ITCC Pediatric Preclinical POC Platform – Sofia ref.: 116064

Deliverables

Websites, patent fillings, videos etc. (2)

Final draft and Submission of a paper for publication

Migration

International scientific symposium on : “Preclinicalevaluation in pediatric oncology drug development”

Migration

Publications

Peer reviewed articles (16)

Phenotypic profiling with a living biobank of primary rhabdomyosarcoma unravels disease heterogeneity and AKT sensitivity

Author(s): Gabriele Manzella, Leonie D. Schreck, Willemijn B. Breunis, Jan Molenaar, Hans Merks, Frederic G. Barr, Wenyue Sun, Michaela Römmele, Luduo Zhang, Joelle Tchinda, Quy A. Ngo, Peter Bode, Olivier Delattre, Didier Surdez, Bharat Rekhi, Felix K. Niggli, Beat W. Schäfer, Marco Wachtel
Published in: Nature Communications, 11/1, 2020, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/s41467-020-18388-7

Targeting fibroblast growth factor receptors to combat aggressive ependymoma

Author(s): Lötsch D, Kirchhofer D, Englinger B, Jiang L, Okonechnikov K, Senfter D, Laemmerer A, Gabler L, Pirker C, Donson AM, Bannauer P, Korbel P, Jaunecker CN, Hübner JM, Mayr L, Madlener S, Schmook MT, Ricken G, Maaß K, Grusch M, Holzmann K, Grasl-Kraupp B, Spiegl-Kreinecker S, Hsu J, Dorfer C, Rössler K, Azizi AA, Foreman NK, Peyrl A, Haberler C, Czech T, Slavc I, Filbin MG, Pajtler KW, Kool M, Ber
Published in: Acta neuropathologica communications, 2021 Aug; 142(2), 2021, Page(s) 339-360, ISSN 0001-6322
Publisher: Springer Verlag
DOI: 10.1007/s00401-021-02327-x

Systematic target actionability reviews of preclinical proof-of-concept papers to match targeted drugs to paediatric cancers

Author(s): Nil A. Schubert, Caitlin D. Lowery, Guillaume Bergthold, Jan Koster, Thomas F. Eleveld, Ana Rodríguez, David T.W. Jones, Gilles Vassal, Louis F. Stancato, Stefan M. Pfister, Hubert N. Caron, Jan J. Molenaar
Published in: European Journal of Cancer, 130, 2020, Page(s) 168-181, ISSN 0959-8049
Publisher: Pergamon Press Ltd.
DOI: 10.1016/j.ejca.2020.01.027

Crossing Oceans: Preclinical Collaboration to Improve Pediatric Drug Development

Author(s): Gregory Reaman, Louis Stancato, Gilles Vassal, John M. Maris
Published in: American Society of Clinical Oncology Educational Book, 40, 2020, Page(s) 409-416, ISSN 1548-8748
Publisher: American Society of Clinical Oncology
DOI: 10.1200/edbk_278893

International consensus on minimum preclinical testing requirements for the development of innovative therapies for children and adolescents with cancer

Author(s): Vassal, G. Houghton, P. J. Pfister, S. M. Smith, M. A. Caron, H. N. Li, X. N. Shields, D. J. Witt, O. Molenaar, J. J. Colombetti, S. Schuler, J. Stancato, L. F.
Published in: Mol Cancer Ther, Volume 20, 2021, Page(s) 1462–8, ISSN 1535-7163
Publisher: American Association for Cancer Research
DOI: 10.1158/1535-7163.mct-20-0394

Prognostic value of patient-derived xenograft engraftment in pediatric sarcomas

Author(s): Helena Castillo-Ecija, Guillem Pascual-Pasto, Sara Perez-Jaume, Claudia Resa-Pares, Monica Vila-Ubach, Carles Monterrubio, Ana Jimenez-Cabaco, Merce Baulenas-Farres, Oscar Muñoz-Aznar, Noelia Salvador, Maria Cuadrado-Vilanova, Nagore G. Olaciregui, Leire Balaguer-Lluna, Victor Burgueño, Francisco J. Vicario, Alejandro Manzanares, Alicia Castañeda, Vicente Santa-Maria, Ofelia Cruz, Veronica Celi
Published in: Journal of Pathology – clinical research, Volume 7, Issu 4, July 2021, 2021, Page(s) 338-349, ISSN 2056-4538
Publisher: Wiley
DOI: 10.1002/cjp2.210

