预约演示

更新于：2025-09-24

Cabozantinib (s)-Malate

苹果酸卡博替尼

更新于：2025-09-24

概要

基本信息

药物类型

小分子化药

别名

cabozantinib、Cabozantinib Malate、Cabozantinib s-malate (USAN)

+ [9]

靶点

AXL x RET x ROS1 x TYRO3 x Tie-2 x TrkB x VEGFR1 x VEGFR2 x VEGFR3 x c-Kit x c-Met

作用方式

抑制剂、拮抗剂

作用机制

AXL抑制剂(AXL受体酪氨酸激酶抑制剂)、RET抑制剂(酪氨酸蛋白激酶受体RET抑制剂)、ROS1抑制剂(原癌基因酪氨酸蛋白激酶ROS抑制剂)

治疗领域

肿瘤消化系统疾病内分泌与代谢疾病+ [11]

在研适应症

高分化胰腺内分泌肿瘤胰腺外神经内分泌肿瘤胰腺神经内分泌肿瘤+ [196]

非在研适应症

晚期胆管癌晚期乳腺癌肝外胆管癌+ [39]

原研机构

Exelixis, Inc.

在研机构

National Cancer InstituteIpsen Pharma SAS

Exelixis, Inc.

+ [19]

非在研机构

University of Washington

EMD Serono, Inc.

江苏豪森药业集团有限公司

权益机构

Ipsen SA

Takeda Pharmaceutical Co., Ltd.

Chugai Pharmaceutical Co., Ltd.

+ [7]

最高研发阶段批准上市

首次获批日期

美国 (2012-11-29),

甲状腺髓样癌

最高研发阶段(中国)临床3期

特殊审评优先审评 (美国)、突破性疗法 (美国)、快速通道 (美国)、孤儿药 (美国)

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结构/序列

分子式C32H30FN3O10

InChIKeyHFCFMRYTXDINDK-WNQIDUERSA-N

CAS号1140909-48-3

关联

272

项与苹果酸卡博替尼相关的临床试验

NCT07187869

/ Not yet recruiting临床1期IIT

Modulation of the Bone Immune Microenvironment Following Cabozantinib Treatment of Bone Metastatic Clear Cell Renal Cell Carcinoma: Proof of Concept Study

The goal of our study is to explore how treatments affect the bone immune microenvironment and determine the best treatment options for patients with metastatic kidney cancer to the bone. This could prevent skeletal complications and improve patient outcomes.

开始日期2026-03-31

申办/合作机构

The University of Texas MD Anderson Cancer Center

[+1]

NCT06811116

/ Recruiting临床1/2期IIT

A Phase I/II Trial of Sapanisertib in Combination With Cabozantinib in β-catenin-mutated Hepatocellular Carcinoma (SAPHIRE)

This phase I/II trial studies the side effects and best dose of sapanisertib when given together with cabozantinib, and to see how well they work in treating patients with liver cancer that has spread from where it first started to other places in the body (metastatic) and contains a mutation (change) in the β-catenin gene. Sapanisertib and cabozantinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving sapanisertib and cabozantinib together may work better than giving cabozantinib alone in treating β-catenin-mutated metastatic hepatocellular carcinoma.

开始日期2026-02-10

申办/合作机构

National Cancer Institute

NCT06502171

/ Not yet recruiting临床1期IIT

A Phase 1/1b/2 Study of Cabozantinib in Combination With Selumetinib for Plexiform Neurofibroma in Adults and Adolescents With Neurofibromatosis Type 1

Based on the clinical activity of both selumetinib and cabozantinib as monotherapies in clinical trials, the demonstrated activity of these agents in reduced doses in preclinical studies, and the non-overlapping toxicity profiles, the study will assess the tolerability and efficacy of selumetinib and cabozantinib in combination in participants with NF1 ≥16 years old with progressive and/or symptomatic PN in a phase 1/1b/2 clinical trial.
Trial Design Phase 1 This will be an open label, dose escalation phase. Dose level escalation will be determined by a rolling six design. In this design, up to 6 participants can be enrolled at a given dose level and then evaluated for dose limiting toxicity (DLT) within the DLT window. The DLT window is defined as 16 weeks in this study based on the long half-life of cabozantinib and the desire to have maximum confidence about long-term tolerability of the combination prior to proceeding to the next dose level.
Phase 1b Once the recommended phase 2 dose has been determined in phase 1, an expanded cohort of 12 participants will be enrolled in phase 1b portion of the study.
Phase 2 This will be an open label, single-arm phase using the recommended phase 2 dose.

开始日期2025-10-01

申办/合作机构

The University of Alabama at Birmingham

[+2]

100 项与苹果酸卡博替尼相关的临床结果

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100 项与苹果酸卡博替尼相关的转化医学

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100 项与苹果酸卡博替尼相关的专利（医药）

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2,468

项与苹果酸卡博替尼相关的文献（医药）

2025-12-01·Medicinal Chemistry

Reducing Cabozantinib Toxicity in Renal Cell Carcinoma Treatment through Structural Modifications

Article

作者: Lu, Yongliang ; Guo, Jiaxiang ; Yang, Yu ; Yin, Xiaotao

Background and Objectives::

Cabozantinib, a Tyrosine Kinase Inhibitor (TKI), is widely used in Renal Cell Carcinoma (RCC) therapy but often causes serious side effects such as myelosuppression, immunosuppression, and angiopathy. This study aims to identify key protein targets responsible for the therapeutic efficacy and adverse reactions of cabozantinib and to explore structural modifications to reduce toxicity while preserving efficacy.

Methods::

A non-randomized computational approach was employed, screening 400 potential protein targets using SwissTargetPrediction and ChemBL databases. Molecular docking and Structure-Activity Relationship (SAR) analysis were performed to assess interactions between cabozantinib and identified targets, focusing on structural elements contributing to toxicity.

