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Bone Marrow Transplants May Cure Sickle Cell in Adults
Date:12/9/2009

to 45 with severe sickle cell disease alemtuzumab, a drug used to suppress immune system T-cells; relatively low doses of radiation; and sirolimus, an immune suppressant to fight rejection. Marrow donors were siblings with matched HLA (human leukocyte antigen) markers in their blood.

None of the patients experienced graft-versus-host disease, one of the most common and potentially fatal complications of bone marrow transplants, in which the body rejects the new bone marrow.

After 30 months, all of them are alive, and nine of the patients had successful grafts and are considered cured of sickle cell disease, according to the study.

"It's been transforming for these patients," Tisdale said. "These were the sickest of the sick patients. Some were in the hospital every other week for pain or other crises. Today, some have gone back to school and to work. One patient had a baby."

The last item is important, because in conventional bone marrow transplants, high doses of chemotherapy drugs and radiation typically destroy fertility. However, the lower level of radiation used in the new method does not seem to do this.

The study is published in the Dec. 10 issue of the New England Journal of Medicine.

Conventional bone marrow transplants cure sickle cell disease by first using chemotherapy and radiation to wipe out the person's own marrow, which makes the faulty red blood cells. The marrow is replaced with stem cells from a donor's marrow, which then takes over and begins to produce new, healthy red blood cells.

But when doing the new bone marrow transplants, the researchers noted that not all of the patient's own marrow was wiped out. Some remained and seemed to co-exist with the donor marrow without causing problems, Tisdale said.

"That meant we didn't necessarily have to kill the entire bone marrow of the patient to make this work," Tisdale said, opening the possibility of using an ev
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