Related Experiment Video
Updated: Feb 26, 2026
Detection and Monitoring of Tumor Associated Circulating DNA in Patient Biofluids
Published on: June 8, 2019
Molecular Screening for Cancer Treatment Optimization (MOSCATO-01) in Pediatric Patients: A Single-Institutional
Anne C Harttrampf1, Ludovic Lacroix2,3, Marc Deloger4
1Vectorology and Anticancer Therapies, UMR 8203, CNRS, Univ. Paris-Sud, Gustave Roussy, Université Paris-Saclay, Villejuif, France.
Abstract:
Purpose: This single-institutional feasibility study prospectively characterized genomic alterations in recurrent or refractory solid tumors of pediatric patients to select a targeted therapy.Experimental Design: Following treatment failure, patients with signed consent and ages above 6 months, underwent tumor biopsy or surgical resection of primary or metastatic tumor site. These newly acquired samples were analyzed by comparative genomic hybridization array, next-generation sequencing for 75 target genes, whole-exome and RNA sequencing. Biological significance of the alterations and suggestion of most relevant targeted therapies available were discussed in a multidisciplinary tumor board.Results: From December 2012 to January 2016, 75 patients were included, 73 patients underwent 79 interventions, 56 of which were research biopsies with a low complication rate. All patients were pretreated, 37.0% had a brain tumor, and 63.0% had an extra-cranial solid tumor. Median tumor cell content was 70% (range, 0%-100%). Successful molecular analysis in 69 patients detected in 60.9% of patients an actionable alteration in various oncogenic pathways (42.4% with copy-number change, 33.3% with mutation, 2.1% with fusion), and change in diagnosis in three patients. Fourteen patients received 17 targeted therapies; two had received a matched treatment before inclusion.Conclusions: Research biopsies are feasible in advanced pediatric malignancies that exhibit a considerable amount of potentially actionable alterations. Genetic events affecting different cancer hallmarks and limited access to targeted agents within pediatric clinical trials remain the main obstacles that are addressed in our two subsequent precision medicine studies MAPPYACTS and AcSé-ESMART. Clin Cancer Res; 23(20); 6101-12. ©2017 AACR.
Insights
Feasibility study shows research biopsies in pediatric solid tumors can identify actionable genomic alterations for targeted therapy selection. This approach is feasible and reveals significant genetic changes in advanced cancers.
Area of Science:
- Oncology
- Genomics
- Pediatric Medicine
Background:
- Recurrent or refractory solid tumors in pediatric patients often lack effective treatment options.
- Genomic characterization can identify targets for personalized therapy.
Purpose of the Study:
- To assess the feasibility of prospectively characterizing genomic alterations in pediatric solid tumors.
- To identify actionable targets for selecting precision therapies.
Main Methods:
- Tumor biopsy or resection followed by comparative genomic hybridization array, next-generation sequencing (75 genes), whole-exome, and RNA sequencing.
- Multidisciplinary tumor board review to discuss findings and suggest targeted therapies.
Main Results:
- 75 pediatric patients with solid tumors were included; 69 had successful molecular analysis.
- Actionable alterations were found in 60.9% of patients, including copy-number changes, mutations, and fusions.
- 14 patients received targeted therapies based on genomic findings.
Conclusions:
- Research biopsies are feasible for advanced pediatric malignancies.
- A significant proportion of pediatric solid tumors harbor actionable genomic alterations.
- Future studies (MAPPYACTS, AcSé-ESMART) will address genetic events and access to targeted agents.
