It’s a while since I communicated last and with the summer ahead of us, its time for a short update.
The Victoza - liraglutide study (GLP1 agonist) at Estonia is nearing an end. The rats have received daily treatment for 15 months and were monitored during that time period. This project started after we started Victor on Victoza. The results are encouraging and will be disclosed in scientific papers. The tissues and analyses of this study will continue in the next couple of months at different labs. What we know now tells us that Victoza slows down the degradation due to Wolfram Syndrome. It’s probably the baseline treatment for future Wolfram patients. Thank you, Eye Hope Foundation. However, it’s not stopping the disease nor reversing it.
That’s why my focus over the past months has been to focus researchers towards gene therapies especially the brain-related aspect of the syndrome. Gene therapies could eventually stop it or even regenerate the brain cells. However, the technologies have a long way ahead to reach the patient. Therefore, it requires full attention now if we wish to have something within 5 years from now. During the April we had a Zoom meeting where groups from France, Estonia, Belgium and the USA gathered to discuss it. My goal is to get together a project where the researchers collaborate and undertake high risk tests, so we get a quick progress.
Figure 1: Screenshot of the Spring zoom conference focusing on gene therapies
There are basically three different possibilities:
Correct the genetic defects in the cells in vitro (in a dish) and transplant the corrected cells in the patient
Inject a working gene in the cell in vivo (patient) that works in parallel with the defect gene
Correct in vivo the genetic defect in the patient cells e.g. using CRISPR CAS9
On the first possibility there was an interesting paper published by Washington University, showing that diabetes might be cured in mice by transplanting corrected human beta cells.
Figure 2: Science paper on diabetes cure in mice
Now its key to show that this also can be done for the brain cells primarily. It is my belief that researchers need to collaborate in order to achieve something. I have seen initial seeds of that and will continue insisting on it.
We have limited funding and cannot keep parallel projects alive where similar things are done.
Personally, Victor continues to perform very well at school. His feedback from the teachers is truly amazing and something to be extremely proud of. It is difficult to satisfy his thirst for knowledge. The Corona implications are of course a serious challenge for a weak-sighted person. It is hard to explain to him why such unseen measures were taken for this. Courses happened online on a screen where he almost sees nothing from. Wolfram Syndrome is a much more devastating disease and we could only wish to have a fraction of the budgets. So, your support is needed more than ever!
I attach a picture of last week’s swimming here in Norway where we had the beach for ourselves. Swimming 9 PM in turquoise waters, was no problem.
Figure 3: Victor ready to take his “kveldsbad” (evening bath)
To quote him: “Come on Dad, lets swim. It’s here and now. Tomorrow it might rain.”
We love, admire and take an example to our son!
Enjoy your summer like we will do and feel free to drop me a message if you wish to!