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Pediatric precision oncology: "better three hours too soon than a minute too late"
Mark Marshall1, Jennifer Ivanovich2, Morgan Schmitt1
1Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, United States.
Abstract:
Precision oncology is defined as the selection of an effective treatment for a cancer patient based upon genomic profiling of the patient's tumor to identify targetable alterations. The application of precision oncology toward pediatric cancer patients has moved forward more slowly than with adults but is gaining momentum. Clinical and pharmaceutical advances developed over the past decade for adult cancer indications have begun to move into pediatric oncology, expanding treatment options for young high-risk and refractory patients. As a result, the FDA has approved 23 targeted drugs for pediatric cancer indications, moving targeted drugs into the standard of care. Our precision oncology program is in a medium sized children's hospital, lacking internal sequencing capabilities and bioinformatics. We have developed methods, medical and business partnerships to provide state-of-the-art tumor characterization and targeted treatment options for our patients. We present here a streamlined and practical protocol designed to enable any oncologist to implement precision oncology options for their patients.
Insights
Precision oncology offers targeted cancer treatments for children, expanding options for high-risk patients. This protocol enables oncologists to implement these advanced genomic profiling and treatment strategies.
Area of Science:
- Oncology
- Genomics
- Pediatric Medicine
Background:
- Precision oncology, guided by tumor genomic profiling, is increasingly applied to adult cancers.
- Its adoption in pediatric oncology has lagged but is accelerating due to advances in targeted therapies.
- The FDA has approved 23 targeted drugs for pediatric cancer, integrating them into standard care.
Purpose of the Study:
- To present a practical protocol for implementing precision oncology in pediatric cancer care.
- To enable oncologists in community hospitals to offer advanced tumor characterization and targeted treatments.
- To overcome limitations such as lack of internal sequencing and bioinformatics capabilities.
Main Methods:
- Developing strategic medical and business partnerships.
- Establishing state-of-the-art tumor characterization methods.
- Creating a streamlined protocol for clinical application.
Main Results:
- Successfully implemented a precision oncology program in a medium-sized children's hospital.
- Facilitated access to advanced tumor profiling and targeted treatment options for pediatric patients.
- Demonstrated a viable model for community hospitals to adopt precision oncology.
Conclusions:
- A practical, partnership-driven protocol can enable precision oncology implementation in pediatric settings.
- This approach expands treatment options for young patients with high-risk or refractory cancers.
- The protocol supports the integration of genomic profiling and targeted therapies into standard pediatric cancer care.
