Recommendations of the Global Multiple System Atrophy Research Roadmap Meeting

Ryan R Walsh¹, Florian Krismer², Wendy R Galpern², Gregor K Wenning², Phillip A Low², Glenda Halliday², Walter J Koroshetz², Janice Holton², Niall P Quinn², Olivier Rascol², Leslie M Shaw², David Eidelberg², Pam Bower², Jeffrey L Cummings², Victor Abler², Judy Biedenharn², Gal Bitan², David J Brooks², Patrik Brundin², Hubert Fernandez², Philip Fortier², Roy Freeman², Thomas Gasser², Art Hewitt², Günter U Höglinger², Matt J Huentelman², Poul H Jensen², Andreas Jeromin², Un Jung Kang², Horacio Kaufmann², Lawrence Kellerman², Vikram Khurana², Thomas Klockgether², Woojin Scott Kim², Carol Langer², Peter LeWitt², Eliezer Masliah², Wassilios Meissner², Ronald Melki², Susanne Ostrowitzki², Steven Piantadosi², Werner Poewe², David Robertson², Cyndi Roemer², Dale Schenk², Michael Schlossmacher², Jeremy D Schmahmann², Klaus Seppi², Lily Shih², Andrew Siderowf², Glenn T Stebbins², Nadia Stefanova², Shoji Tsuji², Sharon Sutton², Jing Zhang²

