Our old CF Commercial that plays on MCTV. Still working on this years.

Friday, June 29, 2012

Learning About CF & New Drugs for DDF508 & 2 new local CF families

I got some great news and some not so great news...

The great news is that a Phase 2 clinical trial of Kalydeco in combination with VX-809 showed significant improvements in lung function in people with the most common CF mutation(Delta F508), which Lil' Chris is a double Delta F508!! This is it people!!! This could be our answers to pray!!

If you remember me talking about Kalydeco only working for those with mutations G551D which is only 4% of the population, well VX-809 is what helps the DF508's, so with the combination of them both...the possibilities are hopeful!!! :-)

Here is a video that a fellow CF mom put together to help us all understand what these 2 drugs really do...(note, VX-770 is what Kalydeco used to be called)...

Below is what the CF Foundation posted yesterday about this trial...(click here to learn more)

June 28, 2012
A Phase 2 clinical trial of Kalydeco™ in combination with VX-809 showed significant improvements in lung function in people with the most common CF mutation, according to final results announced today by Vertex Pharmaceuticals Inc.
Both Kalydeco and VX-809, a CF drug in development, are designed to treat the root cause of cystic fibrosis. Vertex developed Kalydeco and VX-809 with significant financial, clinical and scientific support from the Cystic Fibrosis Foundation.
The 56-day study enrolled 109 people, ages 18 and older, with one or two copies of Delta F508. People in the study with two copies of Delta F508 (the most common CF mutation) who received the highest dose of VX-809 combined with Kalydeco showed the greatest improvement in lung function. Vertex released preliminary results from the Phase 2 trial earlier this year.
Based on these final results, Vertex plans to begin a pivotal trial of the combination treatment in people with two copies of Delta F508 in early 2013. Pivotal trials typically aim to gather data that the U.S. Food and Drug Administration (FDA) could use to decide whether to approve a potential drug.
“The improvements seen in lung function are very encouraging, and we are pleased that Vertex plans to move forward quickly with a pivotal trial,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “We still have significant work ahead of us, but the entire CF community can take pride in its role in making this important step possible. We thank the trial volunteers and their families, clinicians and scientists, and our dedicated volunteers and donors across the country.”  
Participants with one copy of the Delta F508 mutation also showed improvements in lung function, compared with those who received a placebo — though smaller than the improvements seen in those with two copies of Delta F508. Vertex said it plans to conduct additional studies of Kalydeco and VX-809 in those with one copy of Delta F508.
About 50 percent of people with CF in the United States have two copies of the Delta F508 mutation; an additional 40 percent of people in the United States have one copy.
The FDA approved Kalydeco in January 2012 for people with the G551D mutation of CF ages 6 and older. About 4 percent of people in the United States have the G551D mutation.
People with CF and their families who have questions about the Phase 2 results can contact Vertex Medical Information at 1-877-634-8789.

Isn't that GREAT news!?!

Now for the not so great news... I recently found out that there are 2 more kids that have gotten diagnosed with CF in our area:'(

One is a cute lil' 9 year old boy. I first found out about him through The Faithful Little Cupcake facebook page. They posted a pic of him doing his Vest while eating one of their yummy cupcakes. So of course I asked if he had CF once I saw the Vest, and of course asked...does he live in Wooster? Then just a coincidence that my friend Stacy A. with Broncheictasis (a lung disease similar to CF) was at a pool party and found out that there was a child there with CF. She of course then talked to his mom and let her know that she could not be near him since she has cultured Pseudomonas. She told his mom about me and surprisingly she already knew about me! She called me "The Famous Michelle who does the CF walk" or something like that :) LOL!! So she requested to be my friend on Facebook and I'm so glad so I can help her out :) Come to find out, she is the same mom from the Faithful Little Cupcake page! Small world:)

The other one is a little girl who was born a couple of months ago. Her sister was in Lil' Chris' preschool. When she was born, they thought there was a mix up with the results b/c the parents aren't CF carriers. The sweat test came back positive though and they are starting treatment today. The parents are going to get re-tested. A girl I used to work with and used to babysit for us, Michelle V., was the one who told me she has CF and recommended I be her friend on facebook. I had no clue who it was until I went to her page on facebook. Since school let out, I've been wondering if she ever got the sweat test done and what the results were. I'm glad Michelle put us together so I can help them out the best that I can.We started Lil' Chris' treatments at just 2 weeks old, so I know what it's like to try to feed an infant with CF and do manual CPT.

I wish these families all the best and I promise I will not let up on finding a CURE!!! I do all this fundraising not just for Lil' Chris, but for ALL current CF families and ALL future CF families!!! WE WILL MAKE CF STAND FOR CURE FOUND!!!

BTW, all my local CF moms who are probably panicking right now...the little boy above is home schooled :) For those who don't know, schooling is so hard for CFers. It was a big decision for us with Lil' Chris. CFers can't be near each other, b/c of cross-contamination, they could spread the bacteria's in their lungs to each other. A nonCFer can't catch it, but to someone who has another lung disease it can be very dangerous. For example, you don't want an older CFer around a younger CFer in fear that the younger one would catch the bad bacterias that the older one has accumulated over the years. 

For Lil' Chris, we had to make a big decision b/c the local public schools had CFers at each one and the one school that didn't...well, it's very old and full of mold which is not good either for a child with a lung disease. So we decided to send him to the local Christian School. Honestly, I'm glad, b/c I've always wanted my kids to go to a Christian school, b/c I loved going to a Christian school as a kid and I don't think I would be the same Christian I am today if I didn't. There are no CFers at this school, but the sister of this newly diagnosed baby girl will be going there. She will be in the all day Kindergarten and Lil' Chris will be in the half day Kindergarten, so next year they will probably be in the same class. We thought we would start him out slow, plus it means one more year of possible less germs to catch, oh and we totally fell in love with this teacher! She knows some friends with CF, so she is aware of how being germs cautious if very important. Anyway, even though they will be in different classes this year, we will still have to be careful that our 2 CFers don't get too close. They say 3 feet is ok, but I've just tried to always keep him far away from any CFer to be safe. So we'll just have to be extra cautious if we go to field trips together or something.

We got back his last throat culture results and it was the same as usual...Staphylococcus areus sensitive to Oxycillen(MSSA). No changes in treatment. YEA for no bad bacterias!!! WOOHOO!! 

Here is a pic of Lil' Chris doing his Vest and holding a pic of his buddy, Spencer, who is serving in the Army right now and was in the Daily Record Newspaper the other day:) GO SPENCER!!

Please continue to pray for good culture results and good lung function and please keep the above new CF families in prayer. They are going to need the strength! Also, don't forget about Phennyman, he is in need of surgery. Lil' Chris' cousin Nathan with Leukemia is visiting from NJ right now and he is doing GREAT!! Praise the Lord!! Thanks so much for all your prayers!

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