Clinical Trials: A Vital Part of Cystic Fibrosis Treament Advancements

Clinical trials are vital part of advancing treatments for CF patients. Fundraising is the genesis for new treatments because it allows researchers to create new treatments, but clinical trials are the bridge from a breakthrough in the lab to a new medicine actually being available on the open market. It is the often overlooked last step in getting a new medication approved by the FDA for the open market. As a consequence, new medications languish for extended periods because the researchers cannot recruit enough participants in the clinical trials.

As a 30 year old with CF I recognize the importance of clinical trials as I have been alive to see many great breakthroughs for CF in my lifetime. I have participated in various clinical / research trials in CF for over two decades. Some of these studies have involved mucociliary clearance, drug studies, observational studies, genetic marker studies and early intervention studies. Currently, I am participating in a study drug clinical trial and thrilled with the ease of taking the study medication.

The CFF drug development pipeline has never looked as promising as it does today. There are several medications with great potential being studied in CF patients. There are studies ranging from CFTR modulation which Vertex is currently leading. Vertex’s Kalydeco was successfully approved after extensive clinical trials in CF patients with G551D mutation. Vertex also has VX-809+Ivacaftor (Kalydeco) and VX-661+Ivacaftor (Kalydeco) in trials for CF patients who are homozygous F508del as well as those who are heterozygous f508del.

Additionally, there are first-ever inhaled antibiotic therapies being studied in CF patients. For example, Savara Pharma’s (www.aerovanc.com) lead product, inhaled dry powder Aero Vanc, is being studied in CF patients who culture MRSA. Aero Vanc could be a huge advancement in the treatment of MRSA CF lung infections. Currently, there are no commercially available inhaled antibiotics for MRSA. Subsequently, those with CF and MRSA currently have to take very strong oral and IV antibiotics for every exacerbation. For those with Pseudomonas, inhaled Levofloxacin and Arikace are also in development in clinical trials. These new inhaled antibiotic therapies have potential in lessening the bacterial burden in CF patients’airways. Thus, leading to reduced airway infections and subsequent irreversible lung damage.

Participating in clinical trials is not as challenging as one may think. Patients are reimbursed for study participation, travel (mileage), parking, food consumed during study visits and sometimes overnight stays at a hotel depending upon distance traveled by patient. The study sponsors do their best to make study participation less inconvenient to CF patients and their families.

Great sources for online searches of CF clinical trials are www.clinicaltrials.gov, www.cff.org drug pipeline, and company websites. A great example of a patient-friendly company website with easy-to-navigate study site info is Savara Pharma’s www.aerovanc.com. Savara makes it easy for patient’s to select their state and click on the pin to obtain contact info for the study coordinator and doctor conducting their trial. Obtaining study site contact information is easier than ever thanks to the internet and especially social media.

Best of luck to you in your efforts of participation in CF research. Just remember to always consult your CF doctor prior to screening or joining any clinical trials.