Accelerating drug development for neuroblastoma - New Drug
Development Strategy: an Innovative Therapies for Children with
Cancer, European Network for Cancer Research in Children and
Adolescents and International Society of Paediatric Oncology
Europe Neuroblastoma project

J 2017

Accelerating drug development for neuroblastoma - New Drug Development Strategy: an Innovative Therapies for Children with Cancer, European Network for Cancer Research in Children and Adolescents and International Society of Paediatric Oncology Europe Neuroblastoma project

MORENO, L., H. CARON, B. GEOERGER, A. EGGERT, G. SCHLEIERMACHER et. al.

Basic information

Original name

Accelerating drug development for neuroblastoma - New Drug Development Strategy: an Innovative Therapies for Children with Cancer, European Network for Cancer Research in Children and Adolescents and International Society of Paediatric Oncology Europe Neuroblastoma project

Authors

MORENO, L. (826 United Kingdom of Great Britain and Northern Ireland), H. CARON (756 Switzerland), B. GEOERGER (250 France), A. EGGERT (276 Germany), G. SCHLEIERMACHER (250 France), P. BROCK (826 United Kingdom of Great Britain and Northern Ireland), D. VALTEAU-COUANET (276 Germany), L. CHESLER (826 United Kingdom of Great Britain and Northern Ireland), J.H. SCHULTE (276 Germany), K. DE PRETER (56 Belgium), J. MOLENAAR (528 Netherlands), A. SCHRAMM (276 Germany), M. EILERS (276 Germany), T. VAN MAERKEN (56 Belgium), J.I. JOHNSEN (752 Sweden), M. GARRETT (826 United Kingdom of Great Britain and Northern Ireland), S.L. GEORGE (826 United Kingdom of Great Britain and Northern Ireland), D.A. TWEDDLE (36 Australia), P. KOGNER (752 Sweden), F. BERTHOLD (276 Germany), J. KOSTER (528 Netherlands), G. BARONE (826 United Kingdom of Great Britain and Northern Ireland), E.R. TUCKER (826 United Kingdom of Great Britain and Northern Ireland), L. MARSHALL (826 United Kingdom of Great Britain and Northern Ireland), R. HEROLD (826 United Kingdom of Great Britain and Northern Ireland), Jaroslav ŠTĚRBA (203 Czech Republic, guarantor, belonging to the institution), K. NORGA (56 Belgium), G. VASSAL (250 France) and A.D.J. PEARSON (826 United Kingdom of Great Britain and Northern Ireland)

Edition

EXPERT OPINION ON DRUG DISCOVERY, ABINGDON, TAYLOR & FRANCIS LTD, 2017, 1746-0441

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30104 Pharmacology and pharmacy

Country of publisher

United Kingdom of Great Britain and Northern Ireland

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 4.692

RIV identification code

RIV/00216224:14110/17:00098862

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1080/17460441.2017.1340269

UT WoS

000405392700004

Keywords in English

Neuroblastoma; drug development; phase I; preclinical testing; clinical trials

Abstract

V originále

Introduction: Neuroblastoma, the commonest paediatric extra-cranial tumour, remains a leading cause of death from cancer in children. There is an urgent need to develop new drugs to improve cure rates and reduce long-term toxicity and to incorporate molecularly targeted therapies into treatment. Many potential drugs are becoming available, but have to be prioritised for clinical trials due to the relatively small numbers of patients. Areas covered: The current drug development model has been slow, associated with significant attrition, and few new drugs have been developed for neuroblastoma. The Neuroblastoma New Drug Development Strategy (NDDS) has: 1) established a group with expertise in drug development; 2) prioritised targets and drugs according to tumour biology (target expression, dependency, pre-clinical data; potential combinations; biomarkers), identifying as priority targets ALK, MEK, CDK4/6, MDM2, MYCN (druggable by BET bromodomain, aurora kinase, mTORC1/2) BIRC5 and checkpoint kinase 1; 3) promoted clinical trials with target-prioritised drugs. Drugs showing activity can be rapidly transitioned via parallel randomised trials into front-line studies. Expert opinion: The Neuroblastoma NDDS is based on the premise that optimal drug development is reliant on knowledge of tumour biology and prioritisation. This approach will accelerate neuroblastoma drug development and other poor prognosis childhood malignancies.

Links

LM2015090, research and development project

Name: Český národní uzel Evropské sítě infrastruktur klinického výzkumu (Acronym: CZECRIN)

Investor: Ministry of Education, Youth and Sports of the CR

Citovat

MORENO, L., H. CARON, B. GEOERGER, A. EGGERT, G. SCHLEIERMACHER, P. BROCK, D. VALTEAU-COUANET, L. CHESLER, J.H. SCHULTE, K. DE PRETER, J. MOLENAAR, A. SCHRAMM, M. EILERS, T. VAN MAERKEN, J.I. JOHNSEN, M. GARRETT, S.L. GEORGE, D.A. TWEDDLE, P. KOGNER, F. BERTHOLD, J. KOSTER, G. BARONE, E.R. TUCKER, L. MARSHALL, R. HEROLD, Jaroslav ŠTĚRBA, K. NORGA, G. VASSAL and A.D.J. PEARSON. Accelerating drug development for neuroblastoma - New Drug Development Strategy: an Innovative Therapies for Children with Cancer, European Network for Cancer Research in Children and Adolescents and International Society of Paediatric Oncology Europe Neuroblastoma project. EXPERT OPINION ON DRUG DISCOVERY. ABINGDON: TAYLOR & FRANCIS LTD, 2017, vol. 12, No 8, p. 801-811. ISSN 1746-0441. Available from: https://dx.doi.org/10.1080/17460441.2017.1340269.

