Holding Out for 3D-Printed Sinuses

By Sydna Marshall

I often suggest that I should model for the artist-rendering of worst-case scenario CF-sinuses. I’ve had countless sinus surgeries and my ENT (“Dr. E”) has tried everything along the way. Dr. E has opened up my cavities by cutting back some of the bone and membranes, he’s put a flap of sorts in the base of my sinus cavities to help the cheek cavities drain better. He’s fixed my septum and he’s removed a huge number of polyps in multiple surgeries, both under general anesthesia and at his office with local anesthetic. At Dr. E’s suggestion, I’ve also undergone the frontal obliteration. All of this, and my sinuses just plain suck. It’s the number one struggle I have as far as managing my CF.

A year ago, I decided it was time to just see him every two weeks to keep things flushed out routinely and help cut down on hospital time and/or IV antibiotics. Pseudomonas has plagued me for ten years now and just like Bob in the movie, What About Bob, my colonization won’t leave. It’s a resilient little bugger! Because my sinuses are so swollen, we’ve had to think outside the box when it comes to numbing methods before they get washed out in-office. Years ago, three sprays of lidocaine did the trick. Now, I get a spray of lidocaine followed by two gauze pads soaked in lidocaine, which sit in my nostrils for thirty minutes or so. After that, I sometimes get the numbing gel and/or a shot of lidocaine directly in my sinus membranes. Just last week we tried tetracaine, which helped tremendously. It takes roughly 90 minutes for everything to work before we can start the process of suction, extraction, and flushing with huge syringes of water. My favorite part of this whole routine is the immense relief when he extracts something with the alligator tool. It’s the very definition of instant gratification and I’ve spent so much time in his office that my husband bought me my own alligator tool to hang as a Christmas ornament on our tree. One of these days I’m going to ask to decorate what is now my plastic bowl for the rinses. Sydna’s Snot Bowl has a nice ring to it!

Many years ago, I upgraded from the sinus rinse bottle to a SinuPulse machine (think Waterpik for your sinuses) as they really need the extra oomph to power through the mucus and crusting from the infection. I highly recommend it if you’re struggling to get relief from the regular sinus rinse bottle. Over the years, I’ve tried it all: silver sprays, essential oils in my rinse, nebulized antibiotics with a sinus nebulizer, and manuka honey. The latest attempt is a compounded drug consisting of two antibacterial meds, an antifungal, and a steroid, which gets mixed in my sinus rinse along with Alkalol and manuka honey. So far, this seems to help tremendously.

So, what does severe sinus disease look like? It looks like routine visits and trying new therapies, often with little change in outcome and/or comfort. It’s an ever-evolving process and I’ve had a long time to accept that with the therapies available now, my sinuses won’t really get better, as I previously thought they would. Back in 2007 when I first started seeing Dr. E, I mistakenly assumed that one sinus surgery would fix my sinus issues and I’d be on my way to relatively normal sinuses going forward. In hindsight, that was a poor expectation on my part as a routine adenoidectomy at 11 led to my CF diagnosis with the discovery of polyps in my sinuses.

Meanwhile, I’m holding out for 3d-printed sinuses!

Sydna lives in Austin, TX with her husband and fur baby. She loves to read, is a part-time practicing yogi, and enjoys cooking!

In CF, Low-dose Antibiotics Aid Bacterial Diversity in Airways, But at a Cost

As published on CF News Today, BY JOSE MARQUES LOPES, PHD

Using lower, or suboptimal, doses of antibiotics to treat lung infections in children and young adults with cystic fibrosis (CF) leads to fewer changes in airway bacterial diversity compared to therapeutic (higher) antibiotic exposure.

The findings also suggested, however, that patients receiving therapeutic doses had greater improvements in lung function.

The research, “Changes in microbiome diversity following beta-lactam antibiotic treatment are associated with therapeutic versus subtherapeutic antibiotic exposure in cystic fibrosis,” was published in the journal Scientific Reports.

