Implementation of mechanism of action biology-driven early drug
development for children with cancer

J 2016

Implementation of mechanism of action biology-driven early drug development for children with cancer

PEARSON, A.D.J., R. HEROLD, R. ROUSSEAU, C. COPLAND, B. BRADLEY-GARELIK et. al.

Basic information

Original name

Implementation of mechanism of action biology-driven early drug development for children with cancer

Authors

PEARSON, A.D.J. (826 United Kingdom of Great Britain and Northern Ireland), R. HEROLD (826 United Kingdom of Great Britain and Northern Ireland), R. ROUSSEAU (840 United States of America), C. COPLAND (826 United Kingdom of Great Britain and Northern Ireland), B. BRADLEY-GARELIK (840 United States of America), D. BINNER (826 United Kingdom of Great Britain and Northern Ireland), R. CAPDEVILLE (756 Switzerland), H. CARON (756 Switzerland), J. CARLEER (56 Belgium), L. CHESLER (826 United Kingdom of Great Britain and Northern Ireland), B. GEOERGER (250 France), P.. KEARNS (826 United Kingdom of Great Britain and Northern Ireland), L.V. MARSHALL (826 United Kingdom of Great Britain and Northern Ireland), S.M. PFISTER (276 Germany), G. SCHLEIERMACHER (276 Germany), J. SKOLNIK (840 United States of America), C. SPADONI (826 United Kingdom of Great Britain and Northern Ireland), Jaroslav ŠTĚRBA (203 Czech Republic, guarantor, belonging to the institution), van den H. BERG (826 United Kingdom of Great Britain and Northern Ireland), M. UTTENREUTHER-FISCHER (276 Germany), O. WITT (276 Germany), K. NORGA (56 Belgium) and G. VASSAL (250 France)

Edition

European Journal of Cancer, Oxford, Elsevier Science Inc. 2016, 0959-8049

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30200 3.2 Clinical medicine

Country of publisher

United Kingdom of Great Britain and Northern Ireland

Confidentiality degree

není předmětem státního či obchodního tajemství

Impact factor

Impact factor: 6.029

RIV identification code

RIV/00216224:14110/16:00092086

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1016/j.ejca.2016.04.001

UT WoS

000377385100014

Keywords in English

Paediatric oncology; Mechanisrn of action; Targeted cancer drug development

Abstract

V originále

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.

Citovat

PEARSON, A.D.J., R. HEROLD, R. ROUSSEAU, C. COPLAND, B. BRADLEY-GARELIK, D. BINNER, R. CAPDEVILLE, H. CARON, J. CARLEER, L. CHESLER, B. GEOERGER, P.. KEARNS, L.V. MARSHALL, S.M. PFISTER, G. SCHLEIERMACHER, J. SKOLNIK, C. SPADONI, Jaroslav ŠTĚRBA, van den H. BERG, M. UTTENREUTHER-FISCHER, O. WITT, K. NORGA and G. VASSAL. Implementation of mechanism of action biology-driven early drug development for children with cancer. European Journal of Cancer. Oxford: Elsevier Science Inc., 2016, vol. 62, JUL 2016, p. 124-131. ISSN 0959-8049. Available from: https://dx.doi.org/10.1016/j.ejca.2016.04.001.

@article{1363068,
   author = {Pearson, A.D.J. and Herold, R. and Rousseau, R. and Copland, C. and BradleyandGarelik, B. and Binner, D. and Capdeville, R. and Caron, H. and Carleer, J. and Chesler, L. and Geoerger, B. and Kearns, P.. and Marshall, L.V. and Pfister, S.M. and Schleiermacher, G. and Skolnik, J. and Spadoni, C. and Štěrba, Jaroslav and Berg, van den H. and UttenreutherandFischer, M. and Witt, O. and Norga, K. and Vassal, G.},
   article_location = {Oxford},
   article_number = {JUL 2016},
   doi = {http://dx.doi.org/10.1016/j.ejca.2016.04.001},
   keywords = {Paediatric oncology; Mechanisrn of action; Targeted cancer drug development},
   language = {eng},
   issn = {0959-8049},
   journal = {European Journal of Cancer},
   title = {Implementation of mechanism of action biology-driven early drug development for children with cancer},
   volume = {62},
   year = {2016}
}

TY  - JOUR
ID  - 1363068
AU  - 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. - Štěrba, Jaroslav - Berg, van den H. - Uttenreuther-Fischer, M. - Witt, O. - Norga, K. - Vassal, G.
PY  - 2016
TI  - Implementation of mechanism of action biology-driven early drug development for children with cancer
JF  - European Journal of Cancer
VL  - 62
IS  - JUL 2016
SP  - 124-131
EP  - 124-131
PB  - Elsevier Science Inc.
SN  - 09598049
KW  - Paediatric oncology
KW  - Mechanisrn of action
KW  - Targeted cancer drug development
N2  - 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.
ER  -

PEARSON, A.D.J., R. HEROLD, R. ROUSSEAU, C. COPLAND, B. BRADLEY-GARELIK, D. BINNER, R. CAPDEVILLE, H. CARON, J. CARLEER, L. CHESLER, B. GEOERGER, P.. KEARNS, L.V. MARSHALL, S.M. PFISTER, G. SCHLEIERMACHER, J. SKOLNIK, C. SPADONI, Jaroslav ŠTĚRBA, van den H. BERG, M. UTTENREUTHER-FISCHER, O. WITT, K. NORGA and G. VASSAL. Implementation of mechanism of action biology-driven early drug development for children with cancer. \textit{European Journal of Cancer}. Oxford: Elsevier Science Inc., 2016, vol.~62, JUL 2016, p.~124-131. ISSN~0959-8049. Available from: https://dx.doi.org/10.1016/j.ejca.2016.04.001.

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