UGA researchers receive NIH contract to create a stem cell-based drug assay for SMA

Researchers at the University of Georgia have been awarded a $425,598 subcontract to develop a human embryonic stem cellderived test for screening drugs capable of treating spinal muscular atrophy, the number one genetic killer of children under the age of two.

The subcontract was awarded through the Spinal Muscular Atrophy (SMA) Project, a model translation program established by the National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health, to accelerate the process of developing safe and effective treatment of SMA.

The goal of the SMA Project is to identify and complete preclinical research and development of candidate therapeutics for treating SMA by late 2007. The UGA team hopes to have the first assay ready in one year.

"All the talk surrounding stem cell research has focused on cell therapy," said Steven Stice, one of UGA's Georgia Research Alliance Eminent Scholars and the project's principal investigator. "We hope that this will be the first use of human embryonic stem cells in human medicine. Our goal is to have an immediate impact on health issues through better ways of identifying promising drug therapies for diseases like SMA."

Spinal muscular atrophy is a group of inherited and often fatal diseases that destroys the nerves necessary for voluntary muscle movement, such as crawling, walking, head and neck control and even swallowing. According to the NIH, one in every 40 people is a genetic carrier of the disease and one in 6,000 babies is born with the disease. Of those children diagnosed before age two, 50 percent will die before their second birthday.

SMA is caused by a defect in the survival motor neuron gene 1 (SMN1), which produces a protein necessary for the development and function of all of the body's motor neurons. In individuals with SMA, limited amounts of SMN protein are provided by a second SMN gene (SMN2) and allow for the correct functioning of most of t

Contact: Kim Carlyle
University of Georgia

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