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.

Amarantus Bioscience has agreed to collaborate with our team to combat blindness

I have great news to share with you. Amarantus Bioscience, a biotech company in San Francisco, has agreed to collaborate with our team to develop a novel treatment for blindness in Wolfram syndrome. It has been challenging to establish this collaboration because of several complicated issues. I was giving up at some point, but the Snow Foundation helped us establish this important collaboration. I would like to give you more detail after I speak with the CEO of the Amarantus. Here is the article published in the Wall Street Journal.
http://online.wsj.com/article/PR-CO-20140620-905760.html

Please read the SF BAY article!

My visit to San Francisco with the Snow Foundation was a big success. I came up with new ideas and strategies to develop treatments for Wolfram syndrome and conduct interventional studies. Our fundraising event was featured by the local news.
http://sfbay.ca/2014/06/16/j-t-snow-leads-fight-against-genetic-disorder/

I will give higher priority to protecting eye cells from cell death during the progression of Wolfram syndrome. To maintain the quality of life of patients, it is incredibly important to confer protection against ER stress-mediated eye cell death. I will articulate our strategy in my next blog.

Update from San Francisco 5: A Cure for Wolfram leads to A Cure for Diabetes and Blindness

My visit to San Francisco with the Snow Foundation was quite fruitful. The highlight of this visit was a fundraising event at Hamlin Mansion. Thanks to JT Snow, so many people came to the event, including Hunter Pence from SF Giants, Robin Lopez from Portland Trail Blazer, 49ers players, and Miss California Crystal Lee. JT Snow, one of our patients Adam Zawan, Stephanie Snow Gebel and I made speeches, and tried to raise awareness of Wolfram syndrome. The event was a big success. The Snow Foundation's booth at the American Diabetes Association attracted many physicians and researchers. I want you to watch this video. http://vimeo.com/82103685

I could establish a few collaborations with biotech companies in SF and physicians at UCSF. We are moving to the next step on our quest for a cure for Wolfram Syndrome. I feel that a cure for Wolfram could lead to a cure for diabetes and blindness. Thank you for your support. 

Update from San Francisco 4

We had a successful fundraising event at Hamlin Mansion in San Francisco last night. Thanks to JT Snow, so many people came to the event, including Hunter Pence from SF Giants, Robin Lopez from Portland Trail Blazer, and Miss California Crystal Lee. I was really impressed by the speech of Adam Zwan who is a patient with Wolfram syndrome. Stephanie Snow Gebel and I also made speeches and tried to raise awareness of Wolfram syndrome. The event was a big success! Thank you all for coming to the event.
http://sfbay.ca/2014/06/16/j-t-snow-leads-fight-against-genetic-disorder/

I want you to watch this video.
http://vimeo.com/82103685

This morning, Stephanie, JT, David, Steve, and I met with a few potential collaborators and discussed our strategies for developing treatments for Wolfram syndrome. Those were stimulating discussions. I will keep on doing my best to make a difference.

Update from San Francisco 3

We had a very productive day yesterday. We had a photo shooting with Lou Brock and JT Snow at the Giants Stadium in the morning. It was a great opportunity to raise awareness of Wolfram syndrome.
https://en.wikipedia.org/wiki/Lou_Brock

After that, we met with many potential collaborators, advisors, and supporters. Thank you all for taking your time. Here is the photo with Stephanie, David, Lou Brock, JT, Steve, Adam, and myself. We will keep on moving forward to beat Wolfram syndrome, the most difficult form of diabetes.

Update from San Francisco 2

My first day in San Francisco was productive. I met with a couple of my collaborators to discuss our strategies. Interestingly, I happened to meet with a girl with type 1 diabetes, Ms. A,  in the coffee shop where I was speaking with my collaborator. She expressed her interest in helping patients with diabetes by sharing her experiences as a patient. I was impressed and encouraged.

As I mentioned in my previous blog, Kerri Sparling has been making a difference by sharing her experiences. She is attending the ADA and plans to visit our Snow Foundation booth #336.
http://sixuntilme.com/wp/

We had a wonderful kick-off dinner with the Snow Foundation last night. I feel that something wonderful is going to happen.

Update from San Francisco 1

I am in San Francisco to attend the American Diabetes Association's meeting, participate in multiple fundraising events, and establish important collaborations. I feel that something wonderful is going to happen.

Press release from JDRF related to MANF

I found a press release from JDRF related to MANF this morning. As I mentioned in my previous blogs, MANF is an attractive target for Wolfram syndrome and type 1 diabetes. In addition to protecting insulin-producing cells from ER stress-mediated cell death, I think that MANF could protect brain cells and eye cells from death. We will keep on working on this molecule.
http://jdrf.org/blog/2014/specific-protein-may-help-beta-cells-survive-in-type-1-diabetes-2/

Flying to San Francisco to combat diabetes!

I am flying to San Francisco tomorrow to attend the American Diabetes Association meeting, establish important collaborations, and participate in multiple fundraising events. I will meet with many people in the next several days, and hope that we can establish important collaborations and raise money for research on Wolfram syndrome and type 1 diabetes. The Snow Foundation has been helping us achieve these goals, and I appreciate their continued support.
http://www.thesnowfoundation.org/

St. Louis Rams Head Coach, Jeff Fisher

Our research on Wolfram syndrome is supported by donations, and Jeff Fisher, St. Louis Rams Coach, helped us last night. Coach Fisher and his colleagues at Rams played at the Celebrity Softball Game last night. Many fans came to the stadium and donated money for the Snow Foundation and other charities. The game was opened by Raquel, one of our patients, pitching the first ball. It was a nice pitch. Thank you again for coming to the important event!

