Clinical trials for a precision therapy based on Dabrafenib and Trametinib showed remarkable results in patients suffering from a rare type of gallbladder carcinoma. The drug combination elicited a positive response in approximately 40% of participants and managed to stabilize the disease in 40 to 45% of the rest.
“Nearly every patient had some tumor reduction“, noted Zev Wainberg, MD, from the University of California Los Angeles, while presenting his findings at the Gastrointestinal Cancers Symposium. “The duration of response at 6 months was 66%. Many of the patients who had stable disease also had durable clinical benefit“.
Even though it accounts for only 5% of all tract tumors overall, BRAF-mutated biliary tract cancer is strongly associated with poor prognosis, having a five-year survival rate of 15%. Standard treatment involves surgery and chemotherapy (generally with Gemcitabine) and offers about 8 months of progression-free survival (PFS) with an overall survival rate (OS) of 11.7 months. The chances of recurrence are considered high.
The treatment was well received and reported no new or unexpected adverse events. The most common reactions were pyrexia, rash, nausea, diarrhea, fatigue, and chills, leading to some dose reductions.
“These results represent the first prospectively analyzed cohort of patients with BRAF V600E-mutated BTC treated with a combination of BRAF and MEK inhibition”, added Dr. Weinberg. “BRAF V600 is one of several actionable driver mutations in this disease, and should be considered for routine testing in patients with BTCs”.
For their study, the team analyzed several baseline tissue samples and sequenced 570 cancer specific genes. “There were a number of genetic alterations seen in these patients, both mutations and amplifications, and there’s little in common between them”, suggesting the theory of a diverse molecular phenotype. In fact, all patients had less than 6 mutations per megabase of genome examined.
With this new insight regarding the tumor microenvironment, especially pertaining to signaling between normal tissue, stromal tissue, and tumor tissue, researchers are expecting new therapeutic strategies to emerge.
The oncology field is continuously evolving, uncovering more information about the diseases while at the same time improving patient care.
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