Anna Brandt, Konrad Klinghammer, Christoph Schultheiss, Lisa Paschold, Claudia Wickenhauser, Marcus Bauer, Anna Bergqvist, Dennis Hahn, Philippe Schafhausen, Mareike Tometten, Markus Blaurock, Henrike Barbara Zech, Chia-Jung Busch, Andreas Dietz, Urs Müller-Richter, Jürgen Alt, Andreas Boehm, Simone Kowoll, Jörg Steighardt, Alexander Lasch, Ingunn Hagen Westgaard, Marita Westhrin, Alexander Stein, Axel Hinke, Mascha Binder
{"title":"pembrolizumab治疗的复发或转移性头颈癌患者的UV1疫苗接种：一项随机多中心2期试验","authors":"Anna Brandt, Konrad Klinghammer, Christoph Schultheiss, Lisa Paschold, Claudia Wickenhauser, Marcus Bauer, Anna Bergqvist, Dennis Hahn, Philippe Schafhausen, Mareike Tometten, Markus Blaurock, Henrike Barbara Zech, Chia-Jung Busch, Andreas Dietz, Urs Müller-Richter, Jürgen Alt, Andreas Boehm, Simone Kowoll, Jörg Steighardt, Alexander Lasch, Ingunn Hagen Westgaard, Marita Westhrin, Alexander Stein, Axel Hinke, Mascha Binder","doi":"10.1016/j.medj.2025.100647","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Human telomerase reverse transcriptase is highly expressed in head and neck squamous cell carcinoma (HNSCC). The FOCUS study examines the role of the telomerase-directed vaccine UV1 in combination with pembrolizumab in patients with recurrent or metastatic (R/M) HNSCC.</p><p><strong>Methods: </strong>The FOCUS trial, a two-armed, open-label, randomized, multicenter phase 2 study, was designed to assess the efficacy and feasibility of UV1 as an add-on to pembrolizumab in the first-line treatment of patients with R/M PD-L1<sup>+</sup> HNSCC. A progression-free survival (PFS) rate at 6 months of 40% was deemed promising for further development in a phase 3 setting. The trial was conducted in 10 centers in Germany (this study was registered at ClinicalTrials.gov: NCT05075122).</p><p><strong>Findings: </strong>From August 2021 to July 2023, 25 patients were enrolled in the pembrolizumab arm and 50 patients in the pembrolizumab + UV1 arm. The PFS rate at 6 months was 40% in the pembrolizumab arm and 30% in the pembrolizumab + UV1 arm. No specific safety signals were observed in the pembrolizumab + UV1 arm apart from a reversible allergic reaction that appeared in one patient. At a median follow-up of 11.3 months, median overall survival was 13.1 months in the pembrolizumab arm and 12.6 months in the pembrolizumab + UV1 arm.</p><p><strong>Conclusions: </strong>The addition of UV1 to pembrolizumab was safe but did not show an efficacy signal in this study population.</p><p><strong>Funding: </strong>The legal sponsor of the trial was the University Medical Center Halle (Saale), Germany and was funded by a grant from Ultimovacs.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100647"},"PeriodicalIF":12.8000,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"UV1 vaccination in pembrolizumab-treated patients with recurrent or metastatic head and neck cancer: A randomized multicenter phase 2 trial.\",\"authors\":\"Anna Brandt, Konrad Klinghammer, Christoph Schultheiss, Lisa Paschold, Claudia Wickenhauser, Marcus Bauer, Anna Bergqvist, Dennis Hahn, Philippe Schafhausen, Mareike Tometten, Markus Blaurock, Henrike Barbara Zech, Chia-Jung Busch, Andreas Dietz, Urs Müller-Richter, Jürgen Alt, Andreas Boehm, Simone Kowoll, Jörg Steighardt, Alexander Lasch, Ingunn Hagen Westgaard, Marita Westhrin, Alexander Stein, Axel Hinke, Mascha Binder\",\"doi\":\"10.1016/j.medj.2025.100647\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Human telomerase reverse transcriptase is highly expressed in head and neck squamous cell carcinoma (HNSCC). The FOCUS study examines the role of the telomerase-directed vaccine UV1 in combination with pembrolizumab in patients with recurrent or metastatic (R/M) HNSCC.</p><p><strong>Methods: </strong>The FOCUS trial, a two-armed, open-label, randomized, multicenter phase 2 study, was designed to assess the efficacy and feasibility of UV1 as an add-on to pembrolizumab in the first-line treatment of patients with R/M PD-L1<sup>+</sup> HNSCC. A progression-free survival (PFS) rate at 6 months of 40% was deemed promising for further development in a phase 3 setting. The trial was conducted in 10 centers in Germany (this study was registered at ClinicalTrials.gov: NCT05075122).