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A cure for Wolfram syndrome could lead to a cure for diabetes and blindness.

Thursday, August 21, 2014

A stem cell-based replacement therapy

As I mentioned in my yesterday's blog, a stem cell-based beta cell replacement therapy has been approved by the FDA. I was so happy and grateful. This is an important step towards a cure for diabetes. The clinical trial for this new therapy will be led by Viacyte, a biotech company in California.

How do they do it? Here is their strategy.
1. Make insulin-producing cells from human embryonic stem cells.
2. Put these insulin-producing cells in a special capsule (device).
3. Implant the capsule under the skin.

What does this mean? If this technology keeps on improving, patients with diabetes don't need insulin injections. I was very happy and felt grateful to the company and investigators who developed this novel therapy.

What should my team do? We should keep on producing induced pluripotent stem cells (iPS cells) from skin cells of patients and create retinal cells from these iPS cells to implant them to patients. We should follow Viacyte's success. 

Thank you for reading this blog. Our friend and colleague, Stephanie Snow Gebel told me the importance of HOPE yesterday. I am HOPEFUL today. I HOPE you will have a wonderful day.