This phase III trial studies how well pafolacianine works for identifying cancerous lesions in children and adolescent patients with primary solid tumors or solid tumors that have spread from where they first started (primary site) to other places in the body (metastatic). Pafolacianine is a fluorescent imaging agent that targets folate receptors which are overexpressed in many cancers and is used with near infrared (NIR) imaging during surgery to identify tumor cells. NIR uses a special camera that uses wavelengths in the infrared range to visualize and locate the tumor cells that are lit up by the pafolacianine. Giving pafolacianine for NIR imaging may work better than other imaging agents in identifying cancerous lesions in pediatric patients with solid tumors.

开始日期2025-04-15

申办/合作机构

Mayo Clinic

NCT06511037

/ Recruiting早期临床1期IIT

The Impact of Pafolacianine Injection for Intraoperative Imaging on Outcomes of Gastrointestinal Cancer Peritoneal Carcinomatosis - a Pilot Study

This is a pilot, single-arm, open label study to evaluate the ability of CYTALUX™ (pafolacianine) to help identify cancerous lesions in subjects with gastrointestinal cancers and peritoneal carcinomatosis during cytoreductive surgery.

开始日期2024-11-13

申办/合作机构

University of California, Irvine

[+1]

NCT06434909

/ Recruiting临床1期IIT

An Investigator Sponsored Study to Investigate the Safety and Efficacy of CYTALUX™ (PAFOLACIANINE) INJECTION for the Intraoperative Imaging of Prostate Cancer

This study is being done to compare how much using Cytalux™ (pafolacianine) with NIR (Near InfraRed) fluorescent imaging improves the detection of malignant (growing in an uncontrolled way) tissue in adult subjects undergoing prostatectomy and lymph node dissection for biopsy confirmed prostate cancer.
The U.S. Food and Drug Administration (FDA) has approved the targeted imaging agent pafolacianine (Cytalux) for use in ovarian cancer (2021) and lung cancer surgery (2022.)

开始日期2024-09-19

申办/合作机构

Indiana University

[+1]

100 项与 Pafolacianine 相关的临床结果

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100 项与 Pafolacianine 相关的转化医学

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100 项与 Pafolacianine 相关的专利（医药）

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项与 Pafolacianine 相关的文献（医药）

2025-08-01·EJSO

Enhancing surgical precision in ovarian cancer with FRα-fluorescence-guided surgery

Review

作者: Scambia, Giovanni ; Ceccaroni, Marcello ; Bogani, Giorgio ; Ferrari, Filippo Alberto ; Francesco, Raspagliesi ; Fagotti, Anna ; Pavone, Matteo ; Bourdel, Nicolas

INTRODUCTION:

Complete cytoreduction is a key prognostic factor in advanced ovarian cancer. Folate receptor alpha (FRα)-targeted intraoperative fluorescence imaging has emerged as a promising tool to enhance identification of tumor localization. Agents like pafolacianine (OTL38) and EC17 improve real-time visualization of malignant lesions, overcoming limitations of conventional methods relying on visual inspection and palpation.

MATERIALS AND METHODS:

we conducted a systematic review to evaluate the safety, efficacy, and feasibility of FRα-targeted fluorescence imaging in ovarian cancer surgery. Studies were identified through comprehensive searches in PubMed, Scopus, and Web of Science. Clinical and preclinical studies assessing FRα-targeted agents with near-infrared or other fluorescence modalities were included. Bias risk was assessed using the Cochrane Risk of Bias Tool for randomized trials and the Newcastle-Ottawa Scale for non-randomized studies.

RESULTS:

Eleven studies, including clinical and preclinical trials, were analyzed. OTL38 significantly improved lesion detection, identifying additional malignant lesions in 33 % of patients undergoing debulking surgery and enhancing detection by 29 % over standard methods, with sensitivity exceeding 85 %. EC17, assessed in smaller studies, identified 16 % more malignant lesions undetected by conventional methods, though autofluorescence was a challenge. Adverse events, predominantly mild, included nausea, vomiting, and transient skin flushing.

CONCLUSIONS:

FRα-targeted imaging may enhance lesion detection during cytoreductive surgery, increasing resection completeness. While EC17 shows feasibility, larger trials support the potential of OTL38. Future research should optimize imaging agents to reduce autofluorescence and assess their impact on survival outcomes.

2025-08-01·BIOMATERIALS

Matrix-designed bright near-infrared fluorophores for precision peripheral nerve imaging

Article

作者: Hubbell, Grace E ; Wang, Lei G ; Montaño, Antonio R ; Barth, Connor W ; Szafran, Dani A ; Gibbs, Summer L ; Masillati, Anas ; Shams, Nourhan A

The FDA's recent approval of pafolacianine, the first molecular targeted contrast agent for fluorescence-guided surgery (FGS), signifies a remarkable milestone in precision medicine. This advance offers new hope for cancer patients by enabling guided removal of cancerous tissues, where completed surgical removal remains a consistent challenge without real-time intraoperative guidance. For optimal surgical outcomes, delicate nerve tissues must be preserved to maintain patient quality of life. Despite advances in the clinical translation pipeline, the development of clinically viable nerve-specific contrast agents for FGS remains a significant challenge. Herein, a medicinal chemistry-based matrix design strategy was applied to effectively generate a synthetic roadmap permitting management of nerve-specificity within the near-infrared (NIR) oxazine fluorophore family. Many of these newly developed fluorophores demonstrated robust nerve-specificity and superior safety profiles, while also offering spectral profiles that are compatible with the clinical surgical FGS infrastructure. Notably, improving observed brightness in vivo enabled exceptional visibility of buried nerve tissue, a priority during surgical procedures. Critically, the lead probe showed a large dosage safety window capable of generating substantial contrast at doses 100x lower than the maximum tolerated dose. Following clinical translation, such NIR nerve-specific fluorophores stand poised to significantly improve outcomes for surgical patients by improving identification and visualization of surface and buried nerve tissues in real time within the surgical arena.

