What is Sickle Cell Disease?
Sickle cell disease is caused by a small change in a hemoglobin protein contained in redblood cells (RBC's). This change causes the shape of the RBCs to beflat and curved into a sickle shape instead of the normal doughnut shape. These sickled cells do not pass smoothly through the smallest blood vessels causing many serious medical problems including blood clots, severe pain, and stroke, as well as heart attack, and liver, kidney and spleen damage.
Bone Marrow Transplantation for Sickle Cell
The abnormal RBC's in sickle cell disease, like all other blood cells, are produced in the bone marrow. A bone marrow transplant (BMT) can cure sickle cell disease by replacing the marrow that is manufacturing the abnormal cells. However, until now BMT has not been widely used for this purpose because severe complications can result. The Institute's approach to BMT may make it much safer by treating the donor bone marrow to remove harmful cells.
Clinical Trial for Sickle Cell
The Institute's clinical trial for sickle cell disease will use partial conditioning and a specially modified bone marrow transplant procedure to create "chimerism". This procedure allows both your own bone marrow and the new transplanted bone marrow to live together in your body. The purpose of this study will be to evaluate the effectiveness of "Mixed Chimerism" to treat sickle cell disease.
Principal Investigator - Roger Herzig, M.D.