Targeted Therapy in Bone and Soft Tissue Sarcoma in Children and Adolescents

Pediatric soft-tissue and bone sarcomas are a heterogeneous group of tumors of mesenchymal origin which affect approximately 1,500 children in the United States each year. Using multimodal therapy (surgery, radiation, and chemotherapy), the overall 5-year survival rate for children with soft-tissue and bone sarcomas is approximately 60%-70%. However, the prognosis for children with metastatic or recurrent disease is poor; and, furthermore, the improvements in the overall cure rate have slowed. It is highly unlikely that further advances in the treatment of pediatric soft-tissue and bone sarcomas will come from traditional cytotoxic chemotherapy. Based on research advances in understanding the biology of pediatric soft-tissue and bone sarcomas, improved cure rates will likely be driven by new types of treatment which target the specific abnormalities within these tumors. These new targeted therapies may include small molecules, antibodies, or other immunotherapies. This review briefly describes the biology of the major types of pediatric sarcomas, discusses potential targets for new therapy, and highlights some recent and current clinical trials using targeted therapy.