One Nation Under a Groove

The OG of funk, George Clinton. There’s not much that hasn’t been said about him. He is a visionary, eccentric and willing to let his freak flag fly high. He has been a powerhouse of music for the past 50+ years. He was born in NC, the moved to New Jersey in the 50’s when he formed his first doo-wop group, Parliament. Over the years, he transitioned from doo-wop, to soul, and then to funk with his bands Parliament and Funkadelic. Along his ride, he cultivated numerous musicians in his groups, from Bootsy Collins to Maceo Parker to Eddie Hazel to Berne Worrell, and on and on. If I named everyone that’s been in his various bands over the years we could be here a while! In the 70’s these two bands toured together as a supergroup, a mad party on stage that came with over the top costumes, props, and even a full sized spaceship called the mothership. Mothership Connection was one of Parliaments best albums, and during the show the mothership would land from above the stage and George would walk off in character as Dr. Funkenstein, strutting off the mothership to the delight of the crowd. Throughout the show there would be numerous southern and theme changes. Their scene was one of decadence, sex, drugs, and funk. It was a party, and throughout the night the crowd felt like they were part of it.

I remember one of the Parliament Funkadelic shows I saw, obviously many years later, up in Milwaukee. They came on at about 9 o’clock, and didn’t stop funking until about 330 or 4am. No set breaks, no stopping the groove. Musicians would rotate on and off without missing a beat. Sometimes there would be as many as 20 or so people on stage. They continued to push the energy, further and further, until everyone on stage and in the audience was ready to collapse. When it was over I was so exhausted and exhilarated all at the same time, like I had been through a great musical journey. It was one of the best shows I had ever seen, and one I’ll never forget. I had a permanent-grin for the next week or so afterwards.

The reason why I bring up One Nation Under a Groove, one of Funkadelics best songs, is because of recent CF news (article is below from FDA). You may have read about Trifekta, a CFTR modulator that should help up to 90% of the CF population, that was quickly approved by the FDA. The reason they were able to fast track the approval was because of the great data in the clinical trials. I was one of the test subjects; most of you know that I’ve been involved in trials for years. This drug approval was the endpoint of the years of ups and downs, failed combinations and subsequent exacerbations, trying to get back to the magical 40% FEV to continue to qualify for the trial. In the past 6-7 months during my “open label,” I have been taking the drug that is now called Trifekta with great improvement of my symptoms. I have been able to avoid hospitalizations for almost 11 months now, which is fantastic. My day to day symptoms are a lot better, and I have been able to feel more like I had hoped for all those years. So why didn’t I tell everybody about this earlier?

There’s a couple reasons for that. I could not give too many specifics or name the drug or the company due to the ongoing trial; if I did that I could have been disqualified. There are some very strict rules that are set in place by the review board to ensure that the drug is beneficial on its own merits and without any bias. So, I was not going to mess that up! The other and more important reason, why it has taken me a few weeks to write about this, is guilt. Guilt about the part of the CF population that cannot take this drug for one reason or another. It’s difficult to talk about your success when plenty of others are struggling to stay healthy. I equate it to survivor’s guilt of a mass tragedy. On one hand, you are grateful and happy for your good fortune. However, you keep that happiness inside, as to not feel like boasting to those who are suffering. That’s how I feel about this drug; I am very happy with the benefit it has given me but I am also sad and feel slight embarrassment sharing that happiness as many other people are continuing to struggle.

My hope is that this great research and therapies that have improved so many lives will continue to gain momentum, until all CF patients can benefit from this cutting age medication. So, to the community out there: keep hope and faith alive, continue to look positively to the future, and don’t give up until a cure is found!!

Article from FDA:

The U.S. Food and Drug Administration today approved Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. Trikafta is approved for patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population.

“At the FDA, we’re consistently looking for ways to help speed the development of new therapies for complex diseases, while maintaining our high standards of review. Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy,” said acting FDA Commissioner Ned Sharpless, M.D. “In the past few years, we have seen remarkable breakthroughs in therapies to treat cystic fibrosis and improve patients’ quality of life, yet many subgroups of cystic fibrosis patients did not have approved treatment options. That’s why we used all available programs, including Priority Review, Fast Track, Breakthrough Therapy, and orphan drug designation, to help advance today’s approval in the most efficient manner possible, while also adhering to our high standards. The FDA remains committed to advancing novel treatment options for areas of unmet patient need, particularly for diseases affecting children.”

Cystic fibrosis, a rare, progressive, life-threatening disease, results in the formation of thick mucus that builds up in the lungs, digestive tract, and other parts of the body. It leads to severe respiratory and digestive problems as well as other complications such as infections and diabetes. Cystic fibrosis is caused by a defective protein that results from mutations in the CFTR gene. While there are approximately 2,000 known mutations of the CFTR gene, the most common mutation is the F508del mutation.

Trikafta is a combination of three drugs that target the defective CFTR protein. It helps the protein made by the CFTR gene mutation function more effectively. Currently available therapies that target the defective protein are treatment options for some patients with cystic fibrosis, but many patients have mutations that are ineligible for treatment. Trikafta is the first approved treatment that is effective for cystic fibrosis patients 12 years and older with at least one F508del mutation, which affects 90% of the population with cystic fibrosis or roughly 27,000 people in the United States.

The efficacy of Trikafta in patients with cystic fibrosis aged 12 years and older was demonstrated in two trials. The first trial was a 24-week, randomized, double-blind, placebo-controlled trial in 403 patients who had an F508del mutation and a mutation on the second allele that results in either no CFTR protein or a CFTR protein that is not responsive to ivacaftor or tezacaftor/ivacaftor alone. The second trial was a four-week, randomized, double-blind, active-controlled trial in 107 patients who had two identical F508del mutations.

