New study tests drug combination for treatment of biliary tract cancer

Anatomy of the extrahepatic bile ducts

Anatomy of the extrahepatic bile ducts; drawing shows the liver, right and left hepatic ducts, gallbladder, cystic duct, common hepatic duct (hilum region), common bile duct (distal region), extrahepatic bile duct, pancreas, and small intestine. An inset shows the liver, bile ducts, and gallbladder.
Image credit: Terese Winslow

Adults with biliary tract cancer with a mutation of the KRAS gene may be eligible to participate in a clinical trial at the NIH Clinical Center.

Tim F. Greten, M.D., Deputy Chief of the Thoracic and GI Malignancies Branch, is leading a study of a drug combination that may prolong survival in adults with biliary tract cancer (BTC). BTC is a cancer that arises from the bile ducts that carry bile, a digestive fluid, through the liver. It’s a rare, but fast-growing cancer that often has an abnormal or mutated KRAS gene. There are few treatment options for BTC. Study participants will take trametinib and hydroxychloroquine (HCQ) tablets every day in 28-day cycles. Trametinib blocks the action of MEK, a signaling chemical that helps cells multiply and survive. However, cancer cells can get around trametinib’s MEK blockade by boosting a process called autophagy. Autophagy is the body’s way of eliminating damaged cells in order to generate new cells. HCQ inhibits autophagy so that cancer cells damaged by trametinib can’t regenerate themselves. In pre-clinical studies, the combination of trametinib and HCQ showed ability to shrink tumors. Researchers want to study  how this drug combination  may be effective in  BTC with mutated KRAS gene. identifier: NCT04566133

NCI Protocol ID: NCI-20-C-0152

Official Title: Combination of Trametinib (MEK Inhibitor) and Hydroxychloroquine (HCQ) (Autophagy Inhibitor) in Patients with KRAS Mutation Refractory Bile Tract Carcinoma (BTC)

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Posted on Tue, 08/17/2021