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The Research: A Cure on the Horizon

Bone marrow transplantation can cure sickle cell disease, but fewer than 15% of sickle cell patients can find a matching donor and it is highly risky: the fatality rate is 10% and the rate of serious complications is more than 50%. Though it is a cure, it is not an option for most patients; fewer than 300 bone marrow transplants have been performed for sickle cell disease.

Alan Flake, M.D., director of the Center for Fetal Research at CHOP, has spent nearly 30 years developing a pioneering procedure that promises a true cure. It involves a series of two bone marrow stem cell transplants from the mother to her child the first while the child is still in utero, so the child’s body recognizes the mother’s cells as its own, and a second after birth to cement the process. With normal bone marrow, the child’s body will produce normal red blood cells avoiding all the painful, life-limiting symptoms of sickle cell disease.

Our Goal

CHOP’s Center for Fetal Diagnosis and Treatment is the largest and most comprehensive fetal treatment program in the world, capable of performing early prenatal diagnosis and fetal bone marrow transplants. To proceed with a pilot clinical trial, Dr. Flake and his team must first prove the procedure can be applied reliably and safely in research models and satisfy regulatory requirements from the FDA. They hope to begin clinical trials in humans in three years. The cost for supporting Dr. Flake’s work through the conclusion of the clinical trial is $5 million.

This technique would be revolutionary for sickle cell disease and beyond, as it also can be applied to almost all other immunodeficiency disorders.