As we approach the fall season, it is wonderful to reflect on some of the amazing research presented this year, most recently at the ASCO 2023 annual meeting. One of the major highlights of ASCO this year included the results of HER2/neu-directed novel therapies in biliary cancer. Shubham Pant, MD, MBBS, from MD Anderson Cancer Center, presented the results of the HERIZON-1 clinical trial of the bispecific antibody zanidatamab in HER2/neu-expressing biliary cancers. A total of 80 patients with immunohistochemistry test score of 2+ or 3+ biliary cancers received this agent; the overall response rate was 41.3%, with a median response duration of 12.9 months and median time to first response of 1.8 months. These impressive results were associated with tolerable adverse events (diarrhea was the most common).
The BILCAP study has established the standard for adjuvant therapy in biliary tract cancer, and the analysis of genetic alterations from the study population was eagerly awaited. It is interesting to note that although FGFR2 fusions have been associated with an improved overall survival, no such correlation was noted in this study. EGFR amplification was associated with a worse clinical outcome and may represent a therapeutic target. Circulating tumor DNA may have a role in disease monitoring after resection, as was demonstrated by Changhoon Yoo, MD, PhD, from Asan Medical Center, in a correlative analysis of the STAMP adjuvant chemotherapy trial in Korea. Vaibhav Sahai, MBBS, MS, from the University of Michigan, presented the results of the BilT-02 phase 2 trial of the poly-ADP ribose polymerase inhibitor rucaparib and the PD-1 inhibitor nivolumab as maintenance therapy following first-line platinum-based chemotherapy in advanced biliary tract cancer. Although the trial did not meet the prespecified progression-free survival end point, the favorable overall survival argues for a randomized prospective trial in this setting.
Finally, this letter would not be complete without recognizing the remarkable efforts of the Cholangiocarcinoma Foundation’s (CCF) CholangioConnect initiative. This innovative program plays a crucial role in connecting caregivers with mentors and offers support through calls, emails, and virtual meetings. Patty Maxin assumed the role of the inaugural Program Manager for CholangioConnect, and under her leadership, the program has expanded its reach to 31 countries, fostering a network of more than 1000 patients and caregivers. We extend our heartfelt congratulations to Patty and the CCF for this exceptional accomplishment!
Sincerely,
Milind M. Javle, MD
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