Novel therapeutics peptide targeting C16orf74 in biliary tract cancer

Biliary tract cancer (BTC) is a highly aggressive malignancy with limited therapeutic options and poor prognosis. Chromosome 16 open reading frame 74 ( C16orf74 ) gene has been associated with tumor progression and poor outcomes. This study aimed to evaluate the prognostic significance of C16orf74 in BTCs. To this end, 146 surgically resected BTC specimens were retrospectively analyzed. BTC cell lines were used to assess the biological role of C16orf74 through RNA expression analysis, cell proliferation assays, and migration and invasion studies. The therapeutic efficacy of a dimer-blocking (DB) peptide was evaluated in vitro and in vivo using a subcutaneous tumor model in nude mice. High C16orf74 expression was observed in 45% of BTC cases and significantly associated with poorer overall survival (5-year survival: 13.6% vs. 30.0%, p =0.033). Multivariate analysis identified both C16orf74 expression and lymph node metastasis as independent prognostic factors (relative risk 3.67). In BTC cell lines, DB inhibited proliferation, migration, and invasion in C16orf74 high-expressing cells. In vivo, DB significantly reduced tumor volume in a subcutaneous BTC model. C16orf74 is a poor prognostic marker for BTCs, and its inhibition using DB demonstrates therapeutic potential. Targeted therapies against C16orf74 could improve outcomes for BTC patients, particularly those with high expression.