Parents and Children’s Hospital researchers await results on an experimental leukemia gene therapy

Marie McCullough, Inquirer Staff Writer Posted: Monday, March 25, 2013, 3:01 AM

Aaron and Christal Walker live in dread that their daughter will get sick, and in dread that she won't.

Six days ago, Avrey Walker, 9, of Redmond, Ore., became the seventh child to receive an experimental gene therapy for leukemia at Children's Hospital of Philadelphia.

She will soon suffer several days of fevers, nausea, headaches, maybe worse - if the therapy works as it should, marshalling her immune T cells to fight her cancer.

Four of the first five children to undergo treatment and get lab results are cancer-free, according to their families and doctors.

"The doctors said it would take seven to 10 days" for the flulike reaction to begin, Avrey's father said last weekend. "So we're just waiting and watching intently."

The medical and human drama of the T-cell therapy, developed at the University of Pennsylvania, is unfolding in ways the defy the staid traditions of scientific research. On Monday, the New England Journal of Medicine fast-tracked online publication of a paper about Children's first two pediatric patients. But those results - and more - have been out for months, released by the researchers at a conference, or by the families.

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Parents and Children's Hospital researchers await results on an experimental leukemia gene therapy

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