Skip to content

    Children's Health

    Font Size
    A
    A
    A

    Gene Therapy Works for 'Bubble Boy' Disease

    9 Years Later, 14 Out of 16 Kids With the Rare Disease Leading Normal Lives

    How the Children Fared continued...

    The 16 children with SCID who got the gene therapy ranged in age from 6 months to 3 years. Four of them had the ADA-deficiency type of SCID. The other kids had the X1 form of SCID. Those are the two most common types of SCID.

    For most of the children, gene therapy was a success. But one boy who had the X1 form of SCID developed treatment-related leukemia. The complication was not unexpected, Gaspar says, because four children with the X1 from of SCID in a French study had developed leukemia after getting the gene therapy.

    Gaspar says researchers learned from those cases and have modified the treatment in hopes of reducing the risk for patients with the X1 form of the disorder.

    Kohn says gene therapy should be considered the treatment of choice for children with ADA-deficient SCID who do not have perfect bone marrow donor matches. It may prove to be a better choice for patients with perfect donor matches, too, he says.

    As for the X1 form of the disease, Kohn says it remains to be seen if the new approach to gene delivery works and has less risk of leukemia.

    Lessons learned from the SCID trials have spurred studies to find effective gene-based treatments for other blood cell diseases, including sickle cell anemia, Kohn notes.

    “The history of gene therapy research can be summarized as, 'Two steps forward and one step back.' We retrench, we learn, and then we move forward again,” he says.

    “Twenty years ago, nothing was working," Kohn says. "Ten years ago, these treatments started to work, but with complications. The hope is that the next decade will bring highly effective treatments with few complications.”

    1 | 2

    Today on WebMD

    child with red rash on cheeks
    What’s that rash?
    plate of fruit and veggies
    How healthy is your child’s diet?
     
    smiling baby
    Treating diarrhea, fever and more.
    Middle school band practice
    Understanding your child’s changing body.
     

    worried kid
    fitArticle
    jennifer aniston
    Slideshow
     
    Measles virus
    Article
    sick child
    Slideshow
     

    babyapp
    New
    Child with adhd
    Slideshow
     
    rl with friends
    fitSlideshow
    Syringes and graph illustration
    Tool