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A very interesting research topic will be presented at the upcoming NACFC where scientists report a novel approach to treating CF lung disease. They claim that a polymer (called PAAG) which is based on a natural polysaccharide, is able to breakdown unwanted mucus and bacterial biofilms. The...

Drug combination therapies to treat the underlying cause of Cystic Fibrosis are a promising strategy and here is why: Cystic Fibrosis is a result of specific mutations that occurs in the CFTR gene, resulting in a defect CFTR protein. The combination therapies currently being tested are...

Data Driven Medicine is leading us to personalized medicine. Advances in Next Generation Sequencing (NGS) and High Performance Computing (HPC) are enabling P4 medicine: personalized, predictive, preventive and participatory. This new era of medicine ensures every patient is well diagnosed and...

A drug currently in phase 2 clinical trials for COPD and Asthma maybe effective for the treatment of Cystic Fibrosis. Verona Pharma is expected to present some data at the upcoming NACFC on their drug called RPL554, which they claim will help "activate" the CFTR protein. CFTR activation may also...

Someone asked me to explain the difference between mucoid and non-mucoid Pseudomonas aeruginosa (PA) and how this relates to Biofilms. The answer is quite simple really. Most people with CF become infected with the non-mucoid PA strain in their childhood or adolescent years (80% are infected by...

Students at Dundee University are developing a device to help rapid detection and quantification (via green fluorescence) of sputum bacteria such as Pseudomonas aeruginosa or Burkholderia cenocepacia. During a competition (iGEM), they came up with the idea of the "Lung Ranger", made up of the...

Hi all, here is a list of items I came across this week in the media on CF. I covered some of this in more detail in this weeks Newsletter which you either will received in your inbox or can access via our facebook page: https://www.facebook.com/knowcf A lot of these news items link to our...

A recent Lancet review by leading expert Professor Stuart Elborn just came out on the growing concern of antibacterial resistance in cystic fibrosis. He said that more funding and further research are required into antibiotic resistance in order to improve patient outcomes for people with cystic...

If the stock price is any indication, it looks quite likely that Vertex will release some very positive result at the North American Cystic Fibrosis Conference in 2 weeks time on the trials that address the treatment of CFers with double F508del mutation. When Vertex announced top-line...

A New Zealand company is developing a new drug called Citramel, which is claimed to target the biofilms bacteria use to build-up resistance to antibiotics. It just received approved by New Zealand regulators for its use in a clinical study which will start soon pending funding. Citramel is...

It’s uncharted territory! A nice article out yesterday reviewing advances in CF coordinated care, new challenges and new targeted therapies. Some very important points are made! The burden of disease (TIME to treat CF) that CFers and their caregivers have to go through to keep as healthily...

As we approach flu season in the Northern hemisphere, here is a study just out where researchers have concluded that Live Attenuated Influenza Vaccine [LAIV] (The Nasal Spray Flu Vaccine) is well tolerated among children with cystic fibrosis. They said: "Given the clinically severe impact of...

Gene Therapy for Cystic Fibrosis is advancing and we hope to hear some positive results next month from the UK CF Gene Therapy Consortium at the NACF conference. Results will be about the multi-dose trial that is currently going on in the UK via a liposome-based delivery vector and will...

Hi Everyone! Apologies for not updating you on week 37 news. It has been such a busy last 2 weeks for me and Jeanne (imogene). I traveled to the US last week to present with Jeanne at the ePatient connections conference. The presentation went extremely well and the days that followed were none...

Twenty-five years ago this month, the CFTR gene was discovered. Here are some facts: - The CFTR defect is really a very subtle defect and much easier to study then most. - CF is caused by a defect in a single gene, affecting the CFTR protein. - The CFTR protein helps to regulate chloride...

A new drug called "Lynovex" just got approved by FDA that has unique multi-functional, combined properties of breaking down the excessive mucus produced in the airways. The new drug allows for destruction of the bacteria responsible for the recurrent respiratory infections in CF by...

It is so crucial that insurers cover drugs like Kalydeco and here is another milestone for the Kalydeco drug and the CF community. We must educate the payers of healthcare and make them understand that even though Kalydeco costs are high, they offset an enormous treatment cost burden for them as...

ProQR IPO is this week. Their leading product is QR-010 which aims to restore the defect in CFTR protein caused by the del F508 mutation. Specifically, administration into the lungs of a chemically modified oligoribonucleotide that is capable of restoring the RNA sequence coding for CFTR...

THANK YOU! We gave an amazing presentation this morning at the ExL ePatient Connections Conference in Philadelphia and we have this wonderful CF community to thank for driving and motivating us to present. We presented a topic on "participatory healthcare" and why industry should care about...

Exercise is key and often overlooked as a treatment method for CF. Michael Burke, 44, is a lifelong battler with cystic fibrosis. He says: “From my sixth birthday to my 16th birthday, I thought way too much about dying. It was wasteful worrying. Here I am in my 40s, and I’ve run marathons...