28 March 2012

Gin and tonic - I mean, HYPER tonic Saline

Bennett has added a new inhaled medication to his daily regimen.  It's called Hypertonic Saline.  It is one of three inhaled medications he takes daily for healthy lung hygiene.  Hypertonic Saline is breathed in for about 10 minutes once daily during Bennett's chest percussion therapy (aka during his Vest).

Hypertonic Saline is basically salt water in a tiny vial.  This medication was discovered when it became apparent that Cystic Fibrosis sufferers who surf in salty ocean water do better health-wise than their counterparts who do not surf.  Researchers believe that inhaling the salt from the ocean has significant respiratory benefits.  A study later confirmed these findings.  Since this study, Hypertonic Saline has become part of the regular care of those with CF.

We open each vial for use by twisting off their top and squirting it in the nebulizer.  (The compressor is what is in the background of this picture.  The nebulizer is what is connected to Bennett's mask and ultimately what Bennett breathes through.)

Bennett is so very patient during this medication.  It's easy to imagine what it's like to have to breathe salt into your lungs.  If I sit close enough to him, I can breathe it in too.  It can be very irritating to the lungs and causes coughing.  Coughing is very important to those with CF because it helps move that nasty sticky mucus around and out of the lungs.

Shaking tubes.

This piece of equipment (Bennett's Vest) gets used 20 minutes per day.

Bennett sits so quietly during his vest treatment.  He enjoys watching TV during this time.  He prefers to watch Superman, if Daddy is around and will watch it with him (aka if he will navigate around the inappropriate parts).

Smoky vapors = hypertonic saline.  Hypertonic saline can cause a sore throat because it irritates the esophagus tissue.  I would hate to have to do this every day.  What a champ he is!

The crazy thing about Cystic Fibrosis is how much salt is involved with this disease.  Salt is the underlying issue for why the body has such problems.  "In cystic fibrosis, the salt balance in the body is disturbed. Because there is too little salt and water on the outside of the cells, the thin layer of mucus that helps keep the lungs free of germs becomes very thick and difficult to move. And because it is so hard to cough out, this mucus clogs the airways and leads to infections that damage the lungs."  It is obvious why salt is now part of the treatment to relieve symptoms of this disease.  

26 March 2012

Tshirt Orders

So many people have been letting me know they want to buy tshirts!  It means so much that so many friends want to wear "I'm In It For Bennett" on their chest.  I can't wait to see the flood of green at the Walk.  It just touches a mother's heart to imagine something so sweet.  And then, to know people far away who can't even be at the Walk want tshirts too.  Man, my heart wants to explode with gratitude!
Anyway, I wanted to make sure I reposted the tshirt information so nobody accidentally misses out if they want one.

Here's the rundown:
Cost: $15 ($5 from all adult shirts will go to the Cystic Fibrosis Foundation).
Free Shirts: Our family will give your family a free tshirt if you donate $100 or more.
Order Deadline: April 19

Tshirt Specs: Gildan tshirt, Antique Irish Green, black and white ink on the front and white ink on the back. (Note: Children's shirts will be printed on Gildan Irish Green because the "Antique Irish Green" doesn't come in Youth sizes yet.)
Sizes:Youth sizes XS (2-4), S (6-8), M (10-12), L (14-16). Adult S, M, L and XL (contact for more sizes)

To order: Email me at breckgamel at yahoo dot com with your size and address.
Payment options: Check is best but if you have another idea, I'm open! I'll send you the address, just email me.

23 March 2012

Advocacy in Action: The EXXPERT Act

There is a really important bill coming through right now in the House that I have been asked to help share information about...  

It is the "Expanding and Promoting Expertise in the Review of Rare Treatments (EXPERRT) Act" (H.R. 4156) in the House of Representatives. 

This EXPERRT Act legislation aims to enhance the Food and Drug Administration’s (FDA) rare disease drug review process by including systematic consultation with experts and patient advocates.  

