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Stem cells successfully repaired: baby Yanis appears to be the first in the world to be cured thanks to new gene therapy in Leiden
Yanis was born without an immune system. A simple infection could be fatal. At the LUMC, a medical study had just started with a new gene therapy and he was able to participate. Now Yanis’ genes have been repaired. He is the first in the world.
“When the doctor came in, I saw on his face that it was not well. I knew he was sick,” says Saliha Lakbich from Utrecht. Her son Yanis (1) turned out to have a rare birth defect: Rag 1-SCID. As a result, he has no immune system.
Lost son before
Normally this would have been discovered during the heel prick, but Saliha and her husband already knew it was in the genes. Ten years ago they lost a son with the same condition.
Immediately after his birth, Yanis was taken to an isolation room in the Leiden University Medical Center (LUMC). “That was very difficult and heavy,” says Saliha. “Fortunately, we as parents were allowed to be with him.”
No suitable donor
In another case, Yanis would be treated with a stem cell transplant, in which he would receive stem cells from a suitable donor. But there was no suitable one. The family was lucky: research had just started at the LUMC with a new gene therapy and Yanis was able to participate. His parents didn’t hesitate for a moment: “When the doctor told us about the new gene therapy, we immediately agreed. I can’t describe that feeling of joy. Yes, a little bit of joy and also a bit of fear.”
The joy of the parents was not premature. It is now 10 months after the treatment and Yanis now has a well-functioning immune system. “He plays, he moves, if you ask him something, he will tell you,” says father Aachour proudly.
At least as proud are Leiden researchers Frank Staal and Arjan Lankester. Yanis is the first patient in the world to be treated with their new gene therapy. “We have worked on it for a long time, more than 15 years. And when it finally comes to the clinic and turns out to work, that is very special. And we are very proud of that,” says immunologist-stem cell biologist Frank Staal.
Colleague and professor of pediatrics and stem cell transplantation, Arjan Lankester, is also beaming. “You rarely experience such a milestone.”
Stem cells repaired
He explains how the new gene therapy works: “What we did at Yanis is we repaired its own stem cells in the lab, turning them back into healthy stem cells,” he explains.
“These stem cells can then make a healthy immune system again. So Yanis has been his own donor and that now appears to work very well.”
–Researchers Arjan Lankester and Frank Staal, who developed the new gene therapy
Traveling cells
Yanis and his parents had to travel from Utrecht to Leiden for the gene therapy. Arjan Lankester says something has been devised for patients from other countries: “We have now said: we don’t actually want the patient to travel, but we want the cells to travel.”
They are therefore setting up a consortium in which children in Europe can have the cells taken by the doctors in their trusted expertise center in, for example, Germany or Spain. “These are sent to our hospital in Leiden. Frank’s group and his people repair those cells and the cells are sent back to the hospital where the patient is being treated.”
Heal, but keep an eye on
Yanis is currently cured of the immune disorder, but is of course still being closely monitored, the researchers emphasize. “If you have this child evaluated by any doctor or pediatrician and you say, ‘Look at Yanis’ immune system,’ the doctor will say it looks completely normal.”
“You can also achieve that with a traditional stem cell transplant treatment, but it is regularly accompanied by all kinds of complications that result from the fact that you use someone else’s cells. And they can be very appropriate, but they always remain. someone else’s cells.”
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Confidence in the future
Over the next 1 to 2 years, Frank Staal and Arjan Lankester want to perform the same treatment on at least five SCID patients and on that basis conclude whether the treatment is effective, safe and therefore better than the current stem cell transplantation treatment.
Saliha and Aachour Lakbich look confidently to the future with Yanis. “I am very happy, happy because Yanis is doing well. He is cured. And I hope that this will also work for other children in the world.”
