SILVER BULLET x 2

It feels like forever since my first CF related post even though it's only been about three years.  I dunno anymore, is three years a lot?  A little?  Just enough?  I guess I've quieted some because we're still sailing far enough ahead of the brewing storm.  If Charlie and Lola are good, I'm good.  And lucky for us, they've been doing really, really well for a long time.  I was telling my amiga Susie that when it comes to CF, I tend to go in cycles.  I'm cool until about 2-3 weeks before our quarterly clinic visit and then I morph into Sybil.  I just bitch-out if that's even a term.  Every little thing sets me off.  Everything.

And it's all because I've got that leech called cystic fibrosis waiting to suck me dry at every turn.

Well, last week, something big happened.  I'm talking big as in The New York Times big.  It's taken me a full week to even get my brain around it let alone be able to rattle it off on the keyboard.  So here's the 411 after a week's worth of marination in the void that occupies the space once reserved for my brain:

There's a wonder drug out there called VX770.  I wrote about it once here but as quick as it posted to my feed I let go of it; let it fade back into white noise, just another prelude to a dream.  Well, last week this same wonder drug made front page of the Foundation's website.  Did you hear about the earthquake in Arkansas this week?  Yeah, well the press release for VX770 is like an 8.0 on my Richter scale.  I shit you not.  You see, this drug is now in Phase III, the final phase, of clinical trials.  You have no idea how tough it is to get a drug to this point.  I've often wondered if winning the lottery would be easier because for all the drugs that make it into the drug pipeline in the first place, it's very few that make it to market.  Very, very few.

So last week's news from the CFF that they were planning to apply for FDA approval of VX770 sent shockwaves through the CF community.  I was actually sitting in my first block class when I read the release.  At first I just sat there, staring at the monitor not believing what I was reading. 

"Patients who took the drug, compared to those on placebo, showed a marked improvement in lung function at 24 weeks, which was sustained for the duration of the 48-week trial."
WHAT?

"Patients also showed improvement across all key secondary endpoints in the study, including reduced likelihood of experiencing a pulmonary exacerbation, decreased respiratory symptoms and improved weight gain. Each of these areas is critically important to the health of people with CF."
NO FUCKING WAY!

"In addition, average sweat chloride levels of patients on VX-770 dropped toward normal levels, while those on placebo did not change — indicating the drug is impacting the underlying defect in CF. Excessive sweat chloride (salt) is a key clinical indicator of CF."
OH.  MY.  GOD.

I jumped up from my stool and clapped my hands together half a dozen times and just kept repeating, "YESSSS! YESSSSSSS!" over and over and over again. And then the tears came and I had to pace around the classroom fanning my eyes so my mascara wouldn't start running down my cheeks. My poor students, bearing witness to the freak who was their teacher.


But here's the catch. 

The VX770 will NOT help Charlie and Lola.  They don't carry the same mutations that this drug is targeted for.  It will instead help just 4% of the CF population.  What's 4% of 30,000?  Anyone got a calculator?  Like 1,200 people?  Hell, let's talk globally.  Let's take 4% of 70,000.  What's that make...2800 and thensome?  I know what you're thinking.  Yeah, she's totally lost it.  Crapping her pants for a drug that's gonna help 1,200 maybe close to 3,000 but not her own kids?  What a dreamer.  No, strike that.  What a moron.

Well hold on.  I'm not done. 

You see, there's another drug by the same company - the VX809 - and they're working on the theory that giving the VX809 along with the VX770 will help CFers who carry at least one copy of the most common CF mutation (the Delta F508).  In essence not one but two silver bullets.

Why hello there Mr.  VX770, I'd like to introduce you to my fucking amazing children, Charlie and Lola.  No, we didn't name them from that stupid cartoon from Disney Channel.  They're nothing at all like that cartoon.  They are two beautiful, wonderfully chatty, hilariously stubborn, lovey-dovey,in your face kids who happen to have CF.  And they also happen to have

a copy of that nasty Delta F508 gene that you're gonna fix.  Got it?  Good.  Oh and by the way, would you mind terribly passing this on to your pal, Mr. 809?  Thanks!

