Gene therapy offered this 7-year-old freedom. The price: a grueling year

Dec. 13, 2023

Shelby Campbell at home in Mechanicsburg, Pa., in late August. Amanda Berg for STAT

PHILADELPHIA — The meds Shelby Campbell needed for her rare blood disorder stopped working just after her sixth birthday. She lost her appetite and was often doubled over in pain. She continued getting blood transfusions but her doctors struggled to manage side effects that threatened her organs. By the time she turned 7, the doctors told her parents they had to do something — soon.

Several months later, Shelby laid in her mother’s arms on a hospital bed, napping. Her medical team — who deemed the late June 2023 day Shelby’s “re-birthday” — gathered around, preparing to administer a life-changing treatment. Her doctor, Tim Olson, connected the first of two syringes to a small tube that led to a port in Shelby’s chest. Both syringes were filled with a translucent, slightly beige liquid. Everyone in the room, holding their breaths, knew it was worth more than if it were liquid gold: $2.8 million, to be exact.

In a stroke of luck, or divine providence as Shelby’s parents see it, this gene therapy for her disease, beta thalassemia, had been approved just the year before. If all went well, it would replace Shelby’s deficient stem cells with ones that produce red blood cells correctly, and Shelby would hopefully never need transfusions again, like nearly 90% of patients in the pivotal clinical trial for the therapy.

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