Affiliations

¹ From Muhammad Ali Parkinson Center (R.R.W.), Barrow Neurological Institute, Phoenix, AZ; Department of Neurology (F.K., G.K.W., W.P., K.S., N.S.), Medical University Innsbruck, Austria; National Institute of Neurological Disorders and Stroke (W.R.G., W.J.K.), NIH, Bethesda, MD; Department of Neurology (P.A.L.), Mayo Clinic, Rochester, MN; Brain and Mind Centre (G.H.), Sydney Medical School, the University of Sydney; UNSW Medicine & Neuroscience Research Australia (G.H.), Sydney; Queen Square Brain Bank (J.H.), UCL Institute of Neurology (N.P.Q.), London, UK; UPS, INSERM, and CHU de Toulouse (O.R.), University de Toulouse; Centre de Référence Maladie Rare contre l'AMS (O.R.), Centre d'Investigation Clinique CIC1436, Départments de Pharmacologie Clinique et de Neurosciences, NeuroToul/COEN Center of Excellence in Neurodegeneration, Toulouse, France; Department of Pathology & Laboratory Medicine (L.M.S.), Perelman School of Medicine, University of Pennsylvania, Philadelphia; Center for Neurosciences (D.E.), The Feinstein Institute for Medical Research, Manhasset, NY; The Multiple System Atrophy (MSA) Coalition, Inc. (P. B., J.B., P.F., L.K., C.L., C.R., S.S.), Charlotte, NC; Cleveland Clinic Lou Ruvo Center for Brain Health (J.L.C.), Las Vegas, NV; Medical Affairs (V.A.), Teva Pharmaceuticals, Frazer, PA; Department of Neurology (G.B.), David Geffen School of Medicine, Brain Research Institute, and Molecular Biology Institute, University of California at Los Angeles; Institute of Neurology (D.J.B.), University of Newcastle upon Tyne, UK; Van Andel Research Institute (P. Brundin), Center for Neurodegenerative Science, Grand Rapids, MI; Center for Neurological Restoration (H.F.), Cleveland Clinic, OH; Defeat MSA (P.F.); MSA Shoe Charity (P.F., C.R.), Saint Clair Shores, MI; Department of Neurology (R.F.), Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA; Department of Neurodegenerative Diseases (T.G.), Hertie Institute of Clinical Brain Research, University of Tübingen; German Center for Neurodegenerative Diseases (DZNE) (T.G.), Tübingen, Germany; Lundbeck LLC (A.H.), Deerfield, IL; German Center for Neurodegenerative Diseases (DZNE) (G.U.H.), Munich; Department of Neurology (G.U.H.), Technical University of Munich, Germany; Neurogenomics Division (M.J.H.), The Translational Genomics Research Institute, Phoenix, AZ; Dandrite & Department of Biomedicine (P.H.J.), Aarhus University, Denmark; Quanterix (A.J.), Lexington, MA; Department of Neurology (U.J.K.), Columbia University Medical Center; Department of Neurology (H.K.), New York University School of Medicine, New York; Whitehead Institute for Biomedical Research (V.K.), Cambridge; Ann Romney Center for Neurologic Disease (V.K.), Department of Neurology, Brigham and Women's Hospital and Harvard Medical School, Boston; Harvard Stem Cell Institute (V.K.), Cambridge, MA; Department of Neurology (T.K.), University of Bonn; German Center for Neurodegenerative Diseases (DZNE) (T.K.), Bonn, Germany; Brain and Mind Centre (W.S.K.), Sydney Medical School, University of Sydney, Australia; Department of Neurology (P.L.), Henry Ford Hospital; Department of Neurology (P.L.), Wayne State University School of Medicine, Detroit, MI; Division of Neurosciences (E.M.), National Institute on Aging/NIH, Bethesda, MD; Centre de Référence Maladie Rare AMS (W.M.), Hôpital Pellegrin, CHU de Bordeaux; Université de Bordeaux (W.M.), Institut des Maladies Neurodégénératives, UMR 5293, Bordeaux; Paris Saclay Institute of Neuroscience (R.M.), CNRS, Gif-sur-Yvette, France; Genentech Inc. (S.O.), South San Francisco; Cedars-Sinai Medical Center (S.P.), Los Angeles, CA; Department of Neurology (D.R.), Vanderbilt University Medical Center, Nashville, TN; MSA NJ (C.R.), Howell, NJ; Prothena Biosciences (D.S.), South San Francisco, CA; Division of Neurology (M.S.), Department of Medicine, The Ottawa Hospital, Canada; Department of Neurology (J.D.S.), Massachusetts General Hospital, Harvard Medical School, Boston, MA; Cure MSA Foundation (L.S.), Fremont, CA; Avid Radiopharmaceuticals (A.S.), Philadelphia, PA; Department of Neurological Sciences (G.T.S.), Rush University Medical Center, Chicago, IL; Department of Neurology (S.T.), Graduate School of Medicine, Tokyo University, Tokyo, Japan; and University of Washington (J.Z.), Seattle. W.R.G. is currently affiliated with Janssen Research and Development, Titusville, NJ. Ryan.Walsh@BarrowNeuro.org.
