-1 receptor accessory protein in stem and progenitor cells and outcome correlation in AML and MDS. Blood. 2012;120(6):1290–8. https://doi.org/10.1182/blood-2012-01-404699 .
doi: 10.1182/blood-2012-01-404699
pubmed: 22723552
pmcid: 3418722
Saxena M, van der Burg SH, Melief CJM, Bhardwaj N. Therapeutic cancer vaccines. Nat Rev Cancer. 2021;21(6):360–78. https://doi.org/10.1038/s41568-021-00346-0 .
doi: 10.1038/s41568-021-00346-0
pubmed: 33907315
Melief CJM, van Hall T, Arens R, Ossendorp F, van der Burg SH. Therapeutic cancer vaccines. J Clin Investig. 2015;125(9):3401–12. https://doi.org/10.1172/JCI80009 .
doi: 10.1172/JCI80009
pubmed: 26214521
pmcid: 4588240
Gubin MM, Artyomov MN, Mardis ER, Schreiber RD. Tumor neoantigens: building a framework for personalized cancer immunotherapy. J Clin Investig. 2015;125(9):3413–21. https://doi.org/10.1172/JCI80008 .
doi: 10.1172/JCI80008
pmcid: 4588307
Xie N, Shen G, Gao W, Huang Z, Huang C, Fu L. Neoantigens: promising targets for cancer therapy. Signal Transduct Target Ther. 2023;8(1):1–38. https://doi.org/10.1038/s41392-022-01270-x .
doi: 10.1038/s41392-022-01270-x
pubmed: 36588107
pmcid: 9805914
Liu J, Fu M, Wang M, Wan D, Wei Y, Wei X. Cancer vaccines as promising immuno-therapeutics: platforms and current progress. J Hematol OncolJ Hematol Oncol. 2022;15:28. https://doi.org/10.1186/s13045-022-01247-x .
doi: 10.1186/s13045-022-01247-x
Bijker MS, van den Eeden SJF, Franken KL, Melief CJM, van der Burg SH, Offringa R. Superior induction of anti-tumor CTL immunity by extended peptide vaccines involves prolonged, DC-focused antigen presentation. Eur J Immunol. 2008;38(4):1033–42. https://doi.org/10.1002/eji.200737995 .
doi: 10.1002/eji.200737995
pubmed: 18350546
Bloch O, Parsa AT. Heat shock protein peptide complex-96 (HSPPC-96) vaccination for recurrent glioblastoma: a phase II, single arm trial. Neuro Oncol. 2014;16(5):758–9. https://doi.org/10.1093/neuonc/nou054 .
doi: 10.1093/neuonc/nou054
pubmed: 24729070
pmcid: 3984567
Steinhagen F, Kinjo T, Bode C, Klinman DM. TLR-based immune adjuvants. Vaccine. 2011;29(17):3341–55. https://doi.org/10.1016/j.vaccine.2010.08.002 .
doi: 10.1016/j.vaccine.2010.08.002
pubmed: 20713100
Liu J, Miao L, Sui J, Hao Y, Huang G. Nanoparticle cancer vaccines: design considerations and recent advances. Asian J Pharm Sci. 2020;15(5):576–90. https://doi.org/10.1016/j.ajps.2019.10.006 .
doi: 10.1016/j.ajps.2019.10.006
pubmed: 33193861
Asada H, Kishida T, Hirai H, et al. Significant antitumor effects obtained by autologous tumor cell vaccine engineered to secrete interleukin (IL)-12 and IL-18 by means of the EBV/lipoplex. Mol Ther J Am Soc Gene Ther. 2002;5(5 Pt 1):609–16. https://doi.org/10.1006/mthe.2002.0587 .
doi: 10.1006/mthe.2002.0587
Wimmers F, Schreibelt G, Sköld AE, Figdor CG, De Vries IJM. Paradigm shift in dendritic cell-based immunotherapy: from in vitro generated monocyte-derived DCs to naturally circulating DC subsets. Front Immunol. 2014;5:165. https://doi.org/10.3389/fimmu.2014.00165 .
doi: 10.3389/fimmu.2014.00165
pubmed: 24782868
pmcid: 3990057
Ad G, Pg C, den Bj VE. Integrating next-generation dendritic cell vaccines into the current cancer immunotherapy landscape. Trends Immunol. 2017;38(8):25. https://doi.org/10.1016/j.it.2017.05.006 .
doi: 10.1016/j.it.2017.05.006
Lim M, Badruddoza AZM, Firdous J, et al. Engineered nanodelivery systems to improve DNA vaccine technologies. Pharmaceutics. 2020;12(1):30. https://doi.org/10.3390/pharmaceutics12010030 .
doi: 10.3390/pharmaceutics12010030
pubmed: 31906277
pmcid: 7022884
Morse MA, Gwin WR, Mitchell DA. Vaccine therapies for cancer: then and now. Target Oncol. 2021;16(2):121–52. https://doi.org/10.1007/s11523-020-00788-w .
doi: 10.1007/s11523-020-00788-w
pubmed: 33512679
pmcid: 7845582
Anguille S, Van de Velde AL, Smits EL, et al. Dendritic cell vaccination as postremission treatment to prevent or delay relapse in acute myeloid leukemia. Blood. 2017;130(15):1713–21. https://doi.org/10.1182/blood-2017-04-780155 .
doi: 10.1182/blood-2017-04-780155
pubmed: 28830889
pmcid: 5649080
DEC-205/NY-ESO-1 fusion protein CDX-1401, poly ICLC, decitabine, and nivolumab in treating patients with myelodysplastic syndrome or acute myeloid leukemia—Full Text View - ClinicalTrials.gov. Accessed 25 June 2023. https://clinicaltrials.gov/ct2/show/NCT03358719 .
Cellular immunotherapy for patients with high risk myelodysplastic syndromes and acute myeloid leukemia - Full Text View - ClinicalTrials.gov. Accessed 25 June 2023. https://clinicaltrials.gov/ct2/show/NCT03083054 .
SL-401 in Combination With Azacitidine or Azacitidine/Venetoclax in Acute Myeloid Leukemia (AML), High-Risk Myelodysplastic Syndrome (MDS) or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) - Full Text View - ClinicalTrials.gov. Accessed 25 June 2023. https://clinicaltrials.gov/ct2/show/NCT03113643 .
Hypomemylating agents combination with eDC therapy for elderly patients with myelodysplastic syndrome—full text view—ClinicalTrials.gov. Accessed 25 June 2023. https://clinicaltrials.gov/ct2/show/NCT04999943 .