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RBA1 is a preclinical small-molecule inhibitor of the RNA-binding protein HuR (ELAVL1), developed for the treatment of glioblastoma (GBM) and other resistant cancers, including lung and breast cancers. It is the lead compound of the resorcinol-benzil-amide (RBA) series. HuR acts as a key post-transcriptional regulator that stabilizes messenger RNAs (mRNAs) encoding pro-inflammatory cytokines, inflammatory enzymes, and acute-phase proteins, thereby promoting cancer hallmarks such as proliferation, invasion, metastasis, and apoptosis resistance. By disrupting the HuR-mRNA interaction, RBA1 induces apoptosis and exhibits cytotoxic activity in glioblastoma patient-derived cell lines, as well as lung and breast cancer cell lines.
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