Monday, November 21, 2011

Minnesota CF Education Day

On Saturday, Casey and I spent the day at the University of Minnesota getting our CF learn on :) I apologize that this post is going to be full of information and not the usual “can you believe Gavin said that” and silly pictures! But I know that many family members/friends follow our blog as their method of keeping up with CF news…so here we go…

It was a long day, jammed packed full of information – and while I left exhausted, I am so thrilled that we finally went and my goal is to go to it every year from here on out.

We began the day with a CF research update – and while I knew about a lot of it, some of the information was brand new and extremely interesting. While studying with pigs, the University scientists have discovered that on average, growth hormones are lower in CF patients. It has been debated if all of the growth delay is simply a loss of nutrition and the constant fight against infection that people with CF deal with every day, or if there is another role that CF plays in the deficiency. They have discovered (through the pigs) that the pituitary gland seems to have normal amounts of RNA (which instructs the body to make the growth hormones) but for some reason, the pituitary gland does not respond to stimulation the same way as a “typical” pituitary gland would. The connection between CF and growth hormones has a still complicated, and not yet understood, correlation but they are working on it! Especially because it is proven that bigger kids = bigger lungs = better overall health!

Gavin’s pulmonologist has been doing a ton of study on CF patients under the age of six. Until the age of six, its pretty impossible to get an accurate measurement of lung function. While our clinic does do infant PFTs those numbers are not as accurate as when you actually blow into a PFT machine – and kids generally cannot do that correctly until the age of six. What she has been studying in a chemical called “desmosine” which may correlate with the lung function of CF patients in the first two years of life. In other words – we may someday be able to determine lung function by measuring the amount of desmosine in a young patients sputum and/or urine without having to do any kind of lung functioning test. Awesome, awesome stuff! Gavin participated in this study – which makes it even more exciting!!!

One of the most interesting points for me, came at this beginning point of the day. The doctors mentioned that CF is being studied in many different areas of the University – including dentistry. Why you ask? Good question, and I was initially confused as well. BUT let me try to explain. A chemical called “agmatine” is found in a CF patients sputum (spit) and makes some bugs grow as a biofilm which makes them even more “tolerant” (resisting) of antibiotics even if that particular bacteria is not normally resistant to those antibiotics. And overtime these bacteria become more and more resistant to antibiotics which equals disaster for those with CF and constant lung infections. BUT, a dentist has discovered a peptide, found in our own sputum, that can actually kill the bacteria – yes, a product found by a dentist, in spit (not a created medicine) can possibly kill these otherwise resistant bacteria. Today (Monday) the scientists/doctors in the CF sector of the school are beginning to see if these peptides can “kill” bacterial pneumonia – this is a huge development in the CF world. So while each speaker was only given a small amount of time to speak – it was extremely interesting to learn that scientists all over the school (veterinarians, dentists etc) are working so hard to gain progress against this awful, awful disease.

Not surprisingly, my favorite part of the day was when one of the doctors began discussing the Vertex drugs. It was also an eye opener that Gavin is really in a small number of CF patients that have the G551d mutation (Just 3-4% of the 30,000 US population). I am constantly torn between this state of wanting to scream my excitement and containing myself with the realization that we’re not yet there for 90+ percent of the CF population. But, when a reputable doctor stood on a podium, in a room full of people dedicated in the fight against this disease, and told us that he was in awe – floored by – the results of VX-770 (Kalydeco)…I wanted to run on stage and give him a hug. Not only was lung function increasing by 10 percent in just TWO weeks, sodium chloride levels were dropping 50 levels into normal readings. Those of you have followed our story may remember Gavin getting a sweat test when he was a newborn. They place a chemical on the skin that causes the body to sweat in that small area and they then test the sodium chloride/salt in that sweat. A reading over 60 is a positive CF result, Gavin’s was over 100 (does not measure severity of the disease). Just think, if Gavin’s number dropped by FIFTY like the doctor mentioned, he would be in normal numbers. Un-be-liev-able. And when I spoke to this doctor during a break, he said that life for my two year old is going to be different. Those testing this drug have lived years with CF, and because of that they have irreparable lung damage – while someone like Gavin does not have any lung damage and is going to be starting with great, pink lungs. Instead of bringing back numbers the goal is going to be keeping lung function high, and to get his body working normally before the damage occurs. Proactive instead of reactive (my favorite saying when it comes to his CF care). I honestly could have spent the entire day talking about Vertex and nothing else…but the hour I did get was pretty amazing. We got to learn how they decided on the “right” dosage – how they discover what dosage is “too much” and how they structure the research categories…fascinating stuff and I am in awe of those who dedicate their lives to finding the necessary control, if not a cure, of this disease.

The news on 809 was also promising – a lot of what they are doing is trying to figure out what dosage of 809 works correctly with what dosage of 770. They are learning that a higher dosage of 770 is needed when combining the two meds to get the desired results with the Ddf508 mutations (vs a lower needed dosage for those with G551d and only 770 alone)…but those balances are all still being tested. So for those of you worried that 809 “isn’t working” please keep your hopes up. They are definitely still tinkering around with the mixes to get the results they need – they are on the right track. It’s not that it isn’t working, it’s that they are trying to figure out how much of the med is needed - and what balance between the two meds are needed - to get things working. Seriously, very close! I can definitely send more of the info I learned along to anyone curious about more on the 809 studies!

On a side note - more and more drug companies are coming on board with finding these correctors and potentiators (809 and 770 respectively) – sadly its because many of these companies are seeing the “dollar signs” that weren’t there before – but we’ll take all the help we can get now!