According to the Cystic Fibrosis Foundation, the bipartisan EXPERRT Act of 2012 would complement FDA’s existing authority by allowing "rare disease experts, academic researchers, health professional organizations, patient advocates and care providers greater opportunity to offer input and advice to the FDA on rare diseases and drugs and by helping FDA officials navigate a rapidly changing scientific landscape, ensuring that they have the most up-to-date, specialized information when making decisions about rare disease treatments under review."

"The EXPERRT Act makes it easier for review staff to access specialized input and expertise on issues like the risks and benefits of potential therapies, the design of clinical trials and medical needs not met by existing treatments.  This increased collaboration will make the FDA review and approval process more efficient, allowing patients to access needed treatments more quickly."

This is a bill that, while it will help those with Cystic Fibrosis, will help all of those with diseases waiting for medications to be approved by the FDA. 

There is an easy way for those in support of this EXPERRT Act can help get it passed:

1. Follow this link and send a pre-written letter to Congress. 
2. It asks your representatives to support a no-cost bipartisan bill that will save many lives.
3. It does this by making the FDA work more efficiently and more effectively
4. It WILL bring a cure to Cystic Fibrosis that much closer when passed.
5. All you need is 2 minutes to have your voice heard and to make a difference

(Thanks to my friend Peter for putting the following simple steps together for me to share!)

The EXPERRT Act is expected to reach the House floor to be voted on in the next two weeks.  So, I am watching closely to see what happens!  

21 March 2012


Since there is no government or pharmaceutical funding for Cystic Fibrosis, families like ours depend on the generosity of others.  This is why fundraising has been one of the biggest roles of the Cystic Fibrosis Foundation and those families who belong to it.  The thought process has been: You can't find a cure without research.  And you can't research without money.

But as more and more drugs are showing promise for those with CF, it is becoming increasingly obvious that the job of the Cystic Fibrosis Foundation is not only to raise money for new medications but to ensure those medications can get quick and efficient FDA approval once they have been shown to be effective.  This is why a new important role of the Cystic Fibrosis Foundation as developed most recently: Advocacy.

I didn't really know what "advocacy" was until I attended the Volunteer Leadership Conference last month.  But once I understood, I realized this is equally as important for Bennett as fundraising for a cure.

For example, as important as it was that scientists were able to discover the drug Kalydeco, it was equally important that the FDA's approval of Kalydeco was fast (fortunately, it took less than 3 months, usually unheard of).  With a disease like Cystic Fibrosis, time is of the essence.  Advocacy prevents the roadblocks that might prevent a drug from being available soon enough.

The way the Cystic Fibrosis Foundation has begun to address the importance of working with the government to prevent roadblocks in the process of getting medications to people more quickly is by designing a "State Advocate" and "District Advocate" program where by volunteers take on the responsibility to contacting their local Representatives and Senators to let those who represent us in government know the needs of those with CF.

After careful thought, I have decided to take on the role of the Cystic Fibrosis Foundation's State Advocate Chair of Northeast Texas (the Dallas/Fort Worth area).  My job will be to stay on top of the legislative issues important to those with Cystic Fibrosis and to stay in contact with the government officials in my area.

So, I am excited to help out in this way.  It feels good to be able to help find a cure for my son that doesn't necessarily involve only trying to raise money.  Plus, I get to put my journalism skills to good use, which I love.  :)

There is, in fact, a really important bill coming down the pipe in the next two weeks.  But that seems like it should be a blog post all of it's own...

Random Thoughts On Bennett

Bennett is quickly moving from a baby to a big boy.  He is two and a half and no longer seems so infantile.  Part of the biggest change in his life is that his language skills are developing daily.  He surprises us daily with his new words and a few sentences.  Our favorite thing is he has a little lisp when he says his "s's".  The word yes is pronounced "Yeth" to him. :)

(This is his typical face when we say, "smile."  I think he thinks that I am saying, "squint!" :))

Bennett calls Oliver "Ah-Ah," as in the beginning sound of Oliver's name.  Bennett can just barely mumble his own name so he usually to refers to himself as "me." (Case in point, Bennett will look around the table at everyone's plate and say: "Ah-Ah, Momma, Daddee, me.")

