Mesenchymal proof of concept

Bone marrow transplantation (BMT) traditionally has been used to replace hematopoietic cells - red and white blood cells - in patients whose own bone marrow has been destroyed through disease or medical treatment such as chemotherapy. Cell therapy companies have recognized for some time that BMT has wider potential because bone marrow contains more than hematopoietic stem cells, but BMT is only now being applied to a range of diseases.

Last week, researchers at St. Jude Children's Research Hospital (Memphis, Tenn.) used allogeneic BMT to treat osteogenesis imperfecta, a bone disease that results from the secretion of a mutant (and defective) type I collagen by osteoblasts. The successful treatment of three children with the disease, published in Nature Medicine, demonstrated that mesenchymal stem cells (MSCs), the precursors to osteoblasts and other cell lineages, can engraft after BMT and give rise to functional mesenchymal cell types...