Rare Brain Disorders Program, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
Sci Transl Med. 2019 Feb 20;11(480). doi: 10.1126/scitranslmed.aan0457.
Glucose is the ultimate substrate for most brain activities that use carbon, including synthesis of the neurotransmitters glutamate and γ-aminobutyric acid via mitochondrial tricarboxylic acid (TCA) cycle. Brain metabolism and neuronal excitability are thus interdependent. However, the principles that govern their relationship are not always intuitive because heritable defects of brain glucose metabolism are associated with the paradoxical coexistence, in the same individual, of episodic neuronal hyperexcitation (seizures) with reduced basal cerebral electrical activity. One such prototypic disorder is pyruvate dehydrogenase (PDH) deficiency (PDHD). PDH is central to metabolism because it steers most of the glucose-derived flux into the TCA cycle. To better understand the pathophysiology of PDHD, we generated mice with brain-specific reduced PDH activity that paralleled salient human disease features, including cerebral hypotrophy, decreased amplitude electroencephalogram (EEG), and epilepsy. The mice exhibited reductions in cerebral TCA cycle flux, glutamate content, spontaneous, and electrically evoked in vivo cortical field potentials and gamma EEG oscillation amplitude. Episodic decreases in gamma oscillations preceded most epileptiform discharges, facilitating their prediction. Fast-spiking neuron excitability was decreased in brain slices, contributing to in vivo action potential burst prolongation after whisker pad stimulation. These features were partially reversed after systemic administration of acetate, which augmented cerebral TCA cycle flux, glutamate-dependent synaptic transmission, inhibition and gamma oscillations, and reduced epileptiform discharge duration. Thus, our results suggest that dysfunctional excitability in PDHD is consequent to reduced oxidative flux, which leads to decreased neuronal activation and impaired inhibition, and can be mitigated by an alternative metabolic substrate.
葡萄糖是大多数使用碳的大脑活动的最终底物,包括通过线粒体三羧酸 (TCA) 循环合成神经递质谷氨酸和γ-氨基丁酸。因此,大脑代谢和神经元兴奋性是相互依存的。然而,支配它们关系的原则并不总是直观的,因为大脑葡萄糖代谢的遗传性缺陷与在同一个体中阵发性神经元过度兴奋(癫痫发作)与基础脑电活动减少共存的悖论有关。丙酮酸脱氢酶 (PDH) 缺乏症 (PDHD) 就是这样一种典型的疾病。PDH 是代谢的核心,因为它引导大部分葡萄糖衍生的通量进入 TCA 循环。为了更好地理解 PDHD 的病理生理学,我们生成了具有大脑特异性降低 PDH 活性的小鼠,这些活性与人类疾病的重要特征相似,包括脑萎缩、脑电图 (EEG) 幅度降低和癫痫。这些小鼠表现出大脑 TCA 循环通量、谷氨酸含量、自发性和电诱发的皮质场电位以及γ EEG 振荡幅度降低。γ 振荡的阵发性降低先于大多数癫痫样放电,便于预测。在脑切片中,快速发射神经元的兴奋性降低,导致在胡须垫刺激后体内动作电位爆发延长。全身性给予乙酸盐后,这些特征部分逆转,乙酸盐增加了大脑 TCA 循环通量、谷氨酸依赖性突触传递、抑制和γ 振荡,并减少了癫痫样放电的持续时间。因此,我们的结果表明,PDHD 中的功能失调性兴奋性是由于氧化通量降低引起的,这导致神经元激活和抑制受损,并可以通过替代代谢底物得到缓解。