Bennett takes his enzymes like a champ these days and is very compliant with his treatments and medications.  This may be because he is still very much in love with his pacifier (ack!).  Bennett is ready to be potty trained, if I could just get focused enough to do it.  His drink of choice is apple juice, which he calls, "Ap Jew."

The boys are best friends and play well together.  Bennett seems to want to play with Oliver more than Oliver wants to play with him.  But, overall, they really interact with each other very easily.  They rarely fight (knock on wood).

Bennett *loves* stuffed animals, tends to want to kiss and hug everything and anything (he is very affectionate) and especially loves playing like a "fish" in the bathtub.  His favorite outdoor activity is to swing.  That child could be swung all day long, if I'd push him!

It's interesting to see in what ways the boys are similar and in what ways the boys are different: Oliver has always loved playing with cars.  Bennett likes them too.  But Bennett really enjoys little toy men and action figures.  While Oliver would prefer to play alone quietly, Bennett prefers to play with someone, especially if he can get them to wrestle or tease with him.  Bennett has a great sense of humor.  Both boys love to be read to.  Oliver wants to learn about tornadoes and electricity.  But Bennett is most interested in firetrucks and Superman.

I am so thankful that Bennett's health has finally come to a place where we don't have to think about Cystic Fibrosis every day.  We are now able to start focusing on new things, like his developing friendships and learning about the world around him.

Overall, Bennett is thriving these days.  I continue to look forward to seeing his little personality develop as he is learning to use words to tell us his thoughts!

14 March 2012

A new medication for Bennett?

{EDIT as 10.21.12: Updated information on the medication Ataluren was provided recently at the North American Cystic Fibrosis Conference.  Information provided indicates Ataluren is not a medication that will likely come to the market due to complications with the medication for those who have participated in Ataluren trials.}

One of my favorite things the CF Foundation provides for families of those suffering from CF is the CF Pipeline.  The "CF Pipeline" is a like a guide of what medications are currently being researched and where they are with regards to being available for Cystic Fibrosis patients.  It makes it very easy for families to see which medications are "coming down the pipeline."

This is an old pipeline image but this is similar to what currently exists.  For an up-to-date pipeline, go to the most current form on the Cystic Fibrosis website.
At this point, there are around 25 different medications in the pipeline.  Some medications are for keeping lungs healthy.  Other meds are to improve digestion.  There are a few medications to counteract lung infections.  But some, the most hopeful of them all, are those that are showing signs they can treat the underlying issue in Cystic Fibrosis.

I have known about the CF Drug Pipeline since we first learned Bennett had CF.  But I haven't been all that interested in it as I haven't felt like it mattered much to us.

This all changed most recently when a CF Mama who has a young girl with the same genetic mutation as Bennett mentioned to me that her daughter (and therefore, my son) might benefit from this new medication called, Ataluren.

In order to have Cystic Fibrosis, one must have two genes that do not work properly.  There are 1800 different mutations that have been identified as not working properly.  Any combination of two of these can show up as being Cystic Fibrosis in a person.

All genetic mutations have names.  Bennett's specific CF genetic mutations are called "DeltaF508" and "621+1G->T."  (They are pronounced "Delta F Five-Oh-Eight" and "Six-Two-One Plus One G to the T."  Yes, it's a mouthful.)

I was surprised when my friend mentioned Ataluren might help Bennett and her daughter Savannah.  I had not heard much about Ataluren and what I had heard was that it helps those mutations "that end with an x".  Neither of Bennett's mutations end with an "x".

But following my friends' lead, I went searching for more information about Ataluren and what I learned was Ataluren helps those with Class I mutations.

Of the 1800 CF mutations that exist, each is categorized by class.  Each class indicates the place where the gene is malfunctioning.  At this point, scientists have identified five different areas of gene malfunction.