We learned more about nutrition – the importance of taking enzymes – the importance with staying in close contact with Gavin’s dietician to continue checking his BMI and keeping it matched up with his enzyme intake etc. We learned about the necessity to stay committed to treatments. The necessity to keep on track with visiting the CF clinic at least four times a year and getting PFTs at least twice a year for those who are able (older than 6). That yes, it can be a pain in the butt to “fit in” the time to get to clinic but that above all, HEALTH is the most important aspect of all.

Lunchtime was fun – I got to meet two other families with young CF children. We also sat at a table with a nurse who had performed Gavin’s sweat tests nearly 3 years ago. She remembered us AND remembered Gavin’s name without prompt. This is exactly why I call our team family. They care about us, remember us, and truly give a damn about our lives outside the CF world. Our team poured over our wedding album – which they had repeatedly asked for. And guess what – the only picture Gavin’s pulmonologist showed during her talks was of…yep, you guessed it…our one and only Gavin! And to be honest – a huge reason why a lot of the information we learned that day was a refresher is because his doctor is so amazing. Whenever we go in she thoroughly explains everything we’re doing, why we’re doing it – what we’ll do if it doesn’t work, and how to confront different circumstances in life. She has an amazing ability to explain things in terms we understand and there is something to be said when your two year old gets excited to see their doctor! But ok, enough gushing!

After lunch we got to see the one and only Josh Mogren perform. After two years of communication between computers and cell phones I finally got to meet the guy face to face. We also got to meet Moganko! He performed with a local a cappella group and they performed songs they created for CF kids about taking their medications/pills and keeping up with their help. I would love to tell you more – but what we got was a special preview – Josh has not released most of what we’ve heard yet…but once he does you know that I’ll share it there! He is definitely someone I cannot imagine living this CF life without – I cannot count the number of times he has been there with amazing advice, whether its about CF or just life in general. The guy is good stuff :)

We also got to hear from a panel of three adults living with Cystic Fibrosis. It was definitely a great opportunity to pick their brains, and to get a grasp of what Gavin will feel/experience as he ages. Some of it was heartbreaking, yes – but I think hearing them will make me a better parent. They expressed their feelings about being pushed to do treatments, to step back and let them live and how to face many situations – one of the best parts of the entire day. Definitely priceless material!

The last part of our day divided the group into discussions about adults or pediatrics. For obvious reasons we chose to listen to the talks about pediatrics :) Gavin’s pulmonologist lead a talk about many of the different medications/antiobiotics being used right now. I won’t get into details on most of them – but it was a nice refresher and great to hear why certain meds cannot be mixed and why some can. Also great to hear more about side affects to look out for. Overall just some great info!  A small part of the day was spent discussing networking between the parents – I couldn’t believe how many parents didn’t network at all. The team did not bring up CysticLife but I sure did! I honestly cannot imagine where I would be right now without the support of my CF family. They are the people I don’t have to explain myself to, they just ‘get it.’ No one but them truly understands our day to day life, and the emotions that come along with it.

All in all it was a long, but amazing day. Kudos to the CF team who gave up another weekend of their time to help out so many CF families – especially after many of them spent many days in Anaheim at the national conference. We seriously appreciate the extra steps they take in keeping us informed and well educated on the best CF care possible.

Oh, I cannot believe I almost forgot this. Guess what. Our clinic was ranked NUMBER ONE…yep, NUMBER ONE in the nation for FEV1 levels (lung function) in pediatrics ages 6-12. Amazing! We are so very, very, very blessed!

And before I leave you…

This month alone, I am aware of SEVEN individuals that have lost their fight against Cystic Fibrosis – one of them being only a few days old. Which is why I believe it is beyond important for every.single.CF parent/patient/significant other to attend some sort of educational conference to learn about this disease. When we learn, we can teach. When we teach we gain interest. When there is interest there are donations. When there are donations RESEARCH IS FUNDED and LIVES ARE EXTENDED. It begins with US. The parents. The patients. The caregivers. It is OUR responsibility to step in and learn about what is happening, what will happen, and what we can do to better the lives of those living with this disease every single day.


  1. Fantastic post!! Thank you so much for sharing. Is it possible for you to send me all the info on 809/770 combo please?

    Email addy is

    Don't feel strange celebrating kalydeco, I'm so so excited for all it will help and know it's only a matter of time for everyone else.

    Gemma ( x

  2. Thanks Jen for this post!! It made me cry!! You are Casey are such great CF parents arming yourselves with as much info as possible and doing 150% of everything for the best interest of Gavin... This info is amazing... and I am so thankful that us CF-ers and CFER parents are crusaders on the front lines raising all these dollars for this disease so that you can attend a conference like this and share all this info... Thank you!! I am so thrilled for kalydeco and what it will do for that boy of yours!!

  3. Thanks for the info! I am SO excited for your family. Such amazing developments, you have every right in the world to be jumping through ceilings these days. We've met with Dr Laguna before with Rylan and I LOVED her. Such an amazing staff you guys have up there :)

    Would you mind sending me the info on the combo drugs? I would appreciate it immensely. We made it to the conference last year, but missed this year :(

    Thanks, again! Happy Turkey Day :)

  4. Thanks for all the great info! It's so exciting to read about all the new studies that are happening.

  5. Great post. LOVED IT!!! Somedays I get excited with the hope about of a future different than the CF of today. This is our prayer that someday CF really will be about being proactive verses reactive. =) Thank you for sharing.

    If you could send me the information ( I would love it. Somedays we just need to see the hope instead of the crud of CF.

  6. Thank for the mention, Jen! The conference was amazing on so many levels for me. I can tell you that this one had a different feel to it than other years where lots of things are rehashed and progress is made, but its still too far to see. Saturday had a lot of great things share that we can actually see. I'm excited for everyone with CF, but most importantly for little peanuts like G-man. Less or no CF is good stuff. ;-)