An experimental gene therapy has cured eight infants with the so-called Bubble Boy Disease, an immune-system deficiency so severe that children with it were at one time kept in total isolation for fear that even a simple common cold could be deadly.
In a major scientific breakthrough, researchers at St. Jude Children’s Research Hospital in Memphis have developed a one-time, personalized treatment used to correct the genetic defect and build fully functioning immune systems in infants with the condition, formally known as X-linked severe combined immunodeficiency, or SCID.
The boys, all toddlers now, have been released from the protective isolation units that dominated their early lives and are able to play like other children their age, said Ewelina Mamcarz, an assistant member of St. Jude’s department of bone marrow transplantation.
“We believe that the patients are cured,” Mamcarz said. “They’re living normal lives, and they have normal, functional immune systems that aren’t any different from yours and mine. They are home, some have started daycare, and they are making antibodies in response to vaccines just like we all do.”
The results involving the first eight boys to receive the treatment, to be published in the April 18 New England Journal of Medicine, are part of a new wave of experimental gene therapies that holds out the promise of cures for an array of inherited diseases, including rare conditions like spinal muscular atrophy and more common ailments such as sickle cell anemia and hemophilia. The aim is to correct defects causing disease at the most basic level by giving patients a functioning copy of a previously missing or defective gene that will live and work indefinitely inside their bodies.
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