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."

THIRTY SEVEN

THIRTY SEVEN.

That number just blows me away.  Has it really taken thirty seven years for me to love my body?  To admit that I'm no longer a natural blonde and probably never was?  To own up to the fact that a size 10 is my version of 'petite'?  Simply stated, it's taken me thirty seven years to accept myself for who I am instead of worrying about who I am not. 

In less than six months I'm going to be turning 38 years old; dangerously close to 40 though a milestone all the same.  It dawned on me the other day that this, my 37th year, is especially poignant in light of the fact that 37 is actually the most recent the life expectancy released by the Cystic Fibrosis Foundation.  I'm no stranger to this statistic, in fact it was one of the first things I Googled when we got Lola's diagnosis: life expectancy and CF.  Where the answer to my search used to make tears well up in my eyes, I'm pleased to report that this is no longer the case.

I've accepted it.  It's there.  It is what it is.
And I've got roughly 32 more years to change it.
Period.

Five years ago, the same year that Charlie was born and long before we even knew he had CF, a pharmaceutical company out east by the name of Vertex submitted to clinical trials this little ole' drug by the name of VX-770.  Shortly thereafter, it submitted a second drug, VX-809, to clinical trials.  The aim of both drugs was to target the basic defect of cystic fibrosis at the cellular level.  Fix the cells, fix the problem.    Evidently, there's a bit more to it than my peas and carrots brain can articulate but that's the nitty gritty.  For those of you who want it spelled out, you can click here for a mini science lesson that does a pretty good job of explaining things if I do say so myself.

As I turn the corner on January and sail into these last six months as a thirty seven year old, I am looking at new beginnings. My glass is full up to the top and spilling over.   I am as hopeful as ever that VX-770 will gain final approval by the FDA and that VX-809 will continue to show promising results in its testing so that it too can move on down and out of the drug pipeline.  I often think about what it will mean not only for those battling CF, but for anyone battling a monogenetic disorder.  Getting a drug or combination of drugs that can punch a disease - can knock it out - at the cellular level is huge.  HUGE.  Could this pending breakthrough by Vertex eventually impact those fighting sickle cell anemia?  Huntington's Disease?  Hemochromotosis?  I can't get my head around it - my heart yes, but not my head.  Not yet.

So on May 21, 2011, five pairs of running shoes will be laced up in Des Moines.  We are participating in Great Strides, the biggest and most important fundraiser for the Cystic Fibrosis Foundation.  I would like to invite you to do the same, to walk a three mile stretch with us on that day, wherever you are.  Donations made to this event go towards funding the research so crucial to finding a control for CF.  Of you I ask not one, not two, but three simple things:

1.  WALK WITH US
     Wherever you are on May 21, 2011, lace up and walk those 3 miles with us.

2.  MAKE A DONATION
     $10, $20 or $200 - every single little red penny will fill the bucket.  Really, it will!

3.  SPREAD THE WORD
    Pass the link to my blog to your Facebook friends.
    Forward this link in an email to everyone you know
    Talk about CF to your colleagues at work, to your friends at church, to your buddy at the gym, to your cashier at the grocery store - tell them about the crazy picklepits lady who can't catch a break from her Fabio, who wrestled a pair of skinny jeans (and lost), who swears she's part Gitana and who would walk to the ends of the earth if it meant a cure for CF...tell 'em all...I don't care...
JUST SPREAD THE WORD!

2011 is here. 
2011 is now.
2011 is it.

Now let's get out there together & grab it!

A Baracuda's Fight

I've never been one to test the waters with a big toe first.  No, not me.  I just plunge in, usually head first too.  Impulsive, yeah that's the word for it.  Impulsive.  Yeah, that's me.

And that's what happened last week.  I was in such a state.  A state of frenzy.  A state of panic.  A state of desperation.  Such a state that I didn't think it through.  No proofreading.  No editing.  Nope, not last week.  I just threw it up there front and center.  The chipin link to Conner's Fund was a hot potato in my hand.   I had to pass it off FAST and so I copiedcutpastedandposted as fast as I could get my little mouse to click.  I impressed myself with my technological skills, I really did.  But in reflection, I'm thinking my haste hindered more than it helped.

I gave you a bun with no burger.  No meat.  Where's the beef, Kelly?  Huh??

What I should have done I'm doing now.  As I have the tendency to get long winded, I've got to keep this to a soundbite.  And here it is:

My friend needs help.  Now.

She has been waiting for seven years for a cure.  But it hasn't come.
She has searched everywhere for alternatives.  But she has not found one that worked.
She has turned her back on death.  But it has found her little boy.

My friend's little boy, her Boy Wonder, is dying.  Conner is dying.  The doctors have given up.  But my friend has not.  She is fighting like a baracuda.  Fighting with every ounce of her being.  She is a ferocious lioness thrashing about to save her cub. 

But he is still dying.

I feel desperation.  I feel grief.  I feel so incredibly helpless.  As a mother I cannot even imagine waking up to her nightmare.  As a CF mom, I know the possibility looms. 

So before my soundbite ends, I implore upon you to jump in.  Jump in with both feet.  Make a huge splash - much like that of this baracuda of a momma.  Make a donation to Conner's account.  All monies collected are going towards his funeral expenses.  Yes, you heard me.  Somewhere way out west, on top of the USA, a mother is guiding her baby to the heavens.  She has written about this journey, this nightmare, on her blog.  Go, see for yourselves.  It's real.  She's real.  Conner's real.  And then pass this along.  Email it, Facebook it, talk about it...but reach out to another so the word will spread.

Because they need our help.  Sarah and her family need our support, our compassion, our prayers.  Now.

Whether it's 5, 10, or 50 bucks it would help immensely.  Please, please, whatever you can do, do. 

And then pray.  Again.

xo
k.