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."
"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.
- Go HERE and click Join My Team. This will register you as a walker on our family's team.
- Once you register as a team member, you be given a login and password. Please use it to make your own fundraising homepage
- 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.
- 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.
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."