Magazine article Science News

Marrow Transplant Fights Bone Disease

Magazine article Science News

Marrow Transplant Fights Bone Disease

Article excerpt

Children born with a hereditary disease called osteogenesis imperfecta can face a lifetime of bone deformities, fractures, and short stature. These children produce faulty collagen--the white, fibrous protein that forms the framework for bone, tendons, and ligaments.

The skeletons of severely affected children are so weak that parents have been known to break a child's leg accidentally while changing a diaper. There is no known cure for osteogenesis imperfecta. Treatment consists of inserting metal rods into the largest bones as reinforcements.

Now, initial findings in a study of three children with the disease who received bone marrow transplants from healthy siblings reveal sharp increases in the recipients' bone mass, fewer fractures, and some height gain. The work, reported in the March NATURE MEDICINE, raises the prospect of a treatment that attacks osteogenesis imperfecta at its core.

Three months after receiving the marrow transplants, only 1.5 to 2.0 percent of the patients' osteoblasts--cells that make collagen--stemmed from donated marrow. Yet that small amount seems to have made a difference, says study coauthor Edwin M. Horwitz, a pediatric hematologist and oncologist at St. Jude Children's Research Hospital in Memphis, Tenn.

Horwitz admits to being "a bit surprised" when analysis showed that the three children had added 21, 28, and 29 grams of bone in the 100 days after the transplant. Healthy children of these ages--two of the patients were 13 months old and the other 32 months old--showing the same modest weight gain would have been expected to add less than 4 g of bone, he says. …

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