Results::

Three primary proteins were identified as responsible for the anti-tumor effects of cabozantinib, while three others were linked to its side effects. Docking analysis revealed that the methoxyphenyl group in cabozantinib formed undesirable hydrogen bonds with toxicity-related proteins. Modulating these off-target interactions by minimizing hydrogen bonding in this region could significantly reduce adverse effects.

Conclusion::

These findings provide structural insights into cabozantinib’s dual effects and suggest optimization strategies for TKI design, offering a pathway toward safer and more effective RCC treatments.

2025-11-03·JOURNAL OF EXPERIMENTAL MEDICINE

A high-throughput zebrafish screen identifies novel candidate treatments for kaposiform lymphangiomatosis (KLA)

Article

作者: Levin, Lotan ; Jabali, Amani ; Paran, Yael ; Yaniv, Karina ; Perlmoter, Gal ; Bassi, Ivan ; Barzilai, Aviv ; Tevet, Yaara ; Geva, Polina ; Leichner, Gil S. ; Otterstrom, Jason J. ; Lambiase, Giuseppina ; Barr, Haim ; Farag, Naama ; Egozi, Shany ; Avivi, Camila ; Long, Jonathan ; Greenberger, Shoshana ; Moshe, Noga

Kaposiform lymphangiomatosis (KLA) is a rare and aggressive disease caused by a somatic activating NRAS mutation (p.Q61R) in lymphatic endothelial cells (LECs). The development of new therapeutic avenues is hampered by the lack of animal models faithfully replicating the clinical manifestations of KLA. Here, we established a novel zebrafish model of KLA by conditionally expressing the human NRAS mutation in venous and lymphatic ECs. Mutant embryos recapitulate key clinical features of KLA, including dilated lymphatics and pericardial edema, which are reversed by trametinib, a MEK inhibitor used in KLA treatment. Leveraging this model in combination with an AI-based high-throughput drug screening platform, we identify cabozantinib, a tyrosine kinase inhibitor, and GSK690693, a competitive pan-Akt kinase inhibitor, as promising candidates for treating KLA. Notably, both drugs normalized sprouting and migration of cultured LECs from a KLA patient. Overall, our novel zebrafish model provides a powerful platform to dissect KLA pathogenesis and identify new therapeutic avenues.

2025-11-01·Journal of Geriatric Oncology

Pilot phase 2 study of the efficacy, safety, and tolerability of first-line cabozantinib for older fragile patients with metastatic renal cell carcinoma: The SOGUG-CABOMAYOR study

Article

作者: Torregrosa, Dolores ; Heras, Lucía ; Llabres-Valenti, Elisenda ; Climent, Miguel A ; Zambrana, Francisco ; Gironés-Sarrió, Regina ; Guzmán, Jose Carlos Villa ; Nuñez, Natalia Fernandez ; Basterretxea, Laura ; Sanchez-Escribano, Ricardo

INTRODUCTION:

The efficacy and tolerability of anticancer drugs may be affected by several factors that change with age. No specific trials of cabozantinib have been conducted in older patients with metastatic renal cell carcinoma (mRCC). The CABOMAYOR study was aimed at evaluating the efficacy in terms of objective response rate in previously untreated fragile older patients with mRCC.

MATERIALS AND METHODS:

An exploratory, phase 2, single-arm study was conducted in patients who were ≥ 70 years with a risk of frailty or who were > 75 years regardless of frailty, had a diagnosis of mRCC with measurable disease, had an ECOG-PS of 0-2, and had not received previous treatment for mRCC. Cabozantinib was orally administered once daily at a dose of 40 mg for 28-day cycles, which could be increased to 60 mg to prevent suboptimal exposure. The primary endpoint was the objective response rate (ORR). Other outcomes included progression-free survival, overall survival and adverse events.

RESULTS:

Thirty-eight patients were screened, and 24 patients were included in the study. Patients had a median age of 78 years (interquartile range [IQR] 75-81), and 12 (50%) had a Geriatric-8 score equal to or less than 14 points (i.e., risk of frailty). The median duration of follow-up was 27.0 (16.6-47.1) months. There were no confirmed complete responses, and seven patients achieved a confirmed partial response, thus leading to an ORR of 29.2% (95% confidence interval [CI]: 11.0-47.4%). The median progression-free survival and overall survival were 2.8 (95% CI: 2.0-3.6) months and 33.7 (95% CI: 20.5-46.8) months, respectively. Fourteen (58.3%) patients required dose reductions, and 17 patients (70.8%) required dose interruptions, mostly due to nonhematologic toxicity. Most of the adverse events were grade 1-2 events. There were three grade 5 adverse events, none of which were considered treatment-related: these events included physical health deterioration, cerebral hemorrhage, or COVID-19-related pneumonia. Serious adverse events occurred in 11 (45.8%) patients, three of which were considered treatment-related.

DISCUSSION:

Our results suggest that cabozantinib is effective as an initial monotherapy for older patients with mRCC with a toxicity profile consistent with that reported in younger adults.

