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.

Základní údaje

Originální název

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

Autoři

Vydání

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

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30200 3.2 Clinical medicine

Stát vydavatele

Velká Británie a Severní Irsko

Utajení

není předmětem státního či obchodního tajemství

Impakt faktor

Impact factor: 6.029

Kód RIV

RIV/00216224:14110/16:00092086

Organizační jednotka

Lékařská fakulta

DOI

https://doi.org/10.1016/j.ejca.2016.04.001

UT WoS

000377385100014

EID Scopus

2-s2.0-84974539624

Klíčová slova anglicky

Paediatric oncology; Mechanisrn of action; Targeted cancer drug development

Štítky

EL OK

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 1. 12. 2016 12:10, Soňa Böhmová

Anotace

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 a 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, roč. 62, JUL 2016, s. 124-131. ISSN 0959-8049. Dostupné z: https://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 = {https://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 a 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, roč.~62, JUL 2016, s.~124-131. ISSN~0959-8049. Dostupné z: https://doi.org/10.1016/j.ejca.2016.04.001.

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