PNRR POC -Implementation of a predictive biomarker of response to regorafenib in glioblastoma

Scritto il 15 Novembre 2023.

The project focus on the validation of a candidate predictive biomarker of response to regorafenib in Glioblastoma (GBM) patients. Regorafenib is orally available inhibitor of multiple kinases (approved for treatment of metastatic colorectal cancer, gastrointestinal stromal tumors, and hepatocellular carcinoma), that show an encouraging activity in terms of overall survival in the treatment of recurrent GBM. The REGOMA translational study indicated that GBM patients with tumors expressing phosphorylated Acetyl-CoA Carboxylase (pACC, a well-known AMPK target) had much better therapeutic response to regorafenib and identified, by RNA sequencing, a molecular signature associated with improved survival upon regorafenib administration. The pACC marker has been patented as predictive biomarker of response to regorafenib in GBM.

The project aims not only to validate pACC in an independent cohort of GBM patients treated with regorafenib, but also to refine the technical assessment of the biomarker and better understand the mechanisms underlying its association with improved therapeutic response to this drug.