2021
Quantitative digital clock drawing test as a sensitive tool to detect subtle cognitive impairments in early stage Parkinson's disease
Schejter-Margalit T, Kizony R, Shirvan J, Cedarbaum J, Bregman N, Thaler A, Giladi N, Mirelman A. Quantitative digital clock drawing test as a sensitive tool to detect subtle cognitive impairments in early stage Parkinson's disease. Parkinsonism & Related Disorders 2021, 90: 84-89. PMID: 34416663, DOI: 10.1016/j.parkreldis.2021.08.002.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAged, 80 and overCase-Control StudiesCognitionCognitive DysfunctionFemaleGenotypeGlucosylceramidaseHumansLeucine-Rich Repeat Serine-Threonine Protein Kinase-2MaleMental Status and Dementia TestsMiddle AgedMutationNeuropsychological TestsParkinson DiseaseReproducibility of ResultsROC CurveSensitivity and SpecificityConceptsColor Trails TestSubtle cognitive declineClock Drawing TestCognitive AssessmentCognitive profileCognitive declineCognitive testsDigital clock drawing testStandardized neuropsychological testsDrawing TestDigital cognitive assessmentSubtle cognitive impairmentCurrent standardized testsClock-drawing testEarly cognitive declineMontreal Cognitive AssessmentTrails TestNeuropsychological testsSubtle impairmentsHealthy controlsLongitudinal Changes in Neuromelanin MRI Signal in Parkinson's Disease: A Progression Marker
Gaurav R, Yahia‐Cherif L, Pyatigorskaya N, Mangone G, Biondetti E, Valabrègue R, Ewenczyk C, Hutchison R, Cedarbaum J, Corvol J, Vidailhet M, Lehéricy S. Longitudinal Changes in Neuromelanin MRI Signal in Parkinson's Disease: A Progression Marker. Movement Disorders 2021, 36: 1592-1602. PMID: 33751655, PMCID: PMC8359265, DOI: 10.1002/mds.28531.Peer-Reviewed Original ResearchConceptsTotal intracranial volumePD patientsHealthy volunteersParkinson's diseaseIntracranial volumeObservational case-control studySubstantia nigra pars compactaDirect noninvasive assessmentDisease-modifying treatmentsEarly PD patientsFemale PD patientsTherapeutic drug trialsCase-control studyNeuromelanin-sensitive MRI techniquesNeuromelanin-sensitive imagingMale patientsFemale patientsPars compactaCohort IICohort IDrug trialsPatientsImaging biomarkersNoninvasive assessmentDisease severity
2020
Metabolic syndrome does not influence the phenotype of LRRK2 and GBA related Parkinson’s disease
Thaler A, Shenhar-Tsarfaty S, Shaked Y, Gurevich T, Omer N, Bar-Shira A, Gana-Weisz M, Goldstein O, Kestenbaum M, Cedarbaum JM, Orr-Urtreger A, Giladi N, Mirelman A. Metabolic syndrome does not influence the phenotype of LRRK2 and GBA related Parkinson’s disease. Scientific Reports 2020, 10: 9329. PMID: 32518334, PMCID: PMC7283235, DOI: 10.1038/s41598-020-66319-9.Peer-Reviewed Original ResearchConceptsMetabolic syndromeParkinson's diseaseLRRK2-NMCLRRK2-PDComponents of MSGBA-Parkinson diseaseMetabolic componentsProdromal Parkinson's diseaseHigh triglyceride levelsIdiopathic Parkinson's diseaseGBA-NMCGBA-PDElevated triglyceridesBlood pressureLRRK2 carriersProdromal featuresTriglyceride levelsPD groupDiseaseDisease statesLaboratory test resultsPrediabetesSyndromeHigh rateTriglycerides
2019
Revisiting the non-Gaucher-GBA-E326K carrier state: Is it sufficient to increase Parkinson's disease risk?
