I think about two S’s all the time, Small molecules (i.e., drugs) and Stem cell-based therapy.
In Wolfram syndrome, there is imbalance of calcium levels between two cellular compartments. It seems like that brain cells and insulin-producing beta cells are sensitive to misregulated calcium homeostasis. Using small molecules, I would like to tip the balance to our favor. We are getting encouraging results.
By the way, I was speaking with Dr. Louis Philipson, Director of the Kovler Diabetes Center at the University of Chicago. He shared with me wonderful ideas on potential treatments for Wolfram syndrome. Dr. Philipson always has intelligent and constructive ideas. The following video is quite encouraging. We will follow Dr. Philipson’s success.