The VX809 is still in Phase II, so it's a little bit farther back in the drug pipeline but so far, things are looking good, very good.  Still, there's a catch:  getting a drug all the way through these trials not only takes good science but Megabucks.  I'm talking gazillions of dollars.  The US Government does not fund fund these clinical trials.  They are funded through donations.  The gala dinners, the WineOpeners, the charity golf outtings, the foot races, the walks...all the way down to the bake offs and can collecting.  In short, all of those fantastic donations.  That's what funds this research.

So I can't be all polite anymore and ask you to consider making a donation.  Now I'm knees to the floor begging you:  Help me Help the Foundation Help us.

And here's how:

1. Go HERE and click Join My Team.  This will register you as a walker on our family's team.
2. Once you register as a team member, you be given a login and password.  Please use it to make your own fundraising homepage 
3. Wanna really be a hero?  Go to www.CFF.org, click on the Great Strides logo and create your own  Charlie & Lola Team and recruit walkers under you.
4. Last but not least, if you haven't yet made a donation, please, please, please make one by clicking HERE.  Anything you can afford, I mean ANYTHING, would just mean the world to me, to us.  

Yesterday, as I was packing a box of dishes, Charlie came up and kneeled beside me.  "Mama, how much longer do I have to do treatments?"  God, what I wouldn't have given to have been able to tell him the answer he was fishing for.  It took everything I had not to snap that dinner plate in two, I swear.  When your child comes to you with hope in his eyes and you have to be the voice of reason, of right, of truth...well, it just so totally sucks.  

So until then, until the press release that makes me own up to No. 15 of my Bucket List, I just have to stick with, "...until we've got the cure, Charlie.  Until we've got the cure."

Kinked

Cystic Fibrosis.  Yeah, I can articulate the basics but when it comes down to the science of it all, I know just enough to be dangerous...very dangerous.  Lucky for me I have a husband who is really good at taking it all in, digesting it, and then spitting it back to me in laymen's terms which helps immensly when it comes to calming me down at the first sniffle of an oncoming cold or the whistling wheeze heard after a too long hug. It's ironic, really, that my husband the NON multitasker, the NON stress basket of the family, Mr. SoLaidBackYou'dBetterCheckMeForAPulse posesses such a skill.  Meanwhile I'm the one schlepping into Clinic with the checklist of questions written out as a laundry list of whatifs and ohbytheways a mile and a half long.

When you boil CF down to the nitty gritty, the bare bones...it's a disease of the cells.  There's a broken one in there.  Outta commission.  Out of Service.  On the fritz.  And man o man, does that jack things up!  Add to it those 1500+ genetic mutations of which you could get two - the whammies - and you'll give yourself a migraine for sure.  You see, it's just two genes - the gift or curse you get from your parents, on which everything rides.  Sick or not sick,  enzymes or no enzymes, lung transplant or not..the lottery that are those two genes can mean the difference bewteen CF or NO CF. 

My simple, unscientific mind - the one that sorts underwear by color and pants from fat to skinny has no patience for this disease.  We can put a man on the moon.  We can click enter and send a ten page document to Europe in less than a hearbeat.  We can sew a finger, a toe, a leg or an arm back on.  But we can't fix this?  Go on now, get in there, dig out that faulty gene and pop in a good one.  One that WORKS for Godsake.  Geesh!  This is why I'm not a scientist, I guess.  Screw The Scientific Method.  Just get to the answer already.  I mean Come On...