This is important because, as scientists discover more about what is malfunctioning in the gene, scientists are more likely to figure out how to solve the problem.  By classifying mutations, medications can be identified to help the problem.

This is the case for the new medication Kalydeco.  It was discovered that Kalydeco can significantly improve the malfunctioning of the gene for those Class III genes.  This is why only 4% of those with Cystic Fibrosis have benefited...because one must have CF due to this specific malfunction in order to benefit.

Ataluren looks to be benefiting those whose malfunction is happening in the area of Class I (premature stop codons in the mRNA).

And, to my surprise, when I did some research, my friend was so right!  Her daughter and Bennett may very well benefit from Ataluren as they both have the gene mutation 621+1G->T which is a Class I mutation.

I spoke with Bennett's doctor about it last week and she agrees we have a reason to be hopeful.

So, what does this mean for Bennett?
First, we don't know for sure if Ataluren is going to help Bennett or Savannah.  But, from what we have read from the research so far, we do not see why it would not be somewhat beneficial to them as they suffer from a Class I CF mutation.

Second, we don't know if Ataluren is ever going to make it to public use.  But we do know that it is currently in Phase 3 Clinical Trials, one step away from being approved by the FDA.

Third, we don't know the results of the Phase 3 Clinic Trials and how affective the drug is.  There has been some talk of side effects on the liver.  And it hasn't been thought to be quite as successful as Kalydeco.  But we have heard good things and believe we should know the results of the Clinic Trials by the end of the summer.

Fourth, we don't know what it will mean even if Ataluren does help Bennett and Savannah, does make it to public use and does show effectiveness.  All we know is we have reason to be hopeful.

Only about 10% of those with CF have a Class I nonsense mutation like Bennett has.  So, like Kalydeco, this means that Ataluren may directly affect only a small portion of people with CF.   However, like is the case with Kalydeco, there is suspension that, if beneficial, Ataluren might be given in addition to other drugs in the pipeline as an effort to help many more people with this disease benefit.

I have other thoughts to share in the future.  But for now, it's worth noting that I am cautiously optimistic that Bennett may benefit from a drug that looks like it may help.  And when living with a disease known to take one's life, any optimism is much appreciated.

I'll post more on this drug as it becomes available.

Here are some other links to information about Ataluren:
* Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonesense Mutation Cystic Fibrosis:
* Chronic ataluren (PTC124) treatment of nonesense mutation cystic fibrosis:

09 March 2012

Bennett's CF Appointment

My friend Teresa said it best when she said going to a quarterly CF appointment is often like "going to the Principal's office and wondering what you did wrong this time."

I was worried, going into yesterday's appointment.  But I left fairly pleased.

First, we addressed his lungs.  Bennett's lungs sounded clear, which is wonderful.  No signs of any lung issues yet.  We did decide to add the daily use of Hypertonic Saline to help continue to keep his lungs healthy.  

Bennett is learning to be more and more brave when it comes to his doctor appointments.  Dr. Cannon is particularly patient when it comes to her littlest patients.  (By the way, no worries, we use the pacifier as a soother since our appointments can be very long and stressful.  But, we're planning to pull the plug on it at three no matter what. :))

Bennett does tremendously better if he has a stuffed animal go first.  When we asked Dr. Cannon if she would mind checking Mr. Fox's ears first, she said, "Sure, I haven't checked a foxes' ears yet today."

Of course, the most important thing on the list the doctor wanted to address was Bennett's weight.

As I expected, Bennett weighed 28 pounds yesterday, the same he's weighed since August of last year.  That's eight months of virtually no weight gain.

Currently, Bennett is in the 30th percentile for weight and 11th percentile for height.  The concern is that his body is simply not growing.

The "X"'s represent Bennett's weight check-ups.  He went from the 50th percentile in August to the 30th percentile in March.

