After nearly 30 years of research, pediatric and fetal surgeon Alan W. Flake, MD, is on the verge of a potential medical breakthrough: a prenatal cure for sickle cell disease using in utero stem cell transplants.

The procedure Dr. Flake has designed is a series of two bone marrow stem cell transplants from the mother to her child.

The first transplant would occur while the child is still in the womb, taking advantage of a unique window of time when the immune system is immature. The fetus’ body then recognizes the mother’s bone marrow as its own, enabling a second, non-toxic bone marrow transplant of stem cells after birth to complete the process.

With normal bone marrow, the child’s body would produce normal red blood cells, avoiding all of the painful, life-limiting symptoms of sickle cell disease. Funding support for this innovative biomedical approach can advance it into a fully realized, unprecedented and enduring scientific achievement: curing sickle cell disease at the beginning of a child’s life. This research has shown success in animal studies and may progress to testing in clinical trials within the next few years.

Children’s Hospital is an international leader in both fetal therapy and the treatment of sickle cell disease and has pioneered numerous medical breakthroughs that have dramatically improved the lives of children. Many of these breakthroughs have then received federal funding support from the National Institutes of Health to help advance research results into new standards of care.

March 2012