"Our study is important because it opens up a promising new avenue for research into a devastating genetic disease," said Dr. Kothary. "Previous research has focused mainly on replacing the defective SMN gene, or replacing the nerve cells that are lost, but our study shows that targeting the biological problems in nerve cells may also be a valuable approach. I also want to emphasize that while these results are quite promising, this is just one study, using an experimental model of SMA, so we will need to do a lot more research to determine if this drug or a similar one might be a good candidate for testing in humans. Even if it is, I believe that SMA is a disease that will be best addressed by using multiple strategies together, including exercise, nutrition and possibly drugs, cells and gene therapies."

"This discovery by Dr. Kothary and his team is very exciting, although as he fully acknowledges, it must be replicated by other researchers, and replicated using FDA-approved drugs related to the one they used, at which point clinical trials in humans affected by SMA could be considered," said Dr. Rod McInnes, Scientific Director of the Institute of Genetics at the Canadian Institutes of Health Research. "At a minimum, however, this excellent research demonstrates that novel drugs, or novel uses of approved drugs, have the potential to alleviate devastating genetic disorders, even ones affecting the nervous system."

SMA affects approximately one in 10,000 births and more than 25,000 people in Canada and the U.S. are currently living with this disease. Severe forms of SMA will cause paralysis and death within the first few years of life, while milder forms can allow survival into adulthood with less serious disabilities.