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University of Cincinnati Academic Health Center
Publish Date: 04/02/01
Media Contact: AHC Public Relations, (513) 558-4553
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New Treatments Patented by UC to be Tested at Children's in Cincinnati

Cincinnati--A paper on two new cystic fibrosis treatments, that were developed and patented by a team of University of Cincinnati (UC) Medical Center researchers will be presented April 2 at the Experimental Biology 2001 meeting from March 31-April 4. The selected paper, entitled "Human ClC-2 Chloride Channels can be Activated: Potential for Therapy in Cystic Fibrosis" was written by a UC Department of Molecular and Cellular Physiology team of researchers led by John Cuppoletti, PhD, professor in the Department of Molecular and Cellular Physiology and professor in the Department of Internal Medicine.

One in every 2,500 children born to Caucasian parents has cystic fibrosis, the most common genetic disease among white Americans. Cystic fibrosis is characterized by reduced chloride ion permeability, defects in how salt and water are transported across cell linings in the airways, pancreas, intestine, sweat glands and other organs. Since water doesn't transport easily, the respiratory and GI tracts become clogged with mucous. This thick build-up of mucus deposits in the lungs leads to increased susceptibility toward pulmonary infections.

There is no cure, although recent years have seen great improvement in maintenance therapy, including effective treatment of secondary infections and new ways to remove the destructive build up of mucus in the airways. The real trick would be to change how salt and water is transported across the tissues so as to prevent the clogging with mucus. Cuppoletti is trying to circumvent the genetic defect that causes the problem: a cystic fibrosis transmembrane regulatory protein (CFTR) known to be a chloride channel.

At Experimental Biology 2001, Cuppoletti reports finding additional chloride channels in the adult human lung and respiratory tissues and also the discovery (and patenting by UC) of two compounds shown to activate these additional channels. Relatively simple medication treatments may be able to "turn on" these other pathways for chloride ion transport, increasing the salt and water flux in tissues to levels closer to those of normal tissues, thus reducing life-threatening complications.

One of the compounds developed by Cuppoletti (and colleagues) is currently approved for testing in healthy individuals and cystic fibrosis patients at the Cincinnati Children's Hospital Medical Center under the direction of Robert Wilmott, MD, UC professor and director of the Division of Pediatric Pulmonary Medicine. If the compounds are found to be safe and effective during this pilot study, then the Cystic Fibrosis Foundation may fund full-scale clinical trials throughout the country.



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