Featured Research

from universities, journals, and other organizations

Insights into new therapy for rare form of cystic fibrosis

Date:
October 29, 2012
Source:
American Society for Biochemistry and Molecular Biology
Summary:
Scientists have established that a drug recently approved by the US Food and Drug Administration to treat a rare form of cystic fibrosis works in an unconventional way. Their results reveal new possibilities for treating various forms of cystic fibrosis.

Scientists at the Hospital for Sick Children in Toronto have established that a drug recently approved by the U.S. Food and Drug Administration to treat a rare form of cystic fibrosis works in an unconventional way. Their results reveal new possibilities for treating various forms of cystic fibrosis.

Related Articles


Cystic fibrosis is an inherited disease afflicting about 70,000 people around the world. Cystic fibrosis patients carry a defective gene that disables or destroys its protein product, which normally regulates the transport of ions across cell borders. When that transport is disrupted, the viscosity of the mucus coating certain organs becomes too thick. A characteristic feature of the disease is thick mucus buildup in the air passages, which causes difficulty breathing and recurring infections.

While the FDA approved the drug VX-770 (also known by the trade names Kalydeco and Ivacaftor) to ease breathing in people with cystic fibrosis caused by a particular mutation in the CFTR protein (the acronym is short for cystic fibrosis transmembrane conductance regulator), exactly how VX-770 worked in those patients was unknown.

Scientists have understood for some time that normal CFTR regulation requires modification of the protein and binding of a small, energy-providing molecule -- adenosine triphosphate, or ATP. But, in their recent Journal of Biological Chemistry "Paper of the Week," Christine Bear and colleagues report that the drug opens both normal and mutant CFTR channels without ATP. Their results indicate that the compound binds to a different site on CTFR than ATP. Significantly, this finding may be useful in developing therapies for cystic fibrosis caused by various CFTR mutations that, like the G551D mutation that was studied, impair ATP-mediated channel regulation.

Bear's group determined how VX-770 works after developing a new experimental system that may have potential for discovering drugs that target the basic defects caused by CFTR mutations, Bear says. The system is useful for identifying compounds that interact with rare mutations such as G551D as well as the major CFTR mutant F508del, she said.


Story Source:

The above story is based on materials provided by American Society for Biochemistry and Molecular Biology. The original article was written by Danielle Gutierrez. Note: Materials may be edited for content and length.


Journal Reference:

  1. P. D. W. Eckford, C. Li, M. Ramjeesingh, C. E. Bear. Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiator VX-770 (Ivacaftor) Opens the Defective Channel Gate of Mutant CFTR in a Phosphorylation-dependent but ATP-independent Manner. Journal of Biological Chemistry, 2012; 287 (44): 36639 DOI: 10.1074/jbc.M112.393637

Cite This Page:

American Society for Biochemistry and Molecular Biology. "Insights into new therapy for rare form of cystic fibrosis." ScienceDaily. ScienceDaily, 29 October 2012. <www.sciencedaily.com/releases/2012/10/121029082417.htm>.
American Society for Biochemistry and Molecular Biology. (2012, October 29). Insights into new therapy for rare form of cystic fibrosis. ScienceDaily. Retrieved March 3, 2015 from www.sciencedaily.com/releases/2012/10/121029082417.htm
American Society for Biochemistry and Molecular Biology. "Insights into new therapy for rare form of cystic fibrosis." ScienceDaily. www.sciencedaily.com/releases/2012/10/121029082417.htm (accessed March 3, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Tuesday, March 3, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Mom Triumphs Over Tragedy, Helps Other Families

Mom Triumphs Over Tragedy, Helps Other Families

AP (Mar. 3, 2015) After her son, Dax, died from a rare form of leukemia, Julie Locke decided to give back to the doctors at St. Jude Children&apos;s Research Hospital who tried to save his life. She raised $1.6M to help other patients and their families. (March 3) Video provided by AP
Powered by NewsLook.com
Woman Convicted of Poisoning Son

Woman Convicted of Poisoning Son

AP (Mar. 3, 2015) A woman who blogged for years about her son&apos;s constant health woes was convicted Monday of poisoning him to death by force-feeding heavy concentrations of sodium through his stomach tube. (March 3) Video provided by AP
Powered by NewsLook.com
Treadmill Test Can Predict Chance Of Death Within A Decade

Treadmill Test Can Predict Chance Of Death Within A Decade

Newsy (Mar. 2, 2015) Johns Hopkins researchers analyzed 58,000 heart stress tests to come up with a formula that predicts a person&apos;s chances of dying in the next decade. Video provided by Newsy
Powered by NewsLook.com
Going Gluten-Free Could Get You A Tax Break

Going Gluten-Free Could Get You A Tax Break

Newsy (Mar. 2, 2015) If a doctor advises you to remove gluten from your diet, you could get a tax deduction on the amount you spend on gluten-free foods. 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