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Updated: Aug 8, 2026

Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
[Drug development in paediatric oncology]
1Universitätsklinikum Münster, Klinik und Poliklinik für Kinder- und Jugendmedizin, Pädiatrische Hämatologie/Onkologie, Münster.
Abstract:
Cure rates in children with cancer exceed 75 % today. Notwithstanding this high success rate, 60-90 % of drugs employed in Paediatric Oncology are used off label. Comprehensive pre-clinical screening of candidate substances may help to generate entity-specific priority lists and to be faster and more selective in feeding promising candidates into the development process of drugs for use in children. The upcoming EU regulation "Better medicine for children" offers professional societies and their trial groups the opportunity to upgrade existing structures and benefit from the expected increase in paediatric clinical trials. Networking joining more national and international institutions and boards may serve to expand the repertoire of clinical trials and to establish powerful structures. Such a network may be an attractive partner for the drug industry and may thus eventually lead to a reduction in off label use in paediatric oncology.
Insights
Pediatric cancer cure rates are high, but many drugs are used off-label. Pre-clinical screening and networking can accelerate drug development for children, reducing off-label use.
Area of Science:
- Pediatric Oncology
- Drug Development
- Clinical Trials
Context:
- Over 75% cure rates in childhood cancer are achieved today.
- However, 60-90% of drugs used in pediatric oncology are prescribed off-label.
- This highlights a significant gap in targeted pediatric drug availability.
Purpose:
- To explore strategies for improving drug selection and development for pediatric cancer.
- To leverage upcoming EU regulations and foster collaboration among research institutions.
- To reduce the incidence of off-label drug use in treating childhood cancers.
Summary:
- Comprehensive pre-clinical screening of drug candidates can create prioritized lists for pediatric use.
- New EU regulations and increased networking among institutions can boost pediatric clinical trials.
- Establishing robust networks can attract pharmaceutical partnerships, aiming to decrease off-label prescribing.
Impact:
- Accelerated development of targeted therapies for pediatric cancer patients.
- Enhanced collaboration between research societies, trial groups, and the pharmaceutical industry.
- Significant reduction in off-label medication use, improving treatment safety and efficacy for children with cancer.
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