![]() ![]() In the experimental treatment, infants received their re-engineered stem cells just 12 days after some of their bone marrow was obtained. Jude’s Department of Bone Marrow Transplantation and Cellular Therapy. “After gene therapy, for example, brain cells do not have a correct copy of the gene,” explained Stephen Gottschalk, who chairs St. It was crucial for the viral vector to not deliver the gene to other kinds of cells-and that’s what the researchers observed. The goal was to insert the gene into stem cells that had come from the patients’ own bone marrow, and those cells would then go on to produce working immune system cells. They engineered this vector to include “insulators” to prevent the vector from disturbing other genes once it integrated into the human genome. They started with a modified HIV vector emptied of the virus and its original contents, and filled it with a normal copy of the IL2RG gene. Jude, experts led by the late Brian Sorrentino, a hematologist and gene therapy researcher, set out to engineer a virus delivery vehicle that wouldn’t have side effects. But when a quarter of the patients in that study developed leukemia, because the modified virus also disrupted the functioning of normal genes, the study was halted and scientists interested in gene therapy for the disorder hit the brakes.Īt St. Nearly 20 years ago, researchers in France reported successfully reconditioning immune systems in SCID-X1 patients using a particular virus to deliver the correct gene to cells. Jude wasn’t the first to try gene therapy for SCID-X1. Simpson enrolled Omarion in the clinical study and Cincinnati Children’s Hospital arranged a private jet to transport her and her son to the research hospital, where they stayed for five months. The strategy: introduce a normal copy of the faulty gene, designated IL2RG, into a patient’s own stem cells, which then go on to produce the immune system components needed to fight infection. Jude Children’s Research Hospital in Memphis, Tennessee, were then developing a bold new procedure. And Omarion, an only child, was not among them.Īs it happened, medical scientists at St. But fewer than 20 percent of patients have had this option. For years, the best treatments for SCID-X1 have been bone marrow or blood stem cell transplantations from a matched sibling donor. Only about 20 to 50 new cases of the SCID-X1 mutation-which accounts for about half of all SCID cases-appear in the United States each year. “I had no idea what would happen to him,” his mother recalled.Īpproximately one in 40,000 to 100,000 infants is born with SCID, according to the Centers for Disease Control and Prevention. As soon as Omarion tested positive for the disorder, an ambulance carried him to Cincinnati Children’s Hospital in nearby Ohio and placed him in isolation, where he remained for the next few months. Such safeguards are necessary because a patient exposed to even the most innocuous germs can acquire infections that turn deadly. Today, technological advances in hospitals provide a kind of bubble, protecting SCID-X1 patients with controlled circulation of filtered air. He died at 12, but his highly publicized life inspired a 1976 TV movie starring John Travolta (Vetter’s personal papers and some of his toys are now part of the collections of the Smithsonian’s National Museum of American History). The “bubble boy” name is a reference to David Vetter, a Texas child born with SCID-X1 in 1971, who lived in a plastic bubble and ventured out in a NASA-designed suit. What they learned was that Omarion was born with a rare genetic disorder called X-linked severe combined immunodeficiency (SCID), better known as the “bubble boy disease.” Caused by a mutated gene on the X chromosome, and almost always limited to males, a baby born with X-linked SCID, or SCID-X1, lacks a working immune system (hence the unusual reaction to vaccination). She took him to the hospital emergency room, where, again, caregivers seemed puzzled by the baby’s bizarre symptoms, which didn’t make any sense until physicians, finally, ordered the right blood test. Then, not long after he received a round of standard infant vaccinations, his scalp was bleeding and covered with green goop, recalled the first-time mother, who was then in her late teens. They said he was allergic to his mother’s milk and told her to stop breastfeeding. Doctors thought he had eczema and cradle cap. Still, within his first couple of months, he was plagued by three different infections that required intravenous treatments. Her son, however, had everything he needed: a nursery full of toys, a crib, a bassinet and a blue octopus blanket. She’d just moved in and hadn’t had time to decorate. His mother, Kristin Simpson, brought her dark-haired newborn home to a mostly empty apartment in Kendallville, about 30 miles to the north. On the morning of April 25, 2018, in Fort Wayne, Indiana, Omarion Jordan came into the world ten-fingers-and-toes perfect. ![]()
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