New treatment option for bile duct cancer

International study involving MHH Professor Arndt Vogel shows improved overall survival with immunotherapy

New cases of bile duct cancer, also known as biliary tumors, are increasing worldwide. The chances of cure are considered poor. The standard therapy for advanced bile duct cancer has so far been based on chemotherapy with the therapeutic agents gemcitabine and cisplatin; treatment with the body's own immune system has been researched for years for this type of cancer. An international research team with the participation of Prof. Dr. Arndt Vogel from Hannover Medical School (MHH) has now been able to prove in the KEYNOTE-966 study that the overall survival of patients is improved with the addition of the immune checkpoint inhibitor pembrolizumab. The results were published in the journal "The Lancet".

"Our study shows a statistically clear improvement in overall survival and a manageable safety profile in patients with bile duct cancer," says Prof. Dr. Arndt Vogel from the Clinical Department of Gastroenterology, Hepatology, Infectious Diseases and Endocrinology and corresponding last author of the study. "Pembrolizumab plus gemcitabine and cisplatin represents a new treatment option in first-line treatment for patients with previously untreated metastatic or unresectable bile duct cancer." KEYNOTE-966 showed particular efficacy in Caucasian patients and confirms that immunotherapy will now be an integral part of systemic therapy for biliary tumors. The KEYNOTE-966 study was the largest global study ever conducted in biliary tumors.

The KEYNOTE-966 Phase 3 trial was conducted at 175 medical centers worldwide and included 1,069 patients. Study participants were randomized to receive either treatment with pembrolizumab plus gemcitabine and cisplatin or a placebo plus gemcitabine and cisplatin. Such a procedure is considered the "gold standard" in science for achieving reliable results.

The results were presented for the first time on April 16, 2023 at the annual meeting of the American Association for Cancer Research (AACR).