Some existing anti-cancer drugs may act in part by targeting RNA, study shows

IMAGE: Professor Matthew Disney led the new study. view more 

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Credit: Scripps Research

JUPITER, FL – June 28, 2018 – Bolstering the notion that RNA should be considered an important drug-discovery target, scientists at Scripps Research have found that several existing, FDA-approved anti-cancer drugs may work, in part, by binding tightly to RNA, the regulators of the basic activities of life within cells. The research offers another approach for tackling diseases that have been considered “undruggable,” including amyotrophic lateral sclerosis (ALS), muscular dystrophy, cystic fibrosis and certain cancers.

“Known drugs made in the era when RNAs were not considered drug targets are, in fact, binding RNA, and causing some of the drug’s effects by modulating targets that were not previously considered,” says chemist Matthew D. Disney, PhD, professor on the Florida campus of Scripps Research, who led the study. “We found broad drug classes that bind RNA. There is reason to believe that not only could known drugs bind RNA in a disease setting, but there is more evidence that one should consider RNA as a target in drug-discovery efforts.”

While the universe of human proteins consists of about 20,000 varieties, the universe of human RNAs is closer to 200,000, potentially offering other effective


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