² From Muhammad Ali Parkinson Center (R.R.W.), Barrow Neurological Institute, Phoenix, AZ; Department of Neurology (F.K., G.K.W., W.P., K.S., N.S.), Medical University Innsbruck, Austria; National Institute of Neurological Disorders and Stroke (W.R.G., W.J.K.), NIH, Bethesda, MD; Department of Neurology (P.A.L.), Mayo Clinic, Rochester, MN; Brain and Mind Centre (G.H.), Sydney Medical School, the University of Sydney; UNSW Medicine & Neuroscience Research Australia (G.H.), Sydney; Queen Square Brain Bank (J.H.), UCL Institute of Neurology (N.P.Q.), London, UK; UPS, INSERM, and CHU de Toulouse (O.R.), University de Toulouse; Centre de Référence Maladie Rare contre l'AMS (O.R.), Centre d'Investigation Clinique CIC1436, Départments de Pharmacologie Clinique et de Neurosciences, NeuroToul/COEN Center of Excellence in Neurodegeneration, Toulouse, France; Department of Pathology & Laboratory Medicine (L.M.S.), Perelman School of Medicine, University of Pennsylvania, Philadelphia; Center for Neurosciences (D.E.), The Feinstein Institute for Medical Research, Manhasset, NY; The Multiple System Atrophy (MSA) Coalition, Inc. (P. B., J.B., P.F., L.K., C.L., C.R., S.S.), Charlotte, NC; Cleveland Clinic Lou Ruvo Center for Brain Health (J.L.C.), Las Vegas, NV; Medical Affairs (V.A.), Teva Pharmaceuticals, Frazer, PA; Department of Neurology (G.B.), David Geffen School of Medicine, Brain Research Institute, and Molecular Biology Institute, University of California at Los Angeles; Institute of Neurology (D.J.B.), University of Newcastle upon Tyne, UK; Van Andel Research Institute (P. Brundin), Center for Neurodegenerative Science, Grand Rapids, MI; Center for Neurological Restoration (H.F.), Cleveland Clinic, OH; Defeat MSA (P.F.); MSA Shoe Charity (P.F., C.R.), Saint Clair Shores, MI; Department of Neurology (R.F.), Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA; Department of Neurodegenerative Diseases (T.G.), Hertie Institute of Clinical Brain Research, University of Tübingen; German Center for Neurodegenerative Diseases (DZNE) (T.G.), Tübingen, Germany; Lundbeck LLC (A.H.), Deerfield, IL; German Center for Neurodegenerative Diseases (DZNE) (G.U.H.), Munich; Department of Neurology (G.U.H.), Technical University of Munich, Germany; Neurogenomics Division (M.J.H.), The Translational Genomics Research Institute, Phoenix, AZ; Dandrite & Department of Biomedicine (P.H.J.), Aarhus University, Denmark; Quanterix (A.J.), Lexington, MA; Department of Neurology (U.J.K.), Columbia University Medical Center; Department of Neurology (H.K.), New York University School of Medicine, New York; Whitehead Institute for Biomedical Research (V.K.), Cambridge; Ann Romney Center for Neurologic Disease (V.K.), Department of Neurology, Brigham and Women's Hospital and Harvard Medical School, Boston; Harvard Stem Cell Institute (V.K.), Cambridge, MA; Department of Neurology (T.K.), University of Bonn; German Center for Neurodegenerative Diseases (DZNE) (T.K.), Bonn, Germany; Brain and Mind Centre (W.S.K.), Sydney Medical School, University of Sydney, Australia; Department of Neurology (P.L.), Henry Ford Hospital; Department of Neurology (P.L.), Wayne State University School of Medicine, Detroit, MI; Division of Neurosciences (E.M.), National Institute on Aging/NIH, Bethesda, MD; Centre de Référence Maladie Rare AMS (W.M.), Hôpital Pellegrin, CHU de Bordeaux; Université de Bordeaux (W.M.), Institut des Maladies Neurodégénératives, UMR 5293, Bordeaux; Paris Saclay Institute of Neuroscience (R.M.), CNRS, Gif-sur-Yvette, France; Genentech Inc. (S.O.), South San Francisco; Cedars-Sinai Medical Center (S.P.), Los Angeles, CA; Department of Neurology (D.R.), Vanderbilt University Medical Center, Nashville, TN; MSA NJ (C.R.), Howell, NJ; Prothena Biosciences (D.S.), South San Francisco, CA; Division of Neurology (M.S.), Department of Medicine, The Ottawa Hospital, Canada; Department of Neurology (J.D.S.), Massachusetts General Hospital, Harvard Medical School, Boston, MA; Cure MSA Foundation (L.S.), Fremont, CA; Avid Radiopharmaceuticals (A.S.), Philadelphia, PA; Department of Neurological Sciences (G.T.S.), Rush University Medical Center, Chicago, IL; Department of Neurology (S.T.), Graduate School of Medicine, Tokyo University, Tokyo, Japan; and University of Washington (J.Z.), Seattle. W.R.G. is currently affiliated with Janssen Research and Development, Titusville, NJ.

