2025
Applying single cell profiling to assess drug anti fibrotic properties in the human precision cut lung slice model of fibrosis
Justet A, Mitash N, Pineda R, Adams T, Balayev A, Abu Hussein N, Ishizuka M, Kim H, Khoury J, Cala-García J, Ahangari F, Yan X, Kaminski N, Königshoff M. Applying single cell profiling to assess drug anti fibrotic properties in the human precision cut lung slice model of fibrosis. Revue Des Maladies Respiratoires 2025, 42: 223. DOI: 10.1016/j.rmr.2025.02.083.Peer-Reviewed Original ResearchAbnormal cell populationsAlveolar epithelial cellsTreated with drugsAnti-fibrotic propertiesSingle cell platformsSingle nuclear RNA sequencingLigand-receptor analysisPreclinical evidencePulmonary fibrosisBasaloid cellsFibrotic propertiesReceptor analysisFibrotic pathwaysCell signaturesClinical trialsProfibrotic genesAnti-fibroticFDA-approved drugsGene signatureAnimal modelsEpithelial cellsGene expression changesDay 5Drug efficacyCell profilesChapter 49 Phase 0 & window of opportunity clinical trials
Reddy A, Bansal U, Lerner S. Chapter 49 Phase 0 & window of opportunity clinical trials. 2025, 249-253. DOI: 10.1016/b978-0-323-90186-4.00009-2.Peer-Reviewed Original ResearchExploratory investigational new drugClinical trialsPhase 1 clinical trialPreclinical animal studiesFood and Drug AdministrationInvestigational new drugOn-target effectsPhase 0 studiesDrug pharmacokineticsDrug efficacyDrug AdministrationAnimal studiesOncology drugsDrug trialsSuboptimal pharmacological propertiesNew drugsDrug approvalDrugDrug approval processCosts associated with drug developmentPharmacological propertiesDrug safetyTrialsDrug developmentTherapeutic areas
2024
How many patients do you need? Investigating trial designs for anti‐seizure treatment in acute brain injury patients
Parikh H, Sun H, Amerineni R, Rosenthal E, Volfovsky A, Rudin C, Westover M, Zafar S. How many patients do you need? Investigating trial designs for anti‐seizure treatment in acute brain injury patients. Annals Of Clinical And Translational Neurology 2024, 11: 1681-1690. PMID: 38867375, PMCID: PMC11251465, DOI: 10.1002/acn3.52059.Peer-Reviewed Original ResearchConceptsRandomized Controlled TrialsAnti-seizure treatmentAcute brain injuryEpileptiform activityBrain injuryTreatment armsModel treatment responsePlacebo-controlled trialAcute brain injury patientsSingle-center cohortDrug efficacy dataBrain injury patientsCohort of adultsDischarge modified Rankin ScaleFeasibility of randomized controlled trialsSample sizePlacebo armTreatment responseDrug doseEA trajectoriesEfficacy dataWorsen outcomesRankin ScaleDrug efficacyPlacebo
2023
In Utero Electroporated Neurons for Medium-Throughput Screening of Compounds Regulating Neuron Morphology
Sokolov A, Aurich M, Bordey A. In Utero Electroporated Neurons for Medium-Throughput Screening of Compounds Regulating Neuron Morphology. ENeuro 2023, 10: eneuro.0160-23.2023. PMID: 37620147, PMCID: PMC10464655, DOI: 10.1523/eneuro.0160-23.2023.Peer-Reviewed Original ResearchConceptsSomatosensory cortexCortical pyramidal neuronsTreatment of epilepsyNeurite overgrowthNeurologic disabilityPyramidal neuronsSoma sizeNovel agentsCircuit alterationsSide effectsMorphologic assessmentMouse neuronsRelated disordersDiseased neuronsMTOR activatorDrug efficacyCandidate therapeuticsNeuronsNeuron morphologyMTOR activityMedium-throughput screeningNeurodevelopmental disordersNeurite lengthMorphologic measurementsDisorders
2021
Influence of immunomodulatory drugs on the gut microbiota
