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news.Cylene Pharmaceuticals announced that research collaborators at the Peter MacCallum Cancer Centre in Melbourne, Australia, have established that RNA Polymerase I (Pol I) activity is essential for cancer cell survival and its inhibition selectively activates p53 to kill tumors.
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The studies were also conducted in in vivo models of blood cancers and confirmed that the drug removed malignant cells from the bloodstream, while allowing normal healthy blood cells to grow, thus differentiating CX-5461 from genotoxic treatments.
According to Cylene, targeting cancer’s dependence upon Pol I to trigger cancer-specific activation of p53 is a new approach to cancer therapy.
Cylene president and CEO William Rice said, "Building on our mechanistic understanding of CX-5461, we have identified specific genetic markers to select patient populations with the most sensitive solid tumor or hematological cancers."
"The combination of cancer’s reliance on Pol I, the impressive preclinical activity of CX-5461, the development of clear predictive and prognostic biomarkers and the novelty of the therapeutic strategy is compelling," Dr Rice added.
"As such, a First-in-Human clinical trial with CX-5461 is planned in collaboration with our colleagues at Peter Mac later this year."
The results suggest that selective activation of a surveillance pathway to activate p53, using Pol I inhibitors such as CX-5641, is likely to be therapeutically useful in the treatment of a range of tumors.