June 25, 2004

Results of new CF drug study offer hope to young patients

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Tate Wilson, a patient at Vanderbilt Children’s Hospital, shown here with his mother, Tracey LenFant, was one of the participants in a study to test a new drug to improve lung function in people with cystic fibrosis. (photos by Dana Johnson)

Results of new CF drug study offer hope to young patients

Vanderbilt Children’s Hospital participated in a study late last year to test a new drug to improve lung function in people with cystic fibrosis (CF). The results are now in, and show the drug has promise as a new type of therapy for the chronic and life-threatening disease.

The new therapy is an inhaled drug manufactured by Inspire Pharmaceuticals Inc. Patients at Children’s Hospital who participated in the study were given either the drug or a placebo to inhale three times a day for a month.

Tate Wilson, an 11-year-old from Smyrna, was one of the participants.

“It wasn’t hard to participate,” Wilson said. “It was a nebulizer treatment, but then I had to write down notes, and that wasn’t so easy.”

Ninety subjects were enrolled in the multi-center, nationwide trial, including five at Children’s Hospital. The participants were divided into five groups according to the dose being studied. The study was designed to find out if the new drug is well tolerated at different doses.

The results showed that each dose was well tolerated, but beyond that, those who took the drug had better lung function after using the drug for four weeks than those who took the placebo.

Chris Harris, M.D., primary investigator for the study at Children’s Hospital, said the results are exciting because it is the first drug to target the base defect in the lungs of CF patients.

“The gene defect in CF causes an important chloride channel in cells lining in the airways to remain closed. That prevents good flow of moisture, and in turn prevents the proper flow of mucous,” said Harris, who is a pulmonologist and assistant professor of Pediatrics at Vanderbilt Children’s Hospital. “[The new drug therapy] opens up a different chloride channel to allow better flow of moisture, and so, better flow of mucous out of the lungs.”

The results were intriguing enough to cause the Cystic Fibrosis Foundation to comment on this early study, and encourage participation in the next phase which should start in the fall.

“We are delighted with these results, which far surpassed the success criteria that were set forth as part of the agreement between Inspire and CFFT [Cystic Fibrosis Foundation Therapeutics, Inc.] to do the study,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation and CFFT. “We are very pleased that Inspire will be moving forward with this exciting potential product, which addresses the basic CF defect.”

For Wilson, finding a way to keep his airways flowing is a matter of life and death. Infections in his sinuses and lungs already plague him.

Wilson’s mother, Tracey LenFant, said she agreed to enroll her son in the study because of the hope that it might help him, or others like him.

“We’ve had 11 years to deal with this,” LenFant said. “We had to sign a seven or eight page release form, but still I thought it was safe. The safety phase of the trial study was completed. You feel a responsibility to those coming up behind him. Hope comes from knowledge, the more knowledge you have, the more hope.”

The trial paid $475 to each participant. Wilson is an honest child, so when he was asked why he’d agreed to take part in a study for an experimental drug therapy, he was quick to say: “For the money!”

Once the study wrapped up in December, Wilson took his money and bought a new pet, an albino corn snake named Roxy.

Wilson doesn’t know yet if he took the drug or the placebo, but he does say he didn’t notice that much difference in his lung function during the study.

The most common adverse event in taking the new drug therapy was cough, which occurred in 46 percent of subjects overall and was comparable across all groups, including placebo.

Inspire Pharmaceuticals plans to develop the new drug therapy as an early intervention therapy. The company has received Orphan Drug Status and Fast Track designations from the Food and Drug Administration (FDA) for the new drug therapy for the CF indication.

In cystic fibrosis, a genetic defect leads to a set of circumstances that make it easier for dangerous bacteria to grow in the lungs. Repeated bouts of infection lead to lung scarring and resistant bacteria, a combination that ultimately proves deadly for many people with CF. Currently, the average life expectancy for someone with cystic fibrosis is about 40 years.