Could This Investigational Drug be the Latest Advancement for Cholangiocarcinoma?

According to a story from, Dr. Tara Seery is an investigator in a phase 3 trial called PROOF, and she has just one goal: finding better treatments for cholangiocarcinoma, a rare cancer affecting the bile ducts. Currently, patients have a very limited scope of treatments to choose from, but Dr. Seery envisions a future where cholangiocarcinoma can be treated effectively with a diverse array of targeted therapies.

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.

Testing a New Treatment

This clinical trial is testing an experimental treatment called infigratinib for cholangiocarcinoma that has metastasized (spread) or is not eligible for surgery can carries a genetic mutation affecting the FGFR2 gene. The drug recently earned the Fast Track designation from the US Food and Drug Administration (FDA), meaning it will be up for evaluation and approval more quickly.

Data from the research process so far has indicated that as many of 40 percent of patients carry mutations that could be therapeutic targets, and around 15 to 20 percent have FGFR2 mutations. This gene change tends to occur more often in white patients who are younger. In the phase 3 study, infigratinib will be compared side by side with the most common chemotherapy regimen of cisplatin and gemcitabine. 

In earlier study’s, infigratinib was able to provide meaningful differences in progression free survival in patients that had been previously treated with multiple other therapies; this trial will evaluate the drug in patients that have not been previously treated. 

FGFR2 is just one of the mutations in cholangiocarcinoma that could be a possible therapeutic target. Dr. Seery highlights the importance of patients seeking molecular testing following their diagnosis to determine what genetic mutations are present.

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