Here are my slides related to potential drugs for Wolfram syndrome. We are studying FDA-approved compounds currently indicated for other diseases to see if they are safe and effective for treating Wolfram syndrome. In the slide 4, I showed the list of drugs that can potentially delay the progression of Wolfram syndrome. 4 out of 8 are FDA approved drugs. This drug screen was based on the mechanism of the disease, which is “dysregulated cellular calcium homeostasis.” (slide 3).
In the slide 6, I showed the efficacy of dantrolene in brain cells differentiated from stem cells derived from Wolfram syndrome patient’s skin cells. You can see the decrease in the signal intensity after the dantrolene treatment, which reflects the suppression of cell death. I also showed the efficacy of dantrolene in the animal model of Wolfram syndrome. The suppression was milder.
We are conducting a dose-escalation study for dantrolene. In parallel, we are testing other drugs. I understand that we are racing against time. We still need to determine the dose, check the safety, and choose the best drug.
Thank you so much for reading this. I hope you have a wonderful day.