2025-03-01·Clinical Lung Cancer

Intraoperative Molecular Imaging With Pafolacianine: Histologic Characteristics of Identified Nodules

Article

作者: Sarkaria, Inderpal S ; Martin, Linda W ; Singhal, Sunil ; Reddy, Rishindra M ; Murthy, Sudish C ; Barret, Tina ; Biro, Timothy G ; Lopez, Alex S ; Rice, David C ; Stevens, Gary

BACKGROUND:

With increased early detection efforts, surgery for early-stage lung cancer is expected to rise. Pafolacianine is the first FDA approved targeted optical imaging agent indicated as an adjunct for intraoperative identification of malignant and nonmalignant pulmonary lesions in adult patients with known or suspected cancer in the lung.

METHODS:

This is a retrospective review of the malignant and nonmalignant lesions identified by pafolacianine with intraoperative molecular imaging (IMI) in the multi-center Phase 2 and Phase 3 ELUCIDATE clinical trials. All lesions meeting the intent to treat criteria from the combined studies were included. Histopathology for malignant and nonmalignant lesions and immunohistochemistry (ICH) for folate receptor alpha (FRα) and folate receptor beta (FRβ), which pafolacianine binds to, were assessed.

RESULTS:

A total of 273 lesions resected from 191 patients were analyzed. The identification of primary and occult malignant lesions with pafolacianine in combination with standard practice was improved (P < .001) when compared to standard practice alone. A range of histologies were demonstrated including adenocarcinoma (primary and metastatic), squamous cell carcinoma, adenoid cystic carcinoma, chordoma, lymphoma, and papillary thyroid cancer. Ninety-two percent (205 of 223) of lesions tested for folate expression were positive for FRα or FRβ expression.

CONCLUSIONS:

While initially intended to identify adenocarcinoma, IMI with pafolacianine targets a broad histological cross-section of malignant and nonmalignant primary and metastatic lesions in the lung. As real-world use expands, additional insight will continue to inform utility of pafolacianine in clinical practice and may broaden clinical applicability.

