Cancer Genetics and Epigenetics, 2025, Vol.13, No.3, 136-144 http://medscipublisher.com/index.php/cge 136 Feature Review Open Access Trends in CAR-T Cell Therapy for Hematologic Malignancies Qiyan Lou, Xiaoying Xu Biotechnology Research Center, Cuixi Academy of Biotechnology, Zhuji, 311800, Zhejiang, China Corresponding author: xiaoying.xu@cuixi.org Cancer Genetics and Epigenetics, 2025, Vol.13, No.3 doi: 10.5376/cge.2025.13.0014 Received: 15 Apr., 2025 Accepted: 22 May, 2025 Published: 13 Jun., 2025 Copyright © 2025 Lou and Xu, This is an open access article published under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Preferred citation for this article: Lou Q.Y., and Xu X.Y., 2025, Trends in CAR-T cell therapy for hematologic malignancies, Cancer Genetics and Epigenetics, 13(3): 136-144 (doi: 10.5376/cge.2025.13.0014) Abstract This study explored the current situation and new trends of chimeric antigen receptor T cells (CAR-T) in the treatment of hematological malignancies. Blood cancers such as acute lymphoblastic leukemia, non-Hodgkin's lymphoma, chronic lymphoblastic leukemia and multiple myeloma pose significant challenges to treatment, especially in cases of recurrence or refractory. Although traditional treatment methods have certain effects, CAR-T therapy is a breakthrough approach. It targets specific tumor antigens (such as CD19 and BCMA) by modifying autologous T cells, thereby achieving effective cytotoxicity that is independent of MHC. In terms of safety, cytokine release syndrome (CRS) and immune effector cell-related neurotoxicity syndrome (ICANS) are the main issues. The coping strategies include the use of tocilizumab and corticosteroids. In the future, the development of multi-target and switchable CAR structures, "off-the-shelf" allogeneic products, and combination therapy with immune checkpoint inhibitors or chemotherapy will be the key directions of innovation. Personalized treatment plans, predictive biomarkers and simplified production processes will be the keys to making CAR-T therapy more accessible and effective. Keywords CAR-T cell therapy; Hematologic malignancies; Multi-target immunotherapy; Antigen escape and T-cell exhaustion; Cytokine release syndrome (CRS) 1 Introduction Hematological malignancies, including acute lymphoblastic leukemia (ALL), non-Hodgkin's lymphoma, chronic lymphoblastic leukemia and multiple myeloma, are a type of cancer with high incidence and mortality rates worldwide. The characteristic of this type of disease is the abnormal and uncontrolled growth of blood or lymphoid cells, which often leads to the deterioration of the condition and poor therapeutic effect, especially for patients with recurrence or difficult-to-cure (Haslauer et al., 2021). Although traditional treatment methods such as chemotherapy, radiotherapy and stem cell transplantation can improve the survival rate of patients, they are often limited due to drug resistance, disease recurrence and serious side effects. This makes the demand for more effective and durable treatment methods still urgent (Chen et al., 2022; Lu and Jiang, 2022; Wang et al., 2023). Chimeric antigen receptor T (CAR-T) cell therapy has greatly changed the treatment situation of hematological malignancies, especially for those patients with recurrent or refractory diseases. By modifying the patient's own T cells to express synthetic receptors targeting tumor-associated antigens (the most typical ones being CD19 and BCMA), CAR-T therapy can precisely and effectively combat tumors without relying on MHC presentation (Haslauer et al., 2021). Since the U.S. Food and Drug Administration (FDA) first approved it for the treatment of B-cell ALL and lymphoma in 2017, CAR-T therapies such as Kymriah, Yescarta and Tecartus, even in patient populations that have undergone extensive pre-treatment, It also demonstrated a relatively high complete response rate and objective response rate (Wang et al., 2023). However, despite these achievements, there are still many problems, including severe toxic reactions (such as cytokine release syndrome, neurotoxicity), antigen escape, short survival time of CAR-T cells, and disease recurrence, etc. This has driven people to continuously study new targets, improve techniques and explore combined treatment strategies (Han et al., 2021; Chen et al., 2022; Lu and Jiang, 2022; Kampouri et al., 2023). This study will explore the incidence and clinical treatment difficulties of CAR-T cell therapy for hematological malignancies, summarize the development history and clinical effects of CAR-T therapy, and at the same time
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