Pearson, A. D. J., Herold, R., Rousseau, R., Copland, C., Bradley-Garelik, B., Binner, D., Capdeville, R., Caron, H., Carleer, J., Chesler, L., Geoerger, B., Kearns, P., Marshall, L. V., Pfister, S. M., Schleiermacher, G., Skolnik, J., Spadoni, C., Sterba, J., van den Berg, H., Uttenreuther-Fischer, M., Witt, O., Norga, K., Vassal, G., Working Grp Paediat Platform, Accel (2016) Implementation of mechanism of action biology-driven early drug development for children with cancer. EUROPEAN JOURNAL OF CANCER, 62. pp. 124-131. ISSN 0959-8049

Full text not available from this repository.

Abstract

An urgent need remains for new paediatric oncology drugs to cure children who die from cancer and to reduce drug-related sequelae in survivors. In 2007, the European Paediatric Regulation came into law requiring industry to create paediatric drug (all types of medicinal products) development programmes alongside those for adults. Unfortunately, paediatric drug development is still largely centred on adult conditions and not a mechanism of action (MoA)-based model, even though this would be more logical for childhood tumours as these have much fewer non-synonymous coding mutations than adult malignancies. Recent large-scale sequencing by International Genome Consortium and Paediatric Cancer Genome Project has further shown that the genetic and epigenetic repertoire of driver mutations in specific childhood malignancies differs from more common adult-type malignancies. To bring about much needed change, a Paediatric Platform, ACCELERATE, was proposed in 2013 by the Cancer Drug Development Forum, Innovative Therapies for Children with Cancer, the European Network for Cancer Research in Children and Adolescents and the European Society for Paediatric Oncology. The Platform, comprising multiple stakeholders in paediatric oncology, has three working groups, one with responsibility for promoting and developing high-quality MoA-informed paediatric drug development programmes, including specific measures for adolescents. Key is the establishment of a freely accessible aggregated database of paediatric biological tumour drug targets to be aligned with an aggregated pipeline of drugs. This will enable prioritisation and conduct of early phase clinical paediatric trials to evaluate these drugs against promising therapeutic targets and to generate clinical paediatric efficacy and safety data in an accelerated time frame. Through this work, the Platform seeks to ensure that potentially effective drugs, where the MoA is known and thought to be relevant to paediatric malignancies, are evaluated in early phase clinical trials, and that this approach to generate pre-clinical and clinical data is systematically pursued by academia, sponsors, industry, and regulatory bodies to bring new paediatric oncology drugs to front-line therapy more rapidly. (C) 2016 Elsevier Ltd. All rights reserved.

Item Type:	Article
Authors (ICR Faculty only):	Pearson, Andy
All Authors:	Pearson, A. D. J., Herold, R., Rousseau, R., Copland, C., Bradley-Garelik, B., Binner, D., Capdeville, R., Caron, H., Carleer, J., Chesler, L., Geoerger, B., Kearns, P., Marshall, L. V., Pfister, S. M., Schleiermacher, G., Skolnik, J., Spadoni, C., Sterba, J., van den Berg, H., Uttenreuther-Fischer, M., Witt, O., Norga, K., Vassal, G., Working Grp Paediat Platform, Accel
Additional Information:	ISI Document Delivery No.: DN9FO Times Cited: 0 Cited Reference Count: 23 Pearson, Andrew D. J. Herold, Ralf Rousseau, Raphael Copland, Chris Bradley-Garelik, Brigid Binner, Debbie Capdeville, Renaud Caron, Hubert Carleer, Jacqueline Chesler, Louis Geoerger, Birgit Kearns, Pamela Marshall, Lynley V. Pfister, Stefan M. Schleiermacher, Gudrun Skolnik, Jeffrey Spadoni, Cesare Sterba, Jaroslav van den Berg, Hendrick Uttenreuther-Fischer, Martina Witt, Olaf Norga, Koen Vassal, Gilles European Union [HEALTH-F2-2011-261474] This publication has received funding from the European Union's Seventh Framework Programme for research, technological development, and demonstration under the project ENCCA-grant agreement no. HEALTH-F2-2011-261474. 0 ELSEVIER SCI LTD OXFORD EUR J CANCER
Uncontrolled Keywords:	Paediatric oncology Mechanisrn of action Targeted cancer drug development ACTIVATING ACVR1 MUTATIONS INTRINSIC PONTINE GLIOMA LOW-GRADE GLIOMAS CHILDHOOD-CANCER PEDIATRIC-PATIENTS ADOLESCENTS LANDSCAPE RELAPSE TRIALS TUMORS
Research teams:	Closed research groups > Paediatric Drug Development & Clinical Trials ICR divisions > Clinical Studies > Paediatric Solid Tumour Biology and Therapeutics ICR divisions > Cancer Therapeutics > Paediatric Solid Tumour Biology and Therapeutics ICR divisions > Molecular Pathology > Paediatric Solid Tumour Biology and Therapeutics
Depositing User:	Barry Jenkins
Date Deposited:	06 Jul 2016 10:06
Last Modified:	06 Jul 2016 10:06
URI:	http://publications.icr.ac.uk/id/eprint/15160

Actions (login required)

View Item