Advances in targeted therapies and emerging strategies for blood cancer treatment

Abstract

Blood cancers, including leukemia, lymphoma, and multiple myeloma, originate within the bone marrow, where the intricate microenvironment presents considerable challenges for conventional therapies such as chemotherapy, immunotherapy, radiotherapy, and hematopoietic stem cell transplantation. These approaches often suffer from poor specificity, low bioavailability, and systemic toxicity, resulting in suboptimal treatment outcomes. In response, significant advances in targeted drug delivery systems, including liposomes, pegylated formulations, and polymeric nanoparticles have been developed to enhance drug stability, prolong circulation time, and improve tumor accumulation while reducing off-target effects. This review provides a comprehensive overview of recent innovations in ligand-directed drug delivery systems for blood cancers. Emphasis is placed on systems functionalized with antibodies, peptides, aptamers, and proteins designed to overcome the barriers of the bone marrow niche and enable selective delivery to malignant cells. Notably, leukemia has emerged as a key model for evaluating these technologies, with promising preclinical and clinical results. However, despite technological progress, critical translational challenges remain. These include biological heterogeneity, variability in target receptor expression, immunogenicity of nanoparticles, and the complexity of scaling multifunctional delivery systems under clinical conditions. Furthermore, current in vitro and in vivo models fail to accurately recapitulate the bone marrow's dynamic physiology, underscoring the need for improved predictive systems. Future perspectives suggest the integration of personalized nanomedicine approaches that adapt to patient-specific genetic profiles and disease states. Additionally, artificial intelligence (AI) and big data analytics are expected to revolutionize delivery optimization, biomarker discovery, and therapy customization. Ultimately, interdisciplinary collaboration is required to bridge the gap between bench and bedside. By addressing current limitations and embracing innovation, the field moves closer to realizing safe, precise, and effective therapies for patients with hematologic malignancies.