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

Thursday, January 16, 2014

MANF, Wolfram, and Diabetes: What is MANF?

Since a biotech company in California licensed my invention on MANF for therapeutics and diagnostics for Wolfram syndrome and Type 1 diabetes, I have been getting a lot of emails on this topic. I would like to briefly explain you about MANF. In short, MANF is one of our targets for developing patient-based therapeutics and diagnostics for Wolfram syndrome and type 1 diabetes.

MANF stands for mesencephalic astrocyte-derived neurotrophic factor. This molecule is created in our body, and it was originally isolated from a type of brain cells called astrocytes. Several years ago, we discovered that MANF was secreted from pancreatic beta cells and neurons when their endoplasmic reticulum calcium was depleted. We thought that this was a potential biomarker for Wolfram syndrome and filed an invention disclosure. Interestingly, we found several articles reporting that MANF can confer protection against neuronal cell death mediated by endoplasmic reticulum dysfunction. So we started testing if MANF might confer protection against pancreatic beta cell death mediated by ER dysfunction, and the preliminary results were positive.

We still have to do a lot of preclinical studies. Also, the company that has licensed our invention is mainly developing drugs for Parkinson's disease. However, it is good news that they are also interested in Wolfram syndrome and type 1 diabetes.