709

项与苹果酸卡博替尼相关的新闻（医药）

2025-09-19

·PRNewswire

Liver Cancer Therapeutics Market Size Surpasses USD 13.16 Billion by 2032, Increasing at a CAGR of 16.5% from 2025 to 2033

AUSTIN, Texas and TOKYO, Sept. 19, 2025 /PRNewswire/ -- According to DataM Intelligence, the global liver cancer therapeutics market size was valued at US$3.36 billion in 2024 and is expected to reach US$13.16 billion by 2033, growing at a CAGR of 16.5% during the forecast period 2025-2033. The liver cancer therapeutics market is entering a transformative phase, driven by the shift from traditional systemic therapies to innovative immunotherapies and targeted agents that offer improved survival outcomes. The growing adoption of combination regimens, particularly checkpoint inhibitors paired with VEGF or TKI therapies, is expected to redefine the treatment landscape and expand the eligible patient pool. At the same time, the rising prevalence of hepatocellular carcinoma worldwide, coupled with strong R&D pipelines and increasing regulatory approvals, positions this market for sustained growth. A significant advancement in liver cancer therapeutics is the approval of combination immunotherapy regimens. Notably, AstraZeneca's Imfinzi (durvalumab) plus Imjudo (tremelimumab) gained global recognition after the HIMALAYA trial demonstrated a meaningful overall survival benefit in patients with unresectable hepatocellular carcinoma. This approval provided a new first-line treatment option beyond tyrosine kinase inhibitors and VEGF-targeted therapies, offering durable survival outcomes for a subset of patients and reshaping the standard of care in advanced HCC. Another key advancement is the expansion of targeted oral therapies, which have improved safety and efficacy profiles. Drugs such as lenvatinib and cabozantinib have demonstrated substantial clinical activity as first- or second-line options, while ongoing research is investigating their use in combination with checkpoint inhibitors. The growing pipeline of next-generation TKIs and precision therapies, tailored to genetic and molecular profiles, is driving innovation, improving patient survival, and reinforcing the importance of oral targeted agents in the long-term management of HCC. Get a Sample PDF Brochure of the Report (Use Corporate Email ID for a Quick Response): The chemotherapy segment, based on therapy type, is expected to account for 39.1% of the liver cancer therapeutics market share. Chemotherapy remains a vital part of the liver cancer therapeutics market, especially for patients with intermediate-stage hepatocellular carcinoma (HCC) or those ineligible for surgical resection or transplantation. Trans-arterial chemoembolization (TACE) is a standard of care used in clinical settings and often combined with targeted therapies. Despite the rise of immunotherapies and precision drugs, chemotherapy remains vital due to its established protocols, lower costs, and widespread availability. Clinical studies are exploring the synergy of chemotherapeutic agents with immunomodulators to prolong survival and delay disease progression. The North American liver cancer therapeutics market was valued at 42.1% market share in 2024. North America is expected to dominate the liver cancer therapeutics market due to its robust healthcare infrastructure, high awareness, and significant investments in oncology research and development (R&D). For instance, in January 2025, researchers at Mount Sinai made a significant breakthrough in treating hepatocellular carcinoma (HCC), a type of liver cancer. Additionally, the rise in liver cancer incidence, driven by NAFLD, obesity, and chronic hepatitis C, has increased early diagnosis rates and demand for advanced therapeutics. The FDA's favorable regulatory pathways, including priority reviews and orphan drug designations, have accelerated drug approval, ensuring rapid market access. The liver cancer therapeutics market in the U.S. dominated the North America market with the largest revenue share in 2024, driven by the increasing investment in pharmaceuticals. The regulatory framework in the U.S. ensures only safe and effective drugs reach the market, which increases trust of patients and healthcare providers in the medicines. Furthermore, the involvement of major players and the launch of novel treatments contribute to its dominance. Regulatory approvals for innovative drugs support market expansion, making the U.S. a leader in liver cancer treatments. This environment fosters continuous innovation and growth. Get Customization in the Report as Per Your Business Requirements: Europe is the second-dominating region in the liver cancer therapeutics market, valued at 34.5% market share in 2024. The liver cancer therapeutics market in Europe is driven by the growing prevalence of hepatitis B and C infections, rising alcohol consumption, and increasing cases of non-alcoholic fatty liver disease (NAFLD). Supportive regulatory approvals for novel immunotherapies, combined with a well-established healthcare infrastructure and government-backed cancer screening programs, are driving the adoption of advanced treatment regimens. In the U.K., a high burden of liver disease linked to obesity, diabetes, and alcohol misuse is accelerating demand for effective HCC treatments. National Health Service (NHS) initiatives to expand early cancer diagnosis, along with faster access schemes for innovative oncology drugs, further drive uptake of immunotherapies and targeted therapies. The Asia-Pacific region in the Liver Cancer Therapeutics Market was valued at 23.5% market share in 2024. This region is witnessing significant growth due to the very high incidence of hepatitis B and C, particularly in China and Southeast Asia, which are leading causes of HCC. Rising investments in healthcare infrastructure, increased clinical trial activity, and expanding patient access to immuno-oncology drugs are driving market growth. In Japan, liver cancer remains a leading cause of cancer mortality, primarily associated with hepatitis virus infections and an ageing population. Strong government support for oncology research, rapid adoption of cutting-edge immunotherapies, and the presence of domestic pharmaceutical players are key factors driving therapeutic innovation and market expansion. For instance, in June 2025, Ono Pharmaceutical Co., Ltd. and Bristol-Myers Squibb K.K. received supplemental approval for their anti-PD-1 antibody, Opdivo, and BMSKK's anti-CTLA-4 antibody, Yervoy, in combination therapy in Japan to expand their use for treating unresectable hepatocellular carcinoma (HCC), a type of cancer. Purchase This Exclusive Report at Just USD 4350 Only: Key Liver Cancer Therapeutics Companies: Top companies in the liver cancer therapeutics market include AstraZeneca, Bayer AG, Eli Lilly, Bristol-Myers Squibb Company, Eisai Co., Ltd., Genentech and among others. Recent Developments in the Market In September 2025, Akeso, Inc. has dosed the first patient in its Phase II registrational trial, evaluating cadonilimab, Akeso's first-in-class PD-1/CTLA-4 bispecific antibody, in combination with lenvatinib for treating advanced hepatocellular carcinoma in patients previously treated with atezolizumab and bevacizumab. In July 2025, Apollo Proton Cancer Centre (APCC) has launched an 'Advanced Liver Cancers Clinic', an integrated clinic for primary liver cancers and liver metastases. Related Reports: Pediatric Oncology Drugs Market Size, Share, Industry, Forecast and Outlook (2024-2031) Cancer Immunotherapy Market Size, Trends & Forecast 2033 Hepatocellular Carcinoma Treatment Market Size, Share, Growth Trends and Forecast Report 2025-2033 About DataM Intelligence 4Market Research: DataM Intelligence 4Market Research is a comprehensive market intelligence platform offering syndicated and customized reports along with expert consulting across multiple industries, including chemicals, healthcare, agriculture, food & beverages, and more. With extensive experience and a strategy-focused approach, DataM provides businesses and individuals with reliable market insights, statistical forecasts, and personalized research solutions to help them make informed decisions and successfully bring innovations to market. To find out more, visit or follow us on Twitter, LinkedIn and Facebook. Contact: Mr. Sai Kiran DataM Intelligence 4market Research LLP Ground floor DSL Abacus IT Park, Industrial Development Area Uppal, Hyderabad, Telangana 500039 USA: +1 877-441-4866 Email: [email protected] Content Source: Visit Our Website: Logo: SOURCE DataM Intelligence 4 Market Research LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

