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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