By Thomas H. Maugh II

 In a study of 10 adults with the blood disease, the procedure cures nine. More tests are needed, but the treatment could be used on other diseases as well.
Researchers have for the first time performed a successful bone marrow transplant to cure sickle cell disease in adults, a feat that could expand the procedure to more of the 70,000 Americans with the disease — and possibly some other diseases as well.

About 200 children have been cured of sickle cell with transplants, but the procedure was considered too harsh for adults with severe sickle cell disease. Now a team from the National Institutes of Health and Johns Hopkins University is reporting today in the New England Journal of Medicine that it has developed a much-less-toxic transplant procedure and used it to cure nine of the first 10 patients studied.

“We really don’t have anything else to offer patients with sickle cell disease” who do not respond to hydroxyurea, the only drug useful in treating it, said the paper’s senior author, Dr. John F. Tisdale of the National Heart, Lung and Blood Institute. “It’s really satisfying to perform a therapy that . . . gets rid of the disease.”

Dr. Kwaku Ohene-Frempong of the Children’s Hospital of Philadelphia agreed: “This is very significant . . . because it opens up the opportunity for adults to be considered for transplantation.”

The main problem is that not enough sickle cell patients have a healthy sibling who is a compatible donor. A sibling donor is normally necessary in sickle-cell transplants to ensure that the patient doesn’t reject the new marrow. The team plans to try using unrelated donors and parents.

Sickle cell disease, which affects primarily people of African descent, is caused by a genetic mutation that gives red blood cells a sickle shape, hindering their ability to carry oxygen and causing them to clog blood vessels. It can cause stroke, severe pain and organ damage — especially to the lungs, kidneys and liver — and can be fatal.

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Tags: , BONE MARROW, Bone marrow transplant 'gets rid of' sickle cell anemia, health

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