Cancer of the biliary tract - is a rare aggressive malignancy with a poor prognosis. A new study shows that in the subgroup of patients with tumors with mutations in the BRAF V600E, treated with a combination of targeted agents has been effective.
Used a combination of drugs - BRAF kinase inhibitor dabrafenib (pharmaceutical company Novartis) and MEK inhibitor trametinib (Novartis) has already been approved for use in melanoma mutations BRAF V600.
The results of the combination of drugs in patients with the BRAF-mutated biliary tract cancer were presented at the Symposium on gastrointestinal cancer (GICS) 2019. The average progression-free survival was 9.2 months, and overall survival - 11.7 months. Efficacy seen in this group of patients is comparable to the efficiency of first-line chemotherapy with gemcitabine and cisplatin.
Statistically overall 5-year survival for patients with biliary tract cancer is about 15%, but the rate of recurrence and prognosis may vary. Currently, treatment involves resection and chemotherapy with gemcitabine and cisplatin.
Dabrafenib patients were given 150 mg twice a day plus trametinib 2 mg once daily and treatment continued until the appearance of toxicity, disease progression or death. Secondary effects of combination therapy was 6 months (range: 2-32 months), and 86% of the cohort continued therapy for more than 3 months. Also, genetic analysis was performed using sequencing target 16 collected at baseline tissue samples. The most common side effects associated with treatment were pyrexia (40%), rash (29%), nausea, diarrhea and fatigue (23%) and chills (20%).
In the study, Heinz-Josef Lenz, MD, professor of medicine and preventive medicine at the Keck School of Medicine of the University of Los Angeles, said the results were impressive.
He noted that tools such as a liquid biopsy, in the future will be able to determine treatment, and stressed the importance of incorporating the practice of sequencing the entire genome and other types of molecular profiling.
"We should consider expanding the sequencing of the entire genome to identify new ways, new genes, mutations and the use of RNA - says Lenz. - There is no doubt that a comprehensive molecular characterization of DNA and RNA will lead to better treatment options and patient identification. "