Abstract
Rhabdomyosarcomas (RMS) are the most common soft tissue sarcomas of childhood. Despite intensified, multimodality treatments, the overall survival for high-risk populations has remained at 5% to 20% over the last decades. Current treatments must be designed to answer the needs of pediatric patients, taking into account the differences between kids and adults, and particularly intended for children. In order to do accomplished this goal, we have developed two different and complementary multifunctional nanomaterials, namely porphyrin-based mesoporous organosilica nanoparticles (PMOsPOR-NPs) and porous silicon nanoparticles (pSiNPs), which have been modified by grafting therapeutic molecules on their surfaces. The particular structure of PMOsPOR-NPs, with a large mesoporosity (5-80 nm) and a framework consisting of J aggregates of porphyrins, allows their loading with different cargoes and their use in two-photon excitation (TPE). pSiNPs have been used as photosensitizer in two-photon excitation photodynamic therapy because they are capable of absorbing light in the near-infrared generating 1O2, and due to their large specific surface, they could load different molecules inside their pores as well as on their surface. We will use a triple approach based on the combination of these nanomaterials with targeting molecules and photodynamic therapy or/and gene delivery. The active targeting using two therapeutic molecules may have multiple benefits for cancer therapy, leading to novel formulations that have been capable of performing imaging, two-photon excitation photodynamic therapy (TPE-PDT), and TPE-induced siRNA delivery.Keywords: Pediatric cancer, target therapies, nanoparticles.