孤儿药临床2期上市批准免疫疗法

2025-09-19

·ioncology

鄢谢桥教授：ST-1898片在晚期肾细胞癌患者中的有效性和安全性的Ib/II期临床数据及启示丨第28届CSCO学术年会

点击蓝字，关注我们编者按：近年来，肾细胞癌的靶向治疗不断取得新突破，多靶点TKI药物研发成为热点。在第28届全国临床肿瘤学大会（CSCO）上，北京大学肿瘤医院/北京高博医院鄢谢桥教授报告了ST-1898片在晚期肾细胞癌患者中的Ib/II期临床研究结果，显示出良好的有效性和安全性。《肿瘤瞭望》特邀鄢谢桥教授就该研究的核心成果及其临床意义进行深入解读。 01 《肿瘤瞭望》：ST-1898是一个多靶点TKI。在肾癌领域，其靶点设计（如c-MET, AXL, VEGFR2等）是针对当前TKI治疗（如舒尼替尼、卡博替尼）的哪些局限性（如耐药、毒性谱）而优化的？其理论上可能带来哪些差异化优势？鄢谢桥教授北京大学肿瘤医院/北京高博医院这个问题非常好。目前国内确实缺乏新型TKI药物，我们在晚期肾癌的后线甚至一线治疗手段仍较为有限。相比之下，国外已批准卡博替尼、仑伐替尼等多靶点药物，这曾令我们非常羡慕。因此，ST-1898的研发令人振奋，我们现在也拥有了自主研发的新型TKI药物。 ST-1898是通过AI药物设计技术平台（SigmaHit®）开发的全新多靶点TKI，具有三大优势。第一，其靶点覆盖广泛，不仅包括传统的PDGFR、VEGFR，还涵盖耐药相关靶点如AXL和c-MET，以及肿瘤细胞生存依赖的c-KIT和TRK等靶点。第二，靶点抑制活性均衡。该药物对各靶点的IC50值集中在0.4–15 nM的较窄区间；而同类药物如卡博替尼的IC50范围较宽，为2–300 nM。这种均衡性有助于多个靶点协同发挥作用。第三，抑制效力更强。卡博替尼的IC50值约为ST-1898的7–20倍，说明ST-1898在分子水平上具有更强的抑制能力。以AXL靶点为例，该靶点常在抗血管生成治疗后发生耐药时被激活。目前针对AXL的策略主要包括三类：一是使用单克隆抗体，但可能存在旁路代偿激活；二是应用GAS6配体抑制剂如贝拉普利；三是使用作用于AXL的TKI，如卡博替尼，但其疗效有限。ST-1898因强效抑制AXL，有望与GAS6配体联合应用，为克服耐药提供新方向。 02 《肿瘤瞭望》：能否分享一下ST-1898在晚期肾癌患者中Ib/II期研究显示的初步有效性和安全性数据？其疗效信号（如ORR, PFS）和不良反应谱与现有的标准TKI治疗相比，展现出哪些异同点？鄢谢桥教授北京大学肿瘤医院/北京高博医院 ST-1898目前已显示出不错的疗效信号。其客观缓解率（ORR）达到10%–20%，疾病控制率（DCR）较高，超过87.5%，中位无进展生存期（PFS）达到6个月以上。这些数据与当前HIF-2‌α抑制剂及第二代TKI药物的疗效大致相当，但由于非头对头比较，仍需谨慎解读。我举几个典型病例。第一例患者既往接受过九线治疗，包括舒尼替尼、培唑帕尼、安罗替尼，以及在国外使用的仑伐替尼、卡博替尼，在第十线使用ST-1898时仍观察到明显疗效。第二例是在25例靶免治疗失败后的患者中，使用ST-1898作为二线治疗，仅1例发生疾病进展，其余24例均维持稳定。第三例是8例非透明细胞肾癌患者，其中2例达到客观缓解，6例疾病稳定，无1例进展。这说明ST-1898凭借其多靶点作用，可能覆盖更广泛的人群，尤其对多线治疗失败或特殊病理类型患者仍具潜力，这为后续研究指明了方向。 03 《肿瘤瞭望》：肾癌治疗已进入“免疫联合”时代。基于ST-1898的作用机制和现有数据，您认为它未来最有可能的开发路径是什么？是作为晚期后线治疗的单一选择，还是有望与免疫治疗联合，挑战一线治疗格局？鄢谢桥教授北京大学肿瘤医院/北京高博医院 ST-1898具有丰富且强效的作用靶点，因此其应用场景非常广泛。在后线治疗中，例如针对AXL和c-MET等介导血管靶向治疗耐药的旁路激活靶点，ST-1898单药或与GAS6配体抑制剂联合使用，显示出较好的应用前景。在一线治疗方面，该药物是否可延长疗效持续时间，也值得进一步探索。此外，ST-1898与免疫治疗、HIF-2‌α抑制剂或mTOR抑制剂的联合，同样具有广阔潜力。除了传统治疗模式之外，ST-1898还可与更多新型治疗策略相结合。例如，目前在北京高博医院开展的多项临床研究中，包括ADC药物、细胞治疗和核素治疗等正在快速发展。ST-1898作为一种强效抗血管生成TKI，基于肾癌以血管生成为主导的机制，与这些新药联合应用有望拓展其治疗领域，不仅用于前、后线治疗，甚至可能在围手术期治疗中发挥重要价值。鄢谢桥教授副主任医师北京大学肿瘤医院泌尿肿瘤内科北京高博医院黑色素瘤与肉瘤内科/泌尿肿瘤内科副主任中国抗癌协会泌尿男生殖系肿瘤专业委员会少见类型肾癌协作组委员中国临床肿瘤学会（CSCO）肿瘤心脏病学专家委员会委员北京医学会肿瘤学分会青年委员会委员北京癌症防治学会头颈部黑色素瘤专业委员会委员北京癌症防治学会头颈肿瘤MDT专业会委员（来源：《肿瘤瞭望》编辑部）声明凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

