Mitoxantrone reshapes RNA, boosting function
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- Danny Incarnato and his team at the University of Groningen published a study on March 23 in Nature Communications showing that small molecules that bind RNA rarely affect its function.
- Mitoxantrone, an FDA‑approved cancer drug, was shown to stabilize a specific RNA shape, altering the RNA folding ensemble and producing functional consequences.
- Risdiplam, the only mRNA‑targeting small‑molecule drug on the market for spinal muscular atrophy, highlights the limited success of RNA‑targeting therapeutics.
- The paper outlines a framework for identifying small‑molecule drugs that reshape RNA structures, calling for broader whole‑cell studies.
- Incarnato emphasizes that future drug design should focus on molecules that can change RNA conformation, not merely bind to it.
Why it matters: Drug developers gain a concrete design principle—target RNA shape change—to create more effective RNA‑targeting therapeutics, while the existing market, limited to a single approved mRNA drug, stands to expand as new molecules are engineered and could address unmet disease needs.
