TY - JOUR
T1 - 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
AU - Moreno, Lucas
AU - Caron, Hubert
AU - Geoerger, Birgit
AU - Eggert, Angelika
AU - Schleiermacher, Gudrun
AU - Brock, Penelope
AU - Valteau-Couanet, Dominique
AU - Chesler, Louis
AU - Schulte, Johannes H.
AU - de Preter, Katleen
AU - Molenaar, Jan
AU - Schramm, Alexander
AU - Eilers, Martin
AU - van Maerken, Tom
AU - Johnsen, John Inge
AU - Garrett, Michelle
AU - George, Sally L.
AU - Tweddle, Deborah A.
AU - Kogner, Per
AU - Berthold, Frank
AU - Koster, Jan
AU - Barone, Giuseppe
AU - Tucker, Elizabeth R.
AU - Marshall, Lynley
AU - Herold, Ralf
AU - Sterba, Jaroslav
AU - Norga, Koen
AU - Vassal, Gilles
AU - Pearson, Andrew D. J.
PY - 2017
Y1 - 2017
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
AB - 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
U2 - https://doi.org/10.1080/17460441.2017.1340269
DO - https://doi.org/10.1080/17460441.2017.1340269
M3 - Review article
C2 - 28604107
SN - 1746-0441
VL - 12
SP - 801
EP - 811
JO - Expert Opinion on Drug Discovery
JF - Expert Opinion on Drug Discovery
IS - 8
ER -