Margaux, the 5-year-old girl forced to live in a bubble saved by European research

Margaux, who today smiles cheerfully and loves his five years, has two birthdays. It's September 20, 2013, when his mother Marijke gave birth to Belgium. The second is September 5, 2014, when researchers from Telethon and San Raffaele in Milan discovered why they were getting sick until they could breathe easily, allowing them to be treated with state-of-the-art gene therapy. developed with European funds. for research.

The story of the small Margaux moved by the public gathered today in Brussels in the plenary room of the European Parliament where President Antonio Tajani gave the floor to the parents of the girl with Luigi Naldini, Director of the Institute of Gene Therapy of the Telethon (Tiget), as part of the high-level conference "Research and Innovation in our daily life", organized by Euratom in collaboration with the EU executive.

Margaux was deficient in the enzyme adenosine deaminase (Ada), a rare immunodeficiency that compromises the immune system to the point of rendering it helpless in the face of any bacterial threat, forcing people to lead isolated lives in sterile environments. That's why the little patients who suffer from it call themselves bubble kids.

"Margaux had his first seizure at five weeks of his life – said Father Hein Moreels in Brussels – after a night at the hospital, the pediatrician sent us home saying that he was not there. After a week, there was a brief new hospitalization and then two months later, one week at a time, during which she was subjected to antibiotic treatment. has something that you can not understand, "others repeated" it's healthy. "They sent her home again."

Then, in the New Year 2014, the most serious crisis occurs. "He was three months old and he was very weak, he could not breathe," recalls his father. Margaux is taken to the University Hospital of Leuven, a city located east of Brussels. Finally, her parents get the right diagnosis: she suffers from Ada. But the bad news is not over. Soon, we understand that a stem cell transplant is unlikely to succeed and that Margaux's younger brother is not a compatible donor.

Hein Moreels and his wife Marijke Geens are put in contact with Alessandro Aiuti, researcher at the Telethon Foundation and medical researcher at San Raffaele in Milan: a way to save Margaux is and remains a state-of-the-art gene therapy at San Raffaele.

When the cure begins, September 5, 2014. "The second anniversary of Margaux, its rebirth," said his father in front of the European Parliament, recalling that this year's Christmas was the first that they could celebrate together. Belgium after a first three-month treatment cycle. In 2015, things improved until, in May 2016, the baby was considered sufficiently cured to start attending kindergarten and to socialize with his clbadmates. Today, few things remind her parents of what Margaux has had.

To quote the words of Professor Luigi Naldini, that of Margaux "is the story of a dream of a new medicine coming true. His genes – explained the doctor – were taken, corrected and reinserted. And now, they produce blood cells that work. His case is one of the first successful treatments. After his gene therapy was put to the test in the world, with more than 100 patients only in Milan. Developing the cure would have been impossible without European funding: the EU believed in the projects of our scientists, ensuring that this treatment became the first case of gene therapy commercialized in the world. A challenge that demonstrates the power of science when networking. "

The Margaux case is one of the biggest successes of European investments in research and innovation. "Without innovation and research, we are not growing, we are not competitive, our citizens are not guaranteed at best," stressed the President of the European Parliament, Antonio Tajani, recalling that "200 million euros have been invested since 1984 ". More than 45,000 researchers participated in the European Horizon 2020 program, which has funded more than 18,000 projects. The Commission has proposed to increase the budget of the program, which will now be called "Horizon Europe", but according to Tajani, this is not enough: "The Parliament's proposal is to increase the current resources by 80 billion to 120 billion euros, "insisted on the need for a" political budget that reflects the priorities of citizens.If Europe has the courage to become a political guide again, it will also have the courage to invest more in crucial sectors to become a protagonist of the world again, but also to protect the quality of life of half a million Europeans ".

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