SBIR Phase I: Targeted Nanoparticle Delivery Agent for Treatment of Adult Leukemia

这个小企业创新研究（SBIR）一期项目旨在探索靶向纳米递送系统在成人白血病患者治疗和疗效监测中的应用。与可以选择手术、放射和常规治疗的实体瘤不同，白血病是血液癌症，由于从一开始就是播散性疾病，预后非常差。在这个项目中，靶向和药物填充的纳米颗粒将在细胞培养中进行优化，以有效杀死肿瘤，然后在小鼠模型中使用已建立的成人白血病体内方法进行验证。预计这种靶向纳米递送系统将增加治疗窗口，最大限度地减少全身治疗相关的毒性，与标准治疗方法相比，显示出更高的安全性。该项目的更广泛/商业影响将是提供一种能够提供多种药物分子（亲水性和疏水性）或使用多种组织靶向配体的药物鸡尾酒的技术。该项目的具体重点是成人急性白血病，目前尚无治愈方法，成人生存率仅为30%。高度稳健，可定制，稳定，易于制造，这种粒子输送系统有望对成人白血病的治疗产生重大影响。

This Small Business Innovation Research (SBIR) Phase I project aims to explore the application of a targeted nanodelivery system for both treatment and efficacy monitoring in patients with adult leukemia. Unlike solid tumors, for which surgery, radiation, and conventional treatments are an option, leukemias are cancers of the blood, which have very poor prognosis due to the fact that they are disseminated diseases from inception. In this project, the targeted and drug-filled nanoparticle will be optimized for effective tumor killing in cell culture, followed by validation using established in-vivo methods for adult leukemia in a mouse model. It is expected that this targeted nanodelivery system will increase the therapeutic window and minimize systemic therapy related toxicity, demonstrating the superior margin of safety compared to the standard methods for treatment.The broader/commercial impact of this project will be the potential to provide a technology that is capable of delivering a wide array of drug molecules (hydrophilic and hydrophobic) or drug cocktails using multiple tissue targeting ligands. The specific focus of this project is adult acute leukemia for which there is currently no curative treatment, and the adult survival rate is only 30%. Highly robust, customizable, stable, and easy to manufacture, this particle delivery system is anticipated to make a major impact on the treatment of adult leukemia.