Featured Research

from universities, journals, and other organizations

Treatment Of Cystic Fibrosis: Encouraging New Results For Miglustat

Date:
July 22, 2009
Source:
CNRS (Délégation Paris Michel-Ange)
Summary:
Miglustat is a drug currently under phase 2 clinical trials on patients suffering from cystic fibrosis. Its potential for treating the disease was discovered in 2006 and researchers can now show that daily, long-term treatment of human cystic fibrosis cells with low doses of miglustat corrects the main pathological abnormalities. They are therefore extremely hopeful that miglustat will prove effective with patients, and become the first drug able to treat the disease rather than the symptoms.

Miglustat is a drug currently under phase 2 clinical trials on patients suffering from cystic fibrosis (1). Its potential for treating the disease was discovered in 2006 thanks to the work of Frédéric Becq's team at the Institute of Cell Physiology and Biology (CNRS/Université de Poitiers), funded by the associations Vaincre la Mucoviscidose, MucoVie66, La Pierre Le Bigaut and ABCF2.

In new work to be published on 1 August 2009 in the American Journal of Respiratory Cell and Molecular Biology, the researchers show that daily, long-term treatment of human cystic fibrosis cells with low doses of miglustat corrects the main pathological abnormalities. They are therefore extremely hopeful that miglustat will prove effective with patients, and become the first drug able to treat the disease rather than the symptoms.

Cystic fibrosis is a genetic disease, transmitted jointly by both parents, and affects around 6000 people in France. It is caused by the dysfunction of a membrane protein (CFTR), present especially in the epithelial cells in the lungs, which controls exchange of water and mineral salts between the cell and the exterior. On the cell level, the disease manifests itself by the absence of chloride secretion, sodium hyperabsorption, deregulation of calcium homeostasis, and heightened inflammatory response. This results in thickening of the mucus that lines the bronchial tubes and the pancreatic ducts, leading to lung infections and digestive disorders. At the current time, there is no treatment that cures cystic fibrosis. In order to alleviate the symptoms, extremely strict daily treatment is necessary.

In 2006, Frédéric Becq's team at the Institute of Cell Physiology and Biology (CNRS/Université de Poitiers) showed that a drug called miglustat restored the activity of the CFTR protein and could thus temporarily correct the specific phenotype characteristic of cystic fibrosis. Used to treat two rare diseases (Gaucher's disease and Niemann-Pick type C disease), its safety and tolerance had already been assessed, and clinical trials could be rapidly begun in September 2007.

In the new study published in American Journal of Respiratory Cell and Molecular Biology, the researchers show that daily treatment of human respiratory cells that are homozygous for the F508del mutation with low concentrations of miglustat leads to progressive, sustained and reversible correction of the diseased phenotype. The researchers cultured diseased human respiratory cells in the presence of miglustat for two months. The correction observed in the cells takes place after 3-4 days, and then stabilizes. When the treatment is stopped, the cells revert to the diseased phenotype. The low doses used (3 micromolars) mean that they can be administered to patients and that their presence in the bloodstream causes no problems.

This study is the first that shows that a cystic fibrosis cell can acquire a sustained non-diseased phenotype when treated daily with a pharmacological agent. The researchers are therefore very optimistic about the results of the clinical trials under way.

(1) The clinical study is being carried out by the Actelion pharmaceutical laboratory on 15 patients suffering from cystic fibrosis and carrying the delta F508 mutation (F508del), which is the most common and the most serious of the mutations affecting children with cystic fibrosis. The results will be known in the coming weeks.


Story Source:

The above story is based on materials provided by CNRS (Délégation Paris Michel-Ange). Note: Materials may be edited for content and length.


Journal Reference:

  1. C. Norez, F. Antigny, S. Noel, C. Vandebrouck, F. Becq. A CF respiratory epithelial cell chronically treated by miglustat acquires a non-CF like phenotype. American Journal of Respiratory Cell and Molecular Biology, August 2009

Cite This Page:

CNRS (Délégation Paris Michel-Ange). "Treatment Of Cystic Fibrosis: Encouraging New Results For Miglustat." ScienceDaily. ScienceDaily, 22 July 2009. <www.sciencedaily.com/releases/2009/07/090721090134.htm>.
CNRS (Délégation Paris Michel-Ange). (2009, July 22). Treatment Of Cystic Fibrosis: Encouraging New Results For Miglustat. ScienceDaily. Retrieved July 24, 2014 from www.sciencedaily.com/releases/2009/07/090721090134.htm
CNRS (Délégation Paris Michel-Ange). "Treatment Of Cystic Fibrosis: Encouraging New Results For Miglustat." ScienceDaily. www.sciencedaily.com/releases/2009/07/090721090134.htm (accessed July 24, 2014).

Share This




More Health & Medicine News

Thursday, July 24, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

New Painkiller Designed To Discourage Abuse: Will It Work?

New Painkiller Designed To Discourage Abuse: Will It Work?

Newsy (July 24, 2014) — The FDA approved Targiniq ER on Wednesday, a painkiller designed to keep users from abusing it. Like any new medication, however, it has doubters. Video provided by Newsy
Powered by NewsLook.com
Doctor At Forefront Of Fighting Ebola Outbreak Gets Ebola

Doctor At Forefront Of Fighting Ebola Outbreak Gets Ebola

Newsy (July 24, 2014) — Sheik Umar Khan has treated many of the people infected in the Ebola outbreak, and now he's become one of them. Video provided by Newsy
Powered by NewsLook.com
Condemned Man's US Execution Takes Nearly Two Hours

Condemned Man's US Execution Takes Nearly Two Hours

AFP (July 24, 2014) — America's death penalty debate raged Thursday after it took nearly two hours for Arizona to execute a prisoner who lost a Supreme Court battle challenging the experimental lethal drug cocktail. Duration: 00:55 Video provided by AFP
Powered by NewsLook.com
China's Ageing Millions Look Forward to Bleak Future

China's Ageing Millions Look Forward to Bleak Future

AFP (July 24, 2014) — China's elderly population is expanding so quickly that children struggle to look after them, pushing them to do something unexpected in Chinese society- move their parents into a nursing home. Duration: 02:07 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