项与 Pafolacianine 相关的新闻（医药）

2025-05-23

·靶点圈

一文读懂：叶酸受体α（FRα）与多种癌症的神秘关联及靶向治疗突破

01叶酸及叶酸转运体概述叶酸代谢与肿瘤药物研发叶酸代谢在人体生物化学中至关重要，参与一碳代谢，有助于三个基本的生物合成途径：从头核苷合成、甲基化反应和氨基酸生产。但在肿瘤发生过程中会被破坏，可为肿瘤细胞的增殖提供物质基础，因此长期以来都是肿瘤药物研发的热门靶点。早期的抗叶酸药物如氨蝶呤及其后续的甲氨蝶呤、培美曲塞等，至今仍在临床广泛应用。叶酸与叶酸转运体叶酸即维生素B9，是人体必需的营养素，缺乏会导致贫血和发育缺陷。饮食中的叶酸无生物活性，需还原为活性四氢叶酸(THF)形式且携带不同的一碳单位，才能参与细胞的生物合成过程。叶酸是亲水性阴离子分子，难以自由穿过生物膜，其进入哺乳动物细胞依赖于还原型叶酸载体(RFC)、质子偶联叶酸转运体(PCFT)和叶酸受体(FRs)三种主要的转运体。图1 三种类型的叶酸转运蛋白叶酸受体(FRs)亚型FRs有四种亚型，分别为FRα、FRβ、FRγ和FRδ，由FOLR1-4基因编码，其中FRα是研究的重点。各亚型在叶酸结合亲和力和组织分布上存在差异。FRβ存在于胎盘、造血组织等，在肿瘤相关巨噬细胞中高表达，可作为M2调节性巨噬细胞极化的生物标志物和潜在免疫治疗靶点；FRγ是分泌蛋白，主要在造血组织表达，功能尚不明确；FRδ缺乏经典的叶酸结合口袋，对叶酸亲和力低，在受精过程中起重要作用，还可能是CD4+T滤泡辅助细胞激活的生物标志物。表1 叶酸受体亚型在正常组织和恶性组织中的分布与表达02FRα的生物学特征概述FRα的结构FRα是一种38-40kDa富含半胱氨酸的糖蛋白，通过羧基末端的GPI锚定在细胞膜上。它具有球状结构，包含一个深而开放的叶酸结合口袋，能高亲和力结合叶酸及其衍生物，这种结构特点为设计叶酸偶联药物和抗FRα疗法提供了基础。图2 FRα和叶酸之间的相互作用示意图FRα的表达在正常生理情况下，FRα主要表达在脉络丛、肺、甲状腺、视网膜和胎盘等极化上皮细胞的顶端或腔面。在病理情况下，多种肿瘤组织中FRα过表达。图3 FRα在恶性和正常组织中的表达FRα的氨基酸序列SignalChainPropeptide1-2424-234235-257MAQRMTTQLLLLLVWVAVVGEAQTRIAWARTELLNVCMNAKHHKEKPGPEDKLHEQCRPWRKNACCSTNTSQEAHKDVSYLYRFNWNHCGEMAPACKRHFIQDTCLYECSPNLGPWIQQVDQSWRKERVLNVPLCKEDCEQWWEDCRTSYTCKSNWHKGWNWTSGFNKCAVGAACQPFHFYFPTPTVLCNEIWTHSYKVSNYSRGSGRCIQMWFDPAQGNPNEEVARFYAAAMSGAGPWAAWPFLLSLALMLLWLLSFRα的生理功能FRα作为叶酸转运体，结合叶酸衍生物，参与叶酸的摄取和转运；可作为转录因子发挥作用；还在胚胎发育中至关重要，可能介导胚胎神经管的形成、母婴间的叶酸转运。缺乏FRα会导致胚胎发育异常、器官功能受损等问题。图4 FRα介导的细胞内吞作用和信号通路FRα在肿瘤中的作用①促进肿瘤细胞代谢：FRα通过为癌细胞提供叶酸，促进肿瘤细胞代谢，进而推动肿瘤发生。然而，由于叶酸稳态主要由RFC控制，FRα在这一过程中的重要性存在争议。②驱动肿瘤发展的转录因子功能：FRα的核转位与癌细胞的增殖以及癌症干细胞的重编程有关，其可激活多个转录因子，如OCT4/SOX2、KLF4、HES1和FGFR4等，这些转录因子分别调节干细胞特性、细胞重编程、转移和多药耐药以及细胞增殖。③与促癌信号通路相互作用：FRα可直接与促进肿瘤发生的信号通路相互作用。例如，叶酸-FRα复合物能借助共受体gp130，以JAK依赖的方式激活致癌的STAT3；FRα还被认为与丝氨酸/苏氨酸激酶ERK1/2相互作用，调节TP53基因转录；在卵巢癌中，敲低FRα可通过下调细胞-细胞粘附分子E-cadherin，抑制细胞增殖和迁移。图5 FRα表达与癌症发展之间的相关性03FRα在癌症诊断和治疗中的应用癌症诊断方面作为诊断标志物：FRα在多种癌症中呈现高表达，像卵巢癌、乳腺癌、子宫内膜癌等，而在正常组织中表达有限，这种差异表达使其可作为诊断癌症的重要生物标志物。例如，通过检测血清中FRα的含量，有助于卵巢癌的早期诊断和病情监测。多种检测手段的核心：多种方法可用于检测FRα活性，实现癌症的精准诊断。如全身单光子发射CT、FRα-靶向对比增强磁共振成像(MRI)等，能在分子和细胞水平可视化癌症变化，为早期检测和个性化治疗提供依据。表2 FRα表达水平升高的癌症类型的各种例子癌症治疗方面指导靶向治疗：FRα的过表达特性使其成为癌症靶向治疗的理想靶点。基于此开发的多种治疗方法，如叶酸偶联物、单克隆抗体等，可将细胞毒性药物精准输送到癌细胞，减少对健康细胞的损害。免疫治疗的重要靶点：针对FRα产生的抗体，可介导免疫细胞对肿瘤细胞的杀伤作用，为癌症免疫治疗提供了新策略。免疫介导的靶向FRα阳性肿瘤的治疗方法，包括过继转移肿瘤特异性T淋巴细胞、双特异性抗体靶向T细胞疗法和免疫细胞因子等，能有效抑制癌细胞的生长和转移。增强治疗效果：通过调节FRα的表达，可以提高抗癌治疗的效果。在卵巢癌治疗中，顺铂与FRα的相互作用影响癌细胞的凋亡和细胞周期，FRα过表达的癌细胞对顺铂更敏感，因此FRα可作为提高卵巢癌对顺铂治疗敏感性的潜在靶点。图6 FRα在癌症诊断和治疗中的潜在应用的未来方向局限性靶向FRα进行癌症诊断和治疗都存在一定局限性。诊断方面，检测方法存在灵敏度和特异性的问题，且放射性标记的叶酸主要成像于脾脏、肝脏和肾脏的FR，其他组织的FR难以检测。治疗方面，虽然副作用较少，但癌症细胞可能对治疗产生耐药性，且治疗费用较高。04FRα的靶向药物及其他应用图7 FRα靶向疗法及其作用机制单克隆抗体如Farletuzumab、KHK2805、MOv19、MOv18-IgG1和MOv18-IgE等，它们通过不同机制发挥抗癌作用，部分已进入临床试验阶段，但结果各异。Farletuzumab是一种人源化IgG1单克隆抗体，通过激活补体依赖的细胞毒性(CDC)等机制发挥抗癌作用，临床试验显示其在部分患者中有效，但在一些试验中未达到预期疗效。KHK2805是一种新型人源化抗体，对FRα不同表位有高亲和力，采用去岩藻糖基化设计，增强ADCC和CDC活性，在卵巢癌临床前模型中展现出显著活性，但后续开发情况不明。MOv19是一种通过杂交瘤技术制备的鼠源IgG2a抗体。