Sarah Goodwin

Kathi Ovnic
Holly Korschun
June 1, 1998

Emory University researchers have been testing a new drug that has demonstrated promising results in treating cystic fibrosis (CF) this country's most common fatal inherited disease among Caucasians. Approximately 1,000 new cases of CF are diagnosed each year in infants, children and young adults, and 30,000 Americans currently are living with the disease. Although the life expectancy for CF patients has risen dramatically within the past 30 years, from 10 years to 31 years, lives are now cut short just as people are settling down to their prime adult years.

The new drug called CPX is one of three promising new drugs that scientists believe may be able to circumvent a defective protein called the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). This protein is present in cells lining the respiratory system and other organs that are responsible for the flow of chloride and sodium. CF is caused by a genetic mutation leading to the defect, which eventually causes an imbalance in the body's salt and water content resulting in a sticky mucus that coats the lungs. Wheezing, coughing and pneumonia become a predictable pattern, along with inflammation and infection. Mucus obstruction of the pancreas leads to digestive problems and poor weight gain.

CPX, along with other new drugs phenylbutyrate and Duramycin, which also appear to be effective in correcting the salt and water imbalance in the lungs, are the first therapies that address the cause of CF rather than just the symptoms. These drugs comprise a new category of treatment strategies termed "gene-assist" therapies, since they treat the cellular defect instead of attempting to replace the defective gene.

A randomized Phase I clinical trial, testing the safety and proper dose level of CPX, was recently completed at Emory and three other centers nationwide in patients ages 18 to 45. A Phase II clinical trial, sponsored by SciClone Pharmaceuticals, Inc., which licenses CPX, is expected to begin later this summer. If the testing is ultimately successful, CPX would be a lifetime maintenance medication administered in pill form.

Emory co-investigators Nael McCarty, Ph.D., assistant professor of physiology and Mark Weatherly, M.D., assistant professor of pediatrics, are directing the clinical trials of CPX at Emory. Other centers include Rainbow Babies & Children's Hospital in Cleveland, the University of Iowa and the University of Washington in Seattle. Emory cystic fibrosis patients are treated through the Egleston Cystic Fibrosis Center at Emory, the first cooperative-care center in the country dedicated to the treatment of cystic fibrosis.

"We are very excited about the potential use of CPX for CF patients, since it addresses the cellular defect exhibited in the largest portion of all CF patients in this country," said Dr. McCarty. "This is an excellent example of how understanding the basic biology of cells in normal subjects and in patients with the disease lays the groundwork for identifying treatment strategies that may save the lives of thousands of individuals."

"I feel CPX is important as one of the first drugs in the new class of "gene assist therapies," Dr. Weatherly said. "Principles learned in this research should apply to therapies for many other genetic diseases."

Editors Note:A patient who participated in this study is available for an interview. Please contact one of the above media representatives.

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