DEVELOPMENT OF CARBOXYFULLERENE DRUGS TO TREAT ALS

描述：肌萎缩侧索硬化症(ALS)，俗称LOU Gerhig病是一种神经退行性疾病， 三万美国人。它的受害者会逐渐瘫痪为马达 大脑皮层、脑干和脊髓中的神经元退化。 不幸的是，目前还没有足够的治疗ALS的方法，而且 它通常是致命的，通常在三到五年内 出现症状。最近，一种有效的新型抗氧化剂和游离 基于羧化C60富勒烯的自由基清除剂 不仅证明了显著延迟发病的能力 症状，也有能力延长预期寿命在一个 家族性ALS转基因小鼠模型的建立。 尽管它显示出作为肌萎缩侧索硬化症药物的巨大前景，但羧基富勒烯 目前化合物太贵，不能在 足够治疗肌萎缩侧索硬化症患者。如果羧基富勒烯 被证明是一种安全有效的治疗ALS的方法，然后数百人 每年需要一公斤的重量。因此，这个项目的目标是 是开发一种新的工艺来经济地生产千克量 目前正在评估的单一羧基富勒烯异构体的 肌萎缩侧索硬化。第一阶段的预期结果是足够的产量 提供临床试验的能力。 建议的商业应用： 羧基富勒烯是一种非常有效的抗氧化剂，可能具有广泛的 一系列用途，如神经保护剂和作为ALS的治疗。他们 也可能被证明对治疗阿尔茨海默病和其他疾病有效 抗氧化剂目前被假定为 很有效。预计到第一阶段结束时，该项目 可以通过销售羧基富勒烯抗氧化剂来商业化 研究人员对其安全性和有效性进行了测试 抗氧化剂。假设进一步的测试表明羧基富勒烯 既安全又有效的化合物，其最终商业目标是 这个项目是为了大规模生产这种化合物作为一种处理 适用于肌萎缩侧索硬化症和其他抗氧化剂应用。仅肌萎缩侧索硬化市场就 该化合物由美国约30,000名患者组成， 在欧洲也有类似的数字。

DESCRIPTION: Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gerhig's disease, is a neurodegenerative disorder that afflicts about 30,000 Americans. Its victims become progressively paralyzed as motor neurons in the cortex, brainstem, and spinal cord degenerate. Unfortunately, there is currently no adequate treatment for ALS, and it is uniformly fatal, typically within three to five years from the onset of symptoms. Recently, a potent new type of antioxidant and free radical scavenger based upon a carboxylated C60 fullerene has demonstrated not only the ability to significantly delay the onset of symptoms but, also the ability to increase the life expectancy in a transgenic mouse model of familial ALS. Although it shows great promise as an ALS drug, the carboxyfullerene compound is currently too expensive and cannot be produced in sufficient quantities to treat ALS patients. If the carboxyfullerene is shown to be a safe and effective treatment for ALS, then hundreds of kilograms per year would be needed. Thus, the goal of this project is to develop a new process to economically produce kilogram quantities of the single carboxyfullerene isomer currently being evaluated for ALS. The anticipated results of Phase I is sufficient production capacity to supply clinical trials. PROPOSED COMMERCIAL APPLICATION: Carboxyfullerenes are very effective antioxidants that may have a wide range of uses such as neuroprotectants and as a treatment for ALS. They may also prove effective in treating Alzheimer's disease and in other applications where antioxidants are currently postulated to be efficacious. It is anticipated that by the end of Phase I, this project could be commercialized by the sale of carboxyfullerene antioxidants to researchers conducting tests of its safety and potency as an antioxidant. Assuming that further testing shows the carboxyfullerene compound to be both safe and effective, the ultimate commercial goal of this project is to produce this compound on a large scale as a treatment for ALS and other antioxidant applications. The ALS market alone for this compound consists of about 30,000 patients in the United States and similar numbers in Europe.