DEVELOPMENT OF CARBOXYFULLERENE DRUGS TO TREAT ALS

治疗 ALS 的羧基富勒烯药物的开发

• 批准号: 2715542
• 负责人: JOHN M ALFORD
• 金额: $ 10万
• 依托单位: TDA RESEARCH, INC.
• 依托单位国家: 美国
• 财政年份: 1998
• 资助国家: 美国
• 起止时间: 1998-08-01 至 1999-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/2715542
• 关键词: amyotrophic lateral sclerosis; antioxidants; drug design /synthesis /production; free radical scavengers; method development; neuropharmacologic agent

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.

描述：肌萎缩侧索硬化症 (ALS)，俗称 Lou 葛希格氏病是一种神经退行性疾病，影响 30,000 名美国人。受害者的运动能力逐渐瘫痪 皮质、脑干和脊髓的神经元退化。 不幸的是，目前还没有足够的治疗 ALS 的方法，并且 它通常是致命的，通常在发生后三到五年内 症状出现。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 不仅表现出显着延迟发作的能力 症状，而且还有增加预期寿命的能力 家族性 ALS 转基因小鼠模型。 尽管它作为 ALS 药物显示出巨大的前景，但羧基富勒烯 目前该化合物过于昂贵，无法在国内生产 足够治疗 ALS 患者。如果羧基富勒烯 被证明是一种安全有效的 ALS 治疗方法，然后数百 每年需要公斤。因此，该项目的目标 是开发一种新工艺来经济地生产公斤数量 目前正在评估的单一羧基富勒烯异构体 肌萎缩侧索硬化症。一期预期产量充足 提供临床试验的能力。 拟议的商业应用： 羧基富勒烯是非常有效的抗氧化剂，具有广泛的用途 用途广泛，例如神经保护剂和 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 目前假设抗氧化剂的应用 有效。预计该项目一期结束时 可以通过销售羧基富勒烯抗氧化剂来实现商业化 研究人员对其安全性和功效进行测试 抗氧化剂。假设进一步测试显示羧基富勒烯 化合物既安全又有效，是其最终的商业目标 该项目旨在大规模生产这种化合物作为治疗剂 用于 ALS 和其他抗氧化剂应用。仅 ALS 市场就 该化合物由美国约 30,000 名患者组成 欧洲也有类似的数字。