Abstract
Chimeric antigen receptor T-cell (CAR-T) therapy represents a groundbreaking advancement in cancer immunotherapy and has significantly transformed the treatment landscape for hematological malignancies. This innovative adoptive cellular therapy involves the genetic engineering of a patient’s T lymphocytes to express synthetic receptors capable of recognizing specific tumor-associated antigens on malignant cells. By enabling T cells to directly identify and eliminate cancer cells in a major histocompatibility complex-independent manner, CAR-T therapy has emerged as a highly effective therapeutic strategy for patients with relapsed or refractory hematologic cancers. In recent years, CAR-T therapies targeting antigens such as CD19 and B-cell maturation antigen (BCMA) have demonstrated remarkable clinical efficacy in diseases including acute lymphoblastic leukemia, diffuse large B-cell lymphoma, and multiple myeloma. Several CAR-T cell products have received regulatory approval and have shown high overall response rates and durable remissions in heavily pretreated patients who previously had limited treatment options.
Despite these encouraging outcomes, CAR-T therapy is associated with several limitations and safety concerns, including cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, antigen escape, complex manufacturing procedures, and high treatment costs. Ongoing research efforts are focused on improving CAR design, enhancing T-cell persistence, identifying novel tumor targets, and developing universal or allogeneic CAR-T cell platforms. Additionally, emerging strategies such as dual-target CAR-T cells, gene-editing technologies, and combination therapies with immune checkpoint inhibitors are being investigated to further improve therapeutic efficacy and reduce adverse effects. This review provides a comprehensive overview of CAR-T cell therapy, including its mechanism of action, structural design, clinical applications in hematological malignancies, approved CAR-T therapies, treatment-related toxicities, current challenges, and future perspectives in the evolving field of cancer immunotherapy.