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更新于：2025-05-07

PRAME

更新于：2025-05-07

基本信息

别名

CT130、MAPE、Melanoma antigen preferentially expressed in tumors

+ [6]

简介

Substrate-recognition component of a Cul2-RING (CRL2) E3 ubiquitin-protein ligase complex, which mediates ubiquitination of target proteins, leading to their degradation (PubMed:21822215, PubMed:26138980). The CRL2(PRAME) complex mediates ubiquitination and degradation of truncated MSRB1/SEPX1 selenoproteins produced by failed UGA/Sec decoding (PubMed:26138980). In the nucleus, the CRL2(PRAME) complex is recruited to epigenetically and transcriptionally active promoter regions bound by nuclear transcription factor Y (NFY) and probably plays a role in chromstin regulation (PubMed:21822215). Functions as a transcriptional repressor, inhibiting the signaling of retinoic acid through the retinoic acid receptors RARA, RARB and RARG: prevents retinoic acid-induced cell proliferation arrest, differentiation and apoptosis (PubMed:16179254).

关联

项与 PRAME 相关的药物

Brenetafusp

靶点

CD3 x PRAME

作用机制

CD3刺激剂 [+2]

在研机构

Immunocore Ltd. [+1]

原研机构

Immunocore Ltd.

在研适应症

黑色素瘤 [+6]

非在研适应症-

最高研发阶段临床3期

首次获批国家/地区-

首次获批日期1800-01-20

IMA-203

靶点

PRAME

作用机制

PRAME抑制剂 [+2]

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段临床3期

首次获批国家/地区-

首次获批日期1800-01-20

Zelenoleucel

靶点

NY-ESO-1 x PRAME x WT1

作用机制

NY-ESO-1调节剂 [+4]

在研机构

Marker Therapeutics, Inc. [+1]

原研机构

Marker Therapeutics, Inc.

在研适应症

急性髓性白血病 [+1]

非在研适应症-

最高研发阶段临床2期

首次获批国家/地区-

首次获批日期1800-01-20

项与 PRAME 相关的临床试验

NCT06572631

/ Not yet recruiting临床1期IIT

Phase 1b Expansion Study of Multi-Antigen Specific CD8+ T Cells After Decitabine-enhanced Lymphodepletion: An Adoptive Cellular Therapy for Patients With Relapsed or Refractory AML or MDS Following an Allogeneic Hematopoietic Cell Transplantation From Matched HLA Donors

This phase I trial tests the safety, side effects and best dose of NEXI-001 when given with decitabine and lymphodepleting chemotherapy in treating patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory) following an allogeneic hematopoietic cell transplantation from a matched donor. NEXI-001 is a type of chimeric antigen receptor T cell therapy in which a patient's T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells. T cells are taken from a patient's blood. Then the gene for a special receptor that binds to a certain protein on the patient's cancer cells is added to the T cells in the laboratory. The special receptor is called a chimeric antigen receptor (CAR). Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion for treatment of certain cancers. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Lymphodepleting chemotherapy, with fludarabine and cyclophosphamide, helps kill cancer cells in the body and helps prepare the body for the new CAR-T cells. Giving NEXI-001 with decitabine and lymphodepleting chemotherapy may be safe and tolerable in treating patients with relapsed or refractory AML or MDS following an allogeneic hematopoietic cell transplantation from a matched donor.

开始日期2025-06-07

申办/合作机构

City of Hope National Medical Center

[+1]

NCT06946225

/ Not yet recruiting临床1期

A First-in-human, Open-label Trial to Evaluate the Combination of ACTengine® IMA203 With mRNA-4203 in Previously Treated, Unresectable or Metastatic Cutaneous Melanoma or Synovial Sarcoma Patients (ACTengine® IMA203-102)

This purpose of this clinical trial is to evaluate the safety, tolerability and anti-tumor activity of IMA203 in combination with different doses of mRNA-4203. The trial includes participants with previously treated unresectable or metastatic cutaneous melanoma (CM) or synovial sarcoma (SS).

开始日期2025-04-01

申办/合作机构

Immatics US, Inc.

