The future is something no one can predict. But I can tell you that based on the past and what was shared at the Volunteer Leadership Conference this past weekend, the future of CF is going to continue to change. Change can sometimes be good and bad, but in this instance, things are looking so positive! Here is a quick update to share the latest and the greatest from the CF Foundation from this past weekend. I’ll share the updates in the order of the event, which was held in Denver, CO. My first time in the state and the view from the hotel didn’t disappoint.
The conference started off with CEO Dr Michael Boyle welcoming everyone to the 19th conference and sharing a stat right away hot off of the 2022 research. The average age for an individual with CF is now 56 years old! 56! It was only a few years ago that the average age was 40. This in itself is incredible!
From there we had an opportunity to listen to the advocacy team, Mary and Jeremy, discuss how March on the Hill went, how easy it is to advocate, what we are advocating for and what the future for CF looks like on Capitol Hill. I feel confident in the CF advocacy team that they will continue to be the squeaky wheel on things that make sense for CF’ers and everyone else in the general population.
From there we were able to head to the Idea Lab break out sessions., where I was asked to be on a panel of individuals to talk about advocating, what that means to me, our family, how I advocate and tell Maddox’s story. It was very cool to be on a panel of 4 people from across the country, learn the different ways we are tied to CF and what we do when we advocate. Envision the room was full of people, because it was! 🙂
From there, we had another opportunity for break out session dedicated to Fundraising Ideas. The table I was at ranged from Penny wars within the CF’ers middle schools, to events at restaurants and complete obstacle courses. The individuals at my table were also CF Foundation staff so they were able to help provide insight on what the foundation can provide to help make passion fundraisers easier.
We then came back together to have 3 Adult CF’ers share what they have witnessed and lived through as an adult with CF. Next, we had a yummy dinner and a party to celebrate.
The next morning is what I enjoyed the most, the medical update from the team who is behind all the complex work that we are all striving and working towards. The Cure. I can say, as of 2023, there is not a true cure for all CF’ers. Right now about 90-94% of the CF population are on some type of modulator. This graphic explains this better than I could ever try to describe the science as it dives in the levels of CF, literally.
Ok, let me try to explain how this graph relates back to Maddox. He has two CF genes: one is the CF gene (DEL508) and with that gene type, he is eligible for modulators, which includes Trikafta. The other modulators (Kalydeco and Orakambi) were only for CF’ers that basically had both genes of DEL508. The other gene type that he has is not eligible for any modulators. The way to ‘fix’ the other gene is through mRNA or Gene editing/replacement. …with me so far?….
So, with him being on Trikafta, it means that it’s helping some of his aspects of his CF diagnosis but not all of it. Which is making perfect sense that he is struggling/still dealing with some CF issues. He is still pancreatic deficient, meaning he still needs his enzymes otherwise he has the worst stomach issues (just like he had before he was on Trikafta). When looking at the rest of him, he is doing great – his spirometry test results are showing he has very strong lung function. He is even gaining weight on his new enzymes. Tammy actually smiled at our last CF appointment in March!
The CF Foundation medical team basically stated they have an idea of what needs to be done, but the delivery of these therapies is the tough part as they need to find the vehicle to get it to the specific area without it being disrupted in transport. Don’t forget, CF’ers have very, thick, sticky mucus that these would need to travel through to fix of not having thick, sticky mucus. And they are working on the best way to get it to the genes, is it a pill or inhaled? Lung specific or systematic?
Essentially, if they can figure out the mRNA, Gene therapy/editing for the remaining 6-10% of CF’ers then almost all CF patients will have the medical advances they need. Sounds so simple when it’s put this way, right? The hopeful part is that they have a plan and they have a strong funding team to support the medical ideas and research.
The medical team shared their pipeline of different things that are already in the works and timelines they are targeting for 2023. Some other interesting graphs showing the number of lunch transplants in CF patients showing the drastic drop in 2020. One other area they are working on are therapies to help eradicate bacteria in CF’ers lungs.
A note of interest I wrote down throughout the presentation: the CF Foundation is one who actually has an ROI and is measurable when looking at the research provided to the drug companies and to the FDA and that the CF is the greatest story in medical research.
The goal in the next five years is that in CF meetings and conferences, the topic of life expectancies won’t be talked about anymore as they will be matching the rest of the general population. There are now 40,000 individuals that have CF and the CF Foundation is working to bring an entire medical group care center to all 40,000 individuals. Thankfully Maddox has had this team approach since his first meeting with the Green Bay Team.
Mental health will now be a strong focus for the CF community, especially having the trust in medical providers. All their lives, the CF Medical team has talked on vest treatments, enzymes and nebulizers to keep everything healthy. When on the modulators, being told, you don’t need to do your treatments any more, is scary as there needs to be a trust that they will not regress in their health. However, there are CF’ers that are already in trials/studies to pull back the amount of therapies that they are doing and are being closely watched/monitored. The early results are showing positive things for these individuals.
There are CF’ers that have been told their whole life to eat as much as you can to pack in the calories that are now transitioning to overweight/obese and need to reprogram how to eat healthier.
We were also told at the conference that CF is no longer an automatic eligibility for Make a Wish, which is a great thing that CF medical advances are being recognized outside of the CF family.
As a parting note for the conference, I was able to meet a ton of other individuals from all over the nation that are on the CF journey. Especially the CF Mamas I met at March on the Hill in 2020 before COVID shut everything down. It’s so refreshing to be able to share the same insurance battles, learning more tricks of the trade as a parent, advocate and fundraiser all while being mom. Not to mention we knew all the songs they were playing at the party Friday night!
Just like any conference, I walked away with a ton of notes, memories and a heart full of hope and a renewed sense why we are advocating, fundraising, participating in studies, and networking within the community. We are all working towards the ultimate goal – the Cure for CF.