Stem cells were harvested from a mother and successfully transplanted into her growing fetus by researchers at the University of California San Francisco (UCSF). The procedure led to the birth of a healthy infant who would have otherwise carried a fatal condition, alpha thalassemia. This is the world’s first clinical trial in which blood stem cells were transplanted before delivery.
The scientists will continue to assess whether the cells will take root in the baby’s bone marrow and continue to create healthy blood cells throughout the life of the child. First, the team had to evaluate the safety of their process.
“We are encouraged by how well she and her mother have tolerated this complex treatment,” said Tippi MacKenzie, MD, a pediatric and fetal surgeon at UCSF Benioff Children’s Hospital San Francisco
The prognosis is poor for fetuses with alpha thalassemia, and there is a low chance of a successful birth. “Her birth suggests that fetal therapy, including fetal transfusions, is a viable option to offer to families with this diagnosis,” MacKenzie said.