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A cure for Wolfram syndrome could lead to a cure for diabetes.

Saturday, August 30, 2014

Small molecules and Stem cells

I have realized that I always think about two things, Small molecules for Wolfram syndrome treatment and Stem cell therapeutics for Wolfram syndrome. I call it SWSW.

We are looking for small molecules (i.e., drugs) that can stop the progression of Wolfram syndrome. We have made significant progress in the last 6 months. My team has been collaborating with 5 pharmaceutical and biotech companies to develop drugs for ER stress-related diseases, including Wolfram and Type 1 Diabetes. Although they are not directly involved in the therapeutic development specifically for Wolfram, their advice is precious. They know how to get the approval from the regulatory agencies. They are very kind. They are very sympathetic and give us candid advice. I wish I had a small biotech company focusing on Wolfram syndrome.

I was thinking about stem cell therapies for Wolfram and Type 1 Diabetes while I was waiting for my iced latte at the coffee shop yesterday. Interestingly, I found a director of our newly formed regenerative medicine center there. So I approached her and talked about regenerative medicine and Wolfram. She offered me to serve on the committee of the new regenerative medicine center. I promised that I would help her develop the center. It was a great conversation. I felt that higher power was helping us. I was so grateful. 

I hope you will have a wonderful weekend. I am sending kind thoughts toward you.