此外，MOv19还被广泛改造用于多种治疗策略，其经改造生成了嵌合抗体ChiMOv19和人源化衍生物M9346A。MOv18-IgG1是与MOv19平行产生的抗体，鼠源MOv18克隆经放射性标记用于评估临床可行性，但患者易产生人抗鼠抗体(HAMA)。改造后的嵌合MOv18抗体可发挥ADCC活性，在早期试验中能有效定位到卵巢癌组织，但后续未再有开发报道。MOv18-IgE是首个针对FRα的嵌合IgE抗体，基于IgE的特性，在临床前模型中展现出良好疗效，但在早期临床试验中发现可能会引发短暂性荨麻疹等副作用。CAR-T细胞疗法通过工程化改造T细胞，使其表达针对FRα的抗原特异性单链可变片段抗体，从而靶向肿瘤细胞。针对FRα的CAR-T细胞疗法在临床前研究中显示出抗癌潜力，但I期试验中因T细胞存活不佳未观察到肿瘤负担减轻，目前研究主要集中在提高T细胞的持久性和疗效上。如MOv19-BBζ等第二代抗FRα-CAR-T细胞构建体，在体内试验中展现出更好的T细胞持久性和抗肿瘤活性，但该疗法面临鼠源scFv序列引发HAMA反应等问题。图8 CAR-T细胞疗法TIL疗法利用从患者肿瘤中收集的T细胞，与FRα靶向分子结合进行治疗。例如ITIL-306，可增强T细胞活性，目前一项评估其安全性和可行性的多癌种1期剂量递增和扩展研究正在进行中。表3 靶向FRα的临床开发中的抗体和T细胞疗法示例抗体-药物偶联物多种ADC药物正在研发中，Mirvetuximab-Soravtansine(MIRV)是首个获批用于治疗FRα阳性铂耐药卵巢癌的ADC药物，由人源化抗FRα单克隆抗体、可裂解连接子和细胞毒性药物DM4组成，但可能导致可逆性眼部不良事件。此外，MORAb-202、Luveltamab-Tazevibulin和STRO-002等ADC药物也在进行临床试验，各自具有独特的结构和作用机制，显示出一定的潜力。图9 FRα的ADC药物结构示意图以及MIRV的作用机制表4 靶向FRα的临床开发中的ADC示例叶酸-药物偶联物(FDCs)将细胞毒性药物与叶酸结合，通过FRα进入细胞发挥作用。Vintafolide是较为成功的FDC，但在III期临床试验中未达到预期效果，其他FDCs如EC131、EC2629等仍处于研究阶段。图10 Vintafolide的结构及其作用机制疫苗以FRα为靶点的疫苗可诱导免疫系统产生抗体，作为维持治疗手段预防疾病复发。早期研究证实了其安全性，与免疫检查点抑制剂联合使用可增强T细胞反应，目前相关试验主要在乳腺癌和卵巢癌患者中开展。表5 以FRα为靶点的疫苗的临床试验小分子-药物偶联物(SMDCs)叶酸-药物偶联物如EC145，在前期试验有一定效果，但III期临床试验未达到无进展生存期终点，且部分药物因毒性问题研发停滞。BGC945是一种针对FRα的胸苷酸合酶抑制剂，在I期临床试验中观察到对部分卵巢癌患者有临床益处。表6 FRα的临床试验清单联合治疗策略与化疗联合：多数FRα靶向单克隆抗体已与化疗联合应用，但尚无单克隆疗法在3期研究中显示出优于标准化疗的生存获益。相比之下，ADC联合化疗的研究相对较少，但部分ADC与化疗联合在临床前研究中显示出更好的抗肿瘤效果，相关临床试验正在进行中，有望确定新的治疗标准。与靶向药物和免疫疗法联合：如MIRV与贝伐单抗、免疫疗法药物的联合治疗，初步研究显示出一定的潜力，但仍需进一步临床试验验证。FRα的其他应用术中肿瘤识别：FDA批准的荧光药物Pafolacianine可与FRα结合，用于术中识别卵巢癌和肺癌的残留或隐藏肿瘤，提高手术的精准性和患者的预后。在卵巢癌手术研究中，33%的FRα阳性患者通过该方法检测出残留癌组织，检测灵敏度达83%，且62.4%的患者实现了完全R0切除，有助于提高手术精准度，改善患者预后。纳米颗粒疗法：FRα在癌细胞中高表达且能被细胞内吞，借助纳米技术可将化疗药物精准递送至癌细胞，增强治疗效果并减少全身副作用。如肝素-叶酸-紫杉醇(HFT)、Nab-紫杉醇-叶酸等纳米颗粒结合物已在多种癌症治疗中显示出优势，提高了药物递送效率和肿瘤杀伤效果，为癌症治疗提供了新策略。预后标志物：多项研究表明，FRα表达水平与多种癌症的预后相关。在乳腺癌、肺癌、子宫内膜癌和卵巢癌等多种癌症中，FRα高表达往往与患者较差的总生存期(OS)相关，且与肿瘤分级、结节数量和FIGO分期等相关。但在三阴性乳腺癌中，其表达与较好的无侵袭性疾病生存期(IDFS)相关，可辅助医生预测患者预后，制定更合适的治疗方案。化疗耐药替代指标：FRα表达与化疗耐药有关，在卵巢癌中，FRα高表达的患者对化疗反应不佳，它可抑制细胞毒性药物诱导的细胞凋亡。它可能通过调节MDM2和p53等分子影响癌细胞对化疗药物的敏感性。未来，FRα有望作为化疗耐药的替代指标，指导临床选择更有效的治疗方案。ctDNA检测：通过ApoStream技术和激光扫描cytometry可检测血液中FRα阳性的循环肿瘤细胞(CTCs)，为FRα表达癌症的诊断、监测和治疗指导提供了一种非侵入性方法，在非小细胞肺癌中，FRα-ctDNA可作为EGFR阳性的替代指标，辅助诊断。05HER3在多种癌症中的异常表达及影响卵巢癌FRα在约80%的原发性和复发性卵巢癌中过表达，其高表达与疾病分期和分级相关，血清sFR可作为EOC复发标志物，可通过下调肿瘤抑制因子cav-1促进肿瘤发生。可作为生物标志物用于疾病监测、判断患者对治疗的反应以及筛选适合靶向治疗的患者。此外，对一线铂类治疗耐药且术后有残留疾病的患者，其FRα水平较高，具有预后价值，且化疗不影响FRα表达，因此FRα拮抗剂成为上皮性卵巢癌的研究靶点，为卵巢癌治疗带来了新希望。宫颈癌和子宫内膜癌宫颈癌恶性转化时，FRα染色情况会发生变化，且其与FR活性相关，FRα及ERK信号蛋白水平与宫颈病变严重程度正相关。在子宫内膜癌中，FRα过表达与预后不良和无进展生存期缩短有关，且FRα表达水平在子宫内膜样腺癌中显著高于子宫内膜增生，可能参与恶性转化，可作为早期干预的靶点。乳腺癌在乳腺癌研究中，FRα状态与分期及其他标志物相关。其过表达与不良预后相关，包括无病生存期降低，通过PET和MRI等成像技术可检测其表达，指导治疗。在I-III期浸润性导管癌中，30%的样本呈FRα阳性，且三阴性乳腺癌(TNBC)与FRα阳性相关性最强，FRα的表达与较好的无侵袭性疾病生存期(IDFS)相关，但对总生存期(OS)的影响存在争议。雌激素受体阳性可能对高FRα水平有保护作用并下调其表达，因此高FRα表达的TNBC患者值得进一步研究并采用靶向抗叶酸疗法。肺癌在肺癌方面，约72%的恶性胸膜间皮瘤FRα水平升高2-4倍，但使用培美曲塞治疗，对FRα高表达患者的疗效改善不明显。