Tuesday, November 20, 2012

Almost a Cure

I've had to explain what cystic fibrosis is to a lot of friends, family, and strangers over the years.  Since Brady started taking Kalydeco, I've found myself struggling with my spiel (a great problem to have!).  Let's examine the facts:

1) Brady still has cystic fibrosis.  He still has the genetic mutations he was born with (G551D, DF508). 

2) He still takes pancreatic enzymes, special vitamins, and several other prescription meds as a result of being born with CF.  His pancreas has suffered "permanent" damage that Kalydeco hasn't corrected.

3) According to the gold standard diagnostic test for CF--the sweat test, Brady no longer diagnoses positive for cystic fibrosis (last test was 17 mmol/L, 17!!!  Well within the normal range). 

4) Kalydeco has corrected the basic defect of cystic fibrosis in Brady's body (dysfunctional CFTR has now become functional).  Functional CFTR leads to thinner mucus, improved mucociliary clearance, and restoration of proper pH in the lungs and digestive tract in the body (CFers typically have an overly acidic environment in their lungs and digestive tract, promoting infection and impairing digestion.  Kalydeco restores the body's correct pH, giving the body innate bacterial killing power.).

5) In CT scan, Brady's lungs currently show "No visible damage" and appear to be functioning completely normally.

6) Today, Brady has absolutely no lung or sinus symptoms of CF :)

7) Kalydeco is anticipated to add decades and immeasurable quality to Brady's life.

8) If Brady stops taking Kalydeco, the symptoms of cystic fibrosis will return almost immediately.

So what does that all mean?  It means my explanation of Brady's condition just got a lot more complicated...I have caught myself using the following phrase recently because I don't know what else to say:
"It is almost like a cure for him." 
I hesitate to include that word at all--cure--because I am really not sure what that means, and because that word holds so much power.  I know that unless you are a part of the CF world, or perhaps have seen a news piece about Kalydeco, most people won't understand why Kalydeco is such a big deal for us.  They won't understand that it has changed everything we do and think about every minute of the day.  I mean, it isn't a real cure, because Brady still has CF and has to take that magic blue pill twice a day or he is back to old-fashioned CF...BUT, as long as he is taking Kalydeco, his body doesn't realize it has CF.  Almost a cure is good enough for me. 
With all this in mind, my husband, Brady's Dr., and I have some decisions to make about how to move forward with treatment.  Brady is currently still doing once daily Pulmozyme and 15 minutes twice daily on the Vest.  We have been very slow and deliberate in removing treatments from his regimen because his safety is ALWAYS our #1 priority.  We don't want to remove any treatment that could potentially still be beneficial to him. Theoretically, and from everything we have clinically observed in Brady--he shouldn't really need ANY lung treatments or lung clearance anymore.  Not even the experts at the NACFC seemed to have a real answer for us on the best path forward with treatment...the ball is in our court.  
The Scenario:
We are planning a big family trip to Spain over the holidays and I have had a goal in my mind of being able to make that trip without Brady's Vest machine. We have been training Brady on the Acapella device as a more travel friendly option for airway clearance, and would still be able to do manual chest PT in the event that he caught a bad virus and was in need of a good beating ;)  We are taking a portable nebulizer and will be continuing his Pulmozyme treatments as usual.  I am anxious to discuss this again with Brady's Dr. at his next appt. (end of Nov.), and see what he thinks!  Feel free to comment your two cents on the topic!  Take the Vest...or not?
The full-length Plenary Sessions from this year's NACFC are now available online and I encourage everyone to watch!   https://wiww.nacfconference.org/plen.archive.html