You have a new set of lungs! What should you expect next?

Cystic Fibrosis Podcast 183:
The Path Forward with Cystic Fibrosis
By Jerry Cahill
In the latest edition of The Path Forward with Cystic Fibrosis, Dr. Arcasoy from Columbia University Medical Center is back to explain what happens after a patient has a double lung transplant. He discusses pain management and the post-transplant care team in detail.
Here’s what to expect immediately pre and post-surgery:
  • Post-surgical care including pain management
  • Medical care that includes antibiotics, antirejection medication, and anti-infection medication
  • Psycho-social recovery assistance
Dr. Arcasoy also explains who your post-transplant care team is and what they do… it’s a lot, so here’s a cheat sheet:
WHO: Medical Transplant Pulmonologist and the Coordinator
WHAT:
Patients will meet with their Post-transplant team once a week for three months, then every 3-4 weeks for a year. At every meeting, the following occurs:
  • Chest x-ray
  • Lab work
  • Pulmonary function test
  • Physical exam
  • Conversation to review medications and overall health & wellness
  • Follow up lab review and medication changes
The schedule for bronchoscopies vary depending on the center, and additional testing can be added at any time deemed necessary.
Remember – every patient’s experience is completely unique! Do not get discouraged; and work with your care team to prepare both mentally and physically for the bumps along the way.

This video podcast was made possible through an unrestricted educational grant from Columbia University Medical Center and the Lung Transplant Project.

You got the call for transplant… Now what happens?

Cystic Fibrosis Podcast 182:
The Path Forward with Cystic Fibrosis
In Jerry Cahill’s latest edition of The Path Forward with Cystic Fibrosis, Dr. D’Ovidio and Dr. Arcasoy from Columbia University Medical Center explain what happens once a patient receives the official phone call for his or her transplant.
They explain dry runs, the transplant surgery, a patient’s first breath, and more! Keep in mind; the overall transplant experience varies greatly among patients, as each case is completely unique.
This video podcast was made possible through an unrestricted educational grant from Columbia University Medical Center and the Lung Transplant Project.

Ground-Breaking Procedure. A major step for science, medicine, the human condition

by Mary Bulman; Independent UK

“Woman spends record six days without lungs thanks to ground-breaking procedure”

Yes you’ve read that correctly.
Yes, it reads six days.

A true miracle! Definitely an understatement.

Though it’s been over a year since this procedure was carried out, it’s one that I believe cannot be shared enough. A huge step for medicine and science- but perhaps a larger one for the human condition and the willingness to live and fight.

“I still don’t believe it happened. It seems very surreal.” says patient Melissa Benoit.
And that’s because it is, Ms. Benoit.

After coming down with the flu the last year 2016, Ms. Benoit was taken from her home in Burlington, Canada to the ICU in a nearby hospital located right outside of Toronto, Canada.  Doctor’s made the spilt decision to go through with a first time procedure in order to save her life. After becoming resistant to most antibiotics, bacteria began to move throughout her body, eventually causing her to lapse into septic shock. One by one her organs started shutting down, due to the decline of her blood pressure.

“Although it had never been carried out before, doctors decided to remove her lungs entirely.”

“What helped us is the fact that we knew it was a matter of hours before she would die,” said Dr Shaf Keshavjee, one of three surgeons who operated on her. “That gave us the courage to say — if we’re ever going to save this woman, we’re going to do it now.”

To learn more about Ms. Benoit and the new breed of surgery that was carried out please continue onto the article below:
https://www.independent.co.uk/news/world/americas/woman-six-days-without-lungs-waiting-list-donor-organ-burlington-ontario-melissa-benoit-world-first-a7547936.html

Potential Therapy for Infections in CF Gets Patent

AB569Arch Biopartners’ treatment candidate for bacterial infections in patients with cystic fibrosis, chronic obstructive pulmonary disease (COPD), and other respiratory conditions, has received a U.S. patent.

The U.S. Patent and Trademark Office issued patent 9,925,206 to the University of Cincinnati, which granted Arch Biopartners an exclusive commercial license on all patents related to AB569. The inventor is Daniel Hassett, PhD, a principal scientist at Arch and professor at the University of Cincinnati College Of Medicine.

“This patent issuance, which protects the composition of AB569, gives Arch a stronger commercial position to pursue treating not just CF patients, but also the millions of other patients that have chronic antibiotic resistant lung infections including those with COPD,” Richard Muruve, CEO of Arch, said in a press release. “It also opens the door for Arch to develop treatments for many other indications where antibiotic resistance is a problem, such as urinary tract infections and wound care.”

