Progression of Huntington’s Disease Slowed In Mice|Could Pave Way For New Method Of Treatment
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 in the journal Nature Communications, could ultimately point to a new method to treat the disease.
The investigators implanted animals with human glial cells derived from stem cells. One of the roles of glia is to tend to the health of neurons, and the results from this new study reveal that replacing sick mouse glia with healthy human cells blunted the progress of the disease and rescued nerve cells at risk of death.
Huntington’s is a hereditary neurodegenerative disease that is most closely characterized by the loss of a specific nerve cell in the brain that plays a critical role in motor control called the medium spiny neurons. Over time, the disease results in involuntary movements, problems with coordination, and cognitive decline and depression. There is currently no way to slow or modify this fatal disease.
“The role that glia cells play in the progression of Huntington’s disease has never really been explored,” explained senior study author Steve Goldman, M.D., Ph.D., co-director of the University of Rochester Center for Translational Neuromedicine and professor at the Center of Basic and Translational Neuroscience at the University of Copenhagen. “This study shows that these cells are not only important actors in the disease but may also hold the key to new treatment strategies.”
“It’s the first time, we’ve conducted this type of transplant, and the results are both positive and surprising,” Dr. Goldman added. “It reveals that diseased mice injected with healthy glia cells live longer, and their condition improves. This is very promising, and it’s only the tip of the iceberg. We hope to be able to conduct further research on whether this method could possibly result in a treatment for Huntington’s.”
The researchers conducted a series of experiments in which they isolated human glial progenitors – the cells in the central nervous system that give rise to astrocytes – from both embryonic stem cells and brain tissue and implanted the cells into the striatum (a part of brain) of mice with Huntington’s disease. Consistent with prior studies, they observed that the resulting human astrocytes out-competed the native glia cells, resulting in mice with native neurons but human glia.
The researchers discovered that human glia transplanted into mice with the Huntington’s disease mutation appeared to keep neurons healthier and extended the animals survival. They also conducted a battery of tests designed to measure the animals’ behavior, memory, and motor skills, and the mice with healthy human glia performed significantly better than untreated mice with Huntington’s disease.
Conversely, when healthy mice were implanted with human glia carrying the genetic mutation that causes Huntington’s, the animals exhibited symptoms of the disease.
Because glial cells have been shown to migrate and proliferate throughout the brain once implanted, the research team believes their recent findings could herald a potential new approach to rescue nerve cells threatened by the disease.
“The partial rescue of deficiencies we observed in this study tells us that there is a significant glial component in Huntington’s disease and that we may be able to improve function and delay progression with glial transplants,” Dr. Goldman concluded.