Goldstein O, Gana-Weisz M, Cohen-Avinoam D, Shiner T, Thaler A, Cedarbaum JM, John S, Lalioti M, Gurevich T, Bar-Shira A, Mirelman A, Giladi N, Orr-Urtreger A. Revisiting the non-Gaucher-GBA-E326K carrier state: Is it sufficient to increase Parkinson's disease risk? Molecular Genetics And Metabolism 2019, 128: 470-475. PMID: 31662221, DOI: 10.1016/j.ymgme.2019.10.001.Peer-Reviewed Original ResearchConceptsPD patientsE326KGBA variantsLRRK2 G2019SDisease riskDisease worldElderly healthy individualsAdditive effectParkinson's disease riskK variantAshkenazi PD patientsCommon genetic risk factorGenetic risk factorsRisk factorsCarrier patientsGBA mutationsPD riskHealthy individualsPatientsCarrier stateGaucher diseaseEarlier AAOAshkenazi originAshkenazi controlsStatistical significanceRandomized phase I clinical trial of anti–α‐synuclein antibody BIIB054
Brys M, Fanning L, Hung S, Ellenbogen A, Penner N, Yang M, Welch M, Koenig E, David E, Fox T, Makh S, Aldred J, Goodman I, Pepinsky B, Liu Y, Graham D, Weihofen A, Cedarbaum JM. Randomized phase I clinical trial of anti–α‐synuclein antibody BIIB054. Movement Disorders 2019, 34: 1154-1163. PMID: 31211448, PMCID: PMC6771554, DOI: 10.1002/mds.27738.Peer-Reviewed Original ResearchConceptsParkinson's disease participantsΑ-synucleinHealthy volunteersParkinson's diseaseHuman-derived monoclonal antibodiesSingle-dose cohortsMost adverse eventsFurther clinical developmentImmunotherapy targetingStudy drugAdverse eventsFavorable safetySingle doseNeuronal dysfunctionSerum ratioDisease progressionCerebrospinal fluidClinical developmentPharmacokinetic parametersPharmacokinetic profileSerum exposureLaboratory assessmentMonoclonal antibodiesDiseaseDose rangeFeasibility and safety of lumbar puncture in the Parkinson's disease research participants: Parkinson's Progression Marker Initiative (PPMI)
Prakash N, Caspell-Garcia C, Coffey C, Siderowf A, Tanner C, Kieburtz K, Mollenhauer B, Galasko D, Merchant K, Foroud T, Chahine L, Weintraub D, Casaceli C, Dorsey R, Wilson R, Herzog M, Daegele N, Arnedo V, Frasier M, Sherer T, Marek K, Frank S, Jennings D, Simuni T, Marek K, Siderowf A, Seibyl J, Coffey C, Tanner C, Tosun-Turgut D, Simuni T, Shaw L, Trojanowski J, Singleton A, Kieburtz K, Toga A, Mollenhauer B, Galasko D, Poewe W, Foroud T, Poston K, Sherer T, Chowdhury S, Frasier M, Kopil C, Arnedo V, Marek K, Daegele N, Casaceli C, Dorsey R, Wilson R, Mahes S, Seibyl J, Salerno C, Coffey C, Caspell-Garcia C, Toga A, Crawford K, Foroud T, Casalin P, Malferrari G, Weisz M, Orr-Urtreger A, Trojanowski J, Shaw L, Singleton A, Foroud T, Foroud T, Montine T, Foroud T, Russell D, Tanner C, Simuni T, Dahodwala N, Mollenhauer B, Galasko D, Poewe W, Giladi N, Factor S, Hogarth P, Standaert D, Hauser R, Jankovic J, Saint-Hilaire M, Richard I, Shprecher D, Fernandez H, Brockmann K, Rosenthal L, Barone P, Espay A, Rowe D, Marder K, Santiago A, Bressman S, Hu S, Isaacson S, Corvol J, Martinez J, Tolosa E, Tai Y, Politis M, Smejdir D, Rees L, Williams K, Kausar F, Williams K, Richardson W, Willeke D, Peacock S, Heim B, Mirelman A, Sommerfeld B, Freed A, Wakeman K, Blair C, Guthrie S, Harrell L, Hunter C, Thomas C, James R, Zimmerman G, Brown V, Mule J, Hilt E, Ribb K, Ainscough S, Wethington M, Ranola M, Santana H, Moreno J, Raymond