Abramson Family Cancer Research Institute and.
Department of Cancer Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
J Clin Invest. 2018 Apr 2;128(4):1300-1316. doi: 10.1172/JCI95864. Epub 2018 Feb 26.
Myc activation is a primary oncogenic event in many human cancers; however, these transcription factors are difficult to inhibit pharmacologically, suggesting that Myc-dependent downstream effectors may be more tractable therapeutic targets. Here, we show that Myc overexpression induces endoplasmic reticulum (ER) stress and engages the inositol-requiring enzyme 1α (IRE1α)/X-box binding protein 1 (XBP1) pathway through multiple molecular mechanisms in a variety of c-Myc- and N-Myc-dependent cancers. In particular, Myc-overexpressing cells require IRE1α/XBP1 signaling for sustained growth and survival in vitro and in vivo, dependent on elevated stearoyl-CoA-desaturase 1 (SCD1) activity. Pharmacological and genetic XBP1 inhibition induces Myc-dependent apoptosis, which is alleviated by exogenous unsaturated fatty acids. Of note, SCD1 inhibition phenocopies IRE1α RNase activity suppression in vivo. Furthermore, IRE1α inhibition enhances the cytotoxic effects of standard chemotherapy drugs used to treat c-Myc-overexpressing Burkitt's lymphoma, suggesting that inhibiting the IRE1α/XBP1 pathway is a useful general strategy for treatment of Myc-driven cancers.
Myc 的激活是许多人类癌症中的主要致癌事件;然而,这些转录因子在药理学上很难抑制,这表明 Myc 依赖性下游效应物可能是更具治疗潜力的靶点。在这里,我们表明 Myc 的过表达通过多种分子机制在各种 c-Myc 和 N-Myc 依赖性癌症中诱导内质网 (ER) 应激并激活肌醇需求酶 1α (IRE1α)/X 盒结合蛋白 1 (XBP1) 途径。特别是,过表达 Myc 的细胞需要 IRE1α/XBP1 信号通路来维持体外和体内的持续生长和存活,这依赖于升高的硬脂酰辅酶 A 去饱和酶 1 (SCD1) 活性。药理学和基因 XBP1 抑制诱导 Myc 依赖性细胞凋亡,这可以通过外源性不饱和脂肪酸得到缓解。值得注意的是,SCD1 抑制在体内模拟了 IRE1α RNase 活性的抑制。此外,IRE1α 抑制增强了用于治疗过表达 c-Myc 的 Burkitt 淋巴瘤的标准化疗药物的细胞毒性作用,表明抑制 IRE1α/XBP1 途径是治疗 Myc 驱动的癌症的一种有用的通用策略。