Cohen I, Ruff WE, Longbrake EE. Influence of immunomodulatory drugs on the gut microbiota. Translational Research 2021, 233: 144-161. PMID: 33515779, PMCID: PMC8184576, DOI: 10.1016/j.trsl.2021.01.009.Peer-Reviewed Original ResearchMeSH KeywordsAdaptive ImmunityCell ProliferationCytokinesFemaleGastrointestinal MicrobiomeHost Microbial InteractionsHumansImmune Checkpoint InhibitorsImmunity, InnateImmunity, MucosalImmunologic FactorsImmunomodulationLymphocytesMaleModels, ImmunologicalSex FactorsTranslational Research, BiomedicalConceptsImmune checkpoint inhibitorsGut microbiotaCheckpoint inhibitorsAutoimmune diseasesMainstay of treatmentType of immunotherapyInflammatory cytokine inhibitorComprehensive literature searchMechanism of actionImmunomodulatory medicationsDrug-induced shiftImmunomodulatory drugsCytokine inhibitorsImmunotherapy effectImmune cellsClinical significanceImmune responseMost immunotherapiesCommensal microbesDrug efficacyImmunotherapyLiterature searchAntiproliferative drugsMedicationsMicrobiotaAccelerating precision anti-cancer therapy by time-lapse and label-free 3D tumor slice culture platform
Xing F, Liu Y, Huang S, Lyu X, Su S, Chan U, Wu P, Yan Y, Ai N, Li J, Zhao M, Rajendran B, Liu J, Shao F, Sun H, Choi T, Zhu W, Luo G, Liu S, Xu D, Chan K, Zhao Q, Miao K, Luo K, Ge W, Xu X, Wang G, Liu T, Deng C. Accelerating precision anti-cancer therapy by time-lapse and label-free 3D tumor slice culture platform. Theranostics 2021, 11: 9415-9430. PMID: 34646378, PMCID: PMC8490519, DOI: 10.7150/thno.59533.Peer-Reviewed Original ResearchConceptsAnti-cancer therapyNew pre-clinical modelsDay of surgeryPre-clinical modelsRNA sequence analysisHigh-efficacy drugsAnti-cancer drug efficacyPD-1PD-L1Personalized medicineBlockade assayAnti-cancer drug discoveryImmune componentsOriginal tumorAutofluorescence featuresDrug efficacyHistological analysisPrecision anti-cancer therapyCancer treatmentDrug responseHigh-throughput drug screeningDrug discoveryTherapyEfficacyCell-based high-throughput drug screening
2020
Immuno-pharmacokinetics of Meglumine Antimoniate in Patients With Cutaneous Leishmaniasis Caused by Leishmania (Viannia)
Gómez MA, Navas A, Prieto M, Giraldo-Parra L, Cossio A, Alexander N, Saravia N. Immuno-pharmacokinetics of Meglumine Antimoniate in Patients With Cutaneous Leishmaniasis Caused by Leishmania (Viannia). Clinical Infectious Diseases 2020, 72: e484-e492. PMID: 32818964, PMCID: PMC8130027, DOI: 10.1093/cid/ciaa1206.Peer-Reviewed Original ResearchConceptsPeripheral blood mononuclear cellsMeglumine antimoniateCutaneous leishmaniasisBlood mononuclear cellsHuman cutaneous leishmaniasisIntracellular drug concentrationImmune gene expressionPK evidenceClinical cureParasite clearanceClinical resolutionDrug regimensMononuclear cellsPharmacodynamic parametersPharmacodynamic relationshipsLeishmania VianniaDrug treatmentTargeted immunomodulationTherapeutic outcomesImmune dynamicsDrug efficacyGene signatureDrug concentrationsProfile of expressionMicrobial kill
2019
Positron-Emission Tomographic Imaging of a Fluorine 18–Radiolabeled Poly(ADP-Ribose) Polymerase 1 Inhibitor Monitors the Therapeutic Efficacy of Talazoparib in SCLC Patient–Derived Xenografts
Laird J, Lok B, Carney B, Kossatz S, de Stanchina E, Reiner T, Poirier J, Rudin C. Positron-Emission Tomographic Imaging of a Fluorine 18–Radiolabeled Poly(ADP-Ribose) Polymerase 1 Inhibitor Monitors the Therapeutic Efficacy of Talazoparib in SCLC Patient–Derived Xenografts. Journal Of Thoracic Oncology 2019, 14: 1743-1752. PMID: 31195178, PMCID: PMC6764879, DOI: 10.1016/j.jtho.2019.05.032.