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September 21, 2011

$4.5M bolsters efforts to treat neurological disease

Funding from public, private sources will fuel several research projects at the Medical Center

The endeavor to find better treatments or perhaps even one day a cure for a host of debilitating and fatal neurological diseases has been bolstered by an influx of funding from a mix of private and public sources.

The laboratory headed by Steven Goldman, chair of the Department of Neurology, has received $4.5 million in new funding to further its efforts to use stem cells and related molecules to treat several feared disorders for which there are currently no cures—including multiple sclerosis, Huntington’s disease, and fatal childhood diseases known as pediatric leukodystrophies.

The new funding, which will support work in the laboratory for the next three to five years, comes from a mix of private and government sources, including the National Multiple Sclerosis Society, the CHDI Foundation for Huntington’s disease research, Biogen Idec, and the National Institutes of Health.

Goldman’s research is at the forefront of attempts to harness the promise of stem cells to benefit patients who suffer from neurological diseases.

Two newly funded projects focus on the molecular events involved in the repair of myelin, a critical substance in the brain that breaks down in conditions such as multiple sclerosis, as well as in a number of hereditary childhood diseases.

In a related project funded by a $1.7 million grant from the National Institutes of Health, Goldman’s team will establish mice whose brains contain some human oligodendrocytes and human myelin in order to recreate in these mice the type of damage that occurs in the brains of MS patients.

A closely related project, for which the lab was granted $670,000 by Biogen Idec, focuses on using these mice with human myelin to study the progression of a rare brain disease, progressive multifocal leukoencephalopathy, which can affect people whose immune systems have been suppressed with medication. In a fourth and related project, supported with a $1.34 million grant by the CHDI Foundation, the team will explore the use of brain cells known as astrocytes to improve the condition of mice with Huntington’s disease.

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