People with CF often have recurrent lung infections that gradually worsen their lung function. Treatment of these infections, known as acute pulmonary exacerbations (APEs), is typically based on antibiotics directed at pathogens such as Pseudomonas aeruginosa. However, repeated dosing with antibiotics has been suggested as the cause of decreased microbial diversity in the airways, which, in turn, could worsen lung function.

Despite current guidelines recommending higher antibiotic dosing regimens in people with CF, data from the U.S. indicate that beta-lactams — which include penicillin — were given in doses below the guidelines 38–53% of the time.

Although CF patients often do not achieve therapeutic doses of antibiotics to clear infections, which means that their blood levels of antibiotics do not increase sufficiently for effective treatment, it remains to be determined whether short courses of subtherapeutic doses alter microbial diversity, compared to therapeutic doses.

Aiming to address this question, researchers recruited 20 patients, ages 1-21, who were treated for APEs with intravenous infusion of beta-lactam antibiotics at Washington, D.C.’s Children’s National Health System.

Four samples of respiratory fluid were collected from each patient — when they were experiencing an APE, when they were doing well, right after antibiotic treatment, and at least 30 days later. Genetic testing determined the type and relative abundance of bacteria in each sample.

Blood samples and data on lung function were also collected during antibiotic treatment. Plasma antibiotic levels and bacterial minimum inhibitory concentrations (MICs) were used to determine therapeutic versus subtherapeutic antibiotic exposure.

Of note, to achieve effective bacterial killing, the serum concentration of an antibiotic must be above the MIC of the bacteria for a certain amount of time. Subtherapeutic exposure was thereby defined as insufficient time above MIC.

A total of 31 APEs were reported over the study period, from March 2015 to August 2016, and only approximately 14 (45%) of the antibiotic courses given were considered therapeutic. Most treatment regimens (25 out of 31 antibiotic regimens administered) included a single beta-lactam antibiotic.

Patients in the therapeutic group (11 patients, median age 9) had better lung function, and were less likely to receive inhaled antibiotics at study start, compared with the nine patients (median age 14) receiving subtherapeutic doses — 45% versus 100%, respectively.

At exacerbation onset, people in the therapeutic group showed a greater trend toward having a normal flora, compared to the subtherapeutic group (43% versus 12%), as well as increased abundance of Gemella, and a decrease in unclassified Enterobacteriaceae.

There was no significant difference in the presence of Pseudomonas aeruginosa or Staphylococcus aureus between the therapeutic and subtherapeutic groups.

As for antibiotic use, participants in the therapeutic group received ceftazidime (a more narrow spectrum beta-lactam) more often (86% versus 41%), and less frequently meropenem (a broad spectrum beta-lactam; 21% versus 59%) than those in the subtherapeutic group.

Those in the therapeutic group also had a significantly shorter time from end of treatment to post-recovery — 51 versus 79 days.

At both end of treatment and post-recovery, patients in the therapeutic group had decreased bacterial diversity in their airways, which contrasted with those receiving subtherapeutic doses — who showed minimal changes or higher diversity more than one month after treatment.

Unlike participants in the therapeutic group, who showed increased or decreased relative abundance of specific bacterial genera compared to baseline, those in the subtherapeutic group showed no changes.

“With the subtherapeutic treatment group, this could represent a ‘basement effect’ where it is harder to decrease diversity when it is already low to start,” Andrea Hahn, MD, the study’s lead author, said in a press release.

Also, “patients in the subtherapeutic group had more advanced disease than those in the therapeutic group, which may influence the findings,” Hahn said.

Data further showed that receiving therapeutic antibiotics was associated with a trend toward greater improvement in lung function.

“Thus, the conclusion should not be drawn that because subtherapeutic antibiotics have less impact on changes in microbial diversity, it could be used as a strategy to prevent declining lung function,” the scientists said. “This is likely a reflection of disease severity, antibiotic exposure, and antibiotic resistance.”

