Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis, or Lou Gehrig's disease, is the most devastating neurodegenerative disease, affecting almost as many patients per year as multiple sclerosis. Patients become progressively paralyzed due to degeneration of the upper and lower motor neurons in the brain and spinal cord. They usually die within two to five years, unable to breathe or swallow. Each year University of Miami physicians care for more than 350 patients with ALS from the United States and other parts of the world. The School of Medicine is also the major center for pharmaceutical drug trials seeking new and effective treatments for the disease.

ALS is caused by a cascade of events, starting with genes that predispose the patient to develop the disease, triggered by some personal or environmental factor(s) that initiate degeneration of the motor neurons, and thereby induce spreading neuronal death. The processes leading to motor neuron degeneration include excess glutamate, oxidative and free radical damage, mitochondrial dysfunction, protein aggregation, and the activation of the cell death pathways. The Department of Neurology has basic research programs concentrating on genetics, mitochondrial abnormalities and protein aggregation in ALS. Funding received from the ALS Research Foundation, the ALS Recovery Foundation, the Muscular Dystrophy Association, the National Institutes of Health and other organizations support this research. Philanthropic gifts allow University scientists to concentrate on promising avenues of research.

Priority needs in the ALS Clinical and Research Program are:

The endowment of a professorship for ALS	$2 million
The endowment of a 5000 square foot research laboratory for ALS in the new science building of the medical school	$2 million
Research equipment for the new ALS Research Laboratory	$1 million
The endowment of research and clinical programs in ALS	$3 million
The endowment of a visiting scientist program, two scientists per year with research support	$1 million

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This is an outline of some of the basic and clinical research that is being undertaken in the University of Miami ALS Clinical and Research Program designed to find the cause and cure of ALS.