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Cortical Cholinergic Pathway Degeneration Reflects Cognitive Impairment and Dementia in Parkinson's Disease 皮层胆碱能通路退化反映帕金森病的认知障碍和痴呆
IF 8.6 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2026-10-20 DOI: 10.1002/mds.70584
Nicola M. Slater, Daniel J. Myall, Reza Shoorangiz, Wassilios G. Meissner, Ian J. Kirk, Ross J. Keenan, Rebecca M. Lee, Kyla‐Louise Horne, Michael R. MacAskill, Campbell J. Le Heron, Toni L. Pitcher, Tim J. Anderson, Tracy R. Melzer, John C. Dalrymple‐Alford
Background Cognitive impairment is a prevalent and debilitating feature of Parkinson's disease (PD). Increasing evidence from postmortem and in vivo positron emission tomography imaging studies supports cholinergic dysfunction in PD. There is, however, little evidence regarding the influence of the structural integrity of cortical cholinergic pathways that emanate from the nucleus basalis of Meynert (NBM). Objectives To investigate the association between cognition and the structural integrity of medial and lateral cortical cholinergic projection pathways in PD. Methods Neuropsychological assessment, T1 imaging, and high angular resolution diffusion imaging (HARDI) were acquired from 108 participants with PD (41 female, mean age 71.3 years) and 41 control participants (22 female, mean age 70.6 years). Streamlines representing three cortical cholinergic pathways (one medial and two lateral) from the NBM were extracted from anatomically constrained tractography. Structural integrity was compared across controls and cognitive subgroups (PD with normal cognition [PD‐N], with mild cognitive impairment [PD‐MCI], and with dementia [PDD]) and using a continuous global measure of cognition. Results In PD, we found evidence that lower cognitive performance was associated with increased mean diffusivity across all pathways, especially the perisylvian component of the lateral pathway. In those individuals with dementia, increased free water fraction and decreased fractional anisotropy and fiber density and cross‐section was detected across all pathways. Conclusions Degeneration of the cortical cholinergic system is associated with cognitive decline in PD. Future work should determine whether pathway degeneration predicts future cognitive decline and dementia. © 2026 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
认知障碍是帕金森病(PD)的一个普遍和衰弱的特征。越来越多的死后和体内正电子发射断层成像研究证据支持帕金森病的胆碱能功能障碍。然而,关于Meynert基底核(NBM)发出的皮质胆碱能通路结构完整性的影响,几乎没有证据。目的探讨帕金森病患者认知与皮层内外侧胆碱能投射通路结构完整性的关系。方法对108例PD患者(女性41例,平均年龄71.3岁)和41例对照组(女性22例,平均年龄70.6岁)进行神经心理评估、T1成像和高角分辨率扩散成像(HARDI)。从解剖受限的神经束造影中提取了代表NBM的三条皮质胆碱能通路(一条内侧和两条外侧)的流线。结构完整性在对照组和认知亚组(认知正常的PD - N、轻度认知障碍的PD - MCI和痴呆的PD - dd)之间进行比较,并使用连续的整体认知测量。结果:在帕金森病中,我们发现证据表明,较低的认知表现与所有通路的平均扩散率增加有关,特别是外侧通路的外围成分。在痴呆患者中,在所有通路中检测到自由水含量增加,各向异性分数和纤维密度和横截面下降。结论皮质胆碱能系统退行性变与帕金森病患者认知能力下降有关。未来的工作应该确定路径退化是否预测未来的认知能力下降和痴呆。©2026作者。Wiley期刊有限责任公司代表国际帕金森和运动障碍学会出版的《运动障碍》。
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引用次数: 0
Habitual Diet, Gut Microbiome Dysbiosis, and α‐Synucleinopathy Conversion in Isolated Rapid Eye Movement ( REM) Sleep Behavior Disorder 孤立性快速眼动(REM)睡眠行为障碍的习惯性饮食、肠道微生物群失调和α‐突触核蛋白病转化
