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Record W4409039558 · doi:10.1038/s44321-025-00212-8

Proteomics and personalized PDX models identify treatment for a progressive malignancy within an actionable timeframe

2025· article· en· W4409039558 on OpenAlexafffund
Georgina D. Barnabas, Tariq Ahmad Bhat, Verena Goebeler, Pascal Leclair, Nadine Azzam, Nicole Melong, Alexis Gom, Seohee An, Enes K. Ergin, Yaoqing Shen, Agustina Conrrero, Andrew J. Mungall, Karen Mungall, Christopher A. Maxwell, Gregor S. D. Reid, Martin Hirst, Steven J.M. Jones, Jennifer A. Chan, Donna L. Senger, Jason N. Berman, Seth J. Parker, Jonathan W. Bush, Caron Strahlendorf, Rebecca Deyell, Chinten James Lim, Philipp F. Lange

Bibliographic record

VenueEMBO Molecular Medicine · 2025
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicProtein Degradation and Inhibitors
Canadian institutionsBC Cancer AgencyMcGill UniversityMcGill University Health CentreUniversity of OttawaCanada's Michael Smith Genome Sciences CentreJewish General HospitalAlberta Children's HospitalUniversity of British Columbia HospitalChildren's Hospital of Eastern OntarioUniversity of CalgaryBC Children's HospitalUniversity of British Columbia
FundersMichael Cuccione FoundationUniversity of British ColumbiaCanada Research ChairsBC Children's HospitalMichael Smith Health Research BCCanadian Institutes of Health ResearchMitacsAlberta Cancer FoundationBC Children’s Hospital FoundationChildren's Hospital Foundation
KeywordsMedicineProteomicsPrecision medicineProteogenomicsMalignancyOncologyPersonalized medicineDiseaseTargeted therapyBioinformaticsProteomeGenomicsInternal medicineCancerPathologyGenomeBiologyGene

Abstract

fetched live from OpenAlex

Genomics has transformed the diagnostic landscape of pediatric malignancies by identifying and integrating actionable features that refine diagnosis, classification, and treatment. Yet, translating precision oncology data into effective therapies for hard-to-cure childhood, adolescent, and young adult malignancies remains a significant challenge. We present the case for combining proteomics with patient-derived xenograft models to identify personalized treatment for an adolescent with primary and metastatic spindle epithelial tumor with thymus-like elements (SETTLE). Within two weeks of biopsy, proteomics identified elevated SHMT2 as a target for therapy with the anti-depressant sertraline. Drug response was confirmed within two months using a personalized chicken chorioallantoic membrane model of the patient's SETTLE tumor. Following failure of cytotoxic chemotherapy and second-line therapy, the patient received sertraline treatment and showed decreased tumor growth rates, albeit with clinically progressive disease. We demonstrate that proteomics and fast-track xenograft models provide supportive pre-clinical data in a clinically meaningful timeframe to impact clinical practice. By this, we show that proteome-guided and functional precision oncology are feasible and valuable complements to the current genome-driven precision oncology practices.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Bench or experimental · Consensus signal: Bench or experimental
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.130
Threshold uncertainty score0.639

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.019
GPT teacher head0.319
Teacher spread0.300 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designBench or experimental
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations3
Published2025
Admission routes2
Has abstractyes

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