INDIANAPOLIS (WISH) — Elin Lewis, a 12-year-old girl from Greenwood, has made history as the first patient in the state to receive a groundbreaking, FDA-approved gene therapy for transfusion-dependent beta-thalassemia, a severe inherited blood disorder that affects approximately 1,500 people in the United States.
Riley Hospital for Children is the first and only health system in Indiana to offer this therapy, and only a small number of health systems nationwide offer the treatment.
Since the age of three, Elin’s life has been defined by receiving monthly treatments that were vital to maintain her hemoglobin levels and organ function, yet they carried the persistent risk of dangerous iron overload, a side effect that can lead to chronic fatigue and weakness to life-threatening complications, including liver damage and heart failure.
The novel therapy uses a a patient’s own blood stem cells, which are collected, genetically modified in a laboratory to include a functional copy of the beta-globin gene, and then infused back into the patient. Once reintroduced, the modified cells engraft in the bone marrow and produce healthy red blood cells with functional hemoglobin…