University of California, San Francisco, pediatric surgeons treated a fetus diagnosed with a lethal form of thalassemia, alpha thalassemia major, with stem cells from her mother’s bone marrow. Born in February, the baby was the first patient enrolled in the world’s first clinical trial using stems cell prior to birth.
Every fetus has the perfect donor: mom. Although it is too early to say how effective the stem cell transplant will be, the doctors are encouraged by how well the baby is doing.
Babies with alpha thalassemia typically die before birth or are stillborn. The healthy birth suggests that fetal therapy is a viable option for treating this diagnosis. Once considered fatal, thalassemia can be managed as a chronic disease. In utero stem-cell transplantion may take it a step further, making it a disease that can be successfully treated before birth.
Click here to read the entire article by Jim Daley, published May 30, 2018 on The Scientist.