A discovery of a drug target and a potential therapy for Wolfram syndrome

Our article on the discovery of a drug target and a potential therapy for Wolfram syndrome has been just published in the Proceedings of the National Academy of Sciences, one of the most respected journals in Science. Our article has been published in the Medical Science section. “This is freely available online.” This is the first major article published by my team at Washington University and the most important article in my career. A calcium-dependent protease as a potential therapeutic target for Wolfram syndrome

Using stem cells derived from our patients’ skin cells and animal models and cell models of Wolfram syndrome, we have discovered a drug target, calpain 2, and identified a potential drug, dantrolene, for Wolfram syndrome. Our team is currently conducting a dose-escalation study to bring this drug to our patients. Instead of directly targeting calpain 2, we are looking for a way to control the upstream event leading to calpain 2 activation. The upstream event is the dysregulation of cellular calcium homeostasis. I will keep you updated about our progress. I would like to thank my colleagues at Washington University including Dr. Hershey, Dr. Marshall, and Dr. White, my colleagues in Canada, US, Europe, Japan, and South America. I also would like to thank patient organizations that raised funds for this research, including the Team Alejandro, the Team Ian, the Ellie White Foundation, and the Jack and JT Scientific Foundation. You can find them in the acknowledgment section of this article. Looking forward with fingers crossed.