@article{1399958,
   author = {Moreno, L. and Caron, H. and Geoerger, B. and Eggert, A. and Schleiermacher, G. and Brock, P. and ValteauandCouanet, D. and Chesler, L. and Schulte, J.H. and De Preter, K. and Molenaar, J. and Schramm, A. and Eilers, M. and Van Maerken, T. and Johnsen, J.I. and Garrett, M. and George, S.L. and Tweddle, D.A. and Kogner, P. and Berthold, F. and Koster, J. and Barone, G. and Tucker, E.R. and Marshall, L. and Herold, R. and Štěrba, Jaroslav and Norga, K. and Vassal, G. and Pearson, A.D.J.},
   article_location = {ABINGDON},
   article_number = {8},
   doi = {http://dx.doi.org/10.1080/17460441.2017.1340269},
   keywords = {Neuroblastoma; drug development; phase I; preclinical testing; clinical trials},
   language = {eng},
   issn = {1746-0441},
   journal = {EXPERT OPINION ON DRUG DISCOVERY},
   title = {Accelerating drug development for neuroblastoma - New Drug Development Strategy: an Innovative Therapies for Children with Cancer, European Network for Cancer Research in Children and Adolescents and International Society of Paediatric Oncology Europe Neuroblastoma project},
   url = {http://dx.doi.org/10.1080/17460441.2017.1340269},
   volume = {12},
   year = {2017}
}

TY  - JOUR
ID  - 1399958
AU  - Moreno, L. - Caron, H. - Geoerger, B. - Eggert, A. - Schleiermacher, G. - Brock, P. - Valteau-Couanet, D. - Chesler, L. - Schulte, J.H. - De Preter, K. - Molenaar, J. - Schramm, A. - Eilers, M. - Van Maerken, T. - Johnsen, J.I. - Garrett, M. - George, S.L. - Tweddle, D.A. - Kogner, P. - Berthold, F. - Koster, J. - Barone, G. - Tucker, E.R. - Marshall, L. - Herold, R. - Štěrba, Jaroslav - Norga, K. - Vassal, G. - Pearson, A.D.J.
PY  - 2017
TI  - Accelerating drug development for neuroblastoma - New Drug Development Strategy: an Innovative Therapies for Children with Cancer, European Network for Cancer Research in Children and Adolescents and International Society of Paediatric Oncology Europe Neuroblastoma project
JF  - EXPERT OPINION ON DRUG DISCOVERY
VL  - 12
IS  - 8
SP  - 801-811
EP  - 801-811
PB  - TAYLOR & FRANCIS LTD
SN  - 17460441
KW  - Neuroblastoma
KW  - drug development
KW  - phase I
KW  - preclinical testing
KW  - clinical trials
UR  - http://dx.doi.org/10.1080/17460441.2017.1340269
L2  - http://dx.doi.org/10.1080/17460441.2017.1340269
N2  - Introduction: Neuroblastoma, the commonest paediatric extra-cranial tumour, remains a leading cause of death from cancer in children. There is an urgent need to develop new drugs to improve cure rates and reduce long-term toxicity and to incorporate molecularly targeted therapies into treatment. Many potential drugs are becoming available, but have to be prioritised for clinical trials due to the relatively small numbers of patients. Areas covered: The current drug development model has been slow, associated with significant attrition, and few new drugs have been developed for neuroblastoma. The Neuroblastoma New Drug Development Strategy (NDDS) has: 1) established a group with expertise in drug development; 2) prioritised targets and drugs according to tumour biology (target expression, dependency, pre-clinical data; potential combinations; biomarkers), identifying as priority targets ALK, MEK, CDK4/6, MDM2, MYCN (druggable by BET bromodomain, aurora kinase, mTORC1/2) BIRC5 and checkpoint kinase 1; 3) promoted clinical trials with target-prioritised drugs. Drugs showing activity can be rapidly transitioned via parallel randomised trials into front-line studies. Expert opinion: The Neuroblastoma NDDS is based on the premise that optimal drug development is reliant on knowledge of tumour biology and prioritisation. This approach will accelerate neuroblastoma drug development and other poor prognosis childhood malignancies.
ER  -

MORENO, L., H. CARON, B. GEOERGER, A. EGGERT, G. SCHLEIERMACHER, P. BROCK, D. VALTEAU-COUANET, L. CHESLER, J.H. SCHULTE, K. DE PRETER, J. MOLENAAR, A. SCHRAMM, M. EILERS, T. VAN MAERKEN, J.I. JOHNSEN, M. GARRETT, S.L. GEORGE, D.A. TWEDDLE, P. KOGNER, F. BERTHOLD, J. KOSTER, G. BARONE, E.R. TUCKER, L. MARSHALL, R. HEROLD, Jaroslav ŠTĚRBA, K. NORGA, G. VASSAL and A.D.J. PEARSON. Accelerating drug development for neuroblastoma - New Drug Development Strategy: an Innovative Therapies for Children with Cancer, European Network for Cancer Research in Children and Adolescents and International Society of Paediatric Oncology Europe Neuroblastoma project. \textit{EXPERT OPINION ON DRUG DISCOVERY}. ABINGDON: TAYLOR \&{} FRANCIS LTD, 2017, vol.~12, No~8, p.~801-811. ISSN~1746-0441. Available from: https://dx.doi.org/10.1080/17460441.2017.1340269.

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