[+1]

NCT06552416

/ Not yet recruiting临床1期

A Phase 1 Study of Allogenic Off-the-Shelf Multi-Tumor-Associated Antigen-Specific T Cell Products (MT-401-OTS) Administered to Patients with Relapsed Acute Myeloid Leukemia or Myelodysplastic Syndromes (RAPID)

This study is a Phase 1 multicenter, open-label study evaluating the safety and efficacy of escalating doses of MT-401-OTS in 2 participant populations: 1) Those with intermediate or high-risk AML per 2022 ELN criteria who have evidence of MRD and/or

开始日期2025-01-15

申办/合作机构

Marker Therapeutics, Inc.

100 项与 PRAME 相关的临床结果

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100 项与 PRAME 相关的转化医学

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0 项与 PRAME 相关的专利（医药）

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655

项与 PRAME 相关的文献（医药）

2025-06-01·Pathology - Research and Practice

Digital quantification of Ki67 and PRAME in challenging melanocytic lesions – A novel diagnostic tool

Article

作者: Steiniche, Torben ; Brogård, Mette Bak ; Nielsen, Patricia Switten ; Lade-Keller, Johanne ; Christensen, Kristina Bang ; Wandler, Anne ; Georgsen, Jeanette Bæhr

The interpretation of immunohistochemical markers in melanocytic lesions possesses difficulties due to expression in non-melanocytic cells and the time-consuming, non-reproducible nature of manual assessment. A digital tool that accurately quantifies Ki67 and PRAME may valuably aid pathologists in the diagnostic classification of melanocytic lesions. The aim of this study was to assess the diagnostic performance of digitally quantified Ki67 and PRAME in challenging melanocytic lesions utilizing double nuclear staining methods for accurate identification of melanocytic cells. We explored the difference in Ki67 and PRAME expression by WHO-lesion-groups and Melanocytic Pathology Assessment Tool and Hierarchy for Diagnosis version 2.0 (MPATH-Dx V2.0). Tissue slides from a cohort of 156 melanocytic lesions were stained with the Ki67/SOX10 double nuclear stain and the PRAME/SOX10 virtual double nuclear stain. Melanocytic cell specific Ki67/SOX10- and PRAME/SOX10-indexes were quantified by AI-driven digital image analysis (DIA) and compared to non-specific Ki67- and PRAME-indexes. The results showed that ROC AUC of the Ki67/SOX10-index was increased compared to the non-specific Ki67-index (p < 0.001), as opposed to the AUC of the PRAME/SOX10-index compared to non-specific PRAME-index (p = 0.090). The medians of digitally quantified Ki67- and PRAME-indexes differed significantly for the overall WHO-groups and MPATH-Dx V2.0 classes (p < 0.001). In conclusion, we found that double nuclear staining improved the diagnostic performance of Ki67, but not PRAME. The combination of digitally quantified Ki67- and PRAME-indexes may potentially serve as a tool for diagnostic classification of challenging melanocytic lesions. The proposed diagnostic tool presents the results visually, graphically, and quantitatively to optimally aid the pathologist.

2025-06-01·American Society of Clinical Oncology Educational Book

Beyond Checkpoint Inhibition: Keeping Therapeutic Options Open

Review

作者: Hundal, Jasmin ; Warrier, Govind ; Mata, Jonaphine R. ; Joshi, Urvashi Mitbander ; Luke, Jason J. ; Schollenberger, Megan D. ; Lipson, Evan J. ; Funchain, Pauline

