A small study has shown huge results: Researchers have found that bone marrow transplants can reverse severe sickle cell disease in adults. Out of 30 adult participants, the transplant worked in 26, and one year later 15 were able to go off the anti-rejection pills they were taking.
"We're very pleased," Dr. John Tisdale, the study's senior author and a senior investigator at the National Institutes of Health, told The Associated Press. "This is what we hoped for."
Sickle cell disease is a genetic disorder that damages hemoglobin in red blood cells, which in turn makes them form sickle shapes that can block blood flow. About 100,000 Americans, primarily blacks, have the disease, which can cause organ damage, anemia, and pain, as well as an early death.
The patients participating in the study underwent radiation and chemotherapy to destroy the bone marrow, which was then replaced with healthy marrow cells from a donor, either a patient's brother or sister. The one downside is that less than 1 out of 4 adults with sickle cell disease have a sibling that makes a good match, but researchers are looking into whether other relatives could be suitable donors.
The treatment is a modified version of transplants that have worked in children, whose bodies have yet to be ravaged by the disease. The results of the study were published Tuesday in the Journal of the American Medical Association.