Multiple system atrophy: a prototypical synucleinopathy for disease-modifying therapeutic strategies.
Identifieur interne : 000910 ( Main/Exploration ); précédent : 000909; suivant : 000911Multiple system atrophy: a prototypical synucleinopathy for disease-modifying therapeutic strategies.
Auteurs : Pierre-Olivier Fernagut [France] ; Benjamin Dehay [France] ; Aline Maillard [France] ; Erwan Bezard [France] ; Paul Perez [France] ; Anne Pavy-Le Traon [France] ; Olivier Rascol [France] ; Alexandra Foubert-Samier [France] ; François Tison [France] ; Wassilios G. Meissner [France]Source :
- Neurobiology of disease [ 1095-953X ] ; 2014.
English descriptors
- KwdEn :
- MESH :
- chemical , metabolism : alpha-Synuclein.
- metabolism : Brain, Multiple System Atrophy.
- pathology : Multiple System Atrophy.
- therapy : Multiple System Atrophy, Parkinson Disease.
- Clinical Trials as Topic, Humans.
Abstract
Despite active fundamental, translational and clinical research, no therapeutic intervention has yet shown convincing effects on disease progression in Parkinson's disease (PD) patients. Indeed, several disease-modification trials failed or proved to be inconclusive due to lack of consistency between clinical rating scales and putative surrogate markers of disease progression, or confounding symptomatic effects of the tested compound. Multiple system atrophy (MSA) is a rapidly progressing orphan disorder leading to severe motor disability within a few years. Together with PD and dementia with Lewy bodies (DLB), MSA belongs to the synucleinopathies, a group of neurodegenerative disorders characterized by the abnormal accumulation of alpha-synuclein. Crucial milestones have been reached for successfully conducting clinical intervention trials in a large number of patients with MSA. In this personal view, we will review evidence, and discuss why MSA could prove the most relevant clinical model for assessing treatments that target mechanisms operating in all synucleinopathies.
DOI: 10.1016/j.nbd.2014.03.021
PubMed: 24727096
Affiliations:
- France
- Aquitaine, Midi-Pyrénées, Nouvelle-Aquitaine, Occitanie (région administrative)
- Bordeaux, Pessac, Toulouse
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CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac</wicri:regionArea><placeName><region type="region" nuts="2">Nouvelle-Aquitaine</region><region type="old region" nuts="2">Aquitaine</region><settlement type="city">Pessac</settlement></placeName></affiliation></author><author><name sortKey="Perez, Paul" sort="Perez, Paul" uniqKey="Perez P" first="Paul" last="Perez">Paul Perez</name><affiliation wicri:level="3"><nlm:affiliation>CHU de Bordeaux, Unité de Soutien Méthodologique à la Recherche Clinique (USMR), Pôle de santé publique, Bordeaux, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>CHU de Bordeaux, Unité de Soutien Méthodologique à la Recherche Clinique (USMR), Pôle de santé publique, Bordeaux</wicri:regionArea><placeName><region type="region">Nouvelle-Aquitaine</region><region type="old region">Aquitaine</region><settlement type="city">Bordeaux</settlement></placeName></affiliation></author><author><name sortKey="Pavy Le Traon, Anne" sort="Pavy Le Traon, Anne" uniqKey="Pavy Le Traon A" first="Anne" last="Pavy-Le Traon">Anne Pavy-Le Traon</name><affiliation wicri:level="3"><nlm:affiliation>Centre de référence atrophie multisystématisée, CHU de Toulouse, Toulouse, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Centre de référence atrophie multisystématisée, CHU de Toulouse, Toulouse</wicri:regionArea><placeName><region type="region">Occitanie (région administrative)</region><region type="old region">Midi-Pyrénées</region><settlement type="city">Toulouse</settlement></placeName></affiliation></author><author><name sortKey="Rascol, Olivier" sort="Rascol, Olivier" uniqKey="Rascol O" first="Olivier" last="Rascol">Olivier Rascol</name><affiliation wicri:level="3"><nlm:affiliation>Centre de référence