Researchers at the Mayo Clinic, Rochester, Minn, 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 [removed]Parkinson’s disease[/removed]. 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 Mayo researchers. The study was published this month in Molecular Neurodegeneration.
Parkinson’s disease is a progressive disorder that affects nerve cells in the part of the brain that controls muscle movement. At least 1 million people in the United States are believed to have Parkinson’s disease, and 2% of the population can expect to develop the disease during their lifetime.
"While our research has not yet been tested on humans, we expect that these findings will lead to an effective treatment for slowing or even halting the progression of Parkinson’s disease," says [removed]Demetrius Maraganore, MD[/removed], a Mayo Clinic neurologist.
Previous studies conducted by Maraganore and Matthew Farrer, PhD, a Mayo Clinic neuroscientist, found that variations in the alpha-synuclein gene result in increased protein production and are sufficient to cause Parkinson’s disease in some families, or otherwise increase the risk for Parkinson’s disease across populations worldwide.
Maraganore and Farrer invented a method to treat Parkinson’s disease by reducing alpha-synuclein expression. Mayo Clinic patented and licensed their invention to Alnylam Pharmaceuticals Inc, Cambridge, Mass, which is leading the effort to commercialize the Mayo invention using Alnylam RNA interference compounds.
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"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 Maraganore. "We envision that the therapy would be delivered through 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. The researchers expect that RNA interference therapy could be beneficial for patients with those conditions.
The study was funded in part by a grant from the Michael J. Fox Foundation. Mayo Clinic has an agreement with Alnylam whereby Mayo has granted Alnylam an exclusive license to certain patents and know-how. Mayo Clinic and inventors of this intellectual property may receive developmental milestone and/or royalty payments pursuant to the agreement.
[Source: Medical News Today]