PDF(Pubmed)
Abstract：
Homogenates of brain tissue from the frontal cortex at autopsy in patients with amyotrophic lateral sclerosis (ALS) showed dramatically reduced levels of the enzyme thiamine pyrophosphatase (TPPase), the enzyme responsible for the conversion of thiamine pyrophosphate (TPP) to thiamine monophosphate (TMP). Additionally, free thiamine (vitamin B1) and TMP levels have been shown to be significantly reduced in the plasma and cerebral spinal fluid (CSF) of patients with ALS. These findings suggest that there is impaired thiamine metabolism in patients with ALS. Impaired thiamine metabolism decreases adenosine triphosphate (ATP) production and is a well-established cause of neurodegeneration. Decreased levels of TPPase, resulting in decreased levels of TMP in the cells of the frontal cortex, might account for the focal neurodegenerative changes observed in motor neurons in ALS. Benfotiamine, a safe, lipid-soluble, highly absorbable thiamine analogue, significantly raises free thiamine, TMP, and TPP levels in the blood. A case in which benfotiamine may have positively impacted the symptoms of a patient with ALS is presented. The use of benfotiamine in patients with ALS appears to be a promising therapeutic option. Considering the severity and the lack of satisfactory treatment options associated with this disease, more research on the effects of benfotiamine on the course of ALS is urgently needed.
摘要：
肌萎缩侧索硬化症（ALS）患者尸检时额叶皮质脑组织的匀浆显示，硫胺素焦磷酸酶（TPPase）的水平显着降低，负责将焦磷酸硫胺素（TPP）转化为单磷酸硫胺素（TMP）的酶。此外,ALS患者血浆和脑脊液(CSF)中游离硫胺素(维生素B1)和TMP水平显著降低.这些发现表明，ALS患者的硫胺素代谢受损。硫胺素代谢受损会降低三磷酸腺苷（ATP）的产生，并且是神经变性的公认原因。TPPase水平降低,导致额叶皮层细胞中TMP水平下降，可能是ALS运动神经元中观察到的局灶性神经退行性变化的原因。苯福硫胺,一个保险箱,脂溶性，高度可吸收的硫胺素类似物，显著增加游离硫胺素，TMP,以及血液中的TPP水平。提出了一种情况，其中苯膦硫胺可能对ALS患者的症状产生了积极影响。在ALS患者中使用苯膦硫胺似乎是一种有希望的治疗选择。考虑到与这种疾病相关的严重程度和缺乏令人满意的治疗选择，迫切需要更多关于苯磺硫胺对ALS病程的影响的研究。
