St. Jude has treated nine patients with this rare disease. Influencing factors Osteopetrosis is an autosomal recessive disorder. This means both parents must carry the gene for this disease to develop in their children. Osteopetrosis appears to have an increased occurrence in Costa Rica and in certain Middle Eastern countries. The only cure that exists for osteopetrosis is bone marrow transplant.
Optimal transplants require donor marrow from a sibling.
Patients must receive a bone marrow transplant early enough in life so complications of osteopetrosis can be reversed or prevented altogether. Because few patients fit this criteria, researchers are looking for alternative therapy. Research Physicians are currently studying the effects of a drug called interferon-gamma. There is a constant search for a new drug that could be effective in treating osteopetrosis. The ideal drug would correct the defect in osteoclasts, making it possible for the bone marrow cavity to form.