Insights into pediatric rhabdomyosarcoma research: Challenges and goals

Marielle E Yohe, Christine M Heske, Elizabeth Stewart, Peter C Adamson, Nabil Ahmed, Cristina R Antonescu, Eleanor Chen, Natalie Collins, Alan Ehrlich, Rene L Galindo, Berkley E Gryder, Heidi Hahn, Sharon Hammond, Mark E Hatley, Douglas S Hawkins, Madeline N Hayes, Andrea Hayes-Jordan, Lee J Helman, Simone Hettmer, Myron S IgnatiusCharles Keller, Javed Khan, David G Kirsch, Corinne M Linardic, Philip J Lupo, Rossella Rota, Jack F Shern, Janet Shipley, Sivasish Sindiri, Stephen J Tapscott, Christopher R Vakoc, Leonard H Wexler, David M Langenau

Research output: Contribution to journalReview articlepeer-review


Overall survival rates for pediatric patients with high-risk or relapsed rhabdomyosarcoma (RMS) have not improved significantly since the 1980s. Recent studies have identified a number of targetable vulnerabilities in RMS, but these discoveries have infrequently translated into clinical trials. We propose streamlining the process by which agents are selected for clinical evaluation in RMS. We believe that strong consideration should be given to the development of combination therapies that add biologically targeted agents to conventional cytotoxic drugs. One example of this type of combination is the addition of the WEE1 inhibitor AZD1775 to the conventional cytotoxic chemotherapeutics, vincristine and irinotecan.

Original languageEnglish
Pages (from-to)e27869
JournalPediatric Blood and Cancer
Publication statusE-pub ahead of print - Jun 21 2019


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