Angie Rojas's cells are about to be returned to her. Taken from her bone marrow five days earlier, genetically altered and nourished in the lab, the cells nestle in the tip of a syringe, about 45 million of them, a pale nib barely visible in the liquid. When the doctor nods and the nurse starts the "push," the cells trickle through an IV lock into the teenager's bloodstream. It is September 1, 2001, and gene therapy is mounting another try.
Patients with a form of severe combined immune deficiency disease (ADA-SCID) have a defect in a gene that is crucial to immune function. The diagram above outlines a recent gene therapy trial that could correct the condition: (1) extract defective cells from the bone marrow, (2) insert a virus bearing a healthy gene into the cells, and (3) inject the altered cells into the patient.
José Rojas gets up from his seat at the window, eager to keep his daughter's face in view. He cranes his head around the monitor at the foot of the bed. Lucy, her mother, stays seated, placid as usual. She has been with Angie in hospital rooms more times than she can count. This time, she hopes, the doctors will arrest the severe immune disorder that has stalked her daughter's life. She knows the treatment is experimental. Pressing close to Lucy is a young cousin, Denise, whom Angie has invited to the hospital. The girl looks around wide-eyed, not sure how to react.
The air outside the window at Childrens Hospital Los Angeles is bright and hot. Palm trees dot the steep hillside. From where the relatives sit, the famous "Hollywood" sign can be seen, large as life. Although the room is full of witnesses besides the family to this unfolding medical drama, there is no buzz or chatter, no lights or cameras. A sense of anticipation is mixed with anticlimax, as if this were a rerun of a movie that wasn't that great to begin with.