atrophie multisystématisée, CHU de Toulouse, Toulouse, France; Department of Clinical Pharmacology, University Hospital and University of Toulouse 3, Toulouse, France; Department of Neurosciences, University Hospital and University of Toulouse 3, Toulouse, France; INSERM UMR825 and CIC9302, Toulouse, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Centre de référence atrophie multisystématisée, CHU de Toulouse, Toulouse, France; Department of Clinical Pharmacology, University Hospital and University of Toulouse 3, Toulouse, France; Department of Neurosciences, University Hospital and University of Toulouse 3, Toulouse, France; INSERM UMR825 and CIC9302, Toulouse</wicri:regionArea><placeName><region type="region">Occitanie (région administrative)</region><region type="old region">Midi-Pyrénées</region><settlement type="city">Toulouse</settlement></placeName></affiliation></author><author><name sortKey="Foubert Samier, Alexandra" sort="Foubert Samier, Alexandra" uniqKey="Foubert Samier A" first="Alexandra" last="Foubert-Samier">Alexandra Foubert-Samier</name><affiliation wicri:level="1"><nlm:affiliation>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac, France; Centre de référence atrophie multisystématisée, CHU de Bordeaux, Pessac, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac, France; Centre de référence atrophie multisystématisée, CHU de Bordeaux, Pessac</wicri:regionArea><wicri:noRegion>Pessac</wicri:noRegion><wicri:noRegion>Pessac</wicri:noRegion></affiliation></author><author><name sortKey="Tison, Francois" sort="Tison, Francois" uniqKey="Tison F" first="François" last="Tison">François Tison</name><affiliation wicri:level="1"><nlm:affiliation>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; 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CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux</wicri:regionArea><placeName><region type="region" nuts="2">Nouvelle-Aquitaine</region><region type="old region" nuts="2">Aquitaine</region><settlement type="city">Bordeaux</settlement></placeName></affiliation></author><author><name sortKey="Dehay, Benjamin" sort="Dehay, Benjamin" uniqKey="Dehay B" first="Benjamin" last="Dehay">Benjamin Dehay</name><affiliation wicri:level="3"><nlm:affiliation>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux</wicri:regionArea><placeName><region type="region" nuts="2">Nouvelle-Aquitaine</region><region type="old region" nuts="2">Aquitaine</region><settlement type="city">Bordeaux</settlement></placeName></affiliation></author><author><name sortKey="Maillard, Aline" sort="Maillard, Aline" uniqKey="Maillard A" first="Aline" last="Maillard">Aline Maillard</name><affiliation wicri:level="3"><nlm:affiliation>CHU de Bordeaux, Unité de Soutien Méthodologique à la Recherche Clinique (USMR), Pôle de santé publique, Bordeaux, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>CHU de Bordeaux, Unité de Soutien Méthodologique à la Recherche Clinique (USMR), Pôle de santé publique, Bordeaux</wicri:regionArea><placeName><region type="region">Nouvelle-Aquitaine</region><region type="old region">Aquitaine</region><settlement type="city">Bordeaux</settlement></placeName></affiliation></author><author><name sortKey="Bezard, Erwan" sort="Bezard, Erwan" uniqKey="Bezard E" first="Erwan" last="Bezard">Erwan Bezard</name><affiliation wicri:level="3"><nlm:affiliation>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac</wicri:regionArea><placeName><region type="region" nuts="2">Nouvelle-Aquitaine</region><region type="old region" nuts="2">Aquitaine</region><settlement type="city">Pessac</settlement></placeName></affiliation></author><author><name sortKey="Perez, Paul" sort="Perez, Paul" uniqKey="Perez P" first="Paul" last="Perez">Paul Perez</name><affiliation wicri:level="3"><nlm:affiliation>CHU de Bordeaux, Unité de Soutien Méthodologique à la Recherche Clinique (USMR), Pôle de santé publique, Bordeaux, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>CHU