In each trial, the primary analysis looked at increases in the percent predicted forced expiratory volume in one second, known as ppFEV1, which is an established marker of cystic fibrosis lung disease progression. Trikafta increased the ppFEV1 in both trials. In the first trial, it increased mean ppFEV1 13.8% from baseline compared to placebo. In the second trial, it increased mean ppFEV1 10% from baseline compared to tezacaftor/ivacaftor. In the first trial, treatment with Trikafta also resulted in improvements in sweat chloride, number of pulmonary exacerbations (worsening respiratory symptoms and lung function), and body mass index (weight-to-height ratio) compared to placebo.

The safety profile of Trikafta is based on data from the 510 cystic fibrosis patients in the two trials. The safety profile was generally similar across all subgroups of patients. Serious adverse drug reactions that occurred more frequently in patients receiving Trikafta compared to placebo were rash and influenza (flu) events. The most common adverse drug reactions included headaches, upper respiratory tract infections, abdominal pains, diarrhea, rashes, increased liver enzymes (alanine aminotransferase and aspartate aminotransferase), nasal congestion, increased blood creatine phosphokinase (an enzyme that can be associated with muscle damage), rhinorrhea (mucus in the nasal cavity), rhinitis (swelling of the mucous membrane of the nose), influenza, sinusitis and increased blood bilirubin (may be caused by problems involving the liver, gallbladder or red blood cells).

The prescribing information for Trikafta includes warnings related to elevated liver function tests (transaminases and bilirubin), use at the same time with other products that are inducers or inhibitors of another liver enzyme called Cytochrome P450 3A4 (CYP3A), and the risk of cataracts. Patients and their caregivers should speak with a health care professional about these risks and any medicines they take before starting treatment.

Patients with cystic fibrosis should speak with a health care professional and have tests performed to understand which gene mutations they have. The presence of at least one F508del mutation should be confirmed using an FDA-cleared genotyping assay prior to treatment. The safety and effectiveness of Trikafta in patients with cystic fibrosis younger than 12 years of age have not been established.

The FDA granted this application Priority Review, in addition to Fast Track and Breakthrough Therapy Designation. Trikafta also received orphan drugdesignation, which provides incentives to assist and encourage the development of drugs for rare diseases. Drugs approved under expedited programs are held to the same approval standards as other FDA approvals. Because of Trikafta’s benefit to the cystic fibrosis community, the FDA reviewed and approved Trikafta in approximately three months, ahead of the March 19, 2020 review goal date. The approval of Trikafta was granted to Vertex Pharmaceuticals Incorporated, which will receive a Rare Pediatric Disease Priority Review Voucher for developing this therapy.

Touch of Grey

So I’m driving down the highway the other day, and this song comes on. Now normally, I’m not a big singer-in-the-car type, but I was really feeling it. So I start to belt out the chorus, ” I will get by, I will survive!” And I felt a little better instantly. It’s the power of music, like a cathartic release from the stresses and difficulties of life.

Touch of Grey is by the Grateful Dead, one of their most recognizable songs. In fact, it was the only top 10 hit for the band (it peaked at #9), which is remarkable considering the wealth of songs the band recorded from 1965 until Jerry’s untimely death in 1995. Their career was so vast yet remained mostly underground to the general population. It seemed that it was a secret to the community of Deadheads, of which I consider myself an honorary member. I was just getting into the Dead in high school and went to “the lot” for their concert at Soldier Field in 1995, if only to experience the scene. It was eye-opening; the sights, sounds, the smells! It was truly a community unto its own, with people who followed the band around the country. They looked out for each other, and forged relationships that were unique to the tour. People sold merchandise, shirts, grilled cheeses, or other things…….just to make enough to get to the show or the next town. At Soldier Field that night I didn’t have a ticket or much money, so I was content to kick it in the lot with some new friends. I always thought I would make it to more shows, but alas it was not to be. Jerry died a little less than a month later, August 9, 1995, of a heart attack. He had been in and out of rehab, had horrible health problems stemming from obesity, diabetes, and longstanding drug addictions. It seemed to be a byproduct of the lifestyle of a traveling musician; some figured out how to take proper care of themselves, but he never did. The band continued on after his death, in many different forms, with many different people trying to fill the void. I have been to quite of few of those ensembles over the years, but it was not the same without Jerry.

Thinking about Touch of Grey, and how it applies to my life now, its kind of an obvious correlation. All I have to do is look in the mirror, and I see a little grey in my beard, a few grey hairs and evidence that I am getting older. Robert Hunter, Jerry’s songwriting partner, wrote this song in reference to the band growing old gracefully. I think about that, and how I’ve tried to evolve over the years. To be a better version of myself, correcting my past mistakes and behavior. Is this a perfect evolution? Of course not! There are plenty of moments where I feel myself reverting to my past, not taking proper care of my health or acting like a child. But I’m conscious of those moments, try to take a deep breath, and stop those tendencies before they take over. It’s not a perfect system, and there are plenty of times when I am not happy with myself. But I try to have more good moments than bad. Like the song said, “Every silver lining has a touch of grey.”

