title: Cerebrospinal fluid biomarkers in trials for Alzheimer and Parkinson diseases.
abstract: Alzheimer disease (AD) and Parkinson disease (PD) are the most common neurodegenerative disorders. For both diseases, early intervention is thought to be essential to the success of disease-modifying treatments. Cerebrospinal fluid (CSF) can reflect some of the pathophysiological changes that occur in the brain, and the number of CSF biomarkers under investigation in neurodegenerative conditions has grown rapidly in the past 20 years. In AD, CSF biomarkers are increasingly being used in clinical practice, and have been incorporated into the majority of clinical trials to demonstrate target engagement, to enrich or stratify patient groups, and to find evidence of disease modification. In PD, CSF biomarkers have not yet reached the clinic, but are being studied in patients with parkinsonism, and are being used in clinical trials either to monitor progression or to demonstrate target engagement and downstream effects of drugs. CSF biomarkers might also serve as surrogate markers of clinical benefit after a specific therapeutic intervention, although additional data are required. It is anticipated that CSF biomarkers will have an important role in trials aimed at disease modification in the near future. In this Review, we provide an overview of CSF biomarkers in AD and PD, and discuss their role in clinical trials.
authors.affiliation: Department of Neurology, Institut d'Investigacions Biomèdiques, Hospital de Sant Pau, Sant Antoni Maria, Claret 167, Barcelona 08025, Spain.
authors.forename: Alberto
authors.initials: A
authors.lastname: Lleó
authors.affiliation: IRCCS Istituto Centro San Giovanni di Dio-Fatebenefratelli, Italy.
authors.forename: Enrica
authors.initials: E
authors.lastname: Cavedo
authors.affiliation: Ospedale Santa Maria della Misericordia, Università di Perugia, Italy.
authors.forename: Lucilla
authors.initials: L
authors.lastname: Parnetti
authors.affiliation: ADxNeurosciences, Belgium.
authors.forename: Hugo
authors.initials: H
authors.lastname: Vanderstichele
authors.affiliation: University of Eastern Finland and Kuopio University Hospital, Finland.
authors.forename: Sanna Kaisa
authors.initials: SK
authors.lastname: Herukka
authors.affiliation: Karolinska Institute, Sweden.
authors.forename: Niels
authors.initials: N
authors.lastname: Andreasen
authors.affiliation: IRCCS Istituto Centro San Giovanni di Dio-Fatebenefratelli, Italy.
authors.forename: Roberta
authors.initials: R
authors.lastname: Ghidoni
authors.affiliation: Universitätsklinikum Erlangen and Friedrich-Alexander-Universität Erlangen-Nürnberg, Germany.
authors.forename: Piotr
authors.initials: P
authors.lastname: Lewczuk
authors.affiliation: Quanterix, USA.
authors.forename: Andreas
authors.initials: A
authors.lastname: Jeromin
authors.affiliation: Karolinska Institute, Sweden.
authors.forename: Bengt
authors.initials: B
authors.lastname: Winblad
authors.affiliation: Aristotle University of Thessaloniki, Greece.
authors.forename: Magda
authors.initials: M
authors.lastname: Tsolaki
authors.affiliation: Medical University of Białystok, Poland.
authors.forename: Barbara
authors.initials: B
authors.lastname: Mroczko
authors.affiliation: Maastricht University, Netherlands.
authors.forename: Pieter Jelle
authors.initials: PJ
authors.lastname: Visser
authors.affiliation: University of Coimbra, Portugal.
authors.forename: Isabel
authors.initials: I
authors.lastname: Santana
authors.affiliation: Karolinska Institute, Sweden.
authors.forename: Per
authors.initials: P
authors.lastname: Svenningsson
authors.affiliation: The Sahlgrenska Academy at University of Gothenburg, Sweden.
authors.forename: Kaj
authors.initials: K
authors.lastname: Blennow
authors.affiliation: Stavanger University Hospital, Norway.
authors.forename: Dag
authors.initials: D
authors.lastname: Aarsland
authors.affiliation: ICN Hospital Clinic i Universitari, IDIBAPS, Spain.
authors.forename: José Luis
authors.initials: JL
authors.lastname: Molinuevo
authors.affiliation: The Sahlgrenska Academy at University of Gothenburg, Sweden.
authors.forename: Henrik
authors.initials: H
authors.lastname: Zetterberg
authors.affiliation: Paracelsus-Elena-Klinik, Germany.
authors.forename: Brit
authors.initials: B
authors.lastname: Mollenhauer
mesh_terms: D000544:Alzheimer Disease / Q000134:cerebrospinal fluid*; D015415:Biomarkers / Q000134:cerebrospinal fluid*; D002986:Clinical Trials as Topic; D006801:Humans; D010300:Parkinson Disease / Q000134:cerebrospinal fluid*
chemical_list: D015415:Biomarkers