There is currently no effective treatment for Wolfram syndrome, creating an urgent need to develop novel therapeutics to stop the progression. We are focusing our efforts on this. However, we need to think ahead. After we stop the progression, we still need to replace damaged tissues and protect remaining tissues from cell death.
- To replace damaged tissues, we need to develop stem-cell based therapy. We need to create clinical-grade stem cells using patients’ own skin cells (not embryonic stem cells).
- To protect remaining tissues from cell death, we plan to use trophic factors. We need to find a simple way to constantly administer trophic factors.
Thank you for reading this blog. I feel that today is the beginning of our next step.