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/