CSCO会议临床结果临床1期

2025-09-18

·ioncology

CSCO名家视点丨李恩孝教授：胆道恶性肿瘤中国专家共识发布，加速我国胆道肿瘤诊疗规范化和精准化进程

编者按 2025年9月10日~14日，第28届全国临床肿瘤学大会暨2025年中国临床肿瘤学会（CSCO）学术年会在山东济南召开。西安交通大学第一附属医院肿瘤内科的李恩孝教授在胆道肿瘤专场的专家共识解读环节中，提出了关于《胆道恶性肿瘤诊疗精准检测与分子诊断专家共识》《胆道恶性肿瘤免疫检查点抑制剂中国专家共识（2025版）》对推动我国胆道肿瘤诊疗规范化和精准化的重要临床意义和实践指导价值。此外，李恩孝教授还参与了神经内分泌肿瘤专场的讨论，并对这一高度异质性、罕见且易误诊疾病的多学科诊疗MDT模式的挑战和机遇进行了深入探讨。专家简介李恩孝教授西安交通大学第一附属医院肿瘤内科学科带头人主任医师，教授，医学博士，博士研究生导师陕西省抗癌协会肿瘤生物治疗专业委员会主委 CSCO 理事CSCO胆道肿瘤专委会副主委、NET专委会副主委和中西医结合专委会常委国家卫健委能力建设和继续教育肿瘤学专委会委员中国老年医学学会肿瘤学分会副会长中国康复医学会肿瘤康复专委会常委中国研究型医院协会精准医学与肿瘤MDT专业委员会副主委中国医疗保健国际交流促进会消化肿瘤综合诊疗学分会和NET专委会副主委CACA肿瘤分子靶向治疗专业委员会，NET专业委员会常委《现代肿瘤医学》《中国肿瘤临床与康复》编委（上下滑动可查看）胆道恶性肿瘤中国专家共识推动我国胆道肿瘤诊疗规范化和精准化梁后杰前任主委和周军教授提到的两项共识是对CSCO胆道肿瘤诊治指南的补充，意义重大，李恩孝教授主要基于以下几点进行阐述：第一、在细胞毒性药物治疗时代，ABC-02、ABC-06等临床研究确定了吉西他滨加顺铂和改良FOLFOX和FLOFIRI方案作为胆道肿瘤细胞毒性药物标准治疗方案的基石。免疫治疗出现之后，KEYNOTE-966、TOPAZ-1临床研究证明标准GC方案联合度伐利尤单抗和帕博利珠单抗使患者的中位生存时间得以延长。TOPAZ-1研究的中位OS为12.8个月，KEYNOTE-966为12.7个月，较对照组分别延长了1.8和1.3个月，两者的三年长期随访的生存率仅为16%和14%，较对照组分别提高了8%和2%，这对于临床医生和患者而言都难以接受，表明细胞毒性药物联合免疫检查点抑制剂PD-1或PD-L1的疗效需要改进。最新的临床研究探索在TOPAZ-1临床研究基础上加CTLA-4单抗能否提高患者的疗效？很遗憾结果是阴性的，另一项类似的研究同样未达到阳性结果。免疫检查点抑制PD-1或PD-L1加标准GC方案延长了患者的生存时间，然而双抗联合标准GC方案并没有取得疗效获益，因此未来双抗联合标准化疗方案用于晚期胆道细胞癌治疗还有待商榷，需要进行临床研究筛选潜在获益人群。第二，胆道肿瘤是消化道肿瘤中靶点最多的瘤种，针对这些靶点基因的TKI药物非常多。尽管胆道肿瘤突变发生率只有1%~20%，相对比较罕见，但是相应的靶向药物层出不穷。例如，抗血管生成药物在肝细胞肝癌领域取得了非常好的临床效果，尤其是贝伐珠单抗联合阿替利珠单抗在IMbrave150研究中取得了明显的生存延长。胆道细胞癌也进行了相同的临床研究IMbrave151，然而令人惋惜的是试验组和对照组的中位生存时间分别为14.6和14.9个月，并未表现出疗效差异。抗血管生成药物贝伐珠单抗在消化道肿瘤尤其是结直肠癌中至今没有很好的疗效预测指标，它在胆道肿瘤中能否联合以及如何联合使用是目前需要思考的问题。目前国内有许多临床研究正在探讨国产抗血管生成TKI药物如阿帕替尼、安罗替尼、多拉非尼和仑伐替尼等与免疫检查点抑制剂的联合使用，但仅限于Ⅰ、Ⅱ期临床研究，还有待循证学证据或者Ⅲ期临床研究进一步证实TKI药物与免疫治疗联合使用的疗效。其他少见或者罕见靶点包括HER2、FGFR、IDH1/2等，这些靶点目前有很多靶向药物。HER2是其中非常重要的一个靶点，目前的研究数据已经证明它与免疫检查点抑制剂联合可以取得很好的疗效。此外，还有临床研究已经证明抗HER2治疗与ADC药物DS8201或者德曲妥珠单抗等细胞毒性药物联合可以取得非常好的疗效。目前，双特异性抗体泽尼达妥单抗（ZW25）是最值得关注的一种新型靶向HER2药物，正处于临床研究阶段，可同时靶向HER2的2个不同表位，对于胆道恶性肿瘤表现出较好的疗效。一项关键Ⅱb期研究（HERIZONBTC01）探索泽尼达妥单抗对于吉西他滨治疗后疾病进展的HER2扩增、不可切除、局部晚期或转移性胆道恶性肿瘤患者的效果以及安全性，结果显示：患者客观缓解率为41.3%，中位缓解持续时间为12.9个月，中位生存时间已经延长到15个月。相信这HERIZON-BTC302和DESTINT-BTC-01两项Ⅲ期临床研究的结果将为HER2阳性晚期胆管细胞癌的治疗带来里程碑式的改变。