• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利:CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验

帕金森病临床试验药物治疗:2024 年更新。

Parkinson's Disease Drug Therapies in the Clinical Trial Pipeline: 2024 Update.

机构信息

Parkinson's Research Advocate, Oxford, UK.

Parkinson's Research Advocate, Sunnyvale, CA, USA.

出版信息

J Parkinsons Dis. 2024;14(5):899-912. doi: 10.3233/JPD-240272.

DOI:10.3233/JPD-240272
PMID:39031388
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11307066/
Abstract

BACKGROUND

For the past five years, our annual reports have been tracking the clinical development of new drug-based therapies for the neurodegenerative condition of Parkinson's disease (PD). These reviews have followed the progress both of "symptomatic treatments" (ST - improves/reduces symptoms of the condition) and "disease-modifying treatments" (DMT - attempts to delay/slow progression by addressing the underlying biology of PD). Efforts have also been made to further categorize these experimental treatments based on their mechanisms of action and class of drug.

METHODS

A dataset of clinical trials for drug therapies in PD using trial data downloaded from the ClinicalTrials.gov online registry was generated. A breakdown analysis of all the studies that were active as of January 31st, 2024, was conducted. This analysis involved categorizing the trials based on both the mechanism of action (MOA) and the drug target.

RESULTS

There were 136 active Phase 1-3 trials evaluating drug therapies for PD registered on ClinicalTrials.gov, as of January 31, 2024. Of these trials, 76 (56%) were classified as ST trials and 60 (44%) were designated DMT. More than half (58%) of the trials were in Phase 2 testing stage, followed by Phase 1 (30%) and Phase 3 (12%). 35 of the trials were registered since our last report, with the remaining 101 trials appearing in at least one earlier report.

CONCLUSIONS

The drug development pipeline for PD remains in a robust state with a wide variety of approaches being developed and evaluated in Phase 1 and 2. Yet again, however, only a limited number of DMTs are transitioning to Phase 3.

摘要

背景

在过去的五年中,我们的年度报告一直在跟踪针对帕金森病(PD)神经退行性疾病的新型药物疗法的临床进展。这些综述既关注了“症状治疗”(ST-改善/减轻疾病症状),也关注了“疾病修正治疗”(DMT-通过针对 PD 的潜在生物学来尝试延缓/减缓进展)。我们还努力根据作用机制和药物类别进一步对这些实验性治疗进行分类。

方法

使用从 ClinicalTrials.gov 在线注册处下载的临床试验数据,生成了一个用于 PD 药物治疗的临床试验数据集。对截至 2024 年 1 月 31 日所有活跃的研究进行了细分分析。该分析涉及根据作用机制(MOA)和药物靶点对试验进行分类。

结果

截至 2024 年 1 月 31 日,ClinicalTrials.gov 上注册了 136 项评估 PD 药物治疗的活跃的 1 期至 3 期试验。这些试验中,76 项(56%)被归类为 ST 试验,60 项(44%)被指定为 DMT。超过一半(58%)的试验处于 2 期测试阶段,其次是 1 期(30%)和 3 期(12%)。有 35 项试验是自我们上次报告以来注册的,其余 101 项试验至少出现在之前的一份报告中。

结论

PD 的药物开发管道仍然处于强劲状态,有多种方法正在 1 期和 2 期进行开发和评估。然而,只有有限数量的 DMT 正在进入 3 期。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/68a4/11307066/3c2bc4743309/jpd-14-jpd240272-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/68a4/11307066/d2db2c448c12/jpd-14-jpd240272-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/68a4/11307066/6237180a7ae7/jpd-14-jpd240272-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/68a4/11307066/3c2bc4743309/jpd-14-jpd240272-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/68a4/11307066/d2db2c448c12/jpd-14-jpd240272-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/68a4/11307066/6237180a7ae7/jpd-14-jpd240272-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/68a4/11307066/3c2bc4743309/jpd-14-jpd240272-g003.jpg

