Eye Hope Foundation

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.





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