Combination immune checkpoint inhibitor therapy (ICI) with ipilimumab (anti–cytotoxic T-lymphocyte–associated protein 4) + nivolumab (anti–PD-1) in untreated, metastatic melanoma has achieved a ten-year melanoma-specific survival of 52%. However, approximately 40%-55% of patients with metastatic melanoma have primary resistance and do not initially respond to anti–PD-1, and an additional 25% of patients develop secondary resistance, exhibiting an initial response followed by disease progression. In PD-1–refractory melanoma, treatment options are limited. Addition of ipilimumab, relatlimab (anti-LAG3), or lenvatinib (VEGFR TKI) has minimal to modest efficacy. Switching to targeted BRAF/MEK inhibition improves survival for BRAF-mutant disease. MEK and KIT inhibitors have limited activity in NRAS- and KIT-mutant metastatic melanoma, respectively. Recently, personalized, autologous tumor-infiltrating lymphocyte therapy has become a US Food and Drug Administration–approved second-line option; lifileucel demonstrates durable response (approximately 30%) in heavily pretreated, metastatic melanoma. Emerging therapeutics that show promising clinical benefit in ongoing clinical trials include novel engineered oncolytic viral and human leukocyte antigen (HLA)–restricted immune-mediated T-cell therapies. As a therapy which is limited to patients who are HLA-A*02:01, T-cell receptor (TCR) engineered T cells (TCR-T) iterates on personalized adoptive cell transfer, and immune mobilizing monoclonal TCRs against cancer are CD3 bispecifics that bind glycoprotein 100 (tebentafusp, approved for metastatic uveal melanoma) or PRAME to activate T cells. Finally, in patients at high risk for immune-related adverse events (irAEs), ICI should still be considered. ICI may be given with modified immunosuppression in patients with autoimmune disease or previous organ transplantation. Cumulative data support safe administration in older patients and in ICI rechallenge for patients with previous irAE.

2025-06-01·American Journal of Ophthalmology Case Reports

Co-occurrence of EIF1AX, SF3B1, or BAP1 variants in uveal melanomas: A case series and review

Article

作者: Schefler, Amy C ; Skrehot, Henry C ; Alsoudi, Amer F

PurposeThe purpose of this study is to present a case series of patients with co-occurrence of either BRCA1 associated protein-1 (BAP1), eukaryotic translation initiation factor 1A, X-chromosomal (EIF1AX), or splicing factor 3B subunit 1 (SF3B1) in the detection and treatment of a uveal melanoma (UM) prior to the development of metastatic disease.ObservationsThis is a retrospective case series of ten patients with UM demonstrating co-occurrence of either BAP1, EIF1AX, or SF3B1 variants treated at a single ocular oncology clinic by a senior ocular oncologist between 2020 and 2024. Charts were reviewed and data on medical history, demographics, tumor characteristics, genetic testing, follow up, as well as fundus photo and B-scan ocular ultrasound were collected. The average age of the patients was 58.5 years old. The mean length of follow up was 18.2 months. Four patients had guanosine nucleotide-binding protein alpha-11 (GNA11) variants and six had guanosine nucleotide-binding protein Q (GNAQ) variants. Four patients had germline BAP1 variants. Four patients had a combination of EIF1AX and BAP1 variants. Three patients had a combination of EIF1AX and SF3B1 variants. Three patients had a combination of SF3B1 and BAP1 variants. Eight UM were gene expression profile (GEP) Class 1A and two UM were GEP Class 1B. Seven UM were preferentially expressed antigen in melanoma (PRAME) negative and three UM were PRAME positive. All patients had cytologic confirmation of the diagnosis of UM: seven had cytology results of spindle cells and three had results of mixed spindle and epithelioid cells. All patients were treated with Iodine-125 (I-125) plaque brachytherapy.Conclusions and importanceWe present a case series of patients with the co-occurrence of EIF1AX, SF3B1, or BAP1. With distinct genomic aberrations, transcriptional features, and clinical outcomes, EIF1AX, SF3B1, and BAP1 are thought to be mutually exclusive. The present case series demonstrates rare exceptions to this general pattern and speculates on the early molecular steps of UM which may lead to these rare mutation combinations.