This is a poor photo of Bennett's growth chart but it shows how he is "leveling out" with his weight (which, for a child who needs to be growing along the curve, this is like losing weight).

We discussed what Bennett was eating and why he wasn't gaining enough weight.  I have been very concerned that Bennett's lack of eating is why he is not gaining.  But I was so relieved when Dr. Cannon said, "he eats like a totally normal toddler!  He grazes, eats a few bites and moves on.  That's completely normal."

What isn't normal is that Bennett (like all CF toddlers) needs 2,000 calories per day, waaay more than a healthy two year old.  So, I learned, it isn't so much that his eating is an issue, it's his body's need for so many calories that is an issue.

Who would ever think these cheeks weren't getting enough food in them?  :)
Dr. Cannon suggested that the way we address this problem is by increasing our nightly g-tube feeds, something that is fairly easy to do.

I voiced my concern that if we start increasing his g-tube feeds, Bennett will slowly stop eating (forcing us to use the g-tube even more).  But the doctor and dietitian both explained they expect to do everything they can to prevent that from happening.  They both encouraged me to wait and see what happens with Bennett and to allow us to address it when it does.  Bennett may eat less.  Bennett may eat the same.

Either way, Dr. Cannon said she didn't think this was a good time to restart the feeding protocol.  (Hallelujah!) She felt that we need to make meal times fun and not stress over how much he is eating.  The reality is, he is eating enough for a child his age, just not enough for his disease.

In addition to increasing his feeds, we are also going to start giving him nutritional supplement juice drinks during the day in lieu of the apple juice he enjoys.  We hope that a Pediasure-like drink can give him more calories on a daily basis.

I cringed to watch Bennett play on the clinic floor.  But it's almost impossible to expect him to stay off the floor when we're stuck in the room for more than 3 hours.  (The empty wrapper is the chocolate candy I gave him while we waited.  He loves himself some Hershey's chocolate!)

Since this was our quarterly visit and not an annual visit, the throat culture he was given (envision a cotton swab briefly placed at the back of your throat) but he was not given any other invasive procedures.  We do our annual x-ray and blood work next visit.

Our loot: nebulizers and masks for breathing treatments.

Outside of the doctor, social worker and dietitian, we also met with the respiratory therapist who gave us some new masks, nebulizers and tubing for us to use until we return to clinic.  It's always fun to go home with "goodies" as a parting gift from Clinic.

So, overall, we had a very good visit.   I am pleased to know that we aren't going to have to change much to begin the process of pushing Bennett's weight up (we'll increase his tube feeds by only 200-300 calories at a time and hope this helps).  I am pleased we continue to go day after day with not yet one lung infection (I consider him incredibly blessed!!!).  And I am pleased that, for the most part, Bennett had no tears at this appointment!

Our big boy earned two Batman stickers for his bravery for which he was very proud.
Lastly, I asked Dr. Cannon about a new medication that is currently in Clinic Trials that might benefit Bennett.  I was glad to hear there is a good chance it might.  I will post more on this later.  But learning this information was like the silver lining in a dark rain cloud.  I am not letting myself get too excited just yet.  But it's starting to really hit me how really close we may be to a "almost-cure" for Cystic Fibrosis.  Will share more about that soon...

07 March 2012

CF Foundation Conference, Part 3: Nuggets of Gold

A common joke among CF families and those of us who participate in Great Strides, the CF Foundation's largest annual fundraiser is...just as soon as you're done raising money for Great Strides, it's time to begin again!

It's a joke but it's not entirely untrue.  Fundraising begins around January and ends around May.  But planning for such an event happens all year long. 

This year-long process with a Spring-time push can quickly cause burn-out among those who do it.  And I'm not an exception.

So, I am so very glad to have been able to attend the CF Foundation's Volunteer Leadership Conference in February as it gave me exactly what I needed in the way of an energy boost to keep going.

In fact, I think it catapulted me even farther!  This year, I have found some dear friends who have come along side me to help me accomplish a bigger goal than ever.  I look forward to sharing more about this in the coming weeks.