在非小细胞肺癌(NSCLC)中，FRα表达与快速细胞周转相关，且与较好的预后有关。FRα在肺腺癌中表达更高，尤其是表皮生长因子受体(EGFR)突变的肿瘤以及非吸烟者和早期阶段的肿瘤。这使得FRα成为NSCLC治疗的一个有潜力的靶点，针对FRα的研究可能为NSCLC患者提供更精准的治疗方案。头颈部鳞状细胞癌头颈部鳞状细胞癌中，FRα阳性与不良临床结局相关，约45%的原发性肿瘤呈FRα阳性，无FRα阳性表达的患者生存期更长，提示FRα高表达是疾病严重程度和死亡率的重要风险因素。胃肠道癌症在胃肠道癌症中，FRα的存在与多种因素相关，可作为预后因素及潜在的治疗靶点。在胃癌中，FRα的表达与预后不良相关，且与淋巴血管浸润和淋巴结外疾病有关。它可作为一种非侵入性筛查工具，用于胃癌的早期诊断，比癌胚抗原(CEA)更具优势。在结直肠癌中，其表达与癌组织相关(33%-44%的癌组织表达，而正常结肠组织或腺瘤仅7%)，且与患者年龄、转移及非高频微卫星不稳定状态有关。研究发现，IV期结直肠癌伴肝转移患者中，术后生存期短的患者FRα表达显著更高。在胰腺癌中，FRα阳性的手术标本患者预后较差，不过其对无进展生存期影响不显著。此外，胰腺癌还主要表达FRβ，可作为分子靶向治疗的考虑对象。其他癌症在间皮瘤、口腔癌、黑色素瘤等癌症中，FRα也呈现高表达状态。在间皮瘤中，其表达情况影响治疗方案的选择；在口腔癌中，可利用FRα将化疗药物靶向递送至癌细胞，增强治疗效果；在黑色素瘤中，纳米颗粒技术借助FRα可提高免疫治疗药物的递送效率，为黑色素瘤的治疗提供新途径。图11 FRα导致细胞生长的作用机制抗体发现服务 & 产品01羊驼免疫&骆驼免疫—自建现代化养殖农场02万亿级天然抗体库产品—轻松DIY科研抗体03配套产品—助您轻松搭建基因工程抗体平台关于仁域生物成都仁域生物成立于2019年1月，是一家专注基因工程抗体技术和天然抗体库开发的公司，拥有优化的噬菌体展示抗体库技术和现代化的骆驼/羊驼养殖免疫基地。可为客户提供14天、100%成功率的先导抗体分子发现服务，彻底解决传统抗体定制的周期长、失败率高、成本高三大难题。目前已经成功完成300+靶点抗体筛选项目！protocol 获取 / 产品咨询邮箱｜find@renyubio.com电话｜19136178673地址｜成都市经开区科技产业孵化园关注我们，小编将持续更新相关内容～参考文献Liu Y, Chen X, Evan T, Esapa B, Chenoweth A, Cheung A, Karagiannis SN. Folate receptor alpha for cancer therapy: an antibody and antibody-drug conjugate target coming of age. MAbs. 2025 Dec;17(1):2470309. Ferrari FA, Ceccaroni M, Scambia G, Fagotti A, Pavone M, Bourdel N, Francesco R, Bogani G. Enhancing surgical precision in ovarian cancer with FRα-fluorescence-guided surgery. Eur J Surg Oncol. 2025 May 2;51(8):110120.Kong B, Zheng W. Mirvetuximab soravtansine: current and future applications. J Hematol Oncol. 2025 Mar 18;18(1):33. Bukowski K, Rogalska A, Marczak A. Folate Receptor Alpha-A Secret Weapon in Ovarian Cancer Treatment? Int J Mol Sci. 2024 Nov 6;25(22):11927. Gonzalez T, Muminovic M, Nano O, Vulfovich M. Folate Receptor Alpha-A Novel Approach to Cancer Therapy. Int J Mol Sci. 2024 Jan 15;25(2):1046. Kokori E, Olatunji G, Komolafe R, Abraham IC, Ukoaka B, Samuel O, Ayodeji A, Ogunbowale I, Ezenwoba C, Aderinto N. Mirvetuximab soravtansine: A breakthrough in targeted therapy for platinum-resistant ovarian cancer. Medicine (Baltimore). 2024 May 17;103(20):e38132.Mai J, Wu L, Yang L, Sun T, Liu X, Yin R, Jiang Y, Li J, Li Q. Therapeutic strategies targeting folate receptor α for ovarian cancer. Front Immunol. 2023 Aug 30;14:1254532. Varaganti P, Buddolla V, Lakshmi BA, Kim YJ. Recent advances in using folate receptor 1 (FOLR1) for cancer diagnosis and treatment, with an emphasis on cancers that affect women. Life Sci. 2023 Aug 1;326:121802.Young O, Ngo N, Lin L, Stanbery L, Creeden JF, Hamouda D, Nemunaitis J. Folate Receptor as a Biomarker and Therapeutic Target in Solid Tumors. Curr Probl Cancer. 2023 Feb;47(1):100917. Nawaz FZ, Kipreos ET. Emerging roles for folate receptor FOLR1 in signaling and cancer. Trends Endocrinol Metab. 2022 Mar;33(3):159-174.

