New Cystic Fibrosis Drug Improves Lung Function
Study Shows Denufosol Delays Changes in Lungs That Lead to Airflow Obstruction
How Denufosol Works continued...
For the next 24 weeks, all participants got denufosol.
The researchers measured exhalation rates and lung volume to assess functioning. They monitored the participants for adverse events such as cough, fever, or sinusitis.
After treatment, the denufosol group had an improvement of 2% in their FEV1 -- forced expiratory volume, a lung function test -- compared to the start of the study. The differences in function between the drug and placebo groups indicated a mild improvement.
The drug did not lead to improvements in other lung function measures or in the rate of infections, a recurring problem for cystic fibrosis patients.
The study, known as TIGER-1, is a phase III trial. Inspire Pharmaceuticals, which markets denufosol, expects to have results from a second phase III trial by early 2011, according to a statement issued by the company.
Accurso was previously a paid consultant for Inspire but currently is not.
The study findings are cause for optimism, according to Bruce Marshall, MD, vice president of clinical affairs for the Cystic Fibrosis Foundation.
He reviewed the study findings for WebMD but was not involved in the study.
"Denufosol is one of the therapies aimed at the root cause in CF," he says via email. "We are optimistic it can keep patients with milder symptoms healthier over the long term."
Another cystic fibrosis expert also finds the results encouraging. "I think the results look promising," Danieli Salinas, MD, a pediatric pulmonologist at Childrens Hospital Los Angeles, who also reviewed the study results.
"The caveat to that is, the results weren't miraculous," she says.
While the 2% is not a large improvement, Salinas puts it in perspective. "In terms of CF, anything we can do to preserve lung function over time, we are taking it."