For babies born with the severe genetic condition known as “bubble boy” disease, a run-of-the-mill common cold can be deadly.
Born without crucial disease-fighting immune cells, they must be kept isolated from the outside world for their own protection. Months in the hospital and being treated for severe infections is the norm. Without treatment, most born with the disease die as infants.
A new experimental medicine is now being called a cure for the condition by researchers at St. Jude Children’s Research Hospital. Ten babies born with the genetic disease, X-linked severe combined immunodeficiency (XSCID), have been successfully treated, with no apparent side effects, the researchers said on Wednesday.
The kids are now making their own immune cells. Nearly all have been able to go home with their families and live normal lives, including attending day care, with one more recently treated child remaining at St. Jude for the time being.
“This is a first for patients with XSCID,” said Dr. Ewelina Mamcarz, of the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy. Mamcarz is the first author on a paper about the results, which reports on the first eight children to get the treatment and is being