But before the VLC Conference becomes a distant memory, I want to document some of the most important nuggets I gained.  I hope to use this as I return to it each year around this time of year...

I heard a quote by a Cystic Fibrosis mom at the conference that said,
"People ask me all the time how I do it.  I don't necessarily know how.  But I know why."  

I love this quote.  I feel it's the answer to a similar question I ask myself, "Why can't I stop doing what I do?"  The answer is: Bennett.  I am his mother, his advocate, his cheerleader.  If I don't do it, who will?

One of the speakers at the Conference reminded us:
People don't give to diseases.  People don't give to organizations.  People give to people.  

Our story, the story of Bennett's fight, is the single most powerful tool for gaining individuals, groups and businesses to join our fight.  People don't care about statistics, about how the disease affects the body.  What they care about is helping someone in need.  This is why we choose to tell our story, through video, through our Facebook and through our blog.  And as Bennett gets bigger, I'll teach him how to tell his story. 

Here's another quote I remember from one of the speakers:
Money buys science.  Science buys life.  

I remember before I began raising money for CF, I thought, "there are so many people and places that want money.  Why must I ask for money for Cystic Fibrosis?"  And yet, I later learned that the answer is: with no government funding and no pharmaceutical companies willing to invest in medications to cure Cystic Fibrosis, it is up to us to raise money to buy the science we need to save Bennett's life.

When people give, they must understand that the money is to buy the science.  The science is what will buy Bennett his life.

The Cystic Fibrosis Foundation is being run well and spending the money money we raise very wisely.  "The CF Foundation spends their money the right way, all towards research."

One of my favorite moments came from a session encouraging volunteers and CFF chapters to consider more corporate sponsorships.  A high-up GEICO representative (GEICO's national organization supports Great Strides nationally) answered a question given to him by the session host: "Why do you partner with the CF Foundation as your charity of choice?"

The GEICO rep (who may have been an VP in the company, I can't remember at this point) said, "We believe that the Cystic Fibrosis Foundation is a good fit for GEICO because it runs its organization much like we do our own.  For example, their headquarters looks alot like ours."

There was some laughter from the audience.  But after a second or two, it was obvious that nobody understood what he was saying.

The host followed up on his answer, "what do you mean by that?  What do you mean when you say the CF Foundation's Headquarters looks alot like GEICO's?"

The corporate GEICO representative replied, "Well, when I first walked in to the Cystic Fibrosis Foundation's headquarters, I was struck by the fact that there isn't much there except for some basic furniture and a bunch of people working.  This made me happy to see.  Because I'm not gonna be out there fundraising to buy the CF Foundation a new foundation for their lobby.  It is obvious by the office, how they spend their money.  The CF Foundation spends their money the right way, all towards research."

I think this may have been the single most important thing I could have heard from the entire Conference.

We are seriously, seriously, not far from managing this disease in a significant life-extending sort of way.

There was lots of very hopeful talk going on at the Conference.  But, at one point, I found myself starting to daydream during Dr. Campbell's "State of Science" Update and trying to comfort myself, "eh, don't get your hopes up, we are still so far away from a cure - Bennett may never benefit from what Dr. Campbell is talking about"... which I had to reply to my apathetic self, "wait a minute, Breck, you're at a *real* CFF function.  You are hearing it from the horses mouth!  It doesn't get any better than this.  When they say we're close, it's ok to actually believe it!!"

I am not alone.
At this conference, I watched as person after person was given an award or recognized for the tremendous work they are doing on behalf of the Cystic Fibrosis Foundation.  I saw example after example of people who spend day after day building partnerships and raising money for all those with CF, including my son!

I could not help but be inspired to see the success that is happening within the organization.  The CF Foundation is working with more pharmaceutical companies.  More corporations are willing to partner with the CF Foundation to raise money.  More local events are meeting and exceeding their fundraising goals (one family raise more than $200,000 in one year alone for their Great Strides event!).

