Happy Halloween

I know Halloween isn't until tomorrow, but I had a few minutes to sit down and share some photos from Ella's Halloween party at school. Ella is dressed up as Jessie the Cowgirl from Toystory 2 and the twins are garden gnomes. I seriously couldn't stop laughing at how ridiculous (and cute) they all looked in their costumes.

We were all set for an uneventful, relaxing weekend when Lily decided Saturday morning to drink half a small bottle of hand sanitizer. I immediately called poison control who said she needed to be taken to the hospital to be monitored, since its 62% alcohol. I was also advised that she might start to stumble, slur her speech and potentially pass out (none of which ever happened, thankfully). Martin took her to the ER where they drew blood and kept an eye on her for a few hours. Her blood alcohol level came back normal so either she didn't actually eat as much as we thought she had, or she's been up to this before and has built up a tolerance (kidding!). Very luckily, she was fine and we were able to carry on with our weekend.

In other very good and exciting news, Drew's latest culture came back NEGATIVE for pseudomonas! Hopefully it remains that way for a while. He did have some Haemophilus but they are going to treat that unless he shows symptoms of being sick, which he is not. Hopefully he can cough that out on his own without antibiotics.

Tomorrow night we will all bundle up and brave the cold for some trick or treating. I don't suspect it will last long but hopefully everyone will enjoy themselves for the short time we spend outside!

a little bird told me...

A Daily Truth from The Brave Girls Club

Life is a trip with a perfect itinerary that we cannot see. Imagine it this way -- that there really IS an itinerary, but you just can't see it. There is an amazing destination, but you just don't know where the stops along the way will happen where you will have to refuel, where your best memories and your greatest lessons will happen.

We don't know that what may seem like useless detours are where we might meet the most amazing people of our lives, or enjoy the best scenery, or learn the most important things.

Sometimes we fail to enjoy the trip because we are so hung up on wanting to see the itinerary. You will have the best times of your life when you just TRUST that you are exactly where you are supposed to be, doing exactly what you are meant to be doing, and that tomorrow you will be lead to the next destination.

You always have been, you always will be.

Remember to travel light, only take what you need with you. It will make things so much easier.

ENJOY YOUR FLIGHT, little birdie!

Clinic Visit

Not much going on around here these days. The weather has been really nice and we've been spending tons of time outside. Its supposed to get cold now, just in time for Halloween. I'll definitely have to post some pictures after that because the kids costumes are CUTE!

We had a clinic visit on Monday and are hoping that we get a good culture back later this week. Hopefully our pseudomonas is gone again and will stay gone this time. Drew's overall health looks good. I got to see his CT scan that the doctor still says shes very happily shocked about. Everyone kind of just expected things to not look good with all the trouble that he had over the past year, but alas, it looked just fine. They said that sometimes in younger kids there is some evidence of bronchiectasis in the upper lobes but he doesn't seem to have any signs of that whatsoever. There wasn't any signs of lung damage and everything looks just the way its supposed to. I'd like to keep it that way.

Drew is currently on a hunger strike. I haven't figured out what he's trying to prove, but the boy won't eat. Ok let me correct that - he won't eat anything but fruit and an occasional corndog. The boy who use to love peanut butter now steers clear of it. He won't eat chicken nuggets, corn dogs, hamburgers, pasta, nothing. His weight isn't down (thank God) but i'm afraid we're headed there. I hear that toddlers go through these stages. Its just a little more challenging for a toddler who needs fat to thrive. We might be turning to smoothies soon.

I'll post an update when we get Drew's culture back, probably on Friday. Until then, I hope that our lives remain uneventful in a good way!

What If

A message from the Public Policy Department of the CFF

What if many people with cystic fibrosis lost the ability to go to their care center? What if they could no longer afford their treatments? We need your help to make sure that the thousands of people with CF who rely on Medicaid never have to answer these questions. Twelve members of Congress could limit the ability of many in the Medicaid program to access their care. Your voice can help ensure that doesn’t happen. Many families in the CF community would struggle to afford care without Medicaid protections. Speak out now! This summer, Congress created a new deficit reduction “supercommittee.” Its 12 members were given broad powers to change the Medicaid program. Medicaid provides health coverage for children and adults with CF who cannot afford other types of insurance. It is often the last resort to make sure they are able go to a CF care center, see a doctor with expertise in CF and afford their inhaled antibiotics, nebulizers, enzymes and other treatments. Supercommittee members need to cut the country’s budget deficit, but it’s up to us to make sure they protect access to the specialized care people with CF need to stay healthy. Help spread the word: Are you on Twitter? Please retweet the CF Foundation’s messages in the coming weeks to each member of the supercommittee. Not on Twitter? No problem! Send a message to all 12 members of the supercommittee now. Thank you for all that you do! Together, we are making a difference in the lives of people with CF.

Great news for the CF Comminuty

Vertex Submits Application to FDA for Approval of VX-770 – First Potential Drug to Target Underlying Cause of Cystic Fibrosis

October 19, 2011

Vertex Pharmaceuticals, Inc., announced today it has submitted an application to the U.S. Food and Drug Administration for a potential new CF therapy, VX-770 — under its new proposed trade name, KALYDECO™.

