DEVELOPMENT OF CARBOXYFULLERENE DRUGS TO TREAT ALS

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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.