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The second European interdisciplinary Ewing sarcoma research summit – A joint effort to deconstructing the multiple layers of a complex disease

Heinrich Kovar _, James Amatruda, Erika Brunet, Stefan Burdach, Florencia Cidre-Aranaz, Enrique de Alava, Uta Dirksen, Wietske van der Ent, Patrick Grohar, Thomas G. P. Grünewald, Lee Helman, Peter Houghton, Kristiina Iljin, Eberhard Korsching, Marc Ladanyi, Elizabeth Lawlor, Stephen Lessnick, Joseph Ludwig, Paul Meltzer, Markus Metzler, Jaume Mora, Richard Moriggl, Takuro Nakamura, Theodore Papamarkou, Branka Radic Sarikas, Francoise Rédini, Guenther H. S. Richter, Claudia Rossig, Keri Schadler, Beat W. Schäfer, Katia Scotlandi, Nathan C. Sheffield, Anang Shelat, Ewa Snaar-Jagalska, Poul Sorensen, Kimberly Stegmaier, Elizabeth Stewart, Alejandro Sweet-Cordero, Karoly Szuhai, Oscar M. Tirado, Franck Tirode, Jeffrey Toretsky, Kalliopi Tsafou, Aykut Üren, Andrei Zinovyev and Olivier Delattre

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Oncotarget. 2016; 7:8613-8624. https://doi.org/10.18632/oncotarget.6937

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Abstract

Heinrich Kovar1,2,*, James Amatruda3,*, Erika Brunet4,*, Stefan Burdach5,*, Florencia Cidre-Aranaz6,*, Enrique de Alava7,*, Uta Dirksen8,*, Wietske van der Ent9,10,*, Patrick Grohar11,*, Thomas G. P. Grünewald12,*, Lee Helman13,*, Peter Houghton14,*, Kristiina Iljin15,*, Eberhard Korsching16,*, Marc Ladanyi17,*, Elizabeth Lawlor18,*, Stephen Lessnick19,*, Joseph Ludwig20,*, Paul Meltzer21,*, Markus Metzler22,*, Jaume Mora23,*, Richard Moriggl24,25,*, Takuro Nakamura26,*, Theodore Papamarkou27,*, Branka Radic Sarikas28,*, Francoise Rédini29,*, Guenther H. S. Richter5,*, Claudia Rossig8,*, Keri Schadler30,*, Beat W. Schäfer31,*, Katia Scotlandi32,*, Nathan C. Sheffield28,*, Anang Shelat33,*, Ewa Snaar-Jagalska10,*, Poul Sorensen34,*, Kimberly Stegmaier35,*, Elizabeth Stewart36,*, Alejandro Sweet-Cordero37,*, Karoly Szuhai38,*, Oscar M. Tirado39,*, Franck Tirode9,*, Jeffrey Toretsky40,*, Kalliopi Tsafou40,*, Aykut Üren40,*, Andrei Zinovyev9,41,42,* and Olivier Delattre9,*

1 Children’s Cancer Research Institute, St. Anna Kinderkrebsforschung, Vienna, Austria

2 Department of Pediatrics, Medical University Vienna, Vienna, Austria

3 Departments of Pediatrics, Molecular Biology and Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX, USA

4 Museum National d’Histoire Naturelle, INSERM U1154, CNRS 7196, Paris, France

5 Children’s Cancer Research Center and Department of Pediatrics, Klinikum rechts der Isar, Technical University and Comprehensive Cancer Center Munich (CCCM), Munich, Germany

6 Unidad de Tumores Sólidos Infantiles, Área de Genética Humana, Instituto de Investigación de Enfermedades Raras, Instituto de Salud Carlos III, Madrid, Spain

7 Institute of Biomedicine of Sevilla (IBiS), Virgen del Rocio University Hospital /CSIC/University de Sevilla, Department of Pathology, Seville, Spain

8 University Children´s Hospital Muenster, Pediatric Hematology and Oncology, Muenster, Germany

9 INSERM U830, Laboratoire de Génétique et Biologie des Cancers, Institut Curie, Paris, France

10 Institute of Biology, Leiden University, Leiden, The Netherlands

11 Van Andel Institute, Center for Cancer and Cell Biology and Helen DeVos Children’s Hospital, Grand Rapids, MI, USA

12 Laboratory for Pediatric Sarcoma Biology, Institute of Pathology of the LMU Munich, Munich, Germany

13 Center for Cancer Rearch, NCI, NIH, Bethesda, MA, USA

14 Greehey Children’s Cancer Research Institute, University of Texas Health Science Center, San Antonio, TX, USA

15 VTT Technical Research Centre of Finland Ltd, Espoo, Finland

16 Institute of Bioinformatics, Faculty of Medicine, University of Muenster, Muenster, Germany

17 Department of Pathology and Human Oncology and Pathogenesis Program, Memorial Sloan-Kettering Cancer Center, New York, NY, USA

