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

Posted: March 26, 2013 at 10:44 am

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.

The first pediatric patient, Emily Whitehead, 7, of Phillipsburg, Pa., who remains in remission after almost dying, was the subject of worldwide headlines in December.

And last week, the larger story - the harnessing of the immune system to fight cancer after decades of trying - broadened beyond Penn and Children's.

Researchers at Memorial Sloan-Kettering Cancer Center in New York published results from five adult leukemia patients treated with an experimental T-cell therapy much like Penn's. Three of them have been in remission for up to two years; one went into remission but died of a blood clot; and one relapsed and died.

Penn's therapy has worked in adults, too, but those seven patients, who had complete or partial remissions, had a less aggressive form of the disease called chronic myelogenous leukemia.

Sloan-Kettering's results are very impressive, Penn researcher David Porter said.

Penn's team, led by Carl June, will soon collaborate with Sloan-Kettering to see which version of the T-cell therapy works better, Porter said.

The two groups use slightly different viral "vectors" to deliver a therapeutic gene into the T cells. That gene programs the T cells to recognize and kill B cells, the blood component that turns malignant in the leukemias.

"One of the issues is how much does the vector contribute to the patient's response," Porter said. "So we'll trade vectors, then give patients T cells made from both vectors."

Penn is also adapting its T-cell therapy to treat solid tumors such as ovarian cancer. Meanwhile, the early success - although in a small number of patients - is stimulating the field of immunotherapy.

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

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