Bacterial infections in the lungs are a serious problem in patients with CF, COPD, or ventilator-associated pneumonia. Cystic fibrosis patients are susceptible to bacterial respiratory infections as a result of abnormal mucus production in the lungs and airways.

In particular, the bacterium Pseudomonas aeruginosa (P. aeruginosa) affects most adult CF patients and 40 percent of CF children ages 6 to 10. The mucoid form of P. aeruginosa is highly resistant to conventional antibiotics and immune-mediated killing. It causes a rapid decline in lung function and a poor overall clinical prognosis.

Antibiotic use in the treatment of CF and COPD patients with chronic bacterial respiratory infections is increasing, which correlates with a higher prevalence of antibiotic-resistant strains.

AB569 is a non-antibiotic therapy made of sodium nitrite and ethylenediaminetetraacetic acid (EDTA), two compounds approved by the U.S. Food and Drug Administration (FDA) for human use. The treatment has a different mechanism of action from antibiotics that may increase effectiveness, Arch believes.

“AB569 has two active ingredients that produce a dramatic and synergistic effect at killing many antibiotic resistant bacteria including Pseudomonas aeruginosa (P. aeruginosa), which commonly causes severe chronic infections in the lungs of cystic fibrosis (CF) and chronic obstructive pulmonary disease (COPD) patients,” Hassett said. “AB569 has the potential to make a significant medical impact on treating infection where traditional antibiotics fail.”

In preclinical experiments, the therapy showed significant ability to kill several types of Gram-negative and Gram-positive bacteria.

The safety and pharmacokinetics of a single administration of nebulized AB569 are now being evaluated in a Phase 1 clinical trial with up to 25 healthy volunteers at the Cincinnati Veterans Affairs Medical Center (CVAMC). Pharmacokinetics refers to how a drug is absorbed, distributed, metabolized, and expelled by the body. Enrollment of volunteers started in February.

If the Phase 1 study provides positive results, the company plans to start a Phase 2 trial to test the effectiveness of AB569 in the treatment of chronic lung infections caused by P. aeruginosa and other bacterial pathogens in CF and/or COPD patients.

AB569 previously received orphan drug status from the FDA for the treatment of CF patients infected with P. aeruginosa, and orphan medicinal product designation from the European Medicines Agency.

For original article, click here.

Study Links CF Patients’ Airway Bacteria with Disease Outcomes

By: Diogo Pinto

Researchers have linked variations in the mix of microorganisms in cystic fibrosis patients’ airways to their disease outcomes.

The findings in the journal PLOS One were in an article titled “Fluctuations in airway bacterial communities associated with clinical states and disease stages in cystic fibrosis.

CF patients typically have particular strains of bacterial and fungus in their airways. The usual bacteria suspects include PseudomonasAchromobacterBurkholderiaHaemophilusStaphylococcus, and Stenotrophomonas.

Other bacteria and fungi also inhabit CF patients’ airways, however. These include anaerobic species that do not need oxygen to grow and spread.

Not only do the microbial communities in CF patients’ airways vary by type of microorganism, but also in the relative abundance of each species.

Researchers decide to see if the prevalence and relative abundance of typical CF pathogens and anaerobic microorganisms play a role in the severity of patients’ disease and their lung function.

They analyzed 631 sputum samples collected over 10 years from 111 patients.

The team classified the stage of patients’ disease on the basis of their lung function scores. The yardstick they used was forced expiratory volume in one second, or FEV1. They considered an early stage of the disease to be an FEV1 score higher than 70, an intermediate stage a score of 40 to 70, and an advanced stage a score lower than 40.

Researchers classified disease aggressiveness — mild, moderate or severe — on the basis of change in FEV1 relative to age.

They discovered a link between variations in the prevalance of the six typical CF pathogens, plus nine anaerobic species, and changes in a patient’s disease stage and lung function.

To continue reading, click here. 

Antibiotic resistance evolution of Pseudomonas aeruginosain cystic fibrosis patients

By Francesca Lucca, Margherita Guarnieri, Mirco Ros, Giovanni Muffato, Roberto Rigoli, and Liviana Da Dalt

Below is a study hoping to define and answer the questions of Pseudomonas aeruginosain, its evolution and the resistance from different antibiotics. The study took place between 2010-2013. Though the study may have some time clauses I believe there are some strong findings for the CF community moving forward.
_________________________________________________________________________________

Introduction

Pseudomonas aeruginosa is the predominant pathogen responsible of chronic colonization of the airways in cystic fibrosis (CF) patients. There are few European data about antibiotic susceptibility evolution of P aeruginosa in CF patients.

