Borch Department of Medicinal Chemistry and Molecular Pharmacology, Purdue University, West Lafayette, IN, USA.
Department of Internal Medicine, University of Iowa, Iowa City, IA, USA.
Commun Biol. 2024 Aug 9;7(1):963. doi: 10.1038/s42003-024-06602-x.
Limiting cellular plasticity is of key importance for the therapeutic targeting of metastatic breast cancer (MBC). Fibroblast growth receptor (FGFR) is a critical molecule in cellular plasticity and potent inhibitors of FGFR enzymatic activity have been developed, but kinase independent functions for this receptor also contribute to MBC progression. Herein, we evaluated several FGFR inhibitors and find that while FGFR-targeted kinase inhibitors are effective at blocking ligand-induced cell growth, dormant cells persist eventually giving rise to MBC progression. To more broadly target FGFR and cellular plasticity, we examined the FGFR1 proximal promoter, and found several sequences with potential to form G-quadruplex secondary structures. Circular dichroism was used to verify formation of G-quadruplex in the FGFR1 proximal promoter. Importantly, use of the clinical G-quadruplex-stabilizing compound, CX-5461, stabilized the FGFR1 G-quadruplex structures, blocked the transcriptional activity of the FGFR1 proximal promoter, decreased FGFR1 expression, and resulted in potent inhibition of pulmonary tumor formation. Overall, our findings suggest G-quadruplex-targeted compounds could be a potential therapeutic strategy to limit the cellular plasticity of FGFR1 overexpressing MBC.
限制细胞可塑性对于转移性乳腺癌 (MBC) 的治疗靶向至关重要。成纤维细胞生长因子受体 (FGFR) 是细胞可塑性的关键分子,已经开发出 FGFR 酶活性的有效抑制剂,但该受体的激酶非依赖性功能也有助于 MBC 的进展。在此,我们评估了几种 FGFR 抑制剂,发现虽然 FGFR 靶向激酶抑制剂可有效阻断配体诱导的细胞生长,但休眠细胞最终仍会持续存在,导致 MBC 的进展。为了更广泛地靶向 FGFR 和细胞可塑性,我们检查了 FGFR1 近端启动子,并发现了几个具有形成 G-四链体二级结构潜力的序列。圆二色性用于验证 FGFR1 近端启动子中 G-四链体的形成。重要的是,使用临床 G-四链体稳定化合物 CX-5461 稳定了 FGFR1 G-四链体结构,阻断了 FGFR1 近端启动子的转录活性,降低了 FGFR1 的表达,并导致肺肿瘤形成的强烈抑制。总的来说,我们的研究结果表明,G-四链体靶向化合物可能是限制 FGFR1 过表达 MBC 细胞可塑性的潜在治疗策略。