Dr. Urano's Blog

Success

Drug Discovery Efforts Update

Our manuscript on the discovery of mechanisms of ER stress-mediated cell death has been just published in Science signaling. This study is part of our continued efforts to develop novel treatments for Wolfram syndrome. The method introduced in our study can be used to test the efficacy of new drugs.

Our drug discovery efforts are moving forward. As I mentioned in my previous blog, we have started an exciting collaboration with the drug development team at National Center for Advancing Translational Sciences, NIH. My team has also joined their patient registry program.

We have started receiving new types of drugs from NCATS. They are sincere and motivated. It is clear to me that they want to help patients with rare diseases, including Wolfram syndrome. We keep on testing these new types of drugs. Our goal is to develop drugs specifically designed for Wolfram. These drugs target the upstream problem in patients with Wolfram, so that these can potentially stop the progression of optic nerve problems, neuronal problems, and endocrine problems.

We are also actively testing FDA-approved drugs (drug repurposing) in our animal models of Wolfram syndrome. We are making progress. I understand that we are racing against time.

Thank you for reading this blog. I always appreciate your encouragement and support.

Take care,

Fumi Urano