Having gone through all I have in life, I try to draw on those experiences and that perspective to then evolve into a better person. I think about all I have now, as well as all the struggles and difficulties (both physical and mental) that have led me to the point where I have grey hairs! If I told my teenage self that I would still be not only kicking but successful at 40 years old, I would have laughed at myself and dismissed the possibility. But here I am. I keep waking up everyday, and moving forward. So I try to keep progressing and growing as a person, a man, a father, a husband, and a CF elder statesman. 🙂

Also, as it is Sept 11th today, I want to take a quick moment to remember the people who were tragically lost on that day, and the first responders and everyone who put themselves at risk to help. That was the best version of America, how we came together as one to pick ourselves off the mat. Hopefully, we as a nation can get back to that America. NEVER FORGET!!

Like a Hurricane

This song seems very appropriate today, as another deadly storm is barreling down on Puerto Rico and Florida. Written and performed by Neil Young, one of the best singer/songwriters of all time, his lyrics can be applied to the masses. That has been one of his great gifts. Some people can write a song or two that relates to people of all backgrounds or generations. Neil Young has been writing those kinds of songs for 50 years. Canadian born, he started his ascent as a part of Buffalo Springfield, co-writing or contributing to great songs like Mr. Soul, For What it’s Worth, and Bluebird with another budding songwriting great Stephen Stills. Their great chemistry led to Neil Young being including in the collaborative group of Crosby, Stills, Nash, and Young. I could write about that group on its own merits, but today I want to focus on Neil Young. He can paint a picture with his words, relatable to most everyone’s experience at some point in their lives. It’s astounding, and he has done it over and over again.

Like a Hurricane depicts a love that was both irresistible and toxic at the same time. While you are in the eye, you feel calm. But once you are blown the the edges of the storm, you can see the intensity and damage that is being done.

You are like a hurricane
There’s calm in your eye
And I’m gettin’ blown away
To somewhere safer where the feeling stays
I want to love you but I’m getting blown away

Now, this does not have to apply to one thing alone. One could relate to many things in life: a lover, a family member, a close friend, or even an illness. I can say that CF has felt like a hurricane at many points in my life, creating chaos and destruction to all aspects of my immediate surroundings. I have lost multiple lovers and close friends over the years, as it can be traumatic to be a bystander. The unrelenting cycle of illness and hospitalizations have created conflict and hurt to my family and marriage. CF can be out of anyone’s control sometimes, like a rabid dog that needs to be put down. But you can only push it down but so far before it comes back seemingly harder and stronger than before.

Sometimes, in the middle of the storm, you can’t assess the damage it is inflicting. Instead, you are just trying to push through and get to the other side. But when you have the ability to step back, you can see the effects, like a boat leaving ripples in its wake. Since I have been a little healthier, and CF hasn’t taken over a majority of my thoughts and concerns for the time being, I have been looking back at the damage. The ones most obviously affected are the ones closest to you: my wife, son, brother and mother. I try to think about their mindset, being concerned about someone they love, watching them struggle while totally helpless to stop the progression. And after a hospitalization and I improve, the people around me can then breathe, but at the same time are holding their breaths for the next storm coming through. Over the years, the storms have been more frequent and severe. (Just like the effects of global warming have caused worsening and more violent weather. It’s a real thing people!!). Since the storm has seemingly passed, even for the moment, it’s hard not to brace for the next one. Yes, the new drug therapy has done wonders. But by no means it is over. I’m not cured. Part of me can’t help wondering when the next illness will hit, and I’m sure the people around me feel the same. What happens to me happens to them as well. So while I’m not metaphorically boarding up my windows for a new storm, the chronic and unrelenting nature of this depicts there is a storm on the horizon. I just don’t know how far away.

Free Fallin’

35 seconds. That’s how long you are in free fall after jumping out of a plane, or at least that’s what they told me. It feels like a lot longer, a mix of adrenaline, fear, and excitement, overloading your senses and distorting all sense of time. And when the parachute opens and you can really take in where you are and how beautiful the scenery is, you are humbled by how monstrous the world is compared to our tiny being. On the way, and afterwards, I had this song in my head.

It seems like an obvious leap that I would choose this song around this experience. One of the quintessential Tom Petty songs, Free Fallin’ is a song that everyone knows and enjoys. The lyrics describe a girl from the suburbs and them falling in love, and riding the roller coaster that is love. There are highs and lows, and all of it is new and exciting. I think we can all relate to that. Tom Petty was always one of my favorites. His gift was taking a simple 3 chord progression, then writing a fantastic song that is catchy, a song one can sing at will and a story that related to them. He had humble beginnings, and always seemed to be down to earth. He was able to relay that into his lyrics, which seemed universal to all of our experiences in life. I was really sad when he died; I think his heart finally gave out from the lifestyle of booze and drugs. He had his problems, but always seemed to bring a great energy in concert. His shows were great, one big sing-a-long where the crowd’s energy would be palpable, rising to a crescendo, giving you chills. Free Fallin’, Breakdown, American Girl, and others. He will be missed.

Speaking of rising crescendos, lets get back to my Free Fallin’. I’ll take you back 10 minutes before I jumped out of a perfectly good airplane. We were standing in the parking lot, and I had my harness on, already anxious and filled with anticipation. Then I see the plane! It looks like it can barely hold 4 people, and was so light I was amazed. My nervous excitement was further building, even more than it had been the entire morning. We take off on a gravel runway, and slowly build up to 10,000 feet where we jumped. I had no concept of how high we were, it was all very overwhelming. I could feel my excitement and fear building to a boiling point. But there was a positive; I felt very secure in my harness, and was attached to my tandem jumper with 3 super solid clips that locked in. Everything was reminiscent of climbing gear, and so that made me feel more comfortable.

