CAR-T cell therapy in AML: recent progress and future perspectives

DiNardo CD, Wei AH. How I treat acute myeloid leukemia in the era of new drugs. Blood. 2020;135:85–96.

Article  PubMed  Google Scholar 

Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, et al. Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia. N Engl J Med. 2018;378:439–48.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Raje N, Berdeja J, Lin Y, Siegel D, Jagannath S, Madduri D, et al. Anti-BCMA CAR T-cell therapy bb2121 in relapsed or refractory multiple myeloma. N Engl J Med. 2019;380:1726–37.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Schultz LM, Baggott C, Prabhu S, Pacenta HL, Phillips CL, Rossoff J, et al. Disease burden affects outcomes in pediatric and young adult B-cell lymphoblastic leukemia after commercial tisagenlecleucel: a pediatric real-world chimeric antigen receptor consortium report. J Clin Oncol. 2022;40:945–55.

Article  CAS  PubMed  Google Scholar 

Kantarjian H, Stein A, Gökbuget N, Fielding AK, Schuh AC, Ribera JM, et al. Blinatumomab versus chemotherapy for advanced acute lymphoblastic leukemia. N Engl J Med. 2017;376:836–47.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Kantarjian HM, DeAngelo DJ, Stelljes M, Liedtke M, Stock W, Gökbuget N, et al. Inotuzumab ozogamicin versus standard of care in relapsed or refractory acute lymphoblastic leukemia: final report and long-term survival follow-up from the randomized, phase 3 INO-VATE study. Cancer. 2019;125:2474–87.

Article  CAS  PubMed  Google Scholar 

Atilla E, Benabdellah K. The black hole: CAR T cell therapy in AML. Cancers (Basel). 2023;15:2713.

Article  CAS  PubMed  Google Scholar 

Fetsch V, Zeiser R. Chimeric antigen receptor T cells for acute myeloid leukemia. Eur J Haematol. 2024;112:28–35.

Article  CAS  PubMed  Google Scholar 

Ritchie DS, Neeson PJ, Khot A, Peinert S, Tai T, Tainton K, et al. Persistence and efficacy of second generation CAR T cell against the LeY antigen in acute myeloid leukemia. Mol Ther. 2013;21:2122–9.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Wang QS, Wang Y, Lv HY, Han QW, Fan H, Guo B, et al. Treatment of CD33-directed chimeric antigen receptor-modified T cells in one patient with relapsed and refractory acute myeloid leukemia. Mol Ther. 2015;23:184–91.

Article  CAS  PubMed  Google Scholar 

Tambaro FP, Singh H, Jones E, Rytting M, Mahadeo KM, Thompson P, et al. Autologous CD33-CAR-T cells for treatment of relapsed/refractory acute myelogenous leukemia. Leukemia. 2021;35:3282–6.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Sallman DA, Elmariah H, Sweet K, Mishra A, Cox CA, Chakaith M, et al. Phase 1/1b safety study of Prgn-3006 Ultracar-T in patients with relapsed or refractory CD33-positive acute myeloid leukemia and HIGHER risk myelodysplastic syndromes. Blood. 2022;140(suppl 1):10313–5.

Article  Google Scholar 

Shah NN, Tasian SK, Kohler ME, Hsieh EM, Baumeister SHC, Summers C, et al. CD33 CAR T-cells (CD33CART) for children and young adults with relapsed/refractory AML: dose-escalation results from a Phase I/II multicenter trial. Blood. 2023;142(suppl 1):771.

Article  Google Scholar 

Curio S, Jonsson G, Marinović S. A summary of current NKG2D-based CAR clinical trials. Immunother Adv. 2021;1:ltab018.

Article  PubMed  PubMed Central  Google Scholar 

Baumeister SH, Murad J, Werner L, Daley H, Trebeden-Negre H, Gicobi JK, et al. Phase I trial of autologous CAR T cells targeting NKG2D ligands in patients with AML/MDS and multiple myeloma. Cancer Immunol Res. 2019;7:100–12.

Article  CAS  PubMed  Google Scholar 

Sallman DA, Kerre T, Havelange V, Poiré X, Lewalle P, Wang ES, et al. CYAD-01, an autologous NKG2D-based CAR T-cell therapy, in relapsed or refractory acute myeloid leukaemia and myelodysplastic syndromes or multiple myeloma (THINK): haematological cohorts of the dose escalation segment of a phase 1 trial. Lancet Haematol. 2023;10:e191–202.

