A new biologic drug administered to certain patients with precision medicine techniques could give new hope in understanding and treating intrahepatic cholangiocarcinoma (ICCA). It is the result of a study by researchers of the National Institute of Gastroenterology Saverio De Bellis of Castellana Grotte (Bari) on a rare and highly malignant tumor, with an unfavorable prognosis, orphan of effective medical therapy.
The only therapeutic possibility today is represented by surgery limited to the early diagnosis, but with an efficacy that is still unsatisfactory for possible relapses. New hopes derive from the precision medicine-based approach which has proposed the use of new biological drugs directed against specific targets (mutation of the IDH1 and FGFR2 genes) usable in patients with appropriate molecular characteristics. The study by the researchers from Bari was published in the international scientific journal Journal experimental clinical cancer research.
“Our study – explains the scientific director of Irccs Gianluigi Giannelli – demonstrates that the NOTCH signaling pathway stimulates CCA cells to express the CD90 receptor on their surface. Furthermore, patients with intrahepatic CCA expressing CD90 have a worse prognosis. However, these are the tumors that most benefit from a therapy with Crenigacestat, a drug that blocks the NOTCH signaling pathway. “
The drug is currently undergoing clinical trials in various solid and liquid neoplasms and the Irccs of Castellaneta is the only site in Puglia where phase 1 clinical trials can be performed.