Featured Research

from universities, journals, and other organizations

Novel approach shows promise for cystic fibrosis

Date:
November 18, 2010
Source:
University of Alabama at Birmingham
Summary:
An investigational drug targeting a defective protein that causes cystic fibrosis has been shown to improve lung function in a small study of CF patients. The drug, VX-770, appeared to improve function of what is known as CFTR -- the faulty protein responsible for CF. It is among the first compounds being developed for CF that specifically targets the root cause of cystic fibrosis.

An investigational drug targeting a defective protein that causes cystic fibrosis has been shown to improve lung function in a small study of CF patients, according to findings published Nov. 18, 2010, in the New England Journal of Medicine. The investigational drug, VX-770, appeared to improve function of what is known as CFTR--the faulty protein responsible for CF. It is among the first compounds being developed for CF that specifically targets the root cause of cystic fibrosis.

Related Articles


Patients who took VX-770 for 28 days showed improvements in several key indicators of cystic fibrosis, including lung function, nasal potential difference measurements and sweat chloride levels. Excessive sweat chloride is a key clinical indicator of CF. The sweat test is the traditional diagnostic test for CF.

"Patients with CF have a defective protein in chloride channels in lung cells that, in effect, causes a door to shut too tightly, ultimately leading to severe infections in the lung" said Steven M. Rowe, M.D., M.S.P.H, assistant professor of medicine at the University of Alabama at Birmingham and lead investigator at the UAB site, one of 16 study sites nationwide for this VX-770 trial. "The data suggest that the drug seems to improve the function of the protein, so that the door opens and closes more properly." UAB also served as the central coordinating center for nasal potential difference testing, an important outcome measure in the trial.

VX-770 was developed by Vertex Pharmaceuticals, in conjunction with the Cystic Fibrosis Foundation, using a cutting-edge technology known as high-throughput screening, an advanced technique for drug discovery. UAB's Gregory Fleming James Cystic Fibrosis Research Center was the first site in the nation to clinically administer VX-770 to CF patients in the trial in May 2007.

Investigators studied 39 patients with CF in the trial who had a specific mutation in the gene known to cause CF. The primary objective of the trial was to demonstrate the safety and tolerability of VX-770. All patients in the trial completed use of the study drug, and adverse events were similar to placebo.

"Nearly a decade ago, the CF Foundation recognized the need to develop new therapies that address the underlying cause of CF and not just the symptoms of the disease. We are encouraged by the data from this Phase 2 trial and see the trial as a milestone in our efforts to discover and develop new treatment options for this disease," said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation.

VX-770 is currently in Phase 3 clinical trials for cystic fibrosis, and, pending data from these studies, Vertex anticipates submitting a new drug application to the FDA in the second half of 2011.

CF is a fatal genetic disease that affects about 30,000 people in the United States and 70,000 worldwide. It is caused by a genetic mutation that causes life-threatening lung infections and premature death. Ten million Americans are symptomless carriers of a defective CF gene.

Other collaborators on the study are the University of Colorado, Denver and Children's Hospital, Aurora; Johns Hopkins Medical Institutions; University of Minnesota; Hospital for Sick Children and the University of Toronto; University of Iowa; Children's Hospital and Case Western Reserve University School of Medicine, Cleveland; University of North Carolina; Stanford University School of Medicine; University of Pittsburgh; Children's Hospital of Philadelphia and University of Pennsylvania School of Medicine; Children's Hospital Boston and Beth Israel Deaconess Medical Center; University of Washington and Seattle Children's Hospital.


Story Source:

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


Journal Reference:

  1. Frank J. Accurso et al. Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation. New England Journal of Medicine, 2010; 363:1991-2003 [link]

Cite This Page:

University of Alabama at Birmingham. "Novel approach shows promise for cystic fibrosis." ScienceDaily. ScienceDaily, 18 November 2010. <www.sciencedaily.com/releases/2010/11/101117184405.htm>.
University of Alabama at Birmingham. (2010, November 18). Novel approach shows promise for cystic fibrosis. ScienceDaily. Retrieved December 22, 2014 from www.sciencedaily.com/releases/2010/11/101117184405.htm
University of Alabama at Birmingham. "Novel approach shows promise for cystic fibrosis." ScienceDaily. www.sciencedaily.com/releases/2010/11/101117184405.htm (accessed December 22, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Monday, December 22, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Touch-Free Smart Phone Empowers Mobility-Impaired

Touch-Free Smart Phone Empowers Mobility-Impaired

Reuters - Innovations Video Online (Dec. 21, 2014) A touch-free phone developed in Israel enables the mobility-impaired to operate smart phones with just a movement of the head. Suzannah Butcher reports. Video provided by Reuters
Powered by NewsLook.com
Earthworms Provide Cancer-Fighting Bacteria

Earthworms Provide Cancer-Fighting Bacteria

Reuters - Innovations Video Online (Dec. 21, 2014) Polish scientists isolate bacteria from earthworm intestines which they say may be used in antibiotics and cancer treatments. Suzannah Butcher reports. Video provided by Reuters
Powered by NewsLook.com
Existing Chemical Compounds Could Revive Failing Antibiotics, Says Danish Scientist

Existing Chemical Compounds Could Revive Failing Antibiotics, Says Danish Scientist

Reuters - Innovations Video Online (Dec. 21, 2014) A team of scientists led by Danish chemist Jorn Christensen says they have isolated two chemical compounds within an existing antipsychotic medication that could be used to help a range of failing antibiotics work against killer bacterial infections, such as Tuberculosis. Jim Drury went to meet him. Video provided by Reuters
Powered by NewsLook.com
Hugging It Out Could Help You Ward Off A Cold

Hugging It Out Could Help You Ward Off A Cold

Newsy (Dec. 21, 2014) Carnegie Mellon researchers found frequent hugs can help people avoid stress-related illnesses. 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:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

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