IF 8.6 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2026-10-17 DOI: 10.1002/mds.70562
Suyi Xie, Yuhua Yang, Shi Tang, Li Zhou, Jing Wang, Joey W.Y. Chan, Brian Or, Ningning Li, Siyi Gong, Zhixuan He, Jianzhang Ni, Yaping Liu, Jihui Zhang, Paul K.S. Chan, Zigui Chen, Sunny Hei Wong, Vincent C.T. Mok, Ngan Yin Chan, Steven W.H. Chau, Yun Kwok Wing, Bei Huang
Background Accumulating evidence indicates that habitual diet, including plant‐based dietary patterns, is associated with neurodegenerative α‐synucleinopathy risk. However, the role of diet and its correlation with the gut microbiome in prodromal α‐synucleinopathy, specifically among patients with isolated rapid eye movement (REM) sleep behavior disorder (iRBD), remains unclear. Objective To evaluate dietary patterns, their interplay with gut microbial dysbiosis, and longitudinal effects on neurodegenerative progression in iRBD patients. Methods We recruited 287 iRBD patients and 208 age‐ and sex‐matched controls. Plant‐based diet indices (PDIs) were derived from food frequency questionnaires. Shotgun metagenomic sequencing was performed in 456 subjects for diet–microbiome analyses. After a median follow‐up of 4.2 years, 45 (15.7%) iRBD patients converted to α‐synucleinopathies. Cox proportional hazards models assessed the associations between diet and neurodegenerative outcomes. Results We found that iRBD patients exhibited lower healthful PDIs (adjusted odds ratio [aOR] 95% confidence interval [95% CI]: 0.48 [0.29–0.80], P = 0.005) but higher consumption of unhealthful plant‐based foods (refined grains, sweets, and sweeteners). Diet–microbiome interaction analysis identified dietary associations for 84.3% of iRBD‐related gut microbes, including co‐occurrence between healthful PDI and Clostridium sp. AF20‐17LB . Longitudinally, four dietary factors and five iRBD‐related microbes were associated with neurodegenerative outcomes; specifically, higher coffee consumption was linked to a lower risk of progression to α‐synucleinopathy (aHR [95% CI]: 0.30 [0.11–0.78], P = 0.01). Furthermore, mediation analyses indicated that the iRBD‐enriched species Ruthenibacterium lactatiformans mediated the association between unhealthful PDI and dementia‐first conversion. Conclusions Habitual diet in iRBD is associated with gut microbiome dysbiosis and predicts neurodegenerative progression. These findings highlight diet–microbiome interactions as modifiable targets for intervention in prodromal α‐synucleinopathy. © 2026 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
越来越多的证据表明,习惯性饮食,包括植物性饮食模式,与神经退行性α -突触核蛋白病的风险相关。然而,饮食及其与肠道微生物组在前驱α‐突触核蛋白病中的作用,特别是在孤立的快速眼动(REM)睡眠行为障碍(iRBD)患者中,仍不清楚。目的评估iRBD患者饮食模式及其与肠道微生物生态失调的相互作用,以及对神经退行性进展的纵向影响。方法:我们招募了287名iRBD患者和208名年龄和性别匹配的对照组。植物性饮食指数(pdi)来源于食物频率调查问卷。对456名受试者进行鸟枪宏基因组测序,以进行饮食微生物组分析。中位随访4.2年后,45例(15.7%)iRBD患者转化为α -突触核蛋白病。Cox比例风险模型评估了饮食和神经退行性结局之间的关系。结果我们发现,iRBD患者的健康指数较低(调整优势比[aOR] 95%置信区间[95% CI]: 0.48 [0.29-0.80], P = 0.005),但不健康的植物性食品(精制谷物、糖果和甜味剂)的摄入量较高。饮食-微生物组相互作用分析发现,饮食与84.3%的iRBD相关肠道微生物有关,包括健康PDI和梭状芽孢杆菌AF20‐17LB之间的共发生。纵向上,四种饮食因素和五种iRBD相关微生物与神经退行性结局相关;具体来说,高咖啡摄入量与进展为α‐突触核蛋白病的风险降低有关(aHR [95% CI]: 0.30 [0.11-0.78], P = 0.01)。此外,中介分析表明,富含iRBD的乳状鲁thenibacterium lactatiformmans介导了不健康PDI和痴呆- first转换之间的关联。结论iRBD患者的习惯性饮食与肠道菌群失调有关,并可预测神经退行性进展。这些发现强调饮食-微生物组相互作用是干预前驱α‐突触核蛋白病的可改变靶点。©2026作者。Wiley期刊有限责任公司代表国际帕金森和运动障碍学会出版的《运动障碍》。
