In the US, Huntington's Disease affects as many people as hemophilia, cystic fibrosis or muscular dystrophy. Typically characterized by involuntary movements and dementia, HD slowly diminishes a person

The research is published in the Proceedings of the National Academy of Sciences (PNAS).

The findings highlight a potential new therapeutic approach for the degenerative brain disorder, which affects approximately 30,000 Americans.

Extended Lifespan

The mice were genetically engineered to develop HD and then treated with a protein called fibroblast growth factor-2, or FGF-2, which previously has been shown to increase the growth of new blood vessels in human clinical trials.

The use of FGF-2 resulted in a 150 percent increase in new cells in brains of the HD mice, compared to a 30 percent increase in mice that were not genetically engineered. Treatment extended the lifespan of the affected mice by 20 percent.

The animals also exhibited improved motor performance, decreased cell death, and a reduction in the amount of toxic aggregates that typically form in the brains of those affected by HD.

Understanding Neurogenesis

"Efforts to understand and encourage neurogenesis, the growth of new neurons, comprise an emerging area of study as we explore potential treatments for neurodegenerative diseases," says Lisa Ellerby, PhD, lead scientist of the study.

"In this case, the new brain cells migrated to the area of the brain affected by Huntington's disease and assumed the features of the type of neuron commonly lost in HD," Ellerby notes.

The FGF-2 was administered subcutaneously (by injection under the skin) to the mice, indicating that the protein can cross the blood-brain barrier, another factor that shows promise in the development of new therapies for the disease, according to Ellerby.

200,000 Americans at Risk

There is currently no effective treatment or cure for HD, which is typically characterized by involuntary movements and dementia. The disease slowly diminishes a person