The following excerpts appeared originally in a press release on Mayo Clinic's website. Click here to view the full article.
"Mayo Clinic researchers have developed a method to reduce the production of alpha-synuclein in the brain. Alpha-synuclein is a protein that is believed to be central to the cause of Parkinson's disease. All patients with Parkinson's disease have abnormal accumulations of alpha-synuclein protein in the brain.
"The new method involves the delivery of RNA interference compounds directly to selected areas of the brain via injection. The RNA interference compounds silence the gene that produces alpha-synuclein, according to the Mayo researchers. The study was published this month in Molecular Neurodegeneration..."
"Our next step with this research is to test the therapy in mice and primates with experimental forms of Parkinson's disease and prove that we are able to stop the disease progression in those animals," says Dr. Farrer. "We are hopeful, as preliminary studies suggest this is possible."
"It is important to note that there are significant hurdles to this therapy. The compound needs to be directly delivered to the brain through a neurosurgical procedure — it cannot be given by mouth or injection into a vein," says Dr. Maraganore. "We envision that the therapy would be delivered through Food and Drug Administration (FDA)-approved devices currently used for deep brain stimulation therapy. The deep brain stimulation would treat the existing symptoms of Parkinson's disease, while the administration of the RNA interference compounds might halt the progression of the disease..."
"Alpha-synuclein protein also accumulates abnormally in other brain degenerations, including multiple system atrophy (MSA) and dementia with Lewy bodies. Therefore, Mayo researchers expect that RNA interference therapy could be beneficial for patients with those conditions."