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  • October 12, 2016

    Research Will Explore New Therapies for Huntington’s Disease

    Image of astocytes

    A new award from the CHDI Foundation will advance promising research that aims to slow the progression of Huntington's disease. The funding, anticipated to total more than $10.5 million over next five years, will help University of Rochester Medical Center (URMC) scientists develop a stem cell-based therapy that swaps sick brain cells for healthy ones.

    The new award will go to the lab of Steve Goldman, M.D., Ph.D., the co-director of the URMC Center for Translational Neuromedicine, which has research operations in both Rochester and at the University of Copenhagen.

    Huntington's is a hereditary neurodegenerative disease characterized by the loss of medium spiny neurons, a nerve cell in the brain that plays a critical role in motor control. As the disease progresses over time and more of these cells die, the result is involuntary movements, problems with coordination, and cognitive decline, depression, and often psychosis. There is currently no way to slow or modify this fatal disease.

    The new award will support research that builds upon findings published by Goldman earlier this year in the journal Nature Communications showing that researchers were able to slow the progression of the disease in mice by transplanting healthy human support cells, called glial progenitor cells, into the animals' brains.

  • October 12, 2016

    Research Will Explore New Therapies for Huntington's Disease

    A new award from the CHDI Foundation will advance promising research that aims to slow the progression of Huntington's disease. The funding, anticipated to total more than $10.5 million over next five years, will help University of Rochester Medical Center (URMC) scientists develop a stem cell-based therapy that swaps sick brain cells for healthy ones.

    The new award will go to the lab of Steve Goldman, M.D., Ph.D., the co-director of the URMC Center for Translational Neuromedicine, which has research operations in both Rochester and at the University of Copenhagen.

    Huntington's is a hereditary neurodegenerative disease characterized by the loss of medium spiny neurons, a nerve cell in the brain that plays a critical role in motor control. As the disease progresses over time and more of these cells die, the result is involuntary movements, problems with coordination, and cognitive decline, depression, and often psychosis. There is currently no way to slow or modify this fatal disease.

    The new award will support research that builds upon findings published by Goldman earlier this year in the journal Nature Communications showing that researchers were able to slow the progression of the disease in mice by transplanting healthy human support cells, called glial progenitor cells, into the animals' brains.

  • June 30, 2016

    Steven Goldman reappointed as codirector of Center for Translational Neuromedicine, Dean Zutes Chair

    Steven Goldman, professor of neurology, has been reappointed as codirector of the Center for Translational Neuromedicine and as the Dean Zutes Chair in Biology of the Aging Brain—both through June 30, 2020. Goldman also retains his joint appointments as professor of neurosurgery and as Distinguished Professor in Neurosciences.

    Goldman is interested in cell genesis and neural regeneration in the adult brain. His lab focuses on the use of stem and progenitor cells for the treatment of neurodegenerative disorders such as Huntington's Disease, as well as for the treatment of glial diseases such as the pediatric leukodystrophies and multiple sclerosis. He has published more than 250 papers in his field—more than 100 as first or senior author—as well as 20 issued patents with more pending.

  • June 7, 2016

    Swapping Sick for Healthy Brain Cells Slows Huntington’s Disease

    Photo of Steven Goldman

    Researchers have successfully reduced the symptoms and slowed the progression of Huntington's disease in mice using healthy human brain cells. The findings, which were published today in the journal Nature Communications, could ultimately point to a new method to treat the disease.

    The research entailed implanting the animals with human glia cells derived from stem cells. One of the roles of glia, an important support cell found in the brain, is to tend to the health of neurons and the study's findings show that replacing sick mouse glia with healthy human cells blunted the progress of the disease and rescued nerve cells at risk of death.

    The role that glia cells play in the progression of Huntington's disease has never really been explored, said Steve Goldman, M.D., Ph.D., co-director of the University of Rochester Center for Translational Neuromedicine. This study shows that these cells are not only important actors in the disease, but may also hold the key to new treatment strategies.

  • February 22, 2016

    Hope, Hype, and Wishful Thinking

    Dr. Goldman

    In a perspective piece appearing in the journal Cell Stem Cell, URMC neurologist Steve Goldman, M.D., Ph.D., lays out the current state of affairs with respect to stem cell medicine and how close we are to new therapies for neurological disorders.

    The dawn of stem cell medicine some 25 years ago was greeted with great enthusiasm, particularly by scientists who study diseases in the central nervous system (CNS).  Many of the diseases found in the brain and spinal cord are degenerative in nature; meaning that over time populations of cells are lost due to genetic factors, infection, or injury.  Because stem cell medicine holds the potential to repair or replace damaged or destroyed cells, scientists have considered these diseases as promising candidates for new therapies.

    However, as with other emerging fields of medicine, the race to cures has turned out to be more of marathon than a sprint.  While scientists have become very adept at manipulating stem and progenitor cells and understanding the complex choreography of genetic and chemical signals that instruct these cells to divide, differentiate, and proliferate, researchers are still grappling with the challenges of how to integrate new cells into the complex network of connections that comprise the human brain.

    Goldman, co-director of the URMC Center for Translational Neuromedicine, takes a sweeping view of where we stand and which CNS diseases may or may not ultimately benefit from future stem cell-based therapies.