For the first time, scientific researchers have been able to essentially regrow brain cells lost to Huntington's disease using stem cells.
The advance, reported in the journal Cell Stem Cell, is based on studies involving laboratory mice with the disease and may have significant implications for Huntington’s patients, said medical investigators from the University of Rochester Medical Center who conducted the research.
"This study demonstrates the feasibility of a completely new concept to treat Huntington's disease, by recruiting the brain's endogenous neural stem cells to regenerate cells lost to the disease," said neurologist Steve Goldman, M.D., co-director of Rochester's Center for Translational Neuromedicine.
Huntington's disease is an inherited neurodegenerative disease that gradually leads to the loss of motor control. The fatal disease, which affects some 30,000 Americans, results in involuntary movements, problems with coordination, cognitive decline, and depression. There is currently no cure or effective treatment.
Dr. Goldman’s work has involved the study of songbirds like canaries that have a unique ability in the animal kingdom to generate new brain cells as adults. This process, called adult neurogenesis, was first discovered by Dr. Goldman and Fernando Nottebohm of the Rockefeller University in the early 1980s.
"Our work with canaries essentially provided us with the information we needed to understand how to add new neurons to adult brain tissue," said Dr. Goldman. "Once we mastered how this happened in birds, we set about how to replicate the process in the adult mammalian brain."
Goldman and his team spent the past decade attempting to unravel the processes that lead to the creation of new brain neurons. For their latest work involving mice, they were able to replicate the complicated process by which stem cells produce new brain neurons in the region of the brain impacted by Huntington's disease.
The researchers were able to significantly extend the survival of the treated mice, in some cases doubling their life expectancy. The researchers also demonstrated that they could replicate this technique in the brains of monkeys, a step that brings the research much closer to clinical trials involving humans.
"We believe that our data suggest the feasibility of this process as a viable therapeutic strategy for Huntington's disease," said Dr. Goldman.
The study was funded by the National Institute of Neurological Disorders and Stroke, the Hereditary Disease Foundation, the CHDI Foundation, and the New York State Stem Cell Research Program.
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