I sat next to people who are doing what I do but in their small town.  These people I met were those who were ever so slightly dangerous when it comes to the challenge of saving the one they love who are slowly losing their ability to breathe.

I am not alone.  There is an army out there pounding the pavement and making some serious great strides for funding a cure for Cystic Fibrosis.

The new CFF logo is adorable and is a great indication that we are at the dawn of a new era in our fight against CF.  The new logo will take about 2 years to full roll out as a cost effective measure (refer to VLC Nugget #4) but has already begun to be used.  Here is a video they showed us:  This is a part of their "Adding Tomorrows" campaign which is their new campaign for corporate sponsors.  They don't show the new logo at the end of this video but it will be coming along here soon.  The bottom line is the CFF is actively recruiting national corporations to work with us on some national partnerships.  Think American Airlines, GEICO, CVS stores, etc.

Here are some other pieces of random information for those who are interested... (Thank you to my friend and fellow CF Mama Kara Kelly for documenting this information at the VLC Conference!!)
* More than 300 prescriptions for Kalydeco have gone out for those 1200 people in the US who have the G551D mutation.
* About 100 people are in the Phase 2b Trial for the Kalydeco/VX-809 combo study that ends this summer.  They hope to have results/data by late summer.  The VX-661 drug was called the backup to VX-809. It is pretty much the same but metabolized differently and they wanted to have the next generation ready to go in case VX-809 doesn't.
* Researchers believe there is a 50% chance that drug VX-809 will work at this point. If it goes to phase 3, they believe that chance goes up to 80%.  They will have data on drug VX-661 at that time as well.  At that point, pharmaceutical company Vertex will decide whether they will go forward with drug VX-809 or VX-661 into phase 3.  They stated they discovered this compound's potential a few years ago. They were asked why this is not listed on the pipeline CFF site and said because it is in a sense the same as 809 in most ways and in test tube phase. They did mention most of these studies in the lab phase are on human cells rather than rats- a big step as it is one less step and targets CFTR directly.
* The results of the VX-770/VX-809 drug shows potential to be even more effective than VX-770 alone and may restore up to 50% of what the cell should be doing.  VX-770 has shown sweat chloride to drop from 100 to 50, on average. They have seen a 10% absolute change in lung function.
* The CFF noted that new antibiotics, anti-inflammatory and nutritional drugs are coming soon.  Some are showing particularly effective in a fight against resistant bacteria.

* Dr. Campbell showed a slide giving an example of what they believe may happen if they give a combination of three drugs (VX-770 + VX-809 + VRT1019691 is being studied, for example).  The slide showed that there is a significant potential that Kalydeco, given with future medicines currently being studied, will make it such that CF symptoms are not clinically present and will drastically add decades to normal lifespan and quality of life.
* They believe Kalydeco will work for other mutations in its category.  They anticipate over the next few years, after trials, adding onto the FDA label, mutations cleared to take it.  They believe this will effect 10-20% of the CF population but didn't mention mutation types. The VX-809 combo could potentially effect 50-90% of the population.
* The CFF gave us what they hope is a reasonable timeline (although it's potential at this point): 2014 drug Ataluren, 2015 add in other mutations that can take Kalydeco (affects 15-20% population), 2016 combo drug potential approved (affects 50-90% population), 2020 work on next generation drugs and making previous mentioned even better.
* Ataluren study is done and data coming soon. This will effect 10% cf population.
* With regard to a cure, the Cystic Fibrosis Foundation communicated that they feel like they now have a road map to the finish line (and are nearing it).
* The Cystic Fibrosis Foundation have reinvested the royalties from drugs like Tobi, enzymes, etc right back to the most promising drug company Vertex.  They also have new partnerships with companies like Genzyme, Pfizer, Vertex, GSK.  They are spending almost 100 million dollars in these companies to study about 6 million compounds!  
* The Cystic Fibrosis Foundation recently opened the first CFF research lab in Bedford, MA where they can do their own research. They believe it is the first type of lab created by a non-profit foundation.
* They noted that a CF patient is often needing a lung transplant by age 30.  They believe that if things continue the way we are going, CF patients may not need one until age 60.  That's pretty significant!  

