A new international clinical trial, SAFIR-ABC10, is offering hope for patients with bile duct cancer (cholangiocarcinoma) by using a precision medicine approach. Sponsored by UCL and UCLH in the UK, the trial aims to improve survival rates by tailoring treatments to the unique genetic profiles of individual tumors.
Bile duct cancer, which includes intrahepatic, perihilar, and distal cholangiocarcinoma, as well as gallbladder cancer, is often diagnosed late, leading to poor outcomes. According to Cancer Research UK, up to 70% of patients die within a year of diagnosis. The SAFIR-ABC10 trial seeks to address this urgent need for more effective treatments.
Trial Design and Treatment Approach
The SAFIR-ABC10 trial will enroll approximately 800 participants worldwide. Patients will undergo comprehensive genetic profiling of their tumors to identify specific targets. Based on these profiles, patients will be offered one or more of seven different anti-cancer therapies that are best matched to their tumor's genetic makeup. The UK arm of the trial is being run by University College London Hospitals (UCLH) and University College London (UCL).
Professor John Bridgewater, UCL Cancer Institute clinical researcher and UCLH consultant medical oncologist, who leads the UK arm of the trial, emphasized the importance of this approach: "With the current standard of care, patients typically only live for one year after treatment begins. So it has become increasingly more urgent for us to try to identify more innovative and effective alternative treatment options."
The trial builds upon recent advances in understanding the molecular characteristics of biliary tract cancers and the development of targeted therapies. Helen Morement, CEO of AMMF - The Cholangiocarcinoma Charity, noted the significance of incorporating both immunotherapy (durvalumab) with standard chemotherapy (gemcitabine and cisplatin) and genomic profiling in this first-line study.
Potential Benefits and Future Implications
Early results from the trial have been promising. Some patients have experienced remission, while others have seen previously inoperable tumors become suitable for surgical removal. Professor Bridgewater highlighted the potential for significantly improved outcomes: "For some of the patients that we will be able to identify as part of this study, the outlook can be incredibly good – several years, if not more, of extremely good quality of life survival, even if your cancer is incurable."
The therapies being evaluated in the SAFIR-ABC10 trial include futibatinib, ivosidenib, zanidatamab, trastuzumab, neratinib, encorafenib, and binimetinib, in addition to chemotherapy and durvalumab. The trial aims to establish new standards for treating bile duct cancer and to demonstrate the value of genetic profiling in guiding treatment decisions.
Dr. Tayyaba Jiwani, science engagement manager at Cancer Research UK, commented: "There is a pressing need for new treatment avenues and, through SAFIR ABC-10, we're proud to support one of the first precision medicine trials for biliary tract cancers...accelerating the development of more personalised, genetically targeted treatments that are more likely to be effective against cancer."
