NACFC 2018 - My Reflection on the excitement of CF Modulators


NACFC didn't feel quite so overwhelming to me this year since this was my third year to attend. I was more familiar with the format, the expectations, the pace of sessions.

But, there was a moment that took my breath away when I least expected it to.  

It was during Plenary 2.  I was sitting in the middle of literally the hugest auditorium I've ever been in.  I was listening to the newest information about CF research.  A picture of a little baby came up on the screen (see what I'm talking about here at the 53:35 mark).  NACFC Speaker Dr. Felix Ratjen had spent the last hour explaining all the success we've seen with the newest CFTR modulator medications and wondered outloud, "how about if we start [these meds] early on in babies, in patients, before the lungs are damaged?""

What if we give newborn babies, still yet free of CF damage, the newest CF drugs available?

It was a hopeful statement.

Suddenly, I had this flash of memory to the previous plenary, Plenary 1, held the night before, when speaker Dr. Lisa Saiman shared other new clinical information in CF by juxtaposing where we were in CF research the decade before.

"Ten years ago..." Dr. Saiman would end up saying over and over again as she explained how much CF meds have changed in a decade.  As Dr. Saiman flashed back to the decade prior, I couldn't help but do so in my own life.

It was almost 10 years ago that Bennett was born (he just turned 9).  At the time, Brian and I, new CF parents, eagerly listened to and read the newest CF research with expectant hope.  At the time, we felt very encouraged by the newest research and genuinely hoped that the newest meds available 10 years ago would prevent CF from damaging his body. And yet, back in the present, it's hard to believe that research is 10 years ago old and no longer relevant. 

Pulmozyme, a medicine that we use routinely nightly in our house (and have for years), had only barely been out of trials and become FDA approved when Bennett was born.  It felt "new" and "exciting" and now, it's commonplace and part of the standard CF protocol.

I looked up again at that picture of the cute newborn baby up on the screen and re-heard the words "how about if we start [these meds] early on in babies, in patients, before the lungs are damaged?"

All of the sudden, that idea no longer gave me hope.  It brought me grief.

"We're losing years!" I felt some deep place in my heart call out. "We've already lost a decade!"

No longer is my family in that group of CF families who are"newly diagnosed."  Our family isn't even in that group of families with "young children with CF" anymore.  

We're in a new place, a wonderfully joyful place for most parents: a place with a tween!  

But, when it comes to CF, time often passes faster than research can answer questions and faster than medicines can be approved.  So, while birthdays in CF are a really big deal because they are a huge gift that we celebrate, they also mark time that has passed without a cure.  

Bennett is in a really good place, considering.  He has had very good lung health compared to many children with CF.  But we know silent lung damage is happening and we know that, statistically, he's losing 2% of his lung capacity per year.  

More drugs are coming.  It's a very exciting time in cystic fibrosis.  But it's a hard pill to swallow that we've also lost nearly 10 years of potential benefit for those drugs.  

Sitting in the auditorium, surrounded by a sea of professionals quietly listening to the newest information on CF, I felt time compress.

New meds are coming.  
But we won't be the lucky ones with newborns.  
We won't be the lucky ones with young children.  
Fingers crossed, in another few years, we'll be the lucky ones with an older child.  

But I want those early years back.  I want to be the one with the newborn who benefits.  And I want to stop the clock until we have more tools in our arsenal.

Might this be what it feels like for those who have gone before me - parents of adults with cystic fibrosis - whose time compression is marked by many more FDA approvals of CF medicines...or, worse, are marked by the time when there were no new CF medicines at all? How difficult it must be for them to hear of the excitement in the CF community and yet to know their child, whom they've had to say goodbye, will not benefit?

My heart breaks for them.  And it breaks for us, all of those with CF still suffering from this awful life-threatening disease.

Can I be sad for time lost, even as I have hope for the future?  As a mother, I can hardly help but wanting Bennett to have never suffered.

This is a very very good time for the CF community.
But, as good as it is, this NACFC just reminded me: 

The clock is still ticking...