122

项与 PRAME 相关的新闻（医药）

2025-04-30

·药时空

DCR达80%！BioNTech公布全球首个mRNA肺癌疫苗临床数据

近日，BioNTech 在2025年AACR大会上首次展示了mRNA疫苗BNT116联合cemiplimab（西米普利单抗，PD1）用于无法接受铂类化疗作为一线治疗的虚弱患者的初期临床研究数据。这项研究名为LuCa-MERIT-1，是一项开放标签、多组别的I期临床试验，致力于考察BNT116单药及其联合用药的疗效与安全性。在此次会上公布的数据集中在BNT116联合西米普利单抗的效果，特别针对不适合进行一线化疗且TPS ≥1%的患者。截至 2024年12月1日，共有20名患者参与了BNT116联合西米普利单抗的治疗方案。结果显示，所有患者都经历了一些与BNT116治疗相关的不良事件，其中13名（65%）的不良事件与西米普利单抗有关，而有3名（15%）患者出现了≥G3级的不良事件，这些事件与BNT116的使用有关，涉及症状包括心动过速、缺氧和高血压。另有2名（10%）患者的≥G3级不良事件仅与BNT116有关。严重不良事件在7名患者（35%）中出现，其中3名（15%）仅与BNT116有关，另外3名仅与西米普利单抗有关。此外，未发现致使患者死亡的不良事件。在疗效方面，数据显示，在接受治疗的 20位患者中，有9名（45%）实现了部分缓解（PR），另外有7名（35%）患者的疾病状态保持稳定。在部分缓解的9名患者中，有2名的PD-L1TPS小于50%。经过确认的客观缓解率为45%，而疾病控制率则达到了80%，同时，中位无进展生存期为9.9个月。ctDNA分析显示，患者在治疗的第3周便已有显著的分子应答反应。BNT116联合西米普利单抗治疗表现出在虚弱的晚期非小细胞肺癌患者中良好的抗肿瘤活性，并能持续引导免疫应答，且安全性可控。BNT116 是由 BioNTech 研制的一种通过静脉注射的未经修饰的 RNA-脂质体治疗性癌症疫苗。它由六种 mRNA 组成，包括CLDN6 、KK-LC-1、MAGE-A3、MAGE-A4、MAGE-C1 和 PRAME ，每种 mRNA 均在非小细胞肺癌中编码一种常见的肿瘤相关抗原（TAA）。识别微信二维码，可添加药时空小编请注明：姓名+研究方向！

临床结果AACR会议疫苗信使RNA

2025-04-30

·求实药社

全球首个mRNA肺癌疫苗！BioNTech公布临床数据：DCR达80%

来源：RNAScript近日，BioNTech 在2025年AACR大会上首次展示了mRNA疫苗BNT116联合cemiplimab（西米普利单抗，PD1）用于无法接受铂类化疗作为一线治疗的虚弱患者的初期临床研究数据。这项研究名为LuCa-MERIT-1，是一项开放标签、多组别的I期临床试验，致力于考察BNT116单药及其联合用药的疗效与安全性。在此次会上公布的数据集中在BNT116联合西米普利单抗的效果，特别针对不适合进行一线化疗且TPS ≥1%的患者。截至 2024年12月1日，共有20名患者参与了BNT116联合西米普利单抗的治疗方案。结果显示，所有患者都经历了一些与BNT116治疗相关的不良事件，其中13名（65%）的不良事件与西米普利单抗有关，而有3名（15%）患者出现了≥G3级的不良事件，这些事件与BNT116的使用有关，涉及症状包括心动过速、缺氧和高血压。另有2名（10%）患者的≥G3级不良事件仅与BNT116有关。严重不良事件在7名患者（35%）中出现，其中3名（15%）仅与BNT116有关，另外3名仅与西米普利单抗有关。此外，未发现致使患者死亡的不良事件。在疗效方面，数据显示，在接受治疗的 20位患者中，有9名（45%）实现了部分缓解（PR），另外有7名（35%）患者的疾病状态保持稳定。在部分缓解的9名患者中，有2名的PD-L1TPS小于50%。经过确认的客观缓解率为45%，而疾病控制率则达到了80%，同时，中位无进展生存期为9.9个月。ctDNA分析显示，患者在治疗的第3周便已有显著的分子应答反应。BNT116联合西米普利单抗治疗表现出在虚弱的晚期非小细胞肺癌患者中良好的抗肿瘤活性，并能持续引导免疫应答，且安全性可控。BNT116 是由 BioNTech 研制的一种通过静脉注射的未经修饰的 RNA-脂质体治疗性癌症疫苗。它由六种 mRNA 组成，包括CLDN6 、KK-LC-1、MAGE-A3、MAGE-A4、MAGE-C1 和 PRAME ，每种 mRNA 均在非小细胞肺癌中编码一种常见的肿瘤相关抗原（TAA）。免责声明：文章内容仅供参考，不构成投资建议。投资者据此操作，风险自担, 关于对文中陈述、观点判断保持中立，不对所包含内容的准确性、可靠性或完整性提供任何明示或暗示的保证。请读者仅作参考，并请自行承担全部。联系我们I-RNA 2025展位火热预定中！扫码立即咨询电话：13816031174（同微信）赞助形式包括但不仅限于演讲席位、会场展位、会刊彩页、晚宴赞助、会议用品宣传等。点击此处“阅读全文”咨询更多！