Objectives

The aim of this study is to evaluate the evolution of antibiotic resistance in the period 2010‐2013 in CF patients chronically colonized by P aeruginosa and to highlight the characteristics of this evolution in patients younger than 20 years.

Methods

Clinical and microbiological data were extracted from two electronic databases and analyzed. Antibiotic resistance was defined according to European Committee of Antimicrobial Susceptibility Testing for levofloxacin, ciprofloxacin, meropenem, amikacin and ceftazidime. The between‐group comparison was drawn with the Chi‐square test for proportions, with the T‐test for unpaired samples for normally distributed data and with Mann‐Whitney test for non‐normally distributed data. Significancy was defined by P < .05.

Results

Fifty‐seven CF patients, including thirteen subjects aged less than 20 years, were enrolled. P.. aeruginosa antibiotic sensitivity decreased significantly for fluoroquinolones, mainly in patients aged <20 years, while it increased for amikacin and colistin. The analysis of minimum inhibitory concentration confirmed these trends. In pediatric patients treated with more than three antibiotic cycles per year, greater resistance was found, except for amikacin and colistin.

Conclusion

An evolution in P aeruginosa antibiotic resistances is observed in the 4‐year period studied. Responsible and informed use of antibiotics is mandatory in CF.
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Read the whole clinical journal here. 

Antibiotic resistance evolution of Pseudomonas aeruginosa in cystic fibrosis patients (2010‐2013) Francesca Lucca,Margherita Guarnieri,Mirco Ros,Giovanna Muffato,Roberto Rigoli,Liviana Da Dalt. First published: 1 April 2018. https://doi.org/10.1111/crj.12787

Lessons From the Road: Avoid the ER

By: Sydna Marshall

A few weeks ago I found myself in the ER at midnight in a new city.  Let me backup the story a bit. I had planned a road trip to Dallas with my bestie (we’ll call her “A” for now) for a beauty conference. Two days before the trip I ran a 102.6 fever for the better part of the day. The morning of, as I was doing my treatments and finishing my packing, I felt the familiar band of pain around my chest. I mentally ruled it out as merely remnants of pain from my blockage earlier in the week and carried on with my morning. I drove the hour north to pick up A and start our trek up to Dallas. We stopped for tacos and queso along the way. I took some pain meds for that persistent and annoying band of pain. A few hours later, we checked into our hotel, picked up our beauty boxes for the conference, and had a happy hour cocktail in the bar followed by sushi in the rotating tower.

Fast forward to 9p: CF changes on a dime. Suddenly, that pesky band of pain that I’ve absently noticed and ignored for the day is front and center. I can barely take a breath, much less a full breath, post-treatment. By 11p, I’ve laid in bed silently crying as the pain spikes up to an 8 and then back to a 6. It dawns on me that the band of pain is pleuritic pain. After texting multiple Cysters and weighing the pain with the inability to breathe, my recent 20% drop in lung functions and the fever I ran earlier, I finally make the decision to wake up A (who is for once sleeping peacefully, without interruptions, in the absence of her four kiddos) and have her drive me to the ER.

We arrive at the ER with this naïve idea that my CF clinic, albeit on-call at this late hour, will communicate with the CF clinic in Dallas. I’d already given A all of the information, phone numbers, and instructions for getting everyone, including my husband Adam, on the same page. Over the course of the evening and early morning hours, hundreds of texts and calls between A, Adam, and the on-call care team at home transpire in an effort to expedite the process.  Since it’s not my first rodeo with pleuritic pain, I’ve already determined before we even got settled in the ER that I desperately need instant-relief pain meds and a chest X-ray. Am I the only one who self-diagnoses? When you’re in and out of the doctor for the litany of health problems in addition to CF, you become the expert on your own body. I digress.

