Han David, Spehar Jonathan M, Richardson Dillon S, Leelananda Sumudu, Chakravarthy Prathik, Grecco Samantha, Reardon Jesse, Stover Daniel G, Bennett Chad, Sizemore Gina M, Li Zaibo, Lindert Steffen, Sizemore Steven T
Department of Radiation Oncology, Arthur G. James Comprehensive Cancer Center, The Ohio State University, Columbus, OH 43210, USA.
Anagenex, 20 Maguire Rd. Suite 302, Lexington, MA 02421, USA.
Cancers (Basel). 2024 Aug 31;16(17):3043. doi: 10.3390/cancers16173043.
Breast cancer (BC) is the most frequent cancer and second-leading cause of cancer deaths in women in the United States. While RAS mutations are infrequent in BC, triple-negative (TN) and HER2-positive (HER2+) BC both exhibit increased RAS activity. Here, we tested the RAS effectors RALA and RALB, which are overexpressed in BC, as tractable molecular targets in these subtypes. While analysis of the breast cancer patient sample data suggests that the RALs are associated with poor outcome in both TNBC and HER2+ BC, our in vivo and in vitro experimental findings revealed the RALs to be essential in only the TNBC cell lines. While testing the response of the BC cell lines to the RAL inhibitors RBC8 and BQU57, we observed no correlation between drug efficacy and cell line dependency on RAL expression for survival, suggesting that these compounds kill via off-target effects. Finally, we report the discovery of a new small molecule inhibitor, OSURALi, which exhibits strong RAL binding, effectively inhibits RAL activation, and is significantly more toxic to RAL-dependent TNBC cells than RAL-independent HER2+ and normal cell lines. These results support the RALs as viable molecular targets in TNBC and the further investigation of OSURALi as a therapeutic agent.
乳腺癌(BC)是美国女性中最常见的癌症,也是癌症死亡的第二大原因。虽然RAS突变在乳腺癌中并不常见,但三阴性(TN)和人表皮生长因子受体2阳性(HER2+)乳腺癌均表现出RAS活性增加。在此,我们测试了在乳腺癌中过表达的RAS效应分子RALA和RALB,作为这些亚型中易于处理的分子靶点。虽然对乳腺癌患者样本数据的分析表明,RALs与三阴性乳腺癌(TNBC)和HER2+乳腺癌的不良预后相关,但我们的体内和体外实验结果显示,RALs仅在TNBC细胞系中至关重要。在测试乳腺癌细胞系对RAL抑制剂RBC8和BQU57的反应时,我们观察到药物疗效与细胞系对RAL表达的生存依赖性之间没有相关性,这表明这些化合物通过脱靶效应发挥作用。最后,我们报告发现了一种新的小分子抑制剂OSURALi,它表现出强烈的RAL结合能力,有效抑制RAL激活,并且对依赖RAL的TNBC细胞的毒性明显高于不依赖RAL的HER2+细胞系和正常细胞系。这些结果支持RALs作为TNBC中可行的分子靶点,并支持进一步研究OSURALi作为一种治疗药物。