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引用次数: 0
TGF ‐β Inhibition Attenuates Tau Accumulation and Neurodegeneration in Progressive Supranuclear Palsy Patient‐Derived Organoids TGF - β抑制减缓进展性核上性麻痹患者衍生类器官中Tau积聚和神经变性
IF 8.6 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2026-10-17 DOI: 10.1002/mds.70574
Mariagrazia Talarico, Andrea Quattrone, Giorgia Lucia Benedetto, Raffaele Covello, Maurizio Martini, Valeria Zuccalà, Giovanni Cuda, Elvira Immacolata Parrotta, Aldo Quattrone
Background Progressive supranuclear palsy (PSP) is a devastating 4‐repeat tauopathy with no approved disease‐modifying treatments. Recent evidence showed increased transforming growth factor‐β (TGF‐β) levels in PSP patient‐derived ventral midbrain organoids (vMOs) and serum neuronally derived exosomes. Objective This study investigated whether TGF‐β pharmacological inhibition could mitigate PSP pathology in vMOs. Methods Ninety‐six vMOs were generated from induced pluripotent stem cells from two PSP‐Richardson's syndrome patients and two healthy control donors (HCs). From day 60 to day 90, PSP organoids were (1) exposed to TGF‐β receptor I inhibitor (SB431542); (2) exposed to recombinant TGF‐β1, TGF‐β2, and TGF‐β3; or (3) untreated. At day 90, several tau species were assessed by immunoblotting; neurodegeneration was evaluated through TUNEL assay and immunofluorescence (tyrosine hydroxylase [TH], neurofilament light chain [NfL]); astrocytic activation (glial fibrillary acidic protein [GFAP]) marker was evaluated by immunofluorescence, and histopathological hallmarks were evaluated by immunohistochemistry. An HC‐derived organoid subgroup was exposed to the treatment conditions to assess potential toxicity. Results At day 90, untreated PSP‐derived vMOs exhibited increased phosphorylated tau levels (Ser396, Thr181, Ser202/Ser205 [AT8]) compared with HC‐derived vMOs, dopaminergic neuron loss (reduced TH, increased NfL), and astrocytic reactivity (increased GFAP), along with PSP characteristic tau deposition. Tau hyperphosphorylation, neurodegeneration, and astrocytic reactivity were all markedly reduced in PSP‐derived vMOs treated with TGF‐β receptor I inhibitor. Conversely, exposure to TGF‐β exacerbated these features. No toxic effects were observed in HC vMOs exposed to treatment conditions. Conclusions Pharmacological inhibition of TGF‐β signaling prevented key PSP molecular alterations in patient‐derived organoids. These findings support further research and pave the way for future investigation of TGF‐β–targeting strategies in clinical trials in patients with PSP. © 2026 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
