Saturday, February 26, 2011

hope.



Last week was a BIG week in the Cystic Fibrosis community! If you have EVER given one dollar to the Cystic Fibrosis Foundation this is what it’s all about. The CURE is near!! Here is the thing, there are different mutations and this drug (VX770) that has the CF community buzzing is aimed at the G551D mutation. Reese does not have this one BUT it is half of the puzzle! This drug used with another on (VX809) they believe is going to fix the underlying defect in 95% of CFers!!! Reese is one of them. The thought that she could be living a more "normal" life in the near future is enough to bring me to tears.
The following information, I got from one of my dearest CF momma friends Sarah's blog. I figured instead of trying to put it into my own words, I would just use the information she shared on her blog (with her permission, of course) as it is stated so clearly and heck, why reinvent the wheel, right?! The information is from an email that she received from another CF mom who sat in on a national conference call regarding this monumental news. All I can say is we have so much hope...


Here is what the mother on the conference call with the CFF shared.


“Phew! What a terrific conference call! SO MUCH INFO! Here’s a recap:The VX-770 trial is now considered a “home-run” for ALL CF PATIENTS! It was done for 48 weeks and patients maintained improvement the ENTIRE TIME (which has never happened in any other study EVER). They targeted G551D patients (all but 1 patient had only 1 copy of it). The reason they selected G551D patients is because the basic defect with them is that their CFTR membrane just needed to be opened, which the 770 does. (They likened it to having a salt shaker that’s clogged and salt can’t come out- the 770 pokes holes in the shaker and the salt can freely come out.) The results of the trial showed 10%-20% improvement in lung function in just 2-4 weeks using the “blue pill”! This improvement was sustained for the entire 48 week trial! The 10-20% is amazing, considering our other awesome drugs such as Pulmozyme, TOBI, and Cayston have showed 3-8% improvement (which we thought was wonderful!). Patients have reported feeling better, breathing easier, and an average of 7 pounds weight gain (compared to less than 1 pound for placebo patients). The pill seems to improve lung function, liver function, and intestinal process since it treats THE BASIC DEFECT OF THE CFTR, not just symptoms as we have been treating. Sweat Chloride levels dropped from avg 100 down to 53 (which 40-60 is Borderline for CF). Hospital stays and need for antibiotics were reduced by 50%! They mentioned that this isn’t the “cure” we are waiting for, but it does dramatically change the game. In a 5-10 year period of time, CF’ers lose about 10% of lung function. This turns back the clock and helps lessen decrease lung function damage, which prolongs their life significantly. It’s too early to say if it will prevent FEV1 decline, but it will certainly cut it by 50-75% hopefully. The drug is being submitted for approval and “fast tracked” so we will hopefully be approved in 6 months. They want to get it to anyone that needs it. Clinical trial patients who were using the Open Label will continue to be on it by signing on to another clinical trial extension. They are also hoping to get all FEV1 patients (less than 40% lung function) with G551D signed on for a clinical trial to use it until it is formally approved. No idea about costs for patients at this time, but they continually said every patient will have access to it. No definitive answers about enzyme adjustments, physio, airway clearance, etc. These all treat SYMPTOMS, which should still be treated, since this pill treats the actual defect. But the need and amount of treatments may decrease. The drug won’t fix scarring already done, but will help stop potential scarring by slowing the rate of decline. They reiterated that a lot of work still needs to be done, and that we are “in the 7th inning of the World Series and just scored a home-run. But the game isn’t over.” This G551D population is 4% of all CF patients. They are continuing to work relentlessly to capture the 96% of other patients who still need help. Dr. Beall said “We shall not rest until our work for everyone is done.” Since this is getting so long, I’m going to comment below this with other tidbits that I heard about, including what this means for DDF508/ nonsense mutations/ and 809 combinations.
They cautioned us to NOT WRITE to FDA to try to get faster approval. They don’t want to barge or coerce FDA, or annoy them. If you want to write a letter, write to your congressmen and ask them to support the FDA by keeping them funded and staffed, increase their budget, and ask them to support faster reviews for ALL drugs, not just ours!
Next, the 770 doesn’t work as well on DDF508 patients, since there’s not enough protein in the membrane to work on. It’s a whole different “fruit” and not able to be compared, other than it did lower sweat chloride levels from 100 to 95 or so. Still not significant, which is why they are now trying 770 with 809.
2 drugs combined is much more difficult to research and “prove” with FDA. Vertex has hired an additional 200 staff to continue research with the combo and packaging the new drug that will be ready for G551D’s. The reason this trial was so successful was due to the tremendous funding and donations that come in from YOU and others- Bill Gates Foundation donated 20 million for it. CFF used 65 million additionally just for this trial.
CFF Board has now earmarked 160 million for “backup” correctors, just in case 809 isn’t the answer. They are expecting the 770/809 trial to have to repeat Phase 2, since there weren’t as big of lung changes as they hoped for.
For other “nonsense” mutations/ “X” mutations where patients are pancreatic sufficient, this 770 is very promising!
Another AWESOME thing the CFF is doing, is a project called CFTR II. They have taken the top 150 disease causing mutations (there are over 1600 CF mutations, but the top 150 make up 95% of all CF population) and they’ve created them in their labs. They are injecting 770 into them and will be watching the outcomes, so then we will know EXACTLY which mutations this will benefit!
No major reactions from patients on the 770, other than a small rash for some, but nothing significant.
For the 770/809 combo, look for 2015-2016 for a possible approval, providing the repeat Phase 2 goes well.
This 770 looks really good for newborns/ infants who are diagnosed with a G551D mutation (or possibly another “nonsense” mutation) and haven’t had lung damage done yet and haven’t started major treatments. Most promising for them since it helps lessen damage and extend the timeline of damage occurring .
They also brought up the PTC 124 trial on X mutations. Enrollment finished in December and the trial also lasts a year. They are VERY OPTIMISTIC and hoping to be celebrating at this time next year for that drug
No data on the effects of 770 on Pseudomonas, other than the hope is that since CFTR function is restored properly, your body will wash it out of the lungs faster and hopefully prevent further colonization. But probably won’t completely eradicate it.
They want everyone to continue participating in clinical trials, including those trials that treat symptoms only. We can’t put all our eggs in one basket and assume that this is it. There could very well be another better drug out there. Beall said “We’re not out of this yet. We still need more shots on goal.” Yes, he is a sports nut. Baseball, Hockey references everywhere! :):)
Phew. I think I got it all. I tried to listen with a sleeping baby next to me and my 2 toddlers wreaking havoc all over my house, so hope I answered everything. OH!! They did list a number for all of you to call with individual questions! They have staffed it with an awesome group who is dedicated to getting your questions answered. The number is 1800-FightCF. You also can submit your questions to ask@cff.org
Oh, and they did reiterate…. TODAY IS A FANTASTIC DAY FOR ALL THE CF COMMUNITY! Biggest breakthrough since discovering the CF gene mutation. ”

