MTL-CEBPA + sorafenib

MTL-CEBPA + sorafenib is a combination therapy under clinical investigation for **advanced hepatocellular carcinoma (HCC)**, especially in patients with prior resistance to systemic therapy and/or of viral hepatitis etiology. MTL-CEBPA is a first-in-class small activating RNA (saRNA) oligonucleotide designed to upregulate expression of the CCAAT/enhancer-binding protein alpha (C/EBP-α), a master regulator of hepatic and myeloid cell differentiation. This activation shifts myeloid lineage towards reduced immunosuppression by myeloid-derived suppressor cells (MDSCs) within the tumor microenvironment, potentially enhancing anti-tumor immune responses and the efficacy of tyrosine kinase inhibitors (TKIs) like sorafenib. Sorafenib is a multikinase inhibitor that targets RAF kinase and vascular endothelial growth factor receptors (VEGFR), blocking tumor cell proliferation and angiogenesis. The combination has demonstrated greater objective response rates and complete response rates in preliminary trials compared to sorafenib monotherapy, and is the subject of ongoing phase 2 clinical trials.

Brand names

Nexavar

Other names

MTL-CEBPA plus sorafenibMTL-CEBPA and sorafenib