In 2015, Alex’s Wish awarded a £50k grant to help progress clinical trials of an oral drug called VBP15, developed by ReveraGen BioPharma to treat Duchenne muscular dystrophy.
The Duchenne Alliance is a consortium of foundations focused on research and therapeutics development for Duchenne muscular dystrophy. Their primary funding arm is through the Duchenne Dashboard. The Duchenne Alliance has funded ReveraGen for a new GMP drug synthesis (3.0 kg), establishing the Phase 2a clinical trial infrastructure, and a murine mdx/DBA biomarker and efficacy pre-clinical trial. Foundations contributing to this include Alex’s Wish, Pietor’s Fight, Micheal’s Cause, Ryan’s Quest and Save Our Sons.
This funding is helping to speed up the Duchenne clinical trials, providing the first funding for setting up the trials in the US and Europe, as well as funding a GMP drug synthesis for these trials. The funding is also enabling innovations in biomarker studies through as benchmarking of VBP15 against Prednisone in mice with muscular dystrophy. These innovations in biomarkers could help speed the development of many other drugs in Duchenne, and other types of muscular dystrophy.
VBP15 has been shown to be effective in animal models of Duchenne muscular dystrophy, and clinical trials in humans are now under way. In studies, the drug reduced inflammation and stabilised cell membranes, both of which are believed to be relevant to treating Duchenne, a disease in which muscle fibre membranes are unstable. The drug is thought to have similar properties to steroids such as Prednisolone and Deflazacort, which up until now have been the cornerstone of treatment for boys with Duchenne. The benefit of VBP15, however, is that it is thought to have significantly fewer side effects than existing steroids, which can be life changing in themselves. Side effects include diabetes, high blood pressure, osteoporosis, short stature and weight gain.