Integrated molecular characterization of patient-derived models reveals therapeutic strategies for treating CIC-DUX4 sarcoma

Author(s): Carrabotta M, Laginestra MA, Durante G, Mancarella C, Landuzzi L, Parra A, Ruzzi F, Toracchio L, De Feo A, Giusti V, Pasello M, Righi A, Lollini PL, Palmerini E, Donatii DM, Manara MC, Scotlandi K.
Published in: Cancer Research, 2022 Feb 15;82(4), 2021, Page(s) 708-720, ISSN 1538-7445
Publisher: AACR
DOI: 10.1158/0008-5472.can-21-1222

Target Actionability Review: a systematic evaluation of replication stress as a therapeutic target for pediatric solid malignancies

Author(s): Kaylee M. Keller; Apurva Gopisetty; Dan Luedtke; Jan Koster; Nil A. Schubert; Ana Rodríguez; Sander R. van Hooff; Damian Stichel; Emmy M. Dolman; Gilles Vassal; Stefan M. Pfister; Hubert N. Caron; Louis F. Stancato; Jan J. Molenaar; Natalie Jäger; Marcel Kool
Published in: Eur J Cancer, Dec 25, 2021; Volume 162, February 2022, 2021, Page(s) Pages 107-117, ISSN 0959-8049
Publisher: Pergamon Press Ltd.
DOI: 10.1016/j.ejca.2021.11.030

A biobank of patient-derived pediatric brain tumor models

Author(s): Sebastian Brabetz, Sarah E. S. Leary, Susanne N. Gröbner, Madison W. Nakamoto, Huriye Şeker-Cin, Emily J. Girard, Bonnie Cole, Andrew D. Strand, Karina L. Bloom, Volker Hovestadt, Norman L. Mack, Fiona Pakiam, Benjamin Schwalm, Andrey Korshunov, Gnana Prakash Balasubramanian, Paul A. Northcott, Kyle D. Pedro, Joyoti Dey, Stacey Hansen, Sally Ditzler, Peter Lichter, Lukas Chavez, David T. W. Jone
Published in: Nature Medicine, 24/11, 2018, Page(s) 1752-1761, ISSN 1078-8956
Publisher: Nature Publishing Group
DOI: 10.1038/s41591-018-0207-3

PCLN-05. A BIOBANK OF PATIENT-DERIVED MOLECULARLY CHARACTERIZED ORTHOTOPIC PEDIATRIC BRAIN TUMOR MODELS FOR PRECLINICAL RESEARCH

Author(s): Sebastian Brabetz, Susanne N Groebner, Natalie Jaeger, Till Milde, Jonas Ecker, Florian Selt, Olaf Witt, Jessica M Rusert, Jan Koster, Sarah E Leary, Xiao-Nan Li, Robert J Wechsler-Reya, James M Olson, Stefan M Pfister, Marcel Kool
Published in: Neuro-Oncology, 20/suppl_2, 2018, Page(s) i155-i155, ISSN 1522-8517
Publisher: Duke University Press
DOI: 10.1093/neuonc/noy059.574

Small-Molecule Dual PLK1 and BRD4 Inhibitors are Active Against Preclinical Models of Pediatric Solid Tumors

Author(s): Natalie Timme, Youjia Han, Shuai Liu, Hailemichael O. Yosief, Heathcliff Dorado García, Yi Bei, Filippos Klironomos, Ian C. MacArthur, Annabell Szymansky, Jennifer von Stebut, Victor Bardinet, Constantin Dohna, Annette Künkele, Jana Rolff, Patrick Hundsdörfer, Andrej Lissat, Georg Seifert, Angelika Eggert, Johannes H. Schulte, Wei Zhang, Anton G. Henssen
Published in: Translational Oncology, 13/2, 2020, Page(s) 221-232, ISSN 1936-5233
Publisher: Neoplasia Press
DOI: 10.1016/j.tranon.2019.09.013

ROCK2 deprivation leads to the inhibition of tumor growth and metastatic potential in osteosarcoma cells through the modulation of YAP activity

