In a major medical advance, scientists have discovered what causes brain cells to die in people with Huntington's disease and six related disorders. The scientists said that in each case an insoluble ball of protein forms in the cell nucleus and kills it. Until now, the cause of cell death in these diseases had not been known. Huntington's disease is a mysterious, inherited malady in which portions of the brain known as basal ganglia atrophy and die.
Victims develop an abnormal gait, as if being drunk, and suffer severe dementia.
The related diseases, which are also inherited, include spinocerebellar ataxia and spinal and bulbar muscular atrophy. They affect different areas of the brain but produce similar symptoms. Researchers said they hoped to learn how to dissolve the balls of protein, thereby delaying or preventing the onset of the disease. We have turned a corner from looking at genes to where we can begin developing real assays for drugs. The findings are exciting for biology, said Dr. Nancy Wexler, president of the Hereditary Disease Foundation, since they provide a common, underlying explanation for all neurodegenerative diseases, including Alzheimer's disease.