Amazing Things

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Lately, I feel like life has been treating me ridiculously well! I fell so blessed! To begin with, I am on day 10 of being 100% compliant with my treatments. I’m also working out as much as I can at the gym and at home. I’m making sure to do a little bit of everything including cardio and strength. I’m on the road to higher weight and lung function!

Yesterday, at work, I had my final evaluation. It was unannounced and slightly stressful, but I am finished with evaluations for the rest of the year! We have two unannounced and two announced throughout the year, and I am so thankful to have all of my evaluations for my first year of teaching behind me!

Finally, Vertex (the company that gave us Kalydeco), announced yesterday they will be starting Phase 3 of their trials of Kalydeco and VX-809 together. The trials are being done with cystic fibrosis patients who have two copies of the DF508 mutation (the most common mutation in those of us with disease). I just happen to have the double copy of that mutation! They have said that if everything goes well, they hope to send it to the FDA for approval in 2014! I could not be more excited about this news and what it could mean for my future as well as thousands of others with the same mutation! This is why I work so hard to raise money for the Cystic Fibrosis Foundation every year.

With all of that being said, I am starting my fundraising campaign. If you are interested in making a donation to the Cystic Fibrosis Foundation, just click on the link below. I will also be selling t-shirts once again. If you would like to order a t-shirt, just let me know what size you need. I charge $20 a shirt. The more that are ordered, the cheaper they are. So any extra money I get from the shirt orders, will go straight to the Cystic Fibrosis Foundation. The shirts come in sizes YXS to 3XL.

 If you would like to read more about the Vertex announcement, just click on the links below.

Vertex Press Release

Cystic Fibrosis Foundation

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.

Exciting Times

So, not that long ago, I posted an article about the new drug that they have been testing that actually treats the cause of CF. Now, that drug only works for about 4% of the CF population, but it is a huge step in the right direction! Today, we got the amazing news that it has been submitted to the FDA for approval!!!! Here is the article:


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.

This article is from the Cystic Fibrosis Foundation's website. You can find more information here:
Vertex Press Release
FAQ's about KALYDECO
FAQ's about the combination of the 2 medicines

Anyways, thought I would share the very exciting news with you all! And by the way, I have very high hopes for the KALYDECO combination with VX-809 because Delta F508 is the mutation that I have.... So let's keep our fingers crossed and our prayers ongoing! :)