PMID: 29237794
PMCID: PMC5772155
DOI: 10.1212/WNL.0000000000004798

Recommendations of the Global Multiple System Atrophy Research Roadmap Meeting

Ryan R Walsh et al. Neurology. 2018.

. 2018 Jan 9;90(2):74-82.

doi: 10.1212/WNL.0000000000004798. Epub 2017 Dec 13.

Authors

Affiliations

¹ From Muhammad Ali Parkinson Center (R.R.W.), Barrow Neurological Institute, Phoenix, AZ; Department of Neurology (F.K., G.K.W., W.P., K.S., N.S.), Medical University Innsbruck, Austria; National Institute of Neurological Disorders and Stroke (W.R.G., W.J.K.), NIH, Bethesda, MD; Department of Neurology (P.A.L.), Mayo Clinic, Rochester, MN; Brain and Mind Centre (G.H.), Sydney Medical School, the University of Sydney; UNSW Medicine & Neuroscience Research Australia (G.H.), Sydney; Queen Square Brain Bank (J.H.), UCL Institute of Neurology (N.P.Q.), London, UK; UPS, INSERM, and CHU de Toulouse (O.R.), University de Toulouse; Centre de Référence Maladie Rare contre l'AMS (O.R.), Centre d'Investigation Clinique CIC1436, Départments de Pharmacologie Clinique et de Neurosciences, NeuroToul/COEN Center of Excellence in Neurodegeneration, Toulouse, France; Department of Pathology & Laboratory Medicine (L.M.S.), Perelman School of Medicine, University of Pennsylvania, Philadelphia; Center for Neurosciences (D.E.), The Feinstein Institute for Medical Research, Manhasset, NY; The Multiple System Atrophy (MSA) Coalition, Inc. (P. B., J.B., P.F., L.K., C.L., C.R., S.S.), Charlotte, NC; Cleveland Clinic Lou Ruvo Center for Brain Health (J.L.C.), Las Vegas, NV; Medical Affairs (V.A.), Teva Pharmaceuticals, Frazer, PA; Department of Neurology (G.B.), David Geffen School of Medicine, Brain Research Institute, and Molecular Biology Institute, University of California at Los Angeles; Institute of Neurology (D.J.B.), University of Newcastle upon Tyne, UK; Van Andel Research Institute (P. Brundin), Center for Neurodegenerative Science, Grand Rapids, MI; Center for Neurological Restoration (H.F.), Cleveland Clinic, OH; Defeat MSA (P.F.); MSA Shoe Charity (P.F., C.R.), Saint Clair Shores, MI; Department of Neurology (R.F.), Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA; Department of Neurodegenerative Diseases (T.G.), Hertie Institute of Clinical Brain Research, University of Tübingen; German Center for Neurodegenerative Diseases (DZNE) (T.G.), Tübingen, Germany; Lundbeck LLC (A.H.), Deerfield, IL; German Center for Neurodegenerative Diseases (DZNE) (G.U.H.), Munich; Department of Neurology (G.U.H.), Technical University of Munich, Germany; Neurogenomics Division (M.J.H.), The Translational Genomics Research Institute, Phoenix, AZ; Dandrite & Department of Biomedicine (P.H.J.), Aarhus University, Denmark; Quanterix (A.J.), Lexington, MA; Department of Neurology (U.J.K.), Columbia University Medical Center; Department of Neurology (H.K.), New York University School of Medicine, New York; Whitehead Institute for Biomedical Research (V.K.), Cambridge; Ann Romney Center for Neurologic Disease (V.K.), Department of Neurology, Brigham and Women's Hospital and Harvard Medical School, Boston; Harvard Stem Cell Institute (V.K.), Cambridge, MA; Department of Neurology (T.K.), University of Bonn; German Center for Neurodegenerative Diseases (DZNE) (T.K.), Bonn, Germany; Brain and Mind Centre (W.S.K.), Sydney Medical School, University of Sydney, Australia; Department of Neurology (P.L.), Henry Ford Hospital; Department of Neurology (P.L.), Wayne State University School of Medicine, Detroit, MI; Division of Neurosciences (E.M.), National Institute on Aging/NIH, Bethesda, MD; Centre de Référence Maladie Rare AMS (W.M.), Hôpital Pellegrin, CHU de Bordeaux; Université de Bordeaux (W.M.), Institut des Maladies Neurodégénératives, UMR 5293, Bordeaux; Paris Saclay Institute of Neuroscience (R.M.), CNRS, Gif-sur-Yvette, France; Genentech Inc. (S.O.), South San Francisco; Cedars-Sinai Medical Center (S.P.), Los Angeles, CA; Department of Neurology (D.R.), Vanderbilt University Medical Center, Nashville, TN; MSA NJ (C.R.), Howell, NJ; Prothena Biosciences (D.S.), South San Francisco, CA; Division of Neurology (M.S.), Department of Medicine, The Ottawa Hospital, Canada; Department of Neurology (J.D.S.), Massachusetts General Hospital, Harvard Medical School, Boston, MA; Cure MSA Foundation (L.S.), Fremont, CA; Avid Radiopharmaceuticals (A.S.), Philadelphia, PA; Department of Neurological Sciences (G.T.S.), Rush University Medical Center, Chicago, IL; Department of Neurology (S.T.), Graduate School of Medicine, Tokyo University, Tokyo, Japan; and University of Washington (J.Z.), Seattle. W.R.G. is currently affiliated with Janssen Research and Development, Titusville, NJ. Ryan.Walsh@BarrowNeuro.org.