Over the course of the 12 hours in the ER, my port is accessed a total of four times, with one of them being a needle repositioning, before we get anywhere. To administer IV medication and run blood tests, two different nurses start dueling peripheral lines, one in my left hand and the other in my right elbow.  Meanwhile, other nurses attempt to get my port working, which won’t flush or draw back blood. My vein blows on one of the lines, and the other is dangerously close. I have a chest X-ray taken, a CT scan with contrast of my lungs, every blood test imaginable, an EKG, several rounds of morphine, two doses of vancomycin and two albuterol treatments.  I’m told I have a potential pulmonary embolism, a virus causing pleurisy, a mucus plug, or sepsis. Twelve hours in, and about 10 minutes after Adam arrives at the Dallas ER, my repeated requests to be moved to my home clinic, care team, and hospital are heard and I’m care-flighted from Dallas back home (Adam has to drive back home). Once admitted to my home hospital, they have me repeat nearly every test the Dallas ER did less than 24 hours prior as none of my medical records transferred with me from the ER. Five days later the medical records from the ER finally make their way to my home hospital and care team.  In the end, it was determined that I had a virus, which accounted for the difficulty in breathing, pleuritic pain, and fever. It was a very long, traumatic, stressful, and a trying 12 hours away from home. And, I missed my conference entirely, but that’s another story.

I’ve since had some time to reflect on this jaunt to the ER. The biggest takeaway for me – CF clinics do communicate but getting the ER to communicate with the CF care team is nearly impossible.  Having a port is a blessing, but it requires orders from your doctor, not just any doctor, to access and use heparin or cath flow in the event that it’s not working properly (or, in my case, repeatedly accessed incorrectly).  I learned that complaining of chest pain at a new hospital where none of my medical records are accessible means a round of tests to rule out heart problems, despite knowing that it’s my lungs. I learned that transferring medical records from one hospital to another is a royal pain in the you-know-what.

Hindsight is always 20/20, but I know I could have avoided the entire debacle if only I had heeded my inner voice the morning I left for Dallas when I first felt the band of pain around my lungs.  For me, it’s often hard to gauge when it’s important to say no and upend plans, especially when it impacts friends and family around me. If a trip to Walgreens completes a vacation in my house, am I an overachiever for my trip to the ER?

How One Conversation Led Me to Being More Intentional About My Life

By: Ella Balasa

Would I ever live long enough to fall in love? Would I be able to graduate college? Would I be remembered for making some kind of impact on the world before I was gone? Would I get to travel to destinations where the breaking waves crashed against a rocky shore and the sea mist sprayed as I breathed deeply, and beside me stood …

Gabriella-Balasa-Beach-Featured-Rectangle

I’m startled back to reality. I sit in a hospital bed, surrounded by my parents in chairs on either side of me. I’m on the lumpy foam mattress, where I sit cross legged and my butt sinks at least 4 inches straining my back and adding to the pain the past few weeks — and this conversation — have caused me. My dad sits, lips pursed as normal when he listens intently. We are all listening to my doctor talk about my declining health, about my recent episode of pneumonia, and what my future may hold.

“No one knows the future,” I think, as the doctor speaks. My mind jumps again to that ocean scene, only it isn’t me standing on the shore, I’m now observing the scene from above, as if in spirit. Observing a couple embrace and I feel a strange sense of sadness, anger, and jealousy.

“It’s time to consider a lung transplant.” Those words, uttered from my pediatric CF doctor 6 years ago, made me, in an instant, think about all the joys of life I hadn’t gotten to experience yet.

Why me? That’s the first thought many people have when they can’t accept the reality of what’s happening. We try to answer unanswerable questions.

Later that summer, my parents and I followed doctors’ advice and scheduled a week-long transplant evaluation. A week of what I still consider to be grueling medical tests, even compared to other lung complications I have developed since. In the end, the transplant evaluators concluded I was not quite in the transplant window at the time. That fall, my health started to stabilize. I started my second year of college and I felt myself withdraw from the world.

To continue reading, visit CFF community blog.

How to be a Hermit in Flu Season – Top 10 Things to do to Avoid Winter Bugs

By: Beth Sufian

In the past month, many newspapers have reported that large numbers of people in the United States have fallen ill from widespread flu in every state except Hawaii.  People with CF are especially vulnerable to flu and other viruses that pop up in winter.  People spend more time indoors so it is easier for flu and viruses to spread.  I remember one of the first articles I read in CF Roundtable was by Joe Kowalski one of the founders of CF Roundtable.  He wrote about being a hermit during winter and how it reduced his incidence of getting sick.  I thought it was an interesting idea and after 18 years of doing a similar thing in winter, I thought I would share my strategies.

