Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta - PubMed

Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta

E M Horwitz et al. Nat Med. 1999 Mar.

Abstract

In principle, transplantation of mesenchymal progenitor cells would attenuate or possibly correct genetic disorders of bone, cartilage and muscle, but clinical support for this concept is lacking. Here we describe the initial results of allogeneic bone marrow transplantation in three children with osteogenesis imperfecta, a genetic disorder in which osteoblasts produce defective type I collagen, leading to osteopenia, multiple fractures, severe bony deformities and considerably shortened stature. Three months after osteoblast engraftment (1.5-2.0% donor cells), representative specimens of trabecular bone showed histologic changes indicative of new dense bone formation. All patients had increases in total body bone mineral content ranging from 21 to 29 grams (median, 28), compared with predicted values of 0 to 4 grams (median, 0) for healthy children with similar changes in weight. These improvements were associated with increases in growth velocity and reduced frequencies of bone fracture. Thus, allogeneic bone marrow transplantation can lead to engraftment of functional mesenchymal progenitor cells, indicating the feasibility of this strategy in the treatment of osteogenesis imperfecta and perhaps other mesenchymal stem cell disorders as well.

Comment in

• Mesenchymal stem cells: no longer second class marrow citizens.

  Gerson SL. Gerson SL. Nat Med. 1999 Mar;5(3):262-4. doi: 10.1038/6470. Nat Med. 1999. PMID: 10086373 No abstract available.

• Osteogenesis imperfecta calls for caution.

  Marini JC. Marini JC. Nat Med. 1999 May;5(5):466-7. doi: 10.1038/8326. Nat Med. 1999. PMID: 10229207 No abstract available.

• Osteogenesis imperfecta calls for caution.

  Bishop NJ. Bishop NJ. Nat Med. 1999 May;5(5):466-7. doi: 10.1038/8528. Nat Med. 1999. PMID: 10229208 No abstract available.

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