核酸药物

2024-12-30

·美迪西Medicilon

美迪西助力合作伙伴南京诺源1类新药培泰菁绿IND获FDA批准

12月20日，南京诺源医疗器械有限公司（以下简称“诺源医疗”）自主研发的1类新药培泰菁绿获得美国食品药品监督管理局（FDA）的新药临床试验（IND）许可！这是继FDA首个靶向叶酸受体造影剂Cytalux（OTL-38）上市后，我国首个叶酸受体靶向造影剂IND许可，标志着该领域的重大突破。培泰菁绿已获中、美、欧、日专利授权，彰显其全球创新实力。上海美迪西生物医药股份有限公司（以下简称“美迪西”）作为诺源医疗的合作伙伴，为培泰菁绿的研发提供了药学研究（包括原料药和制剂）、临床前研究（包括药理学、药代动力学和安全性评价），以及IND注册申报等一站式研发服务，共同加速了新药研发进程。培泰菁绿有望解决OTL-38存在的问题推动中国分子影像领域飞跃针对OTL-38临床假阳性率、稳定性等方面存在的问题，诺源医疗率先倡导药械并举，应用AIDD技术开发的培泰菁绿，作为叶酸α受体靶向荧光造影剂。临床前体外及体内药效学结果表明，培泰菁绿能够选择性地与FRα结合，是与FRβ结合能力的数倍，有望降低临床假阳性率；稳定性研究结果表明，与OTL-38相比将显著改善储存和运输条件，降低成本，促进医疗资源分布的可及性和公平性。培泰菁绿作为原创小分子靶向药物，从实验室研究到成功获得临床试验许可，不仅是诺源医疗在分子影像领域取得的重大突破，也是中国在该领域实现的重要飞跃。这一里程碑式的成就彰显了诺源医疗在精准医疗技术领域的深厚积淀，也为其在分子影像领域取得世界竞争力奠定了坚实基础。 MEDICILON 440+肿瘤药效模型一站式助力肿瘤新药临床前研发在新药研发的激烈角逐中，速度与品质均为制胜的关键。美迪西二十年来凭借对行业动态的持续创新和拓展，成功构建了一站式生物医药临床前研发服务平台。该平台实现了从药物发现、药学研究至临床前研究的无缝对接，形成了一条高效流畅的研发链路，为新药迅速获得临床试验许可提供了坚实支撑。在肿瘤药物研发的进程中，构建一个高效且精确的药效评价模型具有举足轻重的地位。美迪西在这一领域，已构建起一套全面且先进的药效评价体系。该体系涵盖了超过440种肿瘤药效模型，包括118种PD原位模型、同种肿瘤移植模型、异种肿瘤移植模型以及人源化肿瘤移植模型等，积累了丰富的研发实力与宝贵经验。此外，美迪西已建立并完善了针对CAR-T、TCR-T、CAR-NK、溶瘤病毒、抗体（单抗、双抗、多抗等）、siRNA、AAV等免疫疗法的药效评价模型和方法，为创新疗法的研究进程注入了强大动力。美迪西祝贺诺源医疗1类新药培泰菁绿获批FDA临床，期待在临床试验中取得积极结果，也期待该药物早日获NMPA临床试验许可，实现中美双报双批。美迪西将继续创新技术，不断迭代升级肿瘤药物临床前研发一站式服务平台，为全球肿瘤药物的研发注入新动力，赋予新速度。关于诺源医疗南京诺源医疗器械有限诺源医疗是集高端医疗器械和创新型靶向造影剂药品的研发、生产和销售为一体，具有持续创新能力的国家高新技术企业。诺源医疗“药械”并举，专注于为肿瘤精准诊疗提供全链解决方案；聚焦分子荧光、拉曼光谱、人工智能、计算机辅助药物分子设计（CADD）及AI辅助药物设计等核心技术，拥有丰富的产品管线。诺源医疗是江苏省医疗器械行业协会副会长单位、南京医疗器械管理协会常务副会长单位、中国医学装备协会常务理事单位、“2021、2022及2023年度”南京市培育独角兽企业，并在2023年1月获批江苏省博士后创新实践基地。诺源医疗自成立以来，深受国家和江苏省政府重视，诺源医疗牵头完成了“国家十三·五重点研发计划”、“国家重大科研仪器研制项目”、“国家工信部人工智能医疗器械创新任务揭榜”和“江苏省关键核心技术攻关项目”等十余项国家级、省级重大重点攻关项目；截止到目前，已授权国内外发明专利141项。2022年荣获中国发明协会“发明创业奖创新奖”最高奖项：一等奖，2023年荣获沙利文GIL：中国功能性内窥镜创新奖，2024年荣获BioCon Awards年度投资价值创新企业、沙利文GIL：全球靶向分子荧光示踪剂研发领导奖。往期推荐美迪西助力 | 济民可信JM045缓释微球获批临床➠ 美迪西助力 | 济民可信JMX-2002脂质体注射液获批临床➠ 美迪西助力 | 江苏亚尧晚期肿瘤1类新药YY2201片IND获FDA批准➠ 美迪西助力 | 开悦生命针对RNA解旋酶的新药中美双报获批 ➠ 美迪西助力 | 英矽智能MAT2A抑制剂中美双报获批➠ 美迪西助力 | 中国生物上海生物制品研究所创新型ADC药物获批临床➠ 美迪西助力 | 常山药业1类新药CSCJC3456片获批开展临床研究➠ 关于美迪西美迪西（股票代码：688202.SH）成立于2004年，总部位于上海，致力于为全球制药企业、研究机构及科研工作者提供全方位的临床前新药研究服务。美迪西的一站式综合服务以强有力的项目管理和更高效、高性价比的研发服务助力客户加速新药研发进程，服务涵盖医药临床前新药研究的全过程，包括药物发现、药学研究及临床前研究。至2024年6月底，美迪西已为全球超2000家客户提供药物研发服务，参与研发完成的新药及仿制药项目已有490件IND获批临床，与国内外优质客户共同成长。美迪西将继续立足全球视野，聚力中国创新，为人类健康贡献力量！

