Overall survival of recurrent/metastatic head & neck squamous cell carcinoma patients progressing after ≥ 1 line of systemic therapy, treated with MVX-ONCO-1, a novel, first in class cell encapsulation-based immunotherapy: results of SAKK 11/16, a phase IIa trial.

IF 13.5 1区 医学 Q1 HEMATOLOGY
Eugenio Fernandez, Rémi Vernet, Muriel Urwyler, Olivier Von Rohr, Emily Charrier, Marie-Claude Belkouch, Valentin Saingier, Fabien Courtout, Claudio DeVito, Virginie Ancrenaz, Nicolas Dulguerov, Wolfram Karenovics, Julien Grogg, Jessica Renaux, Katrin Gobat, Gisela Müller, Tomas Brezina, Tamara Rordorf, Markus Joerger, Olivier Michielin, Jean Villard, Nicolas Mach
{"title":"Overall survival of recurrent/metastatic head & neck squamous cell carcinoma patients progressing after ≥ 1 line of systemic therapy, treated with MVX-ONCO-1, a novel, first in class cell encapsulation-based immunotherapy: results of SAKK 11/16, a phase IIa trial.","authors":"Eugenio Fernandez, Rémi Vernet, Muriel Urwyler, Olivier Von Rohr, Emily Charrier, Marie-Claude Belkouch, Valentin Saingier, Fabien Courtout, Claudio DeVito, Virginie Ancrenaz, Nicolas Dulguerov, Wolfram Karenovics, Julien Grogg, Jessica Renaux, Katrin Gobat, Gisela Müller, Tomas Brezina, Tamara Rordorf, Markus Joerger, Olivier Michielin, Jean Villard, Nicolas Mach","doi":"10.1186/s40164-025-00703-x","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Over the past two decades, most cancer vaccines have failed to be developed clinically. The lack of efficient priming with specific tumor antigens and/or weak adjuvants may explain this poor success rate. MVX-ONCO-1, a personalized cell-based vaccine, combines inactivated autologous tumor cells and encapsulated allogeneic human cells genetically engineered to produce granulocyte-macrophage colony stimulating factor (GM-CSF). This unique technology allows sustained local delivery of strong adjuvant at the vaccination site. The combination of inactivated autologous tumor cells and potent local adjuvant delivery addresses these two unmet critical steps and may recapitulate in patients the successful combination observed in experimental models.</p><p><strong>Methods: </strong>The SAKK 11/16, a Phase IIa trial with Overall Survival (OS) as the primary endpoint was the first efficacy study evaluating MVX-ONCO-1. Patients with Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC) progressing after at least one line of systemic therapy were enrolled with 50% of patients alive at 26 weeks as the primary objective.</p><p><strong>Results: </strong>In this hard-to-treat population, SAKK 11/16 met the primary endpoint, with 68.8% of patients alive at 6 months. The median OS was 11.4 months, with 32% of the patients alive after 18 months. Complete and partial responses were observed on MVX-ONCO-1 monotherapy. Moreover, all patients who developed a positive DTH reaction to their tumor cells upon vaccination survived at 12 months. Additionally, patients living for more than 12 months had higher circulating antibody titers against tumor-associated antigens. Explorative analysis looking at median OS from the start of anti-PD-1 therapy was 21.7 months. In addition, no new safety signals with no systemic adverse events (AE) related to the treatment and no manufacturing issues were observed in this multicenter trial.</p><p><strong>Conclusions: </strong>These findings suggest that MVX-ONCO-1 can induce a coordinated immune response with clinical benefits as a standalone treatment, leading to prolonged survival. This effect may be enhanced by previous exposure to immune checkpoint inhibitors. Trial registration (ClinicalTrials.gov): NCT02999646.</p>","PeriodicalId":12180,"journal":{"name":"Experimental Hematology & Oncology","volume":"14 1","pages":"113"},"PeriodicalIF":13.5000,"publicationDate":"2025-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12398963/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Experimental Hematology & Oncology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1186/s40164-025-00703-x","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

Abstract

Background: Over the past two decades, most cancer vaccines have failed to be developed clinically. The lack of efficient priming with specific tumor antigens and/or weak adjuvants may explain this poor success rate. MVX-ONCO-1, a personalized cell-based vaccine, combines inactivated autologous tumor cells and encapsulated allogeneic human cells genetically engineered to produce granulocyte-macrophage colony stimulating factor (GM-CSF). This unique technology allows sustained local delivery of strong adjuvant at the vaccination site. The combination of inactivated autologous tumor cells and potent local adjuvant delivery addresses these two unmet critical steps and may recapitulate in patients the successful combination observed in experimental models.