² From Muhammad Ali Parkinson Center (R.R.W.), Barrow Neurological Institute, Phoenix, AZ; Department of Neurology (F.K., G.K.W., W.P., K.S., N.S.), Medical University Innsbruck, Austria; National Institute of Neurological Disorders and Stroke (W.R.G., W.J.K.), NIH, Bethesda, MD; Department of Neurology (P.A.L.), Mayo Clinic, Rochester, MN; Brain and Mind Centre (G.H.), Sydney Medical School, the University of Sydney; UNSW Medicine & Neuroscience Research Australia (G.H.), Sydney; Queen Square Brain Bank (J.H.), UCL Institute of Neurology (N.P.Q.), London, UK; UPS, INSERM, and CHU de Toulouse (O.R.), University de Toulouse; Centre de Référence Maladie Rare contre l'AMS (O.R.), Centre d'Investigation Clinique CIC1436, Départments de Pharmacologie Clinique et de Neurosciences, NeuroToul/COEN Center of Excellence in Neurodegeneration, Toulouse, France; Department of Pathology & Laboratory Medicine (L.M.S.), Perelman School of Medicine, University of Pennsylvania, Philadelphia; Center for Neurosciences (D.E.), The Feinstein Institute for Medical Research, Manhasset, NY; The Multiple System Atrophy (MSA) Coalition, Inc. (P. B., J.B., P.F., L.K., C.L., C.R., S.S.), Charlotte, NC; Cleveland Clinic Lou Ruvo Center for Brain Health (J.L.C.), Las Vegas, NV; Medical Affairs (V.A.), Teva Pharmaceuticals, Frazer, PA; Department of Neurology (G.B.), David Geffen School of Medicine, Brain Research Institute, and Molecular Biology Institute, University of California at Los Angeles; Institute of Neurology (D.J.B.), University of Newcastle upon Tyne, UK; Van Andel Research Institute (P. Brundin), Center for Neurodegenerative Science, Grand Rapids, MI; Center for Neurological Restoration (H.F.), Cleveland Clinic, OH; Defeat MSA (P.F.); MSA Shoe Charity (P.F., C.R.), Saint Clair Shores, MI; Department of Neurology (R.F.), Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA; Department of Neurodegenerative Diseases (T.G.), Hertie Institute of Clinical Brain Research, University of Tübingen; German Center for Neurodegenerative Diseases (DZNE) (T.G.), Tübingen, Germany; Lundbeck LLC (A.H.), Deerfield, IL; German Center for Neurodegenerative Diseases (DZNE) (G.U.H.), Munich; Department of Neurology (G.U.H.), Technical University of Munich, Germany; Neurogenomics Division (M.J.H.), The Translational Genomics Research Institute, Phoenix, AZ; Dandrite & Department of Biomedicine (P.H.J.), Aarhus University, Denmark; Quanterix (A.J.), Lexington, MA; Department of Neurology (U.J.K.), Columbia University Medical Center; Department of Neurology (H.K.), New York University School of Medicine, New York; Whitehead Institute for Biomedical Research (V.K.), Cambridge; Ann Romney Center for Neurologic Disease (V.K.), Department of Neurology, Brigham and Women's Hospital and Harvard Medical School, Boston; Harvard Stem Cell Institute (V.K.), Cambridge, MA; Department of Neurology (T.K.), University of Bonn; German Center for Neurodegenerative Diseases (DZNE) (T.K.), Bonn, Germany; Brain and Mind Centre (W.S.K.), Sydney Medical School, University of Sydney, Australia; Department of Neurology (P.L.), Henry Ford Hospital; Department of Neurology (P.L.), Wayne State University School of Medicine, Detroit, MI; Division of Neurosciences (E.M.), National Institute on Aging/NIH, Bethesda, MD; Centre de Référence Maladie Rare AMS (W.M.), Hôpital Pellegrin, CHU de Bordeaux; Université de Bordeaux (W.M.), Institut des Maladies Neurodégénératives, UMR 5293, Bordeaux; Paris Saclay Institute of Neuroscience (R.M.), CNRS, Gif-sur-Yvette, France; Genentech Inc. (S.O.), South San Francisco; Cedars-Sinai Medical Center (S.P.), Los Angeles, CA; Department of Neurology (D.R.), Vanderbilt University Medical Center, Nashville, TN; MSA NJ (C.R.), Howell, NJ; Prothena Biosciences (D.S.), South San Francisco, CA; Division of Neurology (M.S.), Department of Medicine, The Ottawa Hospital, Canada; Department of Neurology (J.D.S.), Massachusetts General Hospital, Harvard Medical School, Boston, MA; Cure MSA Foundation (L.S.), Fremont, CA; Avid Radiopharmaceuticals (A.S.), Philadelphia, PA; Department of Neurological Sciences (G.T.S.), Rush University Medical Center, Chicago, IL; Department of Neurology (S.T.), Graduate School of Medicine, Tokyo University, Tokyo, Japan; and University of Washington (J.Z.), Seattle. W.R.G. is currently affiliated with Janssen Research and Development, Titusville, NJ.