May 15th was our annual Great Strides Walk for a Cure.  Des Moines managed to pull in over $205,000 - it's most successful walk ever in spite of falling short of it's $225,000 goal.  I was pleased, yet miffed.  Okay, truth be known, yours truly had managed to work herself down to outright pissed by end of the day.  I was pissed that I was pissed...how is that even possible?  Well, for starters I was pissed about the fact that there were several family members who did not donate.  I'm talking CLOSE family members - people to whom I wouldn't think twice about giving a kidney or bone marrow.  Family members whose butts I once wiped.  Family members who call to chit chat on a weekly, sometimes daily basis.  They didn't even chip in a measly 4 quarters.  Come.  On.  Is the economy that bad?  Are they that out of touch?  My college roommates from nearly twenty years ago (20?!  Gulp.), the Facebook "friend" who's actually a complete and total stranger, my busy as a honey bee in June neighbor who I never talk to but see whizzing by in her SUV....they all donated. 

My attitude soured as I took a break from picking apart said family members and thought about the bigger picture.  Moolah.  Bucks.  Cash.  Benjamins.  Dough.  MONEY.  The catchy, Money Buys Science and Science Buys Life line that I pimped prewalk started to weigh heavily on me.  Des Moines had collected almost a quarter of a million dollars and there were how many other cities walking?  Houston, Tampa, Chicago, Seattle...millions upon millions were blowing around, whirling and swirling around and around all in the name of a cure.  Just how much money to you freakin' need to cure end this thing once and for all?  I've heard it takes a cool $800 million to get a new drug conceived, tested, approved and to market.  Are you kidding me?  Are we talking dollars or pesos?  EIGHT HUNDRED MILLION?!?!  For just ONE new drug?  What gives?  Are the cells charging a participation fee for each clinical trial?  Is the new company car for the scientists a Rolls Royce?  Is this whole beast just a conspiracy by the pharmaceutical companies so they can create more (profit making) drugs instead of a cure? Pop this gal a Vicodin quick before her head flips off!

The plane was in a huge nosedive, going down fast and I couldn't find the damned parachute.  Like always, I did what I normally do.  I yelled, no screamed, at my husband about the sink full of dirty coffee cups, slammed every door that I walked through, and then...then I splintered.

You see, I'm not a patient person.  No, not me.  Not at all.  I strum my fingers against the steering wheel, willing the car ahead to go just a little faster so I can make the light.  I open the microwave between 1 second remaining and zero just so I don't have to hear its annoying beep.  It's half impatience and half OCD.  But it's me.  So one would think I would know better than to cruise out to the CFF.ORG website post walk.  Like always, I lie to myself, telling myself that I'm going to look up walk results of friends and acquaintances.  But I'm coming clean - it's a lie.  It's always the same lie - a different excuse maybe but the same lie nonetheless.

Like a fool I'm a much too frequent visitor of http://www.cff.org/.  I'm a fool not because I visit so regularly but because I expect to see something other than the face of the middle aged physician who pledges, "I will keep working toward a cure."  Sorry Buckaroo, no can do.  Nope.  Not good enough.  Each and every time I see his face I let out a heavy sigh to give voice to my disappointment at the absence of what I have been waiting for since diagnosis: "We are Pleased to Announce That The Cure Has Been Found." How many times in fact have I seen that guy's face?  A hundred?  Five hundred?  A thousand?  I don't know.  The point is, it's still there.  His face.  His promise.  And I'm tired of waiting on promises.  I want a cure.  Did you hear me?  I WANT A CURE NOW. 

So pardon me if the apathy of a few has wet my feathers.  I know life is too short to wallow.  I just had to purge it.  I'm human afterall.  My feelings get hurt, I whine a little and then I move on, past all the bullshit and onto the next line item.

The kink in my line is fixed and I can go back to life as I know it...crayola on my leather couches, fundraising for a cure, dog hair on my kitchen floor, a husband who snores the whole night through, more fundraising, students who forget homework that was never done and don't let me forget, more fundraising for a cure...you know the drill. 

Oh, and by the way, thanks for the purge.