de Bordeaux, Unité de Soutien Méthodologique à la Recherche Clinique (USMR), Pôle de santé publique, Bordeaux</wicri:regionArea><placeName><region type="region">Nouvelle-Aquitaine</region><region type="old region">Aquitaine</region><settlement type="city">Bordeaux</settlement></placeName></affiliation></author><author><name sortKey="Pavy Le Traon, Anne" sort="Pavy Le Traon, Anne" uniqKey="Pavy Le Traon A" first="Anne" last="Pavy-Le Traon">Anne Pavy-Le Traon</name><affiliation wicri:level="3"><nlm:affiliation>Centre de référence atrophie multisystématisée, CHU de Toulouse, Toulouse, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Centre de référence atrophie multisystématisée, CHU de Toulouse, Toulouse</wicri:regionArea><placeName><region type="region">Occitanie (région administrative)</region><region type="old region">Midi-Pyrénées</region><settlement type="city">Toulouse</settlement></placeName></affiliation></author><author><name sortKey="Rascol, Olivier" sort="Rascol, Olivier" uniqKey="Rascol O" first="Olivier" last="Rascol">Olivier Rascol</name><affiliation wicri:level="3"><nlm:affiliation>Centre de référence atrophie multisystématisée, CHU de Toulouse, Toulouse, France; Department of Clinical Pharmacology, University Hospital and University of Toulouse 3, Toulouse, France; Department of Neurosciences, University Hospital and University of Toulouse 3, Toulouse, France; INSERM UMR825 and CIC9302, Toulouse, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Centre de référence atrophie multisystématisée, CHU de Toulouse, Toulouse, France; Department of Clinical Pharmacology, University Hospital and University of Toulouse 3, Toulouse, France; Department of Neurosciences, University Hospital and University of Toulouse 3, Toulouse, France; INSERM UMR825 and CIC9302, Toulouse</wicri:regionArea><placeName><region type="region">Occitanie (région administrative)</region><region type="old region">Midi-Pyrénées</region><settlement type="city">Toulouse</settlement></placeName></affiliation></author><author><name sortKey="Foubert Samier, Alexandra" sort="Foubert Samier, Alexandra" uniqKey="Foubert Samier A" first="Alexandra" last="Foubert-Samier">Alexandra Foubert-Samier</name><affiliation wicri:level="1"><nlm:affiliation>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac, France; Centre de référence atrophie multisystématisée, CHU de Bordeaux, Pessac, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac, France; Centre de référence atrophie multisystématisée, CHU de Bordeaux, Pessac</wicri:regionArea><wicri:noRegion>Pessac</wicri:noRegion><wicri:noRegion>Pessac</wicri:noRegion></affiliation></author><author><name sortKey="Tison, Francois" sort="Tison, Francois" uniqKey="Tison F" first="François" last="Tison">François Tison</name><affiliation wicri:level="1"><nlm:affiliation>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac, France; Centre de référence atrophie multisystématisée, CHU de Bordeaux, Pessac, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac, France; Centre de référence atrophie multisystématisée, CHU de Bordeaux, Pessac</wicri:regionArea><wicri:noRegion>Pessac</wicri:noRegion><wicri:noRegion>Pessac</wicri:noRegion></affiliation></author><author><name sortKey="Meissner, Wassilios G" sort="Meissner, Wassilios G" uniqKey="Meissner W" first="Wassilios G" last="Meissner">Wassilios G. Meissner</name><affiliation wicri:level="1"><nlm:affiliation>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac, France; Centre de référence atrophie multisystématisée, CHU de Bordeaux, Pessac, France. Electronic address: wassilios.meissner@chu-bordeaux.fr.