The annual CFF Meeting that Tricia and I attended last week took place at Inspire Pharmaceuticals, Inc. which recently initiated a second Phase 3 clinical trial evaluating denufosol tetrasodium inhalation solution for the treatment of Cystic Fibrosis. A few of the Inspire doctors/scientists spent several minutes explaining this new drug.
The basic issue that CF presents in the lungs is that it causes the thin layer of hydration that keeps normal lungs wet to dry up and become thick and sticky. Normally, this mucus is such that it is able to catch most dust, germs and other foreign stuff in your lungs and quickly move it out of your body. With CF, the mucus is so thick that most of what you breathe in never makes it back out. The mucus traps a lot of junk, and slowly becomes thicker and thicker. This causes a few major problems (and, understand that the following is an extremely simplistic way of explaining...only because it's ultimately too complicated for my brain to comprehend):
1) The thick mucus traps germs and bugs and bacteria and fungus which, if given enough time, can result in some really nasty/harmful sickness. This is why most CFer's become very sick fairly often.
2) The mucus also slowly begins to block the lungs natural ability to both absorb the O2 and get rid of the CO2 from the air being breathed in. This is why most CFer's have difficulty breathing.
Denufosol enhances the hydration and clearance of mucus from the lungs by helping chloride move across the cell membranes of the lungs. This new trial, named TIGER-2 (Transport of Ions to Generate Epithelial Rehydration), is enrolling 350 people with CF aged 5 years and older in the United States and Canada and at additional international sites as the trial proceeds. The trial aims to demonstrate improved lung function and decreased pulmonary exacerbations, and measure the need for other CF medications and quality of life.
So far, the results are extremely promising!
Inspire is one of the few pharmaceutical companies in the US that was organized to specifically develop drugs for Cystic Fibrosis. The CFF has been partnering with Inspire since it's beginning, and is one of the companies that receives funding from the CFF, which, of course, comes from people like you who generously give to help find a cure.
Inspire explained that, after Denufosol hits the market (assuming the trials go well and it is approved for manufacturing and distribution), their next major goal is to find the cure. But, the only way that they'll ever get that far is with additional financial support from the people who give to the CFF.
Please, if you haven't yet, consider joining us as we search for a cure by Donating to the Cystic Fibrosis Foundation.