![]() The modified vector created for the current trial is a self-inactivating gammaretrovirus, designed to deliver its payload effectively while minimizing the chance of inadvertently turning on genes, called oncogenes, which could lead to leukemia. ![]() This outcome was one of several events that together slowed clinical progress in gene therapy for many years. In the prior European trials - which were the first to demonstrate gene therapy’s potential to cure a disease - leukemia appeared two to five years after treatment. ![]() The investigators will continue to monitor the patients for any signs of treatment-related leukemia for 15 years. Left untreated, boys with SCID-X1 usually die of infection before their first birthday. One child died of an overwhelming infection present at the time gene therapy began. Genetic studies of the boys’ new T-cells, which are critical components of the body’s immune system, reveal that the viral vector used to deliver the gene therapy did not lead to an expansion of cells with vector insertions near known cancer-causing genes, raising cautious hopes about the vector’s long-term safety. Gene therapy alone generated functioning immune systems in seven of the eight. A new form of gene therapy for boys with the life-threatening condition known as “bubble boy” disease appears to be both effective and safe, according to a collaborative research team Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and other institutions conducting an international clinical trial.Įarly data suggest that the therapy may help patients avoid the late-developing leukemia seen in a quarter of those with the disease in pioneering gene therapy trials in Europe more than a decade ago.Įight of nine boys recruited to date into the trial are alive between 12 and 38 months after treatment, with none of the infections associated with the disease, more formally known as X-linked severe combined immunodeficiency syndrome (SCID-X1), the research team reported Thursday in the New England Journal of Medicine (NEJM).
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