第三、胆道肿瘤的多靶点检测非常重要，这两项共识及时地展示了多靶点检测的时机、方法和结果判读等内容。对于胆道肿瘤，我们希望初诊时即给予免疫组化、PCR、FISH以及一、二代测序等检测方法，从而发现罕见的靶点，然后针对性地选择已有的靶向药物治疗。国内各医院的发展水平参差不齐，尤其是基层医院有些免疫组化方法也无法达到要求。作为肿瘤医生和从事肿瘤诊断的病理科医生，我们应该从这两项共识中学习如何应用相应的靶点检测。如果无法检测，也应尽可能留取标本进行规范化处理并送往大型中心进行检测，为需要的患者提供精准的治疗。对于检测结果的判读，指南中也已经提出相应的解决方案，需要病理科和肿瘤科医生共同协作开展MDT讨论以解决。最后，李恩孝教授还提到了胆道癌新药应用的问题。目前药物的可及性主要受价格影响。新靶点药物尽管现阶段价格较高，相信随着药物研发上市、推广以及纳入医保后，这些药物的费用可能降低，并提高其可及性。胰腺神经内分泌肿瘤的新药研究进展在消化道肿瘤中，胰腺癌和胆道系统肿瘤是目前治疗最困难的领域。在神经内分泌肿瘤中胰腺神经内分泌肿瘤在国内发病率最高，几乎占所有神经内分泌肿瘤的1/4~1/3。未来胰腺神经内分泌肿瘤的治疗将集中以下几个方面：第一，SSA类药物：PROMID研究已经证实Ki-67<10%的患者对SSA类药物敏感，但高分级（Ki-67>10%）患者疗效差。CLARINET临床研究也显示兰瑞肽在SSTR阳性神经内分泌肿瘤的疗效。由于没有进行头对头的临床研究，因此这两种药物的疗效无法直接进行比较。然而目前医保对长效奥曲肽要求比较严格，必须功能性的神经内分泌肿瘤才能作为一线选择，而兰瑞肽并没有这样的限制。第二、细胞毒性药物：细胞毒性药物目前仅有CAPTEM这一标准治疗方案，并且在全球指南中推荐。国内有一项研究使用替吉奥代替卡培他滨和替莫唑胺进行联合，研究发现这一方案疗效与CAPTEM相近，且减少了手足综合征的发生。第三、TKI药物。最早出现的TKI类药物是m-TOR抑制剂依维莫司和多靶点抗血管生成药物舒尼替尼，这两种药物的出现使胰腺神经内分泌肿瘤的OS明显延长，然而对于非胰腺或者胰腺外的神经内分泌肿瘤疗效并不理想。直到10年后索凡替尼的出现改变了神经内分泌肿瘤治疗的模式。研究证实其在胰腺神经内分泌肿瘤中的近期有效率达到19%，相对于前期所有靶向药物（2%~5%）翻了三倍，同时长期生存得以明显延长，在非胰腺神经内分泌肿瘤也取得了10%的近期有效率，索凡替尼也因此获批胰腺和胰腺外神经内分泌肿瘤的适应症。此外，近期的临床研究也报道了卡博替尼与索凡替尼相近的近期有效率，约为20%左右。这些药物的上市拓宽了TKI药物在神经内分泌肿瘤中的应用。第四、PRRT（放射性核素治疗）：NETTER-1/2研究显示有效率超40%，成为进展期患者重要选择。NETTER-1/2这两项重要的临床研究明显提升了PRRT治疗在神经内分泌肿瘤的地位，有效率达到40%以上，明显优于现有的SSA药物、细胞毒性药物以及TKI类药物。随着PRRT等新药临床研究的开展，将为神经内分泌肿瘤患者在治疗带来很大的机遇。关于这几类药物不同的联合方案，国内从事神经内分泌肿瘤治疗的专家也正在进行相应的研究。神经内分泌肿瘤多学科诊疗MDT模式的挑战和机遇 MDT对于神经内分泌肿瘤的诊疗非常重要。只有在MDT模式下，我们才能进行准确的病理诊断，避免漏诊和误诊。很多基层医院都无法进行CgA、Syn、INSM1等生物标志物的检测，因此专委会倡导建立区域中心化病理会诊平台，统一诊断标准。其次，采用MDT模式能够指导我们制定合理、规范、系统的治疗计划，让患者得到良好管理和治疗。李教授呼吁初诊神经内分泌肿瘤的患者到具有神经内分泌肿瘤MDT团队的医院进行诊治，避免不必要的误诊和管理混乱。同时在适当的时机推荐患者参加相应的新药临床研究，使患者取得更大的获益。李教授在最后还分享了西安交通大学第一附属医院的MDT诊疗经验。西安交通大学第一附属医院在12年前就已经在仵正、王铮等教授的主导下开始进行胰腺内分泌肿瘤的MDT治疗，将病理科医生、影像科医生、放疗科医生、内科医生、外科医生等不同科室的医生整合到一起协同工作，互通有无，以提高罕见神经内分泌肿瘤患者的诊疗效果。对于有条件的中心，必须建立神经内分泌肿瘤MDT的诊疗模式。只有在这种模式下才能真正使患者从中获益。在CSCO现任主委沈峰教授，前任主委梁后杰教授、CACA主委陈洁教授，医促会主委依荷芭莉．迟教授等率领下中国神经内分泌肿瘤专家一直在神经内分泌肿瘤的诊疗道路上不断探索。相信随着国内新药研发数量不断增多，我国神经内分泌肿瘤的治疗将取得长足进步。（来源：肿瘤瞭望消化时讯）声明凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

