Do we need a new drug?

My answer is yes. Our current focus is to choose the best FDA-approved drugs that can delay the progression of Wolfram so that we can immediately start treating our patients (off-label use). Based on our current results, our candidate FDA-approved drugs are not perfect. Because they are designed for other diseases, these drugs should be improved to suppress the abnormal enzymatic activity in Wolfram patients’ cells. So we need to develop new drugs specifically designed for Wolfram. As I mentioned in my previous blogs, new drugs for Wolfram could be effective for other ER stress-related diseases such as type 1 diabetes and potentially ER stress-related eye diseases.