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Funded Studies

Catherine M. Verfaillie, MD

Parkinson's disease affects almost one in one thousand adults. The disease is caused by a still unknown mechanism that destroys the neurons (brain cells) that produce dopamine in the brain. Although symptoms caused by the disease can be alleviated by intake of dopamine, the destruction of the brain cell continues, leading to further deterioration of the symptoms despite drug therapy. Stem cell therapy, in which new young cells that can generate brain cells that produce dopamine, are introduced in the brain of rats or mice have shown great promise. Likewise, some clinical studies in humans have shown that stem cells may be capable of reversing the disease. Stem cells can be obtained from the brain of human or animal fetuses, can be derived from embryonic stem cells, and possibly from adult stem cells. We have identified stem cells in the marrow of humans, mice, and rats that can be induced in the laboratory to generate neuron-like cells that produce dopamine, the cells that are missing in Parkinson's disease. We now propose to further study this phenomenon and to start testing whether transplantation to such adult stem cells derived from bone marrow may be useful in the therapy of this otherwise devastating disease.

Dr. Catherine Verfaillie is currently a Professor of Medicine at the University of Minnesota and the Director of the Stem Cell Institute at the University of Minnesota. She obtained her M.D. degree from the University of Leuven, Belgium in 1982, and did a post-doctoral fellowship at the University of Minnesota. Her major research interests are in stem cell biology. This includes studies that evaluate regulation of proliferation, differentiation, and lineage commitment of normal hematopoietic stem cells as well as multipotent stem cells. In the hematopoietic field, she is interested in cord blood stem cell expansion and gene therapy, as well as in mechanisms underlying the malignant transformation due to the presence of oncogenes such as in chronic myelogenous leukemia. Over the last four to five years, her laboratory has identified stem cells, previously thought to be mesenchymal stem cells, in the bone marrow of humans, mice, and rats that have much greater plasticity than previously detected. She is performing studies aimed at purification, expansion, and induction of differentiation to multiple lineages including endothelium, liver and pancreas, and different cells of the central nervous system including neurons, atrocities, and ologodendrocytes. She was recently named by U.S. World and News Report as one of the ten Innovators of 2001.

Associated Grants

  • Multipotent Stem Cell Therapy for Parkinson's Disease


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