AACR会议疫苗信使RNA临床结果

2025-04-26

·今日头条

《Nature》：癌症复发转移≠无药可救，TCR-T让复发者缓解率达70%

在免疫细胞疗法的创新探索中，TCR-T细胞疗法因在实体瘤治疗中的独特潜力备受关注，目前多项临床研究已进入关键验证阶段。从实验室到临床的跨越中，TCR-T疗法正用科学实证改写“癌转移不可战胜”的固有认知。那么这项疗法在临床实践中的真实效果如何？是否有权威数据支撑呢？以下通过国际权威期刊《Nature Medicine》发布的IMA203（TCR-T）首次I期临床研究展开分析：在40例入组患者中，治疗总缓解率高达52.5%，而高剂量治疗组总缓解率更是高达70%！这组数据不仅为实体瘤免疫治疗奠定重要临床基石，更以直观的疗效改写患者命运：影像学报告上缩小的肿瘤病灶、持续延长的生存曲线，每一个数字背后都是重燃的生命希望！ ▲截图源自“nature medicine” 一、IMA203 TCR-T疗法震撼数据：实体瘤总缓解率52.5%，高剂量组达70%！难治性黑色素瘤患者治疗15个月完全缓解，持续获益超2年！近年来，原发性肿瘤及转移性肿瘤的治疗取得显著进展，早期筛查技术与新型疗法的普及提升了多种癌症的总体生存率。然而对癌症幸存者而言，初次治疗成功后仍需警惕癌症复发转移的潜在风险。尽管手术、放化疗等传统疗法显著延长了生存周期，但在遏制肿瘤转移复发、降低治疗副作用等方面仍有局限，探索更精准高效的治疗手段成为临床迫切需求。 IMA203是一种靶向自体黑色素瘤优先表达抗原（PRAME）的TCR-T细胞疗法，靶向由HLA-A02:01呈递的PRAME衍生肽，适用于治疗HLA-A02⁺且携带PRAME⁺的复发性和/或难治性实体瘤患者（包括黑色素瘤、肉瘤等）。《Nature Medicine》报道的首次人体剂量递增试验（NCT03686124）分为两个阶段：1a期剂量递增阶段（27例患者，总剂量中位数为0.409×109TCR-T细胞）、1b期剂量扩展阶段（总剂量中位数为4.16×109TCRT细胞）。结果显示如下： 1、全队列：全部40例接受IMA203治疗的患者（覆盖黑色素瘤、肉瘤等）中，总缓解率（含未确认/已确认缓解）高达52.5% (21/40)，确认缓解率（cORR）为28.9% (11/38)，中位缓解持续时间为4.4个月（范围：2.4-23.0个月）。 2、 1a期队列（27例）：总缓解率（u/cORR）为48.1% （13/27）， cORR为18.5% （5/27），中位缓解持续时间（mDOR）为4.4个月，中位无进展生存期（PFS）为2.8个月，中位总生存期（OS）为7.5个月。 3、 1b期队列（13例）：总缓解率提升至61.5% （8/13）， cORR达54.5% （6/11），8例缓解患者中 4例持续缓解，其中 2例在治疗1年后仍维持部分缓解；中位随访11.5个月，中位缓解持续时间8.1个月，中位PFS为5.7个月，中位OS未达到。 4、黑色素瘤高剂量亚组（10例）：对1a期和1b期队列中接受DL4/DL5治疗的黑色素瘤患者（n=10）分析显示， au/cORR高达70% （7/10；95%CI：34.8–93.3）， cORR为50% （5/10；95%CI：18.7–81.3）。其中3例患者在治疗9个月（1例）和12个月（2例）后仍持续缓解，mDOR尚未达到。 ▲图源“nature medicine”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除综上，IMA203在多种实体瘤（包括难治性黑色素瘤）中展现良好抗肿瘤活性，且高剂量给药、基线肿瘤负荷较低的患者中，确认缓解率显著更高。一例复发性黑色素瘤患者治疗15个月实现完全缓解，持续缓解超2年！此前，在2024年黑色素瘤研究学会大会上，公布了IMA203 TCR-T疗法针对黑色素瘤的1b期临床更新数据。其中一名51岁男性皮肤黑色素瘤患者（编号A-DL4-03）在接受IMA203治疗15个月后，成功实现完全缓解（CR）。该患者有13年的皮肤黑色素瘤病史，曾先后接受达拉非尼+曲美替尼、帕博利珠单抗、恩拉菲尼+比美替尼等5种系统治疗方案，但病情持续进展。