[HTML][HTML] CX-5461 activates the DNA damage response and demonstrates therapeutic efficacy in high-grade serous ovarian cancer

E Sanij, KM Hannan, J Xuan, S Yan, JE Ahern… - Nature …, 2020 - nature.com
E Sanij, KM Hannan, J Xuan, S Yan, JE Ahern, AS Trigos, N Brajanovski, J Son, KT Chan
Nature communications, 2020nature.com
Acquired resistance to PARP inhibitors (PARPi) is a major challenge for the clinical
management of high grade serous ovarian cancer (HGSOC). Here, we demonstrate CX-
5461, the first-in-class inhibitor of RNA polymerase I transcription of ribosomal RNA genes
(rDNA), induces replication stress and activates the DNA damage response. CX-5461 co-
operates with PARPi in exacerbating replication stress and enhances therapeutic efficacy
against homologous recombination (HR) DNA repair-deficient HGSOC-patient-derived …
Abstract
Acquired resistance to PARP inhibitors (PARPi) is a major challenge for the clinical management of high grade serous ovarian cancer (HGSOC). Here, we demonstrate CX-5461, the first-in-class inhibitor of RNA polymerase I transcription of ribosomal RNA genes (rDNA), induces replication stress and activates the DNA damage response. CX-5461 co-operates with PARPi in exacerbating replication stress and enhances therapeutic efficacy against homologous recombination (HR) DNA repair-deficient HGSOC-patient-derived xenograft (PDX) in vivo. We demonstrate CX-5461 has a different sensitivity spectrum to PARPi involving MRE11-dependent degradation of replication forks. Importantly, CX-5461 exhibits in vivo single agent efficacy in a HGSOC-PDX with reduced sensitivity to PARPi by overcoming replication fork protection. Further, we identify CX-5461-sensitivity gene expression signatures in primary and relapsed HGSOC. We propose CX-5461 is a promising therapy in combination with PARPi in HR-deficient HGSOC and also as a single agent for the treatment of relapsed disease.
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