![]() ![]() ![]() Immediately, he was whisked into a sealed bubble intended as a stopgap measure until a match could be found. Sibling died in infancyīecause the Vetters knew there was a chance David would have the immune condition - a first son had died of SCID in infancy - he was delivered by cesarean section in a sterile operating room at Texas Children’s Hospital. The Vetters hoped their daughter would provide the match. In 1971, the year David was born, the only hope was a bone marrow transplant from a donor whose blood matched perfectly. It afflicts 40 to 50 babies born every year in the United States and is fatal within a year or two without treatment. Vetter said it was “kind of unbelievable it happened to us, plain, ordinary people.” Marveling at his family’s ability to cope, he said it was like “the whole world came crashing down” when David was diagnosed.ĭavid had severe combined immunodeficiency (SCID), an inherited condition in which the patient lacks the white blood cells that fight infection. “Perhaps it was meant to be - that he was the little guy through whom doctors and the world were meant to learn about the immune system.” Vetter said last week in a rare interview about his son. “A lot of kids are alive today because David was here,” David J. Doctors say he contributed enormously to a better understanding of clinical immunology, an understanding that has resulted in better treatment for many diseases involving the immune system.Īt a time when HIV/AIDS was coming onto the scene, David also put diseases of the immune system on people’s radar screen. That quality took a toll on David’s emotional well-being.ĭavid’s medical legacy is less open to debate. While keeping him alive was largely seen as a technological triumph and a valiant effort that gave him and his family 12 years together, some bioethicists argue it was a classic example of doctors promising more than medicine could deliver and creating an unacceptable quality of life. The ethical questions involve David’s role as a sort of living experiment. Today, 25 years to the day since his death, David Phillip Vetter remains one of Houston’s signature stories, his mark still felt in a legacy of vexing ethical questions and medical advances. For 12 years, thanks to news coverage around his birthday, he captured hearts worldwide.” “Most medical stories have a flash-in-the pan quality, but David’s story didn’t go away. “It’s such a great human interest story, how so many people came to care about him,” says James Jones, a former University of Houston historian and author of a forthcoming book on the subject. The researchers say their technique might serve as a template for developing gene therapies for other blood disorders, such as sickle cell disease.The case was truly unique: Never again would a child live a life in such a cocoon. But the new treatment worked to prevent this from happening by including "insulator" genes that essentially block activation of the adjacent genes to prevent them from turning cancerous One concern with gene therapy is that, after inserting a gene into people's DNA, genes that are next to the insertion site may turn cancerous, as happened in prior cases where people developed leukemia. Before this infusion, the patients received a low dose of a chemotherapy drug to help make space in their marrow for the new cells to grow. These cells were then infused back into the patients. ![]() Then, they used the altered version of HIV to insert a working copy of the IL2RG gene into the bone marrow cells. In the new study, the researchers first collected patients' bone marrow. For example, a gene-therapy treatment in the early 2000s resulted in several patients developing leukemia. Some previous attempts to treat SCID-X1 with gene therapy have had serious side effects. He died at age 12, after receiving his transplant. The name "bubble boy disease" comes from the highly publicized case of David Vetter, who was born in 1971 with SCID-X1, and spent most of his life in a plastic bubble while awaiting a bone-marrow transplant, according to CBS. Bone-marrow transplants from unrelated donors are typically less effective and come with greater risks. But fewer than 20% of patients with SCID-X1 have such a donor available, the authors said. The disease can be essentially cured by a bone-marrow transplant from a sibling that is a match in terms of certain immune system proteins. The condition is rare, likely affecting about 1 in 50,000 to 100,000 newborns. SCID-X1 is caused by a mutation in a gene called IL2RG, which is critical for normal immune function, according to the National Institutes of Health. But they will still need to be monitored for a longer period to determine if the treatment is long-lasting and doesn't cause side effects later in life, the researchers said. About 16 months after their treatment, the patients are developing normally and have not experienced serious side effects from the therapy. ![]()
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