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsApoptosisCell ProliferationFemaleFluorine RadioisotopesHumansLung NeoplasmsMiceMice, Inbred NODMice, SCIDPhthalazinesPoly (ADP-Ribose) Polymerase-1Poly(ADP-ribose) Polymerase InhibitorsPositron-Emission TomographyRadiopharmaceuticalsSmall Cell Lung CarcinomaTumor Cells, CulturedXenograft Model Antitumor AssaysConceptsPatient-derived xenograftsPositron emission tomographicPARP inhibitorsTherapeutic efficacyTarget engagementPositron emission tomographic imagingDifferential therapeutic efficacyTumor radiotracer uptakeTumor growth inhibitionEnzyme-linked immunosorbentDose-dependent mannerFluorine-18Oral talazoparibMultiple dosesSingle dosesTotal tumorsPolymerase-1 (PARP-1) inhibitorsPET uptakeRadiotracer uptakeInhibitor of polyIndividual tumorsPET/Drug efficacyTalazoparib treatmentPromising therapeuticsPhenotyping ciliary dynamics and coordination in response to CFTR-modulators in Cystic Fibrosis respiratory epithelial cells
Chioccioli M, Feriani L, Kotar J, Bratcher PE, Cicuta P. Phenotyping ciliary dynamics and coordination in response to CFTR-modulators in Cystic Fibrosis respiratory epithelial cells. Nature Communications 2019, 10: 1763. PMID: 30992452, PMCID: PMC6467870, DOI: 10.1038/s41467-019-09798-3.Peer-Reviewed Original ResearchConceptsHuman airway epithelial cellsEpithelial cellsCystic fibrosis respiratory epithelial cellsAirway epithelial cellsRespiratory epithelial cellsCiliary beat frequencyCiliary beatingRespiratory disordersRespiratory diseaseClinical observationsCystic fibrosisDrug efficacyPilot studyDrug testingEfficient drugsDrugsIndividual donorsEfficacyDrug screeningBeat dynamicsCellsSame genotypeFibrosisDonorsDiseaseSeparating host and microbiome contributions to drug pharmacokinetics and toxicity
Zimmermann M, Zimmermann-Kogadeeva M, Wegmann R, Goodman AL. Separating host and microbiome contributions to drug pharmacokinetics and toxicity. Science 2019, 363 PMID: 30733391, PMCID: PMC6533120, DOI: 10.1126/science.aat9931.Peer-Reviewed Original ResearchConceptsMicrobiome contributionDrug metabolismFunction of bioavailabilityDrug-metabolizing activitySame metabolic transformationsMetabolism of drugsSystemic drugsMetabolite exposureMetabolite absorptionGut microbiotaDrug pharmacokineticsDrug efficacyPharmacokinetic modelDrugsMedical drugsTransit kineticsMetabolismInterpersonal variationMetabolic transformationToxicityMetabolic routesPharmacokineticsMice
2018
Direct-to-Consumer Broadcast Advertisements for Pharmaceuticals: Off-Label Promotion and Adherence to FDA Guidelines
Klara K, Kim J, Ross JS. Direct-to-Consumer Broadcast Advertisements for Pharmaceuticals: Off-Label Promotion and Adherence to FDA Guidelines. Journal Of General Internal Medicine 2018, 33: 651-658. PMID: 29484575, PMCID: PMC5910340, DOI: 10.1007/s11606-017-4274-9.Peer-Reviewed Original ResearchConceptsDTC adsDiabetes medicationsPresentation of risksLabel promotionPrescription drugsBlood pressure reductionFDA guidelinesBladder dysfunctionDiabetic neuropathyResultsOur sampleChronic conditionsInflammatory conditionsAllergic reactionsLabel usesPrescribers' decisionsUnique drugDrug AdministrationDrug risksDrug efficacyPressure reductionOff-label promotionWeight lossAdvertisement claimsConsumer advertisementsDrugs
2017
Augmentation of Anticancer Drug Efficacy in Murine Hepatocellular Carcinoma Cells by a Peripherally Acting Competitive N‑Methyl‑d‑aspartate (NMDA) Receptor Antagonist
