Trop-2 protein as a therapeutic target: A focused review on Trop-2-based antibody-drug conjugates and their predictive biomarkers

⚡ 摘要

Trop-2蛋白作为治疗靶点:基于Trop-2的抗体-药物偶联物及其预测性生物标志物的重点综述

作者 Semir Vranić; Zoran Gatalica 期刊 Bosnian Journal of Basic Medical Sciences 发表日期 2021 ISSN 1512-8601 DOI 10.17305/bjbms.2021.6100 类型 原创研究 (Original Research)

📄 英文摘要 English Abstract

EN

Antibody-drug conjugates (ADCs) represent a new class of highly potent antineoplastic drugs built by attaching a small molecule of an anticancer drug (payload) or another therapeutic agent to an antibody recognizing an epitope on the targeted cells. Trophoblast cell-surface antigen-2 (Trop-2) was originally described in trophoblasts and fetal tissues, but subsequently, its overexpression has been demonstrated in various solid malignancies. Sacituzumab govitecan, a conjugate of anti-Trop-2 antibody and SN-38 payload (an active metabolite of irinotecan), is the first in the class that has been clinically validated and approved by the Food and Drug Administration for the treatment of metastatic triple-negative breast (2020) and urothelial carcinomas (2021). In the current review, we summarize and critically appraise the most recent advances with Sacituzumab govitecan, emphasizing the predictive biomarker analysis.

📄 中文摘要 Chinese Abstract

中文
滋养层细胞表面抗原-2(Trop-2)蛋白作为抗体药物偶联物(ADC)的靶点。Trop-2是一种40-kDa糖蛋白,是最早被描述的细胞内钙信号转导分子。Trop-2的表达最初在滋养层细胞(胎盘)和胎儿组织(如肺)中被发现。随后在皮肤、子宫颈、食管和扁桃体隐窝的正常复层鳞状上皮中也检测到其表达。然而,许多正常组织缺乏或仅呈现低水平的Trop-2蛋白表达(如结肠、肾脏、肝脏、肺、前列腺和乳腺)。异常的Trop-2过表达已在多种实体肿瘤中被描述,包括其正常对应组织中Trop-2表达水平较低的肿瘤(如结直肠癌、肾癌、肺癌和乳腺癌)。高Trop-2表达通常预示不良预后。

📋 英文结构化总结 English Structured Summary

全文整理

EN

Header:

Background Trophoblast cell-surface antigen-2 (Trop-2) protein as a target for antibody-drug conjugates (ADC). Trop-2 is a 40-kDa glycoprotein that was the first described transducer of intracellular calcium signaling. Trop-2 expression was originally described in trophoblasts (placenta) and fetal tissues (e.g. lungs). Its expression was subsequently described in the normal stratified squamous epithelium of the skin, uterine cervix, esophagus, and tonsil crypts. However, many normal tissues lack or show low Trop-2 protein expression (e.g. colon, kidney, liver, lung, prostate, and breast). Aberrant Trop-2 overexpression has been described in various solid cancers, including those with low Trop-2 expression in their normal counterparts (e.g. colorectal, renal, lung, and breast carcinomas). High Trop-2 expression usually confers a poor outcome.

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Methods N/A - Review article

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Results Two different ADCs targeting the Trop-2 protein have been synthesized, including Sacituzumab govitecan (SG) and RN927C. SG is the first in the class that has been clinically validated and approved by the FDA for heavily pretreated metastatic triple-negative breast and urothelial carcinomas. SG (IMMU-132) is a novel, third generation of ADCs composed of a humanized anti-Trop-2 immunoglobulin (Ig)G antibody. Trop-2 expression has also been described in some rare and aggressive malignancies, such as salivary duct carcinomas, anaplastic thyroid carcinomas, uterine/ovarian carcinosarcomas, and neuroendocrine carcinoma of the prostate.

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Data Summary In a meta-analysis by Zeng et al. that included 2569 cancer patients (reflecting 13 common solid malignancies), increased Trop-2 expression was particularly associated with poor overall survival (OS) and disease-free survival outcomes in patients with gastrointestinal and gynecological malignancies. Lower Trop-2 expression has been described in pulmonary and thyroid neuroendocrine neoplasms. We recently reported Trop-2 in ~20% of mammary NEC. Our study on cervical NEC revealed marginal (<5% of tested samples) Trop-2 protein expression.

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Conclusions The authors concluded that a frequent Trop-2 expression in the majority of solid tumors and its association with a poor prognosis provided a good rationale to target Trop-2 for therapeutic purposes. Sacituzumab govitecan (SG), a conjugate of anti-Trop-2 antibody and SN-38 payload, is the first in the class that has been clinically validated and approved by the Food and Drug Administration for the treatment of metastatic triple-negative breast (2020) and urothelial carcinomas (2021).

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Practical Significance Two recently approved indications of anti-Trop-2 ADCs are discussed in the following paragraphs. SG is the first in the class that has been clinically validated and approved by the FDA for heavily pretreated metastatic triple-negative breast and urothelial carcinomas. ADCs represent a new generation of highly potent antineoplastic drugs, with nine ADCs having already entered clinical practice.

