Research advances in the targeted therapy and immunotherapy of Wilms tumor: a narrative review

Wilms tumor is the most common pediatric abdominal solid tumor, and its treatment has been a focus of research. For now, the 5-year survival rate of children with Wilms tumor is about 90%. It is difficult to make further progress simply by the improvement of the existing treatments (multi-modal therapy). Therefore, targeted therapy and immunotherapy which have high accuracy and few side effects began to be considered for the treatment of Wilms tumor. At present, though targeted therapy and immunotherapy are rarely used in the treatment of Wilms tumor except in clinical trials, there are dozens of clinical trials research them around the world. The sites in targeted therapy research are mainly focused on insulin-like growth factor 2 (IGF2) pathway, anti-angiogenesis, phosphoinositide 3-kinase (PI3K)/protein kinase B (AKT) signaling pathway, and some miRNAs, etc. And there are three types of study in Wilms tumor immunotherapy, which are inhibition of the COX-2 pathway, chimeric antigen receptor (CAR)-T cell therapy, and multi-tumor associated antigen (TAA)-specific cytotoxic T lymphocytes (CTL) therapy. Among them, the phase I clinical trial of multi-TAA-specific CTL (MTAA-CTL) therapy has been completed, and the results are very satisfactory. In this narrative review, we review the basic research and relevant clinical research on targeted therapy and immunotherapy for Wilms tumor.

Automated summary

Wilms tumor is a common pediatric abdominal solid tumor, with current treatment mainly focusing on multi-modal therapy. While targeted therapy and immunotherapy show potential in clinical trials, they are less commonly used in Wilms tumor treatment. There are dozens of clinical trials worldwide researching these new therapeutic approaches.