Moresco Monica, Tropeano Concetta Valentina, Romagnoli Martina, Neccia Giulia, Rapone Alessandro, Pizza Fabio, Vandi Stefano, Mignot Emmanuel, Maresca Alessandra, Carelli Valerio, Plazzi Giuseppe
Programma di Neurogenetica, IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna 40139, Italy.
UOC Clinica Neurologica, IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna 40139, Italy.
Brain Commun. 2025 Apr 17;7(2):fcaf125. doi: 10.1093/braincomms/fcaf125. eCollection 2025.
Narcolepsy type 1 (NT1) is a rare neurological disorder characterized by excessive daytime sleepiness and cataplexy, thought to result from an autoimmune process targeting the hypothalamic hypocretin-producing neurons. Aiming to add clues to the latter hypothesis, we investigated circulating cell-free mitochondrial DNA (ccf-mtDNA) levels in cerebrospinal fluid (CSF), a possible biomarker for neurodegeneration, neuroinflammation or immune activation, from 46 NT1 patients with low CSF hypocretin-1, compared with 32 controls. We found significantly increased ccf-mtDNA levels in NT1 patients compared with controls, which negatively correlated with CSF hypocretin-1 concentrations. Additionally, higher ccf-mtDNA levels were observed in patients with elevated number of sleep onset rapid eye movement periods. These observations imply that increased levels of ccf-mtDNA associate with reduced CSF hypocretin-1 concentrations leading to greater alteration in sleep architecture. Furthermore, cytokine profiling in CSF revealed significant changes in interleukins 6 and 18 in NT1 patients, suggesting an active neuroinflammatory process possibly linked to ccf-mtDNA release, thus pointing to a specific inflammatory signature in NT1. These findings hint a potential mitochondrial dysfunction and neuroinflammation in NT1. Further studies are needed to elucidate the underlying mechanisms and how this may reflect on therapy.
1型发作性睡病(NT1)是一种罕见的神经系统疾病,其特征为日间过度嗜睡和猝倒,被认为是由针对下丘脑分泌食欲素的神经元的自身免疫过程所致。为了为后一种假说提供线索,我们研究了46例脑脊液中食欲素-1水平较低的NT1患者脑脊液(CSF)中循环游离线粒体DNA(ccf-mtDNA)的水平,ccf-mtDNA是神经退行性变、神经炎症或免疫激活的一种可能生物标志物,并与32名对照者进行了比较。我们发现,与对照组相比,NT1患者的ccf-mtDNA水平显著升高,且与脑脊液中食欲素-1浓度呈负相关。此外,在睡眠开始时快速眼动期数量增加的患者中观察到更高的ccf-mtDNA水平。这些观察结果表明,ccf-mtDNA水平升高与脑脊液中食欲素-1浓度降低相关,导致睡眠结构发生更大改变。此外,脑脊液中的细胞因子分析显示,NT1患者的白细胞介素6和18有显著变化,提示可能与ccf-mtDNA释放有关的活跃神经炎症过程,从而表明NT1存在特定的炎症特征。这些发现提示NT1中存在潜在的线粒体功能障碍和神经炎症。需要进一步研究以阐明其潜在机制以及这可能如何反映在治疗上。