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Can mouse models for brain tumors inform treatment in pediatric patients?
William A Weiss1, Anu Banerjee
1Departments of Neurology, Pediatrics, and Neurological Surgery, University of California, San Francisco, CA 94143, USA. weiss@cgl.ucsf.edu
Abstract:
Brain tumors represent the most common solid tumor of childhood. Although the histology of many pediatric brain tumors is similar to that of their adult counterparts, significant differences exist with regard to tumor location and response to therapy. The biological and genetic basis for this difference is poorly understood, as tumor tissue is generally unavailable for such studies. While targeted therapies directed against specific molecules active in cancer represents a new arsenal of agents for treating these tumors, such agents are generally not being developed for pediatric cancer in particular. Therefore, new agents for treatment of pediatric glioma must be obtained from compounds being tested against tumors of comparable histology in adult patients. Compounding this problem, although brain tumors are among the most lethal tumors of childhood, their absolute number is relatively small. As a consequence, trials with new agents must be prioritized based on the likelihood that a particular agent or combination of agents will have efficacy in pediatric cancer. Mouse models for brain tumors may help to identify targeted agents, and combinations of agents, effective against these tumors. Such data can be used to prioritize therapies for clinical trials in children with these tumors.
Insights
Pediatric brain tumors differ from adult types, necessitating novel therapeutic strategies. Mouse models can identify effective targeted agents for clinical trials in children with these challenging cancers.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Cancer Biology
Background:
- Pediatric brain tumors are the most common childhood solid tumors, yet exhibit distinct characteristics from adult counterparts regarding location and treatment response.
- The underlying biological and genetic differences are poorly understood due to limited tumor tissue availability for research.
- Targeted therapies are advancing cancer treatment, but specific development for pediatric cancers lags behind.
Purpose of the Study:
- To address the need for effective treatments for pediatric brain tumors, particularly gliomas.
- To explore strategies for identifying and prioritizing novel therapeutic agents for clinical trials in children.
- To leverage preclinical models for advancing pediatric neuro-oncology research.
Main Methods:
- Review of existing knowledge on pediatric versus adult brain tumor differences.
- Discussion of challenges in developing targeted therapies for rare pediatric cancers.
- Emphasis on the potential utility of preclinical mouse models for identifying effective agents and combinations.
Main Results:
- Significant differences in tumor location and therapeutic response exist between pediatric and adult brain tumors.
- Limited availability of pediatric tumor tissue hinders research into biological and genetic underpinnings.
- Mouse models show promise in identifying targeted agents and combinations for pediatric brain tumors.
Conclusions:
- New agents for pediatric glioma treatment must be sourced from adult cancer trials.
- Prioritization of clinical trials requires evidence of efficacy, potentially from preclinical studies.
- Mouse models are crucial for identifying and validating targeted therapies for pediatric brain tumors, guiding future clinical trials.
