CX-5461 is primarily used in cancer research as a selective inhibitor of RNA polymerase I, which plays a key role in ribosomal RNA synthesis. By targeting this pathway, CX-5461 disrupts ribosome biogenesis in rapidly dividing cancer cells, leading to nucleolar stress and activation of p53-dependent and p53-independent tumor suppressor pathways. It has shown potent anti-tumor activity in hematological malignancies and certain solid tumors, especially in cancers with dysregulated MYC expression. Due to its unique mechanism of action, CX-5461 is being investigated in preclinical and early clinical trials as a potential therapeutic agent for cancers resistant to conventional treatments. Its ability to selectively target cancer cell transcription machinery without damaging DNA makes it a promising candidate for novel oncology therapies.