![]() ![]() Over time, his doctors hoped, there would be enough healthy blood stem cells to make Agustín an ample supply of T-cells. When he arrived at Children’s, Agustín’s T-cell count was only 5, far too low to protect him. With luck, the treated cells would turn on the new gene, enabling them to make T-cells. “We are fortunate to have the technical expertise of Dana-Farber’s CMCF and the superb nursing and clinical research staff of Children’s,” said Williams. A few days later, the treated cells were reinfused back into Agustín. The gene was delivered by a specially engineered virus, known as a vector, that infects the cell without spreading in the body. His bone marrow was extracted and purified and the blood stem cells manipulated in Dana Farber’s Connell and O’Reilly Families Cell Manipulation Core Facility (CMCF) to carry the new replacement gene. In December, Agustín underwent the gene therapy procedure. “Someone heard our story and offered us a place to stay.” (Later, the family moved to a Ronald McDonald house.) “We called a radio station in Jamaica Plain that plays tango music, and told our story,” Alberto said. He was admitted for 23 days to Children’s Hematopoietic Stem Cell Transplant Unit. “There is also no time delay in finding a donor and no need for chemotherapy.”Īgustín arrived at Children’s last October with a mouth full of ulcers. ![]() “The main advantage of gene therapy is that patients would receive their own cells, so there is no chance of graft-versus-host disease,” explained Pai, of Children’s Division of Hematology/Oncology. The SCID-X gene therapy trial is one of several now in the pipeline. The Children’s gene-therapy investigators, L-R: Luigi Notarangelo, David Williams, Sung-Yun Pai. And with any bone marrow transplant, there’s a risk of graft-versus-host disease – the donor cells could launch a potentially fatal attack on Agustín himself. His chances could be improved by first giving him chemotherapy to kill the genetically defective cells in his bone marrow, but at the cost of highly toxic side effects. “It was our best possibility,” said Marcela through a translator.Īgustín’s only other option was a bone marrow transplant from a partially matched donor, perhaps one of his parents. participant in the international trial, led by David Williams, chief of Hematology/Oncology at Children’s. Oleastro sent them a sample of Agustín’s blood, and Agustín was accepted as the first U.S. He had met Luigi Notarangelo and Sung-Yun Pai, the trial’s principal investigators at Children’s, at a medical conference. Without intervention, Agustín was likely to die from a bacterial or viral infection before his first birthday.Īgustín’s immunologist in Buenos Aires, Matías Oleastro, heard of a clinical trial recruiting at Children’s Hospital Boston that was attempting to cure SCID-X1 with gene therapy. Finding a matched donor was a daunting problem, and time was limited. But when Agustín was born, their tissue types turned out to be incompatible. When Jeremías was born healthy, the family banked stem cells from his umbilical cord blood to benefit future siblings. The Cáceres’s first son had died from SCID-X1 at the age of 5 months. When Agustín’s relatives came to help out, they had to change their clothes and wash their hands, and couldn’t enter Agustín’s room.Īgustín, born in Argentina, has a form of X-linked Severe Combined Immunodeficiency, or SCID-X1, better known as “ bubble boy disease.” It affects only boys, leaving their bone marrow unable to make T-lymphocytes, the white blood cells that fight infection. Jeremías had to stop attending nursery school, for fear he’d bring home an infection his baby brother might catch. His father Alberto, and his four-year-old brother Jeremías, kept to a separate bedroom. Everyone had to wear masks, gloves and gowns.Īfter that, he went into isolation, along with his mother Marcela, who came out only for meals. Until this month, Agustín Cáceres’s baptism was the only time his family could come close to him. The first U.S.-treated patient with his parents. ![]()
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