A study conducted at the University of Texas has discovered that combination therapy may be a viable treatment for cholangiocarcinoma with BRAF mutations. Researchers treated participants with both dabrafenib and trametinib. Results were positive, showing that this may be an option for patients with treatment-resistant and advanced cholangiocarcinoma.

About Cholangiocarcinoma

Cholangiocarcinoma is bile duct cancer. It is a rare cancer that typically affects people who are over age 50, but it has the potential to develop at any age. It is classified into three different types, depending on where the cancer occurs in the bile ducts. Intraheptic takes place in the part of the duct where the liver is. Due to its proximity to the liver, it is sometimes classified as a type of liver cancer. Hilar occurs right outside of the liver, and distal happens in the duct closest to the small intestine.

If one has cholangiocarcinoma they may experience jaundice, intense itching, fatigue, abdominal pain, white-colored stool, and unintentional weight loss. These symptoms occur due to a mutation in the DNA that cause cells to rapidly grow and multiply, forming a tumor. While the cause of cholangiocarcinoma is unknown, there are known risk factors that increase the likelihood of developing it. Older age, smoking, primary sclerosing cholangitis, chronic liver disease, a liver parasite, and bile duct problems that were present at birth are all risk factors.

Multiple tests may be performed to diagnose cholangiocarcinoma. A doctor may conduct a liver function test, a tumor marker test, an endoscopic retrograde cholangiopancreatography (ERCP), imaging tests, or a biopsy. Additional tests may be necessary to evaluate the extent and stage of the cancer. After a diagnosis is confirmed treatments include surgery, a liver transplant, chemotherapy, radiation, photodynamic therapy, and biliary drainage. Symptomatic care is also important for the treatment of this cancer.

About the Study

Researchers are investigating the combination therapy in an ongoing Phase II trial. It is open label and being conducted across multiple centers in an effort to evaluate safety and efficacy. 43 patients are enrolled, and as of now, 51% of them have achieved an overall response rate. 

Other results include a median progression free survival of 9.1 months, with overall survival at 13.5 months. Every patient experienced an adverse event, whether that was fatigue, nausea, vomiting, or fever. 

The combination therapy has proven to be a viable option for those with an advanced form of the disease, especially since prior treatments have not worked. In fact, BRAF inhibitors, which is what dabrafenib is, have been used as therapies for cholangiocarcinoma in the past with varying degrees of success. Combining these inhibitors with MEK inhibitors, which is what trametinib is, has been used to treat other cancers, which inspired medical professionals to investigate this combination for cholangiocarcinoma.

Hopefully the trial results continue to be positive, as there is an unmet medical need for those with advanced cholangiocarcinoma. If all goes well, this combination therapy could provide a viable treatment option.

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