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 July 23, 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 July 23, 2014).

Share This




More Health & Medicine News

Wednesday, July 23, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Courts Conflicted Over Healthcare Law

Courts Conflicted Over Healthcare Law

AP (July 22, 2014) Two federal appeals courts issued conflicting rulings Tuesday on the legality of the federally-run healthcare exchange that operates in 36 states. (July 22) Video provided by AP
Powered by NewsLook.com
Why Do People Believe We Only Use 10 Percent Of Our Brains?

Why Do People Believe We Only Use 10 Percent Of Our Brains?

Newsy (July 22, 2014) The new sci-fi thriller "Lucy" is making people question whether we really use all our brainpower. But, as scientists have insisted for years, we do. Video provided by Newsy
Powered by NewsLook.com
Scientists Find New Way To Make Human Platelets

Scientists Find New Way To Make Human Platelets

Newsy (July 22, 2014) Boston scientists have discovered a new way to create fully functioning human platelets using a bioreactor and human stem cells. Video provided by Newsy
Powered by NewsLook.com
Gilead's $1000-a-Pill Drug Could Cure Hep C in HIV-Positive People

Gilead's $1000-a-Pill Drug Could Cure Hep C in HIV-Positive People

TheStreet (July 21, 2014) New research shows Gilead Science's drug Sovaldi helps in curing hepatitis C in those who suffer from HIV. In a medical study, the combination of Gilead's Hep C drug with anti-viral drug Ribavirin cured 76% of HIV-positive patients suffering from the most common hepatitis C strain. Hepatitis C and related complications have been a top cause of death in HIV-positive patients. Typical medication used to treat the disease, including interferon proteins, tended to react badly with HIV drugs. However, Sovaldi's %1,000-a-pill price tag could limit the number of patients able to access the treatment. TheStreet's Keris Lahiff reports from New York. Video provided by TheStreet
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