have received many questions related to dantrolene for treating patients with Wolfram syndrome and diabetes. We are still gathering evidence. Here is the summary.
1. Dantrolene could normalize cytosolic calcium levels in a beta cell line and a neuronal cell line deficient in WFS1 gene (cell models of Wolfram syndrome).
2. Dantrolene could prevent hyper activation of calpain 2 and cell death in a beta cell line and a neuronal cell line deficient in WFS1 gene.
3. Dantrolene could prevent cell death in neuronal cells derived from iPS cells of a patient with Wolfram syndrome (a human cell model of Wolfram syndrome).
4. Dantrolene could prevent calpain 2 activation in brain tissues of the brain-specific WFS1 knockout mouse (a mouse model of Wolfram syndrome)
The data gathered so far are encouraging, but we should be careful. Thank you for your patience.
Kindly,
Fumi Urano