CSCO会议

100 项与苹果酸卡博替尼相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10095	苹果酸卡博替尼	L01XE26	DB08875

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
高分化胰腺内分泌肿瘤	欧盟	Ipsen Pharma SAS	2025-07-24
高分化胰腺内分泌肿瘤	冰岛	Ipsen Pharma SAS	2025-07-24
高分化胰腺内分泌肿瘤	列支敦士登	Ipsen Pharma SAS	2025-07-24
高分化胰腺内分泌肿瘤	挪威	Ipsen Pharma SAS	2025-07-24
胰腺外神经内分泌肿瘤	美国	Exelixis, Inc.	2025-03-26
胰腺神经内分泌肿瘤	美国	Exelixis, Inc.	2025-03-26
分化型甲状腺癌	韩国	Ipsen Ltd.	2017-09-26
肾细胞癌	韩国	Ipsen Ltd.	2017-09-26
肝细胞癌	欧盟	Ipsen Pharma SAS	2016-09-09
肝细胞癌	冰岛	Ipsen Pharma SAS	2016-09-09
肝细胞癌	列支敦士登	Ipsen Pharma SAS	2016-09-09
肝细胞癌	挪威	Ipsen Pharma SAS	2016-09-09
晚期肾细胞癌	美国	Exelixis, Inc.	2016-04-25
甲状腺髓样癌	美国	Exelixis, Inc.	2012-11-29

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
实体瘤	申请上市	中国	江苏豪森药业集团有限公司	2020-09-28
难治性分化型甲状腺癌	临床3期	美国	ECOG-ACRIN Medical Research Foundation, Inc.	2024-08-22
难治性甲状腺癌	临床3期	美国	ECOG-ACRIN Medical Research Foundation, Inc.	2024-08-22
转移性骨肉瘤	临床3期	美国	National Cancer Institute	2023-03-03
转移性骨肉瘤	临床3期	澳大利亚	National Cancer Institute	2023-03-03
转移性骨肉瘤	临床3期	加拿大	National Cancer Institute	2023-03-03
转移性骨肉瘤	临床3期	新西兰	National Cancer Institute	2023-03-03
晚期尿路上皮癌	临床3期	美国	National Cancer Institute	2022-06-03
晚期尿路上皮癌	临床3期	加拿大	National Cancer Institute	2022-06-03
膀胱癌	临床3期	美国	National Cancer Institute	2022-06-03