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France; Service de Neurologie, CHU de Bordeaux, F-33604 Pessac, France; Centre de référence atrophie multisystématisée, CHU de Bordeaux, Pessac</wicri:regionArea><wicri:noRegion>Pessac</wicri:noRegion><wicri:noRegion>Pessac</wicri:noRegion></affiliation></author></analytic><series><title level="j">Neurobiology of disease</title><idno type="eISSN">1095-953X</idno><imprint><date when="2014" type="published">2014</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Brain (metabolism)</term><term>Clinical Trials as Topic</term><term>Humans</term><term>Multiple System Atrophy (metabolism)</term><term>Multiple System Atrophy (pathology)</term><term>Multiple System Atrophy (therapy)</term><term>Parkinson Disease (therapy)</term><term>alpha-Synuclein (metabolism)</term></keywords><keywords scheme="MESH" type="chemical" qualifier="metabolism" xml:lang="en"><term>alpha-Synuclein</term></keywords><keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Brain</term><term>Multiple System Atrophy</term></keywords><keywords scheme="MESH" qualifier="pathology" xml:lang="en"><term>Multiple System Atrophy</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Multiple System Atrophy</term><term>Parkinson Disease</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Clinical Trials as Topic</term><term>Humans</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Despite active fundamental, translational and clinical research, no therapeutic intervention has yet shown convincing effects on disease progression in Parkinson's disease (PD) patients. Indeed, several disease-modification trials failed or proved to be inconclusive due to lack of consistency between clinical rating scales and putative surrogate markers of disease progression, or confounding symptomatic effects of the tested compound. Multiple system atrophy (MSA) is a rapidly progressing orphan disorder leading to severe motor disability within a few years. Together with PD and dementia with Lewy bodies (DLB), MSA belongs to the synucleinopathies, a group of neurodegenerative disorders characterized by the abnormal accumulation of alpha-synuclein. Crucial milestones have been reached for successfully conducting clinical intervention trials in a large number of patients with MSA. In this personal view, we will review evidence, and discuss why MSA could prove the most relevant clinical model for assessing treatments that target mechanisms operating in all synucleinopathies.</div></front></TEI><affiliations><list><country><li>France</li></country><region><li>Aquitaine</li><li>Midi-Pyrénées</li><li>Nouvelle-Aquitaine</li><li>Occitanie (région administrative)</li></region><settlement><li>Bordeaux</li><li>Pessac</li><li>Toulouse</li></settlement></list><tree><country name="France"><region name="Nouvelle-Aquitaine"><name sortKey="Fernagut, Pierre Olivier" sort="Fernagut, Pierre Olivier" uniqKey="Fernagut P" first="Pierre-Olivier" last="Fernagut">Pierre-Olivier Fernagut</name></region><name sortKey="Bezard, Erwan" sort="Bezard, Erwan" uniqKey="Bezard E" first="Erwan" last="Bezard">Erwan Bezard</name><name sortKey="Dehay, Benjamin" sort="Dehay, Benjamin" uniqKey="Dehay B" first="Benjamin" last="Dehay">Benjamin Dehay</name><name sortKey="Foubert Samier, Alexandra" sort="Foubert Samier, Alexandra" uniqKey="Foubert Samier A" first="Alexandra" last="Foubert-Samier">Alexandra Foubert-Samier</name><name sortKey="Maillard, Aline" sort="Maillard, Aline" uniqKey="Maillard A" first="Aline" last="Maillard">Aline Maillard</name><name sortKey="Meissner, Wassilios G" sort="Meissner, Wassilios G" uniqKey="Meissner W" first="Wassilios G" last="Meissner">Wassilios G. Meissner</name><name sortKey="Pavy Le Traon, Anne" sort="Pavy Le Traon, Anne" uniqKey="Pavy Le Traon A" first="Anne" last="Pavy-Le Traon">Anne Pavy-Le Traon</name><name sortKey="Perez, Paul" sort="Perez, Paul" uniqKey="Perez P" first="Paul" last="Perez">Paul Perez</name><name sortKey="Rascol, Olivier" sort="Rascol, Olivier" uniqKey="Rascol O" first="Olivier" last="Rascol">Olivier Rascol</name><name sortKey="Tison, Francois" sort="Tison, Francois" uniqKey="Tison F" first="François" last="Tison">François Tison</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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