If approved, it will be the first drug on the market that targets the underlying cause of cystic fibrosis. Therapies available to people with CF to date only treat symptoms of the disease.

The company is seeking approval for the drug in people with cystic fibrosis age 6 and older who carry at least one copy of the G551D mutation of cystic fibrosis.

KALYDECO (kuh-LYE-deh-koh) was discovered in a collaboration between Vertex and the Cystic Fibrosis Foundation, which provided substantial scientific, financial and clinical support throughout the development process.

“The CF Foundation is thrilled that KALYDECO is on track for possible FDA approval in 2012,” said Robert J. Beall, Ph.D., President and CEO of the CF Foundation. “This is a significant step forward in our collaboration with Vertex and is further validation of the CF Foundation’s drug development strategy. We remain committed to accelerating the development of similar targeted medicines that will benefit all people with cystic fibrosis.”

Vertex has asked the FDA for priority review of the potential drug, which, if granted, could shorten the review from 10 to 6 months. The FDA grants priority review status for several reasons, including in situations where a potential drug is considered a major treatment advance.

Results released earlier this year from Phase 3 clinical trials of KALYDECO in people with the G551D mutation of CF showed that those receiving the drug had remarkable and sustained improvements in lung function and other key symptoms of the disease, compared with those on placebo.

As FDA review of the potential drug gets underway, Vertex has set up a program to provide KALYDECO to people age 6 and older with the G551D mutation who are in critical medical need and could benefit from the treatment prior to potential approval.

The expanded access program is designed for people with CF who have highly limited lung function and meet other criteria. (Information about the program is available at CF Foundation-accredited care centers.)

KALYDECO is currently being evaluated in combination with another oral drug in development, VX-809, in people with the most common mutation of CF, Delta F508.

Vertex plans to begin the second part of the Phase 2 KALYDECO and VX-809 clinical trial this month and will evaluate the two drugs over a longer period of time.

Additional Resources

• Read the Vertex press release.
• Read about KALYDECO in these FAQs.
• Learn about the combination trials in these FAQs.

Clarity

After I slept on it, I had some more thoughts on last nights post. First, our situation is not the same as the woman who wrote that article. My heart breaks for her. While I have shared many of her feelings and emotions, we do have hope. We have treatments and therapies that will hopefully prolong Drew's life. We have a future planned. We have plans and ideas and goals, and hopefully will be able to fulfill them all. There is no cure for CF, but there is hope. I hope that Drew lives to be 95 before it "gets him". Its scary and its hard some days, but I try to focus on the positive. We do try to cherish every moment and every memory, but I did that before I ever had Drew too. Our house is a mess. There are usually dishes on the counter and mounds of laundry all over the house. There are also usually 3 kids running around, sometimes naked, squealing with delight and making a giant mess. I try not to let the little, insignificant stuff bother me.

I feel like sometimes people are a little afraid of me. I've had people comment that they shouldn't even talk about their crazy day because its probably nothing compared to mine. We all have our own crazy. People either don't want to talk about CF because they don't understand, or because they're afraid they have to hop on an emotional roller coaster named Erin if they bring it up on the wrong day. I don't always want to talk about it. I deal with it every day, some days better than others. Some days I don't mind when people ask me questions about it and about things. So few people really understand and I get frustrated because I feel like they haven't taken the time to understand. Other friends have distanced themselves. They have no clue what goes on. I don't know why they've left us, but they have and I've become okay with it. I use to get mad when people didn't "get it", but with time I've come to realize that its not their life to get, its mine. As friends, I thought that they would share this with us, but whether they are scared or confused or simply don't understand our situation, they have chosen distance as their mechanism, and I'm okay with that. I've got who I need in my life.

I think that mostly I've settled into our life the way that it is. I only get worked up when I hear a sad story or hear bad news or just have a bad day...just like the rest of us. I do have lots and lots of hope, but it doesn't mean I'm not scared sometimes. I'm not naive to what this disease can and will do to a person, but I'm optimistic. The perspective that the woman who wrote "Notes From a Dragon Mom" provided in her article was emotional, eye opening and inspiring. This is what I felt I shared with her, as the mom of a kid with a disease with no cure:

"Our goals are simple and terrible: to help our children live with minimal discomfort and maximum dignity. We will not launch our children into a bright and promising future, but see them into early graves. We will prepare to lose them and then, impossibly, to live on after that gutting loss. This requires a new ferocity, a new way of thinking, a new animal. We are dragon parents: fierce and loyal and loving as hell. Our experiences have taught us how to parent for the here and now, for the sake of parenting, for the humanity implicit in the act itself, though this runs counter to traditional wisdom and advice."

Different from the author, I have not abandoned the future. I am hopeful for Drew. I'm just a mom who is truly, madly and deeply in love with my son who fights a disease for which there is no cure every day. But I'm hopeful. That's why I fight for him. I'm hopeful.