Current success in childhood cancer therapy improves the survival rate of patients but unfortunately not all childhood cancers have shown successful survival rates and still many types of childhood cancer continue to have a poor prognosis. Furthermore due to the poor specificity of cancer therapies almost two-third of childhood cancer survivals experience side effects such as secondary cancers, heart or lung damage, infertility or chronic hepatitis. Therefore a reliable, non-toxic high therapeutic window targeted therapy is urgently needed to treat childhood cancer patients. In our study a novel nanoparticle (HPLN) was used for the treatment of childhood ALL and Ewing's sarcoma. The tumor specific monoclonal antibody was conjugated to the PLN into which Doxorubicin was uptaken using active loading method. AntiCD19 and antiCD99 antibodies were used for targeting of ALL and Ewing tumor, respectively. These tumor specific HPLNs effectively inhibit tumor growth in a murine model of ALL and Ewing's sarcoma. Removal of targeting antibody or drug eliminates the antitumor effects, which proves this anticancer effect of HPLN is very specific to the target cancer cells and dependent upon drug. Additionally, no abnormalities in liver and kidney function tests, complete blood counts or pathology of major organs are observed from tail-vein administrations. These data provide strong evidence for the safety and efficacy of this targeted HPLN delivery system of anticancer drugs to childhood cancer.

Citation Format: HyungGyoo Kang, Violette Shahbazian, Jon Nagy, Timothy Triche. Targeted therapy of childhood ALL and Ewing's sarcoma using antiCD19 (antiCD99)-Hybrid Polymerized Liposomal Nanoparticles (HPLN). [abstract]. In: Proceedings of the 104th Annual Meeting of the American Association for Cancer Research; 2013 Apr 6-10; Washington, DC. Philadelphia (PA): AACR; Cancer Res 2013;73(8 Suppl):Abstract nr 2750. doi:10.1158/1538-7445.AM2013-2750