Huntington's Disease: Current and Future Therapeutic Prospects

被引：26

作者：

Kieburtz, Karl ^{[1
,2
]}

Reilmann, Ralf ^{[3
]}

Olanow, C. Warren ^{[2
,4
]}

机构：

[1] Univ Rochester, Rochester, NY 14627 USA

[2] Clintrex LLC, Sarasota, FL USA

[3] George Huntington Inst, Munster, Germany

[4] Mt Sinai Sch Med, New York, NY USA

来源：

MOVEMENT DISORDERS | 2018年 / 33卷 / 07期

关键词：

Huntington's Disease; Therapy; PLACEBO-CONTROLLED TRIAL; YAC128 MOUSE MODEL; DOUBLE-BLIND; MUTANT HUNTINGTIN; CEREBROSPINAL-FLUID; PARKINSONS-DISEASE; MINIPIG MODEL; CELL MODELS; INHIBITOR; PREMANIFEST;

D O I：

10.1002/mds.27363

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Huntington's disease is a progressive neurodegenerative disorder for which therapies are woefully inadequate and do not prevent inevitable progression. Currently approved therapies are primarily aimed at treating chorea, but do not address the more clinically meaningful motor, behavioral, and cognitive features of the disease. However, there are a number of promising new therapies that are currently being studied in the laboratory, and in the clinic. This article will review the wide variety of therapies currently being tested, the advances in clinical trials and end points, and the many potentially relevant new targets. (C) 2018 International Parkinson and Movement Disorder Society

引用

页码：1033 / 1041

页数：9

共 92 条

[11] Mutant Huntingtin is Present in Neuronal Grafts in Huntington Disease Patients [J].

Cicchetti, Francesca ;

Lacroix, Steve ;

Cisbani, Giulia ;

Vallieres, Nicolas ;

Saint-Pierre, Martine ;

St-Amour, Isabelle ;

Tolouei, Ranna ;

Skepper, Jeremy N. ;

Hauser, Robert A. ;

Mantovani, Diego ;

Barker, Roger A. ;

Freeman, Thomas B. .

ANNALS OF NEUROLOGY, 2014, 76 (01) :31-42

[12]

Claassen DO, 2017, J CLIN MOV DISORD

[13] The structural correlates of functional deficits in early huntington's disease [J].

Delmaire, Christine ;

Dumas, Eve M. ;

Sharman, Michael A. ;

van den Bogaard, Simon J. A. ;

Valabregue, Romain ;

Jauffret, Celine ;

Justo, Damian ;

Reilmann, Ralf ;

Stout, Julie C. ;

Craufurd, David ;

Tabrizi, Sarah J. ;

Roos, Raymund A. C. ;

Durr, Alexandra ;

Lehericy, Stephane .

HUMAN BRAIN MAPPING, 2013, 34 (09) :2141-2153

[14] The dopaminergic stabilizers pridopidine (ACR16) and (-)-OSU6162 display dopamine D2 receptor antagonism and fast receptor dissociation properties [J].

Dyhring, Tino ;

Nielsen, Elsebet O. ;

Sonesson, Clas ;

Pettersson, Fredrik ;

Karlsson, Jonas ;

Svensson, Peder ;

Christophersen, Palle ;

Waters, Nicholas .

EUROPEAN JOURNAL OF PHARMACOLOGY, 2010, 628 (1-3) :19-26

[15] Design innovations and baseline findings in a long-term Parkinson's trial: The national institute of neurological disorders and stroke exploratory trials in Parkinson's Disease Long-Term Study-1 [J].

Elm, Jordan J. .

MOVEMENT DISORDERS, 2012, 27 (12) :1513-1521

[16] Effect of Deutetrabenazine on Chorea Among Patients With Huntington Disease A Randomized Clinical Trial [J].

Frank, Samuel ;

Testa, Claudia M. ;

Stamler, David ;

Kayson, Elise ;

Davis, Charles ;

Edmondson, Mary C. ;

Kinel, Shari ;

Leavitt, Blair ;

Oakes, David ;

O'Neill, Christine ;

Vaughan, Christina ;

Goldstein, Jody ;

Herzog, Margaret ;

Snively, Victoria ;

Whaley, Jacquelyn ;

Wong, Cynthia ;

Suter, Greg ;

Jankovic, Joseph ;

Jimenez-Shahed, Joohi ;

Hunter, Christine ;

Claassen, Daniel O. ;

Roman, Olivia C. ;

Sung, Victor ;

Smith, Jenna ;

Janicki, Sarah ;

Clouse, Ronda ;

Saint-Hilaire, Marie ;

Hohler, Anna ;

Turpin, Denyse ;

James, Raymond C. ;

Rodriguez, Ramon ;

Rizer, Kyle ;

Anderson, Karen E. ;

Heller, Hope ;

Carlson, Alexis ;

Criswell, Susan ;

Racette, Brad A. ;

Revilla, Fredy J. ;

Nucifora, Frederick, Jr. ;

Margolis, Russell L. ;

Ong, MaryJane ;

Mendis, Tilak ;

Mendis, Neila ;

Singer, Carlos ;

Quesada, Monica ;

Paulsen, Jane S. ;

Brashers-Krug, Thomas ;

Miller, Amanda ;

Kerr, Jane ;

Dubinsky, Richard M. .

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2016, 316 (01) :40-50

[17] Transplanted fetal striatum in Huntington's disease: Phenotypic development and lack of pathology [J].

Freeman, TB ;

Cicchetti, F ;

Hauser, RA ;

Deacon, TW ;

Li, XJ ;

Hersch, SM ;

Nauert, GM ;

Sanberg, PR ;

Kordower, JH ;

Saporta, S ;

Isacson, O .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2000, 97 (25) :13877-13882

[18] Pridopidine for the treatment of motor function in patients with Huntington's disease (MermaiHD): a phase 3, randomised, double-blind, placebo-controlled trial [J].

Garcia de Yebenes, Justo ;

Landwehrmeyer, Bernhard ;

Squitieri, Ferdinando ;

Reilmann, Ralf ;

Rosser, Anne ;

Barker, Roger A. ;

Saft, Carsten ;

Magnet, Markus K. ;

Sword, Alastair ;

Rembratt, Asa ;

Tedroff, Joakim .

LANCET NEUROLOGY, 2011, 10 (12) :1049-1057

[19] Laquinimod rescues striatal, cortical and white matter pathology and results in modest behavioural improvements in the YAC128 model of Huntington disease [J].

Garcia-Miralles, Marta ;

Hong, Xin ;

Tan, Liang Juin ;

Caron, Nicholas S. ;

Huang, Yihui ;

To, Xuan Vinh ;

Lin, Rachel Yanping ;

Franciosi, Sonia ;

Papapetropoulos, Spyros ;

Hayardeny, Liat ;

Hayden, Michael R. ;

Chuang, Kai-Hsiang ;

Pouladi, Mahmoud A. .

SCIENTIFIC REPORTS, 2016, 6

[20] Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice [J].

Garriga-Canut, Mireia ;

Agustin-Pavon, Carmen ;

Herrmann, Frank ;

Sanchez, Aurora ;

Dierssen, Mara ;

Fillat, Cristina ;

Isalan, Mark .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2012, 109 (45) :E3136-E3145

← 1 2 3 4 5 6 7 8 9 10 →