A new drug is shown to be effective in aggressive cancer within the liver

by time news

A clinical trial has shown that a new generation drug is effective in the treatment of intrahepatic cholangiocarcinomaa cancer that affects the internal ducts of the liver and has few therapeutic options in an advanced stage.

The results of this trial, in which researchers from the Vall d’Hebron Institute of Oncology (VHIO)have been published in the New England Journal of Medicine.

Cancer in the bile ducts

Intrahepatic cholangiocarcinoma is a type of cancer that forms in the thin tubes (bile ducts) that carry bile from the digestive fluid and They connect the liver to the gallbladder and the small intestine.

Once chemotherapy was applied when the disease appears, currently there are no other standardized options when that first treatment does not work and the cancer progresses to an advanced stage.

Between 10 and 20% of patients with this disease have an alteration of the FGFR2 gene, which is targeted by this new drug, futibatinib.

clinical benefit

The results of the phase II clinical trial FOENIX-CCA2 show that the drug provides a clinical benefit in patients with intrahepatic cholangiocarcinoma with a fusion in the FGFR2 gene who had already received chemotherapy treatment

About a sample of 103 patients with intrahepatic cholangiocarcinoma inoperable or metastatic and alterations in the FGRF2 gene, 43 of them (42%) responded favorably and the mean duration of response was 9.7 months.

The median progression-free survival (the time between the start of treatment and the regrowth of the tumor) was 9 months and overall survival of 21.7 months.


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The patients’ quality of life remained stable for an average of 9 months after starting treatment.

“These results indicate that we are facing a new precision medicine treatment for patients with intrahepatic cholangiocarcinoma with this determined genetic profile”, in a disease characterized by being “orphan” of treatmentshas highlighted the director of VHIO and co-author of the study, Josep Tabernero

He has indicated that there are other investigations that are testing different treatments for intrahepatic cholangiocarcinoma patients with other genetic profiles, with the aim that science can offer different subtypes of patients the treatment that will provide the most clinical benefit.


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