Lou Gehrig's Disease

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Amyotrophic Lateral Sclerosis (ALS), more commonly known as Lou Gehrig’s disease, is an adult onset neurodegenerative disease. It is characterized by the death of motor neurons in the motor cortex, brainstem, and spinal cord. 90% of all cases of ALS are sporadic with no familial history, while 10% of cases of ALS have familial history. 20% of cases of familial ALS cases are linked to mutations in the SOD1 gene. Currently, the pathogenesis of the disease is unknown. However, multiple studies show that there are several mechanisms contribute to the progression of the disease. These include mitochondrial dysfunction, glutamate excitotoxicity, oxidative stress, axonal dysfunction, reactive astrocytosis, protein aggregation, and mutant SOD1 expression. …show more content…

Riluzole or rilutek, 2-amino-6-trifluromethoxy benzothiazole, is the only medication available to treat ALS. However, it cannot cure the disease, it can only slow down the progression. Riluzole is only moderately affective in slowing the progression because it only prolongs a patient’s life by three months. Early research suggested that Riluzole acted as a inhibitor of glutamate release, however more recent studies suggest that Riluzole has several functions including a potent neuroprotective agent that modulates GABAergic systems as well as an antagonist of protein kinase c and neuronal nitric oxide. It has also been found that Riluzole acts as a Ca2+, Na2+ channel blocker. It also inhibits pertussis toxin/cholera toxin sensitive G-proteins. The main metabolic pathway of Riluzole in humans is the hydroxylation of the primary amine group producing N-hydroxyriluzole. In humans there are two different types of hepatic enzymes engaged in the biotransformation of Riluzole in the liver. These two types of hepatic enzymes are monooxygenases and UDP glucuronosyltransferases. Riluzole is predominately metabolized by CYP1A2 …show more content…

At the Washington School of Medicine, scientists performed an experiment using glial cell line derived neurotrophic factor protein, GDNF, for a single growth-factor therapy. GDNF is a protein that is important in ensuring neuron survival and development in the human body. They performed multiple experiments that were designed to answer whether or not target-derived GDNF, skeletal muscle (myo-GDNF/G93A-SOD1) and centrally derived GDNF, astrocytes in the CNS, could be effective treatments for amyotrophic lateral

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