Children’s Hospital of Philadelphia: Center for Fetal Diagnosis and Treatment
Just 20 years ago, the developing fetus was a mystery to doctors. Around that time, physicians now working at The Children’s Hospital of Philadelphia (CHOP) in the Center for Fetal Diagnosis and Treatment began to look at prenatal diagnosis and treatment more closely. Dr. Alan Flake, Director of the Center, is leading that effort. Prenatal testing is readily available for many genetic disorders affecting the development of a baby. Generally, though, these conditions can only be treated after a baby is born and develops the symptoms of a disease.
Specifically, genetic disorders involving blood cells can, at present, only be cured by bone marrow transplantation. These include relatively common disorders, such as Sickle Cell Disease, but also a host of other rare diseases, such as many types of immunodeficiency disorders. Without a perfectly matched donor, which is very hard to find, these treatments are very toxic to the patient and can cause other severe health problems.
The Albert M. Greenfield Foundation has been supporting the investigations of Dr. Flake and his team toward a novel alternative approach to bone marrow transplantation called In Utero Hematopoietic Stem Cell Transplantation. This approach involves transplanting normal cells that form a baby’s blood cells very early on in the pregnancy. An important advantage is that prenatal treatment would prevent all of the devastating manifestations of these disorders, allowing children to grow and develop normally.
Research in this field has been conducted by the team at CHOP for over 20 years with support from the NIH and other funding agencies. The support of the Greenfield Foundation has accelerated progress toward clinical application of the strategy.
The research team’s goal is to develop a clinical trial for prenatal treatment of Sickle Cell Disease within the next 5 years. The success of the approach would open the door for prenatal treatment of a large number of other genetic disorders.