A multiplex preclinical model for adenoid cystic carcinoma of the salivary gland identifies regorafenib as a potential therapeutic drug

Rákkutatás · Adenoid cystic carcinoma · EGFR

Regorafenib is a potential therapeutic drug for adenoid cystic carcinoma of the salivary gland

Adenoid cystic carcinomas (ACC) are rare salivary gland cancers with a high incidence of metastases. In order to study this tumor type, a reliable model system exhibiting the molecular features of this tumor is critical, but none exists, thereby inhibiting in‑vitro studies and the analysis of metastatic behavior. To address this deficiency, we have coupled an efficient method to establish tumor cell cultures, conditional reprogramming (CR), with a rapid, reproducible and robust in‑vivo zebrafish model. We have established cell cultures from two individual ACC PDX tumors that maintain the characteristic MYB translocation. Additional mutations found in one ACC culture also seen in the PDX tumor. Finally, the CR/zebrafish model mirrors the PDX mouse model and identifies regorafenib as a potential therapeutic drug to treat this cancer type that mimic the drug sensitivity profile in PDX model, further confirming the unique advantages of multiplex system.

ACC is a rare, highly metastatic salivary gland cancer lacking effective systemic therapies; this study establishes a faithful preclinical platform that replicates human tumor biology and demonstrates regorafenib as a promising treatment, thereby accelerating translational research and potential clinical trial design for this orphan disease.

Bizonyítékszint: Korai humán adat. Kis vagy feltáró emberi adat.

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