Intravenous stem cell transplant reverses sickle cell anemia

Kathleen Blanchard's picture

Following a phase I/II study, researchers have found that intravenous stem cell transplant, known as “mini” stem cell transplant, can reverse sickle cell anemia in adults. Intravenous blood forming stem cells were infused from healthy donors in ten patients with severe sickle cell anemia, resulting in normal blood cells and reversal of sickle cell anemia damage to organs.

The findings come from scientists at the National Institutes of Health and Johns Hopkins. Jonathan Powell, M.D., Ph.D., associate professor at the Johns Hopkins Kimmel Cancer Center explains that sickle cell anemia is the result of a defective gene. Providing patients with a “mini” stem cell transplant does not replace the gene, but rather transplants blood stem cells that possess the normal gene.


Sickle cell anemia causes damage to the body because red blood cells are mishapen, reducing the oxygen carrying capacity needed to keep organs healthy. The result can lead to stroke, causes severe pain, and can damage major organs.

Patients in the study who received blood stem cells from matched donors were given rapamycin, to aid the co-existence of the patient’s own cells and those of the donor – in the past stem cell transplants failed because of rejection of donor cells.

Before receiving the “mini” transplant, patients are given rapamycin to suppress immunity. Nine of the ten patients studied have blood stem cells that co-exist with sickle cells, reversing sickle cell anemia. The blood stem cell transplant via intravenous infusion is safer than chemotherapy used to wipe out the immune system prior to bone marrow transplant that can lead to death in some patients. The combination of rapamycin and radiation is followed by the intravenous stem cell implant.

The “mini” transplant is safer than bone marrow transplant, and offers hope for adult patient suffering from sickle cell anemia. Nine out of ten patients were found to have reversal of sickle cell anemia following “mini” stem cell transplant.