MyPathway study proves that HER2 is a viable target for cholangiocarcinoma

This article is from NEJM Journal Watch.
HER2 Is a Viable Target in Biliary Tract Cancer.
HER2 is a viable target for cholangiocarcinoma.
Comment by David H.
Ilson, MD, PhD.
HER2 is targeted in patients with HER2-positive advanced cholangiocarcinoma.
The treatment achieved an objective response rate of 23%
.

More and more evidence supports the routine second-generation sequencing of patients with cholangiocarcinoma to identify genomic abnormalities that can be used as therapeutic targets
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Recently, drugs targeting the fibroblast growth factor receptor (FGFR) and isocitrate dehydrogenase 1 (IDH-1) pathways have been approved by regulatory agencies
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Now, researchers have published the results of a MyPathway study on a cohort of HER2-positive advanced cholangiocarcinoma patients, an open-label Phase 2 study of patients with advanced solid tumors who may have molecular changes that can be used as therapeutic targets.
Basket test
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Patients with advanced cholangiocarcinoma that had previously received treatment, immunohistochemistry, fluorescence in situ hybridization, or next-generation sequencing showed HER2 positive, and patients who had not previously received HER2-targeted therapy received pertuzumab combined with trastuzumab every 3 weeks Touzumab treatment
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Among 39 patients, 41% had previously received one treatment regimen, 44% had previously received two or more treatment regimens, 41% of the primary site was the gallbladder, and 36% of the primary site was intrahepatic or liver In addition, 80% were determined to be HER2 positive through gene amplification
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The response rate (primary endpoint) was 23%, and another 28% of patients reached stable disease
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The median duration of remission was 10.
8 months
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The median progression-free survival was 4.
0 months, and the overall survival was 10.
9 months
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Treatment-related grade 3 or higher adverse events are uncommon (8%)
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Comment This trial shows that in HER2-positive cholangiocarcinoma patients, HER2 is a viable target for the development of new drugs
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The positive result highlights the necessity of routine second-generation sequencing for patients with advanced cholangiocarcinoma
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Javle M et al.
Pertuzumab and trastuzumab for HER2-positive, metastatic biliary tract cancer (MyPathway): A multicentre, open-label, phase 2a, multiple basket study.
Lancet Oncol 2021 Sep; 22:1290.
(https: //doi.
org/10.
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