I’m Drowning – A researcher-patient’s plea for broader inclusion in cystic fibrosis trials

By: Ella Balasa

I’ve always known cystic fibrosis (CF) is a progressive disease; it destroys lung cells, tightens the small airways in the bottom of my chest, and each day takes me closer to the time when it will have ravaged my lungs. I had never really questioned if there was some way this process could be altered. I accepted that it couldn’t.

Recently, however, this has changed. The epicenter of new CF research is the development of medications that will slow, stop, and hopefully even reverse the effects and damage that CF inflicts on the body. The possibility of the cells in my lungs functioning to their full potential — with CF transmembrane conductance regulator protein function restored and working correctly, expelling chloride out of my cells, hydrating the surface of my lungs, and halting the thick sticky mucus that has caused my airways to be enveloped in a suffocating cloak for all these years — is like a feeling of being rescued when you are drowning.

Unfortunately, I am still drowning.

“I’m very sorry, Ms. Balasa, but you will not be able to be a participant in this clinical trial.” This was the response I received during one of my searches for these drug trials. Excited by the possibility of participating, finding one recruiting at my local adult clinic, I reached out to study coordinators and was informed that I met all but one criterion to participate in the studies. This specific criterion has prevented me from prior trial participation involving other investigational medications treating the symptoms of CF, including anti-infectives and anti-inflammatories.

Most CF studies, including phase I, II, and III trials, require a lung function minimum of at least 40% FEV1 (forced expiratory volume in one second). My FEV1 is 25%, so I am excluded from these trials. Many patients face a similar situation. The 40% threshold biases samples toward a young patient population, as this degenerative condition causes steadily decreasing lung function with time. Furthermore, as CF treatment has rapidly progressed and increased patients’ life expectancies, there are now more adults with CF in the U.S. than children, according to the CF Foundation Patient Registry.

As a patient who works in the science field, I started to ask myself: Where does that number come from? Should this one variable be such a deciding factor? Are we getting comprehensive results from these studies if a subset of patients is omitted? Are investigators using eligibility criteria from a prior study without determining whether the exclusions are scientifically justifiable?

To continue reading, please visit MedPage Today.

Positive Data from the CARE CF 1 Clinical Study of Oral Lynovex in Cystic Fibrosis Exacerbations

NovaBiotics Ltd (“NovaBiotics”) announces that its oral therapy for cystic fibrosis (CF), Lynovex®, has met the study objectives of the CARE CF 1 clinical trial.

CARE CF 1 assessed the effects of two weeks of Lynovex treatment as an adjunct to standard of care therapy (SOCT) in CF, compared to placebo plus SOCT. This trial was designed to determine whether the inclusion of Lynovex capsules alongside SOCT lessened the clinical impact of exacerbations in adults with CF, as measured by symptom severity and levels of bacteria and inflammatory mediators in sputum and blood.  CARE CF 1 was a 6-arm study with the primary objectives of determining the optimal dose and regimen of Lynovex in patients with exacerbations of CF-associated lung disease and to further evaluate the safety and tolerability of Lynovex in exacerbating CF patients.  Continue reading Positive Data from the CARE CF 1 Clinical Study of Oral Lynovex in Cystic Fibrosis Exacerbations

Phase 1a study for drug to treat CF regardless of CF Mutation

http://www.businesswire.com/news/home/20171018005403/en/Synspira-Announces-Patient-Dosed-Phase-1a-Study

Synspira Announces First Patient Dosed in Phase 1a Study of SNSP113 in Cystic Fibrosis
— First-in-class drug candidate for treatment of cystic fibrosis regardless of genetic mutation — Continue reading Phase 1a study for drug to treat CF regardless of CF Mutation

New Pre-Clinical Data for Iclaprim

Motif Bio Presents New Pre-Clinical Data for Iclaprim at IDWeek 2017™

https://globenewswire.com/news-release/2017/10/06/1142163/0/en/Motif-Bio-Presents-New-Pre-Clinical-Data-for-Iclaprim-at-IDWeek-2017.html

1. Pre-Clinical Data Support the Potential Use of Iclaprim in the Treatment of Staphylococcus aureus Pneumonia in Cystic Continue reading New Pre-Clinical Data for Iclaprim

Stem cell therapy for lung fibrosis conditions

https://www.sciencedaily.com/releases/2017/08/170803091928.htm

In the journal Respiratory Research, the scientists demonstrated that they could harvest lung stem cells from people using a relatively non-invasive, doctor’s-office technique. They were then able to multiply the harvested lung Continue reading Stem cell therapy for lung fibrosis conditions

Setting the Record Straight About Coughing Up Blood

https://www.cff.org/CF-Community-Blog/Posts/2017/Setting-the-Record-Straight-About-Coughing-Up-Blood/

Hemoptysis, or coughing up blood, is one of the scariest complications of cystic fibrosis, although it doesn’t have to be. “Hemoptysis” is also the name of a heavy metal band, and that is truly scary! Hemoptysis, the medical Continue reading Setting the Record Straight About Coughing Up Blood

More Than 16 mn Fatal Lung Infections in 2015! Know these 5 Foods to Keep Your Lungs Strong

http://food.ndtv.com/food-drinks/more-than-16-mn-fatal-lung-infections-in-2015-know-these-5-foods-to-keep-your-lungs-strong-1722266

Countries including India, China, Nigeria, Pakistan and Indonesia, accounted for over 16 million or half of the global estimated cases of a fatal lung infection called Respiratory Syncytial Virus (RSV) in 2015, researchers led by Continue reading More Than 16 mn Fatal Lung Infections in 2015! Know these 5 Foods to Keep Your Lungs Strong

New cystic fibrosis biotech aims to take a different approach than Vertex

http://www.bizjournals.com/

California-based Synedgen has spun out its experimental cystic fibrosis treatments into a new biotech to be based in the Boston area, with an eye toward complementing existing drugs for the lung disease. Continue reading New cystic fibrosis biotech aims to take a different approach than Vertex