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This is BIG.

Wednesday, February 23, 2011

I read it first thing this morning.  Like every morning, I rolled over in bed a grabbed my cell phone and clicked the "news" app on my smartphone. With one eye opened, I read today's news headlines.

I always get excited to see the words "Cystic Fibrosis" in the news. But I was particularly excited when I read a New York Times article announcing big news for those with CF.

My heart skipped a beat. I could feel excitement bubble up within me. I don't usually get excited about new CF medications.  I don't want to put my hope in a "cure".  But this article seemed to indicate this is something to get excited about.

About mid-morning, I received a mass email from Robert Beall, CEO and President of the Cystic Fibrosis Foundation. I've never received a mass email from Robert Beall...especially one using words like, "great news" and "exciting results."

And then, this afternoon, I received an email from a sweet lady from my local CFF who invited me to an exclusive phone call with CFF "key contributors and other Foundation leaders" regarding the Vertex trial (how the heck I got invited to such a call, I don't know...).

So, I gave myself permission to get excited.  VX-770, a medication that has been in trials for some time now has been shown to work!  This may be one of the first steps to a real significant medication to overcome the affects of Cystic Fibrosis in the body.

This medication won't help Bennett in its current form.  Even if it did, it would not cure him.  But for one of the first times, the success of this medication in clinical trials offers decent hope of effective medications to come within Bennett's lifetime.

Here are the basics of why this is exciting...

A twice-a-day medication, called VX-770, in trials showed that patients' lung function improved, they gained weight, experienced fewer respiratory symptoms and felt substantially better.1  It is now being sent for FDA approval. 


VX-770 affects the underlying cause of Cystic Fibrosis.  So, it's success gives us hope that it can be modified so that all gene mutations can benefit.  However, right now, the drug, in its current form, affects only 4% of those with CF.  This is because VX-770 corrects only one CF gene mutation (there are over 1000 gene mutations). 

VX-770 will not likely directly affect Bennett since he does not have the appropriate gene mutation.  (However, the same pharmaceutical company that makes VX-770, Vertex is working on a medication for the most common CF gene, which Bennett does have.  Again, it will not be a cure...but it may significantly counteract Bennett's progressive lung issues when and if its placed on the market.)

During my conference call, in which I merely listened in while Dr. Bob Beall (CEO/President of the CFF), Rich Mattingly (EVP Medical Affairs of the CFF) and Dr. Preston Campbell (EVP Medical Affairs of the CFF) reviewed the findings of the VX-770 trial, I learned several great pieces of information. 
Dr. Beall referred to today as being "highest up there" compared to the day in 1989 in which the CF gene mutation was discovered (a significant finding for the science community as well as the CF community).  The CF gene was apparently the first of the first three ever discovered.

The VX-770 trial lasted 48 weeks (nearly a year) and found that there was a 10% sustained lung function improvement.  They said it was by far more than what they have seen with any other drug on the market including Pulmozyme (6-8% improvement), TOBI (6-8% improvement) and Cayston (3% improvement).  Dr. Mattingly explained that this was like "turning back the clock" 5-10 years!  Imagine having progressive lung disease but being able to take a pill and go back to the way your lungs were working 10 years ago?!  That is what kind of improvement they see with this drug.

This drug also showed, on average, a 7 pound weight gain.  That's a pretty significant weight gain for a CFer in 48 weeks.  I can't imagine how wonderful that would be if Bennett could gain 7 lbs in a year!  Weight gain and lung function are correlated as is life expectancy. 

Dr. Beall said at 8:05am Eastern today these results were sent Bill Gates to be placed on his desk.  Apparently, Gates was one of the big-time investors of this drug.  Kinda cool to think Bill Gates cares about this news.  Apparently, so do many in the stock market.  Vertex Pharmaceutical company's stock went up significantly shortly after today's news.

In response to this, someone on the conference call asked if people would likely give less to the Cystic Fibrosis Foundation because now our need is less hopeless.  Dr. Beall said that likely the opposite is true.  He said that he believes investors will see what significant things the Cystic Fibrosis Foundation is doing with the money they are being given and people will actually choose to give more.

There was a question about why the G551D mutation was the one that researchers studied.  Dr. Beall explained that it was the easiest mutation to potentially fix.  He called it "the low lying fruit" for researchers in the way of fixing a Cystic Fibrosis gene mutation.  However, Dr. Mattingly reminded those on the call not to be discouraged.  He explained that today's news is significant for everyone with CF because now that we know VX-770 works, scientists can begin to modify the therapy to work with other gene mutations.

Dr. Beall kept calling today's results a "home run."  But he did say "it's like we're in the 7th game of the World Series.  We hit a home run.  But we still have alot left to play."   I thought this was a great analogy.  It is wonderful to gain a sense of perspective of how big today's news is in comparison to previous and future news.  It is a big deal.  But we are in a long long fight.

Nonetheless, the thing I heard most from the men on the conference call - men who are incredibly passionate about what they do and who are acutely aware of the consequences of their work - was their unabashed excitement and genuine thrill over today's findings.  It was obvious that while this news isn't going to cure anyone, it is something to be celebrated.

My favorite statement from the call came at the end when Dr. Mattingly ended our call with this sentence...
 "This is an amazingly good day for all of those with Cystic Fibrosis."

It's exciting to be a part of something big...especially a "big" that may eventually extend my son's life.
Awesome.



To read more about VX-770, click here.

5 Responses to “This is BIG.”

  1. How cool is it that you were ale to be part of that phone call!!! VERY NEAT!

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  2. It worked, I have been trying for days.=)

    I wrote a blog about the press release last night due to the hope that it created. After reading yours today, it was so fun to hear more of the "inside" story. I was wondering if I could post a link to your blog on my post today? I haven't posed it yet, (plan to do so this afternoon). Your blog was awesome. If not, no big deal. So super excited to see what God does in and through this for our kiddos. Exciting

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  3. It worked, I have been trying for days.=)

    I wrote a blog about the press release last night due to the hope that it created. After reading yours today, it was so fun to hear more of the "inside" story. I was wondering if I could post a link to your blog on my post today? I haven't posed it yet, (plan to do so this afternoon). Your blog was awesome. If not, no big deal. So super excited to see what God does in and through this for our kiddos. Exciting

    ReplyDelete
  4. that is so very awesome. i saw that nytimes article also, and immediately thought of you (of course). love ya!

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  5. Breck, I have two boys with CF and we live in the burbs on Chicago. Your name sounds awfully familiar to me (I connected to your blog through Cystic Life). My husband was also part of that call. It sounds like it was completely momentous. I love the "home run in the 7th of the World Series). With 2 boys needing care of this challenging disease, I feel the excitement. We are close. :) Wishing you and your family the best.

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