Multi-Omic Analysis of Rhabdomyosarcoma Points to Drug Targets in Deregulated Pathways

NEW YORK (GenomeWeb) – A team led by researchers at St. Jude Children’s Research Hospital has conducted an integrated analysis of rhabdomyosarcoma (RMS) samples that involved transcriptomic, epigenomic, proteomic, and phosphoproteomic data, leading them to a number of deregulated pathways.

Through subsequent preclinical testing, the researchers also found the WEE1 kinase to be a promising target for treating high-risk RMS.

The study, which is part of the St. Jude-Washington University Pediatric Cancer Genomic Project and was led by senior author Michael Dyer, chair of the Department of Developmental Neurobiology at St. Jude’s, was published in Cancer Cell today.

“This research offers a template for exploring the origins and vulnerabilities of solid tumors by looking not only at somatic mutations, but also at epigenetic changes and ultimately differences in how those changes are manifest in protein expression and activity,” Dyer said in a statement, adding that it “also highlights how preclinical models and extensive preclinical testing can help prioritize and streamline drug development.”

Rhabdomyosarcoma, a childhood solid tumor that involves muscle and soft tissue, affects about 350 patients in the US each year. While the cure rate is 75 percent for patients with localized tumors, survival is only 30 percent for

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