Wednesday, February 15, 2012

Blue Lightning: Partnerships Keynote Emily Schaller on the approval of Kalydeco

At the 2012 Partnerships in Clinical Trials Event, Emily Schaller will be presenting Beating the Odds—Not Only Living with Cystic FibrosisRocking It.On Tuesday, January 31, she received word that the drug she participated in a clinical trial for, Kalydeco, was approved by the FDA, nearly three months ahead of the deadline.  It's the first drug to be approved for the treatment of CF, and targets the root cause of the disease, and specifically targeted at the G551D mutation.  The New York Times has more information.

Upon it's approval, Emily took the time to share her feelings about the approval with the Partnerships in Clinical Trials Community:

I remember exactly what I was doing when I heard that the newest drug for the treatment of cystic fibrosis was approved. A tweet from a friend came to me at 10am on that Tuesday announcing Kalydeco's approval and we were to celebrate the following week when I would be in New York City. Developed by Vertex Pharmaceuticals, Kalydeco, which I named "Blue Lighting" last year is the first and only drug that treats the underlying cause of cystic fibrosis. 

Why Blue Lightning? Well Kalydeco happens to be a little blue pill and I was on the phase 2&3 clinical trial for this drug which at the time was called VX-770. After a few days of starting phase 3 I noticed some incredibly positive changes in my health. This struck almost as fast as lightning. The changes that happened are things I never imagined possible given my life with cystic firbosis I could breathe. My cough was almost obsolete and I felt like a million bucks, a way I can never remember feeling in all my life. 

The approval of this "designer drug" is earth shattering and will change lives like it has mine and the other who participated in these clinical trials. Kalydeco treats a mutation that only 4% of the CF population carries but is opening the doors and paving the way for drugs that will treat the other 96%. We know Kalydeco works and it works extremely well, so everyone must remain hopeful even if they do not qualify for this drug. The CF Foundation, researchers, scientists and pharmaceutical companies are working tirelessly to make sure that everyone will be able to control CF with a pill one day. I cannot wait to get another tweet telling me that another drug is approved that will treat the rest of my CF friends worldwide!


Emily will be telling her story on March 5, 2012 in Orlando at Partnerships in Clinical Trials. She will be joined by Dr. Peter Mueller is CSO, Executive VP-Global Research & Development at Vertex Pharmaceuticals, Inc, who worked closely with the the drug in order to get it approved by the FDA.  If you'd like to attend this session, register to attend Partnerships in Clinical Trials today. Also, as a reader of this blog, mention code XP1700BLOG and receive a discount of 15% off the standard rate!




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