D, Speketer K, Carvajal L, Carvalho S, Croitoru I, Garrido A, Payne L, Viswanth V, Severt L, Facheris M, Soares H, Mintun M, Cedarbaum J, Taylor P, Biglan K, Vandenbroucke E, Sheikh Z, Bingol B, Fischer T, Sardi P, Forrat R, Reith A, Egebjerg J, Hillert G, Saba B, Min C, Umek R, Mather J, De Santi S, Post A, Boess F, Taylor K, Grachev I, Avbersek A, Muglia P, Merchant K, Tauscher J. Feasibility and safety of lumbar puncture in the Parkinson's disease research participants: Parkinson's Progression Marker Initiative (PPMI). Parkinsonism & Related Disorders 2019, 62: 201-209. PMID: 30738748, PMCID: PMC8978879, DOI: 10.1016/j.parkreldis.2018.12.025.Peer-Reviewed Original ResearchConceptsParkinson's Progression Markers InitiativeProgression Markers InitiativeAdverse eventsLumbar punctureParkinson's diseasePD participantsBaseline lumbar puncturePhone one weekSerial lumbar puncturesCommon adverse eventsRelated adverse eventsCerebrospinal fluid analysisEarly Parkinson's diseaseLow back painLongitudinal observation studyPPMI participantsUnderwent collectionBack painOverall incidenceDopaminergic deficiencyProgression biomarkersFemale genderSafety dataHealthy volunteersHigh incidence
2018
FDG PET Parkinson’s disease-related pattern as a biomarker for clinical trials in early stage disease
Matthews DC, Lerman H, Lukic A, Andrews RD, Mirelman A, Wernick MN, Giladi N, Strother SC, Evans KC, Cedarbaum JM, Even-Sapir E. FDG PET Parkinson’s disease-related pattern as a biomarker for clinical trials in early stage disease. NeuroImage Clinical 2018, 20: 572-579. PMID: 30186761, PMCID: PMC6120603, DOI: 10.1016/j.nicl.2018.08.006.Peer-Reviewed Original ResearchConceptsHealthy controlsDisease-related patternsParkinson's diseaseYahr stagePD patientsClinical trialsParkinson's disease-related patternFluorodeoxyglucose positron emission tomographyGender-matched healthy controlsEarly-stage Parkinson's diseaseMild PD patientsEarly-stage diseaseFDG-PET scansMotor symptom scoresPositron emission tomographyStage diseaseMotor symptomsSymptom scoresFDG-PETDisease stagePD progressionLentiform nucleusParacentral gyrusSymptom evaluationPD subjects
2015
Targeting Prodromal Alzheimer Disease With Avagacestat: A Randomized Clinical Trial
Coric V, Salloway S, van Dyck CH, Dubois B, Andreasen N, Brody M, Curtis C, Soininen H, Thein S, Shiovitz T, Pilcher G, Ferris S, Colby S, Kerselaers W, Dockens R, Soares H, Kaplita S, Luo F, Pachai C, Bracoud L, Mintun M, Grill JD, Marek K, Seibyl J, Cedarbaum JM, Albright C, Feldman HH, Berman RM. Targeting Prodromal Alzheimer Disease With Avagacestat: A Randomized Clinical Trial. JAMA Neurology 2015, 72: 1324-1333. PMID: 26414022, DOI: 10.1001/jamaneurol.2015.0607.Peer-Reviewed Original ResearchConceptsNonmelanoma skin cancerObservational cohortProdromal Alzheimer's diseaseProdromal ADClinical trialsPositron emission tomographyAlzheimer's diseaseDiscontinuation ratesCSF biomarkersBiomarker criteriaTreatment phasePlacebo-controlled phase 2 clinical trialSkin cancerGastrointestinal tract adverse eventsKey clinical outcome measuresSerious adverse event ratesΓ-Secretase Inhibitor AvagacestatEmission tomographyPhase 2 clinical trialPotential disease-modifying agentsBrain atrophy ratesLow discontinuation rateAdverse event ratesDisease-modifying agentsGreater brain atrophyItem response theory analysis of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised in the Pooled Resource Open-Access ALS Clinical Trials Database
