1st sickle cell gene therapy patient completes treatment

A 12-year-old boy has become the first patient in the U.S. to receive gene therapy for sickle cell disease, The New York Times reported Oct. 21.

Kendric Cromer began treatment with Bluebird bio’s sickle cell gene therapy in May. Physicians removed stem cells from his bone marrow and sent them to a facility where new, healthy hemoglobin genes were added. It took a few months before the treated cells were ready for administration.

On Sept. 3, Kendric was admitted to Washington, D.C.-based Children’s National Hospital for the final phase of treatment. He underwent intensive chemotherapy to clear his bone marrow, and physicians then infused the modified stem cells back into his body. Kendric stayed in the hospital for 44 days before being released. He must redo his childhood vaccinations and avoid crowds while his immune system remains compromised, but he is expected to return to normal life soon.

Bluebird bio’s $3.1 million gene therapy was approved by the FDA last December, followed by Vertex Pharmaceuticals’ $2.2 million therapy, approved in May. These treatments are available to patients if covered by insurance. Kendric is Bluebird’s first commercial sickle cell gene therapy patient.