Biological Advances and Current Challenges for Pediatric Rhabdomyosarcoma

Hebron, Katie E, Odeniyide, Patience, Wei, Yun, Gryder, Berkley E, Barr, Frederic G, Casey, Dana L, Chen, Eleanor Y, Crompton, Brian D, Dela Cruz, Filemon S, Durbin, Adam D, Ford, Heide L, Gatz, Susanne A, Hatley, Mark E, Henssen, Anton G, Hettmer, Simone, Houghton, Peter J, Kendall, Genevieve C, Khan, Javed, Lupo, Philip J, Patel, Anand G, Pomella, Silvia, Rota, Rossella, Schito, Marco, Schoot, Reineke A, Shern, Jack F, Stanton, Benjamin Z, Stewart, Elizabeth A, Swindlehurst, Cathy A, Thomas, Craig J, Vakoc, Christopher R, Vaseva, Angelina V, Venkatramani, Rajkumar, Wexler, Leonard H, Yustein, Jason T, Hammond, Sharon, Heske, Christine M, Langenau, David M, Linardic, Corinne M, Ignatius, Myron S, Yohe, Marielle E (March 2026) Biological Advances and Current Challenges for Pediatric Rhabdomyosarcoma. Cancers, 18 (6). p. 888. ISSN 2072-6694

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Abstract

Despite comprehensive and multi-modal therapy, outcomes for children and adolescents with rhabdomyosarcoma (RMS) have plateaued over the past four decades. This is not for a lack of progress in the basic and translational studies of RMS. Indeed, advances in animal models and/or patient tissue sample acquisition and analysis have improved our understanding of RMS biology. Large-scale sequencing efforts have generated transcriptomic, genomic, and epigenomic datasets that highlight the heterogeneity of RMS and have the potential to improve prognostication and the application of precision medicine in patients with RMS. However, few of these discoveries have been clinically translated, and limitations to the accessibility, uniformity, and application of these new models and datasets hinder their utility. Here, we discuss how advances in understanding RMS biology, optimization of preclinical models, and strategies for translating basic science discoveries to the clinic can potentially improve outcomes for patients with RMS.

Item Type: Paper
Subjects: diseases & disorders > cancer
diseases & disorders
diseases & disorders > cancer > cancer types > rhabdomyosarcoma
diseases & disorders > cancer > cancer types
CSHL Authors:
Communities: CSHL labs > Vakoc lab
SWORD Depositor: CSHL Elements
Depositing User: CSHL Elements
Date: 10 March 2026
Date Deposited: 30 Mar 2026 12:51
Last Modified: 30 Mar 2026 12:51
PMCID: PMC13023981
Related URLs:
URI: https://repository.cshl.edu/id/eprint/42127

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