Pediatric Tumor Targeting

Developing novel targeted therapies to improve treatment of pediatric solid tumors





Congratulations to Dzhangar and Andrea for their successful PhD thesis defenses

Pediatric sarcomas account for about 15% of pediatric cancers. These sarcomas often display a highly aggressive behavior with early tendency for development of metastasis. Although current treatment regimens, including surgery and chemotherapy, can achieve good response rates, the relapse rate is generally high with extremely poor prognosis.

The significant toxicity associated with current chemotherapies generates late side effects, a major complication in pediatric oncology.

Rhabdomyosarcoma (RMS) is the most common soft-tissue sarcoma in children. The two main distinct histological groups with prognostic significance are embryonal RMS (ERMS) in 55% of patients, and alveolar RMS (ARMS) in 20% of patients with a five-year event-free survival of 70% for ERMS and 23% for ARMS.

ARMS is generally the most aggressive subtype with the poorest prognosis and ERMS presenting with metastasis at diagnosis has only 30% five-year event-free survival.

More effective and less toxic therapies are needed for pediatric sarcomas