Literature DB >> 31222885

Insights into pediatric rhabdomyosarcoma research: Challenges and goals.

Marielle E Yohe1, Christine M Heske1, Elizabeth Stewart2, Peter C Adamson3, Nabil Ahmed4, Cristina R Antonescu5, Eleanor Chen6, Natalie Collins7, Alan Ehrlich8, Rene L Galindo9, Berkley E Gryder1, Heidi Hahn10, Sharon Hammond11, Mark E Hatley2, Douglas S Hawkins12, Madeline N Hayes13, Andrea Hayes-Jordan14, Lee J Helman15, Simone Hettmer16, Myron S Ignatius17, Charles Keller18, Javed Khan1, David G Kirsch19, Corinne M Linardic19, Philip J Lupo4, Rossella Rota20, Jack F Shern1, Janet Shipley21, Sivasish Sindiri1, Stephen J Tapscott22, Christopher R Vakoc23, Leonard H Wexler5, David M Langenau13.   

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.
© 2019 Wiley Periodicals, Inc.

Entities:  

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

Mesh:

Year:  2019        PMID: 31222885      PMCID: PMC6707829          DOI: 10.1002/pbc.27869

Source DB:  PubMed          Journal:  Pediatr Blood Cancer        ISSN: 1545-5009            Impact factor:   3.167


  79 in total

Review 1.  Rhabdomyosarcoma: review of the Children's Oncology Group (COG) Soft-Tissue Sarcoma Committee experience and rationale for current COG studies.

Authors:  Suman Malempati; Douglas S Hawkins
Journal:  Pediatr Blood Cancer       Date:  2012-02-29       Impact factor: 3.167

2.  Clinical and mutational spectrum of highly differentiated, paired box 3:forkhead box protein o1 fusion-negative rhabdomyosarcoma: A report from the Children's Oncology Group.

Authors:  Lisa A Teot; Michaela Schneider; Aaron R Thorner; Jing Tian; Yueh-Yun Chi; Matthew Ducar; Ling Lin; Marcin Wlodarski; Holcombe E Grier; Christopher D M Fletcher; Paul van Hummelen; Stephen X Skapek; Douglas S Hawkins; Amy J Wagers; Carlos Rodriguez-Galindo; Simone Hettmer
Journal:  Cancer       Date:  2018-02-20       Impact factor: 6.860

3.  Comprehensive genomic analysis of rhabdomyosarcoma reveals a landscape of alterations affecting a common genetic axis in fusion-positive and fusion-negative tumors.

Authors:  Jack F Shern; Li Chen; Juliann Chmielecki; Jun S Wei; Rajesh Patidar; Mara Rosenberg; Lauren Ambrogio; Daniel Auclair; Jianjun Wang; Young K Song; Catherine Tolman; Laura Hurd; Hongling Liao; Shile Zhang; Dominik Bogen; Andrew S Brohl; Sivasish Sindiri; Daniel Catchpoole; Thomas Badgett; Gad Getz; Jaume Mora; James R Anderson; Stephen X Skapek; Frederic G Barr; Matthew Meyerson; Douglas S Hawkins; Javed Khan
Journal:  Cancer Discov       Date:  2014-01-23       Impact factor: 39.397

4.  Antitumor effects of a combined 5-aza-2'deoxycytidine and valproic acid treatment on rhabdomyosarcoma and medulloblastoma in Ptch mutant mice.

Authors:  Ines Ecke; Frauke Petry; Albert Rosenberger; Svantje Tauber; Sven Mönkemeyer; Ina Hess; Christian Dullin; Sarah Kimmina; Judith Pirngruber; Steven A Johnsen; Anja Uhmann; Frauke Nitzki; Leszek Wojnowski; Walter Schulz-Schaeffer; Olaf Witt; Heidi Hahn
Journal:  Cancer Res       Date:  2009-01-20       Impact factor: 12.701

Review 5.  Pediatric Rhabdomyosarcoma.

Authors:  Jack F Shern; Marielle E Yohe; Javed Khan
Journal:  Crit Rev Oncog       Date:  2015

6.  MYOD1-mutant spindle cell and sclerosing rhabdomyosarcoma: an aggressive subtype irrespective of age. A reappraisal for molecular classification and risk stratification.

Authors:  Narasimhan P Agaram; Michael P LaQuaglia; Rita Alaggio; Lei Zhang; Yumi Fujisawa; Marc Ladanyi; Leonard H Wexler; Cristina R Antonescu
Journal:  Mod Pathol       Date:  2018-09-04       Impact factor: 7.842

7.  Glycogen synthase kinase 3 inhibitors induce the canonical WNT/β-catenin pathway to suppress growth and self-renewal in embryonal rhabdomyosarcoma.

