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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.

Utah’s Somer Love Battles CF with Kindness and Strength

By Larry Luxner of CF News Today

Somer Love is sitting at her favorite local hangout, the Landmark Grill in suburban Midvale, happily devouring her lunch. Looming on the horizon to the east are Utah’s majestic Wasatch Mountains, but at more than 10,000 feet above sea level, the range is strictly off-limits to Love — as is nearby Park City and its famous ski resorts, at 7,000 feet above sea level.

That’s because cystic fibrosis (CF) has left Love with only 27 percent of normal lung function.

“I haven’t been to Denver since I was a kid, because it’s harder to breathe there. Even Salt Lake City, at 4,500 feet, is hard,” she told Cystic Fibrosis News Today in a recent interview. “Growing up I was fine, because my lungs were healthier. But I think living in Utah at this higher elevation actually helped strengthen my lungs.”

Love, 39, moved here from New Jersey with her parents when she was nine months old. At 11 months she was diagnosed with CF after her mother had heard that babies who have a salty taste when kissed should be taken for testing.

“We were outside playing in a playpen, and I tasted salty,” she said. “My mom called our pediatrician for a sweat test. I was super healthy and fat. The doctor was probably thinking ‘crazy lady from Jersey.’ But she talked him into doing it, and when the results came back, he called my mother crying.”

Growing up, Love received treatment from the Intermountain Cystic Fibrosis Center located at Primary Children’s Hospital and University of Utah Hospital in Salt Lake City.

Both centers are accredited by the Cystic Fibrosis Foundation (CFF) and serve mostly patients from Utah, Idaho, and Wyoming — with some coming from as far away as Montana and Nevada.

Love’s first hospitalization was at age 6; she began taking enzymes at 8, because no drugs were available back then. In eighth grade, she got her first nebulized medication.

‘Love to Breathe’

Because of her disease, she quickly developed a strong sense of activism and self-reliance.

“I’ve always been a really positive person. This stems from my parents, with whom I’m very close,” Love said. “They started the local CF chapter here and were on the board. They did everything in their power to help me. When you’re faced with such a challenge, you can either overcome it, or do nothing and let it overcome you.”

She added: “CF is basically my life, especially now that my disease has progressed. From the minute I wake up till the minute I go to bed, I’m constantly thinking about it. I literally think about every breath I take. I’m actually grateful for CF; it’s a blessing in an ugly disguise. It makes me appreciate things more.”

No surprise, then, that the website Love launched in 2001 is called “Love to Breathe” — a play on her last name.

Among other things, the site contains a blog with nearly 700 entries, a compendium of facts about CF, and dozens of photos, videos, and original paintings. There’s also a page dedicated to the CF activist’s #LoveToBreathe tokens; she’s sent out more than 10,000 of these little medallions to CF patients and supporters in all 50 U.S. states and more than 70 countries.

“I wanted parents to have a place to go for their newly diagnosed children,” she said. “There’s a lot of negativity out there on the Internet, and I wanted people to see somebody being positive. I’m an artist, so I started by sending my paintings to fundraisers all over the country.”

Love said the Internet is especially helpful to CF patients due to the isolating nature of the disease. “We cannot be around any other CF patients, so we connect online,” she said. “My website is a personal endeavor, a way for me to spread love and CF awareness.”

Even so, her main focus is on staying healthy. That means spending six hours a day doing her airway clearance and taking all her medications — about 60 to 80 pills daily. Her morning treatments are one-and-a-half to two hours each, and she must sterilize her “neb cups” afterward.

The transplant option

Love has been taking Symdeko (tezacaftor/ivacaftor and ivacaftor) twice a day ever since the combo therapy — developed by Vertex Pharmaceuticals — was approved in February 2018 by the U.S. Food and Drug Administration. But she must eat 10 to 20 grams of fat beforehand.

“Because of our gastrointestinal problems, we don’t absorb nutrients well, so one of the perks of CF is that we can eat whatever we want,” she said. Even so, Love is a vegetarian. She usually eats five small meals daily, with breakfast being her main meal — and she snacks all day long.

Speaking of food, each November, Love’s parents host an event called Taste of Utah. Tickets cost $200 each, and 30 restaurants around the state participate. Last year, the annual fundraiser generated just over $400,000 toward CF research.

