Cancer Therapies

Expanding The Therapeutic Potential Of Bone Marrow Transplantation (March 1999)

In a ground-breaking article in the March 1999 issue of Nature Medicine, Edwin Horwitz and colleagues at St. Jude's Children's Research Hospital, Tennessee reported an advance that pushes the frontiers of bone marrow transplantation to test a therapy that may offer relief to thousands of patients suffering from painful bone diseases.

Osteogenesis Imperfecta (OI) is a genetic disorder characterized by bones that break easily that affects between 20,000 and 50,000 people in the US. Patients suffer numerous fractures, restricted physical activity and short stature. At present there is no cure for OI and treatment involves extensive surgical and dental procedures to mend fractures. In severe cases, metal rods are inserted through bones for support.

Based on the knowledge that OI is caused by osteoblasts that produce defective type I collagen-the protein matrix that contributes to bone formation-Horwitz's team exploited the ability of mesenchymal stem cells, which are present in bone marrow, to differentiate into osteoblasts that secrete new organic bone matrix and rejuvenate bone.

The researchers infused sibling bone marrow into children suffering from OI that had undergone ablative conditioning therapy. They found changes in bone histology to indicate new bone formation, and clinical improvement during the first 6 months after transfusion-the children suffered fewer fractures and grew in height. The researchers are hoping that "bone marrow transplantation could be used to correct a far wider range of inherited and acquired disorders that now occurs."

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