Nógrádi Bernát, Nógrádi-Halmi Dóra, Erdélyi-Furka Barbara, Kádár Zalán, Csont Tamás, Gáspár Renáta
Department of Neurology, Albert Szent-Györgyi Health Centre, University of Szeged, Szeged, Hungary.
Euan MacDonald Centre for Motor Neuron Disease Research, University of Edinburgh, Edinburgh, UK.
Cell Death Discov. 2024 Jun 19;10(1):291. doi: 10.1038/s41420-024-02055-7.
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder clinically characterized by muscle atrophy and progressive paralysis. Loss of motoneurons and pyramidal cells is thought to be the center piece of the complex and multifaceted ALS pathology, however, the exact mechanisms laying behind motoneuronal cell death in the spinal cord and motor cortex are still unknown. It was originally proposed that apoptosis plays a fundamental role in motoneuronal demise, nonetheless, later it became clear that other forms of regulated cell death, including necroptosis, pyroptosis, ferroptosis, and autophagy-dependent cell death, may also contribute to motoneuron loss. Over the past years, multiple studies aimed to improve our understanding of the contributory role of these mechanisms as well as to offer novel targets for potential therapeutic interventions. The pharmacological inhibition of the ferroptotic pathway and the modulation of the autophagic machinery seem to have particularly promising effects, reducing motoneuron loss and slowing disease progression in transgenic models of ALS. Nevertheless, the potential beneficial effects of necroptosis-targeting interventions were mostly disproven in the latest studies. In this review we aim to summarize the current view on regulated cell death mechanisms that lead to motoneuronal and pyramidal cell degeneration in ALS and showcase their applicability as future drug targets.
肌萎缩侧索硬化症(ALS)是一种致命的神经退行性疾病,临床特征为肌肉萎缩和进行性麻痹。运动神经元和锥体细胞的丧失被认为是复杂且多方面的ALS病理的核心,但脊髓和运动皮层中运动神经元细胞死亡背后的确切机制仍不清楚。最初有人提出凋亡在运动神经元死亡中起基本作用,然而,后来清楚的是,其他形式的程序性细胞死亡,包括坏死性凋亡、炎性小体介导的细胞焦亡、铁死亡和自噬依赖性细胞死亡,也可能导致运动神经元丧失。在过去几年中,多项研究旨在增进我们对这些机制的促成作用的理解,并提供潜在治疗干预的新靶点。铁死亡途径的药理学抑制和自噬机制的调节似乎具有特别有前景的效果,可减少转基因ALS模型中的运动神经元丧失并减缓疾病进展。然而,针对坏死性凋亡的干预措施的潜在有益效果在最新研究中大多未得到证实。在这篇综述中,我们旨在总结目前关于导致ALS中运动神经元和锥体细胞变性的程序性细胞死亡机制的观点,并展示它们作为未来药物靶点的适用性。