📋 中文结构化总结 Chinese Structured Summary

中文

背景:

滋养层细胞表面抗原-2(Trop-2)蛋白作为抗体药物偶联物(ADC)的靶点。Trop-2是一种40-kDa糖蛋白,是最早被描述的细胞内钙信号转导分子。Trop-2的表达最初在滋养层细胞(胎盘)和胎儿组织(如肺)中被发现。随后在皮肤、子宫颈、食管和扁桃体隐窝的正常复层鳞状上皮中也检测到其表达。然而,许多正常组织缺乏或仅呈现低水平的Trop-2蛋白表达(如结肠、肾脏、肝脏、肺、前列腺和乳腺)。异常的Trop-2过表达已在多种实体肿瘤中被描述,包括其正常对应组织中Trop-2表达水平较低的肿瘤(如结直肠癌、肾癌、肺癌和乳腺癌)。高Trop-2表达通常预示不良预后。

方法:

不适用——综述类文章

结果:

已合成两种靶向Trop-2蛋白的不同ADC,包括Sacituzumab govitecan(SG)和RN927C。SG是该类药物中首个经临床验证并获得FDA批准用于重度预处理转移性三阴性乳腺癌和尿路上皮癌的药物。SG(IMMU-132)是一种新型第三代ADC,由人源化抗Trop-2免疫球蛋白(Ig)G抗体组成。Trop-2表达也在一些罕见且侵袭性强的恶性肿瘤中被描述,如唾液管癌、未分化甲状腺癌、子宫/卵巢癌肉瘤和前列腺神经内分泌癌。

数据总结:

在Zeng等人纳入2569例癌症患者(涵盖13种常见实体恶性肿瘤)的荟萃分析中,Trop-2表达升高与胃肠道和妇科恶性肿瘤患者的总生存期(OS)和无病生存期结局较差尤其相关。肺和甲状腺神经内分泌肿瘤中Trop-2表达水平较低。我们近期报道了约20%的乳腺神经内分泌癌(NEC)中存在Trop-2表达。我们对宫颈NEC的研究显示Trop-2蛋白表达极低(<5%的受检样本)。

结论:

作者得出结论,Trop-2在大多数实体肿瘤中的频繁表达及其与不良预后的关联,为靶向Trop-2进行靶向治疗提供了充分的理论依据。Sacituzumab govitecan(SG)是一种抗Trop-2抗体与SN-38有效载荷的偶联物,是该类药物中首个经临床验证并获得美国食品药品监督管理局(FDA)批准用于治疗转移性三阴性乳腺癌(2020年)和尿路上皮癌(2021年)的药物。

实际意义:

以下段落讨论了抗Trop-2 ADC近期获批的两个适应症。SG是该类药物中首个经临床验证并获得FDA批准用于重度预处理转移性三阴性乳腺癌和尿路上皮癌的药物。ADC代表了一代高效抗肿瘤药物,目前已有九种ADC进入临床实践。

📖 中文全文 Chinese Full Text

中文

BJBMS 综述文章 转化与临床

研究

Trop-2蛋白作为治疗靶点:基于Trop-2的抗体药物偶联物及其预测性生物标志物的专题综述

Semir Vranic1,2*,Zoran Gatalica3

摘要

抗体药物偶联物是一类新型高效抗肿瘤药物,其构建方式是将小分子抗癌药物(有效载荷)或其他治疗剂与识别靶细胞表位的抗体连接而成。滋养层细胞表面抗原-2(Trop-2)最初在滋养层细胞和胎儿组织中被发现,但后续研究证实其在多种实体恶性肿瘤中存在过表达。Sacituzumab govitecan(SG)是一种抗Trop-2抗体与SN-38(伊立替康的活性代谢物)有效载荷的偶联物,是该类药物中首个经临床验证并获得美国食品药品监督管理局批准用于治疗转移性三阴性乳腺癌(2020年)和尿路上皮癌(2021年)的药物。在本综述中,我们总结并批判性评价了SG的最新研究进展,重点强调预测性生物标志物的分析。

关键词:抗体药物偶联物;乳腺癌;预测性生物标志物;sacituzumab govitecan;滋养层细胞表面抗原-2;尿路上皮癌

引言

滋养层细胞表面抗原-2(Trop-2)蛋白作为抗体药物偶联物(ADC)的靶点

Trop-2(又称上皮糖蛋白-1、胃肠抗原733-1、膜组分表面标志物-1及肿瘤相关钙信号转导子-2)是位于1p32.1的TACSTD2基因的产物,相关综述参见Goldenberg等人[1]。Trop-2是一种40-kDa糖蛋白,是首个被发现的细胞内钙信号转导子[2,3]。其结构包含一个由274个氨基酸组成的细胞外表皮生长因子样重复区域,该区域具有三个结构域:富含半胱氨酸结构域、甲状腺球蛋白I型结构域和半胱氨酸贫乏结构域[1]。Trop-2蛋白可与多种细胞调节因子相互作用,包括胰岛素样生长因子1、闭合蛋白-1、闭合蛋白-7、细胞周期蛋白D1及蛋白激酶C[1]。

1 卡塔尔大学QU健康学院基础医学科学系,卡塔尔多哈 2 卡塔尔大学QU健康学院生物医学与制药研究单元,卡塔尔多哈 3 美国俄克拉荷马大学医学院病理学系,美国俄克拉荷马州俄克拉荷马城

*通讯作者:Semir Vranic,卡塔尔大学QU健康学院医学院,卡塔尔多哈。电子邮箱:semir.vranic@gmail.com DOI: https://doi.org/10.17305/bjbms.2021.6100 投稿日期:2021年6月1日/接收日期:2021年6月7日/在线发表日期:2021年6月4日 利益冲突声明:作者声明无利益冲突