Rest assured friends, your donations are making a REAL difference in people’s and family’s lives! We are so excited to see what the future holds for our little Reese and all of the other people living with CF. Our job now is just to keep her healthy until we get our hands on these. Lung damage is irreversible. Our cure is coming!!
As the Latin quote says, "Dum spiro, spero," "While I breathe, I hope."

Lovin' Life

...Life cereal, that is :)





But, we are also lovin' life in general as well. Lots of action is starting to take place as we are gearing up for a few different fundraisers and our Great Strides Walk and also a certain little missy's 2nd birthday!!!! I will update you about all of that soon, I promise!
On another note, winter is getting soooooooooo old around here! It seems like every other day we are getting more snow. GRRRRR! Don't get me wrong, I usually am not so pessimistic about winter, because there are parts of it I really do love, but with an active little toddler, it's very difficult to spend our days indoors because of frigid temps. We have been getting out a bit going to storytime and hitting up playdates, but I think it's safe to say that everyone around here is more than ready for Spring. Bring on the sunshine and warmer temps!!!

Thursday, February 17, 2011

My Sweetheart

My little Valentine sweetheart.

FYI...this was pretty much the only picture where she was looking at the camera.

Friday, February 11, 2011

Flashback Friday


My sweet chubber-cheeked little Valentine last year. Oh how I heart her.

Wednesday, February 2, 2011

Wordless (or not so wordless) Wednesday


This picture really just deserved some words...this is our darling daughter helping take down our Christmas tree...with my...ahem...undergarment around her neck! I obviously didn't place the aforementioned item in its proper place and Reese got her hands on it and thought it would be a fun necklace to wear around the house...which she proceeded to do for a good portion of the morning. Oh dear. That girl is tooooooooo funny!!!!
PS...notice I have posted 3days in a row?!?! Record breaking material for me!
PPS...the schools are closed here in our city today due to FRIGID temps...that NEVER happens!!!! BRRRRRRRRRRR!!!!

Tuesday, February 1, 2011

Baby It's COLD Outside!!!

Let me just start off by saying that tonight, the air temp here is supposed to be -30...that's right, you read right...NEGATIVE 30 DEGREES! Then add on the windchill and it could get as low as NEGATIVE 50 DEGREES! Plus, we just got about 6 more inches of snow dumped on us! Geesh...we need to move! I am sooooooo over winter right now! Anyway, because it's been so darn cold, Reese and I have been catching some MAJOR cabin fever. We needed to get OUT OF THE HOUSE so decided to hit up the Kirby Science Center at the Washington Pavilion recently. Our friends Emily and Ruthie gave us a pass there for Christmas and so we thought it would be fun to take the girls on an outing. Now, if you know me well, you know I'm a bit of a germ-a-phob and have been pretty reluctant to take Reese out too much due to the nasty cold and flu season, but quite frankly, we needed a change of scenery! Luckily it wasn't too busy and the girls were able to kind of run and explore without having to fight through a crowd of kids. I think both Reese and Ruthie loved it and I'm excited to go back!










The hot air balloon was Reese's favorite part. She's obsessed with balloons. Hey, who isn't really? :)