入组前，其颈部淋巴结存在23mm的目标病灶，盆腔骨骼和肺部亦有非目标病灶。接受IMA203 TCR-T细胞治疗后，根据RECIST1.1标准评估，输注后24个月内持续无进展，病灶缩小78.3% 。研究者通过正电子发射断层扫描（PET）成像发现，输注后第15个月已达到代谢完全缓解（CR）（详见下图）。尤为令人振奋的是，截至数据截止时，该患者持续缓解已超过两年。 ▲图源“Immatics”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除二、TCR-T横扫宫颈癌、食管癌、尿路上皮癌、骨肉瘤，一例患者转移灶完全缓解持续29个月《临床肿瘤学杂志》报道了一项TCR-T细胞治疗转移性癌症的临床研究，结果振奋人心：1例转移性宫颈癌患者经治疗后实现持续≥29个月的客观完全缓解，另有3例不同癌种患者（食管癌、尿路上皮癌、骨肉瘤）在最高剂量治疗中出现持续4-19个月的客观部分缓解。本次研究共纳入17例转移性癌症患者，接受TCR转导的CD4⁺T细胞治疗，其中8例在剂量递增期接受低剂量治疗，9例在最高剂量组接受高剂量治疗。结果显示：TCR转导CD4⁺T细胞疗法在转移性宫颈癌中展现持久完全缓解潜力，在食管癌、尿路上皮癌、骨肉瘤中亦能诱导短期至中期的部分缓解，为多种实体瘤的免疫治疗提供了新方向。其中有几例患者的情况值得广泛关注： 6号宫颈癌患者其中6号宫颈癌患者：曾接受放疗及6个周期的顺铂治疗，后出现锁骨上淋巴结复发（PET阳性+活检证实）。遂入组接受2.7×10⁹个TCR转导CD4⁺T细胞治疗。结果显示：治疗后锁骨上淋巴结转移灶完全消失，且该缓解持续29个月。PET复查显示，该患者剩余的淋巴结呈阴性且缩小至＜1cm （详见下图）。 ▼转移性宫颈癌患者（患者6）在接受TCR-T细胞治疗前（左图）与治疗后29个月（右图）的颈部计算机断层扫描（CT）对比 ▲图源“JCO”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 9号食管癌患者 9号食管癌患者：既往接受放疗、FOLFOX方案（亚叶酸-氟尿嘧啶-奥沙利铂）及卡培他滨治疗后，纵隔及食管旁淋巴结出现部分缓解（PR）。 11号尿路上皮癌患者 11号尿路上皮癌患者：原发肿瘤累及左输尿管，并伴肝及腹腔淋巴结转移，经手术和化疗后进展，遂接受TCR-T治疗。结果显示：治疗后，主动脉周围淋巴结及肝转移灶持续19个月部分缓解，仅残留少量病灶（详见下图）。 ▼转移性尿路上皮癌患者（患者11）在接受TCR-T细胞治疗前（左图）与治疗后18个月（右图）的MRI对比 ▲图源“JCO”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 16号骨肉瘤患者 16号骨肉瘤患者：历经手术、化疗、靶向治疗（索拉非尼）等多线治疗后，又入组接受TCR-T治疗，结果显示：肺转移灶部分缓解持续4个月（详见下图）。 ▼转移性转移性骨肉瘤患者（患者16）在接受TCR-T细胞治疗前（左图）与治疗后4个月（右图）的胸部CT对比 ▲图源“JCO”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除三、NY-ESO-1 TCR-T疗法疗法改写肺癌治疗困局：多线治疗失败患者获部分缓解，肺癌转移灶缩小76%，生活质量大幅提升 NY-ESO-1作为癌症免疫治疗中极具潜力的靶点，在11.8%-21%的非小细胞肺癌中均有表达，其安全性与有效性已得到广泛关注。近期，《Oncology Letters》报道了一则NYESO1特异性TCR-T细胞疗法治疗晚期非小细胞肺癌（NSCLC）的振奋案例（NCT02457650），成功使肿瘤明显缩小至部分缓解。该研究纳入4例HLA-A2阳性、NY-ESO-1表达的转移性非小细胞肺癌患者，入组后，先接受淋巴细胞清除化疗，随后进行NY-ESO-1TCR-T细胞的过继转移，并配合全身性IL-2治疗。结果显示： 2例取得临床反应。其中，患者1在NY-ESO-1TCR-T细胞后近3个月，达病情稳定（SD）；患者2在治疗4个月后，达到部分缓解（PR）。