SBIR CONCEPT AWARD-NANOSPONGE LINKED DCLK1 INHIBITORS FOR THE TARGETED TREATMENT OF CHOLANGIOCARCINOMA

The proposed product is designed to meet a high unmet medical need for osteosarcoma (OS), a rare pediatric cancer. OS accounts for 2% of childhood cancers (60% of cases occurring in patients who are 10-20 years of age).(1) OS 5-year survival is 70% for non-metastasized disease, 30% for metastasized primarily as a result of its highly metastatic and drug-resistant nature. Cytotoxic chemotherapy and limb amputation is the established treatment for patients. All stages of disease have a huge unmet need. Upon successful completion of this proposal, DCLK1 LLC will have significantly de-risked a new strategy using a novel delivery system and novel compounds for the treatment of OS.The groundbreaking rationale of using the proprietary drug delivery system coupled to novel small molecules is improved solubility, targeting, cellular uptake, and biodegradability, which makes for a potent vector for small molecule delivery. The company will have synthesized 18 new drug delivery/small molecule combinations and will have evaluated lead compounds for preliminary in vivo proof of concept. The proposed product will be structurally and mechanistically novel compared to drug products currently approved or in clinical development, and it has the potential to be transformative to patients as single or combination therapy.