Yet, Love herself doesn’t travel much. In fact, she said, “I’m never gone from my house for more than four hours. There are some days I can’t even get out of bed. I also have asthma, which makes things difficult.”

In addition, Love regularly experiences catamenial hemoptysis, a rare hormonal condition that causes her to cough up blood a couple of days before her period. And although she doesn’t have CF-related diabetes or liver disease, motherhood, she said, isn’t a good option for her.

“I grew up knowing that I wasn’t going to have kids,” she said. “I’ve had many friends with CF who have kids, and their health takes a huge decline. Also, I could never be the kind of mom I would want to be. When your child is sick, your first instinct is to cuddle them. But if we get sick, it’s a two-week stay in the joint.”

With the median age of survival for newborns with CF now in the early 40s, Love realizes that a double lung transplant is in her future — though the prospect of such a serious and potentially risky operation is daunting.

“I’m not quite there yet,” she said. “You can’t be too healthy, and you can’t be too sick. But I would absolutely opt for a transplant when the time is right.”

How Mentorship Helped Me Through the Process of My Lung Transplant

Note from Ella at CF Roundtable: We wanted to let all of our readers know that the upcoming issue will be all about organ transplants! It is full of information from pre and post transplant experiences by those of us with CF. Personally, I am very much looking forward to reading these stories as I have been just recently told I am in the “window” for transplant. It’s terrifying and joyous and a mixture of everything in between. I also will be signing up for this mentorship program because I want talk to those who have been through it! Thank you to the Lung Transplant Foundation for this perfect blog introduction to our next issue.

By Eirik Gumeny

Receiving a lung transplant can be an isolating experience, and no one should have to go through that alone. Mentorship programs are an excellent way to help.

When my wife and I started our journey towards my double lung transplant in 2014, we found ourselves overwhelmed and slightly adrift. We’d been to the orientations and met with the doctors, but something seemed like it was still missing, something we weren’t quite able to put our fingers on.

In hindsight, the answer was simple: pamphlets and professionals are only one half of the transplant experience. Input from other patients is equally – if not more – important. Transplant patients need to know the technical stuff, the ins and outs, yes, but there are things that only someone who’s actually lived through a transplant can help you with, things that only another person in your same position can understand.

That’s where the Lung Transplant Foundation Mentorship program can help.

They were among the first institutions to implement a mentorship program for lung transplant patients, and they were the first to reach out to my wife and me.

Founded as a non-profit organization in 2009 by a group of lung transplant recipients, The Lung Transplant Foundation (LTF) has been raising funds and acting as an advocate for lung transplant research ever since. Nationally recognized, the LTF is a member of the American Thoracic Society’s PAR Council of Public Representatives, and, currently, the only national lung transplant organization that sits on this prestigious council.

Their Mentorship program provides education and emotional support for transplant recipients and their caregivers, via confidential, individualized, one-on-one support for people at all stages of lung transplantation through personal contact with a trained Mentor.

When it comes to receiving a lung transplant, having someone who intimately understands what you’re going through its crucial to the experience. Though mentors can’t – and shouldn’t – replace professional medical advice, just having someone to talk to, someone who has faced the same problems as you, can help immensely.

The Lung Transplant Foundation can be found online at lungtransplantfoundation.org, and you can find more information on their Mentorship program by clicking here.

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!

If you subscribe before tomorrow, January 26, you will be added to the mailing list in time to receive it.

As always, you can subscribe at no cost. 

Runner Won’t Let Cystic Fibrosis Stop Him From Winning a 5K—or Qualifying for Boston

Original article on Runners World

“You just have to focus on what’s in front of you today, and breathe the best you can.”

When he was a kid, Michael Waltrip remembers getting bored at his brother’s soccer game, and asking his dad if he could kill some time running around the track. 

Waltrip was diagnosed with cystic fibrosis (CF) a few days after birth, so his father probably figured he’d last half a lap before breathing issues kicked in. 

But he took so many laps that they both stopped counting. Since then, he’s never really stopped. Not even when the breathing issues really did begin a few years ago.

