CX-5461 is primarily used in research settings to study its potential as a therapeutic agent. It is known to inhibit RNA polymerase I, which plays a crucial role in the synthesis of ribosomal RNA. This inhibition can lead to the disruption of ribosome biogenesis, making it a promising candidate for targeting cancer cells that rely heavily on rapid ribosome production for their growth and proliferation.

In preclinical studies, CX-5461 has shown efficacy in treating certain types of cancers, including hematological malignancies and solid tumors. It has been investigated for its ability to induce apoptosis in cancer cells while sparing normal cells, highlighting its potential for selective cancer therapy.

Additionally, CX-5461 is being explored for its role in combination therapies, where it may enhance the effects of other anticancer drugs. Its mechanism of action also makes it a valuable tool for studying ribosomal biology and the cellular stress response in various experimental models.

Overall, CX-5461 holds significant promise in oncology research and drug development, with ongoing studies aimed at further understanding its therapeutic potential and optimizing its use in clinical settings.