A multimodal precision oncology platform integrating genomics, single-cell transcriptomics, and functional drug screening expands therapeutic options in pediatric brain tumors

Preprint (not peer-reviewed): Huang et al. (University of Utah; senior authors Cheshier SH and Marth GT) describe a tiered framework combining deep whole-genome sequencing, scRNA-seq and ex vivo functional drug screening, applied to 17 children with malignant CNS tumors. Functional screening gave every patient at least one high-confidence targeted-therapy or chemotherapy nomination; scRNA-seq identified on average 37 druggable gene targets per patient, a fourfold increase in high-confidence targeted-therapy recommendations. In an exploratory analysis, functional classifications were directionally consistent with outcomes in 8 of 10 evaluable cases, but combination treatments and radiation precluded causal attribution.

Authors / organisation

Huang X, Qiao Y, Black GS, Sederman CR, Yang H, Musci T, Ludwick A, He G, Youssef O, Gutierrez M, Yang J, Chan PP, Memmott TH, Whipple NS, Bruggers CS, Quinlan A, Moos PJ, Cheshier SH, Marth GT.

Venue

bioRxiv (preprint, not peer-reviewed)

Date

2026-10-14

Type

preprint

Year

2026

DOI

10.64898/2026.10.12.758619

Verification

bioRxiv API details (title, 19 authors, posted 2026-10-14, v1, new results, cancer biology, CC BY, University of Utah, abstract) fetched 2026-10-20; full text not read

Neuro-oncologyscRNA-seqDrug responseTherapyPlatform

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