The cystic fibrosis drug development pipeline is more robust than ever. Currently, there are more than 25 potential therapies in clinical trials that address the symptoms and root cause of this disease, including three new CF therapies now in Phase 3 trials. With this increase in the number of potential therapies, researchers face an increasing need for volunteers who can participate in clinical trials and help move new compounds from clinic to patient. The need for clinical trials participants has doubled in the last year.
The following three therapies are in Phase 3 of development and need more clinical trials participants:
Denufosol (formerly INS37217) — Developed by Inspire Pharmaceuticals, with CF Foundation support, this drug stimulates the movement of chloride in and out of CF cells by circumventing the faulty CFTR protein channel. In earlier trials, inhaled denufosol also demonstrated improvement in lung function, as well as increased mucus clearance.
Aztreonam — A known intravenous antibiotic for many years, aztreonam has been reformulated as an aerosolized medication for CF airways. This drug, developed by Gilead Sciences, Inc., is a much-needed new type of inhaled antibiotic for individuals colonized with Pseudomonas aeruginosa.
Tobramycin Inhalation Powder (TIP) — A new dry powder form of inhaled tobramycin is being tested by Novartis Pharmaceuticals to determine its safety and effectiveness. (The commonly used drug TOBI ® is a liquid form of tobramycin.)
To find out more about participating in these promising studies and the more than 20 others that are underway throughout the country, check with your local CF Foundation care center. Information about the entire CF Foundation drug development pipeline is also available on our Web site, www.cff.org, along with a recently aired Web cast on current clinical research: “Hope for the Future: Update on CF Clinical Research.”