The researchers believe that repeated subtherapeutic courses of antibiotics could lower microbial diversity without clearing infections, causing progressive lung dysfunction. Closer monitoring of antibiotic levels in blood to ensure that each exacerbation is treated with therapeutic-level dosing could be an effective answer, Hahn said.

“What this study shows is that levels of the antibiotics we give probably play a role in patients’ ability to recover baseline diversity,” Hahn said. “If we pay more attention to drug levels when using these types of antibiotics to ensure that dosing is sufficient, we could potentially improve patients’ clinical outcomes over time.”

Window of opportunity for treatment of early cystic fibrosis lung infections

Technical University of Denmark

CF-patients have a genetic defect which results in dehydrated sticky mucous in the lungs, leading to severe and persistent lung infections often caused by Pseudomonas aeruginosa.

The research shows that within the first two to three years after infection with P. aeruginosa, the bacteria are already adapting rapidly to the environment, growing slower and optimizing their fitness to survive.

“Across all of our patients within the first three years, the bacteria on average slow their growth rates significantly and they reduce their susceptibility to ciprofloxacin, a first line drug in treatment of CF-patients. This means that one should pay extra close attention in this period of time to avoid the infection becoming persistent,” says Jennifer Bartell, Postdoc at The Novo Nordisk Foundation Center for Biosustainability (DTU Biosustain) and co-first author.

Looking beyond antibiotic resistance

Clinicians usually focus on detecting antibiotic resistance during infections, and this appears to be an effective way to follow the development of short-term acute infections.

Antibiotic resistant bacteria are identified by their ability to survive above a specific concentration of an antibiotic. The researchers saw a rapid increase in the concentration of antibiotics that the bacteria could tolerate. But at the same time, few bacteria achieved detectable antibiotic resistance in the early infection period of CF. The researchers suspect this pre-resistance adaptation to be an underused marker of progression in the infection. This pre-resistance adaptation likely occurs in other persistent infections, such as chronic obstructive pulmonary disease (COPD).

Besides looking for antibiotic resistance, clinicians also monitor bacterial mucoidity — a trait where bacteria produce a protective, slimy coating, as a marker of a chronic infection.

But according to the new study, bacteria can become persistent and resilient to treatment regardless of the appearance of mucoidity. Other bacterial traits such as the ability to attach to surfaces and aggregate in biofilms — hefty structured layers of adherent cells — evolve more consistently in these persisting infections than mucoidity and may serve as a better sign of early chronic infection.

“We can see which traits might actually be valuable for the clinicians to monitor in addition to antibiotic resistance,” says Lea Sommer, Postdoc at Rigshospitalet and co-first author.

Potential for new diagnostic tools

The researchers identified these important evolving traits of P. aeruginosa by screening 443 isolates from 39 young cystic fibrosis (CF) patients over a ten-year period and mapping traits adapting in tandem using statistical modeling approaches. Usually, studies focus on bacterial isolates collected from older CF-patients with chronic infections, who have become multi-drug resistant and already have adapted to the human lungs.

These results emphasize that trait evolution measurements are important and should not be neglected, even though genomic tests are advancing.

“In this early phase, the bacteria change a lot and become much more robust, but the doctors do not necessarily see this with current clinical measurements,” says Lea Sommer.

Going forward, the researchers wish to find out how the adapting bacteria respond to a larger panel of antibiotics that are used to treat patients. Armed with this comprehensive map of evolutionary pathways, clinicians would have a much better chance of categorizing the infection and, hence, take the necessary precautionary steps.

“In the clinic, doctors would potentially be able to take a single patient’s bacterial screening data and analyze how this patient responds to the current treatment. As we gain experience with more patients, it will be easier to assess what can be done to stop the transition to chronic infection, “says Jennifer Bartell.

Thus, this could pave the way for developing more fine-tuned personalized treatments for all patients suffering from continuous persisting infections, such as CF, COPD, and perhaps diabetics with chronically infected wounds.

