DEVELOPMENT OF CARBOXYFULLERENE DRUGS TO TREAT ALS

描述：肌萎缩侧索硬化症（ALS），通常称为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， 皮层、脑干和脊髓中的神经元退化。 不幸的是，目前没有足够的治疗ALS， 它是一致致命的，通常在三到五年内，从 出现症状。最近，一种有效的新型抗氧化剂和免费的 基于羧基化C60富勒烯的自由基清除剂， 不仅能显著延缓 症状，但也有能力增加预期寿命， 家族性ALS的转基因小鼠模型。 虽然它显示出作为ALS药物的巨大前景， 目前，该化合物过于昂贵，无法在 足够的量来治疗ALS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， 每年需要100公斤。因此，本项目的目标 是开发一种新的工艺， 目前正在评估的单一羧基富勒烯异构体 人症第一阶段的预期结果是生产充足 提供临床试验。 拟定商业应用： 羧基富勒烯是非常有效的抗氧化剂， 一系列的用途，如神经保护剂和治疗ALS。他们 也可以证明有效治疗阿尔茨海默病和其他疾病， 抗氧化剂目前被认为是 灵验。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， 研究人员对它的安全性和效力进行测试， 抗氧化剂假设进一步的测试显示碳富勒烯 化合物既安全又有效，最终的商业目标是 这个项目是大规模生产这种化合物， 用于ALS和其他抗氧化剂应用。仅ALS市场 这种化合物在美国由大约30，000名患者组成， 欧洲的类似数字。

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.