Pediatric Blood & Cancer: Insights into pediatric rhabdomyosarcoma research: Challenges and goals

Published On: June 21, 2019
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Marielle E Yohe 1, Christine M Heske 1, Elizabeth Stewart 2, Peter C Adamson 3, Nabil Ahmed 4, Cristina R Antonescu 5, Eleanor Chen 6, Natalie Collins 7, Alan Ehrlich 8, Rene L Galindo 9, Berkley E Gryder 1, Heidi Hahn 10, Sharon Hammond 11, Mark E Hatley 2, Douglas S Hawkins 12, Madeline N Hayes 13, Andrea Hayes-Jordan 14, Lee J Helman 15, Simone Hettmer 16, Myron S Ignatius 17, Charles Keller 18, Javed Khan 1, David G Kirsch 19, Corinne M Linardic 19, Philip J Lupo 4, Rossella Rota 20, Jack F Shern 1, Janet Shipley 21, Sivasish Sindiri 1, Stephen J Tapscott 22, Christopher R Vakoc 23, Leonard H Wexler 5, David M Langenau 13

Abstract

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.

Keywords: cancer biology; early-phase clinical trials; genomics; rhabdomyosarcoma.

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