Moreno, Lucas ORCID: 0000-0002-0708-1670, Caron, Hubert, Geoerger, Birgit, Eggert, Angelika, Schleiermacher, Gudrun, Brock, Penelope, Valteau-Couanet, Dominique, Chesler, Louis ORCID: 0000-0001-7842-2068, Schulte, Johannes H., De Preter, Katleen, Molenaar, Jan, Schramm, Alexander ORCID: 0000-0001-7670-7529, Eilers, Martin, Van Maerken, Tom, Johnsen, John Inge, Garrett, Michelle, George, Sally L., Tweddle, Deborah A., Kogner, Per ORCID: 0000-0002-2202-9694, Berthold, Frank, Koster, Jan ORCID: 0000-0002-0890-7585, Barone, Giuseppe, Tucker, Elizabeth R., Marshall, Lynley, Herold, Ralf ORCID: 0000-0002-8148-6748, Sterba, Jaroslav, Norga, Koen, Vassal, Gilles and Pearson, Andrew D. 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. Expert. Opin. Drug Discov., 12 (8). S. 801 - 812. ABINGDON: TAYLOR & FRANCIS LTD. ISSN 1746-045X

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Abstract

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.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Moreno, LucasUNSPECIFIEDorcid.org/0000-0002-0708-1670UNSPECIFIED
Caron, HubertUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Geoerger, BirgitUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Eggert, AngelikaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schleiermacher, GudrunUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Brock, PenelopeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Valteau-Couanet, DominiqueUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Chesler, LouisUNSPECIFIEDorcid.org/0000-0001-7842-2068UNSPECIFIED
Schulte, Johannes H.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
De Preter, KatleenUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Molenaar, JanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schramm, AlexanderUNSPECIFIEDorcid.org/0000-0001-7670-7529UNSPECIFIED
Eilers, MartinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Van Maerken, TomUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Johnsen, John IngeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Garrett, MichelleUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
George, Sally L.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Tweddle, Deborah A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kogner, PerUNSPECIFIEDorcid.org/0000-0002-2202-9694UNSPECIFIED
Berthold, FrankUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Koster, JanUNSPECIFIEDorcid.org/0000-0002-0890-7585UNSPECIFIED
Barone, GiuseppeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Tucker, Elizabeth R.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Marshall, LynleyUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Herold, RalfUNSPECIFIEDorcid.org/0000-0002-8148-6748UNSPECIFIED
Sterba, JaroslavUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Norga, KoenUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Vassal, GillesUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Pearson, Andrew D. J.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-224406
DOI: 10.1080/17460441.2017.1340269
Journal or Publication Title: Expert. Opin. Drug Discov.
Volume: 12
Number: 8
Page Range: S. 801 - 812
Date: 2017
Publisher: TAYLOR & FRANCIS LTD
Place of Publication: ABINGDON
ISSN: 1746-045X
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
HIGH-RISK NEUROBLASTOMA; REFRACTORY SOLID TUMORS; BET-BROMODOMAIN INHIBITION; PHASE-I TRIAL; AURORA KINASE; INDUCTION CHEMOTHERAPY; ACTIVATING MUTATIONS; RANDOMIZED-TRIAL; TARGETING MYCN; PATIENTS PTSMultiple languages
Pharmacology & PharmacyMultiple languages
Refereed: Yes
URI: http://kups.ub.uni-koeln.de/id/eprint/22440

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