Bacci ED, Staniewska D, Coyne KS, Boyer S, White LA, Zach N, Cedarbaum JM, Consortium T. Item response theory analysis of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised in the Pooled Resource Open-Access ALS Clinical Trials Database. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2015, 17: 157-167. PMID: 26473473, DOI: 10.3109/21678421.2015.1095930.Peer-Reviewed Original ResearchConceptsAmyotrophic Lateral Sclerosis Functional Rating Scale-RevisedPooled Resource Open-Access ALS Clinical Trials DatabaseClinical trials databasesTrials databasesAmyotrophic Lateral Sclerosis Functional RatingModern test theory approachesLevel of disabilityALSFRS-R itemsMean ageDisability levelFunctional ratingGross motorDisability severitySevere disabilityPatientsConfirmatory factor analysisItem response theory analysisTreatment effectsDisabilityDomain itemsItem response categoriesResponse categoriesALSFRSInstrument's specificitySpecificity
2012
Safety, tolerability and pharmacodynamics of a skeletal muscle activator in amyotrophic lateral sclerosis
Shefner J, Cedarbaum JM, Cudkowicz ME, Maragakis N, Lee J, Jones D, Watson ML, Mahoney K, Chen M, Saikali K, Mao J, Russell AJ, Hansen RL, Malik F, Wolff AA, Team F. Safety, tolerability and pharmacodynamics of a skeletal muscle activator in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2012, 13: 430-438. PMID: 22591195, DOI: 10.3109/17482968.2012.684214.Peer-Reviewed Original ResearchConceptsAmyotrophic lateral sclerosisSingle dosesGlobal ImpressionLateral sclerosisFast skeletal muscle troponin activatorFrequent adverse eventsDose-related fashionLimb muscle strengthMaximum voluntary ventilationDose-dependent benefitMeasures of enduranceAdverse eventsPulmonary functionVoluntary ventilationGeneral fatigueTroponin activatorMuscle strengthPharmacodynamic markersHandgrip endurancePatientsRandom orderMaximal strengthDosesTolerabilityFurther studies
2011
A phase 2 randomized trial of ELND005, scyllo-inositol, in mild to moderate Alzheimer disease
Salloway S, Sperling R, Keren R, Porsteinsson AP, van Dyck CH, Tariot PN, Gilman S, Arnold D, Abushakra S, Hernandez C, Crans G, Liang E, Quinn G, Bairu M, Pastrak A, Cedarbaum JM. A phase 2 randomized trial of ELND005, scyllo-inositol, in mild to moderate Alzheimer disease. Neurology 2011, 77: 1253-1262. PMID: 21917766, PMCID: PMC3179648, DOI: 10.1212/wnl.0b013e3182309fa5.Peer-Reviewed Original ResearchMeSH KeywordsAdministration, OralAgedAged, 80 and overAlzheimer DiseaseAmyloid beta-PeptidesApolipoprotein E4Dose-Response Relationship, DrugDouble-Blind MethodFemaleFollow-Up StudiesHumansInositolMagnetic Resonance ImagingMaleMental Status ScheduleMiddle AgedPeptide FragmentsPlatelet Aggregation InhibitorsTime FactorsTreatment OutcomeConceptsNeuropsychological test batteryAlzheimer's diseaseDose-ranging phase 2 studyAlzheimer's Disease Cooperative Study-ActivitiesClass II trialsClinical efficacy outcomesCSF biomarker resultsScyllo-inositol concentrationsPhase 2 studyPrimary efficacy analysisHigh-dose groupDaily Living ScaleBrain ventricular volumeCoprimary endpointsEarly discontinuationEfficacy outcomesII trialADCS-ADLDose groupEfficacy analysisAcceptable safetyAβx-42Living ScaleOptimal doseTreatment groupsSuitability of the Clinical Dementia Rating‐Sum of Boxes as a single primary endpoint for Alzheimer's disease trials
