Approaches & Applications
Human cell lines, primary cells and stem-cell-derived assays in drug response, safety and translational research.
This collection follows studies using human cells to investigate drug effects, cellular function and disease-related responses. It includes primary cells, established cell lines and stem-cell-derived models, with each article identifying the actual cell source and experimental setting.
Coverage considers what was measured, the relevant comparison and the extent of validation. Related method profiles provide model components, endpoints and published evidence. Organoid and organ-on-chip studies appear here when their human-cell approach is central to the research.
A four-platform comparison combined lipid imaging, liver-function measurements, expression profiling and CYP assays. Lipid accumulation did not uniformly reduce drug metabolism, and differences depended on the model and donor.
A 512-compound screen in patient-derived motor neurons identified resveratrol tetramers with neuroprotective activity. Follow-up work covered 14 iPSC lines and linked protection to changes in neuronal excitability.
A study links sensory-neuron clock state to sodium-channel function. Synchronized human iPSC-derived nociceptors showed clock-state-dependent changes in excitability, and cryptochrome stabilization reduced hyperexcitability.
A preprint reports recovery of selected calcium and potassium currents, repolarization and video-derived contraction after delivery of a Cav1.3 fragment. Sodium-current and hypertrophy-related changes persisted.
Dendritic-cell/T-cell co-cultures and drug-specific clones revealed separate responses to mitiperstat and four metabolites. Most clone activation was consistent with direct pharmacological interaction.
Dual voltage–calcium mapping reveals short-lived reentry under pharmacological long-QT conditions and a subset of more persistent, injury-anchored rotors in human engineered heart tissues.
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.
An academic team co-cultured HepG2 and hepatic stellate LX-2 cells to form a liver tumor spheroid model. Spheroids with a collagen-rich outer layer showed reduced doxorubicin penetration; disrupting that layer improved the drug response in the model.
Researchers used genetic data from 79 people with spinocerebellar ataxia type 3 to design allele-specific antisense oligonucleotides. One candidate lowered mutant ATXN3 by up to 90% in two patient-derived cortical-neuron lines.
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.
A four-center NIH network is studying transporters in the placenta, mammary gland, pediatric intestine and brain. Its published overview describes how organoids, microphysiological systems and computational models contribute to that research strategy.
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.
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.
A spinal-cord-patterned human neural spheroid model followed myelin damage, debris clearance and new myelin formation over weeks. In a small drug comparison, clemastine increased the share of newly formed oligodendrocytes that became myelinating cells, a cellular result rather than evidence of clinical efficacy.
In an IQ Consortium working-group survey of 45 companies in Asia, the United States and Europe, NAM use was most prevalent in early discovery. Uncertainty about regulatory acceptance and human translatability was cited most often as a barrier.
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.
Researchers optimized spheroids from nine breast, ovarian and gastric cancer cell lines before comparing anticancer compounds. Some 3D models appeared more sensitive than their 2D counterparts, while others appeared less sensitive; exposure times also differed.
Cardiomyocytes derived from one CAV3 p.Ala46Thr carrier showed altered protein localization and irregular calcium transients. Fenoterol increased transient frequency, but the pattern remained less regular than in a relative's control cells.
A two-compartment chip assembled from human primary cells maintained a measurable blood–cerebrospinal fluid barrier for up to 15 days. Its first applications compared the distribution of free PEG species and transport of two reference drugs; the latter comparison does not yet establish broad predictive performance.
A Luxembourg team established spheroids from 12 of 19 metastatic colorectal cancer samples. Drug profiling captured several tumor-cell responses, but VEGFR inhibitor readouts did not reflect the small available clinical comparison for antiangiogenic therapy.
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.
