Head and neck squamous cell carcinoma (HNSCC) is the sixth most prevalent cancer worldwide with about 600,000 new cases diagnosed in the last year. The 5-year survival rate of HNSCC has remained stagnant despite advances in the clinical management of this disease. Understanding the molecular pathways that lead to aggressive HNSCC is crucial to identify new “druggable” targets for anti-cancer drug development. Recently, our laboratory showed that miR-107 is significantly reduced in primary HNSCC tumors and HNSCC cell lines. Ectopic expression of miR-107 in CAL-27 HNSCC cells significantly inhibited cell proliferation, DNA replication, clonogenic survival, and tumor growth in nude mice. Our data demonstrate the potential application of miR-107 as a novel anti-cancer therapeutic for HNSCC. In this study, we determine the efficacy of miR-107 encapsulated nanoparticles (NP-miR-107) in a xenograft model of HNSCC. NP-miR-107 was successfully formulated by ethanol injection method. The particle size of NP-miR-107 was 47.8 ± 3.5 nm and zeta potential was +12.1 mV. NP-miR-107 significantly retarded (57.6% inhibition, p<0.02) tumor growth of CAL27 HNSCC tumors compared to NP-miR-control; tumor volume of 340.6 ± 51.6 mm3 and 804.1 ± 181.6 mm3 for the NP-miR-107 group and the NP-miR-control group, respectively. Moreover, Kaplan-Meir analysis showed a survival advantage for the NP-miR-107 treatment group (p=0.039). Our results demonstrate that our nanoparticles are an effective carrier approach to deliver therapeutic miRs to tumors.

Citation Format: {Authors}. {Abstract title} [abstract]. In: Proceedings of the 102nd Annual Meeting of the American Association for Cancer Research; 2011 Apr 2-6; Orlando, FL. Philadelphia (PA): AACR; Cancer Res 2011;71(8 Suppl):Abstract nr 1153. doi:10.1158/1538-7445.AM2011-1153