Stem cell‐based platforms for cancer therapy: Biological mechanisms, therapeutic applications and clinical translation

Background Stem cell‐based strategies are being investigated as therapeutic, supportive and research‐enabling platforms in oncology through context‐dependent tumour‐directed migration, predominantly demonstrated in experimental models, localized therapeutic delivery, immune and microenvironmental modulation, and regenerative functions. However, stem cell platforms differ substantially in their biological properties, therapeutic roles, safety profiles and clinical maturity, and their value cannot be considered independently of tumour context. Methods This narrative review critically examines the major stem cell platforms investigated in oncology, including mesenchymal stem/stromal cells (MSCs), haematopoietic stem cells (HSCs), neural stem cells (NSCs), induced pluripotent stem cells (iPSCs) and embryonic stem cells (ESCs), while distinguishing nonmalignant therapeutic and enabling platforms from malignant cancer stem cells (CSCs), which contribute to treatment resistance and relapse. We compare their therapeutic mechanisms, cancer‐specific applications, translational limitations and levels of clinical evidence across solid and haematological malignancies. Results HSC transplantation is established for selected haematological malignancies, whereas the approved MSC product remestemcel‐L‐rknd treats paediatric steroid‐refractory acute graft‐versus‐host disease as supportive post‐transplant care, not direct anticancer therapy. Tumour‐directed MSC and NSC strategies, engineered HSC immunotherapies, pluripotent stem cell‐derived therapeutic products, and stem cell‐derived extracellular vesicles remain investigational for cancer treatment, although selected approaches have reached early‐phase clinical evaluation. iPSCs and ESCs also provide enabling platforms for cancer modelling, drug evaluation and generation of differentiated cell products. Across platforms, clinical development is constrained by context‐dependent biological activity, route‐dependent biodistribution and uncertain tumour selectivity, product heterogeneity, limited persistence, safety concerns, manufacturing variability and insufficient evidence of durable clinical efficacy. Conclusion Successful translation requires product‐specific validation of therapeutic delivery, safety and clinical benefit. Collectively, current evidence indicates that no single stem cell platform is universally optimal for cancer therapy; rather, therapeutic value is determined by the interaction among platform biology, tumour context, product design and level of clinical evidence.