Pioneering Gene Therapy Freed Her of Sickle Cell. Is a Cure at Hand?

Such treatments are extraordinarily promising and costly. Will the Biden administration commit to spending that could speed clinical trial results?

MESA, Ariz. — Helen Obando, 18 and soon to be a high school senior, nibbled french fries at a cafe and chatted about her future. Dressed in a halter top and shorts, her toenails painted turquoise, she described her plans to enroll in community college, teach hip-hop dance and become a trauma nurse someday. For most of her life, such modest dreams would have been unattainable. Helen was born with sickle cell disease. Since babyhood, she had experienced episodes of searing pain, hospitalization and organ damage, and would have been expected to die in her 40s. But in 2019 she became the first American teenager with sickle cell to be declared free of the disease six months after undergoing an experimental gene therapy meant to cure her. Her symptoms have vanished. She assures teenagers she met at a camp for children with sickle cell: “One day they can have what I had.”