Orkambi... HOPE

"Aaron Stocks, who participated in a trial for Orkambi, said that's certainly the case for him. The 30-year-old Maryland man said the drug gave him a noticeable improvement in lung function and helped him to gain weight, which is difficult for many cystic fibrosis patients. He recalled going on his normal run not long after starting the drug, only to be surprised to the point of tears when he realized how much easier he could breathe and how much farther he could go."

This quote from an article about today's announcement about Orkambi fills me with so much hope! The hope is welling up inside of me and is getting dangerously close to overflowing. I understand that not everyone on Orkambi has seen these same results, but I can't help but hope that I will see similar results. Actually, I have hope that I might even see a one percent increase in my lung function. To me, that would be amazing! Any gain in lung function is a huge victory for me since I fight every single day for every percentage of lung function I have. My lungs are tired, and my body is tired. I can only get them so far without this extra help. This could be the boost my lungs need to help me live a longer, healthier life. That last sentence in the quote, the one that talks about being "surprised to the point of tears" is something that I want to feel. I can't even imagine what that feels like. I just want the opportunity to feel it.

While it's a huge deal for me that Orkambi was approved today, it is an even bigger deal for all of the children who are fighting cystic fibrosis. With new medications like this being approved, their future is so much brighter than mine was at their age. There is so much hope for them to be able to live with their beautifully high lung function for longer.

Is Orkambi a cure? No. But I believe it is a huge step in the right direction. A cure is coming. I can feel it!

Does that mean we can slow down on fundraising or raising awareness? Of course not! It means that I am going to be doing everything in my power to raise more money and more awareness than I already was! There is so much more work to do! We are getting so close, and we can't slow down now.

If you would like to read more about today's announcement, check out these two articles:
Washington Post
Boston Globe

Frustrated but full of hope

Well, today was my clinic appointment at Vanderbilt. Many of you know how hard I have been working this summer to make my lung function and weight go up. Today is day 94 of 100% compliance with my treatments, and I started doing Insanity last week. I was hopeful for today's appointment. I really wanted to see my numbers go up, but I did not set a goal for my lung function due to being disappointed the last few times.

My lung function today was at 48% which is about what it was last time. I was definitely frustrated, but I have been told by quite a few people that sometimes, it takes longer for your hard work to actually show up in your numbers. Keeping that in mind, I'm trying not to be too disappointed with the lack of movement. I still maintained my lung function which is good, and I still have hope that I will see improvement in the future.

Here's the exciting news: I have been working really hard on gaining weight.... I gained 8 pounds!!! My doctor was thrilled to see that and encouraged me to gain a few more pounds. He was also very happy to hear how hard I've been working and felt that I might see more improvement in my numbers next time.

He said my lungs sounded great, and that's all I can really ask for! :) We also talked a little about the hope that the new medications that are in trials right now offer. I'm excited to see what the future holds for me.

My plan moving forward is to just keep doing what I'm doing. I'm going to continue working out everyday and being compliant with my treatments. I'm going to put everything I have into my health so that I have no regrets. I have so many regrets from my college years when I completely neglected my health, and I am determined not to have any of those for this time in my life.

Hope

This past weekend was such a wonderful weekend! As I have for the past 3 years the weekend before the Valentine's Day, I went to the Cure Finders' "A Special Evening... A Special Cause." It is a fancy dinner and dance complete with silent and live auctions in Pigeon Forge. I always enjoy dressing up to go to this fund raiser, but the best part of the weekend came earlier that day.

The guest speaker for the dinner was Richard Mattingly who is the Executive Vice President and Chief Operating Officer of the Cystic Fibrosis Foundation. Saturday afternoon, they had a meet and greet luncheon with him. Mr. Mattingly spoke about the new developments and research going on with cystic fibrosis. He spoke in detail about where the money that I spend my time and efforts raising have been going. They are dishing out millions of dollars to get major drug companies to actually invest in such a small disease. It is a big risk for companies to take on a disease that affects a total of 70,000 people worldwide, but money talks. And that is exactly what has been working.

This is a clip from an article that appeared in the Wall Street Journal:

"To further entice these small firms, the CF Foundation offered millions of dollars to do the drug discovery work. “We took the risk” so the companies would start and projects would develop the momentum, Beall says. The foundation has now given or committed to give $315 million to companies for drug research."

Here is another one from The Boston Globe:

"The Cambridge biotechnology firm FoldRx Pharmaceuticals Inc. will receive $22 million from the Cystic Fibrosis Foundation to develop and commercialize drugs aimed at treating the fatal genetic disease, a disorder of the lungs and digestive system that afflicts 70,000 people worldwide."

Over the years, the Cystic Fibrosis Foundation has gone out of its way to makes sure that these drug companies have no reason to say no. After the success of Kalydeco, Vertex has begun working on a combination of that drug and another called VX-809. This is a combo built for those with the delta F508 mutation (ME!!!). The CF Foundation has dished out more money to get the process to go quicker. Here is the kicker... Right now, if everything goes well with the trials for Kalydeco and VX-809, they are predicting it to get through the FDA by 2016!!!! There is a lot that has to go right for that to happen, but that is huge! Even if it doesn't work that way, they are on their way to finding something that does work! I have never been so hopeful for my future!


With all of that being said, the money that my friends and family have been so generously giving over the years, has been going exactly where it should. Money is the only way we get closer to a cure, and if you would like to contribute to that, click on the link below or the one to the left.

Good Things Coming

There is super exciting news in the world of cystic fibrosis today! Kalydeco, which is the first drug to actually treat the cause of CF instead of just the symptoms got approved by the FDA! It is only effective for 4% of those who have CF, but it is definitely a huge step in the right direction. Can't wait to see what they come up with next, and I have high hopes that it will be for those of us with the most common gene mutation, delta F508. My hopes are high.

This is the article from the Cystic Fibrosis Foundation. FDA Approves Kalydeco (VX-770) — First Drug That Targets the Underlying Cause of CF - CFF

This is the article from the FDA.

This is the article from Vertex which is the company that created Kalydeco.