miRNA combination cancer therapy may limit toxicity and preserve effectiveness

What is it about?

In this study, we described a new approach to designing anti-cancer therapeutics that leverages combinations of miRNA. MiRNA are small, naturally occuring molecules that inhibit groups of mRNA. We applied this method to Ewing Sarcoma, an understudied rare cancer that primarily affects children and young adults.

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Photo by JJ Ying on Unsplash

Photo by JJ Ying on Unsplash

Why is it important?

miRNA therapeutics were considered a promising avenue for new anti-cancer treatments due to their documented anti-tumor activity in vivo and in vitro. Unfortunately, phase I human trials for miRNA therapeutics failed due to severe toxicity. MiRNA toxicity is likely driven in part by the large number of targets that each miRNA represses (sometimes called the "too many targets" problem). Inspired by radiation oncology, we propose a method to limit toxicity by using combinations of miRNA that jointly target mRNA of interest and don't jointly target other mRNA.