ArticleBMC cancer2019
The design, analysis and application of mouse clinical trials in oncology drug development.
Article in BMC cancer, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
41 citing papers in PubMed, 61 citations in OpenAlex.
- On the Choice of Longitudinal Models for the Analysis of Antitumor Efficacy in Mouse Clinical Trials of Patient-derived Xenograft Models.Cancer research communications · 2023Trial
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- An Integrin β6-Targeted Antibody-Drug Conjugate Optimized for Intravesical Delivery to Treat Non-Muscle-Invasive Bladder Cancer.Molecular cancer therapeutics · 2026Article
- Targeted Delivery of a Potent STING Agonist Payload via an Antibody-Drug Conjugate Drives Robust Antitumor Activity in Preclinical Models.Molecular cancer therapeutics · 2026Article
- Co-administered internalizing RGD peptide boosts anti-PD-L1 therapy in hepatocellular carcinoma.JHEP reports : innovation in hepatology · 2026Article
- Next generation protein-corrole bio-assemblies provide effective tumoricidal treatment in a metastatic triple-negative breast cancer model.bioRxiv : the preprint server for biology · 2026Article
- The MEK-RAF molecular glue IK-595 has potent antitumor activity across RAS/MAPK pathway-altered cancers.Nature cancer · 2026Article
- Improved analysis of in vivo drug combination experiments with a comprehensive statistical framework and web-tool.Nature communications · 2025Article
- IL-17A-secreting γδ T cells promote resistance to CDK4/CDK6 inhibitors in HRNature cancer · 2025Article
- m276-SL-PBD eradicates tumors and instigates long-lasting tumor-free survival in Merkel cell carcinoma preclinical models.iScience · 2025Article
- Engineering TCR-controlled fuzzy logic into CAR T cells enhances therapeutic specificity.Cell · 2025Article
- Impact of Subcutaneous Versus Orthotopic Implantations on Patient-Derived Xenograft Transcriptomic Profiles.Cancer research communications · 2025Article
- Article
- Developing transcriptomic biomarkers for TAVO412 utilizing next generation sequencing analyses of preclinical tumor models.Frontiers in immunology · 2025Article
- Mathematical Modeling of Tumor Growth in Preclinical Mouse Models with Applications in Biomarker Discovery and Drug Mechanism Studies.Cancer research communications · 2024Article
- Statistical classification of treatment responses in mouse clinical trials for stratified medicine in oncology drug discovery.Scientific reports · 2024Article
- Anaplerotic nutrient stress drives synergy of angiogenesis inhibitors with therapeutics targeting tumor metabolism.bioRxiv : the preprint server for biology · 2023Article
- SGN-B7H4V, an investigational vedotin ADC directed to the immune checkpoint ligand B7-H4, shows promising activity in preclinical models.Journal for immunotherapy of cancer · 2023Article
- Proteomics in Childhood Acute Lymphoblastic Leukemia: Challenges and Opportunities.Diagnostics (Basel, Switzerland) · 2023Review
- Immunotherapy targeting different immune compartments in combination with radiation therapy induces regression of resistant tumors.Nature communications · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundMouse clinical trials (MCTs) are becoming wildly used in pre-clinical oncology drug development, but a statistical framework is yet to be developed. In this study, we establish such as framework and provide general guidelines on the design, analysis and application of MCTs.
methodsWe systematically analyzed tumor growth data from a large collection of PDX, CDX and syngeneic mouse tumor models to evaluate multiple efficacy end points, and to introduce statistical methods for modeling MCTs.
resultsWe established empirical quantitative relationships between mouse number and measurement accuracy for categorical and continuous efficacy endpoints, and showed that more mice are needed to achieve given accuracy for syngeneic models than for PDXs and CDXs. There is considerable disagreement between methods on calling drug responses as objective response. We then introduced linear mixed models (LMMs) to describe MCTs as clustered longitudinal studies, which explicitly model growth and drug response heterogeneities across mouse models and among mice within a mouse model. Case studies were used to demonstrate the advantages of LMMs in discovering biomarkers and exploring drug's mechanisms of action. We introduced additive frailty models to perform survival analysis on MCTs, which more accurately estimate hazard ratios by modeling the clustered mouse population. We performed computational simulations for LMMs and frailty models to generate statistical power curves, and showed that power is close for designs with similar total number of mice. Finally, we showed that MCTs can explain discrepant results in clinical trials.
conclusionsMethods proposed in this study can make the design and analysis of MCTs more rational, flexible and powerful, make MCTs a better tool in oncology research and drug development.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.