Authors:  Eleanor Y Chen; Michael T DeRan; Myron S Ignatius; Kathryn Brooke Grandinetti; Ryan Clagg; Karin M McCarthy; Riadh M Lobbardi; Jillian Brockmann; Charles Keller; Xu Wu; David M Langenau
Journal:  Proc Natl Acad Sci U S A       Date:  2014-03-24       Impact factor: 11.205

8.  Discovery of cancer drug targets by CRISPR-Cas9 screening of protein domains.

Authors:  Junwei Shi; Eric Wang; Joseph P Milazzo; Zihua Wang; Justin B Kinney; Christopher R Vakoc
Journal:  Nat Biotechnol       Date:  2015-05-11       Impact factor: 54.908

9.  Human rhabdomyosarcoma cell lines for rhabdomyosarcoma research: utility and pitfalls.

Authors:  Ashley R P Hinson; Rosanne Jones; Lisa E S Crose; Brian C Belyea; Frederic G Barr; Corinne M Linardic
Journal:  Front Oncol       Date:  2013-07-17       Impact factor: 6.244

10.  Functional genomic screening reveals asparagine dependence as a metabolic vulnerability in sarcoma.

Authors:  Simone Hettmer; Anna C Schinzel; Daria Tchessalova; Michaela Schneider; Christina L Parker; Roderick T Bronson; Nigel Gj Richards; William C Hahn; Amy J Wagers
Journal:  Elife       Date:  2015-10-24       Impact factor: 8.140

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  15 in total

1.  Report and Abstracts of the 18th Meeting of the Interuniversity Institute of Myology: Virtual meeting, October 21-24, 2021.

Authors:  Francesca Grassi; Sestina Falcone
Journal:  Eur J Transl Myol       Date:  2021-11-29

2.  Targeting KDM4 for treating PAX3-FOXO1-driven alveolar rhabdomyosarcoma.

Authors:  Shivendra Singh; Ahmed Abu-Zaid; Hongjian Jin; Jie Fang; Qiong Wu; Tingting Wang; Helin Feng; Waise Quarni; Ying Shao; Lily Maxham; Alireza Abdolvahabi; Mi-Kyung Yun; Sivaraja Vaithiyalingam; Haiyan Tan; John Bowling; Victoria Honnell; Brandon Young; Yian Guo; Richa Bajpai; Shondra M Pruett-Miller; Gerard C Grosveld; Mark Hatley; Beisi Xu; Yiping Fan; Gang Wu; Eleanor Y Chen; Taosheng Chen; Peter W Lewis; Zoran Rankovic; Yimei Li; Andrew J Murphy; John Easton; Junmin Peng; Xiang Chen; Ruoning Wang; Stephen W White; Andrew M Davidoff; Jun Yang
Journal:  Sci Transl Med       Date:  2022-07-13       Impact factor: 19.319

3.  Single-cell analysis and functional characterization uncover the stem cell hierarchies and developmental origins of rhabdomyosarcoma.

Authors:  Yun Wei; Qian Qin; Chuan Yan; Madeline N Hayes; Sara P Garcia; Haibin Xi; Daniel Do; Alexander H Jin; Tiffany C Eng; Karin M McCarthy; Abhinav Adhikari; Maristela L Onozato; Dimitrios Spentzos; Gunnlaugur P Neilsen; A John Iafrate; Leonard H Wexler; April D Pyle; Mario L Suvà; Filemon Dela Cruz; Luca Pinello; David M Langenau
Journal:  Nat Cancer       Date:  2022-08-18

4.  Single-cell imaging of T cell immunotherapy responses in vivo.

Authors:  Chuan Yan; Qiqi Yang; Songfa Zhang; David G Millar; Eric J Alpert; Daniel Do; Alexandra Veloso; Dalton C Brunson; Benjamin J Drapkin; Marcello Stanzione; Irene Scarfò; John C Moore; Sowmya Iyer; Qian Qin; Yun Wei; Karin M McCarthy; John F Rawls; Nick J Dyson; Mark Cobbold; Marcela V Maus; David M Langenau
Journal:  J Exp Med       Date:  2021-08-20       Impact factor: 17.579

5.  Radiation therapy is an important factor to improve survival in pediatric patients with head and neck rhabdomyosarcoma by enhancing local control: a historical cohort study from a single center.

Authors:  Yuan Wen; Dongsheng Huang; Weiling Zhang; Yi Zhang; Huimin Hu; Jing Li
Journal:  BMC Pediatr       Date:  2020-05-29       Impact factor: 2.125

Review 6.  Current and Future Treatment Strategies for Rhabdomyosarcoma.

Authors:  Celine Chen; Heathcliff Dorado Garcia; Monika Scheer; Anton G Henssen
Journal:  Front Oncol       Date:  2019-12-20       Impact factor: 6.244

7.  Multicenter study of risk factors of unplanned 30-day readmissions in pediatric oncology.

Authors:  Kamila Hoenk; Lilibeth Torno; William Feaster; Sharief Taraman; Anthony Chang; Michael Weiss; Karen Pugh; Brittney Anderson; Louis Ehwerhemuepha
Journal:  Cancer Rep (Hoboken)       Date:  2021-02-02

Review 8.  Zebrafish disease models in drug discovery: from preclinical modelling to clinical trials.

Authors:  E Elizabeth Patton; Leonard I Zon; David M Langenau
Journal:  Nat Rev Drug Discov       Date:  2021-06-11       Impact factor: 112.288

9.  KDM3A/Ets1 epigenetic axis contributes to PAX3/FOXO1-driven and independent disease-promoting gene expression in fusion-positive Rhabdomyosarcoma.

Authors:  Lays M Sobral; Hannah M Hicks; Janet K Parrish; Tyler S McCann; Joseph Hsieh; Andrew Goodspeed; James C Costello; Joshua C Black; Paul Jedlicka
Journal:  Mol Oncol       Date:  2020-08-05       Impact factor: 6.603

Review 10.  PRC2: an epigenetic multiprotein complex with a key role in the development of rhabdomyosarcoma carcinogenesis.

Authors:  Stefano Zoroddu; Irene Marchesi; Luigi Bagella
Journal:  Clin Epigenetics       Date:  2021-08-09       Impact factor: 6.551

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