A progressive, genetic disease, CF tends to worsen with age, and mainly affects the lungs and digestive system. The condition hampers the cells producing mucus, sweat, and digestive enzymes, often clogging airways and causing frequent lung infections, inability to gain weight, and difficulty absorbing nutrients.

For Waltrip, the digestive challenges have been lifelong, but he managed to avoid breathing problems until three years ago, when he turned 21. His doctors told him this may not have been simple luck—his running may have played a part in keeping him healthy, he said to Runner’s World.

When you run, the vibration that results from basically micro-jumping over and over is beneficial for the lungs, Waltrip said, because it helps to break up mucus. (Something most runners tend to experience, necessitating the perfect “snot rocket” form.) 

Since that first day of laps around the track, running had been a constant for Waltrip: After playing soccer himself as a kid, he switched to cross country in high school and logged 40 miles a week, then continued by joining the track team in college. 

Running may have helped ward off the breathing issues for some time, but when they did start several years ago, it was frightening. Waltrip recalled an awful feeling of being unable to take a full, deep breath. And that freaked him out.

“Until then, I think I didn’t acknowledge that I had CF because it wasn’t affecting me as much,” he said. “But then I came to the point where I couldn’t fall asleep because I couldn’t breathe properly, and I’d have to sit up and calm myself down for 30 minutes with breathing exercises.”

His running, which usually averaged around 60 miles a week, became much more of a struggle. Some runs would be okay, he said, but most would be filled with constant coughing and shallow breaths just to get the miles in.

“I still kept training, though,” Waltrip said. “I was being stubborn. That went on for six miserable months, before I finally decided that this was a big life lesson in accepting what was happening and getting help.”

His doctor put him on a daily treatment that included a nebulizer geared toward breaking up mucus, as well as a saline solution designed to irritate the lungs. Basically, he explained, your lungs don’t like salt, so they excrete water to balance it, and that helps break up the mucus and push it out. Waltrip also needed to inhale antibiotics, since people with CF who get bacterial infections in the lungs find those super hard to kick.

At the same time, he’s worked to stabilize his weight—an issue that’s always been a challenge. He can’t break down protein and fat very well because of his condition, and adding 60 to 80 miles of running a week on top of that means near-constant eating to keep from being too underweight. 

Despite these obstacles, though, he’s back in run-all-the-laps form. 

Waltrip recently won the Fairfax, Virginia-based Run Your Heart Out 5K in 16:02, and last July, he qualified for Boston during his first marathon attempt in San Francisco. Ditching his stubbornness and embracing daily treatment has helped him stay regimented, he said, and he’ll be at the Boston starting line in April.

“Just to be able to run with CF is kind of crazy and awesome,” he said. “But, like anything, you just have to focus on what’s in front of you today, and breathe the best you can.”

Immunotherapy May Be Promising Strategy to Treat Chronic Infections of P. aeruginosa in CF Patients

Original article on CF News Today

Blocking the interaction between two molecules involved in immune restraining mechanisms — called PD-1 and PD-L1 — may be an effective therapeutic strategy to boost the immune system and treat chronic infections of Pseudomonas aeruginosa in cystic fibrosis (CF) patients, according to a recent study.

The study, “Pseudomonas aeruginosa colonization causes PD-L1 overexpression on monocytes, impairing the adaptive immune response in patients with cystic fibrosis,” was published in the Journal of Cystic Fibrosis.

Chronic lung infections by P. aeruginosa —a highly antibiotic-resistant bacteria — are the leading cause of death among CF patients.

The high frequency of infections in CF patients suggest they have an impaired immune system. Furthermore, previous studies have shown that these patients have what is called an endotoxin tolerance, meaning a reduced responsiveness to pathogens following a first contact, leading to a desensitization of immune cells against them.

Researchers have proposed that the passage of bacteria to the bloodstream, inducing a systemic exposure to their LPS — a major component of the cell wall of some bacteria, including P. aeruginosa, that is recognized by the body’s immune system as foreign —, either attached to the membrane or in a soluble form, may explain the endotoxin tolerance observed in CF patients.

Recent evidence has shown that PD-L1 is a marker of endotoxin tolerance. PD-L1/PD-1 interaction has emerged as an important mechanism in the immune response, where the binding of PD-L1, present in many cells, to its PD-1 receptor on immune T-cells prevents the activation of T-cells — a type of white blood cell that fights infections and cancer cells.