进行性核上性麻痹(PSP)是一种破坏性的4次重复的tau病变,目前尚无批准的疾病修饰治疗方法。最近的证据表明,PSP患者源性腹侧中脑类器官(vMOs)和血清神经源性外泌体中转化生长因子β (TGF - β)水平升高。目的探讨TGF - β药物抑制是否能减轻vMOs的PSP病理。方法用2例PSP - Richardson综合征患者和2例健康对照供体(hc)的诱导多能干细胞生成96个vmo。从第60天到第90天,PSP类器官(1)暴露于TGF‐β受体I抑制剂(SB431542);(2)暴露于重组TGF‐β1、TGF‐β2和TGF‐β3;或(3)未经治疗。在第90天,用免疫印迹法评估几种tau蛋白;采用TUNEL法和免疫荧光法(酪氨酸羟化酶[TH]、神经丝轻链[NfL])评价神经变性;免疫荧光法检测星形细胞活化(胶质纤维酸性蛋白[GFAP])标志物,免疫组化法检测组织病理标志。HC衍生类器官亚组暴露于治疗条件下以评估潜在毒性。结果在第90天,与HC来源的vMOs相比,未经治疗的PSP来源的vMOs表现出磷酸化的tau水平(Ser396, Thr181, Ser202/Ser205 [AT8]),多巴胺能神经元损失(TH减少,NfL增加)和星形细胞反应性(GFAP增加),以及PSP特征的tau沉积。TGF - β受体I抑制剂处理的PSP衍生vMOs中,Tau过度磷酸化、神经变性和星形细胞反应性均显著降低。相反,暴露于TGF‐β加剧了这些特征。暴露于处理条件下的HC vMOs未观察到毒性作用。结论TGF - β信号的药理抑制可阻止患者源性类器官中关键的PSP分子改变。这些发现支持了进一步的研究,并为未来在PSP患者的临床试验中研究TGF - β靶向策略铺平了道路。©2026作者。Wiley期刊有限责任公司代表国际帕金森和运动障碍学会出版的《运动障碍》。
{"title":"TGF ‐β Inhibition Attenuates Tau Accumulation and Neurodegeneration in Progressive Supranuclear Palsy Patient‐Derived Organoids","authors":"Mariagrazia Talarico, Andrea Quattrone, Giorgia Lucia Benedetto, Raffaele Covello, Maurizio Martini, Valeria Zuccalà, Giovanni Cuda, Elvira Immacolata Parrotta, Aldo Quattrone","doi":"10.1002/mds.70574","DOIUrl":"https://doi.org/10.1002/mds.70574","url":null,"abstract":"Background Progressive supranuclear palsy (PSP) is a devastating 4‐repeat tauopathy with no approved disease‐modifying treatments. Recent evidence showed increased transforming growth factor‐β (TGF‐β) levels in PSP patient‐derived ventral midbrain organoids (vMOs) and serum neuronally derived exosomes. Objective This study investigated whether TGF‐β pharmacological inhibition could mitigate PSP pathology in vMOs. Methods Ninety‐six vMOs were generated from induced pluripotent stem cells from two PSP‐Richardson's syndrome patients and two healthy control donors (HCs). From day 60 to day 90, PSP organoids were (1) exposed to TGF‐β receptor I inhibitor (SB431542); (2) exposed to recombinant TGF‐β1, TGF‐β2, and TGF‐β3; or (3) untreated. At day 90, several tau species were assessed by immunoblotting; neurodegeneration was evaluated through TUNEL assay and immunofluorescence (tyrosine hydroxylase [TH], neurofilament light chain [NfL]); astrocytic activation (glial fibrillary acidic protein [GFAP]) marker was evaluated by immunofluorescence, and histopathological hallmarks were evaluated by immunohistochemistry. An HC‐derived organoid subgroup was exposed to the treatment conditions to assess potential toxicity. Results At day 90, untreated PSP‐derived vMOs exhibited increased phosphorylated tau levels (Ser396, Thr181, Ser202/Ser205 [AT8]) compared with HC‐derived vMOs, dopaminergic neuron loss (reduced TH, increased NfL), and astrocytic reactivity (increased GFAP), along with PSP characteristic tau deposition. Tau hyperphosphorylation, neurodegeneration, and astrocytic reactivity were all markedly reduced in PSP‐derived vMOs treated with TGF‐β receptor I inhibitor. Conversely, exposure to TGF‐β exacerbated these features. No toxic effects were observed in HC vMOs exposed to treatment conditions. Conclusions Pharmacological inhibition of TGF‐β signaling prevented key PSP molecular alterations in patient‐derived organoids. These findings support further research and pave the way for future investigation of TGF‐β–targeting strategies in clinical trials in