Thursday, June 20

First Patient Begins Sickle Cell Gene Therapy That F.D.A. Approved – Generic English

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Last week, Kendric came prepared for the stem cell collection — he has spent many weeks in this hospital being treated for pain so severe that on his last visit, even morphine and oxycodone could not control it. He brought his special pillow with a Snoopy pillowcase that his grandmother gave him and his special Spider-Man blanket. And he had a goal.

“I want to be cured,” he said.

Bone marrow stem cells, the source of all the body’s red and white blood cells, are normally nestled in a person’s bone marrow. But Kendric’s doctors infused him with a drug, plerixafor, which pried them loose and let them float in his circulatory system.

To isolate the stem cells, staff members at the hospital inserted a catheter into a vein in Kendric’s chest and attached it to an apheresis machine, a boxlike device next to his hospital bed. It spins blood, separating it into layers — a plasma layer, a red cell layer and a stem cell layer.

Once enough stem cells have been gathered, they will be sent to Bluebird’s lab in Allendale, N.J., where technicians will add a healthy hemoglobin gene to correct the mutated ones that are causing his sickle cell disease.

They will send the modified cells back three months later. The goal is to give Kendric red blood cells that will not turn into fragile crescent shapes and get caught in his blood vessels and organs.

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