Four Year Old Girl Provided Life-Saving Bone Marrow for her Brother

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A little four year old girl named Khloe was a hero to her infant brother by providing bone marrow for him. Kayla Land, mother of the two children posted in an update that they are both doing “great”. Khloe’s infant brother, Colton was diagnosed with severe combined immunodeficiency (SCID) when he was about a week old. Also referred to as “bubble boy” disease. Kayla Land and her husband were hoping that their older daughter would be a match because she was older, and better understood what was going on. But they were lucky enough that one of their daughters could help Colton. After the surgery the doctor had told Kayla Land that they were able to get enough bone marrow from her daughter for two additional transplants. This way they can freeze it and use it for Colton in the future if he needed it. Colton’s doctor further told Good Morning America that Colton was “incredibly lucky.”

The condition is fatal, usually within the first year or two of life, unless infants receive immune-restoring treatments, such as transplants of blood-forming stem cells, gene therapy, or enzyme therapy. More than 80 percent of SCID infants do not have a family history of the condition. However, development of a newborn screening test has made it possible to detect SCID before symptoms appear, helping ensure that affected infants receive life-saving treatments. SCID in infants, makes them appear healthy at birth but they are extremely susceptible to infections. The disorder is caused by mutations in different genes involved in the development and function of immune cells. Standard treatment involves stem cell transplantation, with the ideal donor being a sibling who is a close tissue match. The transplants are considered life-saving, but often only partially restore immunity, according to NIAID.

The NIAID has shared that studies also have shown that gene therapy can be an effective treatment for some types of SCID, including X-linked SCID. In gene therapy, stem cells are obtained from the patient’s bone marrow, the normal gene is inserted into the stem cells using a carrier known as a vector, and the corrected cells are returned to the patient. Early efforts to treat X-linked SCID with gene therapy successfully restored children’s T-cell function, but approximately one-quarter of the children developed leukemia two to five years after treatment. Scientists suspect that the vectors used in these studies activated genes that control cell growth, contributing to leukemia. Newer gene therapy strategies use modified vectors that appear effective and safe. NIAID researchers are using a novel gene therapy approach to successfully treat older children and young adults with X-linked SCID.