1. can you introduce yourself?
My name is Ludivine Verboogen, I founded the F101G with my husband Romain Alderweireldt and I am the mother of Aurelien who has Marfan syndrome.
2. can you explain to us what is the project 101 genomes Marfan?
P101GM consists of setting up a bioinformatics platform that crosses phenotypic data, i.e. the complete medical record of patients and genomic data, i.e. the complete genetic mapping of a cohort of 101 patients with Marfan syndrome.
By cross-crossing these data, we hope to better explain the variability of the disease, which is enormous, and eventually identify protective genes that could be replicated by drugs. I will give a concrete example: we take two individuals with Marfan syndrome who have the same mutation but they have very different impairments, one is very slightly affected and the other is severely affected. By studying their genetic mapping thus completes their genome, we can realize that in the one who has little reach, there is a gene that is active and that protects it from the Marfan mutation which is the same.
If we can identify this protective gene and we find that this protective gene produces such a protein, we could try to create a drug that replicates the effects of this protein that would give to patients with more affected patients.
3. Why did you create the Fondation 101 Génomes ?
At birth, our son Aurélien was diagnosed with Marfan early on. We realized that medical solutions existed, but my husband and I would like to allow all Marfans patients to have better living conditions by giving them access to treatments and medications much earlier in life. children are also very targeted , which could allow them to avoid heavy surgeries such as the heart, back, eyes and could really reduce the severity of their symptoms.
4. What do you expect from Project 101 Marfan Genomes?
Our dream to my husband and I is to advance the research by 10 years. A geneticist explained to us that by creating this platform, we will make available to researchers and scientists a database that they do not have access to today, it would allow them to make a 10-year advance in research.
From a research point of view, Marfan's disease is not an orphan disease because there are many researchers working on marfan's disease around the world. This interest of scientists may be explained because it is a cardiovascular disease and that cardiovascular disease is unfortunately among those that kill the most. So it may be interesting to study Marfan syndrome for other diseases as well. Here, researchers, all researchers, regardless of their country, their language, their university, are provided with a database with safe and free access.
Some of these researchers have been working on Marfan syndrome for years. Once they have access to this new information, as some of them may already know what they are looking for, they may have confirmation of their intuitions. We dream that this could allow new drugs to be released, hopefully soon enough.
5. What are the key factors for the success of the Project?
We have assembled a fantastic team of scientists, that's fundamental. They're hyper-specialized people. The two reference centres in the world for Marfan syndrome are Ghent and Baltimore - which has very close ties with Antwerp. So in our misfortune, we were lucky enough to be in Belgium close to hyper specialized and hyper-skilled centres in the field. We also need an excellent bioinformatics platform, we are still negotiating with partners who are at the forefront of new technologies. We also need a good communication with regard to the outside world, trying to have a simple and accessible message because today new technologies really open the way to personalized medicines, to propose treatments different and better to care for people. And of course, we have to raise funds to fund this platform.
Founder of the F101G