SBIR-STTR Award

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.

This Small Business Innovation Research Phase II project will develop targeted nanoparticle delivery systems capable of delivering conventional cytotoxics preferentially to tumor cells. While therapeutic nanoparticles have been introduced into clinical practice (Abraxane(TM) and Doxil(TM)) and others are in development, none have been developed that show tumor target specificity (as opposed to increased affinity) or deliver multiple therapeutic agents (whether conventional cytotoxic or targeted agents). The hypothesizias underlying this project is that targeted delivery of cancer therapy via highly modular and synthetically robust nanoparticles can improve therapeutic response while minimizing systemic toxic effects of therapy for virtually any cancer. In the Phase I study, the innovation of this approach was demonstrated by dramatically reducing the tumor burden in a transgenic mouse model of adult leukemia with our proprietary nanoparticle agents. The technical objectives in this Phase II proposal are designed to refine and optimize the nanoparticle formulation to treat adult leukemia. This Phase II proposal will enable the completion a critical phase of the preclinical studies for the nanoparticle delivery vehicle to anticipate applying for IND status for this new drug with the FDA. The broader impact/commercial potential of this project offers a great opportunity to add value to nanomaterials. The broader market for nanotech-enabled drug-delivery therapeutics was just over $1 billion in 2010 and is projected to mushroom to $136 billion by the year 2021, with a compound annual growth rate (CAGR) estimated at 49.96% over the same period. Within the U.S. alone, there are an estimated 274,930 people living with leukemia. In 2011, it is estimated that 44,600 people (25,320 men and 19,280 women) were diagnosed with leukemia, including 10 times as many adults as children and adolescents. Also in 2011, 21,780 of those already afflicted with leukemia were expected to die from the disease. The American Cancer Society projects even higher figures for 2012, estimating that 47,150 people in the U.S. will be diagnosed with leukemia and that approximately 23,540 patients will die from it. The nanoparticle delivery system proposed here is well positioned to advance the drug delivery field on a scientific level by demonstrating efficient delivery of therapeutics to leukemia cells. Combined with effective tumor delivery this technology could facilitate highly effective, ?personalized? cancer therapy that is not currently possible with routine systemic therapy.