NACFC in Phoenix

I had the opportunity to attend the North American Cystic Fibrosis Conference (NACFC) in Phoenix in October 2023 with the ACH staff. I am the parent representative for a research study being done at ACH on the use of acid reflux medication. Working with the ACH staff has been great and they have always made me feel incredibly welcome so when I was invited to go with them, I jumped at the chance. 

Let me say first off that I do not have a medical background and all that I am about the discuss I learned at the multiple sessions that I attended.  If you want more detailed information on something I can direct you to the presenter.

CF can be a pretty isolating disease because we cannot interact with other CF families due to the risk of sharing CF germs. I’ve gotten to know some of the parents of other children with CF and they’ve all been great, but it’s still sad that we can’t do life together. Each day of the conference, there was a plenary session where all of us came together into the same room to hear a presentation. It was amazing to be in a room with over 4000 people that were all there to support CF and just see how many people care and are working towards making this disease better. As a caregiver of someone with CF, you’re only real interactions are when you go to clinic so it was really neat to see kind of behind the curtain at the next advances and treatments that are coming down the pipeline.  Some of the questions/things we have wondered about our son’s CF were verified / confirmed during some of the sessions I attended.

For a long time CF was seen as a child’s disease because many individuals did not reach adulthood.  Thankfully that is not the case anymore.  With that some major changes are being seen.  Lifestyle related behavior is becoming increasingly important in CF care.  Things like exercise and nutrition are being looked at differently.   

Such as how certain medications affect their appetite which then affect their weight and growth and the pros and cons to both.  Our son was on inhaled Tobramycin for 4 years due to culturing psuedomonas.  He would inhale it via a nebulizer every other month.  He would lose weight every month he was on it because it affected his appetite.  For a long time high calorie high fat diets were pushed to get them to gain weight due to their low absorption rate.  Now that individuals with CF are living longer they are shifting that to a healthier diet but also trying to maintain the nutrients to gain weight.  One thing that really stuck out to me during one of the sessions on nutrition was when a dietitian said “if you had to swallow gravel before you ate every meal it would probably affect your appetite too” in reference to small children swallowing enzymes.  She also mentioned how they are trying to make the enzymes smaller and less of them so they do not fill up on pills & applesause.

The use of the vest, which is mainly an American treatment is being looked at to see if an individual can replace that with running or another high cardio workout instead.  In a few studies individuals said they would love to exercise but that they do not have time to do it.  They said they would much rather go for a 30 min run outdoors than be hooked to their vest for 30 mins indoors.  The data is still relatively new but they hope that with the introduction of the CF modulators people with CF may be able to trade their vest for exercise.  This is talking about when they are healthy.  If they are sick the vest may be needed to prevent / break up the mucus in their lungs.  The researchers also found that mental health improved for participants that were able to exercise vs just doing the vest.

Mental health was a big topic at multiple sessions I attended.  While the modulators have helped dramatically with the physical component of CF they are causing some disturbances with the brain.  Some people with CF are experiencing depression and other mental health concerns.  They are finding that in children the behavior is being expressed as anger, which we have seen a little bit with our son.  They are experimenting with the current dosing to see how they can tweak it to help.   One way is they had a few participants switch their doses so they hit their low point in the middle of the night when they were sleeping as opposed to the middle of their work day.

Another session I attended looked at the rate of severe exacerbations / hospitalizations has decreases significantly since 2020.  Some of that is due to everyone being home due to covid but they also think it is because of the CF modulators that many people were able to start taking.  The introduction of the modulators has been incredible but has made it harder to collect cultures at patients visits.  Basically they have to cough up phlegm and that is tested to see if anything is growing in their lungs.  The negative cultures seem like a great thing except there is some worry that bacteria are still growing and hiding in pockets of the lungs, the patient is just not coughing it up for it to test positive.

The CF community has been asking for other treatments besides the use of antibiotics since many of these bacteria have become resistant to antibiotics so the foundation has been conducting studies on it.  During the second plenary they discussed this in detail.  They are trying to tailor the therapy for each patient and bacteria instead of immediately using antibiotics.  A few of the ways they are doing that is with phage therapy - viruses grown in a lab that are matched with the specific bacteria a person is growing with the hope that the phage will overtake the bacteria.  There are many other drug developments in the pipeline which is so encouraging.  If you want more detailed information on those I can get you that material.        

The big questions CFF is asking now are:

How will CF infections change in the future

How should we think about infections in people who are taking Modulators? And those that are not?

How can we use antibiotics most intelligently in either case?

And Are there effective new antimicrobials with less toxicity or side effects?

I was exhausted at the end of each day I think not only from all of the information, but also, I think it had an emotional component to it of just everything I attended, was talking about my son in someway. I would say to any parent or caregiver that ever has the opportunity to attend I would go , I left feeling very hopeful and knowledgeable about what the future holds in the sense of belonging and family was amazing

I want to thank each of you for supporting CF and helping to raise money and awareness of this genetic disease.  With your help the CF Foundation was able to raise $225.2 Million dollars in 2022.  The majority of that went to Treatment of CF complications, Clinical research support and Genetic Therapies and treatment of nonsense / rare mutations & CF modulators like Trikafta. As a parent of child with CF you support means more than you know.