值得一提的是，患者2的疗效尤为显著：这是一位44岁，HLA-A2阳性且携带EGFR突变的NY-ESO-1晚期肺腺癌（LADC）的女性，卡氏功能状态评分（KPS）仅50分，存在咯血、胸痛等症状。既往曾先后接受6周期多西他赛+卡铂化疗、吉非替尼+厄洛替尼靶向治疗，均告失败。2015年9月CT显示：右肺门、纵隔、右胸膜、右肝叶及肝包膜广泛转移，此时临床上已无更合适的治疗方案。但天无绝人之路，该患者右肺肿瘤活检证实NY-ESO-1强阳性，符合TCR-T治疗条件，遂得以入组接受NY-ESO-1 TCR-T细胞治疗。令人振奋的是，首次TCR-T细胞输注后43天CT显示：肺原发灶从95×86×54mm缩小至64×44×54mm（体积减少约56%）；肝转移灶由19.8×19.6×20mm缩小至10×10×10mm（体积减少约76%）；胸水吸收，肺复张，依据RECIST1.1标准达到部分缓解（PR）。同时，患者的卡氏功能状态评分（KPS）从50分提升至90分，咯血、胸痛症状显著缓解，生活质量大幅提升。 ▲图源“Oncology Letters”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除四、小编寄语 TCR-T治疗创新性地开创了“抗癌+抗病毒”双效合一的治疗范式。近年来，科研团队正加速探索其在病毒相关性实体瘤领域的应用，尤其是乙肝病毒引发的肝细胞癌，以及人乳头瘤病毒（HPV）相关的宫颈癌值得振奋的是，随着首款TCR-T细胞治疗产品成功获批上市，标志着这一前沿技术正式迈入临床应用阶段！我们期待未来有更多突破性抗癌治疗获批，助力实体瘤患者实现长期带瘤生存的希望。五、参考资料 [1]Wermke M,et al.Autologous T cell therapy for PRAME+ advanced solid tumors in HLA-A* 02+ patients: a phase 1 trial[J]. Nature Medicine, 2025: 1-10. https://www.nature.com/articles/s41591-025-03650-6 [2]Lu Y C,et al.Treatment of patients with metastatic cancer using a major histocompatibility complex class II–restricted T-cell receptor targeting the cancer germline antigen MAGE-A3[J]. Journal of Clinical Oncology, 2017, 35(29): 3322-3329. https://ascopubs.org/doi/10.1200/JCO.2017.74.5463?url_ver=Z39.882003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed [3]Xia Y,et al.Treatment of metastatic nonsmall cell lung cancer with NYESO1 specific TCR engineeredT cells in a phase I clinical trial: A case report[J]. Oncology letters, 2018, 16(6): 6998-7007. https://www.spandidos-publications.com/10.3892/ol.2018.9534 [4]https://www.globenewswire.com/news-release/2024/10/10/2961129/0/en/Immatics-Announces-Updated-Phase-1b-Clinical-Data-on-ACTengine-IMA203-TCR-T-Targeting-PRAME-in-Melanoma-Patients-and-Provides-Update-on-Upcoming-SUPRAME-Phase-3-Trial.html 本文为全球肿瘤医生网原创，未经授权严禁转载

细胞疗法临床结果免疫疗法临床1期