Original article: https://www.sciencedaily.com/releases/2019/03/190304121505.htm

Nutrition – Patient Task Force Meeting

By: Melissa Shiffman

Growing up, I had an unhealthy relationship with food. My brother and I both were diagnosed with Cystic Fibrosis as children. He was pancreatic insufficient, painfully thin and took handfuls of enzymes. Meal times were filled with constant fighting as my parents tried to force him to eat. Meanwhile, I was pancreatic sufficient with a very healthy appetite. Our body shapes could not be more different! He was skin and bones and I was chubby. As I got older, “chubby” was socially unacceptable. I was twelve the first time I went on the “hotdog diet!”

I have no real memory of fresh fruit and vegetables being readily available. Instead, our refrigerator was filled with high calorie, high fat foods to encourage my brother to eat: Kit Kats, Nestle Crunch, ice cream, Sustacal puddings. I was told not to eat his food, so I snuck it and ate in my room. I gained more weight. My brother made fat faces to get under my skin as I sucked in my cheeks back to him.

Like a normal pre-teen, I became self conscious about my appearance.Everyone talked about fad diets and losing weight. I absorbed that being thin was ideal. By the end of 8th grade, I convinced myself that, if I read the ingredients in things like Oreos, I would find a way to be disgusted. Eventually, I talked myself out of eating most foods except for grapefruit and canned tuna without mayo. I ate minimally and cut out sweets. I lost a lot of weight, started to look more like my brother (but less sickly) and revelled in the compliments on my weight loss.  

I know this is not the normal CF story. However, it is so important to recognize our relationship with food and to rectify our lack of education on eating well when you are underweight or gaining weight on modulators. Therefore, I am looking forward to Dr.Tonja Gonska (gastroenterologist at Sick Kids Hospital in Toronto) discussing healthy eating vs. high fat/high caloric intake, body composition vs. BMI and how nutrition plays a role in healthy weight gain for women with CF at the CFReSHC Patient Task Force meeting on Thursday March 21, 2019 from 3-5 pm EST.

Attendees can share their personal experiences, help develop potential research questions to be addressed by the CFReSHC research advisory panel and receive an Amazon gift card for their time.  For more information on CFReSHC and for login information email CFReSHC at info@CFReSHC.org.  

‘We’re still waiting’: As cystic fibrosis drugs deliver new hope, not everyone is being swept up by scientific progress

By Andrew Joseph for STAT News

CAMBRIDGE, Mass. — The tiny round one is vitamin K. There’s a gel cap (vitamin D), a two-tone capsule (that one protects his liver), a square pill (generic Singulair), and more — seven pill bottles sharing space on his dresser with a “Criminal Law and Its Processes” textbook that’s thick enough to be a weapon.

Josh Hillman, a 23-year-old Harvard Law student from Alabama, has cystic fibrosis, the progressive genetic disease that causes frequent lung infections and wears on other organs. He has to pop enzymes with every meal to maximize the nutrients his body absorbs. While he sleeps, 1,200 calories of a nutritional shake drip through a tube directly into his stomach. There are the inhaled drugs — sometimes antibiotics, always a mucus thinner — that he breathes in through a nebulizer, and a blue vest that slips on, inflates, and vibrates, a 30-minute shaking session that he does twice a day to help clear the mucus that gunks up his airway and lungs.

“It makes it a little bit difficult to write,” he said, his Southern accent rumbling like his wheeled desk chair was careening across cobblestones.

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But for all that is part of Hillman’s daily regimen — and that’s what he does when he’s healthy — it’s what’s missing that’s just as notable: one of the new cystic fibrosis treatments that have started to transform the lives of others with the condition.STAT Plus: Exclusive analysis of biotech, pharma, and the life sciences.

Those medications, the first to be tuned to the genetic mutations that cause the disease, have helped people experience fewer flare-ups and hospitalizations. They have also brought the relief of simply feeling better and breathing easier. The drugs have been touted as a testament to what’s possible with precision medicine treatments, which target the roots of diseases instead of just addressing symptoms.