patients with PSP. © 2026 The Author(s). <jats:italic>Movement Disorders</jats:italic> published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.","PeriodicalId":213,"journal":{"name":"Movement Disorders","volume":"2 1","pages":""},"PeriodicalIF":8.6,"publicationDate":"2026-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149198939","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Severe Parkinson's Disease Linked to SNCA Locus Triplication Is Associated with Expression Changes in Platelets: A Case Report 重度帕金森病与SNCA基因座三倍复制与血小板表达变化相关:一个病例报告
IF 8.6 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2026-10-15 DOI: 10.1002/mds.70529
Susanna Jiménez‐Román, David Adamuz, Jorge Mena, Abril Tuset, Mar Mallo, Alex Menendez, Daniela Samaniego, Lourdes Ispierto, Dolores Vilas, Pau Pastor, Katrin Beyer
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引用次数: 0
Reply to “Evaluation of Rare MCOLN1 Variants in a Large Cohort of α‐Synucleinopathies” 回复“α‐突触核蛋白病大队列中罕见MCOLN1变异的评估”
IF 8.6 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2026-10-14 DOI: 10.1002/mds.70569
Chenxin Ying, Xinhui Chen, Zhidong Cen, Xinghua Feng, Nan Jin, Jiaxiang Li, Xinchen Wang, Wei Luo
{"title":"Reply to “Evaluation of Rare MCOLN1 Variants in a Large Cohort of α‐Synucleinopathies”","authors":"Chenxin Ying, Xinhui Chen, Zhidong Cen, Xinghua Feng, Nan Jin, Jiaxiang Li, Xinchen Wang, Wei Luo","doi":"10.1002/mds.70569","DOIUrl":"https://doi.org/10.1002/mds.70569","url":null,"abstract":"","PeriodicalId":213,"journal":{"name":"Movement Disorders","volume":"31 1","pages":""},"PeriodicalIF":8.6,"publicationDate":"2026-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149172916","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evaluation of Rare MCOLN1 Variants in a Large Cohort of α‐Synucleinopathies α‐突触核蛋白病大队列中罕见MCOLN1变异的评估
IF 8.6 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2026-10-14 DOI: 10.1002/mds.70568
Yuan Gao, Ruwei Ou, Dejiang Pang, Yi Xiao, Qirui Jiang, Shichan Wang, Jiyong Liu, Yushan Tan, Xiaokai Zhou, Qianqian Wei, Wei Song, Xueping Chen, Chunyu Li, Jing Yang, Yiyuan Cui, Bi Zhao, Junyu Lin, Huifang Shang
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引用次数: 0
Vitamin B6 Deficiency and Status Epilepticus: A Preventable Cause of Death in People with Parkinson's Disease 维生素B6缺乏和癫痫持续状态:帕金森病患者可预防的死亡原因
IF 8.6 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2026-10-14 DOI: 10.1002/mds.70580
Karlo J. Lizarraga, Andres M. Kanner, Anthony E. Lang
{"title":"Vitamin B6 Deficiency and Status Epilepticus: A Preventable Cause of Death in People with Parkinson's Disease","authors":"Karlo J. Lizarraga, Andres M. Kanner, Anthony E. Lang","doi":"10.1002/mds.70580","DOIUrl":"https://doi.org/10.1002/mds.70580","url":null,"abstract":"","PeriodicalId":213,"journal":{"name":"Movement Disorders","volume":"10 1","pages":""},"PeriodicalIF":8.6,"publicationDate":"2026-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149172915","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cerebellar Network Organization in Parkinson's Disease: Functional Connectivity across Motor and Cognitive Circuits 帕金森病的小脑网络组织:运动和认知回路的功能连接
