He went over lots of good info, mainly including 2 drugs that are soon to be approved by the FDA and available to patients. These 2 drugs take care of the root of the problem in people with Cystic Fibrosis. They are called the Potentiator VX-770 and the Corrector VX-809. These drugs have the potential to add 10-15 years of life to patients with CF!!!!
Last Thursday 11/12/09, Chris and I went to a Cystic Fibrosis Volunteer Appreciation Reception. The special guest speaker was Robert Beall, the President and CEO of the Cystic Fibrosis Foundation! Exciting, right?!?! I know! It was such an honor to meet him and shake his hand. It is thanks to his dedication and the doctors and scientist hard work that my son's life expectancy is 37 instead of 5!! After the reception, I went up to him and asked to get a picture with him, and then we talked for a minute and I told him about my blog and how I talk to CFers all over the world! He seemed impressed for me only having a 2 year old with CF and doing so much already:)
The Potentiator VX-770 is in Phase 3, the Definitive Trial, and the Corrector VX-809 is in Phase 2, the Human Safety and Efficacy Trial. Drugs have to go through 3 phases which costs millions of dollars to do. Thanks to the money we raise through Great Strides, the Cystic Fibrosis Foundation can fund these drugs. Lil' Chris wouldn't be as healthy as he is today if it weren't for the CFF. Currently there are 30 drugs in the pipeline. Only 2 up until today have been approved, and I personally know many CFers that use TOBI. There are 13 drugs to be approved by the FDA within the next 3 years!!! Good news!! 4 of those drugs are in the last stage, phase 3. Great news!!
What are so special about the VX-770 and VX-809 drugs?? They could stop the disease at the cellular level. The basic defect in those with CF is that the chloride can't get out of the cell, CFTR. In a normal cell, proteins are transported to the outside of the cell, allowing chloride to flow in and out of the cell(see pic above). In a CF cell, nonworking proteins are transported to the outside of the cell and don't allow chloride to flow. Instead a thick, sticky mucus is formed. Robert Beall said, "Can't we just find a pill that will open up these chloride channels?" Well, the VX- 770 and the VX-809 do!!!
The VX-770 increases the opening(gating) of the CFTR channels with just a pill!! Right now is it working for those with G551D mutations, but Beall believes that in time it can work for other mutations as well. In a CF cell successfully treated with VX-770, the drug binds to the nonworking proteins on the outside of the cell, allowing chloride to flow and prevent mucus from forming. The VX-770 increased CFTR activity in Preclinical studies, and after a 2 week clinical trial, they were at normal levels!! Amazing! Their lung function improved 12%!! They are doing this study on children as young as 6 years of age and up! I didn't know they did these kinds of studies on children so young!
The VX-809 increases the number and function of CFTR channels. It is for those with double delta F508 mutations, which is what Lil' Chris has. The VX-809 helps CFTR to get to the membrane and helps get chloride through. These clinical trials are for those who are 18 and older. This one is very similar to the VX-770. Instead of increasing the opening so the chloride can flow, it makes a NEW opening to let the chloride flow. They do the same thing, but just in 2 different ways.
Robert Beall told us that "CF patients loose 2-3% of lung function EVERY YEAR. There is an 80% chance of drugs working when in phase 3. These drugs could add decades to CF patients lives! This is the most exciting time in our history, and we are closer than ever to treating the basic defect in CF!"
We need to pray, pray, pray that we raise enough money to keep these drugs moving forward to find the CURE for Cystic Fibrosis. These drugs could potentially be the CURE for Lil' Chris and other young CFers. It may not be the cure for older CFers, b/c it can't undo any lung damage that has already happened. But it can help it from getting any worse, which could still increase their life expectancy. Also, a lot less pills and treatments which is a HUGE PLUS!!! This all equals to a better quality of life:)
Other exciting news that he shared with us, is that they are teaming up with Warner's Brothers and CVS. CVS will promote movies that benefit Cystic Fibrosis! This will be huge awareness for the public! The more awareness there is about CF, the faster we will get to a CURE:)
Click here to read more about these drugs and to read about a fellow blogger I know, Somer Love, who is 30 with CF. Click here to read her blog.
Click here and donate today and do your part to help these drugs move a lil' faster to becoming Lil' Chris' CURE!
Click here to join a CF Panel if you are a CFer or a caregiver of a CFer and want to help find a cure for CF and get PAID to do it! Don't forget to mention my name;) Thank you to Traci Liberto for being the last one to mention my name:) A few days ago they called to update my info and I got $25! YEA!!! So far, I have earned $475 for basically doing nothing!! It's easy money to raise for Great Strides for a CURE;-)
With your help, CF will stand for CURE FOUND!!