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03937219 (COSMIC-313, CTgov)	临床3期	转移性肾细胞癌	855	Ipilimumab+Nivolumab+Cabozantinib (Cabozantinib + Nivolumab + Ipilimumab)	築醖醖醖鑰簾顧餘製鏇(蓋構鏇餘鏇顧獵鏇鏇夢) = 醖窪夢製網顧齋衊鹽獵齋網簾膚蓋顧簾壓鬱醖 (製鑰網鏇選糧獵襯遞膚, 獵糧醖壓鏇鬱壓築範餘 ~ 鹽鹽築糧積夢遞憲顧膚) 更多	-	2025-09-10
NCT03937219 (COSMIC-313, CTgov)	临床3期	转移性肾细胞癌	855	Placebo+Ipilimumab+Nivolumab (Placebo + Nivolumab + Ipilimumab)	築醖醖醖鑰簾顧餘製鏇(蓋構鏇餘鏇顧獵鏇鏇夢) = 網積壓膚壓顧壓鬱鹹獵齋網簾膚蓋顧簾壓鬱醖 (製鑰網鏇選糧獵襯遞膚, 製築憲簾網齋鏇積壓遞 ~ 蓋範膚繭顧窪鑰醖襯遞) 更多	-	2025-09-10
NCT01896479 (EXAMINER, CTgov)	临床4期	甲状腺髓样癌	247	Placebo capsule+Cabozantinib (Cabozantinib (XL184) 60 mg)	觸艱鑰糧顧鹽鏇艱範餘(憲簾憲憲衊願築獵鏇淵) = 憲選顧襯餘憲壓膚積繭膚蓋膚廠構糧獵壓醖鏇 (簾壓夢壓範膚鑰齋膚網, 淵衊鬱衊簾積窪構簾簾 ~ 鏇艱構鹽簾壓夢遞選顧) 更多	-	2025-09-02
NCT01896479 (EXAMINER, CTgov)	临床4期	甲状腺髓样癌	247	Cabozantinib (XL184) 140 mg (Cabozantinib (XL184) 140 mg)	觸艱鑰糧顧鹽鏇艱範餘(憲簾憲憲衊願築獵鏇淵) = 淵築襯積鏇鏇獵窪淵顧膚蓋膚廠構糧獵壓醖鏇 (簾壓夢壓範膚鑰齋膚網, 艱鏇醖鬱繭憲淵膚醖憲 ~ 醖艱顧膚憲獵鏇齋膚觸) 更多	-	2025-09-02
NCT03957551 (CTgov)	临床1/2期	转移性黑色素瘤	28	Cabozantinib+Pembrolizumab (Phase I: Cabozantinib and Pembrolizumab (40mg))	積顧顧餘壓壓鬱襯淵夢 = 獵壓網艱壓淵鏇願夢鏇鹹齋鹽構積齋艱廠顧製 (壓觸鑰壓構艱襯選膚鹽, 餘簾獵鬱衊壓構淵衊廠 ~ 壓網淵顧積膚鬱襯廠繭) 更多	-	2025-08-24
NCT03957551 (CTgov)	临床1/2期	转移性黑色素瘤	28	Cabozantinib+Pembrolizumab (Phase I: Cabozantinib and Pembrolizumab (60mg))	積顧顧餘壓壓鬱襯淵夢 = 鏇壓構艱繭範鬱構鏇鏇鹹齋鹽構積齋艱廠顧製 (壓觸鑰壓構艱襯選膚鹽, 獵鏇醖窪膚鹹顧衊獵簾 ~ 鹹築願憲簾膚鬱構繭願) 更多	-	2025-08-24
NCT03375320 (CABINET, NEWS) 人工标引	临床3期	晚期神经内分泌肿瘤	296	Cabometyx	鹽構糧構繭壓網選選鑰(積遞鏇遞鬱獵選鹽蓋蓋) = 觸築衊齋鏇鬱繭願淵壓構廠憲製襯範襯顧壓衊 (觸選糧範壓製鬱製醖積 ) 更多	积极	2025-08-14
NCT03375320 (CABINET, NEWS) 人工标引	临床3期	晚期神经内分泌肿瘤	296	Placebo	鹽構糧構繭壓網選選鑰(積遞鏇遞鬱獵選鹽蓋蓋) = 選範壓積遞構廠窪網齋構廠憲製襯範襯顧壓衊 (觸選糧範壓製鬱製醖積 ) 更多	积极	2025-08-14
NCT04164979 (CTgov)	临床2期	进展期胃腺癌 \| 胃腺癌 \| 转移性胃食管腺癌 ... 更多	34	Cabozantinib+Pembrolizumab	衊鹽鏇獵廠齋築顧衊鹽 = 鑰鑰簾壓積遞蓋願醖築憲範鏇築願齋糧選製鏇 (獵選鏇鹽餘鹹顧夢淵積, 鹹顧繭範網壓選齋鹹憲 ~ 襯選鏇鏇齋鏇齋衊繭衊) 更多	-	2025-08-11
NCT02302833 (Natalie, CTgov)	临床2期	区域性肾上腺嗜铬细胞瘤 \| 肾上腺嗜铬细胞瘤	21	Cabozantinib	構鹹糧鬱膚積衊膚蓋觸 = 憲夢糧夢製衊膚齋衊鑰範齋壓憲築願蓋製壓壓 (醖艱繭積築觸鑰鹹鹹窪, 壓襯選築鏇簾鬱憲醖蓋 ~ 鏇糧獵繭顧鏇獵鹹願壓) 更多	-	2025-07-31
NCT04220229 (CTgov)	临床1/2期	肢体肉瘤	6	Radiation Therapy+Cabozantinib S-malate	範廠獵積齋觸構糧觸淵 = 範膚鏇願夢築齋觸衊積餘獵鬱蓋憲齋淵積製觸 (憲醖襯壓顧製艱築顧壓, 願鹹夢網製糧願淵遞餘 ~ 艱網範鬱繭艱鹽衊積構)	-	2025-07-17
NCT05007613 (ESMO_GI2025)	临床2期	转移性食管鳞状细胞癌二线	24	Cabozantinib 40mg	醖醖餘築簾選繭廠鬱艱(憲鬱願願遞繭繭構窪鏇) = 構膚襯衊壓蓋廠廠網窪艱鑰願選蓋積蓋齋選鏇 (簾願襯壓膚網廠積夢願, 11.7% ~ 60.1) 更多	积极	2025-07-03
EUCTR2019-004728-39-DE (IKF-t018 AURORA, ESMO_GI2025)	临床2期	一线	22	Cabozantinib 60 mg/day	鑰鏇遞鏇鑰鏇構願襯廠(淵餘鏇鹽餘壓鹹鏇繭蓋) = DOR could not be analyzed 積繭網築淵獵鏇衊鑰憲 (願築選衊繭鹹選窪鬱窪 )	积极	2025-07-03
NCT05536141 (ARC-20, PipelineReview) 人工标引	临床1期	肾细胞癌	42	Casdatifan 100mg QD + Cabozantinib 60mg QD	襯繭築鏇觸窪鏇範鏇糧(壓壓簾繭蓋積鏇網襯淵) = 憲製積簾構簾壓積願築觸膚窪憲齋選鏇製衊憲 (艱糧膚鏇廠鬱構構繭遞, 26 ~ 67) 更多	积极	2025-06-02