MANF, Wolfram, and Diabetes #6: Protecting remaining tissues

Yesterday, I articulated our strategy for protecting remaining beta cells and brain cells with MANF.
1. Inject recombinant MANF to patients.
2. Stabilize endogenous MANF
3. Activate a receptor for MANF

I feel that these are realistic plans. The same strategy has been applied to GLP-1, a molecule produced in our guts, and GLP-1 related drugs are widely and successfully used for patients with type 2 diabetes.
http://www.medscape.com/viewarticle/751632

We are currently focusing on "recombinant" MANF, and hope to expand our research in this area.

MANF, Wolfram, and Diabetes #5: Protecting remaining tissues

We have been trying to target MANF to protect remaining pancreatic beta cells and brain cells in patients with Wolfram syndrome (beta cells and brain cells) and type 1 diabetes (beta cells). 

All of us have this molecule in our bodies, and our strategy is to enhance the effect of MANF. There are at least three ways to do it.

1. Inject recombinant MANF to patients.
We can crate MANF in the tissue culture dish using biotechnology. We then purify this "artificial" MANF and use it for the treatment.

2. Stabilize endogenous MANF
There is continuous process of loss and replacement of MANF in our bodies. MANF is degraded after certain amount of time in our cells. If we find a way to stabilize MANF, we can enhance the effect of MANF.

3. Activate a receptor for MANF
Many secreted proteins in our bodies have their "receptors." These receptors mediate the effect of their partner secreted proteins. For example, we have "insulin" receptors to mediate the effect of insulin. We are trying to identify a receptor for MANF, so that we can develop a drug that can activate this receptor.

I will continue this topic.

MANF, Wolfram, and Diabetes #4: Protecting remaining tissues

We are trying to target MANF to protect remaining pancreatic beta cells and brain cells in patients with Wolfram syndrome (beta cells and brain cells) and type 1 diabetes (beta cells). 

Although we hold an intellectual property related to MANF in the context of endoplasmic reticulum (ER) stress, this molecule was originally identified by Dr. John Commissiong in 2003. His research team discovered a novel neurotrophic factor in a type of brain cells. 
http://www.ncbi.nlm.nih.gov/pubmed/12794311

Dr. Commissiong has established a biotech company and has been trying to use MANF for the treatment of Parkinson disease. Recently, my team and others revealed that MANF is induced by ER stress and confers protection against ER stress-mediated cell death. Because beta cell death in Wolfram and type 1 diabetes is mediated by ER stress, MANF is an attractive drug target for these disorders. I will articulate our strategy in my next blog.

"Meeting" with Kerri Sparling. Do you know her?

I have been inspired, encouraged, and empowered by patients with Wolfram syndrome and type 1 diabetes.

1. http://www.laduenews.com/society/charities-nonprofits/the-jack-and-j-t-snow-scientific-research-foundation/article_2623d048-c42a-558e-8d59-4c9deb04478b.html

2. http://wolframsyndrome.blogspot.com/2014/05/denver-paris-and-saint-louis.html

I happened to connect with Kerri Sparling yesterday through one of our patients' mother, M. Who is Kerri? Kerri is the pioneer who started using the social media for sharing her experiences as a type 1 diabetes patient. Her blog, Six Until Me, has been read by many people, including patients, doctors, researchers, and etc. I believe that she has empowered many people and is making a difference. My blog is linked to her blog. I recommend you check her site!

http://sixuntilme.com/wp/

MANF, Wolfram, and Diabetes #3: Protecting remaining tissues

I am getting many questions about MANF, and many people are looking at my blogs related to this topic.

http://wolframsyndrome.blogspot.com/2014/01/manf-wolfram-and-diabetes-what-is-manf.html
http://wolframsyndrome.blogspot.com/2014/04/manf-wolfram-and-diabetes-part2.html

We became interested in MANF several years ago. Although we have not published any articles related to MANF, we have been accumulating data. The reason we are actively studying MANF is that this molecule may be able to protect remaining pancreatic beta cells and brain cells in patients with Wolfram syndrome and type 1 diabetes. I will tell you more about our strategy in my next blog.

http://wolframsyndrome.blogspot.com/2014/05/four-pillars-to-provide-cure-for.html

What is "off-label use"?

I received several emails related to "off-label use." "Off-label use" means that an FDA-approved drug is used for an unapproved indication. Once a drug has been approved for one indication, physicians are free to prescribe it for any other indications that in their professional judgment is both safe and effective.

We are working very hard to identify the best FDA-approved drug that can potentially delay the progression of Wolfram. The reason I have not provided the details of our candidate drugs is that you could ask your physician to prescribe any of those candidate drugs for off-label use. I understand that the time of waiting is the hardest part of anything. I read the blog written by one of our patients, Adam Zwan. We need to bring you a safe treatment, but I also understand that the timing is critical.

http://www.thesnowfoundation.org/living-wolfram-syndrome-adam-zwan-9/