Eye Hope Foundation

Eye Hope Foundation – Christmas 2018 update

Dear friends,


During our last letter, I reported on the increasing popularity of GLP1-agonist to treat Wolfram Syndrome in animal models. These results triggered me to arrange a workshop in Leuven during last week (19-20th of November) where our researchers from Belgium met with Dr. M. Plaas and Dr. A. Teresmaa for Estonia. The goal of the meeting was to align the different ongoing research projects on GLP1 agonist on mice, rat and human 😊. Prof. G. Bultynck and Prof. C. Verfaillie presented their projects and where introduced to our growing Wolfram research community (close to 15 people in Belgium only!).

Prior to the meeting, I asked Victor to draw a picture for the researchers. Below is what I call an artist impression of the research world, a mouse getting an injection with some medicines by the doctors… The drawing is also signed and dated, the original stayed behind at the labs!

The workshop concluded with a general impression that GLP1 agonists are probably the most promising drugs for Wolfram patients that are currently available. Mice and rats don’t develop diabetes if the GLP1 agonist treatment is started early enough. Furthermore, Victor has now had a significant number of days where he has been insulin and pump-free like he had no diabetes. Of course, we still need to monitor the food and it doesn’t allow for “un-healthy” kind of lifestyle. However, we are short of words to describe how it feels after 4 years with meal-to-meal management of carbohydrates and insulin. We can only wish that this will continue for ever, but that probably asking too much. As a side-effect we rather must fight against vomiting and hypoglycaemic events, although those have been diminishing during the last weeks. I am happy to discuss our findings with other potential parents of WFS1 children, don’t hesitate to contact me on lode.carnel at gmail.com.

Besides diabetes, the community is still hopeful for the neuroprotective effects of GLP1 agonists that could delay the vision and brain related issues.

Since Victor is the first kid with Wolfram Syndrome that is on the drugs, the result also impressed the researchers. Dr. M. Plaas described Victor as his hero when he found out what his animal-based research has led to. The initial results from Victor seem to go beyond what the researchers thought was possible.

I am happy that I took the risk of putting my son on this medication. I hope that this short time optimism will translate into long term rewards as well.

After my 38 years on this planet, I am however very down-to-earth, and I realise that Wolfram patients will need more than a pharmaceutical treatment. The best result I am willing to accept is a status-quo due to medication. Therefore, it was interesting to meet with Prof. Bultynck who will analyse wolfram syndrome on a molecular level. This will be the basis on which we will find better and more targeted molecules in the future (https://gbiomed.kuleuven.be/english/research/50000618/50753344).

Finally, we met Prof. Verfaillie her team consisting of four persons: Catherine, Arefe (principal researcher), Kirsten and Thomas. All of them or working on a CRISPR CAS9 gene therapy technology suited for Wolfram Syndrome. While this project carries probably the highest risk and the longest timeline, it also has the highest potential and hence should be started now. It has the potential to cure the genetic defect on a cell level. Initial results are exciting, and we need to investigate how we can fund this project going forward. It carries also a potential for several other rare diseases!


With this I hope I gave you some insight into our research and I want to thank all the researchers that contributed to this event. Thanks Mariana, Evy, Lies, Geert, Tim, Lode, Lieve, Mario, Anton, Catherine, Arefe, Kirsten and Thomas!  Keep up the good work, going from research to having Victor on a medication in 1,5 years must be close to record-breaking! It proves that Eye Hope Foundation is making a difference already now.

I hope to reflect at the end of next year with the same satisfaction about our projects as has been the case this year.


Therefore, I also want to wish all readers and contributors to Eye Hope Foundation a happy end of the year and the best wishes for 2019! Go and spread the Eye Hope Foundation word!


With best regards,


Eye Hope Foundation – Christmas 2018 update


Choose Eye Hope Foundation as your good cause during 'The Warmest week' by Stu Bru

Eye Hope Ivzw is just like last year one of the charities for Studio Brussels’ X-mas fundraising De Warmste Week. Studio Brussel is one of the most popular Belgian radio stations and each year they collect a large sum for charity. During the warmest week they broadcast 24/7 with information about the fundraising events performed and the charities which are supported.

This year participants to the ‘Warmathon (one of the actions of the warmest week) kan pick the charity which they would like to support directly at the registration. Both in Ghent (22nd Dec. 2018) and in Bruges (23rs Dec. 2018) people will run, jogg, walk,… to support Eye Hope iVZW. Hopefully we will be able to post some nice pictures of the event in the coming weeks.


