2022
Trial of Cinpanemab in Early Parkinson’s Disease
Lang A, Siderowf A, Macklin E, Poewe W, Brooks D, Fernandez H, Rascol O, Giladi N, Stocchi F, Tanner C, Postuma R, Simon D, Tolosa E, Mollenhauer B, Cedarbaum J, Fraser K, Xiao J, Evans K, Graham D, Sapir I, Inra J, Hutchison R, Yang M, Fox T, Budd Haeberlein S, Dam T. Trial of Cinpanemab in Early Parkinson’s Disease. New England Journal Of Medicine 2022, 387: 408-420. PMID: 35921450, DOI: 10.1056/nejmoa2203395.Peer-Reviewed Original ResearchConceptsEarly Parkinson's diseasePrimary end pointSecondary end pointsWeek 52Parkinson's diseaseEnd pointControl groupUnified Parkinson's Disease Rating Scale (UPDRS) total scoreDisease pathogenesisHuman-derived monoclonal antibodiesΑ-synucleinCommon adverse eventsPhase 2 trialDisease-modifying treatmentsMDS-UPDRS scoresLack of efficacyDAT SPECT imagingAdjusted mean differenceMovement Disorder SocietyParkinson's disease pathogenesisScale total scoreAdverse eventsIntravenous infusionDAT-SPECTDisease progression
2021
Longitudinal 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
2018
Targeted Therapies for Parkinson's Disease: From Genetics to the Clinic
Sardi SP, Cedarbaum JM, Brundin P. Targeted Therapies for Parkinson's Disease: From Genetics to the Clinic. Movement Disorders 2018, 33: 684-696. PMID: 29704272, PMCID: PMC6282975, DOI: 10.1002/mds.27414.Peer-Reviewed Original ResearchConceptsParkinson's diseaseGreat unmet medical needDisease-modifying treatmentsNew therapeutic approachesUnmet medical needClinical stageClinical trialsRelentless progressionTherapeutic approachesPotential therapyClinical developmentTherapeutic paradigmMedical needDiseaseGenetic variantsPD geneticsNew arsenalTreatmentGenetic discoveriesKey outstanding questionsTherapySymptomsPathologyProgressionTrials