Focus my attention to a cure for Wolfram syndrome - a small piece of paper

My visit to Tokyo was fruitful. I had a chance to write my wish on a small piece of paper and hang it on bamboo because a Japanese Star Festival was approaching. I will keep on focusing my attention to a cure for Wolfram syndrome.

Focus your attention to a cure for Wolfram syndrome

Something interesting just happened. I was revising my grant application related to the therapeutic development for Wolfram syndrome. The following picture suddenly popped up on my computer screen although I did not push any key. ENERGY FLOWS WHERE ATTENTION GOES!

Update from Tokyo, Japan 2

I was interviewed by editors of "Resident Notes", a journal for young doctors, today. I thought it was a great opportunity for me to raise awareness of Wolfram syndrome in young physicians. So I mainly talked about Wolfram syndrome. I explained them why I started working on Wolfram, told them about the disease, and emphasized the importance of giving patients hope. In addition to developing new treatments, I will keep on raising awareness of Wolfram, diabetes, and blindness.

Update from Tokyo, Japan

I just gave a lecture on Wolfram syndrome at the National Center for Child Health and Development, Tokyo, Japan. This is the largest children's hospital in Japan. I met with motivated doctors and researchers. I was surprised to find out that genetic testing for Wolfram is not covered by health insurance in Japan. We need to keep on raising awareness of Wolfram syndrome.

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 will use MANF protein derived from human brain 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. This will be a wonderful collaboration. Please see the press release in the Wall Street Journal.
http://online.wsj.com/article/PR-CO-20140620-905760.html

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. So we will 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? By a new method developed by a Japanese physician-scientist, we created stem cells from patients’ skin cells! We have chosen eye cells to test the efficacy of MANF first because 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. 

I will share with you the significance of this collaboration tomorrow.