‘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.

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!

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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.

Vertex Pharmaceuticals opens expanded San Diego research center with focus on cystic fibrosis

By Bradley J. Fikes

Vertex Pharmaceuticals opened its new San Diego research center Monday, starting a new chapter in a decades-long quest to not only treat but cure cystic fibrosis.

In 18 years, three drugs for the lung-ravaging disease have emerged from Vertex’s San Diego center and more are in the pipeline.

The first, Kalydeco, was approved in 2012. It is the first drug that treats the underlying cause of the disease. The second, Orkambi, was approved three years later. And the third, Symdeko, was approved in February.

These drugs can benefit about half of all patients with the incurable disease. In the next several years, Boston-based Vertex hopes its drugs can help nearly all patients live longer, healthier lives.

Cystic fibrosis is caused by a genetic defect that allows a buildup of thick mucus in the lungs, and other internal organs. This mucus clogs airways and promotes the growth of bacteria. The average lifespan of patients is 37 years, up from 20 years in 1980. Treatments include antibiotics to fight lung infections and mucus-thinning drugs.

The new 170,000 square-foot building on Torrey Pines Mesa more than doubles the company’s space. The center includes cell culturing equipment to grow lung cells from patients, to be used for drug screening. A 4,000 square-foot incubator suite will serve outside collaborators.

Asides from cystic fibrosis, the staff will work on other serious diseases.

Among the speakers Monday morning was a veteran in the fight against cystic fibrosis: Jennifer Ferguson, who has two children with the disease, Ashton and Lola. Both her children are taking Vertex drugs, and both were present with her at the event.

With these drugs and the promise of better therapies ahead, she says Ashton and Lola have a good chance of growing up and leading their own lives. She urged all Vertex employees to think of themselves as part of a team to cure the disease.

Ferguson, of San Diego, found out about the work from the Cystic Fibrosis Foundation. The foundation had invested $30 million in startup Aurora Biosciences to find therapies.

In 2001, Vertex purchased Aurora for $592 million in stock, the same year Ashton was diagnosed. The research went on under Vertex, and Ferguson became quite familiar with the research team.

“The Cystic Fibrosis Foundation asked me to come speak, to show them what it’s like to have a little child with CF,” she said. “So I came here about 17 years ago with him as a 6-month-old.”

At that time, many cystic fibrosis patients never reached adulthood.

“I had a hard time keeping it together,” Ferguson told the audience of that long-ago visit.

“But I looked in the staff’s faces — and some of you are still here — and I thought, I’m going to put my faith and trust in your hands, in your brains. And I was able to let go of my worry, because you were on the case.”

Ferguson started visiting every few years to check on what progress was being made, first with Ashton, and later including Lola. She also raises money for the Cystic Fibrosis Foundation.

Both her children have shown improvement since starting the Vertex drugs, Ferguson said. But they still need to go through a daily regimen of clearing out their lungs.

From medications, the research frontier has advanced to investigations into a cure. That means fixing the genetic defect, which can come in several variations, inside living patients.

That cure might come from the hot new gene editing technology called CRISR. In 2015, Vertex allied with startup CRISPR Therapeutics to develop curative therapies.

This post was originally published on The San Diego Union-Tribune

7 Things Your Partner with CF Probably Isn’t Telling You

By Hannah Buck

Being in a committed, loving, long-term relationship is a distinctly intimate experience. It is the most intimate experience of humanhood many would argue. To see a person walking by and say to them, “Hey, you’re fairly OK-looking. Would you like to hold hands for forever and accrue debt until we die?” is to truly know companionship. That, and watching each other poop.

Chronic illness makes dating a thoroughly more vulnerable experience, and not just for the patient. You see, sickness affects everyone involved. It accelerates everything. Sickness makes daily life complicated (e.g. planning dates that aren’t physically taxing or one partner relying more on the other for help with everyday chores) and the future even less promising than it usually is. When you or your partner has a condition like cystic fibrosis, the bleak reality of your situation is sorely evident. It’s inescapable. One of you will die much sooner than the other. And with that intense reality flashing its lights 24/7, it can be tempting to hold things in.

I don’t speak for all people with CF in writing the following list — but by sharing what I’ve withheld in past romantic relationships, I hope to make you laugh, open your eyes, and help you become a better partner to the person whose hand you like to hold. Enjoy.

Things your partner with CF probably isn’t telling you

1. They’ve been wetting your bed for a while.

Have you ever rolled over in the middle of the night to feel a damp spot on the mattress? Has your girlfriend been known to spontaneously wash your sheets and comforter out of the goodness of her heart? Yeah, sorry to break it to you, it’s not because she’s an angel. It’s because she has coughing-induced, premature incontinence, and she doesn’t want you to know.

2. Their antibiotics give them diarrhea.

It just happens, OK! We don’t ask for this! Antibiotics have one mission: to kill. This includes good gut bacteria, which unfortunately messes up our tummies. Make your partner’s day by surprising them with a bottle of probiotic-rich kombucha to get things back on track.

3. They’d prefer if you looked away during their cough attacks.

Coughing ain’t cute. Yes, yes, I know you love them, but try to put yourself in your partner’s shoes. If you were red in the face, foaming at the mouth, hunched over like the Notre Dame character, and spewing phlegm like a swampy sprinkler, would you want the love of your life to gaze longingly upon you? Probably not. Give ’em space.

4. They wish you visited them in the hospital more.

They just feel too guilty to say it out loud. It is unspeakably lonely to sit in a small room and face the same wall every day. Please, even if they insist they’re fine, be there for them. Stop by. Make the time. If you can’t do that, text, call, or video chat. While 24 hours pass by in a snap in the outside world, in the hospital, the hours drag like you wouldn’t believe.

5. It makes them really happy when you randomly flex your CF knowledge.

Showing your partner that you care about them enough to not only learn about their disease but about how it’s treated is an instant way to grow closer. For many of us, the only people we have to confide in about this part of our lives is our medical team, our family, and occasionally other CFers (but only online). Take the time to learn what’s what — get the medication names right, make yourself an ally, and demonstrate that the two of you are teammates in this fight.

6. Explaining what’s “wrong” with them to other people makes their life so much easier.

With this one, I want to repeat my disclaimer: I am speaking for myself, and every person with CF is different. Please talk to your partner before taking this advice.

With that said, I have always found it to be an incredible relief when my partner discreetly says, “She has something called CF, so she coughs a lot. It’s normal. So anyway … ” and then changes the subject when I have a cough attack around people who don’t know me. Explaining myself is something I’ve had to do my entire life, so having someone else do it for me is a treat I savor every time.

7. They don’t feel worthy of your love, and they feel guilty about loving you.

I hope this one isn’t true for you guys. In my case, it is, and I suspect it’ll be a lifelong battle. Having an incurable illness is a heavy burden to bear, but when you’re born with it, there’s no other option. Putting it on another person, though, that’s different. That isoptional. And it can feel impossible to justify exposing the person whose hand you like holding to that level of lifelong pain.

This post originally appeared on CF News Today.