IF 8.6 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2026-10-10 DOI: 10.1002/mds.70564
Chi‐Ying R. Lin,Thamires N.C. Magalhães,Shayla S. Yonce,Ihika Rampalli,Rory Mahabir,Jessica A. Bernard,and the Parkinson's Precision Medicine Initiative
Abstract Background The cerebellum is increasingly recognized as a key contributor to cognitive reserve and network organization in Parkinson's disease (PD). However, the relationship of cerebello‐cortical and cerebello‐basal ganglia connectivity with cognitive status and nigrostriatal integrity remains incompletely understood. Methods Resting‐state functional magnetic resonance imaging (fMRI) data from 172 participants with PD in the Parkinson's Precision Medicine Initiative were analyzed. Cerebello‐basal ganglia and cerebello‐cortical connectivity were investigated using region of interest (ROI)‐to‐ROI and seed‐to‐voxel approaches. Cerebellar connectivity was examined in relation to cognitive status, age, disease duration, motor severity, dopaminergic medication exposure, and contralateral putaminal striatal binding ratio (SBR). Results PD participants demonstrated robust positive cerebello‐basal ganglia connectivity, particularly between lobule VI/vermis VI and the pallidum. Greater age was associated with widespread reductions in cerebello‐basal ganglia coupling. Longer disease duration was associated with stronger connectivity within cerebellar and basal ganglia circuits, and among cognitively unimpaired participants, stronger lobule V connectivity with temporal regions. After adjustment for contralateral putaminal SBR, cognitively unimpaired participants generally demonstrated greater connectivity between left cerebellar regions and frontal networks, whereas cognitively impaired participants showed greater connectivity from posterior cerebellar and vermal regions, including regions implicated in cognitive processing, to temporal, parahippocampal, occipital, parietal, and somatosensory regions. Conclusions Cerebellar functional connectivity in PD shows region‐specific differences across cognitive status rather than uniform network loss with cognitive impairment. These findings support further investigation of cerebellar functional connectivity as a candidate biomarker of cognitive resilience and network adaptation in PD. © 2026 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
小脑在帕金森病(PD)的认知储备和网络组织中扮演着重要的角色。然而,小脑-皮质和小脑-基底神经节的连通性与认知状态和黑质纹状体完整性的关系仍不完全清楚。方法分析172名帕金森病患者的静息状态功能磁共振成像(fMRI)数据。使用感兴趣区域(ROI)到感兴趣区域和种子到体素的方法研究小脑-基底节区和小脑-皮质的连通性。小脑连通性与认知状态、年龄、疾病持续时间、运动严重程度、多巴胺能药物暴露和对侧壳核纹状体结合比(SBR)有关。结果PD参与者表现出强大的小脑-基底神经节阳性连接,特别是在第六小叶/第六蚓和苍白球之间。年龄越大,小脑-基底神经节偶联性普遍降低。较长的疾病持续时间与小脑和基底神经节回路内更强的连通性有关,在认知未受损的参与者中,与颞区更强的小叶V连通性有关。在调整对侧壳层SBR后,认知功能受损的参与者通常表现出左小脑区域和额叶网络之间更大的连通性,而认知功能受损的参与者则表现出从小脑后区和颞区(包括涉及认知加工的区域)到颞、海马旁、枕、顶叶和体感觉区域更大的连通性。PD患者的小脑功能连通性表现出不同认知状态的区域特异性差异,而不是认知障碍患者的统一网络丧失。这些发现支持进一步研究小脑功能连通性作为PD认知弹性和网络适应的候选生物标志物。©2026作者。Wiley期刊有限责任公司代表国际帕金森和运动障碍学会出版的《运动障碍》。
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引用次数: 0
