Featured Research

from universities, journals, and other organizations

Device implanted in brain has therapeutic potential for Huntington's disease

Date:
June 21, 2012
Source:
IOS Press BV
Summary:
Studies suggest that neurotrophic factors, which play a role in the development and survival of neurons, have significant therapeutic and restorative potential for neurologic diseases such as Huntington's disease. However, clinical applications are limited because these proteins cannot easily cross the blood brain barrier, have a short half-life, and cause serious side effects. Now, a group of scientists has successfully treated neurological symptoms in laboratory rats by implanting a device to deliver a genetically engineered neurotrophic factor directly to the brain.

The tip of the EC biodelivery system, a straw-like device that is implanted in the brain of patients, contains living cells which are genetically modified to produce a therapeutic factor. The membrane enclosing the cells allows the factor to flow out of the device and into the patient’s brain tissue. This way, areas deep within the brain affected by Huntington’s disease can be treated to delay or prevent the disease.
Credit: Jens Tornøe, NsGene A/S, Ballerup, Denmark

Studies suggest that neurotrophic factors, which play a role in the development and survival of neurons, have significant therapeutic and restorative potential for neurologic diseases such as Huntington's disease. However, clinical applications are limited because these proteins cannot easily cross the blood brain barrier, have a short half-life, and cause serious side effects. Now, a group of scientists has successfully treated neurological symptoms in laboratory rats by implanting a device to deliver a genetically engineered neurotrophic factor directly to the brain.

They report on their results in the latest issue of Restorative Neurology and Neuroscience.

Researchers used Encapsulated Cell (EC) biodelivery, a platform which can be applied using conventional minimally invasive neurosurgical procedures to target deep brain structures with therapeutic proteins. "Our study adds to the continually increasing body of preclinical and clinical data positioning EC biodelivery as a promising therapeutic delivery method for larger biomolecules. It combines the therapeutic advantages of gene therapy with the well-established safety of a retrievable implant," says lead investigator Jens Tornøe, NsGene A/S, Ballerup, Denmark.

Investigators made a catheter-like device consisting of a hollow fiber membrane encapsulating a polymeric "scaffold," which provides a surface area to which neurotrophic factor-producing cells can attach. When implanted in the brain, the membrane allows the neurotrophic factor to flow out of the device, as well as allowing nutrients in. Dr. Tornøe and his colleagues used the neurotrophic factor Meteorin, which plays a role in the development of striatal projection neurons, whose degeneration is a hallmark of Huntington's disease. The scientists engineered ARPE-19 cells to produce Meteorin and used those that produced high levels of Meteorin in their experiment.

The EC biodelivery devices were implanted in the brains of rats followed by injection with quinolinic acid (QA), a potent neurotoxin that causes excitotoxicity, a component of Huntington's disease. They tested three different implant types: devices filled with the high-producing ARPE-19 cells (EC-Meteorin), devices with unmodified ARPE-19 cells (ARPE-19), and devices without cells. Motor dysfunction was tested immediately prior to injection with QA and at two and four weeks after injection.

The research team found that the EC-Meteorin devices significantly protected against QA-induced toxicity. Rats with EC-Meteorin devices manifested near normal neurological performance and significantly reduced loss of brain cells from the QA injection compared to controls. Analysis of the Meteorin-treated brains showed a markedly reduced striatal lesion size. The EC biodelivery devices were found to produce stable or even increasing levels of Meteorin throughout the study. Meteorin diffused readily from the biodelivery device to the striatal tissue.

"Huntington's disease can be diagnosed with high accuracy by genetic testing. Pre-symptomatic administration of a safe therapeutic treatment providing sustained delay or prevention of disease would be of great benefit to patients," says Dr. Tornøe. "With additional functional and safety data, tests in animals larger than the rat to study distribution, and more accurate disease models to evaluate the therapeutic potential of Meteorin, we anticipate that EC biodelivery can be developed as a platform technology for targeted therapy in patients with Huntington's disease."


Story Source:

The above story is based on materials provided by IOS Press BV. Note: Materials may be edited for content and length.


Journal Reference:

  1. J. Tornøe, M. Torp, J.R. Jørgensen, et al. Encapsulated Cell-Based Biodelivery of Meteorin is Neuroprotective in the Quinolinic Acid Rat Model of Neurodegenerative Disease. Restorative Neurology and Neuroscience, 30(2012) 225-275 DOI: 10.3233/RNN-2012-110199

Cite This Page:

IOS Press BV. "Device implanted in brain has therapeutic potential for Huntington's disease." ScienceDaily. ScienceDaily, 21 June 2012. <www.sciencedaily.com/releases/2012/06/120621125552.htm>.
IOS Press BV. (2012, June 21). Device implanted in brain has therapeutic potential for Huntington's disease. ScienceDaily. Retrieved August 2, 2014 from www.sciencedaily.com/releases/2012/06/120621125552.htm
IOS Press BV. "Device implanted in brain has therapeutic potential for Huntington's disease." ScienceDaily. www.sciencedaily.com/releases/2012/06/120621125552.htm (accessed August 2, 2014).

Share This




More Mind & Brain News

Saturday, August 2, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Work Can Be Stressful, But Is Unemployment Worse?

Work Can Be Stressful, But Is Unemployment Worse?

Newsy (Aug. 1, 2014) — A new study shows stress at work can be hard on your health, but people who are unemployed might be at even greater risk of health problems. Video provided by Newsy
Powered by NewsLook.com
Google (Kind Of) Complies With 'Right To Be Forgotten Law'

Google (Kind Of) Complies With 'Right To Be Forgotten Law'

Newsy (July 31, 2014) — Google says it is following Europe's new "Right To Be Forgotten Law," which eliminates user information upon request, but only to a certain degree. Video provided by Newsy
Powered by NewsLook.com
Stroke Signs: Three Hour Deadline

Stroke Signs: Three Hour Deadline

Ivanhoe (July 31, 2014) — Sometimes the signs of a stroke are far from easy to recognize. Learn from one young father’s story on the signs of a stroke. Video provided by Ivanhoe
Powered by NewsLook.com
Grain Brain May Be Harming Us

Grain Brain May Be Harming Us

Ivanhoe (July 31, 2014) — Could eating carbohydrates be harmful to our brain health? Find out what one neurologist says about changing our diets. Video provided by Ivanhoe
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