Amy Norton HealthDay Reporter Posted: Thursday, October 9, 2014, 5:00 AM <\/p>\n<\/p>\n
(HealthDay News) -- A new form of gene therapy may offer a safe and effective way to treat \"bubble boy\" disease -- a severe immune deficiency that is fatal unless treated in infancy. <\/p>\n
Researchers have long known that gene therapy can cure the disease, known medically as severe combined immunodeficiency, or SCID. Over a decade ago, trials in Europe showed that gene therapy worked -- but five of the 20 children treated developed leukemia (a type of cancer) within two to five years, according to background information in the study. <\/p>\n
In the new trial, reported in the Oct. 9 New England Journal of Medicine, researchers refined the gene therapy approach to hopefully negate the leukemia risk. <\/p>\n
Eight of nine children who received the therapy are still alive one to three years later, the investigators report. And so far, none has developed leukemia. <\/p>\n
It's too early to say the therapy carries no leukemia risk, cautioned researcher Dr. David Williams, a pediatric hematologist\/oncologist at Dana-Farber Cancer Institute and Boston Children's Hospital. <\/p>\n
\"We'll continue to follow these children for 15 years,\" Williams said. <\/p>\n
But based on the early results, he noted, the tweaked gene therapy appears as effective at generating a functional immune system as the earlier form of treatment. <\/p>\n
SCID refers to a group of rare genetic disorders that all but eliminate the immune system, according to the Immune Deficiency Foundation (IDF). That leaves children at high risk of severe infections. <\/p>\n
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Gene Therapy Shows Potential for 'Bubble Boy' Disease<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" Amy Norton HealthDay Reporter Posted: Thursday, October 9, 2014, 5:00 AM (HealthDay News) -- A new form of gene therapy may offer a safe and effective way to treat \"bubble boy\" disease -- a severe immune deficiency that is fatal unless treated in infancy. Continue reading