Cystic Fibrosis Testing
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The Nemours Aerosol Research Laboratory in Orlando was founded in 1999 to evaluate delivery of inhaled therapies. The delivery of drugs directly to the lungs by the inhaled route improves efficacy and reduces side effects for treatment of lung diseases such as cystic fibrosis (CF).
Many of the new drugs are for treatment of CF lung disease. The CF Foundation estimates that there are more than two-dozen new drugs in development for the disease, and many require inhalation. Some of the new drug entities include inhaled antibiotics, airway wetting and clearance agents, long-acting bronchodilators, antioxidants, gene-modifying agents, and gene replacement therapy.
With so many treatments for CF that require inhalation, the time-burden on the patient is so great that non-adherence with therapy becomes a real problem. Therefore, this laboratory explores efficient new technologies to shorten treatment times and participates in clinical research projects designed to improve treatment adherence and study new therapies.
Nemours is in a unique position to advance the progress in inhaled drug development. Researchers have the capability of testing various drugs and delivery devices in the lab and have the clinical population and experience to carry that research forward into phase 1 through phase 3 testing. This laboratory also has close ties to industry both on the device side and the drug-development side and often acts as an intermediary for companies seeking to find the most efficient way to aerosolize their new drugs, thus achieving the maximum benefit for children (and adults) with chronic lung diseases. With expertise in aerosol science as well as clinical experience, researchers can offer the pharmaceutical and biotech industries significant value-added services that 1) reduce drug-development time, 2) offer new therapies to Nemours patients, and 3) reduce the overwhelming burden of treatment on patients with chronic lung diseases.
Examples of the Aerosol Lab partnering with industry for drug development include the in vitro assessment of delivery devices for Pulmozyme®, tobramycin, levofloxacin and a-1 antitrypsin for CF, and nitrite for pulmonary hypertension. On the clinical side, the lab was the leading enrolling site for phase 1 and 3 studies of inhaled tobramycin powder and phase 1 and 2 studies of inhaled levofloxacin.
The unique strategy employed by the Aerosol Lab for clinical trials has led to success in enrollment and has allowed investigators to participate in the data analysis and present the data from multicenter trials both at meetings and in publications. It opens up new avenues for patients to experience cutting-edge therapies and give them hope for a better tomorrow. It also has allowed Nemours to participate on a national and international level to affect the care of children with CF and respiratory diseases.