Related Experiment Video
Updated: Aug 15, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
New approaches to drug development in pediatric oncology
Peter C Adamson1, Susan M Blaney
1The Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA. adamsonp@mail.med.upenn.edu
Abstract:
This article reviews the changing paradigm of new drug development for childhood cancer. Although there have been dramatic improvements in the treatment and outcome for childhood cancer over the past 50 years, in some cancers, only limited progress has been made. New strategies to improve the outcome for poor-prognosis tumors and to minimize the long-term sequelae associated with therapy are required. Such strategies include a new pediatric-specific initiative for the preclinical testing of anticancer drugs for childhood malignancies, and ongoing research into the molecular heterogeneity of childhood tumors to facilitate the evaluation of molecularly targeted drugs. In addition, continued investigation into the genetic basis underlying interpatient variability in drug exposure and response is a priority. The goal of these initiatives is to improve the overall outcome for childhood cancer while minimizing the short and long-term sequelae associated with current treatment strategies.
Insights
New strategies are needed to improve outcomes for pediatric cancers, especially poor-prognosis tumors. Research focuses on targeted drugs and understanding genetic variations to enhance childhood cancer treatment effectiveness and reduce side effects.
Area of Science:
- Pediatric Oncology
- Pharmacology
- Cancer Genomics
Background:
- Childhood cancer treatment has advanced significantly over 50 years.
- Progress in treating some childhood cancers remains limited, necessitating new therapeutic approaches.
- Minimizing long-term side effects of cancer therapy is a critical goal.
Purpose of the Study:
- To review the evolving landscape of new drug development for pediatric malignancies.
- To highlight the need for improved strategies for poor-prognosis childhood tumors.
- To emphasize the importance of reducing treatment-related sequelae in children.
Main Methods:
- Review of current pediatric-specific drug development initiatives.
- Discussion of research into the molecular heterogeneity of childhood tumors.
- Examination of investigations into genetic factors influencing drug response.
Main Results:
- A pediatric-specific preclinical drug testing initiative is proposed.
- Understanding molecular heterogeneity can guide the development of targeted therapies.
- Investigating genetic variability is crucial for personalized treatment.
Conclusions:
- New drug development strategies are essential for advancing childhood cancer care.
- Targeted therapies and personalized medicine hold promise for improving outcomes.
- Reducing the long-term impact of treatment is a key objective for future research.
Related Concept Videos
Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption
Pharmacokinetics in Pediatric Patients: Drug Metabolism
Pharmacokinetics in Pediatric Patients: Drug Distribution
Pharmacokinetics in Pediatric Patients: Drug Excretion
Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
Drug Discovery: Overview
