Eye Hope Foundation

Spring update 2021

Dear Friends,

It’s almost half a year since our last update. We hope that you all are in good health and doing well.

In the meantime, we have read and analysed the R&D projects that we received after our call for projects and selected to continue with two of them.

One project solely funded by Eye Hope Foundation is continuing the work by M. Plaas in Estonia on the WFS1 rat. In my opinion this is still the best animal model available to date of Wolfram Syndrome. The project will evaluate neutrophic factors, look at GLP1 / GIP co-agonists and gene therapy as a treatment for Wolfram syndrome. With their established baseline from the previous projects, they have a good comparison base.

Another project was picked in cooperation with the Snow Foundation where we will fund part of the work by B. Delprat and his team in Montpellier. The project also uses gene therapy for administering a protein in a mouse model of WFS1. In parallel the group looks also at a zebrafish model of WFS1.

We wish that we could fund many more projects but after a corona year we are happy we can contribute at least a bit of funding to these two groups.

Besides trying to find a better treatment and managing our daily lives, we as parents have a duty to prepare our kids for the future. Victor is a very positive boy and attending sixth grade in the local international school. As a preparation for the transition to the Middle Years Program, he completed his exhibition project. As a topic of choice, he focused on different types of syndromes (“No surprise”). As part of his work, he performed a 24 km sponsored walk for Eye Hope Foundation collecting a significant amount of funding. He got excellent grades and below are screenshots of his first and last page.

We are very proud of his attitude in life.

On behalf of Eye Hope Foundation,

Lode Carnel

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The 28th of februari

The 28th of februari ... 

Worldwide it is "rare diseases day"...

A day to write you a letter.

A day to thank you for all the support you gave us. 

We told you the story of Victor, of Bertha, of Pim and a lot of others. They have Wolfram Syndrome. Most doctors have never heard about Wolfram, an ultrarare disease. It determines the rest of their lives and the lives of their families. 

There is no cure (yet), the future is unpredictable, people with similar conditions don't live next door, a treatement does not exist (yet) …….

We started Eye Hope Foundation to get some hope and to give some hope. 

To contact researchers aroud the world, to financially support them by setting up some research to find a treatement or a cure.

We have already reached a lot, but unfortunately the end is not yet in sight. 

2020 was a cruel year!

We lost our loved ones in their battle against covid. We couldn't meet each other, we couldn't hug each other. 

Eyeopener, Golf charity cup, the barbecue of V-Care, Maldegem ment for Eye Hope, all those events that couldn't happen! We missed a lot of fundings...

And still you were there with your phonecall, your message, ...

And still you kept on supporting us by donating  to our fund. 

Thanks to you all, we keep on having faith, we keep on supporting the scientific researchers.

Thanks to you all, Eye Hope Foundation is still the leading force behind the research done worldwide on Wolfram Syndrome. 

We hope you are all doing very well!                                                                                                                      

Kind regards, 

www.eyehopefoundation.org

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Autumn update 2020

Dear Friends,

 

Hopefully you are doing fine and taking care of your closest during this health crisis.

As foundation founders, it’s easy to be frustrated over many things. We collect far too little money, we can fund only a limited number of projects, the foundation work comes on top of all daily issues with our children, researchers are years away from a solution etc. etc.

However, we have the duty to our children and families to look at it from another angle.  There are plenty of positives as well, but only if you actively pursue them.

During the past half year, we launched our call for research projects and received more than ten well described proposals.  Several of them made a lot of sense and we wish that we could fund all of them.

In the selection of the projects we focused on three things:

Realism
Timeframe towards patients
Experience in the field
Animal model

After careful consideration, we selected the project of Dr. M. Plaas, Estonia to be funded. The project will run for two years and focus on novel treatments based on gene therapy and novel molecules. We had an excellent collaboration during the past couple of years and we are looking forward to continuing that. Their rats are in our opinion the most reliable animal model available within the wolfram research environment. If co-funding by other patient groups allows, we might evaluate to co-fund other projects as well over the next couple of months.

Therefore, we reach out to other foundations to approach us. Together, we are stronger!

To summarise, we are proud of several things.

Proud of the Eye Hope Foundation for allowing us to search for research projects. Since our start we have been the driving force for Wolfram Syndrome research around the world.

Proud of the three Eye Hope families and many Eye Hope supporters for collecting funding for our organisation. They are unique within the Wolfram field!

Proud of the progress in research during the last years and the perseverance of our researchers.

On a personal note, I am proud of the positivism of my son, his sisters and my wife! They help me through our daily issues and that’s only possible with their positive attitude!

Eye hope we will find good treatment alternatives.

Take care and stay safe,

On behalf of Eye Hope Foundation,

Lode Carnel

 

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Call for research 2020

Dear all, 


I am very happy that I can announce on behalf of Eye Hope Foundation a new call for research projects on Wolfram Syndrome.
This year has been quite difficult for fundraising but nevertheless we were able to put this through.

We have put a deadline for the 15th of September to collect potential projects.

Don't hesitate to reach out to me in case of questions!

Lode
 

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Summer update 2020

Dear all,

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!

 

Cheers,

Lode

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Autumn 2019 update

Dear Wolfram society,

It’s the time of the year again for a short update on our activities during the past 6 months. Thanks to your continued support we have continued to fund a couple of very interesting projects in Belgium, the USA and Estonia. Without your contributions to our fundraising this wouldn’t be possible.

During the past months, an initiative has been initiated in the US to create a Wolfram Syndrome Research Alliance bringing together researchers, patients and medical doctors. As part of this work, an overview was created of currently ongoing research and treatments in Wolfram Syndrome with the different phases.

Figure 1: Wolfram Syndrome Research and Treatment Pipeline, made by Sarah Gladstone.

