This July 2019 image provided by the Sarah Cannon Research Institute shows Victoria Gray on her infusion day during a gene editing trial for sickle cell disease at the Sarah Cannon Research Institute and The Children’s Hospital At TriStar Centennial in Nashville, Tenn. Ms. Gray, the first patient to test the treatment, shared her experience with researchers at a scientific conference in 2023. She described suffering with terrible bouts of pain since childhood and receiving high-dose pain medications and sometimes blood transfusions. She described feeling she “was being reborn” the day she got the gene therapy.
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Eureka!
FDA approves milestone treatments for sickle cell disease
Two breakthrough gene therapies can now be used to treat and possibly cure sickle cell anemia, the genetic blood disorder that afflicts 100,000 mostly Black Americans and 20 million people worldwide. But the announcement from the Food and Drug Administration of approval of the treatments — the first use of medicines to address an inherited disease — drew cheers and caution flags from those in the field.
