Featured Research

from universities, journals, and other organizations

New Drug Hope For Cystic Fibrosis Patients

Date:
September 10, 2008
Source:
University of Bristol
Summary:
A new drug therapy may represent a tremendous step forward in the treatment of some 70,000 cystic fibrosis patients worldwide. One of the researchers said, "The early results with VX-770 suggest that drug therapies which target defects at the root of the disease have the potential to improve greatly the quality of life of CF patients."

A new drug therapy may represent a tremendous step forward in the treatment of some 70,000 cystic fibrosis (CF) patients worldwide, Dr David Sheppard from the University of Bristol told an audience at the BA Festival of Science in Liverpool on September 9.

Speaking at the conference, Dr Sheppard said: ‘The early results with VX-770 suggest that drug therapies which target defects at the root of the disease have the potential to improve greatly the quality of life of CF patients.’

At the moment there is no cure for CF – a common single-gene disorder in the UK. The disease, which affects about 8,000 people in the UK and 70,000 people worldwide, is due to a defective gene that causes ducts and tubes in the body to become blocked by thick, sticky mucus. This mucus affects the lungs, pancreas, the intestines, the liver and the reproductive organs.

One of the most recognisable symptoms of CF is ‘salty sweat’, caused by the failure of the sweat ducts to reabsorb salt. Existing treatments only alleviate symptoms, for example, physiotherapy to clear the air passages, antibiotics for lung infections and enzymes to aid the digestion of food.

The defective gene disables or destroys a protein known as CFTR. To date around 1,500 genetic defects have been found in this protein. In general, genetic defects cause harm in two ways – some stop the protein from travelling to its correct destination in cells, whereas others prevent the protein from working properly.

Dr Sheppard’s research group, supported by the Cystic Fibrosis Trust, investigates how new drugs restore function to defective CFTR proteins and EuroCareCF, of which Dr Sheppard is the coordinator, works to promote CF therapy development in Europe.

The new drug therapy (VX-770) was developed by Vertex Pharmaceuticals and will tackle the ‘functional’ defect. It has been tested on CF patients in the US who carry a genetic defect known as G551D. European trials are expected in the future. Early results are very encouraging. Patients who received 150mg twice a day saw the concentration of salt in their sweat decrease by almost 50 per cent and lung function improve by 10 per cent.

The Cystic Fibrosis Foundation funded the discovery and early development work of VX-770 in collaboration with Vertex Pharmaceuticals. To date the Foundation has invested $79 million in the project. Results of the trials can be found here. Dr Sheppard’s research group and other academic groups and companies are also working to develop new drugs that tackle defects at the root of CF.

Current life expectancy for CF patients is between 30 and 40 years of age in Western Europe and the US but is considerably lower in other parts of the world. One in 25 people in the UK is a carrier of a defective CF gene, making CF one of the most common life-shortening, childhood-onset, inherited diseases.


Story Source:

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


Cite This Page:

University of Bristol. "New Drug Hope For Cystic Fibrosis Patients." ScienceDaily. ScienceDaily, 10 September 2008. <www.sciencedaily.com/releases/2008/09/080909074100.htm>.
University of Bristol. (2008, September 10). New Drug Hope For Cystic Fibrosis Patients. ScienceDaily. Retrieved September 2, 2014 from www.sciencedaily.com/releases/2008/09/080909074100.htm
University of Bristol. "New Drug Hope For Cystic Fibrosis Patients." ScienceDaily. www.sciencedaily.com/releases/2008/09/080909074100.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