SCID-X1 is an immune system disorder commonly known as 'bubble boy' disease. A new treatment for it is being called a 'cure.'
Dannie Hawkins dreamed for her precious baby nephew, Ja'Ceon Golden, to experience the simple joys of childhood -- swimming in pools during the summer, going to birthday parties, playing in parks filled with people, having a pet -- without risk.
Yet because of his illness, going outdoors and into crowds of people would put him at serious risk of deadly infections, and so Hawkins accepted that as Ja'Ceon's reality -- until now.Kristin Simpson, as any mother would, wished for her baby son, Omarion Jordan, to grow into a healthy boy who could venture outdoors and not live at risk of getting gravely ill. Now, she says, he can.
Mukherjee on the 'revolution' of gene therapy 01:43A team led by researchers at St. Jude Children's Research Hospital in Memphis developed an experimental gene therapy that involves harvesting bone marrow stem cells from a child with SCID-X1, inserting the normal gene that is missing in these patients into those cells, and then infusing them back into the child so the cells repopulate and restore the immune system.Tod Lubitch in the 1976 movie"The Boy in the Plastic Bubble.
'Bubble girl' is allergic to life 04:02The newly developed gene therapy, performed in combination with a chemotherapy called busulfan, was described in a study published in the New England Journal of Medicine on Wednesday. Both Ja'Ceon and Omarion participated in a clinical trial to test the therapy, and it proved successful in treating SCID-X1 in both.
The researchers are investigating how this gene therapy could be used to treat patients of older ages and with other types of disease.For instance,"the same vector that was manufactured here at St. Jude is currently being used at National Institutes of Health to treat older children who had an unsuccessful bone marrow transplant," Mamcarz said.
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