Author(s): Cinzia Zucchini, Maria Cristina Manara, Camilla Cristalli, Marianna Carrabotta, Sara Greco, Rosa Simona Pinca, Cristina Ferrari, Lorena Landuzzi, Michela Pasello, Pier-Luigi Lollini, Marco Gambarotti, Davide Maria Donati, Katia Scotlandi
Published in: Journal of Experimental & Clinical Cancer Research, 38/1, 2019, ISSN 1756-9966
Publisher: J Exp Clin Cancer Res
DOI: 10.1186/s13046-019-1506-3

TERT expression is susceptible to BRAF and ETS-factor inhibition in BRAFV600E/TERT promoter double-mutated glioma

Author(s): Lisa Gabler, Daniela Lötsch, Dominik Kirchhofer, Sushilla van Schoonhoven, Hannah M. Schmidt, Lisa Mayr, Christine Pirker, Katharina Neumayer, Carina Dinhof, Lucia Kastler, Amedeo A. Azizi, Christian Dorfer, Thomas Czech, Christine Haberler, Andreas Peyrl, Rajiv Kumar, Irene Slavc, Sabine Spiegl-Kreinecker, Johannes Gojo, Walter Berger
Published in: Acta Neuropathologica Communications, 7/1, 2019, ISSN 2051-5960
Publisher: BioMed Central
DOI: 10.1186/s40478-019-0775-6

Increased Anaplastic Lymphoma Kinase Activity Induces a Poorly Differentiated Thyroid Carcinoma in Mice

Author(s): Hannah Kohler, Soeren Latteyer, Georg Sebastian Hönes, Sarah Theurer, Xiao-Hui Liao, Sandra Christoph, Denise Zwanziger, Johannes H. Schulte, Jukka Kero, Hendrik Undeutsch, Samuel Refetoff, Kurt W. Schmid, Dagmar Führer, Lars C. Moeller
Published in: Thyroid, 29/10, 2019, Page(s) 1438-1446, ISSN 1050-7256
Publisher: Mary Ann Liebert Inc.
DOI: 10.1089/thy.2018.0526

Synergistic activity of BET inhibitor MK-8628 and PLK inhibitor Volasertib in preclinical models of medulloblastoma

Author(s): Youjia Han, Sven Lindner, Yi Bei, Heathcliff Dorado Garcia, Natalie Timme, Kristina Althoff, Andrea Odersky, Alexander Schramm, Andrej Lissat, Annette Künkele, Hedwig E. Deubzer, Angelika Eggert, Johannes H. Schulte, Anton G. Henssen
Published in: Cancer Letters, 445, 2019, Page(s) 24-33, ISSN 0304-3835
Publisher: Elsevier BV
DOI: 10.1016/j.canlet.2018.12.012

Single-Cell RNA-Seq Reveals Cellular Hierarchies and Impaired Developmental Trajectories in Pediatric Ependymoma

Author(s): Johannes Gojo, Bernhard Englinger, Li Jiang, Jens M. Hübner, McKenzie L. Shaw, Olivia A. Hack, Sibylle Madlener, Dominik Kirchhofer, Ilon Liu, Jason Pyrdol, Volker Hovestadt, Emanuele Mazzola, Nathan D. Mathewson, Maria Trissal, Daniela Lötsch, Christian Dorfer, Christine Haberler, Angela Halfmann, Lisa Mayr, Andreas Peyrl, Rene Geyeregger, Benjamin Schwalm, Monica Mauermann, Kristian W. Pajtler
Published in: Cancer Cell, 38/1, 2020, Page(s) 44-59.e9, ISSN 1535-6108
Publisher: Cell Press
DOI: 10.1016/j.ccell.2020.06.004

Book chapters (1)

Ewing Sarcoma PDX Models

Author(s): Didier Surdez, Lorena Landuzzi, Katia Scotlandi, Maria Cristina Manara
Published in: Ewing Sarcoma - Methods and Protocols, 2226, 2021, Page(s) 223-242, ISBN 978-1-0716-1019-0
Publisher: Springer US
DOI: 10.1007/978-1-0716-1020-6_18

Thesis dissertations (1)

Molecular and preclinical evaluation of patient-derived orthotopic xenograft models of pediatric brain tumors

Author(s): Sebastian Brabetz
Published in: 2018
Publisher: Univesity of Heidelberg