But then, just as I’m starting to feel comfortable, he shouts “1 minute” and they opened the door to the plane!!! Holy shit was my first reaction……a blast of cold air and then I could really see the ground below, A LONG WAY DOWN!!!! I tried to control my breathing, but it was nearly impossible. So I put my feet on the ledge as instructed and looked down. Everything was so small, and I had a moment of hesitation. The tandem jumper, doing this all day every day, wisely gave me a nudge forward rather than let me stew upon my current position. And we were off……Free Fallin’! The blast of wind was exhilarating, and we settled into nice body position quickly, and I was able to really take it in and enjoy. I screamed in delight the entire free fall. When he finally pulled the chord for the parachute, I was surprised that it was not more jarring; it didn’t even hurt! I had imagined it jerking me and causing issues with my port, but I was able to position the shoulder straps laterally so they were not directly over the hub. The slow descent afterwards was relaxing and breaktaking, taking in the landscape and the mountains in the distance. I was able to “drive” the parachute, taking little turns and helping us down to the landing spot. Nice and smooth. For the next hour or so, I felt the adrenaline pumping through me like I was hooked up to a generator, my hands and feet slightly tingly. It was an experience to remember, and an item checked off my bucket list!

Happy 4th of July everyone! Happy to be alive and healthy, and living life 🙂

A Day in the Life

This song, the last track on Sgt. Peppers Lonely Heart Club Band, by The Beatles, is a classic. The whole album from front to back is an all time favorite, as it seamlessly blends from song to son as if it is one. The climax is A Day in the Life, written primarily by John Lennon. He was inspired by the newspaper articles at the time, in 1967. Also, it seemed, this song was inspired by their experiences with LSD. They were experimenting heavily then, and had various people pulling them in all kinds of directions. They met the Dalai Lama in 1966, which then inspired many different sounds, like incorporating the sitar and other Eastern instruments. When they were recording Sgt. Pepper’s, they wanted to be more Avant-Garde and pushing the boundaries.

That became very clear at the end of A Day in the Life, as they hired a 40 piece orchestra to build this crescendo to the final note. The movement builds, highlighted by the violins, slowly increasing their pitch and volume until you think they can’t go any higher without breaking all of their strings. Then, it finally releases into an E-major chord played simultaneously on 3 pianos, stretching out endlessly in a slow fade (actually it’s about 40 seconds!) At the end of it all, it leaves you breathless, wondering how in the hell they came up with all of these innovative sounds and recording techniques that had never been tried. This is one example of The Beatles’ genius, and their everlasting legacy.

I picked this song because of the title. I wanted to share a recent speech I gave at the National Jewish Research Conference. They asked me to give my perspective on the CD experience and how it pertains to research. Thus, A Day in the Life was perfect! I wasn’t able to upload the video, but there’s a link to audio here. Call it a mini podcast!


I will start writing more frequently soon, I promise. Right now I am enjoying the summer with my family. Peace and love to all.


Here Comes the Sun

So, I’m sitting here in Mexico, poolside. It’s almost 9am, and probably already 85 degrees or so. We are going to start drinking soon, because why not? We are on vacation with some close friends, all sharing a house for a few days. All this morning, this song seems to be on a loop in my head, no matter what song is playing through the speaker. Here Comes the Sun is one of my all time favorite songs, one of George Harrison’s gems and part of the opus album that is Abbey Road. It’s such a positive song, with his acoustic melody setting the mood. He said he wrote it in Eric Clapton’s backyard on a nice spring day. They were the best of friends at the time; oddly enough Eric was madly in love with George’s wife Patty Boyd. His emotional pain was the inspiration for the album Layla and accompanying love songs. I digress, but back to the point! Here Comes the Sun just makes me happy, as it should you. George’s wonderful acoustic guitar and arrangement compliment the lyrics. The words speak of a glimmer of hope after despair:

Little darling, it’s been a long cold lonely winter
Little darling, it feels like years since it’s been here
Here comes the sun
Here comes the sun, and I say
It’s all right

As I hear this song of positivity and hope I think about my current state of mood and health. I have been in a clinical trial for some time now, relating to the CFTR modulators (see further explanation below). Look more closely at the next generation modulators since it relates to my current trial. I can’t discuss all the particulars, like the company or drug name, because the data has not been fully collected and submitted to the FDA. However, since I finished the last phase, I recently entered what’s called “open label.” That means that regardless of my double blind group for the trial, I now receive the drug until it is available on the open market. Boy, have I felt the difference right away! I started coughing relentlessly for about 5 days, lots of nastiness coming up that probably had been in there for quite a long time. Once that was over, I feel so clear! It brought to mind a few other songs: I Can See Clearly Now by Johnny Nash, Breathe by Pink Floyd, Sun is Shining by Bob Marley, among others. Suffice to say, it is a drastic improvement. My PFTs have increased quite a bit as well; the FEV1 (forced exploratory volume in 1st second) has gone from 40 to 50% in just a couple of weeks! And my overall volume capacity has improved by 0.5L, which is quite significant. I can exercise much easier, and have a tolerance for more cardio. I want to springboard off this increase, and get back to the mountains for some hiking and climbing. It has been more difficult the past couple of years, which is frustrating since we live in the foothills!

