Denufosol… the next miracle drug?

Let’s hope so!

There’s been a lot of buzz about the promise of this exciting drug to enter the realm of cystic fibrosis treatments. As it enters its second Phase III trial, the excitement is palpable. Will this change the future of the cystic fibrosis in our youngest population, potentially staving off the effects of lung damage by helping to prevent it in the first place? While the future of denufosol remains unclear for the moment, the long term implications of potentially finding such a drug is giving hope to thousands.  Another reference here.

This news burst follows closely on the heels of another major advancement for those with cystic fibrosis who have what is known as nonsense mutation (approximately 10% of the CF population).  A drug known as PTC124 continues to show promise for these patients as well as some patients with a certain form of Muscular Dystrophy.

In hopes of continuing the momentum and helping to find a cure, I have once again activated my team, Ally’s Allies,  to join the Cystic Fibrosis Foundation’s Great Strides fundraiser.  Join my team, form a team of your own, or simply donate.  Your support makes the difference for these and other drugs to be discovered and used to help save lives… thank you for your help!

Secondhand smoke is deadly. Dangerous. Downright dirty. So what’s the news? Check out this JAMA abstract to see the facts of the effects of secondhand smoke on those with CF.  Another article here.

For those living with cystic fibrosis (or other lung diseases, for that matter), secondhand smoke is, essentially, the devil. Now, I’m not here to tell you to quit smoking (though you should), but I am asking you to consider the effects on those around you the next time you light up.

…on my heart and in my mind Tricia, Nate and Gwyneth. You’ll remember a few posts back when I posted “To be in Love…” about Tricia’s journey toward a double lung transplant was suddenly halted when they discovered that they were pregnant with Gwyneth. I have seen many miracles in the last four years during our own medical journey with Allison; but never have I seen anyone flourish and shine in their steadfast Faith the way I’ve been witnessing Nate’s awesome ability to chronicle their very personal and private journey. When you get a moment and have some time to really get sucked into something heartwarming, be sure to check out their blog.

… or not? That was the question.

When you “live in chronicity” (or parent someone who does), you exist somewhere between those people who can make plans and keep them and those forced to mark every “commitment” down in pencil. For example, I recently took a trip out West to visit another CF care center (details to come soon). Up until the very moment I left for the airport I couldn’t be sure whether or not I would be able to go. Would Allison be able to hold out until her doctor’s appointment on Tuesday (and is it okay for me to not go, and send my husband instead?), or would all hell break loose before then? Obviously, my choice would be to stay with her when I know that she needs me, but given the bigger picture, and the task that lay before me on my trip, that was ultimately for her benefit as well. (Okay, I’ll admit a sanity break was in order for me, too.)

I went, I learned, and discovered that, in fact, life can somewhat be managed via Blackberry and conference calls. While I strolled down First Avenue, I got up-to-the-minute details of the doctor appointment and was able to contribute my two cents from thousands of miles away. However, it wasn’t the same as being there.

The news from that appointment: she has to get better in 14 days on oral antibiotics, or we’re putting her in… as in, admitting her to the hospital for no less than 14 days for IV antibiotics, lots of chest physio (to clear her lungs), rest and nutrition. Nothing like a little pressure hanging over your head. You may or many not be home for two weeks in the next several days. Left in limbo, you don’t know whether to buy groceries (watch those perishables), pack your bags, or beg to just be admitted now and get it over with. Get the the laundry caught up, make sure the bills are paid, call your friend and inform her that you may or may not be able to help her out after all (sorry about that ~ I know we set this up months ago, but what’s a mom to do?), and sit and wait. I can’t RSVP for the bachelorette party and bridal shower for a dear friend three hours away because I just don’t know what is going to happen.

Living in chronicity extends far beyond what happens in your immediate family; you learn who your friends are based on their reactions when you have to cancel plans at the last minute and when extended family lives in limbo with you because you just don’t know what could be coming down the pike. Not to say, however, that life is ALWAYS like this. We just hit a pretty rough patch every few years. And the reality is that no one is guaranteed anything. Ever.

So how does someone “Live in Chronicity?” With all of the humor, determination and stamina that one can possibly muster. Fueled, of course, by the lovely and innocent reason behind it all:

These are exciting times indeed!

The CFF wins again… sort of. It’s no small feat for the Cystic Fibrosis Foundation’s care center network to be recognized by The National Institutes of Health as a model for the delivery of care for those with a chronic disease. Even more surprising for some was the bold move to make public the data on each of their care centers. Read the highlights here.

I directly attribute the great achievements and recognition of the CFF’s efforts in providing quality care while using innovative means to search for a cure to the extraordinary vision of the President and CEO of the Cystic Fibrosis Foundation ~ Robert J. Beall, Ph.D. I’ve had the honor of meeting Dr. Beall on a few different occasions and I have to say I’m massively impressed with his dedication to finding a cure for my daughter. I realize he’s the frontman for a much larger team of players, but it means something more when you’ve actually seen his passion up close and personal. Which is why, when I get the form letter email from the Foundation about what Dr. Beall has been up to, I take note of what he has to say. I hope you’ll take this plea seriously, too. Today’s form letter read something like this:

Dear Friend,

Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation, testified today before the Small Business Committee of the U.S. House of Representatives. He urged the committee to support research of rare diseases, such as cystic fibrosis.

Click here to view Dr. Beall’s testimony.

Specifically, Dr. Beall asked Congress to reauthorize the Small Business Innovation Research (SBIR) program, which provides grants to small biotech companies. He also urged Congress to dedicate a portion of these grants to companies interested in building drug discovery and development programs for CF and other rare diseases. Congress must reauthorize the SBIR program before it expires in September.

You too can make a difference—and inspire others to take action—by building a powerful CF Advocacy Task Force. Here’s how:

1. Register to be an Advocate.
   You will be notified when your involvement can make a critical difference.
2. Ask 5 friends or family members to sign up.
   As Advocates, they will help ensure that our agenda makes an impact in Washington, D.C. There is power in numbers.

Thank you for supporting our important mission. Together, we are adding tomorrows every day to the lives of those with CF. Sincerely,

Cystic Fibrosis Foundation
6931 Arlington Road
Bethesda, MD 20814
(800) FIGHT CF
E-mail: publicpolicy@cff.org
Web: www.cff.org/GetInvolved/Advocate

P.S. Remember—register to be an Advocate and ask others to sign up!

Did you realize that for most people living with CF, gaining weight is a tremendous struggle? Many of us complain about having to lose weight, reduce our food intake and watch what we eat to be healthier… can you imagine the if the opposite were true? Sure, it sounds tempting initially ~ to be able to eat virtually anything you want in an effort to maintain your health.

However, as this article points out, consuming enough calories in a day can be, well, consuming.

*Side Note:  While the article points out that it gives the person with CF pretty much carte blanche to eat even the emptiest of calories, more recent research for the CF diet has emphasized the use of “good” fats versus “bad” fats and trying to consume larger quantities of “healthier” calories, including a diet rich in fruits and vegetables.