These are the different fundraising events for Eye Hope iVZW  which are for the moment ongoing as part of the Warmste week:

-Kiwanis YP Wervik @ Kerstmarkt Geluwe (Zo 09 dec - 14:30 tot 20:30)
-Janssen Pharmaceutica Warmste week (Ma 12 nov tot Vr 21 dec)
-Janssen Pharmaceutica Kerstmarkt (Di 11 dec - 11:00 tot 15:00)
-Orodée opening (Do 29 nov tot Za 01 dec)
-Warmste soep (Ma 05 nov tot Do 20 dec)
-Bondgenootje van gemeente Zonnebeke en de Warmste Week (Di 30 okt tot Wo 19 dec)
-Kerstmarkt in het Jan Yperman Ziekenhuis (Ma 17 dec tot Do 20 dec)
-De Warmste Thuis (Do 18 okt tot Di 18 dec)

-De warmste week van Geotechniek - more information


More information about the different events can be found on the website of de Warmste Week: https://dewarmsteweek.stubru.be/goededoelen/eye-hope-ivzw


Autumn Update

Dear all,


The last time we gave an update on our foundation was after the Wolfram workshop in Paris.

Since then several fundraising events have happened enabling us to raise a substantial amount.  We are immensely grateful to all people contributing and supporting our foundation in that way.

Thanks to that funding, our foundation has funded three projects during the last months.


The group of Pof. C. Verfaillie has been granted an initial budget to start their gene therapy work using adapted CRISPR/CAS9 technology. Currently, although the project is started, there is an ongoing activity to recruit the best researchers. If this project shows promising results, it could be a cure for blindness related to rare genetic diseases.


At the ULB, initial experiments have been done with drug repurposing. Using the GLP1 agonist on the mice models of WFS1 disease, promising results were shown for diabetes phenotype. It remains to be analysed how the effect on nervous systems and hence vision will be. The excellent quality is reflected in an oral presentation that was held on the European diabetes conference (https://www.easd.org/virtualmeeting/home.html#!resources/glp-1-analogues-protect-beta-cells-in-models-of-wolfram-syndrome-12312e34-0d39-499d-a4b2-d56622c38989).


Our support in the US at the lab of Prof. F. Urano has also progressed. We are hopeful that MANF carries the potential to regenerate in the future some of the vision losses. Fumi Urano posts regular updates on his blog (https://wolframsyndrome.dom.wustl.edu/dr-uranos-blog/).


We are also pleased that the lab of L. Moons has been able to finance the second phase of their project. She will continue to investigate the effects of the treatment on the eyes and brains of the animal models.


Other interesting news is the article that was recently published about use of GLP1 agonist in WFS1 rats showing a positive effect. https://www.nature.com/articles/s41598-018-28314-z Dr. M. Plaas also developed the WFS1 mouse we are investigating. A collaboration is planned between Dr. Plaas and the ULB / KUL groups.


Eye Hope Foundation is also please to learn that another group of KULeuven, the group of Prof. G. Bultynck has obtained a grant to do research on Ca2+ signalling in WFS1-syndrome (https://gbiomed.kuleuven.be/english/research/50000618/50753344/introduction-1).

Finally, Prof. T. Barrett is close to start his clinical trial using Sodium Valproate during the next month. More information about the clinical trials can be found on http://www.wolframsyndrome.co.uk/clinical_trial.php.


On the private side, Victor has started in the beginning of October on a new medicine using GLP1 agonist to reduce the progression of the syndrome. He is the second patient worldwide with WFS1 that is going for this treatment. His vision deteriorates very fast. Since we discovered he was a WFS1 patient in autumn 2015, his vision dropped from 35 to less than 5%. So basically, he cannot read anymore posing an endless list of practical problems to tackle. That brings us to braille-learning and becoming member of the association for blind people.

Nevertheless, it doesn’t stop him yet, he is still going to cinemas, swimming and playing football. He is walking with Nordic walking sticks and his energy and positivity motivates us to keep on fighting for a cure every day. He learned the braille alphabet in less than a month and will proceed now to his first books!


We do hope from the deepest of our heart that the treatment we are currently doing, will give us time to continue our work.

We will never stop and do everything possible to give children with Wolfram a joyful future!

Our work is only possible to your contributions and activities. Thank you for giving us all hope.


Best regards,



Update on the research on Wolfram Syndrome

With Wolfram symposium of Paris just behind us and 2018 almost halfway through, I thought is was time to give you an update on the supported projects and their evolution.

Every 2 years we have this great Wolfram-conference in Paris. Wolfram researchers from all over the world come here to present the results they achieved and the goals they have set for the years to come. In 2016 it was my first time to attend at this symposium. Now, 2 years later, I am very happy to see that the Eye Hope Foundation has made a difference in the research on Wolfram Syndrome and has been able to accelerate the research and to give the financial support to the scientists who are trying to find a cure . 

