Dr. Fumihiko Urano Washington University School of Medicine, USA
January 31, 2026
Dr. Fumihiko Urano
Dear Friends,
I hope the new year has started well for you and your family. Thank you, as always, for being part of the Wolfram syndrome community. Your trust, patience, and partnership mean a great deal to us. Everything we do in the clinic, the clinical trial unit, and the laboratory is driven by a single purpose: to improve the lives of individuals and families living with Wolfram syndrome. Our shared goal is CURE4WOLFRAM, and every visit, study, and experiment moves us one step closer. I would like to share where we are now, what we are learning, and how these efforts are coming together as we move into 2026.
New Drugs and Supplements
We are actively developing a systematic platform to identify medications and supplements that may benefit individuals with Wolfram syndrome. Using patient derived induced pluripotent stem cells, we generate brain cells in the laboratory that closely reflect the biology of Wolfram syndrome. These cells allow us to directly test existing drugs, supplements, and new compounds to see whether they improve cell survival, reduce stress responses, support mitochondrial function, or restore healthier cellular balance. Our highest priorities include antioxidants, sigma 1 receptor agonists, NAD activators, idebenone, GLP-1 receptor agonists, and other compounds that target endoplasmic reticulum stress and mitochondrial dysfunction. We also have a long list of additional candidates based on scientific rationale and emerging evidence. This platform allows us to evaluate potential therapies before moving toward clinical studies. We plan to expand this effort and will continue to share updates as we learn more.
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