A New Approach to SMA Treatment

Scientists from the Ottawa Hospital Research Institute (OHRI) and the University of Ottawa (uOttawa) have discovered that a drug called fasudil can extend the average lifespan of mice with Spinal muscular atrophy (SMA) from 30.5 days to more than 300 days, but unlike the majority of compounds currently being researched for SMA treatment, this one takes a different approach.

Most of the drugs currently in development tend to target the genetic defficiency that causes SMA, Fasudil however targeted muscle cells. The study published in BioMed Central’s open access journal BMC Medicine, by Dr. Rashmi Kothary, his graduate student Melissa Bowerman and others, showed that fasudil-treated SMA mice survived for an average of more than 300 days, compared to just 30.5 days for untreated SMA mice.

However, the average lifespan of fasudil-treated SMA mice was still only about half as long as that of normal mice. Fasudil-treated SMA mice also had larger muscle fibres than the untreated SMA mice, and they behaved more normally with respect to grooming and other regular activities. However, they did not perform any better in strength and balance tests and they still had low numbers of motor neurons, which is typical for SMA.

“Our study is important because it expands a new area of research into SMA, which could lead to the development of new treatments,” said Melissa Bowerman. “Of course, this research is still at the early stages and although we found that fasudil could significantly increase lifespan in a mouse model of SMA, this drug couldn’t correct all the problems in these mice, and it had serious side effects when used at higher doses.”

“A number of groups are working to develop fasudil-like compounds with fewer side effects, and we’re very excited to see how these perform in our models, and hopefully in human SMA clinical trials some day,” said Dr. Kothary “However, we continue to believe that SMA is a disease that will best be addressed using multiple strategies together, including nutrition and possibly drug, cell and gene therapies.”


Complete story on uOttawa website.