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Gene Therapy could be Safe and Effective in Treating Bubble Boy Syndrome

Update Date: Oct 09, 2014 03:44 PM EDT

According to the early results from a clinical trial, a form of gene therapy might be an effective treatment option for patients with severe combined immune deficiency (SCID). SCID, more commonly known as Bubble Boy syndrome, is a condition that occurs when the immune system is compromised and becomes extremely vulnerable to infections. The disease can be fatal if it is left untreated during infancy.

The trial enrolled nine infant boys diagnosed with X-linked SCID who had serious infections at the time of the therapy. One of them passed away. X-SCID is caused by a gene mutation known as IL2G. Gene therapy involves replacing that mutation with a healthy version of it. The cells then go on to create normal immune-system cells that can protect the body from infections.

So far, eight of the boys are still alive 16 to 43 months post treatment. Unlike earlier trials involving gene therapy, none of these patients have developed leukemia. However, the team stated that the cancer risk was not eliminated. In the previous SCID trials, five out of the 20 patients went on to develop leukemia. The patients who survived were considered cured of the disease.

"The early results are encouraging, but this needs more time to mature," commented Dr. Richard O'Reilly, chief of pediatric bone marrow transplant at Memorial Sloan-Kettering Cancer Center in New York City, who was not a part of the trial. "Many of the children had significant infections, and would be at high risk of [dying] with a stem cell transplant.

"We'll continue to follow these children for 15 years," researcher Dr. David Williams, a pediatric hematologist/oncologist at Dana-Farber Cancer Institute and Boston Children's Hospital, said according to Philly.

The team wants to enroll more patients with a target of 20 boys. The trials' results were published in the New England Journal of Medicine.

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