Tippi MacKenzie, MD, a pediatric and fetal surgeon at UCSF Benioff Children’s Hospital San Francisco, is conducting the world’s first clinical trial using blood stem cells transplanted prior to birth. By transplanting cells from the mother, this technique could offer a way to bypass rejection by the fetal immune system. Her work is aimed at treating alpha thalassemia, a normally fatal fetal condition.

The clinical trial brings together the unique expertise of both UCSF Benioff Children’s Hospitals. The UCSF Fetal Treatment Center, in San Francisco, is the birthplace of fetal surgery and continues to offer life-saving interventions for fetuses with multiple birth defects. UCSF Benioff Children’s Hospital Oakland established its thalassemia program in 1991 to provide multidisciplinary care to the growing number of thalassemia patients.

Vichinsky, who oversaw the treatment for the first baby born after receiving an in utero stem cell transplant in the clinical trial, said future plans may include using in utero stem cells to treat beta thalassemia, the more common type of the disease, as well as sickle cell anemia and other serious and life-threatening genetic disorders.

“Once-fatal blood diseases can now be managed as chronic diseases,” Vichinsky said. “In utero stem-cell transplantation may take it one step further: to successfully treat blood diseases before birth.”