In Wolfram syndrome, insulin-producing pancreatic beta cells and brain cells selectively degenerate. Other cells, such skin cells and immune cells, are healthy. Why are beta cells and brain cells so sensitive to the loss of function of Wolfram gene?
It has been recently suggested that beta cells and brain cells have many common features. My hunch is that brain cells and brain-like cells, including beta cells, are sensitive to dysregulated calcium homeostasis. So if we can modulate cellular calcium homeostasis using small molecules, we can potentially delay the progression of Wolfram syndrome. We have preclinical data supporting this model. We can disclose the information soon.
Thank you for reading this. I hope you have a wonderful day.
Kindly,
Fumi Urano
http://wolframsyndrome.dom.wustl.edu/
