November 2016

Duchenne UK is delighted to announce a £72,000 grant to Solid Biosciences to develop statins as a potential treatment option for Duchenne Muscular Dystrophy.

In 2015, Drs Stan Froehner and Nick Whitehead at the University of Washington, published a paper on the potential benefits of statins in DMD. They tested a commonly used statin called Simvastatin. In early pre-clinical models of DMD, short term administration showed reduced skeletal muscle damage and enhanced muscle function.  Long-term administration in the same models showed an improvement of overall muscle health.

“The Simvastatin program is another example of our commitment to leaving no stone unturned in our search to find effective therapies for DMD,” said Gilad Hayeem, President of Solid Biosciences. “The program could not have been possible without the generous grant from Duchenne UK, one of our fearless advocacy partners in the fight to beat this disease.”

Solid and its collaborators at the University of Washington are currently performing several preclinical studies to better characterize the potential risks and benefits of statins and inform the optimal molecule to move into clinical studies for DMD.

Emily Crossley and Alex Johnson from Duchenne UK said:

“We are delighted to be partnering with Solid on this new project, and are grateful to them for their speed and determination to drive this forward. Testing existing medicines to see if they could be effective in treating Duchenne Muscular Dystrophy is an exciting approach known as repurposing, and it’s something we are actively pursuing. The advantage of this approach, compared to the development of an entirely novel compound, is that the development time has the potential to be cut dramatically, because these medicines are already approved as safe for use in humans.”

Mechanism: DMD is characterized by chronic inflammation, oxidative stress and fibrosis. Statins, which are already approved for cardiac indications, have been shown to inhibit these harmful processes.  In early preclinical models of DMD, Simvastatin reduced muscle damage and enhanced muscle function.

Impact on DMD: Simvastatin, along with other statins, may provide skeletal and cardiac muscle function benefits in DMD patients.  Preclinical studies are underway to better help characterize the risks and benefits of statins in DMD  and inform the optimal molecule to move into clinical studies.

Read more from Solid Biosciences here.