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Updated: May 22, 2025

Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo
Published on: February 6, 2015
Ex Vivo Drug Screening: An Emerging Paradigm in the Treatment of Childhood Cancer
Anees Ahmed1, Ellen Cox1, Louis Lane1
1Sheffield Ex vivo Group, Division of Clinical Medicine, School of Medicine and Population Health, Faculty of Health, The University of Sheffield.
Abstract:
Developing and providing the right therapy for the right patient (or personalized targeted treatments) is key to reducing side-effects and improving survival in childhood cancers. Most efforts aiming to personalize childhood cancer treatment use genomic analysis of malignancies to identify potentially targetable genetic events. But it is becoming clear that not all patients will have an actionable change, and in those that do there is no additional way to determine if treatments will be effective. Ex vivo drug screening is a laboratory technique used to test the effects of various drugs or compounds, on biological tissues or cells that have been removed from an organism. This information is then used to predict which cancer treatments will be most effective based on the therapeutic response in the tissue or cells removed from that individual. Its utility in personalizing treatments in childhood cancer is increasingly recognized. In this review we describe the different methods for ex vivo drug screening and the advantages and disadvantages of each technique. We also present recent evidence that ex vivo screening may have utility in a variety of childhood malignancies including an overview of current clinical trials appraising its use. Finally, we discuss the research questions and hurdles that must be overcome before ex vivo screening can be widely used in pediatric oncology.
Insights
Personalized medicine for childhood cancers is advancing. Ex vivo drug screening tests drug effectiveness on patient tumor cells, offering a way to predict successful treatments and improve outcomes.
Area of Science:
- Pediatric Oncology
- Cancer Therapeutics
- Pharmacology
Background:
- Personalized medicine aims to tailor treatments for pediatric cancer patients.
- Genomic analysis identifies some targetable genetic events, but effectiveness prediction remains a challenge.
- Ex vivo drug screening offers a complementary approach to predict treatment efficacy.
Purpose of the Study:
- To review methods for ex vivo drug screening in childhood cancers.
- To discuss the advantages and disadvantages of different ex vivo screening techniques.
- To present evidence and ongoing clinical trials on ex vivo screening utility.
Main Methods:
- Review of existing literature on ex vivo drug screening methodologies.
- Analysis of current clinical trials investigating ex vivo screening in pediatric malignancies.
- Discussion of technical and research challenges for clinical implementation.
Main Results:
- Ex vivo drug screening tests drug responses on patient-derived tumor cells.
- This method can predict treatment effectiveness when genomic alterations are insufficient or non-actionable.
- Evidence suggests utility across various childhood cancers, with ongoing clinical trials.
Conclusions:
- Ex vivo drug screening shows promise for personalizing pediatric cancer therapy.
- Further research and overcoming implementation hurdles are necessary for widespread adoption.
- This approach could significantly improve treatment selection and patient survival rates.