Here is a list of the top 10 things I do to try and reduce the likelihood of getting sick in winter. I know some people are already anxious about getting sick and this blog post is not meant to increase anxiety.  My hope is that one or more of these strategies may help some of you stay healthy during the winter.

Please share any effective strategies you use in the comments section below

1.Take Your Own Pen                                                                                                              On your next trip to the store watch as people take the pen at the checkout and sneeze or cough right on the pen. When you go to the store, doctor’s office or any other public place where you may need to sign something bring your own pen. It is easy to find pens with a stylus cap to use in stores that use a screen for signatures.

2. Take a Small Bottle of Hand Gel and a N-95 Mask                                                    If you find you have touched a surface that has been used by many like a door handle then make sure you have a bottle of hand sanitizer so that you can clean your hands.  In addition, keep a N-95 mask in your purse or backpack.  If you find yourself in a space with a person or many people who are coughing or sneezing you can quickly put on the mask.  If you feel self-conscious about wearing a mask just remember the last time you were sick and that should put those thoughts to the back of your mind.

3. Wear Gloves                                                                                                                        Wearing gloves can help you avoid germs when out in public.  While it is advised you should not shake hands with people this is a hard habit to break.  Wearing gloves allows you to shake hands and lower the risk of passing germs to yourself.  However, you need to make sure you wash the gloves frequently.

4. Step up your Treatments                                                                                               It is hard to avoid sick people if you work in an office or in a job that exposes you to the public so it is important to make sure you are doing your daily CF treatments.  In a perfect world, everyone with CF would do all the breathing treatments prescribed each day without missing any doses.  In reality, things get in the way.  Most people with CF tell me they normally skip a lot of treatments each week.  During winter it is important to reduce the number of missed treatments.  Medicine cannot work if it stays in the bottle.   People with CF often say “I do not have time to do my treatments”.  I think the opposite, I do not have time to get sick so I must make time to do my treatments.  If you are working in an office or going to school it is hard to avoid people who are sick but taking good care of yourself can reduce the chance of catching a winter bug.  Also, make sure you go for quarterly CF Care Center visits so that your CF Care team can monitor your health.

To make treatment time more enjoyable find something you really like to do and do it during treatment time.  If possible make that the main time you do the activity.  For example, if you like watching movies or playing video games make treatment time the time you watch movies or play games.  It takes discipline but can really help decrease missed treatments. Listening to music while doing treatments also helps to reduce the noise of the machines and can make treatment time relaxing.  Some people meditate while doing treatments and report it has a calming effect.

5. Avoid Crowds/ Avoid Sick Visitors                                                                             In the late 1990’s I was on and off IV’s many times due to illness.  I realized that often I finished a round of IV’s and would then go to a party or a big meeting and would be sick within 3 days.  When I started restricting my contact with sick people during winter and beyond my own incidence of illness decreased.  My close friends know they should cancel a lunch date with me if they think they may be sick or someone in their house is sick.  I still go out to lunch with friends but in winter I avoid big gatherings. For example, if my daughter’s school is having a meeting of parents I make sure I sit toward the front or back (depending on the room) on the side and not in the middle of the group.  But if I know the meeting will be in a small room with the potential of having a lot of people in attendance I send my husband to the meeting and stay home.

6. Exercise at Home                                                                                                           For me, going to a public gym or exercise class during winter makes me nervous.  I used to attend a yoga class that I enjoyed.  During the winter months half the class was sneezing and coughing and I decided that was not a good place for me to be exercising.  The same thing happened at a local gym.  Now I use yoga videos and step up the number of times I walk my dog.  I know in some places it is too cold to walk outside.  If you have to go to an indoor gym try to go at an off time.

7. Shop at Off Times                                                                                                      Once winter starts I become very disciplined about when I shop.  I love a certain grocery store in Houston that has beautiful food but it can be mobbed on the weekend and at lunchtime.  The other day I drove to the grocery store at 11 am but saw the parking lot was full.  I was tempted to just “run in” because I had driven there and needed a few things.  But I turned the car around and headed home.  I find that when the store first opens at 8 am there are very few shoppers so that is the best time for me to go.  If you work or go to school and this is not possible see if someone else can get things for you.   Some stores now have a way for you to order things online and then pick up the bagged items at the store. This fairly new service can be very helpful to people with CF.