Targeting and blocking this interaction has been shown to promote T-cell activation, and to be associated with a protective effect against infections.

However, the potential role of PD-L1/PD-1 interaction in CF, and especially in CF patients infected by P. aeruginosa, remains unexplored.

Researchers in Madrid evaluated the levels of PD-L1 and PD-1 in CF patients, and the potential association between PD-L1/PD-1 interaction and P. aeruginosainfection.

The team analyzed the levels of PD-L1 in monocytes — a type of white blood cell that is in the frontline of infection, and communicates with T-cells — and of PD-1 in T-cells of 32 CF patients, 19 of them infected by P. aeruginosa, and of healthy volunteers. These cells also were grown in the lab to assess their proliferation and immune responses.

Results showed that CF patients had significantly higher levels of PD-L1 in monocytes and PD-1 in T-cells than healthy people, and that PD-L1 levels — both in monocytes and in a soluble form in the blood — were even higher in CF patients infected with P. aeruginosa.

These data, combined with the fact that CF patients infected with P. aeruginosaalso showed a significant impairment in T-cell proliferation, highlighted an association between P. aeruginosa infection and an increased suppression of T-cells.

In accordance, additional analysis showed that endotoxin tolerance in monocytes was stronger in CF patients infected with P. aeruginosa than in those who were not.

Interestingly, the addition of an antibody that blocks PD-L1/PD-1 interaction to T-cells from patients infected with the bacteria significantly increased the proliferation of T-cells to levels almost comparable to those of healthy people and patients without the infection, suggesting this type of approach could revert the impaired immune response and benefit these CF patients.

The team also found evidence strengthening the hypothesis that bacteria/LPS presence in the blood induces increased levels of PD-L1 and endotoxin tolerance, as the levels of PD-L1 in monocytes from healthy people were significantly increased when in contact with isolated LPS of P. aeruginosa, or with blood of CF patients infected with the bacteria.

This PD-L1 increase with blood of infected CF patients was reduced when monocytes were pretreated with colistin (polymyxin E), a potent antibiotic that neutralizes or absorbs LPS. CF patients treated with colistin had lower levels of soluble PD-L1 in their blood.

These results suggest that colistin could be used to reduce the effects of bacteria passage to the bloodstream in CF patients infected with P. aeruginosa. Nonetheless, the team noted that future clinical trials are needed to confirm this hypothesis.

Overall, based on the results, the team concluded that “Pseudomonas aeruginosa colonization in patients with CF was associated with PD-L1 overexpression and impaired T-cell response, and LPS from this pathogen induced the observed phenotype.”

“Altogether, our results suggest the possibility of studying antibiotics and antiPD-1/PD-L1 antibody combination as a new potential treatment to provide clinical benefits to patients with P. aeruginosa colonization,” the researchers added.

Patients with lung disease could find relief by breathing in messenger RNA molecules

Original article on Science Daily.

Messenger RNA, which can induce cells to produce therapeutic proteins, holds great promise for treating a variety of diseases. The biggest obstacle to this approach so far has been finding safe and efficient ways to deliver mRNA molecules to the target cells.

In an advance that could lead to new treatments for lung disease, MIT researchers have now designed an inhalable form of mRNA. This aerosol could be administered directly to the lungs to help treat diseases such as cystic fibrosis, the researchers say.

“We think the ability to deliver mRNA via inhalation could allow us to treat a range of different disease of the lung,” says Daniel Anderson, an associate professor in MIT’s Department of Chemical Engineering, a member of MIT’s Koch Institute for Integrative Cancer Research and Institute for Medical Engineering and Science (IMES), and the senior author of the study.

The researchers showed that they could induce lung cells in mice to produce a target protein — in this case, a bioluminescent protein. If the same success rate can be achieved with therapeutic proteins, that could be high enough to treat many lung diseases, the researchers say.

