(Vienna, 24 June 2025) An international study involving MedUni Vienna and the University Hospital Vienna shows that additional immunotherapy with pembrolizumab significantly prolongs disease-free survival in patients with locally advanced, resectable squamous cell carcinoma of the head and neck. The results, currently published in the top journal The New England Journal of Medicine, pave the way for approval of the therapy, which could become the new standard treatment for this common cancer.
In the Phase III clinical trial, patients with resectable head and neck squamous cell carcinoma received pembrolizumab pre- and postoperatively in addition to standard therapy, which consists of surgery followed by radiation or chemoradiotherapy. Pembrolizumab is a so-called immune checkpoint inhibitor that can switch off various brakes on the immune system and enable the body's own defences to recognise and fight cancer cells. The study participants were given the drug both before and after surgery, over a total of 17 treatment cycles. The study was conducted at 192 medical centres worldwide, including the Division of Oncology at the Department of Medicine I at MedUni Vienna and the University Hospital Vienna.
The 714 participating patients were randomly assigned to either the pembrolizumab group or the control group receiving standard treatment. The aim of the study was to investigate whether the additional immunotherapy could delay disease recurrence, tumour progression or death, as measured by the so-called event-free survival time. "With the additional application of pembrolizumab, the event-free survival increased from an average of 26.9 months to 59.7 months," reports Thorsten Füreder from the Division of Oncology at MedUni Vienna and Vienna General Hospital, who participated in the study.
Potential new standard of care
Head and neck squamous cell carcinoma is a malignant tumour that is difficult to treat and develops in the mucous membranes of the mouth, throat, larynx or nose from squamous cells – protective cells that line the surface of these areas. Treatment involves a combination of surgery, radiation therapy or chemoradiotherapy. "These standard therapies have remained largely unchanged for around two decades, so the new study results represent an important advance. Immunotherapy with pembrolizumab has primarily been used in recurrent or metastatic stages of the disease," says Matthias Preusser, Head of the Division of Oncology at MedUni Vienna and University Hospital Vienna. "The current study now confirms that this type of immunotherapy can also be effective before and after surgery and defines a potentially new standard of care for head and neck tumours," adds Thorsten Füreder. The researchers expect approval in Europe in three to four months.
Publication: The New England Journal of Medicine
Neoadjuvant and Adjuvant Pembrolizumab in Locally Advanced Head and Neck Cancer
Ravindra Uppaluri, Robert I. Haddad, Yungan Tao, Christophe Le Tourneau, Nancy Y. Lee, William Westra, Rebecca Chernock, Makoto Tahara, Kevin J. Harrington, Arkadiy L. Klochikhin, Irene Braña, Gustavo Vasconcelos Alves, Brett G. M. Hughes, Marc Oliva, M.D., Iane Pinto Figueiredo Lima, Tsutomu Ueda, Tomasz Rutkowski, Ursula Schroeder, M.D., Paul-Stefan Mauz, Thorsten Fuereder, Simon Laban, Nobuhiko Oridate, Aron Popovtzer, Nicolas Mach, Yevhen Korobko, Diogo Alpuim Costa, Anupama Hooda-Nehra, Cristina P. Rodriguez, R. Bryan Bell, Cole Manschot, Kimberly Benjamin, Burak Gumuscu, and Douglas Adkins, M.D., for the KEYNOTE-689 Investigators
DOI: 10.1056/NEJMoa2415434
https://www.nejm.org/doi/full/10.1056/NEJMoa2415434