SBIR Phase I: Targeting the Root Cause of Ewing’s Sarcoma

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The broader impact of this Small Business Innovation Research (SBIR) Phase I project is to treat pediatric patients affected with Ewing’s Sarcoma by targeting the root cause of the disease. Using a novel approach, new therapeutics will be developed to specifically inhibit the protein that causes Ewing’s Sarcoma and thereby extend the lifespan of patient. The approach will enable understanding of the molecular underpinnings of this devastating disease. Importantly, findings from this study may be able to treat diseases driven by similar proteins, providing new medication for patients with few available treatment options. The successful outcome of the project would lead to the commercialization of the first-in-classinhibitor to target the oncogenic fusion protein, EWS-FLI1, to treat Ewing’s Sarcoma. In addition, the technology could provide valuable data about drugging oncogenic proteins in other disesases such as prostate cancer and breast cancer.This project will aid in characterizing potential therapies for Ewing’s Sarcoma. The goals include: (1) determining the toxicity profile of novel inhibitors across a panel of human cells, (2) measuring the drug specificity to the target protein, and (3) determining the efficacy of the lead molecule in mice. The data from this study will add to the understanding of drugging fusion proteins which are validated drivers of many diseases such as prostate cancer, breast cancer, and Ewing’s Sarcoma.This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.