Characterization of the biological properties of adult stromal cells is essential for a better understanding of their normal function in adults as well as to revealing their potential in treating disease. Stem cells are so named because they are like the stems on a tree that can produce new leaves and flowers each year. Each stem cells has the remarkable property that it can divide so as to produce a perfect copy of itself together with a second cell that can become a “workhorse” cell of the body such as a bone cell or a nerve cell. Because the stem cell produced by the division is a perfect copy of the original stem cell, stem cells seem to be able to divide and live indefinitely, perhaps forever. Understanding stem cells is now one of the most important problems of biology. The Center staff is working at the forefront of research on stem cells using cutting edge technologies to define them in terms of the genes they express. Also, they have developed new procedures that make it possible to begin with a small sample of stem cells from a patient’s bone marrow and grow extremely large numbers of the cells in the laboratory. The ability to grow the cells rapidly, in turn, makes it possible to gene engineer the cells with simple techniques that do not involve use of a virus.
To test the cell’s potentials for therapy of patients, the Center is using the adult stromal stem cells to treat mice and rats that represent models of human diseases. Plans are being made to carry out similar experiments in non-human primates at the nearby Tulane University Primate Center, the largest N. I. H. sponsored primate center in the country. In one series of experiments, cells are transplanted into mice that undergo repeated spontaneous bone fractures because of a genetic defect. The aim of these experiments is to determine if the stem cells can travel to the site of a bone fracture and strengthen the bone and prevent further fractures. Results from these studies should define the most effective ways the cells can be used to treat human bone diseases, such as osteogenesis imperfecta and osteoporosis. In other experiments, the Center staff is pursuing their discovery that the adult stem cells can differentiate into cells that make up the brain. Therefore, the cells are being transplanted directly into the brains of mice that exhibit progressive neurodegeneration due to lack of a critical protein. The aim of these experiments is to determine if the stem cells can replace the missing protein and reverse the degeneration of the brain. If the experiments succeed, they will suggest that the cells can be used to treat serious neurological diseases in children such as Tay-Sachs disease. Other studies are evaluating if the stem cells can replace brain cells lost in common diseases of adults. Promising preliminary results were recently obtained in a rat model for parkinsonism. Similar experiments are underway to test the effectiveness of the cells in animal models for Alzheimer’s disease and brain tumors (gliomas). The procedures for the experiments in animal models are being developed so as to conform to reporting requirements of the Food and Drug Administration and other agencies in order that the therapies can be introduced as clinical trials in patients as soon as possible