Ari Csilla, Poff Angela M, Held Heather E, Landon Carol S, Goldhagen Craig R, Mavromates Nicholas, D'Agostino Dominic P
Department of Molecular Pharmacology and Physiology, Hyperbaric Biomedical Research Laboratory, Morsani College of Medicine, University of South Florida, Tampa, Florida, United States of America.
PLoS One. 2014 Jul 25;9(7):e103526. doi: 10.1371/journal.pone.0103526. eCollection 2014.
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease, is a neurodegenerative disorder of motor neurons causing progressive muscle weakness, paralysis, and eventual death from respiratory failure. There is currently no cure or effective treatment for ALS. Besides motor neuron degeneration, ALS is associated with impaired energy metabolism, which is pathophysiologically linked to mitochondrial dysfunction and glutamate excitotoxicity. The Deanna Protocol (DP) is a metabolic therapy that has been reported to alleviate symptoms in patients with ALS. In this study we hypothesized that alternative fuels in the form of TCA cycle intermediates, specifically arginine-alpha-ketoglutarate (AAKG), the main ingredient of the DP, and the ketogenic diet (KD), would increase motor function and survival in a mouse model of ALS (SOD1-G93A). ALS mice were fed standard rodent diet (SD), KD, or either diets containing a metabolic therapy of the primary ingredients of the DP consisting of AAKG, gamma-aminobutyric acid, Coenzyme Q10, and medium chain triglyceride high in caprylic triglyceride. Assessment of ALS-like pathology was performed using a pre-defined criteria for neurological score, accelerated rotarod test, paw grip endurance test, and grip strength test. Blood glucose, blood beta-hydroxybutyrate, and body weight were also monitored. SD+DP-fed mice exhibited improved neurological score from age 116 to 136 days compared to control mice. KD-fed mice exhibited better motor performance on all motor function tests at 15 and 16 weeks of age compared to controls. SD+DP and KD+DP therapies significantly extended survival time of SOD1-G93A mice by 7.5% (p = 0.001) and 4.2% (p = 0.006), respectively. Sixty-three percent of mice in the KD+DP and 72.7% of the SD+DP group lived past 125 days, while only 9% of the control animals survived past that point. Targeting energy metabolism with metabolic therapy produces a therapeutic effect in ALS mice which may prolong survival and quality of life in ALS patients.
肌萎缩侧索硬化症(ALS),也被称为卢伽雷氏病,是一种运动神经元的神经退行性疾病,会导致进行性肌肉无力、瘫痪,并最终因呼吸衰竭而死亡。目前尚无治愈ALS的方法或有效的治疗手段。除了运动神经元退化外,ALS还与能量代谢受损有关,这在病理生理上与线粒体功能障碍和谷氨酸兴奋性毒性相关。迪安娜疗法(DP)是一种代谢疗法,据报道可缓解ALS患者的症状。在本研究中,我们假设三羧酸循环中间产物形式的替代燃料,特别是DP的主要成分精氨酸-α-酮戊二酸(AAKG)以及生酮饮食(KD),会提高ALS小鼠模型(SOD1-G93A)的运动功能并延长其生存期。给ALS小鼠喂食标准啮齿动物饮食(SD)、KD,或含有由AAKG、γ-氨基丁酸、辅酶Q10和富含辛酸甘油三酯的中链甘油三酯组成的DP主要成分的代谢疗法的饮食。使用预先定义的神经学评分标准、加速转棒试验、爪握耐力试验和握力试验对ALS样病理进行评估。还监测了血糖、血液β-羟基丁酸酯和体重。与对照小鼠相比,喂食SD+DP的小鼠在116至136日龄时神经学评分有所改善。与对照组相比,喂食KD的小鼠在15和16周龄时在所有运动功能测试中表现出更好的运动性能。SD+DP和KD+DP疗法分别使SOD1-G93A小鼠的存活时间显著延长了7.5%(p = 0.001)和4.2%(p = 0.006)。KD+DP组63%的小鼠和SD+DP组72.7%的小鼠存活超过125天,而对照组只有9%的动物存活超过该时间点。用代谢疗法靶向能量代谢在ALS小鼠中产生了治疗效果,这可能会延长ALS患者的生存期和提高其生活质量。