I think a lot about a new platform for endoplasmic reticulum disease and other rare diseases.
The new clinic requires new expertise in addition to conventional neurology, ophthalmology, and endocrinology. I feel that regenerative therapy and modern medical genetics based on high-throughput sequencing are the key players.
I found an interesting video describing regenerative medicine.
https://www.youtube.com/watch?v=v-Q2kghHwq4
I hope you will have the best week in your life.
Some people say that it is not realistic to develop a novel therapy to stop the progression of eye manifestations in Wolfram syndrome. I don’t agree.
It is possible. If we can modulate ER stress in retinal cells, we can slow or stop the progression of optic nerve atrophy in Wolfram syndrome. I have found a biotech company in Seattle that is developing novel therapeutics for “sight-threatening” eye diseases. If they can do it, we can do it. http://acucela.com/About-Us
The biotech I mentioned above is taking a very interesting strategy. They are trying to use a tablet (i.e., a pill) for delaying the progression of eye diseases.
https://clinicaltrials.gov/ct2/show/NCT02130531?term=acucela&rank=1
I was envisioning that I would use ophthalmic suspensions (eye drops) or intravitreal injections (injection into eyes) for stopping eye manifestations in Wolfram syndrome, but we should also think about using pills.
It is difficult not to imagine what the future may hold when medical breakthroughs are on the horizon. Conversations took place during the last Wolfram clinic regarding clinical trials next summer. Clinical trials are fascinating movements forward in the lives of Wolfram Syndrome patients.
After hearing the news that research is going well and treatments are a possibility, I cannot stop thinking about how my life with Wolfram Syndrome may turn a corner in the years to come. Chasing glucose levels, vision loss, hearing loss, kidney failure, and many other complications due to Wolfram Syndrome will all be dealt with in simpler terms if clinical trials are a success. I think a unique treatment for Wolfram and or even diabetes would be as big as Bill Gates and Microsoft.
Even though having high hopes and dreaming of what could be can set a person up to be further disappointed; hope is one of the driving forces behind life’s remarkable occurrences. I dream everyday about getting a full nights rest and not having to wake up every two hours to check my glucose, as well as not experiencing mood swings due to glucose fluctuations all day. Also, I hope to be able to see and hear well enough to drive myself where I need to go and be able to not hassle with visual and hearing difficulties at work. Then there is the hope of being able to eat a well balanced dies and not eliminate certain food groups so that I can stay out of the emergency room. It is so close I can feel it because if Dr. Urano’s medical breakthrough is a success all my prayers will be answered.
Regenerative Medicine and Regenerative Therapy are the same concepts. Regenerative Therapy focuses on the clinical application of regenerative medicine. Regenerative therapy aims at growing tissues and organs from stem cells derived from patients in the laboratory and implanting them to patients.
When we establish our ER disease clinic, we should include “regenerative therapy” into our platform in addition to endocrinology, neurology, and genetics.
We have established more than 100 stem cell lines from Wolfram patients. We are currently differentiating them into eye cells and brain cells. There are many ongoing clinical trails using stem cells. My goal is to join them as soon as possible. There are many hurdles and challenges, and we need to solve them one by one.
http://clinicaltrials.gov/ct/search?term=stem+cell&submit=Search
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