04 March 2012

Ugh. Bennett's Weight.

Bennett has his CF Clinic appointment this week.  I'm dreading it.

Bennett hasn't gained any weight for about 8 months now.  He's 26 pounds.  His size 2T pants are falling off of him.  His ribs are showing, so much so that I am gentle with him when I tickle him for I'm afraid it will hurt.  It takes everything within me not to get anxious about it.

He is eating well, for the most part.  But it's just not enough.  He needs to eat 2,000 calories per day - what I eat - and he's not getting that by any means.

Up until now, our doctors have been cautious but not overly concerned.  But the longer he goes no gaining weight, the worse it is for his development and his disease.  I know this.  So I fully expect them to be concerned when I meet with them this week.

As I wrote in an earlier post, I have chosen not to get too worked up about it until we see the doctors.  But, as our appointment approaches, the anxiety creeps in more.  I find myself anxious because I realize that the decision of what to do ultimately rests with myself and Brian.  No matter whatever the doctors recommend, we still have to be the ones who decide what we're going to do about it.

Here are what I see our options are:
* Cut back g-tube completely with hopes that Bennett will get hungrier and eat more by mouth.
* Put back into place the Feeding Clinic protocol we learned this summer forcing him to eat by mouth three to four times daily.
* Utilize the g-tube more at night, which will likely make him even less hungry during the day and slow his oral eating.
* Utilize the g-tube more at night and during day, which will likely make him even less hungry and couple potentially stop his oral eating.

Our GI doctor recently suggested that we try this first, to cut back the g-tube.  But I know in my heart that this is not the best decision.  If he isn't eating now, his hunger won't convince him to eat more.  I wish it would.  But lack of appetite and issues with gaining weight are hallmarks of Cystic Fibrosis.

Putting back the Feeding Clinic protocol into place has huge ramifications for other areas in our life.  In one way, it feels much like trading one issue for another.  If we choose to do this, we will absolutely have to stop participating in many of our daily activities and will have to begin regulating meals to an almost unmanageable (to me) level.  I am very glad we now have the skills to do this if we need but I dread actually having to do this.  It seems like a good idea on one level but it's incredibly disruptive to our entire family on another.

I expect that the doctor is going to suggest increasing his night time feedings.  This is very doable and something we are happy to do.  But we know that his day-time eating will suffer.  Night g-tube feedings can significantly affect the daytime hunger pattern.

The worst case senario is to use the feeding tube at night and during the day.  I don't expect this to be the case, at this point.  But, if Bennett chooses to cut back his daytime eating even more, it can be a slippery slope that causes us to have to increase feedings during day and night.

One of the things I have to do is to remind myself that Bennett isn't physically a normal child.  He has CF.  This isn't going to change.  And while I'd like for his life to be as "normal" as possible, normalcy comes behind being healthy.

So, I have to face my fear that we're going to need to increase his g-tube or even put the Feeding Clinic protocol back in place.  I know that I must focus on the overall goal of keeping Bennett happy and healthy for the future, not just what makes sense right now.  But it's hard to do as there are so many things we must balance, between Oliver's needs, each of our needs, Bennett's emotional/social needs, etc.

Brian and I would greatly appreciate prayers with regards to wisdom of what to do in this situation.  Our prayer is that the doctors will have helpful suggestions on what to do, and that what they recommend will not be something that jeopardizes other needs we must balance in Bennett's life.  We just want to make the best decision for Bennett, as well as for our family.

It feels like we've been here before.  And we have.  And, I'm afraid, this isn't going to be the last time we're here again, I'm sure.  But each time we're here, it doesn't seem to feel much better.  Decisions are tough.  It's tough to fight this beast that is wanting to swallow up my child.