Methods: The SAKK 11/16, a Phase IIa trial with Overall Survival (OS) as the primary endpoint was the first efficacy study evaluating MVX-ONCO-1. Patients with Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC) progressing after at least one line of systemic therapy were enrolled with 50% of patients alive at 26 weeks as the primary objective.

Results: In this hard-to-treat population, SAKK 11/16 met the primary endpoint, with 68.8% of patients alive at 6 months. The median OS was 11.4 months, with 32% of the patients alive after 18 months. Complete and partial responses were observed on MVX-ONCO-1 monotherapy. Moreover, all patients who developed a positive DTH reaction to their tumor cells upon vaccination survived at 12 months. Additionally, patients living for more than 12 months had higher circulating antibody titers against tumor-associated antigens. Explorative analysis looking at median OS from the start of anti-PD-1 therapy was 21.7 months. In addition, no new safety signals with no systemic adverse events (AE) related to the treatment and no manufacturing issues were observed in this multicenter trial.

Conclusions: These findings suggest that MVX-ONCO-1 can induce a coordinated immune response with clinical benefits as a standalone treatment, leading to prolonged survival. This effect may be enhanced by previous exposure to immune checkpoint inhibitors. Trial registration (ClinicalTrials.gov): NCT02999646.

Abstract Image

Abstract Image

Abstract Image

复发/转移性头颈部鳞状细胞癌患者在接受≥1线全身治疗后的总生存率,MVX-ONCO-1是一种新型的,首创的基于细胞包埋的免疫疗法:SAKK 11/16,一项IIa期试验的结果。
背景:在过去的二十年中,大多数癌症疫苗的临床开发都失败了。缺乏特异性肿瘤抗原和/或弱佐剂的有效启动可能解释了这种低成功率。MVX-ONCO-1是一种个性化的基于细胞的疫苗,结合灭活的自体肿瘤细胞和包被的异体人细胞,通过基因工程产生粒细胞-巨噬细胞集落刺激因子(GM-CSF)。这种独特的技术允许在接种部位持续地局部递送强佐剂。灭活的自体肿瘤细胞和有效的局部佐剂递送的结合解决了这两个未满足的关键步骤,并且可能在患者身上重现实验模型中观察到的成功结合。方法:SAKK 11/16,一项以总生存期(OS)为主要终点的IIa期试验,是第一个评估MVX-ONCO-1疗效的研究。复发/转移性头颈部鳞状细胞癌(R/M HNSCC)患者在接受至少一条全身治疗后进展,其中50%的患者在26周存活为主要目标。结果:在这个难以治疗的人群中,SAKK 11/16达到了主要终点,68.8%的患者在6个月时存活。中位OS为11.4个月,其中32%的患者在18个月后存活。在MVX-ONCO-1单药治疗中观察到完全和部分缓解。此外,所有接种疫苗后对肿瘤细胞产生DTH阳性反应的患者在12个月后存活。此外,存活超过12个月的患者针对肿瘤相关抗原的循环抗体滴度更高。探索性分析观察抗pd -1治疗开始的中位总生存期为21.7个月。此外,在这项多中心试验中,没有观察到新的安全信号,没有与治疗相关的系统性不良事件(AE),也没有观察到生产问题。结论:这些研究结果表明,MVX-ONCO-1可以诱导协调的免疫反应,作为独立治疗具有临床益处,可延长生存期。这种作用可能因先前暴露于免疫检查点抑制剂而增强。试验注册(ClinicalTrials.gov): NCT02999646。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
CiteScore
12.60
自引率
7.30%
发文量
97
审稿时长
6 weeks
期刊介绍: Experimental Hematology & Oncology is an open access journal that encompasses all aspects of hematology and oncology with an emphasis on preclinical, basic, patient-oriented and translational research. The journal acts as an international platform for sharing laboratory findings in these areas and makes a deliberate effort to publish clinical trials with 'negative' results and basic science studies with provocative findings. Experimental Hematology & Oncology publishes original work, hypothesis, commentaries and timely reviews. With open access and rapid turnaround time from submission to publication, the journal strives to be a hub for disseminating new knowledge and discussing controversial topics for both basic scientists and busy clinicians in the closely related fields of hematology and oncology.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:604180095
Book学术官方微信