相似文献

1
Parkinson's Disease Drug Therapies in the Clinical Trial Pipeline: 2024 Update.帕金森病临床试验药物治疗:2024 年更新。
J Parkinsons Dis. 2024;14(5):899-912. doi: 10.3233/JPD-240272.
2
Parkinson's Disease Drug Therapies in the Clinical Trial Pipeline: 2023 Update.帕金森病临床试验药物治疗:2023 年更新。
J Parkinsons Dis. 2023;13(4):427-439. doi: 10.3233/JPD-239901.
3
Parkinson's Disease Drug Therapies in the Clinical Trial Pipeline: 2022 Update.帕金森病临床试验药物治疗:2022 年更新。
J Parkinsons Dis. 2022;12(4):1073-1082. doi: 10.3233/JPD-229002.
4
Parkinson's Disease Drug Therapies in the Clinical Trial Pipeline: 2021 Update.帕金森病临床试验药物治疗:2021 年更新。
J Parkinsons Dis. 2021;11(3):891-903. doi: 10.3233/JPD-219006.
5
Parkinson's Disease Drug Therapies in the Clinical Trial Pipeline: 2020.帕金森病临床试验药物治疗:2020 年。
J Parkinsons Dis. 2020;10(3):757-774. doi: 10.3233/JPD-202128.
6
Modeling of Parkinson's Disease Progression and Implications for Detection of Disease Modification in Treatment Trials.帕金森病进展的建模及其对治疗试验中疾病修饰检测的影响。
J Parkinsons Dis. 2024;14(6):1225-1235. doi: 10.3233/JPD-230446.
7
Disease Modification in Parkinson's Disease: Current Approaches, Challenges, and Future Considerations.帕金森病的疾病修饰:当前方法、挑战和未来考虑。
Mov Disord. 2018 May;33(5):660-677. doi: 10.1002/mds.27360. Epub 2018 Apr 11.
8
Twelve Years of Drug Prioritization to Help Accelerate Disease Modification Trials in Parkinson's Disease: The International Linked Clinical Trials Initiative.十二载药物优先化助力帕金森病疾病修饰临床试验提速:国际关联临床试验倡议。
J Parkinsons Dis. 2024;14(4):657-666. doi: 10.3233/JPD-230363.
9
Folic acid supplementation and malaria susceptibility and severity among people taking antifolate antimalarial drugs in endemic areas.在流行地区,服用抗叶酸抗疟药物的人群中,叶酸补充剂与疟疾易感性和严重程度的关系。
Cochrane Database Syst Rev. 2022 Feb 1;2(2022):CD014217. doi: 10.1002/14651858.CD014217.
10
Disease modification in Parkinson's disease.帕金森病的疾病修饰。
Drugs Aging. 2011 Aug 1;28(8):605-15. doi: 10.2165/11591320-000000000-00000.

引用本文的文献

1
Lysine-Targeting Inhibitors of Amyloidogenic Protein Aggregation: A Promise for Neurodegenerative Proteinopathies.靶向赖氨酸的淀粉样蛋白聚集抑制剂:对神经退行性蛋白质病的前景
JACS Au. 2025 Aug 11;5(8):3680-3700. doi: 10.1021/jacsau.5c00269. eCollection 2025 Aug 25.
2
Multifaceted roles of mammalian heat shock factor 1 in the central nervous system.哺乳动物热休克因子1在中枢神经系统中的多方面作用
Cell Stress Chaperones. 2025 Aug 15;30(5):100109. doi: 10.1016/j.cstres.2025.100109.
3
Association of Medication Use and 8-Year Mortality Risk in Patients With Parkinson Disease: Drug-Wide Trial Emulation.

