Preclinical drug development for childhood cancer.
BACKGROUND
More effective drugs are needed to treat poor prognosis paediatric malignancies. Development of anticancer agents for childhood cancers faces several unique challenges compared with their adult counterparts.


METHODS
We demonstrate how recent advances in preclinical drug development may overcome these difficulties and challenges. We explain the role of academia, regulators and industry in this field, address issues with preclinical models and illustrate several examples of biology-driven drug development in childhood cancers.


RESULTS
Increased knowledge about preclinical drug development in paediatric oncology including different preclinical models, established preclinical research networks, and relationships among academia, industry and regulators, as illustrated by several examples of targeted agents in childhood solid malignancies.


CONCLUSIONS
It is anticipated that emerging advanced preclinical models and testing platforms will provide a more efficient, biologically-driven rationale to support the use of targeted therapies in several malignancies such as neuroblastoma, medulloblastoma or high grade glioma which account for the majority of deaths related to childhood cancer.
The Royal Marsden NHS Foundation Trust, Children and Young People's Unit, Sutton, Surrey, UK. Lucas.Moreno@icr.ac.uk
Lucas
L
Moreno
Louis
L
Chesler
Darren
D
Hargrave
Suzanne A
SA
Eccles
Andrew D J
AD
Pearson