Inserm U 1127, CNRS UMR 7225, Sorbonne Universités, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau et de la Moelle épinière, ICM, Paris, France.
AP-HP, Pitié-Salpêtrière University Hospital, Department of Genetics, Paris, France.
J Neurosci Res. 2017 Nov;95(11):2236-2243. doi: 10.1002/jnr.24111. Epub 2017 Jul 8.
Triheptanoin is an odd-chain triglyceride with anaplerotic properties-that is, replenishing the pool of metabolic intermediates in the Krebs cycle. Unlike even-chain fatty acids metabolized to acetyl-CoA only, triheptanoin can indeed provide both acetyl-CoA and propionyl-CoA, two key carbon sources for the Krebs cycle. Triheptanoin was initially used in patients with long-chain fatty acid oxidation disorders. The first demonstration of the possible benefit of triheptanoin for brain energy deficit came from a patient with pyruvate carboxylase deficiency, a severe metabolic disease that affects anaplerosis in the brain. In an open-label study, triheptanoin was then shown to decrease nonepileptic paroxysmal manifestations by 90% in patients with glucose transporter 1 deficiency syndrome, a disease that affects glucose transport into the brain. P magnetic resonance spectroscopy studies also indicated that triheptanoin was able to correct bioenergetics in the brain of patients with Huntington disease, a neurodegenerative disease associated with brain energy deficit. Altogether, these studies indicate that triheptanoin can be a treatment for brain energy deficit related to altered anaplerosis and/or glucose metabolism. © 2017 Wiley Periodicals, Inc.
三庚酸甘油酯是一种奇数链甘油三酯，具有补充克雷布斯循环代谢中间产物的作用，即补充代谢中间产物库。与仅代谢为乙酰辅酶 A 的偶数链脂肪酸不同，三庚酸甘油酯确实可以提供乙酰辅酶 A 和丙酰辅酶 A，这是克雷布斯循环的两个关键碳源。三庚酸甘油酯最初用于长链脂肪酸氧化障碍患者。三庚酸甘油酯可能有益于大脑能量不足的首次证明来自于丙酮酸羧化酶缺乏症患者，该患者患有严重的代谢疾病，影响大脑的氨甲酰磷酸合成。在一项开放标签研究中，三庚酸甘油酯在葡萄糖转运蛋白 1 缺乏综合征患者中使非癫痫性阵发性发作表现减少了 90%，该疾病影响葡萄糖向大脑的转运。质子磁共振波谱研究也表明，三庚酸甘油酯能够纠正亨廷顿病患者大脑的生物能量，亨廷顿病是一种与大脑能量不足相关的神经退行性疾病。总之，这些研究表明，三庚酸甘油酯可能是治疗与氨甲酰磷酸合成和/或葡萄糖代谢改变相关的大脑能量不足的一种方法。© 2017 Wiley Periodicals, Inc.
