Featured Research

from universities, journals, and other organizations

Therapy for incurable childhood disease alpha-mannosidosis may be in sight

Date:
December 8, 2010
Source:
Christian-Albrechts-Universitaet zu Kiel
Summary:
Alpha-mannosidosis is a rare childhood disease which causes symptoms such as mental retardation or hearing loss and is linked to a lower life expectancy. A European research team has developed a form of therapy as part of the Alpha-Man project, which will receive around six million euros from the European Union for its next stage of trials. This will enable the first clinical trials to be conducted on people.

Typical cell modification in the rare Alpha-mannosidosis with sugar build-up (white).
Credit: © Paul Saftig

Alpha-mannosidosis is a rare childhood disease which causes symptoms such as mental retardation or hearing loss and is linked to a lower life expectancy. A European research team headed by Professor Paul Saftig from Christian-Albrechts-Universitδt zu Kiel (CAU) has developed a form of therapy as part of the Alpha-Man project, which will receive around six million euros from the European Union for its next stage of trials. This will enable the first clinical trials to be conducted on people.

Alpha-mannosidosis is a dangerous hereditary disease which is caused by an enzyme deficiency. This in turn leads sugar to build up in cells, impairing the cell function. "Children who are affected by this disease often appear normal at birth, but their condition worsens increasingly over time. Until now there was no possibility of stopping this development," explains project manager Paul Saftig. "The therapy being developed in our project can drastically increase life expectancy and quality of life."

The Alpha-Man project is building on two earlier project phases in which research was conducted over a period of roughly ten years, initially at the molecule level and most recently with studies on mice. It was possible to develop the enzyme Lamazym as a therapeutic agent in the course of these projects. "Our approach is to introduce the new agent into the patient's bloodstream, from where it is absorbed by the cells and can replace the missing enzyme. Until now, this method of treatment has shown itself to be the most successful," says Kiel co-coordinator Dr Judith Blanz.

The clinical trials on patients with Alpha-mannosidosis are to prove how safe and efficient this method is. As soon as the three-year test phase is over, the researchers hope to be able to make the treatment accessible to all those affected by the disease. At the same time, it may be possible to obtain fundamental knowledge of other neurodegenerative diseases, i.e. slowly progressive diseases of the nervous system, in the event of success.

The forerunner of the Alpha-Man project was the "Euroman" project, which ran from 2001 to 2004 and received 1.6 million euros in support from the European Union. The project was a systematic and multi-discipline approach to research and therapy of the hereditary lysosomal storage disorder Alpha-mannosidosis. This was followed by the Hue-Man project from 2006 to 2009 (Towards the development of an effective enzyme replacement therapy for human Alpha-mannosidosis), which was provided with a total of 3.2 million euros.

The project is a cooperation of organisations from Germany, Denmark, Belgium, England, France, Norway and Poland.

Further information is available at: http://www.alpha-man.eu


Story Source:

The above story is based on materials provided by Christian-Albrechts-Universitaet zu Kiel. Note: Materials may be edited for content and length.


Cite This Page:

Christian-Albrechts-Universitaet zu Kiel. "Therapy for incurable childhood disease alpha-mannosidosis may be in sight." ScienceDaily. ScienceDaily, 8 December 2010. <www.sciencedaily.com/releases/2010/12/101208083533.htm>.
Christian-Albrechts-Universitaet zu Kiel. (2010, December 8). Therapy for incurable childhood disease alpha-mannosidosis may be in sight. ScienceDaily. Retrieved September 2, 2014 from www.sciencedaily.com/releases/2010/12/101208083533.htm
Christian-Albrechts-Universitaet zu Kiel. "Therapy for incurable childhood disease alpha-mannosidosis may be in sight." ScienceDaily. www.sciencedaily.com/releases/2010/12/101208083533.htm (accessed September 2, 2014).

Share This




More Health & Medicine News

Tuesday, September 2, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Snack Attack: Study Says Action Movies Make You Snack More

Snack Attack: Study Says Action Movies Make You Snack More

Newsy (Sep. 2, 2014) — You're more likely to gain weight while watching action flicks than you are watching other types of programming, says a new study published in JAMA. Video provided by Newsy
Powered by NewsLook.com
U.N. Says Ebola Travel Restrictions Will Cause Food Shortage

U.N. Says Ebola Travel Restrictions Will Cause Food Shortage

Newsy (Sep. 2, 2014) — The U.N. says the problem is two-fold — quarantine zones and travel restrictions are limiting the movement of both people and food. Video provided by Newsy
Powered by NewsLook.com
Doctors Fear They're Losing Battle Against Ebola

Doctors Fear They're Losing Battle Against Ebola

AP (Sep. 2, 2014) — As a third American missionary is confirmed to have contracted Ebola in Liberia, doctors on the ground in West Africa fear they're losing the battle against the outbreak. (Sept. 2) Video provided by AP
Powered by NewsLook.com
Tech Giants Bet on 3D Headsets for Gaming, Healthcare

Tech Giants Bet on 3D Headsets for Gaming, Healthcare

AFP (Sep. 2, 2014) — When Facebook acquired the virtual reality hardware developer Oculus VR in March for $2 billion, CEO Mark Zuckerberg hailed the firm's technology as "a new communication platform." Duration: 02:24 Video provided by AFP
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