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Lithium Eases Symptoms of Fatal Neurological Disorder

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Lithium Eases Symptoms of Fatal Neurological Disorder

 
 

Dr. Huda Zoghbi views a test tube with a mouse embryo alongside a research assistant.

Led by Huda Y. Zoghbi, M.D., director of Jan and Dan Duncan Neurological Research Institute at Texas Children's and Howard Hughes Medical Institute Investigator, studies in mice have shown that lithium, widely used to treat mood disorders in humans, can provide relief from the symptoms of a fatal brain disease, spinocerebellar ataxia type 1.

The recent findings, published in May 2007 in the journal Public Library of Science (PLoS) Medicine, are important because they suggest it may be possible to use lithium to alleviate the debilitating deteriorations in motor coordination, learning and memory manifested by this disease.

“The results are very exciting,” explained Zoghbi, also professor of pediatrics, neuroscience, and molecular and human genetics at Baylor College of Medicine. “It's really hard to improve multiple symptoms (in a condition). Lithium seems to improve several in this case, not just one.” Zoghbi co-discovered the gene that causes spinocerebellar ataxia type 1, which is one of a group of nine human neurodegenerative diseases caused by a similar type of genetic defect.

Because lithium is already widely used in humans, the work of Zoghbi and her colleagues could be tested in humans far faster than a new drug. Although lithium will not cure the disease and may not extend the lifespan of patients, its potential use as an agent to treat the symptoms of a devastating nervous system disorder is promising as the research suggests it can ease symptoms for patients who currently have no other therapeutic options.

"I'm cautiously optimistic," said Zoghbi. "Not everything that works in mice works in humans. But this is a safe drug already in use in humans, and we've shown that it improves multiple symptoms in our model."

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