Cells Enhance Marrow Transplants
Treatment with bone marrow mesenchymal cells has the potential to enhance the therapeutic effects of bone marrow transplantation in patients with osteogenesis imperfecta (OI), also known as brittle bone disease, according to researchers at St. Jude’s Children’s Research Hospital (501St. Jude Place, Memphis, TN 38105; Tel: 901/495-3300; Website: www2. stjude.org). Mesenchymal cells are bone-marrow derived stem cells that have the ability to form body tissues such as bone, cartilage, muscle and fat. The findings of the St. Jude’s team were published in the June 25 issue of the journal Proceedings of the National Academy of Sciences.
OI is a genetic disorder characterized by bones that break easily, often from little or no apparent cause. The disease can be mild to severe and affects between 20,000 and 50,000 Americans. OI is caused by a genetic defect that affects the production of collagen, the major protein of the body’s connective tissue. Patients with OI have either less collagen or a poorer quality of collagen than healthy individuals, leading to weak bones that fracture easily. Current treatment is directed toward preventing or controlling the symptoms, maximizing independent mobility and developing optimal bone mass and muscle strength.
“This is the first human trial to clearly show the therapeutic potential of mesenchymal cells and represents a significant step forward in the development of cellular therapies,” says Edwin Horwitz, a member of the department of Hematology-Oncology at St. Jude and the lead investigator for the research.
For the study, infusions of gene-marked, donor-marrow derived mesenchymal cells were used to treat five children, ranging in age from 2 years, 10 months to 4 years, 9 months, who had previously undergone standard bone marrow transplantation for severe OI. The mesenchymal cells were harvested from the bone marrow of patients’ transplant donors.
The patients received an initial infusion of mesenchymal cells 18 to 34 months after bone marrow transplant. After a second infusion, a few weeks later, all of the patients showed engraftment in one or more sites, including bone, skin and bone marrow. Additionally, the children experienced an acceleration of growth during the first six months post-infusion. The growth rates ranged from 60% to 94% of the predicted growth for children unaffected by OI. The patients had experienced growth rates of 0% to 40% in the six months prior to the infusions.
“For kids with OI, our study represents hope for an improved treatment program and someday a cure,” Horwitz says. “For all people with diseases of mesenchymal tissue such as bone, cartilage or muscle, our study represents the potential for new cellular therapy that may improve our general treatment strategies.”
The treatment methodology used in the study is based on the research of Darwin Prockop, who has investigated the genetic mutations that cause OI. Prockop is a faculty member at the Tulane Center for Gene Therapy in New Orleans, Louisiana.
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