Until this month, Agustín Cáceres’s baptism was the only time the boy’s family could come close to him. Everyone had to wear masks, gloves and gowns. Agustín, born in Argentina, has a form of X-linked Severe Combined Immunodeficiency, or SCID-X1, better known as “bubble boy disease.” The condition affects only boys, leaving their bone marrow unable to make T-lymphocytes, the white blood cells that fight infection.
Agustín’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. He had met the trial’s principal investigators at Children’s, Luigi Notarangelo and Sung-Yun Pai, at a medical conference.
Oleastro sent them a sample of Agustín’s blood, and Agustín was accepted as the first U.S. participant in the international trial, led by David Williams, chief of Hematology/Oncology at Children’s. “It was our best possibility,” said his mother, Marcela, through a translator.
Read the full story at Children’s Hospital’s Vector blog.
