CLINICAL TRIAL: CLINICAL TRIAL OF HIGH DOSE COQ10 IN ALS

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 肌萎缩侧索硬化症（ALS）是一种神经系统的毁灭性疾病。 它会导致控制肌肉体积和运动的神经细胞逐渐丧失。 患者的所有肌肉都变得越来越虚弱，包括呼吸、吞咽和说话所需的肌肉。 ALS通常在发病后3至5年内导致死亡。 研究结果表明，线粒体，细胞的动力，在ALS中受到影响。其他数据表明，“自由基”，非常活性氧，有助于神经细胞损伤。 辅酶Q10（CoQ 10）是一种天然营养素，有助于线粒体功能和清除自由基。 它延长了ALS小鼠模型的存活时间，并帮助了帕金森病患者，帕金森病是另一种由神经细胞丢失引起的疾病。 在两项小型研究中，我们给ALS患者服用CoQ 10，发现它被身体吸收良好，安全且耐受性良好。 因此，辅酶Q10是一种很有前途的治疗ALS的药物。 我们建议对ALS患者进行CoQ 10临床试验。 审判将分两个阶段进行。 在第一阶段，我们将确定两种剂量中最好的一种。 我们根据小型研究的结果选择了两种剂量。 在第二阶段，我们将测试辅酶Q10是否比安慰剂更有效，安慰剂是一种不含任何活性成分的糖丸。 每例患者的治疗期为9个月。 所有参与者将被允许服用利鲁唑，这是目前唯一批准的ALS治疗方法。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Amyotrophic lateral sclerosis (ALS) is a devastating disease of the nervous system. It leads to progressive loss of those nerve cells that control muscle bulk and movement. Patients develop increasing weakness in all muscles, including those needed for breathing, swallowing and speaking. ALS typically leads to death within 3 to 5 years of onset. Research results implicate that mitochondria, the powerhouse of the cell, are affected in ALS. Other data suggests that "free radicals", very reactive oxygen species, contribute to the nerve cell damage. Coenzyme Q10 (CoQ10), a natural nutrient, helps mitochondrial function and scavenges free radicals. It has prolonged survival in a mouse model of ALS and it has helped patients with Parkinson's disease, another disease caused by nerve cell loss. In two small studies we have given CoQ10 to patients with ALS and found that it was well absorbed by the body and also safe and well tolerated. Therefore, CoQ10 is a promising treatment for ALS. We propose a clinical trial with CoQ10 for patients with ALS. The trial will have two stages. In a first stage we will determine the best of 2 doses. We have selected the two doses based on the results of our smaller studies. In a second stage we will test whether CoQ10 is more effective than placebo, a sugar pill without any active ingredient. The treatment period for each patient will be 9 months. All participants will be allowed to take riluzole, the only currently approved treatment for ALS.