Abstract
Cancer remains the leading cause of disease-related death in children. For the many children who experience relapses of their malignant solid tumors, usually after very intensive first-line therapy, curative treatment options are scarce. Preclinical drug testing to identify promising treatment elements that match the molecular make-up of the tumor is hampered by the fact that (i) molecular genetic data on pediatric solid tumors from relapsed patients and thus our understanding of tumor evolution and therapy resistance are very limited to date and (ii) for many of the high-risk entities, no appropriate and molecularly well-characterized patient-derived models and/or genetic mouse models are currently available. However, recent regulatory changes enacted by the European Medicines Agency (class waiver changes) and the maturation of the RACE for Children act with the FDA, will require a significant increase in preclinical pediatric cancer research and clinical development must occur. We detail the outcome of a pediatric cancer international multistakeholder meeting whose output aims at defining an international consensus on minimum preclinical testing requirements for the development of innovative therapies for children and adolescents with cancer. Recommendations based on the experience of the NCI funded PPTP/C (www.ncipptc.org) and the EU funded ITCC-P4 public private partnership (https://www.itccp4.eu/) are provided for the use of cell-based and mouse models for pediatric solid malignancies, as well as guidance on the scope and content of preclinical proof-of-concept data packages to inform clinical development dependent on clinical urgency. These recommendations can serve as a minimal guidance necessary to jumpstart preclinical pediatric research globally.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 1462-1468 |
| Number of pages | 7 |
| Journal | Molecular cancer therapeutics |
| Volume | 20 |
| Issue number | 8 |
| DOIs | |
| State | Published - Aug 2021 |
Funding
G. Vassal reports grants from Innovative Medicine Initiative during the conduct of the study. S.M. Pfister reports grants from IMI-2 funded project together with Roche, Eli-Lilly, Pfizer, Bayer, and PharmaMar during the conduct of the study. H.N. Caron reports other support from Roche during the conduct of the study as well as other ITCC-P4 is funded by IMI2 grant agreement no. 116064 ITCC-P4. The September 2018 workshop was funded by Solving Kids\u2019 Cancer, Neuroblastoma UK, Sohn Conference Foundation and ITCC-P4. We thanks all participants for their valuable contribution in designing the international consensus: Sandy Azzi-Hatem, Kladiola Berdufi, Patricia Blanc, Trandafir Bogdan-Adrian, Anna-Lisa Bottcher, Stefano Cairo, Hubert Caron, Michela Casanova, Louis Chesler, Silvia Chioato, Marta Colletti, Sara Colombetti, Emilie Dass\u00E9, Emmy Dolman, Gregory Donoho, Jarno Drost, Angelika Eggert, Stephen Erickson, David Frost, Patrizia Gasparini, Johannes Gojo, Ralph Graeser, Peter Houghton, Yann Jamin, Kaylee Keller, Leona Knox, Marcel Kool, Jan Koster, Heinrich Kovar, Andrew Kung, Raushan Kurmasheva, Xiao-Nan Li, Caitlin Lowery, Donna Ludwinski, Florence Meyer-Losic, Jan Molenaar, Massimo Moro, Andy Pearson, Stefan Pfister, Gregory Reaman, Ana Rodriguez, Jana Rolff, Gudrun Schleiermacher, Nil Schubert, Julia Schu\u20ACler, David Shields, Malcolm Smith, Louis Stanaato, Birgit Steckel-Hamann, Didier Surdez, Beverly Teicher, Gilles Vassal, Cl\u00E9mence Vieules, Marco Wachtel, Scott Weinrich, Frank Westermann, Olaf Witt, Dieter Zopf, and Danny Zwijnenburg.
ASJC Scopus subject areas
- Oncology
- Cancer Research
