Saturday, November 5, 2011

Honor the gift

Let me preface this entry by saying that today I am feeling so tired, so I hope this makes sense.  In other words, the day after day of being super excited and not sleeping or eating normally is catching up to this old lady.  I am a pretty emotional person also, and the last few days have been quite overwhelming.  About 10 o'clock this morning, I hit a wall and thought I was going to just pass out in the hallway...Fortunately, the very next presentation I saw was about VX-770 and apparently that particular subject works like crack for me.  I see a big crash coming soon and as amazing as the last few days have been, I miss Brady, my husband, and my juicer.  I don't have a lot of time to write before the closing events this evening so I'll jump right in.

Plenary session: Pulmonary Exacerbations
1st of all, the drug development pipeline has been updated and changed format on the www.cff.org website.  It contains more information than ever before and I encourage you to explore the new, more interactive format.  The pulmonary exacerbations talk first went into the difficulty of defining what exactly constitutes an exacerbation.  They would like to establish a standardized method of identification and treatment among care centers.  Currently, the protocol of treatment almost always includes a round of either oral or IV antibiotics.  Seems logical, but data showed that in many cases, bacterial growth rebounded right back to pre-flare-up growth levels within a short time after treatment, even when FEV1 had increased, which begs the question: Is this the right course of action?  They also addressed questions about how bacteria move or migrate throughout the airways.  Do infections "slough" off one area of the lung and migrate to another?  How do they spread and disperse?  Also, which bugs (of the many they may see in culture) are the real trouble makers in terms of destruction of lung function?  This talk presented a ton of questions, but not many answers.  It was actually pretty surprising to me that so little is known about what causes pulmonary exacerbations and how to treat them.  The speaker presented question after question about exacerbations and ended up with a slide that read exactly "We know a little more than Jack."  As in, we know slightly more than jack shit about this and we need more studies.  They have lots of ideas for learning more about this basic concept of risk factors that promote exacerbations and the most effective ways to treat them.  They hope to learn from COPD exacerbation model and continued research projects are underway. 

Next, I attended a talk about Key Advancements along the CF Drug Development Pipeline.  They described what went wrong with Denufosol(an inhaled treatment that attempted to correct for CFTR dysfunction by activating an alternate channel (calcium channel) to transport chloride ions.  In short, the trials were poorly designed and seemed to use some pretty creative statistical analysis to get as far as they did in trials.  This talk felt sort of like an apology.  Next up, they commented on where we are with Bronchitol.  Bronchitol has already been approved for use in Europe, and is thought to have a similar line of function as Hypertonic Saline.  Rather than inhaled salt, Bronchitol is a large molecule sugar alcohol that comes in the form of an inhaled powder(Similar to Advair, which saves a ton of time with a single inhalation vs. 30 or so min on neb with Hypersal).  They plan to get this drug approved in the US soon, but they weren't sure when.  They also couldn't comment on its efficacy above and beyond what is seen with Hypersal.  In my mind, if it works as well as Hypersal but can be taken in a fraction of the time, it would improve quality of life enough to make it worth it.  I'm sure we will be hearing more about this soon here in the US.  This is where I hit that wall I mentioned earlier.  Fortunately, next up was the data about VX-770.  There was no new data, but rather a review of the trials that led to the FDA application.  You would think that I would be getting used to seeing these stats by now, but something about those graphs on those huge projector screnes of dramatically dropping sweat chloride scores alongside similarly dramatic spikes in lung function put me in tears...again.  It is nearly impossible to wrap your mind around a concept like this when you have been dealing with CF for any length of time.  The more tired I get, the harder it is for me to control my emotions and act professional.  I've been screaming on the inside the entire time I've been here and as I sat in that darkened room of completely subdued scientists and physicians...it was all I could do to keep myself seated and quiet.  I wanted to stand up and give an old school fist pump and woot with each new graph displayed.  I also wanted to kick the lady next to me because she didn't even seem to be paying attention.  It just never ever gets old.  My hands still shake and sweat, my breathing goes all funky, and I'm pretty sure my eyes bug out when VX-770 is the topic of conversation.  I know getting VX-770 to where it is today has taken decades of collaboration between Vertex and the CFF, but it is truly is amazing that it was discovered at all.  Dr. Beall told me yesterday that when they first began screening for compounds to treat the basic defect of CF, VX-770 was in one of the first few thousand compounds they screened.  In the millions of compounds that they have screened since, nothing has come close to being as pharmacologically perfect.  Imagine setting out to find a single burned out light bulb in all of Orange County and then finding it on the very first block you search.  Finding that compound was more than lucky, it was a miracle.  The last talk in this workshop was given by a researcher from Pfizer, one of the largest pharmaceutical companies in the world, who has now thrown themselves into the arena of CF small molecule research.  Vertex now has company in searching for new and better functioning corrector compounds.  Pfizer has its own databases of compounds that haven't previously been screened by anyone and they hope to find some gems there.   They want to find the "next generation" of correctors that work specifically on the two misfolding sites I described on F508 yesterday.  They believe that a two drug combo of correctors working synergistically will provide the best results and are setting out the find those burned out lightbulbs.  Let's all hope they get as lucky as Vertex did, so they can begin the process of clinical trials...like NOW NOW NOW!

Next up was a special luncheon attended by CF patients, families, and certain caregivers.  The face of healthcare is changing.  Patients and providers can both do more to improve the quality of care, especially for chronically ill patients like those with CF.  I didn't see this coming, but this talk hit me like a ton of bricks.  "Tiffany Christensen is the CEO of Sick Girl Speaks, Inc. She was born with cystic fibrosis and has received two life-saving double lung transplants. Today she is a national public speaker, the author of “Sick Girl Speaks!” and “We are the Change: Transforming the Healthcare Experience through Partnership.” http://www.sickgirlspeaks.com
Yes, you read that right.  She has undergone not one, but two double lung transplant procedures.  It is amazing to me that she has spent most of her life very ill and close to death.  There was so much life in that tiny lady that her body seemed too petite to possibly contain all the energy and positivity that she exuded.  I'm planning an entire blog entry centered on the tools she taught about communication between patient and provider.  I don't have the time to go into detail now as the closing event for the Conference begins in less than an hour, but I do want to say this.  She described her drive to do something meaningful with her life as a way to "honor the gift."  As soon as she said the words, tears sprung into my eyes and the words haven't escaped my mind for a second today.  Honor the gift.  She knows how lucky she is to have been given the gift of a second...and a third chance at life.  She now works to train medical students, medical staff, and patients on how to improve communication in healthcare, especially in regard to end of life issues.  Brady doesn't need a transplant, but VX-770 certainly feels like a gift to me.  I'm not sure exactly how, but I will be spending the rest of my life trying to "honor the gift" I've been given through continued work and advocacy for the CF community.  I'm so humbled I can barely stand how lucky I am.  I have so much more to say, but I have am out of time for now.  I fly out at 7:45 a.m. tomorrow morning.

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