![]() ![]() David died in 1984 after an unsuccessful bone marrow. “These foamy virus vectors, because of the way they behave biologically, are proposed to be safer than the previous gene therapy vectors used in human patients,” says Felsburg.īy the end of the five-year project, Felsburg and his research partners hope to have enough data from trials in dogs and mice to win approval for clinical trials of the foamy virus therapy in humans. The movie is based on the real-life story of David Vetter, a SCID patient who literally lived in a plastic bubble for 12 years to avoid germs. With the new funding, Felsburg and the other researchers will test a new approach, using a type of retrovirus called a foamy virus, to deliver a “good” copy of the SCID-affected gene into patients. While previous gene therapy trials have advanced to the stage of human clinical trials, problems have emerged. “The dog is the mirror image of what happens in humans with the disease,” he says. Though other researchers have created a mouse model of XSCID, Felsburg says the immunologic defect in the mice differs from that of the human and dog, and dogs have remained a superior model species for evaluating the efficacy of treatments. Studies of these dogs-dubbed “bubble bassets,” although beagles are now the main breed used-have led to a few different approaches to treatment, using bone marrow transplants and various types of gene therapy, where the dogs receive healthy copies of the defective gene. ![]() To better comprehend the underlying cause of the disease and to test possible treatments, Felsburg and colleagues developed a model of X-linked SCID in basset hounds. In addition, many newborns contract an infection before the disease is even diagnosed. Bone marrow transplants can save their lives, but the transplants require a genetically matched donor, usually a sibling. Untreated, baby boys often die before the age of 2. In these patients, the B and T cells-important players in fighting disease-are low in number and function poorly, rendering babies helpless to infection. X-linked SCID affects only boys, impacting one of every 30,000 newborns. A new funding award of $12 million over the next five years from the National Institutes of Health will enable Felsburg, along with colleagues at the Fred Hutchison Cancer Research Center and several other institutions, to focus on a novel type of gene therapy that might safely treat the condition. Peter Felsburg, Trustee Professor of Immunology at Penn’s School of Veterinary Medicine, has been studying the most common form of the disease, X-linked SCID, for more than 25 years. David Vetter, the boy who lived in a plastic bubble until his death at the age of 12, suffered from a disease known as Severe Combined Immunodeficiency, or SCID. Now, a gene therapy advance described in a small New England Journal of Medicine study seems to have safely produced a robust immune response in eight young patients, offering families and doctors new hope.Made famous by the television show “Seinfeld,” the real “bubble boy” was no joke. Severe combined immunodeficiency (SCID), also known as the bubble boy disease, is a rare genetic disease that affects people around the world. Still, previous gene therapy techniques either did not fully restore immune function or resulted in side effects as serious as leukemia. Such donations can improve patients’ health, but they often can’t produce full immunity, the NIH says - which is why gene therapy has seemed so promising. But since many patients do not have a sibling who’s a match, doctors often resort to other donors. Until now, the best treatment for these difficult cases relied on using bone marrow transplants from tissue-matched siblings to restore immunity. In one famous case, a boy with SCID, David Vetter, lived in a sterile plastic “bubble” until his death at age 12 - hence the disease’s nickname. The condition, which strikes up to one in 50,000 newborns, primarily affects boys and requires extreme measures to prevent infection. Researchers used an experimental gene therapy to develop a possible treatment for a rare and deadly immune disorder known as “bubble boy” disease, the National Institutes of Health (NIH) announced Wednesday.īecause of a gene mutation, babies who are born with X-linked severe combined immunodeficiency (X-SCID) do not develop immune cells properly, leaving them highly susceptible to infections. ![]()
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