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

SBIR 第一阶段：针对尤文氏肉瘤的根本原因

• 批准号: 2233404
• 负责人: Ali Ozes
• 金额: $ 27.5万
• 依托单位: ALTAY THERAPEUTICS, INC.
• 依托单位国家: 美国
• 项目类别: Standard Grant
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-15 至 2024-08-31
• 项目状态: 已结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=2233404&HistoricalAwards=false
• 关键词: SBIR; Phase; Targeting; Root; Cause

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.

这个小企业创新研究（SBIR）第一阶段项目的更广泛的影响是通过针对疾病的根本原因来治疗患有尤文肉瘤的儿科患者。使用一种新的方法，新的治疗方法将被开发出来，以特异性地抑制导致尤文肉瘤的蛋白质，从而延长患者的寿命。这种方法将使人们能够理解这种毁灭性疾病的分子基础。重要的是，这项研究的发现可能能够治疗由类似蛋白质驱动的疾病，为几乎没有治疗选择的患者提供新的药物。 该项目的成功结果将导致第一个针对致癌融合蛋白EWS-FLI 1的抑制剂的商业化，以治疗尤文肉瘤。此外，该技术还可以提供有关在前列腺癌和乳腺癌等其他疾病中使用致癌蛋白的有价值的数据。该项目将有助于描述尤文肉瘤的潜在治疗方法。这些目标包括：（1）确定新型抑制剂在一组人细胞中的毒性特征，（2）测量药物对靶蛋白的特异性，和（3）确定先导分子在小鼠中的功效。这项研究的数据将增加对药物融合蛋白的理解，这些药物融合蛋白是许多疾病（如前列腺癌、乳腺癌和尤文氏肉瘤）的有效驱动因素。该奖项反映了NSF的法定使命，并通过使用基金会的知识价值和更广泛的影响审查标准进行评估，被认为值得支持。