As a result of this increase, I have been able to enjoy this vacation, without CF dominating every aspect of it. That has been the trend lately, we have big plans for a good vacation, and at some point CF rears its ugly head and takes over. Whether I get sick, or have a bad coughing episode, or I am unable to participate fully in the activities we have planned, and on, and on. There have been more times like this than I care to count. But not this time, not this vacation. And that gives me more hope for the future, something I haven’t had in a long time. So a quick thanks to all the people at the National Jewish research clinic and the CF clinic who have made this possible. For continuing to push me, to make me my best so as to qualify for these trials. Because these new meds have let me see a new day ahead. One where I don’t need as many hospitalizations and have less frequent exacerbations. As the song goes, “Here comes the sun, and I say, it’s all right!”

Information about CFTR Modulators (provided by the CF Foundation)

Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that affect the production of the CFTR protein. When the CFTR protein is not made correctly, it affects the balance of salt and fluids inside and outside of the cell. This imbalance leads to thick, sticky mucus in the lungs, pancreas, and other organs.

The Cystic Fibrosis Foundation supports the development of drugs that target specific defects in the CFTR protein. As a group, these drugs are called modulators because they are intended to modulate the function of the CFTR protein so that it can serve its primary function: to create a channel for chloride (a component of salt) to flow across the cell surface.

When proper chloride flow is reestablished, mucus becomes rehydrated inside the lungs and other organs. Although modulators can’t yet completely restore proper chloride flow, they can improve the flow enough to relieve symptoms for people with CF.

There are three main types of CFTR modulators:

  1. Potentiators
  2. Correctors
  3. Amplifiers


The first type of CFTR modulator is called a “potentiator.” Potentiators help chloride flow through the CFTR protein channel at the cell surface. The CFTR protein is shaped like a tunnel that can be closed by a gate. Potentiators hold the gate open so chloride can flow through.

The drug ivacaftor (Kalydeco®) is a potentiator. This drug can help patients with gating and conduction mutations in CFTR. It also works on residual function and splice mutations where an insufficient amount of normal protein is present. In all of these mutations, some CFTR protein reaches the surface of the cell. However, either not enough protein reaches the cell surface, or the protein does not allow enough chloride to flow through. By holding the gate on the CFTR protein open, potentiators allow more chloride to flow through and reduce the symptoms of CF.


The next type of CFTR modulator is called a “corrector.” Correctors help the CFTR protein to form the right 3-D shape so that it is able to move — or traffic — to the cell surface.

Almost half of people with CF have two copies of the F508del mutation, which prevents the CFTR protein from forming the right shape. The corrector drugs lumacaftor and tezacaftor help the CFTR protein to form the right shape, traffic to the cell surface, and stay there longer. But, even with lumacaftor and tezacaftor, only about a third of the CFTR protein reaches the cell surface, so by itself it can’t reduce the symptoms of CF.

Additionally, the proteins that do reach the cell surface do not open sufficiently to allow chloride to pass out of the cell. But, if a corrector is used in combination with a potentiator — such as ivacaftor — to hold the gate on the CFTR protein open, enough chloride can then flow to reduce the symptoms of CF. The combinations of lumacaftor/ivacaftor (Orkambi®) and tezacaftor/ivacaftor (Symdeko®) are therefore used to treat people with two copies of the F508del mutation. (Tezacaftor/ivacaftor also can be used to treat people with a single copy of one of 26 specified mutations –regardless of their other mutation.)


The last type of CFTR modulator is called an “amplifier.” Amplifiers increase the amount of CFTR protein that the cell makes. Many CFTR mutations produce insufficient CFTR protein. If the cell made more CFTR protein, potentiators and correctors would be able to allow even more chloride to flow across the cell membrane. Amplifiers, which are being developed and tested, are not yet available.

Next-Generation Modulators

Ivacaftor and lumacaftor are sometimes called “first-generation modulators” because they were the first modulators approved to treat people with CF. Tezacaftor, approved in Feruary 2018, is also considered a first-generation modulator.

“Next-generation” modulators are new and potentially more effective CFTR modulators. The next-generation CFTR correctors currently in clinical trials target different problems caused by the F508del mutation to further improve CFTR folding and increase the amount of CFTR trafficked to the cell surface. The goal of next-generation therapies, which will likely be part of a triple combination therapy, is to develop treatments that benefit more people, including individuals with a single F508del mutation.

Individually, each of the three drugs (which will likely first be composed of ivacaftor, tezacaftor, and a next-generation modulator) in a triple combination therapy addresses a different aspect of the defective CFTR protein. When used in combination, the results may be better than using one or two of the drugs alone. Already, several next-generation modulators in development have the potential to be significantly more effective than current FDA-approved modulators.

Next-generation modulators may also benefit more people with CF than first-generation modulators. The first-generation drug combination lumacaftor/ivacaftor can only help people with two copies of the F508del mutation, or about 50 percent of the people with CF. But, early clinical trial results suggest that several of the next-generation modulators could benefit people with CF who have only one copy of the F508del mutation. Because nearly 90 percent of the CF population has one or two copies of the F508del mutation, next-generation modulators would enable a much larger number of people with CF to have a modulator treatment.

Don’t Stand So Close to Me (Five Feet Apart)

I chose this song more for its ironic title than the content. It’s a song by the Police, released in 1980, apparently about a female student and a school teacher who have a forbidden sexual chemistry. While I like the song I don’t particularly like the content and background of the lyrics. The topic has been sung about before, like in Van Halen’s Hot for Teacher. The fact that Van Halen and the Police were awesome bands that doesn’t make it okay. We have all heard in the news about inappropriate relationships between female or male teachers and their students, and the consequences that resulted. I won’t glorify that any further, but will just say I liked the title of the song as it relates to the topic I really wanted to talk about, Five Feet Apart.

