Approaches & Applications
Research on patient-derived and stem-cell-derived organoids for drug response, safety and disease modeling.
Explore research using patient-derived or stem-cell-derived organoids to study drug response and disease biology. The collection spans cancer models and models of non-malignant tissues, while keeping their origins, composition and research purposes distinct.
Articles explain the tested treatment, comparison and outcome within the available original evidence. Method profiles give further detail where the source supports it. Coverage distinguishes experimental drug sensitivity, comparisons with patient outcomes and prospective evaluation.
A study connects aloperine binding and VPS34 lactylation to disrupted endosomal trafficking in lung cancer cells. Patient-derived organoids provided additional evidence of a stronger response to aloperine plus cisplatin than to either agent alone.
Single-cell analysis identified conserved epithelial programs associated with survival and resistance. Patient-derived organoid experiments then shifted platinum responses through SERCA2 activation or CCDC6 inhibition.
Assembloids from six patients reproduced their corresponding immunotherapy-response patterns. Component exchange and perturbations connected tumor WNT2B, fibroblast activin A and stem-like CD8 T cells, with low stromal LRP6 linked to resistance.
A paired comparison found medium-dependent responses to 33 compounds in three pancreatic and colorectal tumor organoid models. Switching medium partly reversed sensitivity, but the study did not test prediction of patient outcomes.
A review describes glioblastoma organoids as human systems linking molecular profiling with functional treatment tests. It identifies unresolved issues in perfusion, immune components, matrix consistency and clinical validation.
A Westlake University team trained an image-analysis framework with fluorescence-derived labels, then used bright-field images to estimate viability and death patterns. A 24-compound A2780 screen revealed responses that bulk ATP measurements alone did not distinguish.
A Research Square preprint quantified uptake of 13 drugs in human-cell blood–brain barrier organoids. For measurable drugs, organoid uptake ratios correlated with published mammalian brain-to-plasma data, although the reference data and organoids themselves varied.
A review of bright-field and phase-contrast imaging finds different strengths and limits in rule-based, classical machine-learning and deep-learning analysis of spheroids and organoids. Dataset shifts, segmentation failures and two-dimensional projections remain practical concerns.
A bioRxiv preprint describes Matrigel-droplet tumor organoids used to screen more than 5,000 clinical compounds and run pooled CRISPR perturbations. The authors report candidate drug opportunities beyond genomic profiling and a UXS1-linked cell-state change in a stomach cancer model.
Researchers exposed human hippocampal organoids to valproic acid for four days, then profiled mRNA and miRNA. They found 19 altered miRNAs and tested selected target sequences in HEK-293T reporter cells, with limited replication from one iPSC line.
Isogenic human liver organoids with LIPA loss reproduced lipid accumulation and multicellular inflammatory and biliary responses with increased fibrogenic gene expression. Restoring LIPA in hepatocyte-like cells attenuated these molecular signals.
Researchers linked APOE4-associated lipid accumulation in human oligodendrocytes to altered Wnt/GSK3B signaling. A GSK3B inhibitor reduced lipid droplets in cell cultures and increased myelination in brain organoids; the findings remain preclinical.
Roche used patient-derived colorectal tumor assembloids to compare its engineered FAP-targeted IFNγ agonists with recombinant IFNγ and an untargeted reference. The targeted constructs induced some immune signals with less PD-L1, while Ki67 and CD8 migration changes did not reach significance.
A Journal of Clinical Investigation review compares iPSC-derived neurons, cocultures, organoids and directly converted neurons for neurodegeneration research. The authors distinguish a model’s cellular complexity from its ability to retain an older donor’s biological age.
A microbe–gut–cartilage chip linked a probiotic-exposed intestinal module to human cartilage organoids. Five enriched metabolites were subsequently tested on cartilage organoids made from pooled cells from three osteoarthritis donors.
Expression-based models trained on 2D cancer cultures retained a weak ranking signal in independent organoid cohorts. Their added value over tumor-lineage information in the blinded Sanger cohort remained uncertain.
Both patient-derived intestinal organoid cultures tested showed increased swelling after exposure to elexacaftor/tezacaftor/ivacaftor (ETI), but a retrospective 12-person cohort did not show consistent improvement across clinical measures.
Researchers exposed human cortical organoids to acetaminophen for five days and followed structural, transcriptomic and network outcomes for several months. Small early transcriptional changes did not translate into detectable differences in later cellular composition or the measured functional endpoints.
An academic team established organoids from 15 of 18 glioblastoma samples and recovered matched primary cells during processing. A two-patient temozolomide comparison illustrates exploratory use of the models, with very limited clinical follow-up.
Semaglutide partly protected human forebrain organoids against the structural and metabolic effects of cholesterol stress. The response was more limited in older cultures, where some calcium activity measures improved without restoring tissue size or several disease-associated markers.
A six-drug study combines human intestinal organoid injury, epithelial turnover and pharmacokinetic models. Accounting for local intestinal exposure improves agreement with reported clinical diarrhea for drugs whose plasma concentrations understate the modeled intestinal effect.
A platform built around a flexible microelectrode array and scoop-shaped well combines electrical recordings with video of tissue motion. Organoids from healthy donors and patients with Duchenne muscular dystrophy show different structural features and responses to hERG blockade.
Two hiPSC-derived proximal tubule models show how transporter localization relates to directional drug transport and inhibitor responses. Transport activity and longer-term cell viability do not necessarily favor the same model.