Many of the projects in the overview, have been initiated or supported by our Foundation.

Since the announcement that Victor is currently taking Victoza (also called Liraglutide or GLP1 agonist), I have been contacted by numerous parents of WFS1 kids. Several dozens of kids are now on Victoza. According to my knowledge, the results are uniformly positive resulting in an increased quality of life at least. Especially the diabetes part becomes manageable. In order to gather the knowledge and experience at these different individuals, I created a Facebook group called “GLP1 for WFS1 kids” (https://www.facebook.com/groups/517126908859489/). Before starting however, its recommended to visit your endocrinologist.

Despite the positive results we observe, there is little known on the neuroprotective effect (eyes, hearing and brain) of liraglutide on the longer term. Therefore, we initiated since the beginning of this year a project in Estonia (Tartu) to investigate the long-term effects of liraglutide on WFS1-deficient rats. This project is running for almost 2 years according to the lifetime of the rat. During the past week, Victor and me visited the research director Dr. Mario Plaas and his co-workers Dr. Anton Terasmaa and Kadri Seppa in Tartu. The project investigates the effects of liraglutide on most phenotypes (diabetes, eyes, ears and brain) and is unique in its kind. Currently it's sponsored monthly by Eye Hope Foundation and we are always looking for funding in order to continue this project. I believe it's key to our society and the results will be presented at conferences of neuroscience.

Liraglutide is in best case slowing down the degeneration which is still our main priority. We discussed also several ideas to go to the next step which is recovery of functions. It's my ultimate hope we can initiate projects on this as well in a near future!

Figure 2: Victor entering the cleanroom in Tartu with A. Terasmaa

Figure 3: Inspection of WFS1 and Wild-type rat by Victor

Figure 4: Visionary drawing as an inspiration to the team, made by Victor.

Figure 5: Final dinner in Tartu, Estonia, with research team.

As a last point, during March next year, the French Wolfram association will organise its bi-annual Wolfram syndrome workshop in Paris where researchers have a chance to interact. The research that is sponsored by Eye Hope Foundation will again be strongly represented and provide major highlights to the conference!

To conclude, things on Wolfram research are moving, still too slow for me, but at least faster than ever before. Time is our worst enemy!

Thanks for reading and take care,

Lode

Eye Hope Foundation – Autumn 2019 update

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Eye Hope Foundation– Spring 2019 update

Dear Friends, Dear Wolfram Syndrome Community,

 

In this newsletter, I wanted to give an overview of the research initiatives we took with our foundation during the last six months.

These initiatives are always discussed and evaluated internally. During the last months, I received significant assistance by Dr. Ir. Yvan Gilliams, a retired engineer that is devoting a large part of his pension to understanding the mechanisms underlying Wolfram Syndrome and supporting Eye Hope Foundation.

 

During January 2019, we decided to donate money to a project in the labs of Prof. G. Bultynck (GBIOMED - KU Leuven) at the KU Leuven. The project will investigate the role of Ca2+ signalling in cell models of Wolfram Syndrome. It will allow us to conclude the underlying mechanism and hence allow us to focus on those molecules that primarily target that pathway. The project will be executed by an experienced Ph.D. student and will also receive European funding.

 

The lab in Brussels is continuing their research with GLP1 agonist on a cell and a mouse model of Wolfram syndrome. Their work was the basis to put my son last October on Victoza (a commercial GLP1 agonist). Their results as well as the results on Victor were presented last week at a conference in the UK. Marianas work won a presentation award (Figure 1). Victor his diabetes is currently managed by Victoza only, allowing us to remove the insulin pump. For effects of GLP1 on the neurodegeneration its too early to conclude anything both on animal and on “Victor’s” level.

Figure 1: Mariana Esteve presenting the work on Wolfram Syndrome at the Islet Study Group and Beta Cell Workshop
 
Therefore, we have started a project in Estonia in the beginning of the year. The group led by Dr. M. Plaas (www.etis.ee/CV/Mario_Plaas/eng?lang=ENG&tabId=CV_ENG) and Dr. A. Terasmaa (https://www.etis.ee/CV/Anton_Terasmaa/est?lang=ENG&tabId=CV_ENG) will investigate the effects of GLP1 agonist on a rat model of Wolfram Syndrome. This project will cover almost 2 years and allow us to make conclusion on the neuroprotective effects of GLP1 agonist. The animals were born in January and started the administration of GLP1 agonist during last month. Both researchers have a long experience within the Wolfram field and were driving forces behind the various animal models.

 

Figure 2: Anton Terasmaa with the WFS1 rats that are receiving GLP1 agonist

 

The projects that we are sponsoring are closely followed by the Wolfram community. The groups we are sponsoring are visited by other foundations that are evaluating to sponsor them as well. I am also pleased to have received a meaningful donation from another Wolfram foundation to fund our Estonian project. The more such collaborative funding we receive the more useful our research will be!

Furthermore, it a great pleasure to note that several WFS1 patients other than Victor have starting to take GLP1 agonist to slow down progression of Wolfram Syndrome. Patients from Iran, Italy, Germany, Spain and USA have been contacting me to know our experiences. In my humble opinion it’s the most promising available drug currently on the market to combat Wolfram Syndrome. The projects that we are doing now will allow us to conclude on the degree of protection it offers against the various symptoms.

Thanks to all the supporters and volunteers helping our foundation grow towards a meaningful organisation. The results thus far prove that a small foundation like Eye Hope can make a difference when the scarce resources are used wisely! And on the latter, you can be sure that I will guard over it!

 

With best regards,

Lode Carnel

Eye Hope Foundation – Spring 2019 update

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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,

Lode

Eye Hope Foundation – Christmas 2018 update

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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

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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,

Lode

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