4 targeted therapies for Bile Duct Cancer(Cholangiocarcinoma)

Targeted therapies are drugs that specifically target certain proteins or genetic mutations in cancer cells, which can help to slow down or stop their growth. In cholangiocarcinoma, some of the targeted therapies that have been studied or used include:

FGFR inhibitors

Some cholangiocarcinomas have alterations in the FGFR2 gene. Drugs that target these alterations, such as pemigatinib and infigratinib, have shown promise in treating patients with these specific genetic mutations.

Infigratinib^[2]

On May 28, 2021, the Food and Drug Administration granted accelerated approval to infigratinib (Truseltiq, QED Therapeutics, Inc.), a kinase inhibitor for adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.

Futibatinib^[3]

On September 30, 2022, the Food and Drug Administration granted accelerated approval to futibatinib (Lytgobi, Taiho Oncology, Inc.) for adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.

IDH inhibitors

Mutations in the IDH1 or IDH2 genes can be found in a subset of cholangiocarcinoma patients. Ivosidenib is an IDH1 inhibitor that has been studied for use in this population.

Ivosidenib^[4]

On August 25, 2021, the Food and Drug Administration approved ivosidenib (Tibsovo, Servier Pharmaceuticals LLC) for adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

Others

This is not an exhaustive list, and new targeted therapies are continuously being researched and developed. It is important to note that not all cholangiocarcinoma patients will be suitable for targeted therapies, as they are often specific to certain genetic mutations or proteinsling.

EGFR inhibitors

Epidermal growth factor receptor (EGFR) is a protein found on the surface of some cancer cells, including some cholangiocarcinomas. Drugs targeting EGFR, such as erlotinib, have been studied in cholangiocarcinoma with varying results.

BRAF inhibitors

Some cholangiocarcinomas have mutations in the BRAF gene. Targeted therapies like vemurafenib or dabrafenib, which inhibit the BRAF protein, have been used in combination with other drugs, such as MEK inhibitors, to treat patients with these mutations.

Immune checkpoint inhibitors

Some cholangiocarcinomas express PD-L1, a protein that can be targeted by immune checkpoint inhibitors like pembrolizumab or nivolumab to help the immune system recognize and attack cancer cells.

Multi-kinase inhibitors

Drugs like regorafenib, sorafenib, and cabozantinib, which target multiple kinases involved in cancer cell growth and survival, have been studied in cholangiocarcinoma with mixed results.