As many of you know I enjoy keeping up with everything going on in the CF world. I’ve been following the CF drug pipeline and have become very close with one of the top CF specialists in the world. We often e-mail eachother and ofcoure I always have questions to ask him! Here’s some great info:Denufosol: This is the one year long study I participated in from summer 2006 to summer 2007. It’s a P2Y2 agonist. It was taken 3 times a day via a Pari Nebulizer. I was in a trial where you get 6 months placebo or Denufosol followed by 6 months of Denufosol. I benefitted from Denufosol. It’s currently designed and being studied in CF patients with mild to moderate CF lung disease. It hasn’t been studied in severe CF lung disease- YET. The results will be out from the study I participated in summer 2008. If results show Denufosol was effective in the phase 3 clinicial trial it will become available under the Compassionate Use program through the FDA. I will get to be in the compassionate use program because I participated in the phase 3 trial. I’ll keep you posted on this one. It was developed by doctors at my CF center, and the doctor I’m very close with. Denufosol used to be called INS37217 and it’s been in the works for over 10 years.

Bronchitol: It is made by Pharmaxis. It’s being developed and studied in Austrailia. It’s studied in many different lung disease such as Bronchiectassis, COPD, Emphysema and Cystic Fibrosis. Bronchitol is made from a sugar alchohol called Mannitol. Bronchitol,, is a proprietary formulation of mannitol administered as a dry powder in a convenient hand-held inhaler. It is designed to hydrate the lungs, restore normal lung clearance mechanisms, and help patients clear mucus more effectively. Pharmaxis is developing Bronchitol for the management of cystic fibrosis and chronic obstructive lung diseases including bronchiectasis and chronic bronchitis. Bronchitol,, aims to restore the ability of patients’ lungs to clear mucus by promoting salt and water movement through airway cells to the lung surface. Clearing the lungs of mucus and restoring lung hygeine may reduce infections and the consequent risk of scarring. For those with cystic fibrosis, maintaining the airway surface liquid at normal levels may reduce the pulmonary effects of their disease to a point where lung failure is no longer of primary concern. Bronchitol is currently in phase 3 clinicial trials and Pharmaxis has submitted info to the United States FDA. So, shortly after it’s approved in Austrailia it should be available in the USA.

Azetronam Lysine for inhalation: This is an anti-pseudomonal antibiotic. It’s made from the IV antibiotic called Azactam. It was developed by Gilead Sciences (who also recently teamed up with Parion to develope another medication for CF). It’s used by the Pari Eflow Neb. It’s a very small Neb, much like the size of a hamburger. It’s designed to deliver more efficiently than any other Nebulizer on the market. Also, it’s much faster because it breaks down the particle size of the drugs. Eflow was developed special for Azetronam. This medication will take less than 5 minutes to Nebulizer (much better than our current 15 to 20 minute Nebbed treatments each time!). Warning: Pari Eflow was not designed to be used with other medications such as Tobi. If Eflow is used with Tobi you will get a drug overdose and risk ototoxicity to your kidneys and Vestibular system. Gilead Sciences have submitted a New Drug Application to the United States FDA and are waiting approval. Azetronam is currently avaliable through a Compassionate Use Program for CF patients with low lung function, resistant to many other antibiotics and are in need of medications to treat their Pseudomonas. I believe Azetronam is on schedule to be approved early 2008 for Pseudomonas therapy. It can be alternated with the Tobi 28 day cycle if your doctor prescribes it to you. Which would be more power against Pseudomonas infections.

TIP: This is Tobi inhaled Powder. It’s currently being studied to treat Pseudomonas infections in our lungs, but without the LONG Neb treatments that we currently have with Tobi. This is in clinical trials and the powder Tobi is at higher doses than Nebulized Tobi. It’s shown effective against Pseudomonas thus far in studies. However, I’m curious to see if it shows more effective than the Nebbed Tobi since with Nebbed Tobi we have a compressor to push the medication deeper into our lungs compared to the inhaled powder (much like Advair). It’s possible the Tobi Inhaled Powder will eventually replace the Nebulized Tobi depending on if it’s as effective as the Nebbed formulation.

Parion 552- It’s being developed by Parion Sciences which is also developed by CF doctors at my CF center. It was designed to correct the CF ion transport defects by acting primarily on abnormal sodium reabsorption. Recently Parion teamed up with Gilead Sciences (the developers of Azetronam and Pari Eflow) to form a new better formulation of Parion 552 called Parion-680. Studies will begin in humans next summer with this new formulation. This will also be used in conjunction with Hypertonic Saline and according to my source it will be used with the great Pari Eflow Neb to make treatment times very short & more effective.

The next 5 years in Cystic Fibrosis research is going to be the most exciting in years. The reason is because we’ve never had a drug pipeline like the one we have now. The money that we all raise for the Cystic Fibrosis Foundation is really making a difference in our lives. We haven’t had a new drug for Cystic Fibrosis in over a decade. That’s a long time. CF has always had a harder time raising money because there isn’t enough people who know about CF, and it’s not like Cancer where you can just say the word “cancer” and people know what it is and want to help. There’s only 30,000 CF patients in the USA. Approximately 70,000 in the world. However, 2500 babies+ are born with Cystic Fibrosis every year.

I hope I have explained the mentioned CF potential medications so everyone can understand them. I have many CF friends who just don’t understand all this medical terminology so I wanted to break it down for everyone. I don’t know why..but I seem to have a gift with understanding Medical Lingo. So, I wanted to share some info with all my beloved CF family!

Hopefully in the next several years we can add several more years or decades to a CF persons lifespan and add to the average life expectancy. We don’t know what tomorrow will hold, but the CFF is adding tomorrows everyday! We just need to live life to the fullest, enjoy each day, be thankful for the blessings, and dance in the rain!