Adjuvant Chemoradiation and Immunotherapy for Extrahepatic Cholangiocarcinoma and Gallbladder Cancer: A Randomized Clinical Trial
In August 2021, Tibsovo (ivosidenib), the first IDH inhibitor for cholangiocarcinoma, was approved by the FDA.
The TargetCancer Foundation (TCF) has invested $1.1 million in cholangiocarcinoma research since 2011, focusing on basic research tools, driver mutations, and treatment options. TCF’s support enabled the development of a comprehensive cell line bank, patient‑derived xenografts, and mouse models. Key discoveries include the functional impact of IDH1/IDH2 mutations (10‑15% of intrahepatic cholangiocarcinoma) and FGFR2 alterations (≈15%). These findings underpinned clinical trials that led to the first FDA‑approved targeted therapies: Tibsovo (ivosidenib), an IDH inhibitor approved August 2021, and Pemazyre (pemigatinib), an FGFR2 inhibitor approved April 2020. TCF remains active in researching resistance mechanisms and integrating immunotherapy. Several high‑impact papers in journals such as *Cancer Discovery* and *Nature* were supported by TCF grants.
Tibsovo’s approval represents the first approved precision medicine therapy for cholangiocarcinoma, improving survival for patients with IDH1/2‑mutated tumors.
Evidence level: Korai humán adat. Kis vagy feltáró emberi adat.
Related signals
- The Cholangiocarcinoma in the Young (CITY) Study: Tumor Biology, Treatment Patterns, and Survival Outcomes in Adolescent Young Adults With Cholangiocarcinoma
- Scientists build cell line atlas of deadly cancers of the bile ducts and gallbladder
- Cholangiocarcinoma Workup: Laboratory Studies, Imaging Studies, Procedures
- New EASL Clinical Practice Guidelines on extrahepatic cholangiocarcinoma
- An open‑label study of pemigatinib in cholangiocarcinoma: final results from FIGHT‑202