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Paul D. Robbins

University of Pittsburgh Schools of the Health Sciences

University Of Pittsburgh Stem Cell Research Funded

PITTSBURGH, January 17, 2003 Two faculty members of the University of Pittsburgh School of Medicine have received grants totaling more than $2 million from the National Institute of Arthritis and Musculoskeletal and Skin Diseases at the National Institutes of Health to fund studies that target the use of stem cells to treat disease.

They are Christopher Niyibizi, Ph.D., research associate professor in the Ferguson Laboratory for Orthopaedic Research in the department of orthopaedic surgery and cell biology and physiology; and Johnny Huard, Ph.D., Henry J. Mankin Associate Professor of Orthopaedic Surgery. Dr. Huard also is an associate professor in Pitts department of molecular genetics and biochemistry, deputy director of the McGowan Institute for Regenerative Medicine and director of the Growth and Development Laboratory at Childrens Hospital of Pittsburgh.

Dr. Niyibizi, who also is a faculty member of the McGowan Institute, has been awarded $1.26 million for a study, Stem Cell Therapy for Diseases of Bone in a Mouse Model. This study is evaluating the use of stem cells derived from adult bone marrow for the treatment of osteogenesis imperfecta (OI), a hereditary disorder of the connective tissue related to collagen production that can be marked by extreme fragility of the long bones and a bluish color of the whites of the eyes. A person with OI can break a rib by coughing, or a leg by rolling over during sleep.

Osteogenesis imperfecta can be a devastating illness really, a constellation of illnesses, said Dr. Niyibizi. People who have it can suffer hundreds of fractures throughout a lifetime in a truly disabling way.

Dr. Niyibizi is principal investigator for the study, which focuses on mice that are born with a severe form of OI that mimics the human condition. Dr. Niyibizi and his colleagues are investigating the effect of using bone marrow-derived stem cells to treat these mice. Previous studies have shown that mesenchymal stem cells isolated from bone marrow and infused into recipient animals persist in bone and differentiate into bone cells. Mesenchymal stem cells are undifferentiated cells that can become cells related to the connective tissue, including bone, cartilage, fat and muscle as well as brain and epithelial cells. The current study aims to determine characteristics of engraftment by these cells and their contribution to the animals bone structure.

While the number of people affected by OI in the United States is unknown, estimates from the Osteogenesis Imperfecta Foundation range from 20,000 to 50,000. There is currently no cure, although treatment options are available ranging from medications to metal rod insertion surgery to strengthen skeletal support. Treatment of chronic fractures, physical therapy and the use of mobility aids such as wheelchairs and braces also are common.

In addition to Dr. Niyibizi, other investigators involved in the study include Janey Whalen, Ph.D.; Paul Robbins, Ph.D.; and Molly Vogt, Ph.D., all of the University of Pittsburgh School of Medicine; and Jeffrey Hollinger, Ph.D., Carnegie Mellon University.

This and other proposed studies will lead to the design of human clinical trials using bone marrow-derived cells for the treatment not only of OI, but also other bone-related disorders such as osteoporosis .

Dr. Huard has been awarded $1.2 million over five years for a study, Muscle Regeneration Through Stem Cell Transplantation, that focuses on using a unique population of muscle stem cells from healthy newborn mice to deliver dystrophin, a key protein for muscle function, into mice born with a genetic muscle-wasting disease similar to Duchenne muscular dystrophy. In humans, this genetic disease causes muscle weakness and early death because of respiratory or cardiac failure.

Studying the behavior of these cells after transplant, we found some very exciting things, said Dr. Huard. Not only did the donor cells continue to grow and make dystrophin in the recipient, but they also apparently failed to provoke an immune response, which would protect them from rejection.

In previous studies, transplant of these muscle-derived stem cells also improved muscle regeneration in recipient mice, Dr. Huard said. The current study targets further investigation of the use of such cell transplants to discover ways to improve prior results in muscle regeneration and dystrophin delivery.

Dr. Niyibizi and Dr. Huards studies also will further knowledge of basic stem cell behavior and function.

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