In addition to developing therapeutics and diagnostics for Wolfram syndrome and seeing and speaking with Wolfram syndrome patients, I speak with medical officers and scientific officers of pharmaceutical and biotech industries almost every day. Why?
One of our advisors once said to me, “You are doing RESEARCH, not developing therapeutics.” I was a little shocked. After I listened to her explanation, I completely understood what she meant. I should learn how to bring our drugs, small molecules, genetically-modifed cells, and diagnostic devices to our patients. I should secure my intellectual properties, have a clear and reasonable strategy to go through the regulatory processes, and design a fair and realistic clinical trial plan. I learn new things every day, and I feel that we are moving into the right direction. When I moved to Washington University and took over Dr. Permutt’s program, I could not see the clear path. Now I can see it. I have wonderful colleagues at Washington University. My sincere gratitude to the Snow Foundation and Stephanie Snow Gebel. She is my colleague and friend. They are closely working with other patient organizations including the World Wide Society of Wolfram Syndrome Families and the Ellie White Foundation For Rare Genetic Disorders. Our highest priority right now is to bring a “drug” that can potentially delay the progression of the disease to our patients as soon as possible. Our ultimate goal is a cure. I can see roadblocks, but we should keep on moving. A cure for Wolfram can lead to a cure for diabetes.
I feel so grateful and hopeful today. I feel empowered. I hope you have a wonderful weekend.