What Are Your Remaining Medical Needs Post-Trikafta?

With the calendar turning to October this week (thank God 2020 is coming to an end), we will be celebrating one year of Trikafta’s availability in the United States. It goes without saying that a lot has happened since then – the most significant thing to celebrate is the CF community’s seemingly overnight transformation.

Not long after Trikafta received FDA approval, though, I wrote a blog entitled “Adults with Cystic Fibrosis Cannot Be Forgotten Amidst Trikafta’s Success” where I argued that we are on the precipice of several different and distinct CF populations going down respective life courses. There are, of course, the ~10% of the CF population cannot use CFTR modulators based on genotype. Beyond that, though, there are folks who are post-transplant and are thus dealing with very different disease manifestations than the classic CF symptoms, there are the adults who have lived through a lifetime of harsh disease progression before finally gaining access to a disease modifying drug (I’d put my self in that group) and then finally there is going to be a rapidly growing segment of people with CF who have not yet faced rapid progression before starting a disease modifier. That last group includes children irrespective of their genotype that have been granted access to CFTR modulators and adults who have lived with very mild cystic fibrosis.

Since I wrote that blog, many people with CF and their families have reached out to me about their shared concern. What happens when the healthy CF population begins to outnumber the older, sicker population?

It’s an interesting dichotomy, and one that I have not been able to find any evidence of elsewhere in medicine (if you know of another patient population that has faced a similar juxtaposition, by all means let me know!). My thoughts on this were resurfaced last week when we all received the tremendous news that Kalydeco received approval for infants 4-6 months old. I then tweeted the following:

This has to be the closest thing to a curative therapy going in CF. Starting on such an effective CFTR modulator at that age should delay respiratory disease progression for a long, long time.

If there is any downside in this, it’s that the rapidly growing “healthy” CF population will start to outweigh the older, sicker CF population. I fear investment into symptom management for those of us with advanced disease could begin to wain as returns look less attractive [with a shrinking] X.

My thoughts embedded in that tweet are a mix of some mathematical forecasting, hope that introducing CFTR modulators at a young age will be the closest thing to a livable cure and some health economics. If there is interest, I am happy to do a deeper dive in to that tweet in a follow-on blog, but for now that is all probably outside the scope of what I hope to achieve here. To be clear, starting infants on Kalydeco (and hopefully soon some of the other Mods) is the goal we have all be striving for. This advance is a tremendous success and one we all should celebrate. I still get chills thinking about how the CF I grew up with is quickly becoming something lost in the past.

My concern is for those of us who still deal with any degree of CF symptoms. Symptom managers aren’t exactly blockbuster drugs like modifiers or curative therapies. What is going to be done for the older population, and those who can’t use modulators, until our next breakthrough? A cure is a very complex ask in the multisystem disorder that is CF. The heartbreaker is that there is some growing evidence that investors are growing bearish on CF symptom managers. Again, I will dive into that a little more in a follow-on blog if we get interest.

So, I want to know what those symptoms are. What are our remaining unmet needs?

For me, the severity of my CF has all but gone away. That said I still have some day to day annoyance, not to mention decades of fibrosis in my lungs. I still cough some mucus up with morning treatments. I have occasional hemoptysis, but nothing like I used to have. I definitely have inflammation problems. Survivor’s guilt is an issue that hasn’t gone away, and, frankly, I am not sure it ever will. I have procedural anxiety after decades of traumatic medical experiences. I also have some GI issues [read: some days I poop a lot]. My biggest issue, though, is the multidrug resistant pseudomonas that calls my lungs home.

Time for some free response! Let me know what you think in the below SUPER BRIEF survey. Click the link below.

CF Symptoms Survey