One step closer to a drug treatment for cystic fibrosis

October 12, 2010

A University of Missouri researcher believes his latest work moves scientists closer to a cure for cystic fibrosis, one of the world's most common fatal genetic diseases.

The has published findings by Tzyh-Chang Hwang, a professor in the School of Medicine's Department of Medical Pharmacology and Physiology and the Dalton Cardiovascular Research Center. The publication has been recognized as the "paper of the week" for the journal, meaning Hwang's work is considered to be in the top 1 percent of papers reviewed annually in terms of significance and overall importance.

Hwang's work focuses on the two most common among approximately 1,500 mutations found in patients with . These two mutations cause specific chloride channels in the cell, known as the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) chloride channels, to malfunction. This ultimately leads to repeated pneumonia, the primary cause of most deaths associated with cystic fibrosis.

"The normal function of a cell is to pass across the at a very fast speed," Hwang said. "We know some signaling molecules elicit this reaction, much like a hand signals an automatic water faucet to dispense water. But in the case of cystic fibrosis, that signal is no longer detected by the mutated channel protein. Through some mechanisms we still don't quite understand, malfunction of this channel protein eventually leads to in the lung, which is believed to be responsible for the most severe symptoms of cystic fibrosis."

The most recent study found that manipulating the sensor of the channel protein can significantly rectify the malfunction of the mutated channel, thus opening the door to a drug design that may eventually be a "real cure," Hwang said.

"We could help a lot of patients if we can utilize the power of computer simulations and structure-based drug design to discover new therapeutical reagents for cystic fibrosis, but it's very expensive to do this kind of research in an academic institute," Hwang said.

Explore further: Breathe deep: Which patients could benefit from inhaled steroids in cystic fibrosis?

Related Stories

Defective signaling pathway sheds light on cystic fibrosis

February 14, 2010

In a study that could lead to new therapeutic targets for patients with the cystic fibrosis, a research team from the University of California, San Diego School of Medicine has identified a defective signaling pathway that ...

At last, a living model for an important body channel

October 7, 2010

( -- Ion channels provide a way for key molecules to cross into cells, are the means for many swift physical reactions and regulate the movement of fluid across internal cavities in our bodies. When chloride ions ...

Recommended for you

Moonlighting molecules: Finding new uses for old enzymes

November 27, 2015

A collaboration between the University of Cambridge and MedImmune, the global biologics research and development arm of AstraZeneca, has led researchers to identify a potentially significant new application for a well-known ...

A new form of real gold, almost as light as air

November 25, 2015

Researchers at ETH Zurich have created a new type of foam made of real gold. It is the lightest form ever produced of the precious metal: a thousand times lighter than its conventional form and yet it is nearly impossible ...

Getting under the skin of a medieval mystery

November 23, 2015

A simple PVC eraser has helped an international team of scientists led by bioarchaeologists at the University of York to resolve the mystery surrounding the tissue-thin parchment used by medieval scribes to produce the first ...


Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.