UGA researchers have been awarded a $425,598 subcontract to develop a human embryonic stem cell-derived test for screening drugs capable of treating spinal muscular atrophy, the leading genetic killer of children younger than age 2.
The subcontract was awarded through the Spinal Muscular Atrophy Project, a model translation program established by the National Institute of Neurological Disorders and Stroke 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,” says 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.