Coley N, Andrieu S, Jaros M, Weiner M, Cedarbaum J, Vellas B. Suitability of the Clinical Dementia Rating‐Sum of Boxes as a single primary endpoint for Alzheimer's disease trials. Alzheimer's & Dementia 2011, 7: 602-610.e2. PMID: 21745761, DOI: 10.1016/j.jalz.2011.01.005.Peer-Reviewed Original ResearchConceptsClinical Dementia Rating SumCo-primary endpointsPrimary endpointSingle primary endpointCDR-SBInternal responsivenessFunctional impairmentADAS-CogDisease trialsExternal responsivenessAlzheimer's diseaseAlzheimer's Disease Assessment Scale-cognitive subscaleFloor/ceiling effectsCDR-SB changeDisease-modifying trialsModerate AD patientsDaily Living ScaleAlzheimer's disease trialsConvergent validityInternal consistencyAD trialsDisease stageAD patientsInstrumental activitiesMean change
2010
Phase 1 Study of Aflibercept Administered Subcutaneously to Patients with Advanced Solid Tumors
Tew WP, Gordon M, Murren J, Dupont J, Pezzulli S, Aghajanian C, Sabbatini P, Mendelson D, Schwartz L, Gettinger S, Psyrri A, Cedarbaum JM, Spriggs DR. Phase 1 Study of Aflibercept Administered Subcutaneously to Patients with Advanced Solid Tumors. Clinical Cancer Research 2010, 16: 358-366. PMID: 20028764, PMCID: PMC4211604, DOI: 10.1158/1078-0432.ccr-09-2103.Peer-Reviewed Original ResearchConceptsAdvanced solid tumorsSolid tumorsDrug-related grade 3Vascular endothelial growth factor trapDose of afliberceptDose-escalation studyDose-proportional increaseInjection site reactionsPhase 1 studyManageable side effectsVascular endothelial growth factorWarrants further evaluationFavorable pharmacokinetic profileProgression of diseaseNovel antiangiogenic agentsEndothelial growth factorCommon toxicitiesStable diseasePulmonary embolismCerebral ischemiaSubcutaneous dosesSafety profileSingle doseSite reactionsSubcutaneous formulation
2009
A Phase I Study of Intravitreal Vascular Endothelial Growth Factor Trap-Eye in Patients with Neovascular Age-Related Macular Degeneration
Nguyen QD, Shah SM, Browning DJ, Hudson H, Sonkin P, Hariprasad SM, Kaiser P, Slakter JS, Haller J, V. D, Mieler WF, Chu K, Yang K, Ingerman A, Vitti RL, Berliner AJ, Cedarbaum JM, Campochiaro PA. A Phase I Study of Intravitreal Vascular Endothelial Growth Factor Trap-Eye in Patients with Neovascular Age-Related Macular Degeneration. Ophthalmology 2009, 116: 2141-2148.e1. PMID: 19700196, DOI: 10.1016/j.ophtha.2009.04.030.Peer-Reviewed Original ResearchMeSH KeywordsAgedAged, 80 and overChoroidal NeovascularizationFemaleFluorescein AngiographyFollow-Up StudiesHumansInjectionsMacular DegenerationMaleMaximum Tolerated DoseReceptors, Vascular Endothelial Growth FactorRecombinant Fusion ProteinsTomography, Optical CoherenceTreatment OutcomeVisual AcuityVitreous BodyConceptsVEGF Trap-EyeAge-related macular degenerationVascular endothelial growth factor (VEGF) Trap-EyeNeovascular age-related macular degenerationNeovascular AMDChoroidal neovascularizationOptical coherence tomographyFoveal thicknessIntravitreal injectionVisual acuityMacular degenerationMean increaseArea of CNVExcess foveal thicknessSingle intraocular injectionPrimary end pointSerious adverse eventsPhase III trialsHigh-dose groupInterventional clinical trialsActive choroidal neovascularizationNumber of patientsHuman immunoglobulin G (IgG) FcVEGF receptor 1VEGF family membersAn exploratory study of the safety, tolerability and bioactivity of a single intravitreal injection of vascular endothelial growth factor Trap-Eye in patients with diabetic macular oedema