Asha Patel, a former MIT postdoc who is now an assistant professor at Imperial College London, is the lead author of the paper, which appears in the Jan. 4 issue of the journal Advanced Materials. Other authors of the paper include James Kaczmarek and Kevin Kauffman, both recent MIT PhD recipients; Suman Bose, a research scientist at the Koch Institute; Faryal Mir, a former MIT technical assistant; Michael Heartlein, the chief technical officer at Translate Bio; Frank DeRosa, senior vice president of research and development at Translate Bio; and Robert Langer, the David H. Koch Institute Professor at MIT and a member of the Koch Institute.

Treatment by inhalation

Messenger RNA encodes genetic instructions that stimulate cells to produce specific proteins. Many researchers have been working on developing mRNA to treat genetic disorders or cancer, by essentially turning the patients’ own cells into drug factories.

Because mRNA can be easily broken down in the body, it needs to transported within some kind of protective carrier. Anderson’s lab has previously designed materials that can deliver mRNA and another type of RNA therapy called RNA interference (RNAi) to the liver and other organs, and some of these are being further developed for possible testing in patients.

In this study, the researchers wanted to create an inhalable form of mRNA, which would allow the molecules to be delivered directly to the lungs. Many existing drugs for asthma and other lung diseases are specially formulated so they can be inhaled via either an inhaler, which sprays powdered particles of medication, or a nebulizer, which releases an aerosol containing the medication.

The MIT team set out to develop a material that could stabilize RNA during the process of aerosol delivery. Some previous studies have explored a material called polyethylenimine (PEI) for delivering inhalable DNA to the lungs. However, PEI doesn’t break down easily, so with the repeated dosing that would likely be required for mRNA therapies, the polymer could accumulate and cause side effects.

To avoid those potential side effects, the researchers turned to a type of positively charged polymers called hyperbranched poly (beta amino esters), which, unlike PEI, are biodegradable.

The particles the team created consist of spheres, approximately 150 nanometers in diameter, with a tangled mixture of the polymer and mRNA molecules that encode luciferase, a bioluminescent protein. The researchers suspended these particles in droplets and delivered them to mice as an inhalable mist, using a nebulizer.

“Breathing is used as a simple but effective delivery route to the lungs. Once the aerosol droplets are inhaled, the nanoparticles contained within each droplet enter the cells and instruct it to make a particular protein from mRNA,” Patel says.

The researchers found that 24 hours after the mice inhaled the mRNA, lung cells were producing the bioluminescent protein. The amount of protein gradually fell over time as the mRNA was cleared. The researchers were able to maintain steady levels of the protein by giving the mice repeated doses, which may be necessary if adapted to treat chronic lung disease.

Broad distribution

Further analysis of the lungs revealed that mRNA was evenly distributed throughout the five lobes of the lungs and was taken up mainly by epithelial lung cells, which line the lung surfaces. These cells are implicated in cystic fibrosis, as well as other lung diseases such as respiratory distress syndrome, which is caused by a deficiency in surfactant protein. In her new lab at Imperial College London, Patel plans to further investigate mRNA-based therapeutics.

In this study, the researchers also demonstrated that the nanoparticles could be freeze-dried into a powder, suggesting that it may be possible to deliver them via an inhaler instead of nebulizer, which could make the medication more convenient for patients.

TranslateBio, a company developing mRNA therapeutics, partially funded this study and has also begun testing an inhalable form of mRNA in a Phase 1/2 clinical trial in patients with cystic fibrosis. Other sources of funding for this study include the United Kingdom Engineering and Physical Sciences Research Council and the Koch Institute Support (core) Grant from the National Cancer Institute.

Losing disability insurance is a possible nightmare for those with CF

Original article on CBS This Morning

Megan Willis lives with cystic fibrosis, a deadly disease that causes extensive lung damage. The 22-year-old said she spends around six hours a day administering medications and therapy, and that the disease frequently causes infections and other complications.

With the condition, Willis qualified as a disabled adult for Social Security benefits on living expenses. About 10 million other Americans too disabled for work also get the stipend, called Disability Insurance. More importantly, having Social Security gave Willis access to Medicaid, which paid her annual health care costs of over $100,000.

But in March, Social Security sent her a letter saying her health had improved since the last review of her case and that she was able to work. This was news to Willis.

“My health has only gotten worse in the past year,” Willis told CBS News chief medical correspondent Dr. Jon LaPook.

