B7-H3 COME POTENZIALE BERSAGLIO PER L'IMMUNOTERAPIA BASATA SUI CAR CONTRO I SARCOMI PEDIATRICI DELL’OSSO E DEI TESSUTI MOLLI

Bone and soft tissue sarcomas (BSTS) are rare cancers, accounting for approximately 12% of pediatric malignancies. Despite advances in multimodal therapy, the prognosis for children with high-grade, refractory, or metastatic BSTS remains poor, highlighting the urgent need for more effective and less toxic treatments. Identifying tumor-specific chimeric antigen receptor (CAR) target molecules is key to successful CAR-based therapies, and B7-H3 is an attractive target for pediatric tumors due to its restricted expression and crucial biological roles.

This study aims to explore B7-H3 redirected CAR-CIK immunotherapy for pediatric sarcomas, including osteosarcoma, rhabdomyosarcoma, and other refractory or relapsing tumors. First, we will assess B7-H3 expression in available BSTS cohorts and preclinical models to explore its potential as a biomarker. The anti-sarcoma activity of B7-H3 CAR-CIK lymphocytes will be tested in both in vitro and in vivo BSTS models, including patient-derived and immunocompetent models.

Our goal is to generate proof-of-concept data for translating this approach into clinical applications, potentially benefiting other pediatric solid tumors. The findings will inform the design of future clinical studies, making this research translational with a strong clinical focus.