V D, Nguyen QD, Shah SM, Browning DJ, Haller JA, Chu K, Yang K, Cedarbaum JM, Vitti RL, Ingerman A, Campochiaro PA. An exploratory study of the safety, tolerability and bioactivity of a single intravitreal injection of vascular endothelial growth factor Trap-Eye in patients with diabetic macular oedema. British Journal Of Ophthalmology 2009, 93: 144. PMID: 19174400, DOI: 10.1136/bjo.2008.138271.Peer-Reviewed Original ResearchConceptsVEGF Trap-EyeDiabetic macular edemaSingle intravitreal injectionVascular endothelial growth factor (VEGF) Trap-EyeExcess foveal thicknessFoveal thicknessIntravitreal injectionOptical coherence tomographyMacular edemaUnrelated serious adverse eventsMedian baseline BCVASerious adverse eventsBaseline BCVAETDRS lettersAdverse eventsRetinal thicknessVisual acuityOcular toxicityOutcome measuresBCVAMedian improvementPatientsCoherence tomographyAdditional studiesObservation period
2006
A Phase I Trial of an IV-Administered Vascular Endothelial Growth Factor Trap for Treatment in Patients with Choroidal Neovascularization due to Age-Related Macular Degeneration
Nguyen QD, Shah SM, Hafiz G, Quinlan E, Sung J, Chu K, Cedarbaum JM, Campochiaro PA, Group C. A Phase I Trial of an IV-Administered Vascular Endothelial Growth Factor Trap for Treatment in Patients with Choroidal Neovascularization due to Age-Related Macular Degeneration. Ophthalmology 2006, 113: 1522.e1-1522.e14. PMID: 16876249, DOI: 10.1016/j.ophtha.2006.05.055.Peer-Reviewed Original ResearchMeSH KeywordsAgedAged, 80 and overChoroidal NeovascularizationDose-Response Relationship, DrugFemaleFluorescein AngiographyHumansInfusions, IntravenousMacular DegenerationMaleMaximum Tolerated DoseMiddle AgedReceptors, Vascular Endothelial Growth FactorRecombinant Fusion ProteinsRetinaTomography, Optical CoherenceVisual AcuityConceptsVascular endothelial growth factor trapAge-related macular degenerationVEGF TrapRetinal thicknessVisual acuityMacular degenerationEarly Treatment Diabetic Retinopathy Study protocolNeovascular age-related macular degenerationDiabetic Retinopathy Study protocolPlacebo-controlled clinical trialDoses 2 weeksDose-limiting toxicityPhase I trialMean percent changeVEGF receptor 1Optical coherence tomographyAdverse eventsI trialDose groupChoroidal neovascularizationStudy protocolClinical trialsStudy populationMultiple administrationsTherapeutic window
2005
NT-3 promotes nerve regeneration and sensory improvement in CMT1A mouse models and in patients
