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Cholangiocarcinoma Emerges as Key Target of FGFR Inhibitor Research

Angelica Welch
Published: Wednesday, Dec 19, 2018
Sameek Roychowdhury, MD, PhD

Sameek Roychowdhury, MD, PhD
Recent data have shown that fusions in FGFR1/2/3 can be targetable in many cancers, particularly in cholangiocarcinoma. FGFR inhibitors are now being tested in clinical trials of patients with these fusions.


OncLive: What is the importance of FGFR inhibitors in cholangiocarcinoma?

Roychowdhury: FGFR gene fusions, sometimes called translocations or rearrangements, have emerged as a significant target for therapy, not only in cholangiocarcinoma, but also in other cancer types. The most common gene fusion in cholangiocarcinoma that is intrahepatic is FGFR2, but we have also seen FGFR1 and FGFR3 in other cancer types, such as urothelial carcinoma, pancreatic cancer, and breast cancer. For each of these cancer types, especially cholangiocarcinoma, early trials involving inhibitors of the FGFR gene, which is a receptor tyrosine kinase, [have] emerged.

-driving genetic alterations. Finding these fusions has allowed patients to qualify for an FGFR inhibitor of some kind and benefit [from it]. As we [do genetic testing on] more and more patients, we are going to find more candidates for testing.

Should FGFR inhibitors be tested in a tumor-agnostic fashion?

As a principle, companies developing and driving FGFR inhibitors are interested in getting these drugs to patients who will benefit. My understanding is that they are interested in patients who have marker-positive cancer, regardless of cancer type. There are definitely disease-specific studies; however, these markers are not limited to patients who have a certain cancer type. We are seeing them in multiple tumor types, and patients are benefitting. The landscape of FGFR drug development isn't limited to 1 or 2 cancer types; any cancer type with a driving alteration in FGFR could be a candidate.
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