Rare genetic disease cured thanks to stem cells

Rare genetic disease: the story of Alex, saved by stem cells

The story of Alex, saved by stem cells

“Many times I wondered why me, what had I done wrong to deserve all this suffering and only now have I understood it: I had to witness the success of science, the triumph of solidarity, the primacy of love and victory over a rare genetic disease such as Hemophagocytic Lymphohistiocytosis that wanted to tear me away from all of you.”

These are the words that the parents of baby Alex – Alessandro Maria Montresor, the child who underwent a hematopoietic stem cell transplant from a parent a month ago, shared on their social networks to thank all the people who contributed and supported their family in such a difficult time.

Alex, born in England to Italian parents, was taken to the Bambino Gesù hospital in Rome, where Franco Locatelli’s team, head of the Oncohaematology and Transfusional Medicine department, wanted to try everything for everything. Hopes for the child were placed in the innovative technique of transplantation, which was submitted on December 20, 2018. The technique developed and applied in the Bambino Gesù hospital for the first time in the world in 2014, provides for the manipulation of stem cells from the most compatible parent to avoid the risk of rejection.

The father’s cells, the most compatible among the parents, were manipulated and infused into the child and, explained the hospital, “have perfectly grafted”. The transplantation process “can be said to have been successfully concluded”. In fact, after more than 30 days from transplantation, the cells “are adequately repopulating the hematopoietic and immune system of the patient”.