Despite that, her Social Security benefits were terminated and she lost Medicaid. Since she lives in Florida, one of 14 states without expanded Medicaid, she had no other way to get it. Her family can’t afford private insurance.

While the medical bills mounted, she began law school.

“I don’t want to just stay stagnant and you know, depressed, looking at the four walls of my room,” Willis said. “I want to move up in the world even if you know it’s going to be hard.”

Willis also contacted attorney Beth Sufian, who runs the Cystic Fibrosis Legal Hotline and has cystic fibrosis herself. Sufian said, “We’ve seen a five-times increase in the number of people with cystic fibrosis that have been reviewed in the past 18 months. And we think that Social Security is targeting young people with chronic illness in an effort to reduce the number of people getting benefits.”

By law, disability claims are periodically reviewed to see if recipients are still eligible for benefits. Over the last decade, to combat a backlog, full medical reviews quadrupled to an expected 900,000 this year.      

“It really is a life or death situation for all of our clients when they lose their benefits,” Sufian said.   

In Willis’ case, Social Security ultimately reconsidered and she was able to get back on disability in November. She was hospitalized around Thanksgiving, and Medicaid kicked in and the bills were covered, her mother Wendy said.

But Willis’s lawyer still has about 200 pending cases of people with cystic fibrosis who are first getting reviewed and those who are appealing.

***

Losing disability insurance can have devastating consequences for those with CF. Our very own Vice President, Beth Sufian, helps many individuals get on and stay on disability. For the full story go to https://www.cbsnews.com/…/what-happens-when-someone-loses-…/

Insufficient antibiotics available for cystic fibrosis patients: Study

Turns out, the majority of patients with cystic fibrosis may not achieve blood concentrations of antibiotics sufficiently high enough to effectively fight bacteria responsible for pulmonary exacerbations, thus leading to worsening pulmonary function.

Cystic fibrosis, a genetic condition that affects about 70,000 people worldwide, is characterised by a buildup of thick, sticky mucus in patients’ lungs. There, the mucus traps bacteria, causing patients to develop frequent lung infections that progressively damage these vital organs and impair patients’ ability to breathe.

A recent study led by researchers at Children’s National Health System shows that it’s impossible to predict solely from dosing regimens which patients will achieve therapeutically meaningful antibiotic concentrations in their blood. The findings were published online in the Journal of Pediatric Pharmacology and Therapeutics.

These infections, which cause a host of symptoms collectively known as pulmonary exacerbations, are typically treated with a combination of at least two antibiotics with unique mechanisms. One of these drugs is typically a Beta-lactam antibiotic, a member of a family of antibiotics that includes penicillin derivatives, cephalosporins, monobactams and carbapenems.

Although all antibiotics have a minimum concentration threshold necessary to treat infections, Beta-lactam antibiotics are time-dependent in their bactericidal activity. Their concentrations must exceed a minimum inhibitory concentration for a certain period. However, study’s lead author Andrea Hahn explained that blood concentrations of Beta-lactam antibiotics aren’t typically tracked while patients receive them.

Since antibiotic dosing often doesn’t correlate with cystic fibrosis patients’ clinical outcomes, Dr. Hahn and other researchers examined whether patients actually achieved serum antibiotic concentrations that are therapeutically effective.

In addition, all the patients underwent pulmonary function tests at the start of their exacerbations and about once weekly until their antibiotic therapy ended.

Using the data points, the researchers constructed a model to determine which patients had achieved therapeutic concentrations for the bacteria found in their respiratory secretions. They then correlated these findings with the results of patients’ pulmonary function tests. Just 47 per cent of patients had achieved therapeutic concentrations. Those who achieved significantly high antibiotic exposure had more improvement on their pulmonary function tests compared with patients who didn’t.

Paradoxically, they discovered that although each patient received recommended antibiotic doses, some patients had adequately high serum antibiotic concentrations while others did not.

Another way to ensure patients receive therapeutically meaningful levels of antibiotics is to develop new models that incorporate variables such as age, gender, and creatinine clearance–a measure of kidney function that can be a valuable predictor of metabolism–to predict drug pharmacokinetics.

Using findings from this research, Dr. Hahn adds, Children’s National already has implemented an algorithm using different variables to determine antibiotic dosing for patients treated at the hospital.

Original article here.