@article{1c567b4e80644aeb9355818b0938fabf,
title = "Insights into pediatric rhabdomyosarcoma research: Challenges and goals",
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",
author = "Yohe, {Marielle E.} and Heske, {Christine M.} and Elizabeth Stewart and Adamson, {Peter C.} and Nabil Ahmed and Antonescu, {Cristina R.} and Eleanor Chen and Natalie Collins and Alan Ehrlich and Galindo, {Rene L.} and Gryder, {Berkley E.} and Heidi Hahn and Sharon Hammond and Hatley, {Mark E.} and Hawkins, {Douglas S.} and Hayes, {Madeline N.} and Andrea Hayes-Jordan and Helman, {Lee J.} and Simone Hettmer and Ignatius, {Myron S.} and Charles Keller and Javed Khan and Kirsch, {David G.} and Linardic, {Corinne M.} and Lupo, {Philip J.} and Rossella Rota and Shern, {Jack F.} and Janet Shipley and Sivasish Sindiri and Tapscott, {Stephen J.} and Vakoc, {Christopher R.} and Wexler, {Leonard H.} and Langenau, {David M.}",
note = "Funding Information: We thank the clinicians, basic scientists, and translational researchers as well as patients, their families, and representatives from five RMS patient advocacy groups (Summer{\textquoteright}s Way, Focus on Rhabdo, Friends of TJ, Super Sam Foundation, Rein in Sarcoma, and Sarcoma Patient Alliance Coalition) that assembled for the Summer{\textquoteright}s Way RMS workshop in May 2018. Many are authors on this consensus article. The authors of this article certify they have no conflicts of interest. We are also grateful to S.X. Skapek for attendance at the Summer{\textquoteright}s Way Rhabdomyosarcoma workshop and critical reading of the manuscript. This was the third such international workshop to follow those previously held at the Banbury Center at Cold Spring Harbor Laboratory in May of 201469 and the National Cancer Institute in April of 2015. This work was supported by R24OD016761 (D.M.L.), R01CA154923 (D.M.L.), R01CA215118, (D.M.L.), R01 R01CA226926 (D.M.L), and the Liddy Shriver Sarcoma Initiative. Funding Information: We thank the clinicians, basic scientists, and translational researchers as well as patients, their families, and representatives from five RMS patient advocacy groups (Summer's Way, Focus on Rhabdo, Friends of TJ, Super Sam Foundation, Rein in Sarcoma, and Sarcoma Patient Alliance Coalition) that assembled for the Summer's Way RMS workshop in May 2018. Many are authors on this consensus article. The authors of this article certify they have no conflicts of interest. We are also grateful to S.X. Skapek for attendance at the Summer's Way Rhabdomyosarcoma workshop and critical reading of the manuscript. This was the third such international workshop to follow those previously held at the Banbury Center at Cold Spring Harbor Laboratory in May of 2014 and the National Cancer Institute in April of 2015. This work was supported by R24OD016761 (D.M.L.), R01CA154923 (D.M.L.), R01CA215118, (D.M.L.), R01 R01CA226926 (D.M.L), and the Liddy Shriver Sarcoma Initiative. Publisher Copyright: {\textcopyright} 2019 Wiley Periodicals, Inc.",
year = "2019",
month = oct,
doi = "10.1002/pbc.27869",
language = "English (US)",
volume = "66",
journal = "Medical and Pediatric Oncology",
issn = "1545-5009",
publisher = "Wiley-Liss Inc.",
number = "10",
}