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 at the Jacksonville, Fla. and Rochester, Minn. campuses.
“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 Demetrius Maraganore, M.D., a Mayo Clinic neurologist.
Previous studies conducted by Dr. Maraganore and Matthew Farrer, Ph.D., 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. Drs. 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. Alnylam is leading the effort to commercialize the Mayo invention using Alnylam RNA interference compounds.
“For this study, we developed a lead compound of small interfering RNAs,” says Heather Melrose, Ph.D., a Mayo Clinic neuroscientist and a lead author of this study. “By infusing this into the brains of mice we were able to effectively reduce the production of alpha-synuclein in the brain. The therapy produced gene silencing that lasted up to three weeks after treatment, and the mice exhibited no ill effects. These are desirable characteristics of a drug therapy ultimately intended to treat disease in humans.”
The study was published this month in Molecular Neurodegeneration. The full news release can be viewed at: http://www.mayoclinic.org/news2008-rst/5087.html.
Image of a mouse brain. The shaded portions represent the 'genetic message' for the alpha-synuclein gene. The right side of the brain was infused with the RNA interference compound and the gene has been effectively silenced.
Drs. Maraganore and Farrer describe this research.
6 Comments
I’m very pleased that you are doing this research and am excited with what you have accomplished. My brother is 69 years old and he is experiencing the beginning of Parkinsons disease, so this finding is very welcome. I am going to forward the link to this article to him this morning.
My mother was diagnosed with Parkinson’s only three years ago.(she has probably had it much longer than that) We just moved her to be closer to my brother in Tampa, Florida. Are there any specialists in the area whom someone would recommend? I believe she needs a fresh look at her disease and what we might be able to do to slow its progression.
Jennifer – and others interested. In Jacksonville, we can offer standard clinical appointment for your mother with our specialist – Dr. Z. Wszolek (the ws are silent). In Tampa, Dr. Hauser is a movement disorders specialist and is referred by Dr. Wszolek. Please contact us at 904-953-2299 for more information.
My 57 year old son has Parkinsons and is on medication. So far his speech is fine and there are days when he walks as normally as anybody else his age. He drives a car. However he sleeps poorly. This, I understand is one of the symptoms of the disease. He uses his stationary bicyle for 1/2 hour a day which he feels is helpful. With new drugs emerging daily and the fond hope that stem cells will soon be available, am I too optimistic that he will start to improve with all the research occuring to stop the disease from progressing.
This seems to me to be along the same lines as the work done earlier at the San Diego UC School of Medicine, whose findings were published in CELLULAR, MOLECULAR AND DEVELOPMENTAL NEUROSCIENCES, vol 19, no 13, August 2008> of course, the latter dealt specifically with MSA, but both studies dealt with the agglomeration of alfa-synuclein as possible causes. The Mayo clinic report gives a means of perhaps dealing with MSA directly by injections into the brain.
Your comments are appreciated.
Warren Smith
Kick-ass post, amazing looking website, added it to my favs!
One Trackback
[...] takes it a step further. Instead of using it simply as a platform for sharing press releases, the institution is using social media to the fullest extent to share stories about the life-changing work it’s doing. In one recent example, the Mayo Clinic posted a [...]