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Wednesday, February 23, 2011

CF Update / Positive Results for VX-770 Study!

Lil' Chris finished his Bactrim last night for his cough, but of course....he is STILL coughing!!! Arggggg!! So I called the CF clinic today and left a message. This is the first time this has happened, so I'm curious as to what they are going to do...maybe refill the Bactrim, or wait a little to see how he does, or wait and then put him on something different??? IDK. Do they even HAVE to wait?? I guess I'll find out when they call me back. Hopefully they call today!! His cough isn't super bad or all the time, but when he does cough...it doesn't sound the greatest. It just makes you sink and wonder what's going on in those lungs of his. I just pray it's not "the bad bacteria"...Pseudomonas.

Oh, and he also had another bad night on the 19th where he was throwing up again. We don't think he had any soy protein, so not sure why he keeps doing this. That makes 3 times in the last couple months I think. I wonder if we need to adjust his enzyme intake. I'll be asking the dr on 3/16 when we go for clinic visit. The pic above is him in between throwing up. Notice the trash can right next to him?? lol! If it's right next to him, he never gets it on the floor. He's pretty good about knowing when it's coming and when to get to the trash can. He's had enough practice, that's for sure. Poor kid.

Our snow FINALLY almost all melted a few days ago when we had like 60 degree weather, but that didn't last long!! We got hit again on Monday! Of course I had to drive home from work at the worst time!! But...I lived to tell about thankfully!! It was about 4-5 inches on top of 1 inch of ice and coming down hard! Cars were sliding all over the road, including me! Unfortunately, any way I go to my house there is a hill going down and up;( This time the roads were so bad that I started sliding down one hill sideways. I thought I was going to slide into the cop car at the bottom that was blocking the way coming up the hill I guess b/c cars were just sliding down it. Crazy, Crazy, but I made it home:) The Angels guided me the whole way!! Thank you Lord!! The pic above is the next day after my hubby snow plowed:) Thanks Honey!!

Now the following is some really encouraging news from the Cystic Fibrosis Foundation(what your donations for Great Strides fund). Lil' Chris' mutations are Double Delta F508(most common), but it's still promising for him...(click here for the whole article)...


Phase 3 Study of VX-770 Shows Positive Results

February 23, 2011

The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis, showed promising results in a Phase 3 clinical trial.

The trial was designed to evaluate patients age 12 and up who carry at least one copy of a CF mutation called G551D. The study included 161 patients who received at least one dose of VX-770 or placebo.

Patients who took the drug, compared to those on placebo, showed a marked improvement in lung function at 24 weeks, which was sustained for the duration of the 48-week trial.

Patients also showed improvement across all key secondary endpoints in the study, including reduced likelihood of experiencing a pulmonary exacerbation, decreased respiratory symptoms and improved weight gain. Each of these areas is critically important to the health of people with CF.

In addition, average sweat chloride levels of patients on VX-770 dropped toward normal levels, while those on placebo did not change — indicating the drug is impacting the underlying defect in CF. Excessive sweat chloride (salt) is a key clinical indicator of CF.

VX-770 is being developed by Vertex, and was discovered in collaboration with the CF Foundation, which provided substantial support to Vertex throughout the development process, including an approximately $75 million investment.

About four percent of people with CF carry the G551D mutation. More studies are needed to determine whether other CF mutations may benefit from VX-770.

“These results are highly encouraging. They provide scientific evidence that support our long-standing belief that targeting the underlying defect of CF may have a profound effect on the disease,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “We have much more to do to end the suffering caused by this disease, but these data are extremely exciting, especially for people with the G551D mutation and their families. The results also offer significant hope that a similar approach to treatment may help the majority of patients living with CF.”

The Phase 3 data support Vertex’s plan to submit a New Drug Application for VX-770 to the U.S. Food and Drug Administration (FDA) in the second half of 2011. Generally, the FDA takes between 6 and 12 months to review and rule on a drug application.

“The Cystic Fibrosis Foundation has played an instrumental role in our more than 10-year effort to discover and develop potential new CF medicines such as VX-770,” said Matthew W. Emmens, chairman, president and CEO of Vertex. “The data announced today reflect a significant investment of time, dollars and scientific expertise from both Vertex and the CF Foundation, and we look forward to working closely with the Foundation as we seek to bring VX-770 to people with CF.”

Said Preston W. Campbell III, M.D., executive vice president for medical affairs of the Cystic Fibrosis Foundation: “As a physician who has treated CF patients for many years, the VX-770 results are more than just numbers — they represent hope.” He added, “It’s not surprising that patients felt better on the drug because of the magnitude of lung function improvement and weight gain. These are important clinical outcomes, and the fact they were maintained through 48 weeks is very encouraging.”

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