临床申请免疫疗法细胞疗法

2024-12-10

·PRNewswire

On Target Laboratories Appoints Bill Peters as CEO

WEST LAFAYETTE, Ind., Dec. 10, 2024 /PRNewswire/ -- On Target Laboratories, Inc., a leading biotechnology company specializing in the development of intraoperative molecular imaging agents to target and illuminate cancer during surgery, today announced the appointment of Bill Peters as president and chief executive officer. Peters has also joined the company's board of directors. He succeeds Ben Lundgren, who served as CEO since 2023 and was with the company for nearly six years. Continue Reading Bill Peters, president and chief executive officer, On Target Laboratories "Ben has been a pivotal force in advancing the commercialization of CYTALUX, building our Sales and Marketing organization, establishing strategic partnerships, and forging key customer relationships. We are deeply grateful for his leadership," said Scott Huennekens, Executive Chairperson of the Board at On Target Laboratories. "We are excited to welcome Bill as our new CEO. His impressive track record of driving success at large organizations uniquely positions him to scale On Target and continue delivering value for both our customers and investors." Peters brings more than 20 years of leadership experience in the medical technology sector, including a decade of executive leadership at ConMed. Most recently, he served as President of the Advanced Surgical and Advanced Endoscopic Technologies businesses in ConMed's General Surgery division, where he played a crucial role in driving a significant portion of the company's sales and profit growth. Under his leadership, General Surgery completed three successful acquisitions, launched innovative new products and emerged as the primary growth engine for the company. Prior to ConMed, Peters held senior commercial roles at Mako Surgical Corporation, which was acquired by Stryker in 2013, and at Intuitive Surgical. Earlier in his career, he also held sales leadership positions at Stryker Corporation. Peters holds a Bachelor of Business Administration in Finance from Ohio University. "I am both honored and excited to take on this leadership role at such an impactful company," said Peters. "The work we are doing to provide patients with groundbreaking technology and renewed hope is incredibly inspiring. I look forward to working alongside our exceptional team to advance our mission to illuminate cancer intraoperatively so it can be removed completely." About On Target Laboratories, Inc. On Target Laboratories discovers and develops targeted intraoperative molecular imaging agents to illuminate cancer during surgery. Their molecular imaging technology, based on the pioneering work of Philip S. Low, PhD, Purdue University's Presidential Scholar for Drug Discovery and the Ralph C. Corley Distinguished Professor of Chemistry, is comprised of a near-infrared dye and a targeting molecule, or ligand, that binds to receptors overexpressed on cancer cells. The imaging agents illuminate the cancerous tissue, which may enable surgeons to detect more cancer that otherwise may have been left behind. CYTALUX, the company's first product, received FDA approval of a New Drug Application for ovarian cancer in November 2021 and a supplemental New Drug Application for lung cancer in December 2022. CYTALUX targets the folate receptors commonly found on many cancers, binds to the cancerous tissue, and illuminates under near-infrared light. A single dose of the agent is administered via intravenous infusion prior to surgery and assists surgeons in visually identifying additional cancerous tissue to be removed during the operation. For more information, visit and . Media Contact: On Target Laboratories, Inc. Jessica Todd 513-325-5706 [email protected] SOURCE On Target Laboratories, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

高管变更并购

100 项与 Pafolacianine 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Pafolacianine	-	DB15413

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
肺癌	美国	On Target Laboratories LLC	2022-12-16
造影剂	美国	On Target Laboratories LLC	2021-11-29
卵巢癌	美国	On Target Laboratories LLC	2021-11-29

