In the first trial, in 1993, they used cord blood, treating it with the healthy ADA gene and hoping enough of them would “take” to rebuild an immune system. His group has been researching the best way to treat SCID with gene therapy for more than two decades. The success was a long time coming for Kohn as well. Those words…besides the birth of my children, that day will always be the best day in my life.” Kohn came in and told me, ‘It worked.’ It worked. “After the transplant of this miraculous tube of stem cells, which literally took five minutes, we had to just wait and see for a good six weeks,” says Padilla-Vaccaro. MORE: Woman Receives First Stem Cell Therapy Using Her Own Skin Cells And because they were made from her own cells, her body wouldn’t reject them. The idea was that by transplanting these healthy ADA-containing cells back into Evangelina, she would soon be making her own healthy immune cells. Kohn treated them with gene therapy, co-opting a modified virus to carry the healthy ADA gene so it could infect the stem cells from Evangelina’s bone marrow. It contained the stem cells that go on to develop into all of the cells in the blood and immune systems. When she was two months old, Evangelina was admitted to UCLA and had bone marrow drawn from her tiny hip. “We would be trying to fix one problem and getting another,” she says. Padilla-Vaccaro and her husband, Christian, were considering unrelated donors but were concerned about the risk of rejection. But despite being her twin, Annabella wasn’t a blood match for her sister, nor were her parents. The only treatments for SCID are bone marrow transplants from healthy people, ideally a matched sibling the unaffected cells can then repopulate the immune system of the baby with SCID. Now with a newly-restored immune system, Evangelina lives a normal and healthy life. Christian and Alysia Padilla-Vaccaro and their healthy twins Annabella (left) and Evangelina. The Vaccaros decided to treat Annabella in the same way that they cared for Evangelina “They were crawling and playing with each other, and every toy they sucked on, they stuck in each other’s hands and each other’s mouth, so we couldn’t take one outside to have a grand old time and potentially bring something back that could harm her sister,” says Padilla-Vaccaro. SCID is caused by a genetic mutation in the ADA gene, which normally produces the white blood cells that are the front lines of the body’s defense against bacteria and viruses. MORE: Gene-Therapy Trial Shows Promise Fighting ‘Bubble Boy’ Syndrome And they took showers and changed clothes as soon as they entered the house. We couldn’t take them outside to take a breath of fresh air, because there is fungus in the air, and that could kill her.”īoth parents wore masks at work to lower the chances they would be exposed to germs that they might bring back home. “Our children did not physically see our mouths until then because we were masked all the time. Until Evangelina and her sister Annabella were 11 months old, “We were gowned and masked and did not go outside,” says their mother Alysia Padilla-Vaccaro. Known to doctors as adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), it’s better known as “bubble boy” disease, since children born with the genetic disorder have immune systems so weak that they need to stay in relatively clean and germ-free environments. Donald Kohn, says that the strategy could also be used to treat other gene-based disorders such as sickle cell anemia. She’s one of 18 children who have been treated with the cutting-edge therapy, and the study’s leader, Dr. But doctors at University of California Los Angeles (UCLA) Broad Stem Cell Research Center gave her a new treatment, using her own stem cells, that has essentially cured her disease. Evangelina, now two, is alive today because she saved herself with her own bone marrow cells.Įvangelina, a twin, was born with a severe immune disorder caused by a genetic aberration that makes her vulnerable to any and all bacteria and viruses even a simple cold could be fatal. Alysia Padilla-Vaccaro and Christian Vaccaro owe their daughter’s life to stem cells.
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