This is a book, written by Rachael Lippincott and collaborated on by many, about 2 teenagers with CF (Will and Stella). They are polar opposites: Stella is neurotic about her treatments and even developed an app to remind her to do and take everything on time. Will is more of a rebel, rarely doing his therapies and stating as soon as he turns 18 he will leave the hospital to live out his days on his own terms. He has a ticking clock since he has colonized a deadly bacteria, B. Cepacia (see explanation below). The main difficulty with this bacteria is its resistance to most or all antibiotics.

This is the backdrop of them “meeting” in the hospital. I say that in quotes because the isolation and infection control measures for us cystics in the hospital are extreme, to say the least. CF patients are isolated in their own rooms, and all medical personnel must wear full disposable gowns and sanitize in and out of every room. I am always astounded by the amount of gowns that are used just for me everyday, probably upwards of 50! Then if a CFer wants to leave their room, they must wear a mask and stay 6 feet apart from other patients. During the story, Will and Stella end up liking each other, and Stella decides to “steal” one foot of distance thereby making them Five Feet Apart. I won’t spoil the story or the ending, so you will have to read the book or go see the movie.

There has been an ongoing debate in the CF community about this book/movie, with wildly different points of view. Some believe that the CF isn’t accurately portrayed in the movie, and that is just a backdrop to the romantic storyline (which is also unrealistic). Others believe that while the CF and hospital depiction may not be fully accurate, it is important to show the mindset of a CF patient in the hospital. Also it could help raise awareness and publicity not only for the illness but how patients and their loved ones cope with this debilitating disease. I land somewhere in the middle; truth be told I only read the book and haven’t seen the movie. I think the book probably more accurately portrayed the CF hospital environment and the mindset of a patient, as books can offer a better imagery than a movie. However, I believe the movie is a good thing overall. So let’s discuss from both sides.

There are a few things that might turn people in the CF community off to this film. As seen in some of the trailers, both of the characters do not wear masks at all times, and are given free reign over the hospital to wander as they please. This may or may not be accurate depending on the institution, although the mask rule is not a variable and CF patients must wear them when around other CFers! I have been in hospitals where I was able to roam, and some when they wouldn’t allow me to leave the room let alone the floor. So it’s hard to judge that aspect. But, the romantic element to the movie is probably exaggerated and fantasized. Like anyone in the hospital is in the mood for love; the whole reason you are in the hospital in the first place…….is because you are sick!! And not just sick, but on oxygen, hooked up to IVs, fatigued, fighting with your body. Some people are waiting for lung transplants, or even too sick to be considered for one. I know when I’ve been at my lowest, I wasn’t thinking about hooking up with other people who are inpatient for exacerbations. There is too much else going on: treatments, exhaustion, effects of strong antibiotics, etc. Safe to say, it’s not a pretty picture. I’m just saying, the hospital romance seems very Hollywood. I’m sure that other elements of the hospital experience aren’t accurate, even though they consulted Claire Wineland. She was a CF activist and advocate who sadly died recently from complications of a lung transplant.

That brings me to the other side of this arguement, and the biggest positive of this movie: awareness and publicity. Any awareness and publicity for CF is a good thing in my view, as society as a whole does not know much about this disease. Even people who might know the basics, or know someone who has CF, they probably are unaware about the hospital experience, the daily difficulties and complications that come with this disease. Seeing it on the big screen, all that comes into play both physically and emotionally, is hard to relay to people. While this film may be slightly glorified, it’s not as dark as other movies centered around CF. When I was little, there was a film Alex, written by Frank Deford about his daughter Alex and her life. She died in 1980, and her short life was riddled with hospitalizations and complications. It was a very tough movie to see, so tough that I wasn’t allowed to watch it when I was young. Then there is A Fault in Our Stars, a more recent film with a similar pretext to Five Feet Apart. Two teenagers, one with B. Cepacia, fall in love online and get married despite all the warnings against. They both died shortly after. And………that’s about it. There haven’t been a lot of movies centered around CF, because it is so hard to depict given the subject matter. Five Feet Apart may not be the most real or depressing movie about CF, but it will show people some of the difficulties and hardships people struggle with their entire lives. They might tell their friends, or try to learn more about it. If more people know about CF, then more money can be raised for research, which can lead to a cure. CF can someday stand for Cure Found.

So go see the film. Bring your friends. Read the book. Learn more about this disease.

Watch the movie trailer below:

Information about B. Cepacia from the CF foundation:

Burkholderia bacteria are often resistant to many antibiotics, which makes them difficult to treat once they infect the lungs. However, some species may be successfully treated with combinations of antibiotics.

Basic infection prevention and control practices reduce the risk of getting or spreading B. cepacia. These bacteria pose very little medical risks to healthy people. However, some people who have a serious illness such as cancer or acquired immunodeficiency syndrome (AIDS) may be at risk of an infection from these bacteria.

How B. cepacia complex species affect people with CF varies. Researchers do not yet know why some people with CF are more likely to get B. cepacia than others.

In many people with CF, infection with B. cepacia may not worsen lung disease. In up to one-third of people infected with B. cepacia, the rate of lung function decline appears to be only slightly faster. However, for a smaller number of people, B. cepacia can cause a rapid decline in lung function and health. This can lead to more severe lung disease and maybe death.

Among the different species of B. cepacia complex, some may be more harmful than others (for example, B. cenocepacia and B. dolosa). The Cystic Fibrosis Foundation is working with researchers to learn more about the B. cepacia complex and help identify new treatments.

