Departments of Cellular and Molecular Pharmacology (D.K., S.-M.K., Y.L., M.A.C.), Pharmaceutical Chemistry (M.A.C.), and Bioengineering and Therapeutic Sciences (M.A.C.) and The Liver Center (M.A.C.), University of California San Francisco, San Francisco, California; and Clinical Pharmacology Program, Division of Cardiology, Department of Medicine, Center for Tobacco Control Research and Education, University of California, San Francisco, California (P.J.).
Departments of Cellular and Molecular Pharmacology (D.K., S.-M.K., Y.L., M.A.C.), Pharmaceutical Chemistry (M.A.C.), and Bioengineering and Therapeutic Sciences (M.A.C.) and The Liver Center (M.A.C.), University of California San Francisco, San Francisco, California; and Clinical Pharmacology Program, Division of Cardiology, Department of Medicine, Center for Tobacco Control Research and Education, University of California, San Francisco, California (P.J.)
Mol Pharmacol. 2019 Nov;96(5):641-654. doi: 10.1124/mol.119.117069. Epub 2019 Sep 6.
The hepatic endoplasmic reticulum (ER)-anchored monotopic proteins, cytochromes P450 (P450s), are enzymes that metabolize endobiotics (physiologically active steroids and fatty acids), as well as xenobiotics including therapeutic/chemotherapeutic drugs, nutrients, carcinogens, and toxins. Alterations of hepatic P450 content through synthesis, inactivation, or proteolytic turnover influence their metabolic function. P450 proteolytic turnover occurs via ER-associated degradation (ERAD) involving ubiquitin (Ub)-dependent proteasomal degradation (UPD) as a major pathway. UPD critically involves P450 protein ubiquitination by E2/E3 Ub-ligase complexes. We have previously identified the ER-polytopic gp78/AMFR (autocrine motility factor receptor) as a relevant E3 in CYP3A4, CYP3A23, and CYP2E1 UPD. We now document that liver-conditional genetic ablation of gp78/AMFR in male mice disrupts P450 ERAD, resulting in statistically significant stabilization of Cyp2a5 and Cyp2c, in addition to that of Cyp3a and Cyp2e1. More importantly, we establish that such stabilization is of the functionally active P450 proteins, leading to corresponding statistically significant enhancement of their drug-metabolizing capacities. Our findings, with clinically relevant therapeutic drugs (nicotine, coumarin, chlorzoxazone, and acetaminophen) and the prodrug (tamoxifen) as P450 substrates, reveal that P450 ERAD disruption could influence therapeutic drug response and/or toxicity, warranting serious consideration as a potential source of clinically relevant drug-drug interactions (DDIs). Because gp78/AMFR is not only an E3 Ub-ligase, but also a cell-surface prometastatic oncogene that is upregulated in various malignant cancers, our finding that hepatic gp78/AMFR knockout can enhance P450-dependent bioactivation of relevant cancer chemotherapeutic prodrugs is of therapeutic relevance and noteworthy in prospective drug design and development. SIGNIFICANCE STATEMENT: The cell-surface and ER transmembrane protein gp78/AMFR, a receptor for the prometastatic autocrine motility factor (AMF), as well as an E3 ubiquitin-ligase involved in the ER-associated degradation (ERAD) of not only the tumor metastatic suppressor KAI1 but also of hepatic cytochromes P450, is upregulated in various human cancers, enhancing their invasiveness, metastatic potential, and poor prognosis. Liver-specific gp78/AMFR genetic ablation results in functional protein stabilization of several hepatic P450s and consequently enhanced drug and prodrug metabolism, a feature that could be therapeutically exploited in the bioactivation of chemotherapeutic prodrugs through design and development of novel short-term gp78/AMFR chemical inhibitors.
内质网锚定的单跨膜蛋白细胞色素 P450(P450s)是代谢内源性物质(生理活性甾体和脂肪酸)以及外源性物质(治疗/化疗药物、营养素、致癌物和毒素)的酶。肝 P450 含量的改变通过合成、失活或蛋白水解周转影响其代谢功能。P450 蛋白水解周转通过内质网相关降解(ERAD)发生,涉及泛素(Ub)依赖性蛋白酶体降解(UPD)作为主要途径。UPD 关键涉及 P450 蛋白由 E2/E3 Ub 连接酶复合物的泛素化。我们之前已经确定内质网多跨膜 gp78/AMFR(自分泌运动因子受体)是 CYP3A4、CYP3A23 和 CYP2E1 UPD 中的相关 E3。我们现在证明,在雄性小鼠中肝条件性遗传敲除 gp78/AMFR 会破坏 P450 ERAD,导致 Cyp2a5 和 Cyp2c 的统计学显著稳定,此外还包括 Cyp3a 和 Cyp2e1。更重要的是,我们确定这种稳定是功能性活性 P450 蛋白,导致其药物代谢能力相应的统计学显著增强。我们的发现,以临床相关的治疗药物(尼古丁、香豆素、氯唑沙宗和对乙酰氨基酚)和前药(他莫昔芬)作为 P450 底物,表明 P450 ERAD 破坏可能影响治疗药物反应和/或毒性,值得认真考虑作为潜在的临床相关药物相互作用(DDI)的来源。因为 gp78/AMFR 不仅是一种 E3 Ub 连接酶,也是一种细胞表面促进转移的致癌基因,在各种恶性肿瘤中上调,我们发现肝 gp78/AMFR 敲除可以增强相关癌症化疗前药的 P450 依赖性生物激活,这在未来的药物设计和开发中具有治疗意义和值得注意。意义:细胞表面和内质网跨膜蛋白 gp78/AMFR 是促转移自分泌运动因子(AMF)的受体,也是一种 E3 泛素连接酶,不仅参与肿瘤转移抑制因子 KAI1 的内质网相关降解(ERAD),还参与肝细胞色素 P450 的 ERAD,在各种人类癌症中上调,增强其侵袭性、转移潜能和预后不良。肝特异性 gp78/AMFR 遗传敲除导致几种肝 P450 的功能性蛋白稳定,从而增强药物和前药代谢,这一特征可通过设计和开发新型短期 gp78/AMFR 化学抑制剂来增强化疗前药的生物激活,从而在治疗上得到利用。