In a study supported by The Michael J. Fox Foundation and the National Institutes of Health, researchers from Columbia University Medical Center (CUMC) have found potential biomarkers and drug targets for the more common, non-inherited form of Parkinson's disease.
In the inherited form of the progressive movement disorder, people have extra copies of the gene for alpha-synuclein, a protein that can damage the nerve cells, particularly in the part of the brain that controls movement. Through carrying out gene mapping and expression analysis, the researchers found that mutations in non-familial Parkinson's disease still affect alpha-synuclein, creating longer versions of the protein, which then accumulate in the brain. The researchers also found that exposure to toxins associated with Parkinson's increased the levels of the abnormal, longer transcript form of alpha-synuclein, suggesting that this could show how both environmental and genetic factors impact the disease.
This elongated version of the protein appears in the blood, and could be used as biomarkers to diagnose the disease, as well as provide a surrogate endpoint in clinical trials.
"There is a tremendous need for a biomarker for Parkinson's, which now can be diagnosed only on the basis of clinical symptoms. The finding is particularly intriguing, but needs to be validated in additional patient groups," said Dr. Asa Abeliovich, of CUMC, in a statement.
Drugs that reduce the accumulation of elongated alpha-synuclein transcripts in the brain could have potential in treating Parkinson's disease, and the research team has identified a number of potential candidates.
- read the press release
- see the abstract