18 Department of Pediatrics and Department of Pathology, University of Michigan, Ann Arbor, MI, USA

19 Center for Childhood Cancer and Blood Disorders, Nationwide Children’s Hospital, and the Division of Pediatric Hematology/Oncology/BMT, The Ohio State University, Columbus, OH, USA

20 Department of Sarcoma Medical Oncology, MD Anderson Cancer Center, Houston, TX, USA

21 Genetics Branch, Center for Cancer Research, National Cancer Institute, Bethesda, MD, USA

22 Pediatric Oncology and Hematology, University Hospital Erlangen, Erlangen, Germany

23 Department of Pediatric Oncology, Sant Joan de Déu Hospital, Barcelona, Spain

24 Ludwig Boltzmann Institute for Cancer Research, Vienna, Austria

25 Institute of Animal Breeding and Genetics, University of Veterinary Medicine and Medical University, Vienna, Austria

26 Division of Carcinogenesis, The Cancer Institute, Japanese Foundation for Cancer Research, Tokyo, Japan

27 University of Glasgow, School of Mathematics and Statistics, Glasgow, UK

28 CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria

29 INSERM UMR957, Université de Nantes, Nantes, France

30 Department of Pediatrics Research, MD Anderson Cancer Center, Houston, TX, USA

31 Department of Oncology and Children’s Research Center, University Children‘s Hospital, Zurich, Switzerland

32 CRS Development of Biomolecular Therapies, Experimental Oncology Lab, Rizzoli Institute, Bologna, Italy

33 Department of Chemical Biology and Therapeutics, St. Jude Children’s Research Hospital, Memphis,TN, USA

34 Department of Molecular Oncology, British Columbia Cancer Research Centre, Vancouver, British Columbia, Canada

35 Department of Pediatric Oncology, Dana-Farber Cancer Institute and Boston Children’s Hospital, Boston, MA, USA

36 Department of Developmental Neurobiology, St. Jude Children’s Research Hospital, Memphis, TN, USA

37 Division of Hematology and Oncology, Department of Pediatrics, Stanford University, Stanford, CA, USA

38 Department of Molecular Cell Biology, Leiden University Medical Center, Leiden, The Netherlands

39 Sarcoma Research Group, Molecular Oncology Laboratory, Bellvitge Biomedical Research Institute (IDIBELL), L’Hospitalet de Llobregat, Barcelona, Spain

40 Department of Oncology, Georgetown University School of Medicine, Washington, DC, USA

41 INSERM, U900, Paris, France

42 Ecole des Mines ParisTech, Fontainbleau, France

* These authors have contributed equally to this work

Correspondence to:

Heinrich Kovar, email:

Keywords: Ewing sarcoma, epigenetics, development, therapy, microenvironment

Received: October 20, 2015 Accepted: January 14, 2016 Published: January 18, 2016

Abstract

Despite multimodal treatment, long term outcome for patients with Ewing sarcoma is still poor. The second “European interdisciplinary Ewing sarcoma research summit” assembled a large group of scientific experts in the field to discuss their latest unpublished findings on the way to the identification of novel therapeutic targets and strategies. Ewing sarcoma is characterized by a quiet genome with presence of an EWSR1-ETS gene rearrangement as the only and defining genetic aberration. RNA-sequencing of recently described Ewing-like sarcomas with variant translocations identified them as biologically distinct diseases. Various presentations adressed mechanisms of EWS-ETS fusion protein activities with a focus on EWS-FLI1. Data were presented shedding light on the molecular underpinnings of genetic permissiveness to this disease uncovering interaction of EWS-FLI1 with recently discovered susceptibility loci. Epigenetic context as a consequence of the interaction between the oncoprotein, cell type, developmental stage, and tissue microenvironment emerged as dominant theme in the discussion of the molecular pathogenesis and inter- and intra-tumor heterogeneity of Ewing sarcoma, and the difficulty to generate animal models faithfully recapitulating the human disease. The problem of preclinical development of biologically targeted therapeutics was discussed and promising perspectives were offered from the study of novel in vitro models. Finally, it was concluded that in order to facilitate rapid pre-clinical and clinical development of novel therapies in Ewing sarcoma, the community needs a platform to maintain knowledge of unpublished results, systems and models used in drug testing and to continue the open dialogue initiated at the first two Ewing sarcoma summits.


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