Inhaled antibiotic for cystic fibrosis patients debuts
The first inhaled antibiotic approved by the U.S. Food and Drug Administration went on the market this month, offering a new treatment option to patients with cystic fibrosis.
"We're excited about this new drug because it gives hope for patients with cystic fibrosis that they can live longer and more meaningful lives," said Dr. Preston W. Campbell, associate professor of Pediatrics and medical director of the Cystic Fibrosis Treatment and Research Center at Vanderbilt Children's Hospital.
Campbell, one of two physician witnesses who testified before the FDA during the drug's approval process, chaired a Cystic Fibrosis Foundation consensus committee that developed a nearly 50-page document outlining how cystic fibrosis specialists would use the medication among their patients.
The drug, tobramycin or "tobi" for short, is the first antibiotic designed to be inhaled. While the FDA has approved its use in CF patients, the concept of inhaled antibiotics may be used in the treatment of other diseases, Campbell said.
A second, potentially even more effective inhaled antibiotic is expected to be tested in CF patients soon, he said.
The most important advantage of tobi is that it delivers high doses of medication directly to the site of infection in the lung, Campbell said. That improves action of the drug against the infection and reduces the risk that the bacteria will develop resistance against the medication.
In addition, aerosol delivery of the medication eliminates the side effects ‹ most commonly hearing and kidney damage ‹ seen with systemic administration of tobramycin, Campbell said.
In clinical trials, the new drug resulted in an average 15 percent improvement in lung function, which can make a substantial difference in quality of life, he said.
"The massive degree of infection that these patients often have in their lungs makes them feel truly terrible," Campbell said.
"Tobi is not a cure ‹ we're still working on a cure through other approaches ‹ but it is a life extender and a quality of life improver for patients with cystic fibrosis."
Cystic fibrosis is the most common, serious inherited disease among the white population, appearing in about 1 in 2,500 live births, Campbell said. One in 20 adults carries the defective gene.
The disease is characterized by recurrent serious lung infection, along with subsequent progressive lung deterioration, and failure to thrive.
Over the past two decades, improved understanding of the disease and corresponding advances in treatment have changed the demographics of the disease. As recently as the 1970s, children with CF could expect to live only until about age 10. Today, the average CF patient can expect to live to age 30 and beyond, Campbell said.
"In the year 2000, half of the patients with CF will be adults," Campbell said.
Now that tobi is available, Campbell said that many of Vanderbilt's patients will be transitioned onto the new drug.
"We expect about 80 of our patients will go on the new drug over the next few months," he said.