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 30, 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 30, 2014).

Share This




More Health & Medicine News

Wednesday, July 30, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Health Insurers' Profits Slide

Health Insurers' Profits Slide

Reuters - Business Video Online (July 30, 2014) Obamacare-related costs were said to be behind the profit plunge at Wellpoint and Humana, but Wellpoint sees the new exchanges boosting its earnings for the full year. Fred Katayama reports. Video provided by Reuters
Powered by NewsLook.com
Concern Grows Over Worsening Ebola Crisis

Concern Grows Over Worsening Ebola Crisis

AFP (July 30, 2014) Pan-African airline ASKY has suspended all flights to and from the capitals of Liberia and Sierra Leone amid the worsening Ebola health crisis, which has so far caused 672 deaths in Guinea, Liberia and Sierra Leone. Duration: 00:43 Video provided by AFP
Powered by NewsLook.com
At Least 20 Chikungunya Cases in New Jersey

At Least 20 Chikungunya Cases in New Jersey

AP (July 30, 2014) At least 20 New Jersey residents have tested positive for chikungunya, a mosquito-borne virus that has spread through the Caribbean. (July 30) Video provided by AP
Powered by NewsLook.com
Xtreme Eating: Your Daily Caloric Intake All On One Plate

Xtreme Eating: Your Daily Caloric Intake All On One Plate

Newsy (July 30, 2014) The Center for Science in the Public Interest released its 2014 list of single meals with whopping calorie counts. Video provided by Newsy
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