Journal Article

DKFZ-2026-00085

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Ex vivo drug sensitivity profiling to complement molecular profiling in pediatric precision oncology.

Schoonbeek, M. C. ; Gestraud, P. ; Vernooij, L. ; Boltjes, A. ; Amo-Addae, V. ; van Luik, M. ; Del Nery, E. ; Bellini, A. ; Chua, E. ; Swaak, S. ; Looze, E. J. ; Pietiäinen, V. M. ; Turunen, L. L. ; Saarela, J. S. ; Schueler, J. ; Indersie, E. ; Gürgen, D. ; Scotlandi, K. ; Eggert, A. ; Bouarich, R. ; Bourdeaut, F. ; Zaidi, S. ; Surdez, D. ; Carcaboso, Á. M. ; Geoerger, B. ; Iddir, Y. ; Saint-Charles, A. ; Saberi-Ansari, E. ; Cavalli, F. ; Gopisetty, A.DKFZ* ; Rief, E. M. ; Caron, H. N. ; Stancato, L. ; Vassal, G. ; Pfister, S.DKFZ* ; Koster, J. ; Eising, S. ; van Hooff, S. R. ; van den Boogaard, M. L. ; Schleiermacher, G. ; Molenaar, J. J.

2026
Springer Nature [London]

Abstract: Pediatric patients with high-risk extra-cranial solid tumors face a 5-year survival rate below 50%. As molecular profiling alone is insufficient to guide treatment at relapse, complementary strategies like drug screening are urgently needed. We evaluated short-term drug screening as a rapid, reliable method to assess drug sensitivities in pediatric solid tumors using ex vivo cultures from previously established patient-derived xenograft (PDX) models. Ex vivo drug screening was performed within 14 days of receipt across two institutes, testing 77-224 compounds depending on cell availability. Drug responses were consistent across institutes (n = 6), and effective compounds were reproducibly identified in a replicate model. Tumor type-specific responses were observed. In neuroblastoma, ALK-mutation status did not correlate with ALK-inhibitor response, whereas correlations with transcriptomic changes were observed. Timepoint-specific drug sensitivities were observed in serial Ewing sarcoma models. Overall, drug hits were identified in 94% of screens (n = 63), broadening treatment options for 88% of cases without targetable alterations (n = 11). In case of a targetable event, drug screening refined compound choice. Ex vivo drug screening is a fast and feasible method, providing insights into compound efficacy and enabling quick identification of functional treatment suggestions. Ex vivo drug screening should be integrated into a future next-generation diagnostic platform for pediatric solid tumors, combined with genomics and transcriptomics.

Note: epub

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Contributing Institute(s):

1. B062 Pädiatrische Neuroonkologie (B062)
2. DKTK HD zentral (HD01)

Research Program(s):

1. 899 - ohne Topic (POF4-899) (POF4-899)

Appears in the scientific report 2026

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