Article  CAS  PubMed  Google Scholar 

Sallman DA, Brayer J, Sagatys EM, Lonez C, Breman E, Agaugué S, et al. NKG2D-based chimeric antigen receptor therapy induced remission in a relapsed/refractory acute myeloid leukemia patient. Haematologica. 2018;103:e424–6.

Article  PubMed  PubMed Central  Google Scholar 

Sauter CS, Borthakur G, Mountjoy L, Rotta M, Liu H, Murthy HS, et al. A Phase 1 study of NKX101, a chimeric antigen receptor natural killer (CAR-NK) cell therapy, with fludarabine and cytarabine in patients with acute myeloid leukemia. Blood. 2023;142(suppl 1):2097.

Article  Google Scholar 

Mardiros A, Dos Santos C, McDonald T, Brown CE, Wang X, Budde LE, et al. T cells expressing CD123-specific chimeric antigen receptors exhibit specific cytolytic effector functions and antitumor effects against human acute myeloid leukemia. Blood. 2013;122:3138–48.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Luo Y, Chang L-J, Hu Y, Dong L, Wei G, Huang H. First-in-man CD123-specific chimeric antigen receptor-modified T cells for the treatment of refractory acute myeloid leukemia. Blood. 2015;126:3778.

Article  Google Scholar 

Budde L, Song JY, Kim Y, Blanchard S, Wagner J, Stein AS, et al. Remissions of acute myeloid leukemia and blastic plasmacytoid dendritic cell neoplasm following treatment with CD123-specific CAR T cells: a first-in-human clinical trial. Blood. 2017;130(suppl 1):811.

Article  Google Scholar 

Yao S, Jianlin C, Yarong L, Botao L, Qinghan W, Hongliang F, et al. Donor-derived CD123-targeted CAR T cell serves as a RIC regimen for haploidentical transplantation in a patient with FUS-ERG+ AML. Front Oncol. 2019;9:1358.

Article  PubMed  PubMed Central  Google Scholar 

Naik S, Madden RM, Lipsitt A, Lockey T, Bran J, Rubnitz JE, et al. Safety and anti-leukemic activity of CD123-CAR T cells in pediatric patients with AML: preliminary results from a Phase 1 trial. Blood. 2022;140(suppl 1):4584–5.

Article  Google Scholar 

Sallman DA, DeAngelo DJ, Pemmaraju N, Dinner S, Gill S, Olin RL, et al. Ameli-01: a phase I trial of UCART123v1.2, an anti-CD123 allogeneic CAR-T cell product, in adult patients with relapsed or refractory (R/R) CD123+ acute myeloid leukemia (AML). Blood. 2022;140(suppl 1):2371–84.

Article  Google Scholar 

Peters DT, Savoldo B, Grover NS. Building safety into CAR-T therapy. Hum Vaccin Immunother. 2023;19:2275457.

Article  PubMed  PubMed Central  Google Scholar 

Loff S, Dietrich J, Meyer JE, Riewaldt J, Spehr J, von Bonin M, et al. Rapidly switchable universal CAR-T cells for treatment of CD123-positive leukemia. Mol Ther Oncolytics. 2020;17:408–20.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Wermke M, Kraus S, Ehninger A, Bargou RC, Goebeler ME, Middeke JM, et al. Proof of concept for a rapidly switchable universal CAR-T platform with UniCAR-T-CD123 in relapsed/refractory AML. Blood. 2021;137:3145–8.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Ehninger G, Kraus S, Sala E, Metzelder SK, Vucinic V, Fiedler W, et al. Phase 1 dose escalation study of the rapidly switchable universal CAR-T therapy Unicar-T-CD123 in relapsed/refractory AML. Blood. 2022;140(suppl 1):2367–8.

Article  Google Scholar 

Wermke M, Metzelder S, Kraus S, Sala E, Vucinic V, Fiedler W, et al. Updated results from a phase I dose escalation study of the rapidly-switchable universal CAR-T therapy UniCAR-T-CD123 in relapsed/refractory AML. Blood. 2023;142(suppl 1):3465.

Article  Google Scholar 

Tashiro H, Sauer T, Shum T, Parikh K, Mamonkin M, Omer B, et al. Treatment of acute myeloid leukemia with T cells expressing chimeric antigen receptors directed to C-type lectin-like Molecule 1. Mol Ther. 2017;25:2202–13.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Liu F, Pinz K, Ma Y, Wada M, Chen K, Ma G, et al. First-in-human CLL1-CD33 compound CAR T cells as a two-pronged approach for the treatment of refractory acute myeloid leukemia. HemaSphere. 2018;2:29.

Google Scholar 

留言 (0)

沒有登入
gif