接受过≥1线全身治疗后病情进展的复发/转移性头颈部鳞状细胞癌患者，采用新型的、同类首创的基于细胞封装的免疫疗法MVX-ONCO-1进行治疗的总生存期：IIa期试验SAKK 11/16的结果

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.

作者信息

Fernandez Eugenio, Vernet Rémi, Urwyler Muriel, Von Rohr Olivier, Charrier Emily, Belkouch Marie-Claude, Saingier Valentin, Courtout Fabien, DeVito Claudio, Ancrenaz Virginie, Dulguerov Nicolas, Karenovics Wolfram, Grogg Julien, Renaux Jessica, Gobat Katrin, Müller Gisela, Brezina Tomas, Rordorf Tamara, Joerger Markus, Michielin Olivier, Villard Jean, Mach Nicolas

机构信息

Division of Oncology, Geneva University Hospitals, Geneva, Switzerland.

Swiss Cancer Center Léman (SCCL), Geneva and Lausanne, Switzerland.

出版信息

Exp Hematol Oncol. 2025 Aug 31;14(1):113. doi: 10.1186/s40164-025-00703-x.

DOI:10.1186/s40164-025-00703-x

PMID:40887652

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.

摘要

背景

在过去二十年中，大多数癌症疫苗未能在临床上得到开发。缺乏对特定肿瘤抗原的有效启动和/或弱佐剂可能解释了这种低成功率。MVX-ONCO-1是一种个性化的细胞疫苗，它将灭活的自体肿瘤细胞与经过基因工程改造以产生粒细胞-巨噬细胞集落刺激因子（GM-CSF）的封装异体人类细胞相结合。这种独特的技术允许在接种部位持续局部递送强效佐剂。灭活的自体肿瘤细胞与有效的局部佐剂递送相结合解决了这两个未满足的关键步骤，并可能在患者中重现实验模型中观察到的成功组合。

方法

SAKK 11/16是一项以总生存期（OS）为主要终点的IIa期试验，是评估MVX-ONCO-疫苗的首次疗效研究。纳入了至少接受一线全身治疗后病情进展的复发性/转移性头颈部鳞状细胞癌（R/M HNSCC）患者，主要目标是26周时50%的患者存活。

结果

在这个难以治疗的人群中，SAKK 11/16达到了主要终点，6个月时68.8%的患者存活。中位总生存期为11.4个月，18个月后32%的患者存活。在MVX-ONCO-1单药治疗中观察到了完全缓解和部分缓解。此外，所有在接种疫苗后对其肿瘤细胞产生阳性迟发型超敏反应（DTH）的患者在12个月时均存活。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。从抗PD-1治疗开始观察的中位总生存期的探索性分析为21.7个月。此外，在这项多中心试验中未观察到与治疗相关的新的安全信号，没有全身性不良事件（AE），也没有生产问题。