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Published on: December 8, 2016
Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis
E M Horwitz1, D J Prockop, L A Fitzpatrick
1Cell and Gene Therapy Program, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.
Insights
Allogeneic bone marrow transplants in children with osteogenesis imperfecta led to functional mesenchymal progenitor cell engraftment. This approach shows promise for treating this genetic bone disorder and others.
Area of Science:
- Regenerative Medicine
- Genetics
- Orthopedics
Background:
- Mesenchymal progenitor cell transplantation could treat genetic bone disorders.
- Clinical evidence supporting this concept is currently limited.
- Osteogenesis imperfecta is a genetic disorder characterized by defective type I collagen production by osteoblasts.
Purpose of the Study:
- To evaluate the feasibility and initial outcomes of allogeneic bone marrow transplantation (BMT) in children with osteogenesis imperfecta (OI).
Main Methods:
- Allogeneic bone marrow transplantation was performed in three children diagnosed with osteogenesis imperfecta.
- Engraftment of donor mesenchymal progenitor cells was assessed.
- Bone mineral content, growth velocity, and fracture frequency were monitored post-transplantation.
Main Results:
- Successful engraftment of donor osteoblasts (1.5-2.0%) was observed within three months.
- Histologic analysis of bone specimens revealed new, dense bone formation.
- Patients showed significant increases in total body bone mineral content (21-29g) compared to predicted values.
- Improvements in growth velocity and a reduction in fracture frequency were noted.
Conclusions:
- Allogeneic bone marrow transplantation facilitates the engraftment of functional mesenchymal progenitor cells in patients with osteogenesis imperfecta.
- This strategy demonstrates feasibility for treating osteogenesis imperfecta.
- The findings suggest potential applicability to other mesenchymal stem cell disorders.
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.
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