Gynther M, Silvestri I, Hansen J, Hansen K, Malm T, Ishchenko Y, Larsen Y, Han L, Kayser S, Auriola S, Petsalo A, Nielsen B, Pickering D, Bunch L. Augmentation of Anticancer Drug Efficacy in Murine Hepatocellular Carcinoma Cells by a Peripherally Acting Competitive N‑Methyl‑d‑aspartate (NMDA) Receptor Antagonist. Journal Of Medicinal Chemistry 2017, 60: 9885-9904. PMID: 29205034, PMCID: PMC5788303, DOI: 10.1021/acs.jmedchem.7b01624.Peer-Reviewed Original ResearchConceptsMurine hepatocellular carcinoma cellsHepatocellular carcinoma cellsN-methyl-D-aspartate receptor antagonistCompetitive N-methyl-D-aspartate (NMDA) receptor antagonistMultidrug resistanceCarcinoma cellsCancer cellsCommon solid tumorsNMDA receptor inhibitionIntrinsic multidrug resistanceReceptor antagonistReceptor inhibitionAnticancer drug efficacyUnderlying biological mechanismsSolid tumorsDrug efficacyReduced expressionCytotoxic actionSorafenibStructure-activity studiesMDR transportersAnticancer drugsBiological mechanismsCellsPromising strategyGeneric atorvastatin is as effective as the brand-name drug (LIPITOR®) in lowering cholesterol levels: a cross-sectional retrospective cohort study
Loch A, Bewersdorf J, Kofink D, Ismail D, Abidin I, Veriah R. Generic atorvastatin is as effective as the brand-name drug (LIPITOR®) in lowering cholesterol levels: a cross-sectional retrospective cohort study. BMC Research Notes 2017, 10: 291. PMID: 28716156, PMCID: PMC5514478, DOI: 10.1186/s13104-017-2617-6.Peer-Reviewed Original ResearchConceptsStatistically significant differenceEffective management of hyperlipidemiaHealth care costsSignificant differenceUniversity of Malaya Medical CentreRetrospective cohort studyHDL cholesterol levelsManagement of hyperlipidemiaLipid lowering effectsIncreased health care costsAtorvastatin formulationsLowering cholesterol levelsSwitching patientsCare costsLDL cholesterolMethodsThis cross-sectionalCohort studyLipid profileTotal cholesterolGeneric atorvastatinDrug efficacyAtorvastatinMedical CentreCholesterol levelsPatients
2016
A prospective comparison of ER, PR, Ki67 and gene expression in paired sequential core biopsies of primary, untreated breast cancer
Hadad SM, Jordan LB, Roy PG, Purdie CA, Iwamoto T, Pusztai L, Moulder-Thompson SL, Thompson AM. A prospective comparison of ER, PR, Ki67 and gene expression in paired sequential core biopsies of primary, untreated breast cancer. BMC Cancer 2016, 16: 745. PMID: 27658825, PMCID: PMC5034430, DOI: 10.1186/s12885-016-2788-x.Peer-Reviewed Original ResearchIngenuity Pathway AnalysisBreast cancerCore biopsyDrug therapyOperable breast cancerSequential core biopsiesUntreated breast cancerHormone receptor statusPrimary breast cancerPathway analysisPR immunohistochemistryUntreated patientsReceptor statusPrimary cancerNon-treatment controlBiomarker statusProspective comparisonBiomarker changesDrug interventionKi67 scoreBiopsyBiomarker effectsDrug efficacyKi67MRNA expression
2014
Application of a Poisson distribution quality control measure to the analysis of two human hookworm drug treatment studies in Ghana
Kotze AC, Dobson RJ, Humphries D, Wilson M, Cappello M. Application of a Poisson distribution quality control measure to the analysis of two human hookworm drug treatment studies in Ghana. International Journal For Parasitology Drugs And Drug Resistance 2014, 4: 64-70. PMID: 24596670, PMCID: PMC3940077, DOI: 10.1016/j.ijpddr.2014.01.001.Peer-Reviewed Original ResearchDrug treatmentFaecal egg count reduction testDrug efficacyPre-treatment samplesDrug treatment studiesOverall drug efficacyStudy subjectsSignificant prevalenceEgg excretionPoisson distribution analysisTreatment studiesEfficacySignificant increaseEgg count reduction testSubjectsTreatmentControl measuresSuch subjectsReduction testStoolCasesPoisson distribution methodExcretionPrevalenceQuality control measuresCardiovascular pharmacogenetics of anti-thrombotic agents and non-steroidal anti-inflammatory drugs.