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
肺结节	临床3期	美国	On Target Laboratories LLC	2020-03-26
FOLR1阳性卵巢癌	临床3期	美国	On Target Laboratories LLC	2018-01-26
FOLR1阳性卵巢癌	临床3期	荷兰	On Target Laboratories LLC	2018-01-26
肺腺癌	临床2期	美国	On Target Laboratories LLC	2017-05-04
肺腺癌	临床2期	荷兰	On Target Laboratories LLC	2017-05-04
叶酸受体α 阳性铂类耐药的卵巢上皮癌	临床2期	美国	On Target Laboratories LLC	2014-12-01
子宫内膜癌	临床2期	荷兰	On Target Laboratories LLC	2014-08-18
肾细胞癌	临床2期	荷兰	On Target Laboratories LLC	2014-08-18
胃肠道肿瘤	临床1期	美国	University of California, Irvine	2024-11-13
腹膜癌	临床1期	美国	University of California, Irvine	2024-11-13

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临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02872701 (CTgov)	临床2期	肺癌 \| 肺腺癌 \| 肺结节	100	Endoscopic or Thoracic Surgery+OTL38 for Injection	衊繭繭鹹窪膚憲簾鬱醖 = 顧齋獵艱鏇廠醖廠範餘衊網鏇觸衊廠憲壓選築 (膚願築壓獵範鬱廠選襯, 膚範觸築夢製繭積膚艱 ~ 壓獵蓋鑰夢繭觸網網築) 更多	-	2023-03-24
NCT04241315 (Elucidate, CTgov)	临床3期	肺癌 \| 肺结节	112	Near infrared camera imaging system+OTL38 for Injection (Near-Infrared Imaging Group)	餘壓淵鹹鏇蓋醖膚淵觸(醖鬱鬱餘觸夢窪餘築齋) = 齋廠餘鑰夢鏇艱願廠繭簾製鹹獵餘願鑰鏇壓醖 (鹽願獵淵蓋鏇衊鑰簾淵, 繭構選構衊艱鑰夢遞餘 ~ 淵願鹹窪築願網鬱鬱憲) 更多	-	2023-03-23
NCT04241315 (Elucidate, CTgov)	临床3期	肺癌 \| 肺结节	112	OTL38 for Injection (No Imaging Group)	餘壓淵鹹鏇蓋醖膚淵觸(醖鬱鬱餘觸夢窪餘築齋) = 艱遞廠鑰顧鑰觸艱繭選簾製鹹獵餘願鑰鏇壓醖 (鹽願獵淵蓋鏇衊鑰簾淵, 願顧襯網壓製憲襯襯鹹 ~ 願淵鑰壓願蓋膚積蓋窪) 更多	-	2023-03-23
NCT03180307 (Study 006, Pubmed) 人工标引	临床3期	FOLR1阳性卵巢癌 FR Positive	150	Pafolacianine	廠醖襯衊鏇觸蓋構醖鹽(簾鹹鹽衊鏇鑰遞蓋願築) = 鏇鏇鹽衊範鏇夢膚顧膚選醖夢簾獵襯觸膚衊顧 (觸觸糧觸遞醖廠夢廠蓋, 24.3 ~ 42.7) 更多	积极	2022-09-07
NCT03180307 (Study 006, Pubmed) 人工标引	临床3期	FOLR1阳性卵巢癌 FR Positive	150	Pafolacianine (patients who underwent interval debulking surgery)	-	积极	2022-09-07
NCT02317705 (CTgov)	临床2期	叶酸受体α 阳性铂类耐药的卵巢上皮癌	48	Laparotomy+OTL38	膚餘夢觸淵簾鹹鬱觸觸(憲鹽鹹繭鬱窪觸簾簾築) = 繭鏇遞衊鑰觸鏇製築淵夢鑰積鑰壓艱蓋蓋鬱齋 (鏇淵構蓋淵簾艱淵壓繭, 觸網壓鏇醖鑰構遞顧艱 ~ 選廠鹽製築膚艱艱蓋繭) 更多	-	2022-06-21
NCT03180307 (ASCO2021)	临床3期	FOLR1阳性卵巢癌	150	Pafolacianine sodium	獵網製網鹹餘願壓觸願(壓鹹鏇襯鑰鬱觸構衊鬱) = 4.7% 鑰鬱壓繭遞繭願鹽獵鹹 (廠觸製製壓顧壓餘淵遞 ) 更多	-	2021-05-28
NCT02645409 (CTgov)	N/A	肾细胞癌	14	Intraoperative fluorescence imaging system+OTL38 (Partial Nephrectomy)	窪願範網壓膚觸鬱觸網 = 製醖衊衊醖糧積蓋範餘廠齋觸積鹹膚衊遞顧壓 (願襯齋網糧窪醖鏇範鑰, 構製範鏇襯蓋鏇淵製繭 ~ 蓋網艱構選壓選醖鹹顧) 更多	-	2020-01-07
NCT02645409 (CTgov)	N/A	肾细胞癌	14	Intraoperative fluorescence imaging system+OTL38 (Radical Nephrectomy)	範網鏇糧繭觸憲鏇鑰選(糧蓋齋糧鬱衊積積蓋網) = 餘窪襯餘網選壓簾憲觸衊簾膚餘襯願醖淵製觸 (壓積蓋鏇窪餘簾網鑰顧, 窪選廠醖窪遞壓憲鹹簾 ~ 網艱糧製廠顧醖糧鬱網) 更多	-	2020-01-07
NCT02317705 (Pubmed \| Obstet Gynecol Surv) 人工标引	临床2期	FOLR1阳性卵巢癌 folate receptor-alpha positive	44	OTL38	齋壓遞襯獵壓選鏇淵範(構鹽淵艱廠齋願廠鑰觸) = infusion reaction, nausea, vomiting, and abdominal pain 網鏇選衊醖窪選糧繭蓋 (網積膚衊鹽憲製遞築製 )	积极	2019-10-01