Yes! Such exciting and positive news announced last week about the clinical trials that are going on for Dean's mutation.
Here is the link to the article and I'll summarize the excitement:
A little science to explain what is going on...Cystic fibrosis is caused by the mutation to the CFTR gene which hinders the transfer of water and salt across cells affecting both the digestive and respiratory systems. To manage the digestive system properly, Dean takes pancreatic enzymes before snacks, meals and milk since his pancreas doesn't produce the enzymens needed to digest fat. On the respiratory side, oatmeal-like mucus is produced in the mucus producing organs and since last August, we've been giving Dean daily respiratory treatments to prevent a buildup of mucus in his lungs as well as thin any mucus that is making a home in his lungs. We do this to help him breathe easier and because bacteria like to breed in warm, sticky environments and we're trying to prevent the bacteria from causing any damage to his lungs. So, back in January, the FDA granted approval for Kalydeco, which is the first drug for CF that doesn't treat the symptoms but rather treats the disease by targeting the CFTR gene by helping to open the cell "gates" to allow better transport of ions across cell membranes. Dean's issue though is two fold. Not only do the cell gates not open but the protein can't even get close enough to the cell surface to get out! So, the Cystic Fibrosis Foundation partnered with Vertex and developed a drug (now called VX-809) that they intend use to get the CFTR protein to the cell surface, where Kalydeco can then work it's magic, open the gate and allow for the correct flow of ions. On Thursday, final results from the phase 2 clinical trial of the combination of these drugs were released which showed significant improved in lung function for patients that had the same mutation that Dean has. Lung function is measured by how much air can be expelled from the lungs in 1 second. Although Dean is too young to get measured for this now, these trials in CFers 18+ with the same set of mutations showed very promising results. So, the next step is the pivotal phase 3 trial which will be the mose rigorous evaluation of the safety and efficacy of the combination of these 2 drugs in patients who have the double deltaF508 (in preparation for another FDA filing).
We are beyond hopeful and prayerful that God guides these scientists to continue making the future look bright for our son and so many others with this disease. We are so thankful for all of your support and wanted to explain what $75million of research dollars can do for one little man and 30,000 other people in the United States alone. It's the passion of the people that care so much to do everything to help us raise money that is allowing this to happen. As a "orphan" disease, meaning one given very limited federal funding, the Cystic Fibrosis Foundation is at the mercy of private donations and the families and friends of those affected to raise money. We truly won't stop until CF stands for Cure Found. Thank you for helping us. Our next fundraiser for Team Eat. Pray. Dean, Chillin' & Grillin' for a Cure is on July 14th from 3-10pm. Please say you'll come and be a part of this amazingly huge part of our lives. We thank God for you and for your kindness and your prayers.
With love and appreciation,
Jim & Erin
check out http://www.eatpraydean.com/ for the schedule of events on July 14th
email us at firstname.lastname@example.org if you will join us and need a link to the evite
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|Dean 2 years old|
|the awesome kids that will do anything to help Dean-o|
"God is not unjust; he will not forget your work and the love you have shown him as you have helped his people and continue to help them.” ~Hebrews 6:10 (NIV)