Leveraging Immunotherapy in Acute Myeloid Leukemia Treatment: Opportunities and Challenges

Acute myeloid leukemia (AML) is a heterogeneous group of malignant clonal disorders originating from hematopoietic stem or progenitor cells. Despite significant advances in understanding its pathogenesis and the development of novel therapeutic agents, the overall prognosis for AML patients remains poor. Immunotherapies have demonstrated remarkable success in other hematologic malignancies and are increasingly being explored in AML. However, their clinical application remains challenging, owing to multiple factors such as limited target specificity, high disease heterogeneity, and the immunosuppressive bone marrow microenvironment (BMME). In this review, we provide a comprehensive overview of recent advances in four major immunotherapeutic strategies for AML: adoptive cell therapy, bispecific antibodies, immune checkpoint modulation, and cytokine-based therapies. We further discuss the key biological and clinical challenges that limit their efficacy, including antigen selection, tumor heterogeneity, and microenvironmental immune suppression, along with emerging strategies to overcome these obstacles. With continued progress in both basic and clinical research, immunotherapy holds great potential to achieve greater specificity, improved safety, and more durable responses, ultimately improving outcomes for patients with AML.