Sir Run Run Shaw Hospital, Zhejiang University School of Medicine, Hangzhou, China.
National Center for Advancing Translational Sciences (NCATS), NIH, Bethesda, Maryland.
Mol Cancer Ther. 2019 Jul;18(7):1265-1277. doi: 10.1158/1535-7163.MCT-18-1241. Epub 2019 May 15.
Targeting of extrinsic apoptosis pathway by TNF-related apoptosis-inducing ligand (TRAIL) is an attractive approach for cancer therapy. However, two TRAIL drug candidates failed in clinical trials due to lack of efficacy. We identified 17-hydroxy wortmannin (17-HW) in a drug repurposing screen that resensitized TRAIL's response in the resistant colon cancer cells. The deficiency of caspase-8 in drug-resistant cells along with defects in apoptotic cell death was corrected by 17-HW, an inhibitor of PIK3C3-beclin 1 (BECN1) complex and autophagy activity. Further study found that BECN1 significantly increased in the TRAIL-resistant cells, resulting in increased autophagosome formation and enhanced autophagy flux. The extracellular domain (ECD) of BECN1 directly bound to the caspase-8 catalytic subunit (p10), leading to sequestration of caspase-8 in the autophagosome and its subsequent degradation. Inhibition of BECN1 restored the caspase-8 level and TRAIL's apoptotic response in the resistant colon cancer cells. An analysis of 120 colon cancer patient tissues revealed a correlation of a subgroup of patients (30.8%, 37/120) who have high BECN1 level and low caspase-8 level with a poor survival rate. Our study demonstrates that the increased BECN1 accompanied by enhanced autophagy activity is responsible for the TRAIL resistance, and a combination of TRAIL with a PIK3C3-BECN1 inhibitor is a promising therapeutic approach for the treatment of colon cancer.
肿瘤坏死因子相关凋亡诱导配体(TRAIL)靶向细胞外凋亡途径是癌症治疗的一种有吸引力的方法。然而,由于缺乏疗效,两种 TRAIL 药物候选物在临床试验中失败。我们在药物再利用筛选中发现 17-羟基wortmannin(17-HW),它可以使耐药结肠癌细胞对 TRAIL 的反应重新敏感。耐药细胞中 caspase-8 的缺乏以及凋亡细胞死亡的缺陷,通过抑制 PIK3C3-beclin 1(BECN1)复合物和自噬活性的 17-HW 得到纠正。进一步的研究发现,BECN1 在 TRAIL 耐药细胞中显著增加,导致自噬体形成增加和自噬流增强。BECN1 的细胞外结构域(ECD)直接与 caspase-8 催化亚基(p10)结合,导致 caspase-8 被隔离在自噬体中并随后降解。抑制 BECN1 可恢复耐药结肠癌细胞中 caspase-8 的水平和 TRAIL 的凋亡反应。对 120 例结肠癌患者组织的分析表明,有一组(30.8%,37/120)患者具有高 BECN1 水平和低 caspase-8 水平,与生存率降低相关。我们的研究表明,增加的 BECN1 伴随着增强的自噬活性是 TRAIL 耐药的原因,TRAIL 与 PIK3C3-BECN1 抑制剂的联合应用是治疗结肠癌的一种有前途的治疗方法。