$5 Million Funds Stem Cell Work Aimed at MS, Other Diseases

July 29, 2005

A research group at the University of Rochester Medical Center has received new grants totaling more than $5 million to continue its work exploring the use of stem cells to treat several diseases, including multiple sclerosis, Huntington’s disease, and several fatal children’s disorders.

The new funding will allow neurologist Steven Goldman, M.D., Ph.D., to expand his research group of more than two dozen people exploring the use of stem cells to treat a host of common and rare neurological disorders. Goldman, a professor in the Department of Neurology and chief of its Division of Cell and Gene Therapy, moved his group from Cornell’s Weill Medical College in New York City to Rochester two years ago. The new funding pays for equipment and salaries, and allows Goldman to attract additional researchers with specific expertise in stem cell biology.

Goldman’s research centers on new ways to replace or replenish crucial cells in the brain and spinal cord. A new five-year grant of $1.76 million from the National Institutes of Health is supporting the group’s efforts against Huntington’s disease, a fatal neurological disease for which there is currently no cure and little treatment. Goldman is exploring new ways to compel the brain to make more of the kind of brain cells that die in patients with Huntington’s disease, causing their symptoms. A symphony of molecular signals normally directs the growth of brain cells from neural stem cells, and Goldman is exploring ways to control those signals to actually replace those neurons lost because of disease.

Goldman’s laboratory has also just received a grant for $500,000 from the National Multiple Sclerosis Society, to explore potential new treatments for MS. Currently, there are medications to try to slow the disease, but there is nothing to treat the underlying damage to nerves that occurs in all MS patients and causes symptoms such as fatigue and weakness. Goldman is part of an international team of experts brought together with MS Society funding for the next five years to explore ways to repair the nerve damage caused by the disease. This new funding adds to recent grants to Goldman’s team of $590,000 from the NMSS, and $1.8 million from NIH, each intended to explore ways to repair the damage caused by the disease.

In addition, funding from two private foundations – a type of support that is crucial for many researchers using stem cells – is supporting his research on several rare brain diseases of children. The grants, totaling $410,000, come from the Children’s Ataxia Telangiectasia Foundation and the Children’s Neurobiological Solutions Foundation. The funds will allow Goldman’s group to pursue research that holds promise for a group of rare, usually fatal diseases known as pediatric leukodystrophies. These diseases include disorders such as Krabbe’s, Pelizaeus-Merzabacher and Tay-Sachs diseases – though individually rare, collectively the diseases kill thousands of children every year.

In this study, Goldman’s team will build on its previous work restoring nerve function in brain tissue by restoring a crucial substance known as myelin, which is lost in these diseases. Myelin is a fatty substance that covers nearly all the nerve cells in our bodies, like insulation wrapped around a wire, and helps signals in the nervous system move crisply from one point to another. In the laboratory, Goldman has shown that by injecting human “progenitor cells” into the brain under the right conditions, they can evolve into cells that make new myelin. Using this approach, last year he was able to replenish myelin around the nerves in extensive regions in the brains of laboratory mice. With the new funding the team will continue its experiments, with the goal of laying the foundation for a treatment for children afflicted with these disorders, which currently are incurable and untreatable.

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