It is not always known how people with CF become infected with B. cepacia complex. Research has shown that people with CF can get B. cepacia from others who are infected with these bacteria. The germs spread either by direct contact, such as kissing, or indirectly from touching objects with the germs, such as doorknobs. This is known as cross-infection and can happen in social settings like events, gatherings or meetings.

In some cases, shared infection was not found in the lungs of a person with CF until two years after being exposed to someone else who was infected with the germ.

For many people with CF, infection with B. cepacia complex cannot be traced back to exposure to another infected person. In these cases, infection may have occurred by exposure to Burkholderia in the natural environment.

For the Love of Money

I was listening through a funk mix the other day, and this gem came on, courtesy of The O’Jays. Written by the songwriting pair Gamble and Huff who was largely credited with creating the Philadelphia sound of the 60s and 70s, it is a song that anyone can enjoy. Whether you’re a funkateer or not, or are part of different generations (millennials, Gen Xers, baby boomers, etc.), anybody listening to this song starts bobbing their collective heads to the rhythm. Starting with a hi-hat and funky guitar, then slowly added with a great baseline, and the melodic voices of the O’Jays that was very reminiscent of the Temptations. Everyone knows the words to this timeless classic; they hold true as much today as they did in 1973.

For the love of money
People will steal from their mother
For the love of money
People will rob their own brother

For a small piece of paper it carries a lot of weight

Call it lean, mean, mean green

Almighty dollar

While I was listening, I related this to a current and very important matter: drug prices. This is an issue that affects not only the CF community, but American society in general. Anyone with Diabetes who may need insulin, to someone with and auto-immune disease such as Rheumatoid Arthritis or Crohn’s disease, to a person with cancer. These people are clinging to hopes of any new medications that may be available, that in turn may increase not only their life expectancy but also quality of life. Mari and I were watching Hasan Minhaj’s new show on Netflix, Patriot Act, while he was discussing this very topic. America has the highest drug prices of any developed country, causing certain people to prioritize whether or not they can get their needed medication due to overwhelming increase in costs. People are traveling to other countries and smuggling back their meds, or spacing out their required daily medications and not taking them as prescribed because they cannot afford the refills. That is an incredulous statement for the richest country in the world, don’t you think?

For me, a person who with CF, I can relate to these struggles. While we were not poor growing up, I can only imagine the sacrifices my parents made in order for me to receive the proper care and all the newest treatments available. I know I probably didn’t appreciate it at the time, but I sure do now as an adult. There have been more than a couple times that I have not been able to afford my medications, due to a variety of reasons. Whether it was a poor insurance policy that only covered a minor percentage of my medications (most CF meds are top tier without any generic substitute), or not being able to shell out the hundreds of dollars for nutritional supplements or vitamins/minerals that are not covered on any insurance, or going to collections for the rising number of monthly payments to hospitals and clinics, the list goes on. All the while, I make a good living….this fact makes it more difficult to swallow. How can people with chronic illness afford be expected to succeed in this system when they can’t even afford what they need to survive?

This discussion becomes even more relevant to the CF community in the age of CFTR modulators. It’s a new class of drug that has been undergoing trials over the past several years. In short, it helps regulate the sodium chloride pump and prevents the buildup of sticky mucus congregating in the lungs. It’s important to point out that these drugs are not cures, but can significantly impact lives of those with CF. There are different classes of modulators, dependent on the specific genetic mutation of the person with CF.

Orkambi is one such drug, one that targets those with CF who have 2 forms of the most common genetic mutation (F508Delta). It is now on the market, at the low, low cost of approximately $100,000 per year!! Sounds like a bargain, right?! Not so much say the insurance companies. Certain companies, both private or state/federally regulated, have determined that the cost does not justify the possible benefit of this medication. In turn they are denying coverage for these meds. Imagine this……..someone who has lived their whole life just getting by until a possible life-altering medication comes by, and when it finally does it isn’t covered. What would you do? Most people, myself included, could not even fathom shelling out many thousands of dollars per month in order to afford the drug. This is another example of CF literally beating the life out of you…….even when there is hope it comes with heavy strings. Are you supposed to go into bankruptcy just to live? What kind of life is that? How is the government going to help these people?? Oh yeah……..they’re not! They are just going to try to reduce people’s access to the medical care and aide they need by reducing the qualifications for open market insurance and Medicaid expansion. It’s exhausting and infuriating!!

It all comes down to money: how much the drug companies can make, and how many politicians they can have in their pocket to influence their profit margins. Like the song says, “For a small piece of paper, it carries a lot of weight.” Truth!

Thinking Out Loud

I wanted to write a post to my wife, Mari, for Valentine’s Day. Both of us think that Valentine’s Day is more of a “Hallmark Holiday,” and thus we don’t really celebrate it. However, I thought that this would be a nice gift, a way to express how much she means to me.

Our life is rather complicated, more than most people our age. Largely, that has to do with CF rearing its ugly head more and more as time goes on. Add to that both of our careers, our teenage son, and managing our everyday life and it all adds up to…….A LOT!! Sometimes, we both feel like our head is going to explode, and we both just want to run far away from it all. But we don’t (obviously); I’d like to think that has to do with the deep love and respect we have for each other. I know for myself that she has made me a better man, husband, father, son, and person with chronic illness. So this song expresses a deep love that will last through thick and thin; this just so happens to be one of her favorites!

