Goals and progress in August 2016

I would like to accomplish the following three goals: #1 Improve Clinical Care, #2 Raise Awareness, and #3 Provide a Cure for Wolfram syndrome. I feel that we have been making steady progress in #1 and #2. #3 is the most challenging one. To achieve #3, we need to first delay the progression of the disease so that we have more time to develop regenerative therapies and gene therapies. To accomplish this milestone, I have been focusing on “repurposing” one of the FDA-approved drugs (i.e., existing drugs) for the treatment of patients with Wolfram syndrome. We have been making steady progress on this. As I reported before, we discovered that dantrolene sodium currently approved for the treatment of spasticity (muscle stiffness) could delay the progression of Wolfram syndrome in animal and cell models. We obtained the orphan drug designation of dantrolene sodium for the treatment of Wolfram syndrome from the US FDA early in 2016. Once I get the approval from our medical center, we can commence a clinical trial of dantrolene sodium for the treatment of Wolfram syndrome. I plan to monitor safety, vision, neurological functions, and diabetes in our patients. The Snow Foundation and the Ellie White Foundation plan to financially support this trial. I am also trying to get more funds from the National Institutes of Health to recruit more patients. You can find the information here. Please stay tuned.