Featured Research

from universities, journals, and other organizations

Promising new approach to treating debilitating nervous system disease

Date:
October 17, 2011
Source:
Loyola University Health System
Summary:
A groundbreaking study suggests what could become the first effective treatment for a debilitating and fatal disease of the central nervous system called SCA1.

A groundbreaking study in the journal Nature Medicine suggests what could become the first effective treatment for a debilitating and fatal disease of the central nervous system called SCA1.

The study, based on an animal model, found that the disease is linked to low levels of a multipurpose protein called VEGF. Researchers found that in mice that had SCA1, replenishing this protein lead to significant improvements in muscle coordination and balance.

Ameet R. Kini, MD, PhD, an associate professor in the Department of Pathology and Cardinal Bernardin Cancer Center of Loyola University Chicago Stritch School of Medicine, is a co-author of the study.

SCA1 (spinocerebellar ataxia type 1) is an inherited disease caused by a mutation of a single gene. The disease affects 1 to 2 people per 100,000 of population. The first symptoms are usually lack of muscular control of the hands and trouble with balance while walking. Later symptoms can include difficulty swallowing, indistinct speech, neuropathy, spasticity, weakness and memory problems. The disease generally proves fatal within 10 to 30 years, and there currently is no effective treatment, according to the National Ataxia Foundation.

In the first part of their study, researchers conducted several tests that demonstrated that mice with SCA1 had low levels of VEGF (vascular endothelial growth factor). In the brain, VEGF stimulates the growth of blood vessels and works in other ways to keep brain cells healthy.

Researchers then tested whether boosting VEGF levels would benefit mice that had SCA1. They boosted VEGF two ways: By genetically engineering mice to produce more of the protein and by delivering VEGF directly to the brain.

Mice that had SCA1 were given a performance test called Rotarod, which measures balance and coordination. The test, which is somewhat like a log-rolling competition, measures how long a mouse can stay atop a rolling cylinder.

Researchers compared diseased mice that had been treated with VEGF to diseased mice that received no treatment. The VEGF group performed significantly better than the untreated mice -- and in some cases nearly as well as healthy mice that did not have SCA1. In addition, microscopic examinations found significant improvements in the brains of diseased mice that had been treated with VEGF.

The findings suggest that reversing low VEGF levels "may be a potentially useful treatment in patients with SCA1," researchers wrote.

The results also could prove relevant to other ataxia disorders "and possibly even other neurodegenerative syndromes," researchers wrote.

However, Kini cautioned that while the study findings are promising, they do not necessarily apply to humans. It would require clinical trials in patients to prove that replenishing VEGF levels is a safe and effective treatment, Kini said.

The study was jointly conceived and designed by Kini and his colleagues Marija Cvetanovic, PhD, (first author) and Puneet Opal, MD, PhD, (corresponding author) of Northwestern University Feinberg School of Medicine. Other co-authors are Jay M. Patel, BS, of Northwestern and Hugo H. Marti, MD, of the University of Heidelberg.

The study was funded by the National Institutes of Health, National Organization for Rare Disorders, Brain Research Foundation and National Ataxia Foundation.


Story Source:

The above story is based on materials provided by Loyola University Health System. Note: Materials may be edited for content and length.


Journal Reference:

  1. Marija Cvetanovic, Jay M Patel, Hugo H Marti, Ameet R Kini, Puneet Opal. Vascular endothelial growth factor ameliorates the ataxic phenotype in a mouse model of spinocerebellar ataxia type 1. Nature Medicine, 2011; DOI: 10.1038/nm.2494

Cite This Page:

Loyola University Health System. "Promising new approach to treating debilitating nervous system disease." ScienceDaily. ScienceDaily, 17 October 2011. <www.sciencedaily.com/releases/2011/10/111016132040.htm>.
Loyola University Health System. (2011, October 17). Promising new approach to treating debilitating nervous system disease. ScienceDaily. Retrieved April 18, 2014 from www.sciencedaily.com/releases/2011/10/111016132040.htm
Loyola University Health System. "Promising new approach to treating debilitating nervous system disease." ScienceDaily. www.sciencedaily.com/releases/2011/10/111016132040.htm (accessed April 18, 2014).

Share This



More Health & Medicine News

Friday, April 18, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Obama: 8 Million Healthcare Signups

Obama: 8 Million Healthcare Signups

AP (Apr. 17, 2014) President Barack Obama gave a briefing Thursday announcing 8 million people have signed up under the Affordable Care Act. He blasted continued Republican efforts to repeal the law. (April 17) Video provided by AP
Powered by NewsLook.com
Is Apathy A Sign Of A Shrinking Brain?

Is Apathy A Sign Of A Shrinking Brain?

Newsy (Apr. 17, 2014) A recent study links apathetic feelings to a smaller brain. Researchers say the results indicate a need for apathy screening for at-risk seniors. Video provided by Newsy
Powered by NewsLook.com
Could Even Casual Marijuana Use Alter Your Brain?

Could Even Casual Marijuana Use Alter Your Brain?

Newsy (Apr. 16, 2014) A new study conducted by researchers at Northwestern and Harvard suggests even casual marijuana use can alter your brain. Video provided by Newsy
Powered by NewsLook.com
Thousands Of Vials Of SARS Virus Go Missing

Thousands Of Vials Of SARS Virus Go Missing

Newsy (Apr. 16, 2014) A research institute in Paris somehow misplaced more than 2,000 vials of the deadly SARS virus. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins