An experimental drug targeting a genetic variant linked to Parkinson's disease had no effect on patients, according to the results of a new clinical trial published in The Lancet Neurology.
An experimental drug targeting a genetic variant linked to Parkinson's disease had no effect on patients, according to the results of a new clinical trial published inParkinson's disease, a brain disorder that causes uncontrollable movements such as shaking, stiffness and difficulty with balance, affects nearly one million people in the U.S., according to the Parkinson's Foundation.
Patients with GBA1 mutations may have a more aggressive course of Parkinson's disease. GBA1 is known to control the production of glucocerebrosidase, an enzyme essential for lysosomal function, i.e., breaking down "Parkinson's is not a single-gene disease. However, there are a number of genetic variants that are clearly associated with an increased risk of Parkinson's disease, and GBA1 is very high on the list," said Tanya Simuni, MD, the Arthur C. Nielsen, Jr., Research Professor of Parkinson's Disease and Movement Disorders, director of the Parkinson's Disease and Movement Disorders Center, and a co-author of the study.
"Despite the fact that, biologically, the drug did what it was supposed to do and reduced the level targeted enzyme, the findings of the study were negative," Simuni said."The study was very well designed and executed, and the result gives us confidence to say that the drug did not work because this particular way of modifying the GBA1 pathway does not work, unequivocally.", Simuni said, and future research will focus on targeting the gene in different ways.
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