</p><p><strong>Findings: </strong>From August 2021 to July 2023, 25 patients were enrolled in the pembrolizumab arm and 50 patients in the pembrolizumab + UV1 arm. The PFS rate at 6 months was 40% in the pembrolizumab arm and 30% in the pembrolizumab + UV1 arm. No specific safety signals were observed in the pembrolizumab + UV1 arm apart from a reversible allergic reaction that appeared in one patient. At a median follow-up of 11.3 months, median overall survival was 13.1 months in the pembrolizumab arm and 12.6 months in the pembrolizumab + UV1 arm.</p><p><strong>Conclusions: </strong>The addition of UV1 to pembrolizumab was safe but did not show an efficacy signal in this study population.</p><p><strong>Funding: </strong>The legal sponsor of the trial was the University Medical Center Halle (Saale), Germany and was funded by a grant from Ultimovacs.</p>\",\"PeriodicalId\":29964,\"journal\":{\"name\":\"Med\",\"volume\":\" \",\"pages\":\"100647\"},\"PeriodicalIF\":12.8000,\"publicationDate\":\"2025-04-11\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Med\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1016/j.medj.2025.100647\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"MEDICINE, RESEARCH & EXPERIMENTAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Med","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.medj.2025.100647","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
UV1 vaccination in pembrolizumab-treated patients with recurrent or metastatic head and neck cancer: A randomized multicenter phase 2 trial.
Background: Human telomerase reverse transcriptase is highly expressed in head and neck squamous cell carcinoma (HNSCC). The FOCUS study examines the role of the telomerase-directed vaccine UV1 in combination with pembrolizumab in patients with recurrent or metastatic (R/M) HNSCC.
Methods: The FOCUS trial, a two-armed, open-label, randomized, multicenter phase 2 study, was designed to assess the efficacy and feasibility of UV1 as an add-on to pembrolizumab in the first-line treatment of patients with R/M PD-L1+ HNSCC. A progression-free survival (PFS) rate at 6 months of 40% was deemed promising for further development in a phase 3 setting. The trial was conducted in 10 centers in Germany (this study was registered at ClinicalTrials.gov: NCT05075122).
Findings: From August 2021 to July 2023, 25 patients were enrolled in the pembrolizumab arm and 50 patients in the pembrolizumab + UV1 arm. The PFS rate at 6 months was 40% in the pembrolizumab arm and 30% in the pembrolizumab + UV1 arm. No specific safety signals were observed in the pembrolizumab + UV1 arm apart from a reversible allergic reaction that appeared in one patient. At a median follow-up of 11.3 months, median overall survival was 13.1 months in the pembrolizumab arm and 12.6 months in the pembrolizumab + UV1 arm.
Conclusions: The addition of UV1 to pembrolizumab was safe but did not show an efficacy signal in this study population.
Funding: The legal sponsor of the trial was the University Medical Center Halle (Saale), Germany and was funded by a grant from Ultimovacs.
期刊介绍：
Med is a flagship medical journal published monthly by Cell Press, the global publisher of trusted and authoritative science journals including Cell, Cancer Cell, and Cell Reports Medicine. Our mission is to advance clinical research and practice by providing a communication forum for the publication of clinical trial results, innovative observations from longitudinal cohorts, and pioneering discoveries about disease mechanisms. The journal also encourages thought-leadership discussions among biomedical researchers, physicians, and other health scientists and stakeholders. Our goal is to improve health worldwide sustainably and ethically.
Med publishes rigorously vetted original research and cutting-edge review and perspective articles on critical health issues globally and regionally. Our research section covers clinical case reports, first-in-human studies, large-scale clinical trials, population-based studies, as well as translational research work with the potential to change the course of medical research and improve clinical practice.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
