As I mentioned repeatedly in my previous blogs, my highest priority right now is to start interventional studies using currently approved drugs. This strategy is called “drug repurposing.” This is an attractive therapeutic option because the development of novel therapeutic compounds takes a lot of time and money, which can be avoided by the use of readily available FDA approved drugs. In the treatment of Wolfram syndrome, drugs can be targeted to several areas of endoplasmic reticulum (ER) dysfunction. Currently we are conducting a mouse trial using a modulator of ER calcium homeostasis. If we get a positive result in this mouse trial, we will move to interventional studies. I keep my fingers crossed.
Thank you for your continued support. I will keep on doing my best.