Adult beta cells proliferate? How about eye cells and brain cells?

I talked about stem cell-based therapy yesterday. In parallel, we are seeking a way to increase the proliferation of remaining insulin-producing cells (i.e., pancreatic beta cells) and eye cells.

Is it possible? Growing evidence indicates that adult human beta cells can proliferate under certain conditions. The challenge is that adult human beta cells have very low proliferation rates. So we need to find a way to "increase" the proliferative potential of adult human beta cells. Interestingly, significant levels of proliferation occur during pregnancy.

We are considering leveraging our potential to increase the proliferation of our beta cells and neuronal cells. One of our candidate molecules is MANF. MANF is produced in our brain cells. The following article shows that MANF can increase the proliferation of "rodent" beta cells. Our next step is to develop a way to activate MANF and increase the proliferation of human beta cells and eye cells.
http://www.ncbi.nlm.nih.gov/pubmed/24726366

Thank you for reading this blog. I hope you will have a wonderful Sunday! I am sending kind thoughts toward you.

Small molecules and Stem cells

I have realized that I always think about two things, Small molecules for Wolfram syndrome treatment and Stem cell therapeutics for Wolfram syndrome. I call it SWSW.

We are looking for small molecules (i.e., drugs) that can stop the progression of Wolfram syndrome. We have made significant progress in the last 6 months. My team has been collaborating with 5 pharmaceutical and biotech companies to develop drugs for ER stress-related diseases, including Wolfram and Type 1 Diabetes. Although they are not directly involved in the therapeutic development specifically for Wolfram, their advice is precious. They know how to get the approval from the regulatory agencies. They are very kind. They are very sympathetic and give us candid advice. I wish I had a small biotech company focusing on Wolfram syndrome.

I was thinking about stem cell therapies for Wolfram and Type 1 Diabetes while I was waiting for my iced latte at the coffee shop yesterday. Interestingly, I found a director of our newly formed regenerative medicine center there. So I approached her and talked about regenerative medicine and Wolfram. She offered me to serve on the committee of the new regenerative medicine center. I promised that I would help her develop the center. It was a great conversation. I felt that higher power was helping us. I was so grateful. 

I hope you will have a wonderful weekend. I am sending kind thoughts toward you.

I feel grateful because...

I receive a few hundred emails from patients with Wolfram syndrome and diabetes every week. As I mentioned in my previous blogs, I respond to every single email. It is my privilege to work for you. So please feel free to contact me.

I was grateful yesterday. I had a few meetings, and I could feel that things are going into the right direction. The unmet medical needs should be fulfilled and patients should receive the best care. On my way home, I remembered Dr. Rossini's words. He was my mentor. "Fumi, we should always offer help to our patients. Before they leave, please make sure if they still need help. Please make sure you offer help with compassion." He always emphasized the importance and power of "kindness" and "compassion".

I hope you will have a wonderful weekend. Thank you for reading this. I am here for you.

Collaboration with Dr. Gil Leibowitz

I am very grateful today. We will officially work with Dr. Gil Leibowitz at Hadassah Medical Center in Jerusalem on Wolfram syndrome, and this will be funded by the US-Israel Binational Science Foundation in the next four years. We will develop and test a new treatment for Wolfram syndrome. 
http://www.hadassah-med.com/doctors/prof-leibowitz-lev-gil.aspx

Dr. Leibowitz is my long-term collaborator. He is a dedicated physician and scientist, and a very kind and reserved person. I first met with him at a diabetes meeting in France several years ago. I presented our work on Wolfram syndrome and Dr. Leibowitz showed his data on ER stress in beta cells. He mentioned that he had patients with Wolfram syndrome. Dr. Leibowitz has conducted a small scale clinical trial on Wolfram syndrome.
http://clinicaltrials.gov/show/NCT01302327

I feel grateful today and appreciate this opportunity. Thank you, thank you, thank you. I hope you will have a wonderful day.

Chasing the cure for Wolfram syndrome

I'd like to propose a two-step strategy to achieve a cure for Wolfram syndrome. The first step is to stop the progression using a small molecule (i.e., a drug) and the second step is to replace the damaged tissues using a stem cell technology. In parallel, we try to restore the functions of remaining pancreatic beta cells, eye cells, and brain cells using a small molecule or biologics such as MANF. A new technology called a "genome editing" can be combined with a "gene therapy." All of these are ongoing in my team.

I believe that these are all achievable goals. John Crowley, a father of two children with Pompe disease, and William Canfield, MD, PhD, a Pompe disease researcher, worked together, established a biotech, and discovered a treatment for the disease. Mr. Crowley could save his children's lives and is now looking for a cure. If they can do it, we can do it.

https://en.wikipedia.org/wiki/John_Crowley_(biotech_executive)
https://en.wikipedia.org/wiki/William_Canfield