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04-02-2008, 08:50 AM | #1 | |||
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In Remembrance
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Orphan News :: Treatment & Health
April 2, 2008 Lithium delays progression of amyotrophic lateral sclerosis By Evangelia Kallivretaki, CheckOrphan Good news for Amyotrophic Lateral Sclerosis (ALS) patients. A recent clinical trial showed that lithium, a chemical element used as a mood stabilizer in psychiatric disorders, can significantly delay the progression of the disease. Amyotrophic lateral sclerosis (ALS), also called Lou Gehrig's disease, is a devastating neurodegenerative disease which leads to death within 3-5 years from diagnosis. ALS attacks motor neurons (nerve cells that control muscles) and eventually results in a range of disabilities caused by the weakening of the affected muscles. In 90-95 percent of ALS cases, there is no clear risk factor involved, whereas the other 5-10 percent of the cases are genetically inherited. About 20 percent of the familial cases are caused by a mutation of the gene coding for the enzyme superoxide dismutase 1 (SOD1). There is no cure yet for ALS, and existing treatment aims at relieving the symptoms. That is why the latest research results from the team led by Dr. Francesco Fornai from the University of Pisa in Italy offer a ray of hope. Dr. Fornai’s team showed that lithium administration significantly decreased disease progress. Their research appeared this February in the Proceedings of the National Academy of Science (PNAS). Although the exact mechanism that causes ALS is not completely elucidated, damaged motor neuron mitochondria seem to be central to disease etiology. Mitochondria are small organelles that produce energy for the cell. When mitochondria are damaged they accumulate inside the cell along with other proteins leading to a so-called aggregate formation. Once this happens, microglia, the cells that exert the immune response of the nervous system, become active and mark the affected motor neurons for destruction. Motor neuron death is what eventually causes disease symptoms. Using a mouse model of ALS, Dr. Fornai’s team showed that the effects of lithium are brought about by delayed motor neuron death. Furthermore, his team could show that lithium treatment could delay motor neuron death by rescuing the affected motor neuron mitochondria, decreasing the formation of protein aggregates and reducing the activation of microglia. CheckOrphan learned that in comparison to riluzole, the only FDA approved medication specific for ALS, which reduces damage to motor neurons by decreasing glutamate (an excitatory neurotransmitter) levels, lithium additionally stabilized the quality of life until the end of the two year study. Most importantly, none of the patients died during the study, whereas 30 percent of the patients in the control group did. Lithium delays progression of amyotrophic lateral sclerosis Francesco Fornai, Patrizia Longone, Luisa Cafaro, Olga Kastsiuchenka, Michela Ferrucci, Maria Laura Manca, Gloria Lazzeri, Alida Spalloni, Natascia Bellio, Paola Lenzi, Nicola Modugno, Gabriele Siciliano, Ciro Isidoro, Luigi Murri, Stefano Ruggieri†, and Antonio Paparelli Contact: Francesco Fornai Department of Human Morphology and Applied Biology Department of Neuroscience, Clinical Neurology University of Pisa 56100 Pisa, Italy Source: Checkbiotech More Related Articles Hope among patients with ALS may take a variety of forms CytRx responses to FDA regarding ALS drug, Arimoclomol ALS Aggregates are composed of only one protein New gene for Lou Gehrig's disease identified 2-way cell talk provides clues about neuromuscular disease Orphan drug designation for AX200 for the treatment of Amyotrophic Lateral Sclerosis Avicena Group to present at 10th annual BIO CEO & Investor Conference Targeting astrocytes slows disease progression in ALS http://www.checkbiotech.org/orphan_N...px?infoId=2935
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