The catch is that cystic fibrosis is not caused by one mutation, or a handful, but more than 1,500 different rearrangements in the code for the gene known as CFTR. The cutting-edge treatments — there are three available now and a fourth, still-experimental medication expected to be approved, all from Vertex Pharmaceuticals — cover the mutations held by some 90 percent of CF patients.

That leaves up to 10 percent of people whose diseases are advancing without a powerful defense to slow them down. Hillman is among them.

“This is such a concrete fact of your life — it’s always going to be here, it’s never going away,” he said matter-of-factly in describing his condition.

The new therapeutic landscape for CF has created disparities among patients who, until 2012, were all treated the same way. Patients who are not eligible for the new medications have been left wondering if the kinds of treatments available to others will reach them in their lifetimes. And there is a time imperative: Only half of people who are born today with CF will live into their late 40s.Related: Vertex cystic fibrosis drug combination shows strong results in pivotal clinical trials

It’s a reminder that with complicated genetic diseases, progress, while rightly celebrated, doesn’t always extend to everyone at the same time.

“It makes me so happy to see other people with CF thriving and getting help. It brings me more joy than I can even explain,” said Stacy Carmona, 32, who works in patient advocacy for a specialty pharmacy, takes more than 40 pills a day, and spoke of the “panic” of feeling left behind. “But there’s that other side of it, where I so desperately want to catch up to them.”

As she’s gotten older, Emily Kramer-Golinkoff, 34, whose lungs function at about 30 percent the level of healthy organs, has started talking with doctors about a transplant.

“Watching them have not just new hope, but like new lives, new capabilities, to start thinking about retirement funds and things like that, I’ve never had that luxury,” she said. “We’re still waiting, and we’re sinking. For us, this disease is still the same killer.”

Head over to STAT News for the remainder of this story.

Sharing Life with CF on Social Media

By Ella Balasa for CF News Today

“Ella, you were at a bar?! I thought you were going to the hospital?”

I received this perplexed (and perhaps skeptical) text after I posted a picture to social media of me at a local brewery for a cystic fibrosis-related fundraising event. I was smiling with a group of girls and the caption stated that I will go to Duke University to begin evaluation tests for lung transplantation due to my rapid health decline.

What that photo didn’t hint was that it took 4 liters of oxygen to walk from the car into the bar. I didn’t take a sip of alcohol the entire time I was there. I was holding the nasal cannula in my lap just long enough for the photographer’s shot. Though I’m shown smiling, only minutes later I coughed uncontrollably as tears welled in my eyes.

But could I actually be that sick if I’m showing pictures of myself looking completely normal?

I have briefly shared on social media the realities of the last eight weeks because I want my fears and experiences understood and my plight sympathized by those close to me. The past two months included IV antibiotics, three hospitalizations, constant supplemental oxygen use, and the quick decision to begin lung transplant evaluation. I have tried to show this reality with photos of me using oxygen, as the cannula and tank really are a couple of the only things that make this disease visible.

CF has become a significant part of my life, and my public image would be a façade if I didn’t share these details. But I don’t want my social media to be the diary of a sick girl. I get anxious just wondering what others would think about me if I did that.

In this social media world, some people know us only by our online presence, which can misconstrue the realities of life. The shared glimpses don’t represent the details of actual day-to-day life. I fear that others will judge me and doubt my sincerity and genuineness due to the anonymity of the internet. I don’t want to seem volatile, inconsistent, or as if I misrepresent my life.

Perhaps images of oxygen use with captions expressing my hardship contrast too sharply with subsequent posts showing me somewhat recovered and smiling with a group of friends during an evening out. It’s not that I’ve had a dramatic recovery, but rather that I’m simply not quite as out of breath on those days. Most people close to me or in the CF community understand how unpredictable life with this disease can be, but those at a distance often don’t. I want to portray my life accurately, yet I also don’t want my CF to consume my social media.

Truthfully, I’ve only ever been given an outpouring of support, sincere concern, and care from my family, friends, the CF community, and even those I don’t know. Many people want to uplift and support me. Through connecting to people with CF online and joining a community of people who understand all I share, I am starting to feel more comfortable about sharing the ups and downs of my life with a chronic illness.

