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一种新的帕金森病疾病修饰药物试验方法。

A new approach to disease-modifying drug trials in Parkinson's disease.

机构信息

Department of Neurology and Clinical Neurosciences, Cambridge Center for Brain Repair, University of Cambridge, Cambridge, United Kingdom.

出版信息

J Clin Invest. 2013 Jun;123(6):2364-5. doi: 10.1172/JCI69690.

DOI:10.1172/JCI69690
PMID:23728166
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3668828/
Abstract

Translating new findings in the laboratory into therapies for patients is a slow and expensive process. The development of therapies for neurodegenerative diseases is further complicated by the difficulty in determining whether the drug truly retards the slow degenerative process or provides only symptomatic benefit. In this issue, Aviles-Olmos et al. describe a first in Parkinson's disease (PD) patient study using a drug previously approved for diabetes treatment. In addition to suggesting that the drug may indeed be disease modifying in PD, their innovative approach suggests there may be more rapid and inexpensive avenues for testing novel therapies in PD.

摘要

将实验室的新发现转化为患者的治疗方法是一个缓慢而昂贵的过程。神经退行性疾病治疗方法的发展进一步复杂化,因为很难确定药物是否真的延缓了缓慢的退行性过程,还是仅提供了症状上的益处。在本期中,Aviles-Olmos 等人描述了使用先前批准用于糖尿病治疗的药物对帕金森病 (PD) 患者进行的首次研究。除了表明该药物实际上可能对 PD 具有疾病修饰作用外,他们的创新方法还表明,在 PD 中测试新疗法可能有更快、更经济的途径。

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2
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J Parkinsons Dis. 2013 Jan 1;3(3):231-9. doi: 10.3233/JPD-139000.

本文引用的文献

1
Exenatide and the treatment of patients with Parkinson's disease.依西那肽与帕金森病患者的治疗。
J Clin Invest. 2013 Jun;123(6):2730-6. doi: 10.1172/JCI68295.
2
Trophic factor gene therapy for Parkinson's disease.神经营养因子基因治疗帕金森病。
Mov Disord. 2013 Jan;28(1):96-109. doi: 10.1002/mds.25344.
3
Fetal dopaminergic transplantation trials and the future of neural grafting in Parkinson's disease.胎儿多巴胺能移植试验与帕金森病神经移植的未来。
Lancet Neurol. 2013 Jan;12(1):84-91. doi: 10.1016/S1474-4422(12)70295-8.
4
Exendin-4 reverts behavioural and neurochemical dysfunction in a pre-motor rodent model of Parkinson's disease with noradrenergic deficit.Exendin-4 逆转了具有去甲肾上腺素能缺陷的帕金森病前运动型啮齿动物模型中的行为和神经化学功能障碍。
Br J Pharmacol. 2012 Dec;167(7):1467-79. doi: 10.1111/j.1476-5381.2012.02100.x.
5
Sham neurosurgical procedures in clinical trials for neurodegenerative diseases: scientific and ethical considerations.神经退行性疾病临床试验中的假神经外科手术:科学和伦理考虑。
Lancet Neurol. 2012 Jul;11(7):643-50. doi: 10.1016/S1474-4422(12)70064-9.
6
Etiology and pathogenesis of Parkinson's disease.帕金森病的病因和发病机制。
Mov Disord. 2011 May;26(6):1049-55. doi: 10.1002/mds.23732.
7
AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial.AAV2-GAD 基因治疗晚期帕金森病:一项双盲、假手术对照、随机试验。
Lancet Neurol. 2011 Apr;10(4):309-19. doi: 10.1016/S1474-4422(11)70039-4.
8
Role of brain imaging in early parkinsonism.脑成像在早期帕金森病中的作用。
BMJ. 2011 Feb 23;342:d638. doi: 10.1136/bmj.d638.
9
Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial.AAV2-神经生长因子基因治疗帕金森病:一项双盲、随机、对照试验。
Lancet Neurol. 2010 Dec;9(12):1164-1172. doi: 10.1016/S1474-4422(10)70254-4. Epub 2010 Oct 20.
10
Trials of neuroprotective therapies for Parkinson's disease: problems and limitations.帕金森病神经保护疗法的试验:问题与局限性。
Parkinsonism Relat Disord. 2010 Jul;16(6):365-9. doi: 10.1016/j.parkreldis.2010.04.008. Epub 2010 May 14.