本文引用的文献

1
[Not Available].[无可用内容]
Alzheimers Dement (N Y). 2024 Apr 24;10(2):e12465. doi: 10.1002/trc2.12465. eCollection 2024 Apr-Jun.
2
Trial of Lixisenatide in Early Parkinson's Disease.利西拉肽治疗早期帕金森病的试验。
N Engl J Med. 2024 Apr 4;390(13):1176-1185. doi: 10.1056/NEJMoa2312323.
3
A biological classification of Parkinson's disease: the SynNeurGe research diagnostic criteria.帕金森病的生物学分类:SynNeurGe 研究诊断标准。
帕金森病患者药物使用与8年死亡风险的关联:全药物试验模拟
Neurology. 2025 Aug 12;105(3):e213783. doi: 10.1212/WNL.0000000000213783. Epub 2025 Jul 11.
4
Investigational Gene Therapies for Parkinson's Disease.帕金森病的研究性基因疗法
CNS Drugs. 2025 Jul 10. doi: 10.1007/s40263-025-01203-6.
5
Combining light-induced aggregation and biotin proximity labeling implicates endolysosomal proteins in early α-synuclein oligomerization.结合光诱导聚集和生物素邻近标记表明内溶酶体蛋白参与早期α-突触核蛋白寡聚化。
iScience. 2025 Jun 6;28(7):112823. doi: 10.1016/j.isci.2025.112823. eCollection 2025 Jul 18.
6
Current strategies in the management of dementia with lewy bodies and future directions based on disease pathophysiology.路易体痴呆的当前管理策略及基于疾病病理生理学的未来方向。
Mol Neurodegener. 2025 Jul 1;20(1):78. doi: 10.1186/s13024-025-00856-7.
7
A community-led initiative to de-risk and advance Parkinson's disease therapeutic targets.一项由社区主导的降低帕金森病治疗靶点风险并推动其发展的倡议。
NPJ Parkinsons Dis. 2025 Jun 20;11(1):179. doi: 10.1038/s41531-025-01039-3.
8
Wrist accelerometry and machine learning sensitively capture disease progression in prodromal Parkinson's disease.手腕加速度测量法和机器学习能够灵敏地捕捉前驱期帕金森病的疾病进展。
NPJ Parkinsons Dis. 2025 Jun 17;11(1):171. doi: 10.1038/s41531-025-01034-8.
9
Engineered sequestrins inhibit aggregation of pathogenic alpha-synuclein mutants.工程化的隔离蛋白抑制致病性α-突触核蛋白突变体的聚集。
Front Immunol. 2025 May 16;16:1574755. doi: 10.3389/fimmu.2025.1574755. eCollection 2025.
10
LRRK2-mediated mitochondrial dysfunction in Parkinson's disease.帕金森病中由LRRK2介导的线粒体功能障碍
Biochem J. 2025 May 28;482(11):BCJ20253062. doi: 10.1042/BCJ20253062.
Lancet Neurol. 2024 Feb;23(2):191-204. doi: 10.1016/S1474-4422(23)00404-0.
4
A biological definition of neuronal α-synuclein disease: towards an integrated staging system for research.神经元α-突触核蛋白病的生物学定义:建立研究用综合分期系统。
Lancet Neurol. 2024 Feb;23(2):178-190. doi: 10.1016/S1474-4422(23)00405-2.
5
Multi-ancestry genome-wide association meta-analysis of Parkinson's disease.多族裔帕金森病全基因组关联荟萃分析。
Nat Genet. 2024 Jan;56(1):27-36. doi: 10.1038/s41588-023-01584-8. Epub 2023 Dec 28.
6
Huntington's Disease Drug Development: A Phase 3 Pipeline Analysis.亨廷顿舞蹈症药物研发:一项3期管线分析
Pharmaceuticals (Basel). 2023 Oct 24;16(11):1513. doi: 10.3390/ph16111513.
7
Transforming Parkinson's Care in Africa (TraPCAf): protocol for a multimethodology National Institute for Health and Care Research Global Health Research Group project.将帕金森病护理转化至非洲(TraPCAf):英国国家卫生与保健研究院全球健康研究组的一个多方法学项目方案。
BMC Neurol. 2023 Oct 19;23(1):373. doi: 10.1186/s12883-023-03414-0.
8
Using F-AV-133 VMAT2 PET Imaging to Monitor Progressive Nigrostriatal Degeneration in Parkinson Disease.使用 F-AV-133 VMAT2 PET 成像监测帕金森病中的进行性黑质纹状体变性。
Neurology. 2023 Nov 27;101(22):e2314-e2324. doi: 10.1212/WNL.0000000000207748.
9
DOPA decarboxylase is an emerging biomarker for Parkinsonian disorders including preclinical Lewy body disease.DOPA 脱羧酶是帕金森病的一个新兴生物标志物,包括临床前路易体病。
Nat Aging. 2023 Oct;3(10):1201-1209. doi: 10.1038/s43587-023-00478-y. Epub 2023 Sep 18.
10
Defining the causes of sporadic Parkinson's disease in the global Parkinson's genetics program (GP2).在全球帕金森病遗传学项目(GP2)中确定散发性帕金森病的病因。
NPJ Parkinsons Dis. 2023 Sep 12;9(1):131. doi: 10.1038/s41531-023-00533-w.