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The University of Rochester Medical Center has opened the doors on a new facility that will enable researchers to create, study, and ultimately use stem cells and their offspring in early-phase experimental human therapies. The Upstate Stem Cell cGMP Facility – which will be used by academic and private-sector scientists from across the state – was created with $3.5 million in support from the Empire State Stem Cell Board.

“One of the critical barriers to moving cell-based therapies into clinical trials is the requirement that these cells be manufactured in a facility that meets strict federal requirements,” said Steve Dewhurst, Ph.D., chair of the URMC Department of Microbiology and Immunology and principal investigator for the state grant. “Without this resource, much of this science remains stuck in the lab.”

In a new study appearing this month in the Journal of Neuroscience, researchers have unlocked the complex cellular mechanics that instruct specific brain cells to continue to divide. This discovery overcomes a significant technical hurdle to potential human stem cell therapies; ensuring that an abundant supply of cells is available to study and ultimately treat people with diseases.

One of the major factors that will determine the viability of stem cell therapies is access to a safe and reliable supply of cells, said University of Rochester Medical Center (URMC) neurologist Steve Goldman, M.D., Ph.D., lead author of the study. This study demonstrates that -- in the case of certain populations of brain cells -- we now understand the cell biology and the mechanisms necessary to control cell division and generate an almost endless supply of cells.

When the era of regenerative medicine dawned more than three decades ago, the potential to replenish populations of cells destroyed by disease was seen by many as the next medical revolution. However, what followed turned out not to be a sprint to the clinic, but rather a long tedious slog carried out in labs across the globe required to master the complexity of stem cells and then pair their capabilities and attributes with specific diseases.

In a review article appearing today in the journal Science, University of Rochester Medical Center scientists Steve Goldman, M.D., Ph.D., Maiken Nedergaard, Ph.D., and Martha Windrem, Ph.D., contend that researchers are now on the threshold of human application of stem cell therapies for a class of neurological diseases known as myelin disorders -- a long list of diseases that include conditions such as multiple sclerosis, white matter stroke, cerebral palsy, certain dementias, and rare but fatal childhood disorders called pediatric leukodystrophies.

Stem cell biology has progressed in many ways over the last decade, and many potential opportunities for clinical translation have arisen, said Goldman. In particular, for diseases of the central nervous system, which have proven difficult to treat because of the brain's great cellular complexity, we postulated that the simplest cell types might provide us the best opportunities for cell therapy.

A previously unrecognized system that drains waste from the brain at a rapid clip has been discovered by neuroscientists at the University of Rochester Medical Center. The findings were published online August 15 in Science Translational Medicine.

The highly organized system acts like a series of pipes that piggyback on the brain's blood vessels, sort of a shadow plumbing system that seems to serve much the same function in the brain as the lymph system does in the rest of the body -- to drain away waste products.

"Waste clearance is of central importance to every organ, and there have been long-standing questions about how the brain gets rid of its waste," said Maiken Nedergaard, M.D., D.M.Sc., senior author of the paper and co-director of the University's Center for Translational Neuromedicine. "This work shows that the brain is cleansing itself in a more organized way and on a much larger scale than has been realized previously.

"We're hopeful that these findings have implications for many conditions that involve the brain, such as traumatic brain injury, Alzheimer's disease, stroke, and Parkinson's disease," she added.

After four years as chair of the Department of Neurology, Steven Goldman, M.D., Ph.D., is stepping down to resume research duties full time as Co-Director of the Center for Translational Neuromedicine.

Dr. Goldman will remain an active member of the Neurology faculty, increasing his research focus and commitment, while Robert G. Holloway Jr., M.D., M.P.H., Professor of Neurology, will serve as Acting Chair of the Department as a national search for permanent leadership is launched.