Thinking Out Loud, by Ed Sheeran, is a love song about growing old together and letting the love grow with age, like a fine wine. It has become one of the most successful singles of all time, having been streamed over a half a BILLION times and was number one on the charts for over a year. Sheeran sings this song as if he is older than he really is, like an old soul that has experienced this deep and long lasting love. In truth, he is only in his 20’s and probably has not. But that’s the power of music; being able to channel something that you may or may not have done or felt in life into something that resonates with the masses. In turn, the listener can interpret the song and relate that into their own life. This song is pretty transparent in its meaning of everlasting love, and has thus become a staple on the wedding circuit. Here are a few lyrics:

When your legs don’t work like they used to before
And I can’t sweep you off of your feet
Will your mouth still remember the taste of my love
Will your eyes still smile from your cheeks
And darling I will be loving you ’til we’re 70
And baby my heart could still fall as hard at 23

Cause honey your soul can never grow old, it’s evergreen
Baby your smile’s forever in my mind and memory

So this song is for you Mari. Not only are you incredibly beautiful, but you’re smart, funny, and possess an inner strength that is unmatched. You are my wife and partner, have had to navigate waters not many people can understand. Being a spouse of someone with CF means sitting by and watching the person you love go through immeasurable obstacles and life-threatening infections. All the while, that person has to manage the entirety of the life built together, as the hospitalizations grow in frequency and intensity, the ability to work and support becomes less and less, and the world you built together gets flipped upside down over and over and over……….. It’s exhausting, and the spouse often gets overlooked for all of the sacrifices made. Even if a person knew some of the hardships of CF they could not be ready for all it entails as the progression takes hold.

Mari, you are the love of my life, and my rock through all this crazy and maddening ride we call the CF train. I cannot imagine my life without you by my side, and these words cannot fully express how much you mean to me. I hope that we grow old enough to make Ed Sheeran’s words come true.

Happy Valentine’s Day!!

Fly Away

So I’ve been gone from this blog for a bit, since I was released the hospital. I just needed some time away from it all. CF encompasses so much of my daily life, especially leading up to and during any hospitalizations. When I get out, and I’m feeling more at my baseline, I try to not think about my illness for a while. I was hoping for a little more of a “vacation” from CF, but it wasn’t cooperating. It only lasted a week. I developed another cough and my day to day difficulties crept back in well before I was ready for them. Now, I’m back to increasing my therapies to reduce my cough, trying to prevent another downslide and keep myself out of the hospital. And this is my life, every freakin day of it. I’m just tired of the grind.

As my symptoms started to creep back in, I was trying to figure out how to express my frustrations and thoughts about wanting to escape CF. I was in my car driving, and this song came on, Fly Away by Lenny Kravitz. It’s a song about escaping the difficulties of the day, even for a while, and getting away with the people you love. While that is not steeped in reality for most, who cannot just charter a flight to a great getaway on a whim, it is more the thought that works for me. Trying to escape my CF, even for a little while in my mind, to help make the bad or frustrating days a little easier. I learned early on in life that even 10 minutes of meditation, sitting alone and focusing on nothing but breathing can make a big difference. Ironic, it seems, as breathing is the cause of most of my difficulties! Even though it is difficult to do, I feel a little more balanced when I’m able to. So….I’m listening to this song as I’m feeling really frustrated about my cough and symptoms. Not only is the song a great message, but Lenny in general is a great person of positivity with a great story to tell.

Let’s face it: Lenny Kravitz is really cool. He so cool, he’s a bad mutha……..Shut yo mouth! (Couldn’t help a shaft reference there). Kravitz grew up in New York to a white Jewish TV producer and African American actress mother, Roxie Roker (She was on the Jeffersons). Growing up in this world, he was exposed to more culture than most people his age. Immediately he was drawn to music, influenced heavily by funk, jazz, blues, etc. He started in the mid 80s with his debut Let Love Rule, but didn’t make a huge commercial mark until Are You Gonna Go My Way. He had long dreads and sung about love and peace. It didn’t hurt he married Lisa Bonet, who was super popular at the time as a star of The Cosby Show (when it was on top of the world well before everyone knew about Mr. Cosby). He was totally cool; guys wanted to be him and girls (I assume) wanted to be with him. By the time he came out with his 5th album, on which Fly Away was a track, his stardom had continued to rise. Yet he continued to be philanthropic, and to spread his message of peace and love, which he continues today.

Back to the story at hand. I think about trying to get away from things. Not that my life is bad, by any means. I feel extremely lucky and fortunate, having found the love of my life, Mari. We have built a great life for us and our son in Chicago and now in Denver, and I have made it to 40 years old. I have a wonderful extended family and great, supportive friends. I am a medical professional and a CF advocate. Most days I am happy to take all that on; but on other days I don’t want to be that person. I don’t want CF to rule my life, I don’t want to take care of others; I just want to go away with my family somewhere far away. To where I’m healthy and can run around and play without coughing fits. To where I don’t have to worry about getting a minor illness that could catapult me into the hospital before I know it. To where I can just be, living a regular existence without the constant roller coaster that is CF.

But I know that’s not reality, so I have to get it in small doses. Whether that’s not being involved in CF and advocacy for a few days to a week, or not being in constant communication to everyone about the status of my health, or even just taking a day where I don’t do anything! (Although even those days I have to do my treatments, so I can’t even escape then!! UGH!). Sometimes, we all need a break. To get away, or fly away in this case. Hopefully, the people around you will see and allow for this. Because CF or chronic illness or just life is hard, and we all need a break sometimes.

I promise not to take too many breaks from this blog, but if I do you will know why 🙂

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