The scientists we have funded (Dr. M. Esteve, Dr. J. Van Houcke, Prof. C. Verfaillie, Prof. F. Urano, Prof. T. Barrett and Dr. C. Delettre) all presented their results at the workshop that was located in the proximity of the famous Eiffeltower

The research has clearly advanced. The progress is visible on the three aspects:

  1. First of all they try to delay the progression of Wolfram Syndrome by using existing approved drugs.Three different medicines showed promising results on human patients with Wolfram Syndrome although none is conclusive at this point of time. More research is needed to conclude which currently tested medicine is the most effective for human patients. 

  2. Second strategy is stopping the progression of Wolfram Syndrome through gene-therapies or new mini-molecules. This strategy has a longer timeframe. Initial results on cell level and animal models were shown but are still preliminary and more research is needed. 

  3. Third and last point to handle is to find a cure for the damage through regenerative medicine. On this third step Prof. C. Verfaillie showed us a very promising presentation on CRISPR-CAS9. This is a technology to correct gene defects, dealing with the actual core of the problem. 

As you can see, despite the promising results and the clear progress in past 2 years, a lot of work still has to be done. Therefore funding is mandatory.

Our foundation has shown its ability to achieve success during the past years. But there is more, I am happy to announce that the Eye Hope Foundation will be funding three new projects during Summer 2018.

The first project is a follow-up project at the ULB where a side-by-side comparison of different medicines and combinations thereof will be done on cell-and WFS1 mice-models.

The second project will support the research at Washington University on MANF as a regenerative medicine for e.g. visual impairment.

Last but not least, the Eye Hope Foundation has decided to fund the lab of Prof. C. Verfaillie at the KULeuven to provide a gene correction of the WFS1 mutation using CRISPR-CAS9 technology.

All these projects will be described in more detail at a later stage.

I am also thankful to have met these excellent researchers from around the world as well as the other patient-organizations, giving us hope and motivation to carry on our efforts.

I am optimistic we will find a treatment against Wolfram Syndrome.

The only question is when will it happen? And will our kids still be able to take advantage of it?

Thanks for reading and for your contributions,

On behalf of Eye Hope Foundation,

Lode Carnel.





Damme Golf Charity cup


Last weekend we could again enjoy the Damme golf charity cup.

Again the revenues of this party were partly donated to the Eye Hope Foundation. 17,000 euros was collected to support the research into a cure for Wolfram's syndrome !! Thanks to the organization, the sponsors and the participants who made this possible!



Please support Victor's teacher, Alexander Cadden, in his challenge this weekend!

Victor's teacher, Alexander Cadden, was inspired by the incredible positivity that Victor shows in his classes.

Besides the wonderful thirst for knowledge and an eagerness to learn about and engage with the world around him, Victor has tought his teacher that nothing is impossible.  As someone tells Victor that something isn't possible, his answer is always the same; 'Why not?' For Victor, the only failure would be to not have tried in the first place. 

So Victor became his inspiration in taking on this challenge. 

In Grenland, Norway, there is a challenge called the Ten-Tops-run. The aim in this challenge is to climb 10 out of a possible 17 hills between May 1st and November 1st.

This year mister Alexander will be climbing all 17 tops and to add a bit more of a challenge, he will aim to do this in one single weekend!

(9th– 10th June).

He will walk over 90km in ascents and descents together with his friend, Simon.

The first goal with this challenge is to inspire Victor, the boy who inspires him.

The second is that he wants to raise some money for the Eye Hope Foundation and to give some hope to the parents and children who are battling this cruel disease. 

YES you too can support Mr. Alexander! No amount is too small for such an extraordinary attempt! Donate now!

You can read all all about this challenge here.

Many thanks to Alexander Cadden for being a great teacher for Victor, and to support him in his daily struggle with Wolfram's disease. 

We wish him the best of luck in his challenge this weekend. 


Aoigems Vier warms our hope

The 'warmest activity' of Aoigems vier raised 17.000 euro in total of wich 8500 euro was donated to our Eye Hope foundation.

With this gift not only the 'warmest week' got warmer, our hope was given a boost too! 

We will keep on fighting ... for hope ... for a disease inhibitor ... for a cure for all children suffering from Wolfram Syndrome. 

Thank you Aoigems Vier!


Wonderfull day and great result on the Skimathon by Snowplus

By organising the Skimathon, Snowplus had the intention to give Bertha an unforgettable day. Besides this first goal they wanted to raise money for our Eye Hope Foundation.

The first target was reached on saturday. Bertha was shining and had the day of her life. For 1 day she forgot all about her worries. Her parents Stephan and Sophie saw how much their daughter enjoyed the day! 

After some calculations, Snowplus had to conclude that their second intention was a great succes! The skimathon raised no less than 23.854,78 EURO for Eye Hope Foundation. 

We would like to thank everyone with Snowplus to support our battle against Wolfram Syndrome so much!

Photo by Frankie and Fish