Published on in CHOP News
A team of researchers at The Children’s Hospital of Philadelphia’s Center for Fetal Research is seeking to cure sickle cell disease before birth by performing stem-cell transplants in utero.
Alan Flake, MD
Stem-cell transplantation can make a big difference in the lives of patients suffering from sickle-cell disease. This latest research, led by Alan Flake, MD, director of the Center for Fetal Research, is seeking to extend the age range of patients that can benefit from this treatment to those in the womb.
Dr. Flake’s team has successfully treated sickle-cell disease in utero in mice and dogs by transplanting stem cells in the fetus. He is now running a trial in monkeys, and hopes to start a clinical trial in human fetal patients within the next two years.
In a procedure guided by ultrasound, Flake and his colleagues take stem cells from the mother and inject them into the bloodstream of the fetus. Because the immune systems of the fetus and the mother are naturally tolerant of one another, no destruction of the fetal immune system is required.