Experimental Drug Displays Potential for Treating Intrahepatic Cholangiocarcinoma in Clinical Trial

According to a story from Business Wire, Basilea Pharmaceutica, Ltd., a partner of ArQule, Inc., recently released interim results from its Phase 2 clinical trial testing the company’s investigational drug derazantinib. In this trial, the treatment was being tested as a therapy for intrahepatic cholangiocarcinoma. The data indicates that derazantinib has significant potential in treating this cancer and it continues to display favorable tolerability and safety characteristics as indicated in earlier testing.

About Cholangiocarcinoma

Cholangiocarcinoma is a form of cancer that affects the bile ducts. More specifically, the epithelial cells of the bile duct are the ones affected by this disease. The cause is not directly known; many patients who develop the disease also have no known risk factors, but there are some factors that are known to increase risk, such as primary sclerosing cholangitis, chronic liver diseases such as hepatitis, certain genetic disorders such as Caroli’s syndrome, infection with Helicobacter bacteria, and certain liver flukes, a type of parasitic animal that infiltrates the bile ducts and feeds on bile. Symptoms include fever, weight loss, jaundice, itching, and abdominal pain. Treatment may include surgery, chemotherapy, radiation, or liver transplant. Transplant is usually only useful if the disease is detected early; surgery is the best chance for curing cholangiocarcinoma. This disease is rapidly lethal if surgical removal is not possible. To learn more about cholangiocarcinoma, click here.

Interim Results

In this ongoing study of 42 patients, a total of 29 patients were currently eligible for evaluation. In this group of participants, the objective response rate achieved was 21 percent. However, the overall disease control rate was 83 percent. Disease control rate describes who had at least a partial response to treatment with derazantinib or whose disease was stabilized. 

About Derazantinib

Derazantinib is classified as an FGFR kinase small molecule inhibitor. It displays this activity against multiple types of FGFR kinases. These kinases play a critical role in the growth and proliferation of cells. Alterations of the kinases have the potential to be valuable therapeutic targets in a number of cancers, including cholangiocarcinoma. Alterations such as gene fusions may be present in as many as 22 percent of intrahepatic cholangiocarcinoma cases, so patients that express these alterations should potentially benefit from treatment with derazantinib.


Share this post

We believe rare disease patients are people, not a diagnosis. Through education, awareness and some humor, we help patients, caregivers and support persons by providing relevant and often inspirational news and stories.
Our goals are to share stories, cultivate strong community, provide the latest medical findings, connect people and pioneer production of patient worthy information. Help us attain these goals by telling us a little bit about yourself!

© Copyright Patient Worthy

Close Menu