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Published on: September 18, 2013
Preclinical drug development for childhood cancer
Lucas Moreno1, Louis Chesler, Darren Hargrave
1The Royal Marsden NHS Foundation Trust, Children and Young People's Unit, Sutton, Surrey, UK. Lucas.Moreno@icr.ac.uk
Importance Of The Field:
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.
Areas Covered In This Review:
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.
What The Reader Will Gain:
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.
Take Home Message:
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.
Insights
Developing new anticancer drugs for children presents unique challenges. Advances in preclinical drug development and collaborative efforts are paving the way for more effective targeted therapies in paediatric oncology.
Area of Science:
- Paediatric Oncology
- Drug Development
- Cancer Therapeutics
Background:
- Childhood cancers require novel therapeutic strategies due to poor prognosis and unique developmental aspects.
- Anticancer drug development for paediatric malignancies faces distinct challenges compared to adult cancers.
Purpose of the Study:
- To review recent advances in preclinical drug development for paediatric malignancies.
- To highlight strategies for overcoming challenges in childhood cancer drug development.
- To illustrate biology-driven drug development in paediatric oncology.
Main Methods:
- Review of current preclinical models and their limitations.
- Analysis of the roles of academia, regulatory bodies, and industry.
- Examination of established preclinical research networks and collaborations.
Main Results:
- Enhanced understanding of preclinical models and their application in paediatric oncology.
- Successful examples of targeted agents in childhood solid malignancies.
- Strengthened collaborations among academia, industry, and regulators.
Conclusions:
- Advanced preclinical models and testing platforms are crucial for efficient, biologically-driven drug development.
- Targeted therapies show promise for malignancies like neuroblastoma, medulloblastoma, and high-grade glioma.
- Improved preclinical strategies are essential to combat leading causes of childhood cancer mortality.
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Clinical Trials: Overview
Drug Discovery: Overview
Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption
Pharmacokinetics in Pediatric Patients: Drug Metabolism
Pharmacokinetics in Pediatric Patients: Drug Excretion

