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@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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