# Utilization of Peripheral Blood Exosomes to Detect Genetic and Protein Markers in Pediatric Solid Tumor Patients

> **NIH NIH R21** · LURIE CHILDREN'S HOSPITAL OF CHICAGO · 2021 · $120,373

## Abstract

PROJECT SUMMARY
The diagnosis of a pediatric solid tumor currently requires a tissue biopsy, which is an invasive
procedure that is not able to capture the heterogeneity present within the entire tumor. These
children then typically receive a combination of therapies, with response determined by serial
imaging. This approach is complicated by exposure to radiation during imaging and
requirements for sedation in younger children. Therefore, superior methods are needed for
diagnosis and for monitoring of response to treatment in children with solid tumors. Recently,
several technologies have been developed to analyze blood samples for the genomic presence
of solid tumors within circulating free DNA or circulating tumor cells. However, their clinical utility
is limited in pediatric neoplasms, which often rely on the detection of fusion transcripts for
diagnosis. Exosomes, which are small, membrane-enclosed vesicles released from cells,
contain mRNA, miRNA, and protein cargo unique to their cell of origin. Importantly, exosomes
protect RNA from degradation and could provide the ideal biomarker for diagnosis, monitoring
treatment response, and detection of early recurrence in children with solid tumors. However,
further evaluation of the methods utilized for exosome isolation and cargo evaluation are
needed to ensure that detection is reliable and reproducible prior to moving forward with the use
of exosomes as biomarkers in the clinical realm. The proposed studies will identify the
appropriate pre-analytic conditions for the reliable isolation of exosomes from the blood of
pediatric patients with solid tumors containing known fusion transcripts identified through routine
diagnostic testing, including Ewing Sarcoma/PNET, synovial sarcoma, alveolar
rhabdomyosarcoma, and desmoplastic small round cell tumor, as well as for the reproducible
detection of tumor-specific markers within the isolated exosomes. We will isolate exosomes
from peripheral blood samples from a total of 50 healthy children and 30 children with newly
diagnosed solid tumors during this study, and will apply clinical laboratory validation concepts of
accuracy, precision, and analytic sensitivity and specificity to establish the reliability and utility of
exosomal cargo as biomarkers in children with solid tumors. The overall goal of this study is to
provide sufficient information to allow for the proposal of studies within the Children’s Oncology
Group cooperative clinical trials to investigate the utility of exosome isolation and cargo
analysis. We anticipate that within 10 years, the analysis of peripheral blood for exosome
characteristics may provide a non-invasive method for establishing the diagnosis, monitoring
treatment response, and detecting early recurrence in children with solid tumors.

## Key facts

- **NIH application ID:** 10226046
- **Project number:** 5R21CA225846-03
- **Recipient organization:** LURIE CHILDREN'S HOSPITAL OF CHICAGO
- **Principal Investigator:** AMY Leanne WALZ
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $120,373
- **Award type:** 5
- **Project period:** 2019-08-15 → 2022-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10226046

## Citation

> US National Institutes of Health, RePORTER application 10226046, Utilization of Peripheral Blood Exosomes to Detect Genetic and Protein Markers in Pediatric Solid Tumor Patients (5R21CA225846-03). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10226046. Licensed CC0.

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