Marching Towards a Revolutionary Therapy for Blindness and Diabetes in Wolfram Syndrome

I am excited by a new collaboration with Amarantus Bioscience to develop a revolutionary therapy for Wolfram syndrome, the most difficult form of blindness and diabetes. We plan to use MANF protein to achieve this goal. Amarantus is a leading biotech company developing MANF-based therapies for neurodegeneration and blindness, and my team has expertise in Wolfram syndrome making for a wonderful collaboration.

Wolfram syndrome is caused by stress in the endoplasmic reticulum (ER), a cell component involved in many vital functions of eyes, brain, and pancreas. Despite the underlying importance of ER stress in Wolfram syndrome, no currently available drugs can control ER stress. MANF has been shown to block ER stress in a number of different cell types related to various diseases, so we plan to test if MANF protein can block ER stress in eye cells created from induced pluripotent stem cells derived from Wolfram syndrome patients.

How did we purify stem cells from Wolfram syndrome patients? Using a new method developed by a Japanese physician-scientist, we created stem cells from patients’ skin cells and induced them to produce chosen eye cells.  This is how we will test the efficacy of MANF.

Blindness is the most devastating quality-of-life issue faced by patients with Wolfram syndrome. If successful, MANF-based therapies for blindness and diabetes will be established because ER stress is an emerging target for both diseases.  With an estimated 10,000 to 30,000 patients worldwide affected by Wolfram’s, we believe this disease can serve as fertile ground for testing MANF as a disease-modifying treatment for Type-1 diabetes that can later be translated to the broader diabetes population. As we evaluate MANF’s ability to protect eye cells, we will be exploring similar activities with beta cells in hopes of demonstrating a strong activity in Wolfram’s patients.

The Jack and JT Snow Scientific Foundation made this collaboration possible. The co-founder, Stephanie Snow Gebel, is an outstanding person. To save her daughter, Raquel, who has Wolfram syndrome, Stephanie created a non-profit organization with a plan to make a difference by promoting a novel therapeutic paradigm for Wolfram syndrome. JT Snow, Stephanie’s brother and a former SF Giants player, has been helping us. I was so moved by Stephanie and JT Snow, and decided to move to Washington University Medical Center to take over the Wolfram syndrome research program established by late Dr. M. Alan Permutt in 2012. Since then, the Snow Foundation and my team have been working together to make a difference by providing a cure for Wolfram syndrome. I have been studying MANF for over 3 years now, and filed my first patents on MANF protection and marker for beta cells several years ago while I was still at University of Massachusetts Medical School. Now that I have finally been able to forge a pact with Amarantus on an area where we share mutual interest, I believe we may begin to see my research properly translated with the help of the stellar drug development team Amarantus has assembled. We are all hopeful that a breakthrough is on the horizon.

 

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Fumihiko Urano, MD, PhD, is currently the Samuel E. Schechter Professor of Medicine and the Director of International Wolfram Syndrome Registry at Washington University School of Medicine.

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