Stitham J, Vanichakarn P, Ying L, Hwa J. Cardiovascular pharmacogenetics of anti-thrombotic agents and non-steroidal anti-inflammatory drugs. 2014, 14: 909-31. PMID: 25109796, DOI: 10.2174/1566524014666140811121109.Peer-Reviewed Original ResearchConceptsNon-steroidal anti-inflammatory drugsAnti-inflammatory drugsCardiovascular pharmacogeneticsAdverse cardiovascular eventsAppropriate treatment regimenAnti-thrombotic agentsNSAID therapyCardiovascular eventsThromboembolic eventsCardiovascular implicationsCardiovascular riskPoor respondersTreatment regimenProspective screeningMyocardial infarctionAdverse reactionsAntithrombotic agentsCardiovascular diseaseHealthcare providersDrug efficacyGenetic biomarkersMedicationsIndividualized predictionInter-individual variationDrugs
2013
Sialorrhea: Anatomy, Pathophysiology and Treatment with Emphasis on the Role of Botulinum Toxins
Lakraj AA, Moghimi N, Jabbari B. Sialorrhea: Anatomy, Pathophysiology and Treatment with Emphasis on the Role of Botulinum Toxins. Toxins 2013, 5: 1010-1031. PMID: 23698357, PMCID: PMC3709276, DOI: 10.3390/toxins5051010.Peer-Reviewed Original ResearchConceptsBotulinum toxinSide effectsAdministration of BoNTLevel B evidencePlacebo-controlled studyCurrent medical managementTroublesome side effectsLevel of evidenceOral agentsCerebral palsyClinical featuresExcessive droolingMedical managementTopical agentsB evidenceBlinded studyMedical treatmentAmerican AcademyClinical notesDrug efficacyNeurodegenerative disordersClinical pointB levelsSalivary glandsType BHyperhidrosis: Anatomy, Pathophysiology and Treatment with Emphasis on the Role of Botulinum Toxins
Lakraj AA, Moghimi N, Jabbari B. Hyperhidrosis: Anatomy, Pathophysiology and Treatment with Emphasis on the Role of Botulinum Toxins. Toxins 2013, 5: 821-840. PMID: 23612753, PMCID: PMC3705293, DOI: 10.3390/toxins5040821.Peer-Reviewed Original ResearchConceptsClass II studiesII studyAxillary hyperhidrosisPalmar hyperhidrosisComparator studiesBotulinum toxinClass IEvidence level BLevel of evidenceClass III studiesOral agentsIII studyClinical featuresTopical agentsComparable efficacyLong lasting effectInjection sessionsBlinded studyAmerican AcademyDrug efficacyClass IIHyperhidrosisEfficacyWorld literatureToxin
2012
Noninvasive Monitoring of Immunosuppressive Drug Efficacy to Prevent Rejection of Intracerebral Glial Precursor Allografts
Gorelik M, Janowski M, Galpoththawela C, Rifkin R, Levy M, Lukomska B, Kerr D, Bulte J, Walczak P, Morgan R. Noninvasive Monitoring of Immunosuppressive Drug Efficacy to Prevent Rejection of Intracerebral Glial Precursor Allografts. Cell Transplantation 2012, 21: 2149-2157. PMID: 22508097, DOI: 10.3727/096368912x636911.Peer-Reviewed Original ResearchConceptsCentral nervous systemBioluminescence imagingAutologous transplanted cellsDevelopment of cell-based therapiesCombination of rapamycinLong-term survivalCell-based therapiesDevelopment of patient-specificGraft toleranceImmunocompetent miceCell transplantationGraft rejectionGlial-restricted progenitorsCell rejectionImmunodeficient animalsPrevent rejectionImmunosuppressed miceImmunocompetent animalsCyclosporin AGrafted areaTransplanted cellsAllograftDrug efficacyInflammatory processMyriad of diseasesDevelopmental expression of drug metabolizing enzymes: Impact on disposition in neonates and young children
Hines R. Developmental expression of drug metabolizing enzymes: Impact on disposition in neonates and young children. International Journal Of Pharmaceutics 2012, 452: 3-7. PMID: 22766445, DOI: 10.1016/j.ijpharm.2012.05.079.Peer-Reviewed Original ResearchConceptsPerinatal changesDrug dispositionPediatric drug safetyYoung childrenDrug metabolizing enzymesAge-dependent changesSignificant interindividual variationAdverse eventsPharmacogenetic factorsHepatic drugFunctional genetic variantsDrug safetyDrug efficacyMetabolizing enzymesPharmacokinetic modelInterindividual variationEnzyme ontogenyDrugsEnzyme expressionChildrenClass 3Genetic variantsMajor determinantClass 1Current knowledge
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