In the last two weeks, my health has stabilized so I can be more active and off of my oxygen tank’s leash. When you see a post with fun captions and a blissful smile, it is because I am truly happy and thankful that I am in this state. I try to remind myself that as long as I share parts of my life with genuine intention, others will believe it and feel my sincerity.

UC Berkeley professor collaborates on study to improve cystic fibrosis treatment

By  Stanley Von Ehrenstein-Smith of The Daily Californian

UC Berkeley professor Terry Machen is collaborating with researchers from Canada to discover new treatments for cystic fibrosis, an incurable genetic disease that cuts decades off the lives of patients.

The research, a collaboration between UC Berkeley and the University of Saskatchewan, or USask, in Canada that began in 2016, is based on a unique method developed by the Canadian researchers to measure fluid secretion in the lungs. Cystic fibrosis is characterized by thick, viscous mucus that clogs the airways and causes bacteria to accumulate, leading to frequent infections that damage the airways.

“This treatment predicates an idea that if you inhale a solution that is more concentrated at the blood, the patient will get this salty solution in the lungs,” said Juan Ianowski, a study co-director and USask assistant professor of physiology. “The salt will cause osmosis or water movement through a concentration gradient and water movement from the blood side into the airways.”

The effects of this treatment change the properties of the mucus to become “normal,” allowing the patient to cough it out or have it moved through cilia, tiny hairlike structures, in the lung. This hypersaline treatment originated in Australia and has been used for decades to alleviate the symptoms of cystic fibrosis.

The research team — co-directed by Ianowski and Julian Tam, a respirologist at USask — found a way to develop this treatment by observing the way in which the hypersaline solution stimulated neuron cells that are present in the lungs rather than solely crediting the solution for its established osmotic effects.

“Neurons that are present in the airway are constantly monitoring the conditions of the airway, (becoming) deactivated in cystic fibrosis,” Ianowski said. “These neuron cells would also contribute to the production of water in the lumen of the airway.”

The research group determined that 50 percent of the liquid produced in the airway when the hypertonic saline is administered comes from the nervous system stimulating the production of fluids.

The impacts of these potential treatments enable cystic fibrosis patients to modulate the amount of the hypertonic saline solution as well as potentially disengage or engage certain aspects of their nervous system to manage a more desirable response.

“The main goal is to come up with new formulations that will allow us to make this treatment last longer and reduce negative reactions in the patient,” Ianowski said. “We want to make the treatment stronger, last longer, or cause it to inhibit some components such as a coughing reaction.”

The research team is funded by Cystic Fibrosis Canada and will be continuing to study developed treatments for cystic fibrosis.

Machen, campus professor emeritus of molecular and cell biology and study contributor, said “amazingly effective” drugs for cystic fibrosis have been made available for patients by pharmaceutical companies including Vertex. The disadvantage, however, is that these drugs cost $300,000 a patient per year.

“It would be a big addition to the field if we could develop something that made things better and is also cheaper,” Machen said.

Stanley von Ehrenstein-Smith covers research and ideas. Contact him at svonehrensteinsmith@dailycal.org and follow him on Twitter at @von_ehrenstein.

CF Roundtable Organ Transplantation Issue is Almost Here!

The special winter CF Roundtable® issue on Organ Transplantation in CF will be out soon.

Subscribe by January 26th to ensure that you receive it. It’s free! 

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This issue is close to 60 pages (a record for us!) and it’s almost here! If you or anyone you know wants to receive their own issue in print or PDF, there’s still time!

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As always, you can subscribe at no cost. 

Aridis Enrolling CF Patients to Test AR-501 in Chronic Lung Infections

By:
ALICE MELÃO

Aridis Pharmaceuticals has enrolled the first healthy participant in its Phase 1/2a clinical trial to evaluate the antibacterial potential of its investigational candidate, AR-501 (gallium citrate), against chronic lung infections in patients with cystic fibrosis (CF).

