The French pharmaceutical company Trophos announced in early March 2014 positive results from a two year-long Phase II clinical trial of the drug Olesoxime in SMA patients.
Specifically developed as a potential treatment for SMA, Olesoxime has been shown to protect nerve cells from damage in cell culture, while improving neuronal growth and function, indicating that it may be beneficial to patients with SMA. The Phase II double-blind trial of Olesoxime included 165 Type II and non-ambulatory Type III SMA patients aged 3-25, and was performed across 22 centres in seven different European countries. The promising results of the trial are expected to lead to further testing of Olesoxime as a potential treatment for SMA, something that Trophos has reported it is currently pursuing.
The successful planning and recruitment for this clinical trial was largely facilitated through the TREAT-NMD SMA patient registries.