The Gut–Brain Axis in Parkinson's Disease: From Clinical Observation toward Mechanism‐Based Precision Medicine 帕金森病的肠脑轴:从临床观察到基于机制的精准医学
IF 8.6 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2026-10-08 DOI: 10.1002/mds.70566
Filip Scheperjans, Per Borghammer, Ronald F. Pfeiffer, Silke Appel‐Cresswell, Ali Keshavarzian
Gastrointestinal dysfunction is increasingly recognized as a core feature of Parkinson's disease (PD), affecting quality of life, nutrition, medication response, and potentially the earliest stages of pathogenesis. This perspective traces the evolution of the gut from a neglected source of non‐motor symptoms to a central component of biological models of PD. Gastrointestinal manifestations are physiologically heterogeneous and require objective phenotyping to distinguish dysphagia, gastroparesis, slow transit, defecatory dysfunction, and overlapping mechanisms. The body‐first/brain‐first framework further suggests that Lewy pathology may follow distinct anatomical trajectories, with early enteric and autonomic involvement defining a subgroup in which gut‐directed biomarkers and therapies may be especially relevant. Alterations of the gut microbiome are reproducibly associated with PD, although geography, constipation, medication, diet, disease stage, and reverse causation complicate interpretation. Emerging work is shifting from taxonomy toward microbial functions and host pathways, including barrier integrity, immune and enteroendocrine signaling, short‐chain fatty acids, glucagon‐like peptide‐1 (GLP‐1), host genetic susceptibility, α‐synuclein aggregation and propagation, and microbial levodopa metabolism. Trials of probiotics, fecal microbiota transplantation, and dietary interventions remain heterogeneous and have not established disease‐modifying efficacy. Progress will require longitudinal studies in prodromal and subtype‐defined populations, objective gastrointestinal measurements, integrated microbial, host‐genetic and other multi‐omics, mechanistic human and animal models, and clinical trials combining target‐engagement markers with meaningful outcomes. Such an approach may enable precision gut‐brain medicine for appropriately selected patients with PD. © 2026 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
胃肠功能障碍越来越被认为是帕金森病(PD)的核心特征,它影响生活质量、营养、药物反应,并可能影响发病的早期阶段。这一观点追溯了肠道从一个被忽视的非运动症状来源到PD生物学模型的核心组成部分的演变。胃肠道表现在生理上是异质性的,需要客观的表型来区分吞咽困难、胃轻瘫、转运缓慢、排便功能障碍和重叠机制。身体优先/大脑优先的框架进一步表明,Lewy病理可能遵循不同的解剖轨迹,早期肠道和自主神经参与定义了一个亚群,其中肠道导向的生物标志物和治疗可能特别相关。尽管地理位置、便秘、药物、饮食、疾病阶段和反向因果关系使解释复杂化,但肠道微生物组的改变与PD具有可重复性的相关性。新兴的研究正从分类学转向微生物功能和宿主途径,包括屏障完整性、免疫和肠内分泌信号、短链脂肪酸、胰高血糖素样肽- 1 (GLP - 1)、宿主遗传易感性、α -突触核蛋白聚集和繁殖,以及微生物左旋多巴代谢。益生菌、粪便微生物群移植和饮食干预的试验仍然存在异质性,尚未确定疾病改变的功效。进展将需要对前驱和亚型定义人群进行纵向研究,客观胃肠道测量,综合微生物,宿主遗传和其他多组学,机械性人类和动物模型,以及结合靶参与标记物和有意义结果的临床试验。这种方法可以为适当选择的PD患者提供精确的肠脑医学。©2026作者。Wiley期刊有限责任公司代表国际帕金森和运动障碍学会出版的《运动障碍》。
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引用次数: 0
KCNA2 ‐Associated Hereditary Spastic Paraplegia: Clinical Characteristics and Rationale for Targeted Treatment KCNA2相关的遗传性痉挛性截瘫:临床特征和靶向治疗的基本原理
IF 8.6 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2026-10-08 DOI: 10.1002/mds.70555
