Florida Teen Leads New Gene Therapy Transplant for Sickle Cell at Holtz children‘s Hospital
USA: Nineteen-year-old Kaden Major has emerged from relentless daily pain after receiving a gene therapy–based transplant at Holtz children‘s Hospital that physicians say has effectively corrected his sickle cell disease. Born with the inherited blood disorder, Kaden is described by clinicians as essentially cured following a procedure performed after the FDA authorized the therapy for pediatric use in late 2023. He is one of the first pediatric patients in Florida to undergo the treatment.
The intervention combined intensive preparatory chemotherapy with a period of isolation while Kaden’s own blood-forming cells were genetically edited and then returned to him, a form of autologous cell transplantation. Doctors emphasize that, unlike traditional bone marrow transplants that rely on donors and carry significant risks, this approach manipulates a patient’s own cells to repair the underlying genetic defect. Medical staff called the shift in technique a major change from earlier donor-dependent options.
The impact on daily life has been dramatic. Kaden’s parents — both of whom have sickle cell anemia — recount a son who once lived in constant pain and frequent hospital crises, and who now experiences no pain, no fatigue, and a restored ability to be active and social. The treatment required Kaden to pause his schooling, but he hopes to resume studies, attend college, and pursue a career in the medical field inspired by his recovery. His family sought to publicize the case to raise awareness that the therapy exists as an option for eligible youngsters.
Physicians and the family stress that access remains the pressing issue. The FDA approval covers children 12 and older, and doctors are hopeful about expanding eligibility to younger patients, but the high cost and uneven insurance coverage present immediate barriers. The team and Kaden’s family are advocating for broader availability so more children with sickle cell disease can benefit from this transplantation-based gene therapy.