The study (NCT03669614) is expected to enroll approximately 48 healthy adult volunteers and 48 adult CF patients with chronic lung infections across 15 sites in the United States.

Participants will be randomized to receive one of three doses of AR-501, or a placebo, self-administered once a week using a hand-held nebulizer.

The company expects to announce results from Phase 1 during the fourth quarter of 2019, and from Phase 2a in the fourth quarter of 2020.

“We are pleased to initiate this exciting program with the first subject enrolled,” Wolfgang Dummer, MD, PhD, chief medical officer of Aridis, said in a press release. “Through this trial, we anticipate safety, pharmacokinetic, and exploratory efficacy data that will enable us to further explore the potential of AR-501 in the treatment of life-threatening bacterial infections in cystic fibrosis patients.”

AR-501 is an inhalable formulation of gallium being developed to treat pulmonary bacterial infections. It works by starving bacteria of iron, and inhibiting their iron-dependent metabolic processes necessary for the infection to progress, a mechanism very different from that of common antibiotics.

Preclinical studies have demonstrated that AR-501 holds a broad antibacterial activity with unique benefits, compared to current standard-of-care antibiotics, working against antibiotic-resistant strains such as Pseudomonas aeruginosa and B. cepaciaaccording to the company.

Also, data from a Phase 2 clinical trial (NCT02354859) showed that intravenous gallium is safe and can effectively improve the lung function of CF patients.

“The recent safety and efficacy demonstration of intravenous gallium from a Phase 2 clinical study in CF patients gives us optimism of the prospect inhaled delivery of gallium (AR-501), which is a more direct, local route of delivery to the site of infection in the lungs and less systemic exposure,” Dummer said.

The new Phase 1/2a trial is being conducted in collaboration with the Cystic Fibrosis Foundation (CFF), and is led by Noah Lechtzin, MD, director of the Adult Cystic Fibrosis Program and associate professor of medicine at Johns Hopkins University.

The U.S. Food and Drug Administration recently granted Fast Track Designation and Qualified Infectious Disease Product Designation (QIDP) to AR-501. These are expected to support and expedite the therapy’s development and regulatory review.

Original article: https://cysticfibrosisnewstoday.com/2018/12/14/aridis-started-enrolling-in-phase-1-2a-trial-to-test-ar-501-antibacterial-potential/

Airway Clearance Vests Fail to Show Measurable Short-term Lung Benefits in Study

A note from CF Roundtable: Please do not stop using your Vest or other HFCWO device because of this impractical study. There are important differences in this study that make it not applicable to CF and therefore, not meaningful for us. First, healthy volunteers without CF were enrolled. Too many adults with CF have experienced significant benefits with these HFCWO devices, not to mention the preventive benefits. To imply these devices worsen lung function when used by a healthy nonCF person vs one with CF – with the usual accompanying inflammation, mucus +/- bronchiectasis, etc – is not practical. Second, these healthy subjects tested all 4 HFCWO devices in one day. Even when I repeat spirometry in one day, my lung function numbers most often decline over time, even with rest periods in between. The researchers tested lung function with a rest period of 15 minutes on these healthy individuals after use of the HFCWO device then moved on to the next device. Very impractical and again – not applicable to use by individuals with CF.

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By Iqra Mumal

A clinical study into high-frequency chest wall oscillation vests — assessing their short-term impact on standard measures of lung function before and during use — challenges the view that these devices work through airflow bias in the lungs, the process responsible for mucus movement when breathing.

Findings, using established tests that include forced vital capacity (FVC), forced expiratory volume (FEV1), and forced expiratory flow (FEF25%-75%), suggest “that the concept of HFCWO vest-induced cephalad airflow bias is not supported by standard spirometry measurements,” researchers concluded. “None of the vest groups showed statistically significant increased airflow in the lungs.” Continue reading Airway Clearance Vests Fail to Show Measurable Short-term Lung Benefits in Study