Christian M. Boßelmann, Martin Vyhnalek, Zuzana Blichova, Ludmila Novotna, Anna Uhrova Meszarosova, Petra Havránková, Elena Kuster, Robert J. Lauerer, Ulrike B.S. Hedrich, Hang Lyu, Mayke Oosterloo, Michael Alber, Ludger Schöls, Karen Müller‐Schlüter, Almut Turid Bischoff, Thomas Klopstock, Georgios Koutsis, Georgia Karadima, Stephanie Efthymiou, Henry Houlden, Ainara Salazar‐Villacorta, Colin A. Ellis, Anna Scheper, Dieter Gläser, Tobias B. Haack, Matias Wagner, Michael Zech, Konrad Platzer, Pasquale Striano, Steffen Syrbe, Rebecca Schüle, Holger Lerche
Background A single recurrent variant, NM_004974.4( KCNA2 ):c.881G>A:p.(Arg294His), causes hereditary spastic paraplegia (HSP) through a unique variant‐specific mechanism. Data from paralogous variants suggest that this may be a druggable mechanism. Objective The objective of this work is to give an overview of the clinical spectrum and course of KCNA2 ‐p.(Arg294His)‐associated HSP (HSP‐ KCNA2 ) and report treatment with eplerenone, a potassium‐sparing diuretic. Methods In this retrospective study, we recruited individuals with the variant of interest through international rare disease networks. Treatment with eplerenone was done as off‐label use, and outcome measures were recorded during routine clinical care. Results We report the clinical characteristics of 23 individuals from 17 families with HSP‐ KCNA2 . We observed a consistent core phenotype of abnormal evoked potentials (100%), cerebellar ataxia (83%), and lower limb spasticity (70%). Childhood‐onset was more commonly associated with epilepsy (odds ratio, 4.16; P = 0.042), and 52% had developmental delay. Progression of motor disability was slow even in childhood onset. We observed full (100%) penetrance and estimated the prevalence to be 0.18 to 0.69 case per million. Treatment with eplerenone was associated with a decrease on the Scale for the Assessment and Rating of Ataxia (Cohen's d = −1.8; −7.9% change from baseline, P = 0.015), but not on the Spastic Paraplegia Rating Scale, whereas stopping treatment was associated with worsening symptoms. At 2‐year follow‐up, four of five (80%) individuals chose to remain on treatment. Conclusions HSP‐ KCNA2 is a syndrome with a recognizable variant‐specific core phenotype. Our exploratory analyses on eplerenone may serve as rationale for future investigations on targeted treatments. © 2026 International Parkinson and Movement Disorder Society.
背景:单个复发型变异,NM_004974.4(KCNA2):c.881G>A:p。(Arg294His)通过一种独特的变异特异性机制导致遗传性痉挛性截瘫(HSP)。来自相似变异的数据表明,这可能是一种可用药的机制。目的本研究的目的是对KCNA2‐p的临床谱和病程进行综述。(Arg294His) -相关HSP (HSP - KCNA2),并报道了用保钾利尿剂eplerenone治疗。方法在这项回顾性研究中,我们通过国际罕见病网络招募具有感兴趣变异的个体。依普利酮治疗作为标签外用药,并在常规临床护理期间记录结果。结果报告了来自17个家族的23例HSP - KCNA2患者的临床特征。我们观察到异常诱发电位(100%)、小脑性共济失调(83%)和下肢痉挛(70%)的核心表型一致。儿童期发病更常与癫痫相关(优势比为4.16;P = 0.042), 52%有发育迟缓。即使在儿童期发病时,运动障碍的进展也是缓慢的。我们观察到完全(100%)外显率,并估计患病率为0.18至0.69例/百万人。依普利酮治疗与共济失调评估和评分量表的下降相关(Cohen’s d = - 1.8;较基线变化- 7.9%,P = 0.015),但与痉挛性截瘫评分量表无关,而停止治疗与症状恶化相关。在2年的随访中,五分之四(80%)的患者选择继续治疗。结论:HSP - KCNA2是一种具有可识别的变异特异性核心表型的综合征。我们对eplerone的探索性分析可能为未来研究靶向治疗提供依据。©2026国际帕金森和运动障碍学会。
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