Friday, April 16, 2010

PTC124, my thoughts.

So here we are on the verge of a potential cure for CF. Its the thing that I've been hearing is 10 years away for my entire life. And I don't know how to feel about it. Assuming this works would I want it? Now, before half of you out there in blogger land read that and flip out let me explain.

I can't seem to decide how I feel about remaining exactly where I am for the rest of my life. If this works I'm looking at potentially 50 years of THIS. By this I mean mid-50s lung function. Sure I could regain some function back without having mucus plugging up my airways but the damage that has been done isn't going anywhere. Do I want another 50 years of struggling up a flight of stairs, panting while doing laundry, and walking a step behind everyone because I just can't keep up. I might complain about my health but I've never been angry about it because there was always the option of a transplant somewhere on my horizon. I would think "I might feel like crap now, but someday I'll have new lungs and it will be worth it." Would I like to live another 50 or so years? Sure. But do I want to live another 50 years feeling like crap? I'm not so sure that I do.

I'm doing this study for all the CF kids who might benefit from it. For the ones that haven't been born yet. This drug might not do a thing, but if it does work there are some kids somewhere down the line that will never have to ask themselves if they want to live the rest of their lives with half their lung function. They'll just live, and CF won't take away their dreams, or their lives.


tdraxler said...


I found your blog while CF-blog hopping today, but I remember you from and cf2chat, and from just being around.

I just wanted to say thank you for doing the study. My Emily (age 4) is one of the kids who stands to benefit from PTC124. I'm grateful to everyone who is working to make it a possibility.

Have a good weekend.

(izemmom on

Chelsea said...

Aww Jess. I don't think PTC 124 "freezes" thinks. from what I read, it just acts as a set of instructions for the CFTR protein to finish salt production, & that's it. I know you can work to regain your lung function (without worrying about other CF stuff holding you back.)

However, I do understand where you are coming from. You're kind of stuck in limbo. - You're lungs are too sick for you to be comfortable; but they aren't sick enough to allow you to be a candidate for transplant.

Remember that this drug will only help the CFTR protein do it's job, it won't preserve your lungs. You will still get sick like a normal person, and your lungs will still get a little more tired each time.

It could really go either way. You could work your butt off & lung function could increase; or the world can make you sick a lot, & you could end up needing new lungs by force anyway.

No matter what though, remember that I'm still terribly new to this. I've researched a ton on PTC 124, but I could still easily be wrong. I'm just giving you some food for thought :)

Devorah Yuda said...

My two daughters are in the PTC124 study. They are both improved in many ways. But beyond that, everyone should remember that noone knows what this drug is capable of, what your body is capable of on this drug down the line, 1 year, 5 years, 10 years. I'm a Lubavitcher Jew and as we like to say, "Think good, and it will be good."

Anonymous said...

Hi. My son, Joey (age 7), has the stop mutation. From the bottom of our hearts, we thank you for being in the study. And, my prayer is that you will benefit from it too! You are a warrior- stay in the fight!!