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’s ability to move, think and communicate.

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’s ability to move, think and communicate.

Those affected eventually become totally dependent on others for their care and usually die from complications, such as choking, heart failure or infection. The disease is hereditary; each child of a person with HD has a 50/50 chance of inheriting the fatal gene.

Approximately 200,000 Americans are believed to be at risk of developing HD, a disease that affects as many people as hemophilia, cystic fibrosis or muscular dystrophy. The symptoms of HD typically begin to appear in mid-life, although the progression of the disease varies among individuals and within the same family.

Therapies Lacking

"We welcome the encouraging knowledge generated by Dr. Ellerby's study that FGF2 improves neurological function and longevity in HD transgenic mice," says Carl Johnson, PhD, Executive Director for Science, Hereditary Disease Foundation.

"We look forward to further studies aimed at clarifying how FGF2 protects either through neurogenesis or through direct neuroprotection or both. These are promising studies and should be pursued," Johnson urges.

"Despite recent advances in understanding the pathogenesis of HD, therapeutics that significantly slow or stop the disease are lacking," adds Nancy Wexler, President of the Hereditary Disease Foundation. "We encourage research that facilitates the discovery and development of therapies and cures for Huntington’s disease."