Related Experiment Video
Updated: May 4, 2026
Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
Targeted therapy in pediatric central nervous system tumors: a review from the National Pediatric Cancer Foundation
Benjamin I Siegel1,2, Prabhumallikarjun Patil3,4, Akul Prakash5
1Brain Tumor Institute and Gilbert Family Neurofibromatosis Institute, Children's National Hospital, Washington, DC, United States.
Abstract:
Central nervous system tumors represent the leading cause of cancer-related mortality in children. Conventional therapies of surgery, radiation, and cytotoxic chemotherapy have insufficient efficacy for some pediatric CNS tumors and are associated with significant morbidity, prompting an ongoing need for novel treatment approaches. Identification of molecular alterations driving tumorigenesis has led to a rising interest in developing targeted therapies for these tumors. The present narrative review focuses on recent progress in targeted therapies for pediatric CNS tumors. We outline the key implicated cellular pathways, discuss candidate molecular therapies for targeting each pathway, and present an overview of the clinical trial landscape for targeted therapies in pediatric CNS tumors. We then discuss challenges and future directions for targeted therapy, including combinatorial approaches and real-time drug screening for personalized treatment planning.
Insights
Pediatric central nervous system (CNS) tumors are a leading cause of childhood cancer death. Targeted therapies offer new hope by focusing on specific molecular pathways driving tumor growth, improving treatment efficacy.
Area of Science:
- Pediatric oncology
- Neuro-oncology
- Molecular medicine
Background:
- Central nervous system (CNS) tumors are the primary cause of cancer-related mortality in children.
- Current treatments like surgery, radiation, and chemotherapy have limited efficacy and significant side effects for pediatric CNS tumors.
- Advances in identifying molecular drivers of tumorigenesis necessitate novel therapeutic strategies.
Purpose of the Study:
- To review recent advancements in targeted therapies for pediatric CNS tumors.
- To outline key cellular pathways implicated in pediatric CNS tumor development.
- To discuss the clinical trial landscape and future directions for targeted treatments.
Main Methods:
- Narrative review of current literature on targeted therapies for pediatric CNS tumors.
- Identification of key molecular pathways driving tumorigenesis.
- Analysis of the clinical trial landscape for targeted agents.
- Discussion of challenges and future directions, including combinatorial therapies and personalized medicine.
Main Results:
- Several molecular pathways are implicated in pediatric CNS tumor development.
- A growing number of targeted molecular therapies are under investigation.
- The clinical trial landscape for these therapies is expanding.
- Combinatorial approaches and personalized treatment planning show promise.
Conclusions:
- Targeted therapies represent a promising frontier for treating pediatric CNS tumors.
- Further research and clinical trials are crucial to optimize efficacy and minimize toxicity.
- Personalized treatment strategies hold potential for improving outcomes in pediatric neuro-oncology.
Related Concept Videos
02:49Cancer Therapies
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
02:57Targeted Cancer Therapies
There are several types of targeted therapies against...
02:56Treatment Resistant Cancers
01:27Tumor Immunotherapy