PMID: 29237794
PMCID: PMC5772155
DOI: 10.1212/WNL.0000000000004798

Abstract

Multiple system atrophy (MSA) is a rare neurodegenerative disorder with substantial knowledge gaps despite recent gains in basic and clinical research. In order to make further advances, concerted international collaboration is vital. In 2014, an international meeting involving leaders in the field and MSA advocacy groups was convened in Las Vegas, Nevada, to identify critical research areas where consensus and progress was needed to improve understanding, diagnosis, and treatment of the disease. Eight topic areas were defined: pathogenesis, preclinical modeling, target identification, endophenotyping, clinical measures, imaging biomarkers, nonimaging biomarkers, treatments/trial designs, and patient advocacy. For each topic area, an expert served as a working group chair and each working group developed priority-ranked research recommendations with associated timelines and pathways to reach the intended goals. In this report, each groups' recommendations are provided.

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Figures

**Figure 1. Preclinical working groups and recommendations (priority-ranked)**
α-syn = α-synuclein; iPSC = induced pluripotent stem cell; MSA = multiple system atrophy.

**Figure 2. Clinical working groups and recommendations (priority-ranked)**
ANS = autonomic nervous system; MSA = multiple system atrophy; SYN = synuclein; UMSARS = Unified MSA Rating Scale.

See this image and copyright information in PMC

References

1. Fanciulli A, Wenning GK. Multiple-system atrophy. N Engl J Med 2015;372:249–263. - PubMed
1. Linder J, Stenlund H, Forsgren L. Incidence of Parkinson's disease and parkinsonism in northern Sweden: a population-based study. Mov Disord 2010;25:341–348. - PubMed
1. Winter Y, Bezdolnyy Y, Katunina E, et al. Incidence of Parkinson's disease and atypical parkinsonism: Russian population-based study. Mov Disord 2010;25:349–356. - PubMed
1. Bjornsdottir A, Gudmundsson G, Blondal H, Olafsson E. Incidence and prevalence of multiple system atrophy: a nationwide study in Iceland. J Neurol Neurosurg Psychiatry 2013;84:136–140. - PubMed
1. Bower JH, Maraganore DM, McDonnell SK, Rocca WA. Incidence of progressive supranuclear palsy and multiple system atrophy in Olmsted County, Minnesota, 1976 to 1990. Neurology 1997;49:1284–1288. - PubMed

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Recommendations of the Global Multiple System Atrophy Research Roadmap Meeting

Affiliations

Recommendations of the Global Multiple System Atrophy Research Roadmap Meeting

Authors

Affiliations

Abstract

Figures

References

Publication types

MeSH terms

Grants and funding

LinkOut - more resources

Full Text Sources

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