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Modeling the developmental origins of pediatric cancer to improve patient outcomes
1Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA 90027, USA jamatruda@chla.usc.edu.
Abstract:
In the treatment of children and adolescents with cancer, multimodal approaches combining surgery, chemotherapy and radiation can cure most patients, but may cause lifelong health problems in survivors. Current therapies only modestly reflect increased knowledge about the molecular mechanisms of these cancers. Advances in next-generation sequencing have provided unprecedented cataloging of genetic aberrations in tumors, but understanding how these genetic changes drive cellular transformation, and how they can be effectively targeted, will require multidisciplinary collaboration and preclinical models that are truly representative of the in vivo environment. Here, I discuss some of the key challenges in pediatric cancer from my perspective as a physician-scientist, and touch on some promising new approaches that have the potential to transform our understanding of these diseases.
Insights
Pediatric cancer treatments combining surgery, radiation, and chemotherapy can lead to long-term health issues in survivors. New research emphasizes understanding cancer
Area of Science:
- Pediatric Oncology
- Cancer Genomics
- Translational Cancer Research
Background:
- Multimodal cancer therapies (surgery, chemotherapy, radiation) improve survival but cause lifelong health problems in survivors.
- Current pediatric cancer treatments inadequately incorporate advances in understanding molecular mechanisms.
- Next-generation sequencing has identified numerous genetic aberrations in tumors, but their functional impact remains unclear.
Purpose of the Study:
- To discuss key challenges in pediatric cancer research and treatment from a physician-scientist's viewpoint.
- To highlight promising new approaches for transforming the understanding and treatment of pediatric cancers.
- To emphasize the need for multidisciplinary collaboration and improved preclinical models.
Main Methods:
- Review of current challenges in pediatric oncology.
- Discussion of advances in next-generation sequencing and cancer genomics.
- Exploration of the role of preclinical models in understanding tumor development.
Main Results:
- Most pediatric cancer patients can be cured with multimodal therapy, but survivors face significant long-term health issues.
- Genetic aberrations cataloged by next-generation sequencing offer potential therapeutic targets.
- Understanding how genetic changes drive cancer requires better preclinical models reflecting the in vivo environment.
Conclusions:
- Transforming pediatric cancer care necessitates a deeper understanding of molecular mechanisms and improved therapeutic strategies.
- Multidisciplinary collaboration and the development of representative preclinical models are crucial for advancing research.
- New approaches hold promise for improving outcomes for children and adolescents with cancer.
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