Sahenk Z, Nagaraja HN, McCracken BS, King WM, Freimer ML, Cedarbaum JM, Mendell JR. NT-3 promotes nerve regeneration and sensory improvement in CMT1A mouse models and in patients. Neurology 2005, 65: 681-689. PMID: 16157899, DOI: 10.1212/01.wnl.0000171978.70849.c5.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAnimalsCharcot-Marie-Tooth DiseaseDisease Models, AnimalDouble-Blind MethodFemaleHumansMaleMiceMice, Neurologic MutantsMice, NudeMice, TransgenicMiddle AgedMyelin ProteinsNerve RegenerationNeurotrophin 3Pilot ProjectsRecovery of FunctionSciatic NeuropathySural NerveTransplantation, HeterologousTreatment OutcomeConceptsNeuropathy Impairment ScoreNT-3 groupNT-3 treatmentNeurotrophin-3Placebo groupAnimal modelsNerve regenerationNude mice axonsQuantitative muscle testingSural nerve biopsyPrimary outcome measurePeripheral myelin proteinPilot clinical studyTooth type 1ANerve biopsyMuscle testingAxonal regenerationClinical studiesImpairment scoresOutcome measuresPreclinical studiesMouse modelEndpoint measuresElectrophysiologic measurementsNude mice
2003
Neurotrophin-3 Improves Functional Constipation
Parkman HP, Rao SS, Reynolds JC, Schiller LR, Wald A, Miner PB, Lembo AJ, Gordon JM, Drossman DA, Waltzman L, Stambler N, Cedarbaum JM. Neurotrophin-3 Improves Functional Constipation. The American Journal Of Gastroenterology 2003, 98: ajg2003312. PMID: 12818279, DOI: 10.1111/j.1572-0241.2003.t01-1-07477.x.Peer-Reviewed Original ResearchConceptsComplete bowel movementsNeurotrophin-3Bowel movementsColon transitFunctional constipationChronic constipationStool frequencyPlacebo-controlled phase II studyTransient injection site reactionsConstipation-related symptomsFrequent adverse eventsPhase II studyThird of patientsInjection site reactionsEnd of treatmentDose-related effectsConstipated subjectsBowel functionPrimary endpointAdverse eventsII studyImproved symptomsWeekly dosingNeurotrophic factorConstipation
2000
Recombinant human neurotrophic factors accelerate colonic transit and relieve constipation in humans
Coulie B, Szarka L, Camilleri M, Burton D, McKinzie S, Stambler N, Cedarbaum J. Recombinant human neurotrophic factors accelerate colonic transit and relieve constipation in humans. Gastroenterology 2000, 119: 41-50. PMID: 10889153, DOI: 10.1053/gast.2000.8553.Peer-Reviewed Original ResearchConceptsNeurotrophic factorStool frequencyColonic transitR-metHuBDNFHealthy subjectsBrain-derived neurotrophic factorHuman brain-derived neurotrophic factorHuman gut motilityOverall colonic transitPassage of stoolRecombinant human brain-derived neurotrophic factorGastrointestinal motor functionSmall bowel transitInjection site reactionsWeeks of treatmentNeurotrophic factor 3Neurotrophin administrationBowel transitRandomized studyGut motilityColonic emptyingSite reactionsMotor functionConstipationHealthy people
1999
A retrospective study of percutaneous endoscopic gastrostomy in ALS patients during the BDNF and CNTF trials
Kasarskis E, Scarlata D, Hill R, Fuller C, Stambler N, Cedarbaum J, in the . B. A retrospective study of percutaneous endoscopic gastrostomy in ALS patients during the BDNF and CNTF trials. Journal Of The Neurological Sciences 1999, 169: 118-125. PMID: 10540019, DOI: 10.1016/s0022-510x(99)00230-0.Peer-Reviewed Original ResearchConceptsPercutaneous endoscopic gastrostomyALS patientsEndoscopic gastrostomyClinical statusRetrospective studyVital capacityNutritional interventionNutritional supplementationRetrospective analysisPEG insertionPatientsBDNF studiesRate of declineDisease statusEarly interventionMarked reductionWeight lossGastrostomyCNTFInterventionReduction of functionSequential measurementsDaysStatusDysphagia