Integration and validation of complementary ex vivo assays for functional precision oncology

Functional precision oncology (FPO) enables individualized therapy selection using patient-derived tumor models, yet the concordance of distinct ex vivo testing strategies remains unclear. Here, we compare two orthogonal drug sensitivity platforms across molecularly characterized models spanning diverse pediatric (n = 13) and adult (n = 6) cancers. A rapid ATP-based assay quantifies viability within three days, whereas a long-term dynamic image-based platform captures microtumor dynamics over two weeks, incorporating pharmacokinetic features. Up to 50 drugs were profiled across both platforms, with additional combinations evaluated in the long-term assay; genomics-guided targeted drugs served as benchmarks. Both approaches robustly distinguished responders from non-responders and showed strong agreement in therapeutic prioritization (96.5% within 95% limits of agreement). The long-term dynamic platform achieved 81% sensitivity and 78% specificity, while resolving response depth and distinguishing cytostatic from cytotoxic effects. A representative sarcoma case highlights clinical relevance: long-term dynamic profiling predicted disease progression, whereas the short-term assay captured early treatment-associated viability effects. These findings establish cross-platform reproducibility in FPO and provide a systematic benchmarking of such approaches. Defining their complementary utility will be essential for integrating FPO strategies into clinical decision-making.

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Publication Details

Journal
npj Precision Oncology
Published
2026-06-17
DOI
https://doi.org/10.1038/s41698-026-01555-2
Primary Topic
Neuroblastoma Research and Treatments
Type
article
Field-Weighted Citation Impact
0.00

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article

Integration and validation of complementary ex vivo assays for functional precision oncology

Ina Oehme, Roland Imle, Heike Peterziel, Robert J. Autry et al.
npj Precision Oncology
Neuroblastoma Research and Treatments
article

Integration and validation of complementary ex vivo assays for functional precision oncology

Ina Oehme, Roland Imle, Heike Peterziel, Robert J. Autry, Sonja Herter, Ana Banito, Constantia Zeiser, Jasmina Paluncic, Mirjam Blattner-Johnson, Dmitry Lupar, Claudia R Ball, Olaf Witt, Juliana P Schulz, Marko Weidmann, H Glimm, Attila Jády, Micaela Freitas, Jens M. Kelm, Martin Schneider, Anna Loboda, Karen Frese
article en

Abstract

Functional precision oncology (FPO) enables individualized therapy selection using patient-derived tumor models, yet the concordance of distinct ex vivo testing strategies remains unclear. Here, we compare two orthogonal drug sensitivity platforms across molecularly characterized models spanning diverse pediatric (n = 13) and adult (n = 6) cancers. A rapid ATP-based assay quantifies viability within three days, whereas a long-term dynamic image-based platform captures microtumor dynamics over two weeks, incorporating pharmacokinetic features. Up to 50 drugs were profiled across both platforms, with additional combinations evaluated in the long-term assay; genomics-guided targeted drugs served as benchmarks. Both approaches robustly distinguished responders from non-responders and showed strong agreement in therapeutic prioritization (96.5% within 95% limits of agreement). The long-term dynamic platform achieved 81% sensitivity and 78% specificity, while resolving response depth and distinguishing cytostatic from cytotoxic effects. A representative sarcoma case highlights clinical relevance: long-term dynamic profiling predicted disease progression, whereas the short-term assay captured early treatment-associated viability effects. These findings establish cross-platform reproducibility in FPO and provide a systematic benchmarking of such approaches. Defining their complementary utility will be essential for integrating FPO strategies into clinical decision-making.

npj Precision OncologyVol. 10(1)
German Cancer Research Center (DE), Heidelberg University (DE), Helmholtz-Zentrum Dresden-Rossendorf (DE), University Hospital Heidelberg (DE), National Center for Tumor Diseases (DE), Hopp Children's Cancer Center Heidelberg (DE), German Centre for Cardiovascular Research (DE), Qiagen (Switzerland) (CH), University Hospital Carl Gustav Carus (DE), Hochschule für Technik und Wirtschaft Dresden – University of Applied Sciences (DE), Nationales Centrum für Tumorerkrankungen Dresden (DE), Deutsches Konsortium für Translationale Krebsforschung (DE), Technische Universität Dresden (DE)
Helsingin Yliopisto, Deutsches Krebsforschungszentrum, Universität Heidelberg, Bundesministerium für Bildung und Forschung, Bundesministerium für Gesundheit, Deutsche Krebshilfe, Deutschen Konsortium für Translationale Krebsforschung, Biocenter Finland
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Openalex Percentile: Top 7%
Neuroblastoma Research and Treatments
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