8. No Airplane Travel                                                                                                         In the late 1990’s I was still traveling in winter. I would finish a course of IV’s and feel good and then a week later I would board an airplane and head to a work meeting, wedding or family event.  Within 3 days of returning from the trip, I would be sick and back on IV’s.  After 3 winters of this cycle of IV’s, travel and getting sick again I realized there was a direct correlation between my travel and getting sick.

My solution was to impose winter travel restrictions.  I do not fly on an airplane in January and February unless I need to travel for medical care.  This year I think I will extend my rule to mid- March given the widespread flu activity and what looks like extended cold weather in many places.  I have been restricting airplane travel since 2000 and have seen great results in terms of my health.  Also by having an absolute rule, no one feels slighted if I miss their wedding or event.  I do wear an N-95 mask when I fly on a plane in other months.  However, I found when I traveled in winter when I got to my destination (especially if the place had cold weather) I still got sick because I came into contact with a lot of sick people.

I travel a lot the rest of the year so having 2 months at home is a treat.  I just cleaned out 28 years of boxes that have accumulated in my attic.  February my goal is to clean and organize my closets.  In Houston where so many lost everything in Hurricane Harvey, it feels good to send things I do not use to those who need help.

9. Rest                                                                                                                                         I have come to the conclusion based on conversions with thousands of people with CF that people with CF do not enough sleep.  For those who work or go to school, there is always a shortage of time as a person tries to do breathing treatments in the morning and night and fit in work and school (or the other way around).  Those who are not attending work or school may find they have interrupted sleep due to coughing, low blood sugar or other health issues which results in exhaustion in the morning.  A decline in health also brings with it the need for more sleep. Sleep is extremely important and helps your body fight off viruses, the flu, and other bugs.  While it seems rare for most CF physicians to talk about the need for sleep it is very important and can really improve health and reduce the chance of getting sick.

10. Stay Connected                                                                                                               In Joe Kowalski’s day there was no Internet, Facebook or Twitter.  Talking on the phone was the way he stayed connected to friends and family during winter.  I make plans to speak to friends or to meet them for coffee or lunch when they feel well.  I also like to plan fun things to do in the spring and summer while I am in my winter cocoon.  I may have to pass up going to a party or an event in winter but I have found the reward of not being sick is worth it.  I look forward to reading of the strategies CF Roundtable Readers use to avoid winter bugs.

 

 

 

A Breath of Fresh Air for Biotechs Working on Cystic Fibrosis Therapies

Researchers from the University of Zurich have determined the structure of a chloride channel, which could be a target for new drugs to treat cystic fibrosis.

Researchers at the University of Zurich have found a new target for future cystic fibrosis treatments. The study, published in Nature, has uncovered the structure of a protein that could help to correct the mechanism underlying the buildup of sticky mucus in patients’ lungs. This could give rise to a new wave of therapeutics for the condition, which at the moment lacks disease-modifying treatments.

Cystic fibrosis is a severe genetic disease affecting the lungs, for which there is currently no cure. It is caused by a malfunctioning chloride channel, CFTR, which prevents the secretion of chloride by cells, leading to the production of thick, sticky mucus in the lung. The condition affects around 70,000 people worldwide, who suffer from chronic infections and require daily physiotherapy.

However, one potential approach to treat cystic fibrosis is to activate the calcium-activated chloride channel, TMEM16A, as an alternative route for chloride efflux. As TMEM16A is located within the same epithelium as CFTR, its activation could rehydrate the mucus layer. The research group used cryo-electron microscopy to decipher the structure of TMEM16A, which is part of a protein family that facilitates the flow of negatively charged ions or lipids across the cell membrane.

The changes that occur in the lungs of cystic fibrosis patients.

TMEM16A is found in many of our organs, playing a key role in muscle contraction and pain perception, as well as in the lungs. It forms an hourglass-shaped protein-enclosed channel, which when bound by positively charged calcium ions, opens to let chloride ions to pass through the membrane.

Current treatments for cystic fibrosis include bronchodilators, mucus thinners, antibiotics, and physiotherapy, which only control symptoms. However, biotechs around Europe are beginning to make progress, with ProQR completing a Phase Ib trial and Galapagos and Abbvie’s triple combination therapy entering Phase I. Antabio has also received €7.6M from CARB-X to develop a new antibiotic against Pseudomonas infections.

The identification of a new target provides patients and biotechs alike with renewed hope of new and effective cystic fibrosis treatments, or even a cure. It will be interesting to see whether small molecules or gene therapy specialists could take advantage of this information.

Original article: https://labiotech.eu/cystic-fibrosis-treatment-target/