A Phase I clinical trial to test the efficacy of genetically modified autologous (the patient’s own) stem cells to treat beta-thalassemia has been initiated. This condition is an inherited disease that affects the production of hemoglobin, which is responsible for carrying oxygen in the body and delivering it to tissues and vital organs. With thousands of new cases every year, this condition often results in fatigue, bone fragility and extreme anemia (a deficiency of iron in the blood). This trial aims to create a groundbreaking protocol that would obtain autologous stem cells from the patients, genetically alter them to produce the missing protein responsible for the condition and, reintroduce the stem cells back into the body through a transfusion.
Gene therapies utilizing stem cells are being developed that may lead to a cure for several genetic diseases. Currently, two clinical trials were announced to treat Cerebral Adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell disease.