The University of Rochester Medical Center has announced a five-year, $10.5 million grant from the CHDI Foundation in support of research to develop a stem-cell-based therapy for Huntington's disease.
Huntington's disease is a hereditary neurodegenerative disease characterized by the loss of medium spiny neurons, nerve cells in the brain that play a critical role in motor control. As the disease progresses and the spiny neurons die, patients develop involuntary movements, problems with coordination, cognitive decline, depression, and often psychosis.
The grant will support the work of URMC researcher and URMC Center for Translational Neuromedicine co-director Steve Goldman, who, with his URMC colleagues, transplanted healthy human support cells, called glial progenitor cells, into the brains of mice with Huntington's. In those experiments, the new glial cells took over for the sick ones and were able to stabilize and even rescue neurons that were being lost to the disease, slowing its progression and allowing the animals to live longer. The new award will enable Goldman’s team to further understand the basic biology of glial cells so that they can refine the process of preparing them for implantation before going to clinical trials.