Brain Research Breakthrough May Lead to Parkinson's Cure

A new discovery linking the blockage of a critical brain pathway to Parkinson’s disease may lead to improved treatments that could potentially reverse the course of the devastating disease.

A new study shows that certain proteins associated with Parkinson’s disease symptoms actually block a critical transport system within the cell.

Researchers say that in people with Parkinson’s and other neurological disorders, these proteins become misfolded and block this biological pathway, producing symptoms such as uncontrollable tremors.

Repairing this pathway may stop or reverse the progression of Parkinson’s disease.

"This gives a whole new direction for understanding what's been going wrong in these patients, and for considering much better strategies for treating people," says researcher Antony Cooper of the University of Missouri, in a news release.

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In the study, published in the journal Science, researchers used cell biology and genetic screening in yeast to isolate and identify the biological pathway blocked by accumulations of misfolded proteins.

The pathway is responsible for preparing and sorting out cellular proteins for secretion and other processes.

Researchers say the findings may also help explain why dopamine-producing brain cells (also known as neurons) are especially susceptible to these abnormal protein accumulations because dopamine is more toxic than other chemical messengers in the brain when not properly transported within cells.

The death of dopamine-producing brain cells lowers dopamine levels in the brain and leads to tremors and other symptoms associated with Parkinson’s disease.

In experiments with laboratory animals, researchers were able to use this discovery to rescue the dying cells, repair the affected pathway, and improve neurological function, which could lead to better treatments for humans with Parkinson’s disease.

"For the first time we've been able to repair dopaminergic neurons, the specific cells that are damaged in Parkinson's disease," says researcher Susan Lindquist of Howard Hughes Medical Institute, in a news release. "These findings are exciting because they tell us we have a platform for discovering new therapeutic strategies and for speeding the process of discovering treatments for these disorders."

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By Jennifer Warner, reviewed by Louise Chang, MD

SOURCES: Cooper, A.Science, June 22, 2006; express online edition. News release, Whitehead Institute for Biomedical Research. News release, Howard Hughes Medical Institute.