Thursday, December 29, 2011
Friday, December 23, 2011
Wednesday, December 21, 2011
There were generally fewer people than normal there when I was there tonight. I think that's awesome, given that Christmas is just 4 days away. Most of the folks that I had known all went home on good terms, and I can only hope that the ones that I didn't know so well also left on a high note. We spent one Easter in the NICU and it was definitely a bummer holiday, so I can't imagine having to be in there for Christmas. But I know that the kids in there need to be in there and are getting the best care they can.
We heard some great news from the Cystic Fibrosis Foundation again today. I got the following email from one of the Public Policy Directors:
I’ve got some great news to share. As you may know, late last week Congress passed the final FY 2012 Appropriations bill - or “omnibus” - at long last completing the budget process for the current fiscal year. Thanks to your hard work, the CF Foundation has achieved three of our main policy priorities in the budget, no small feat considering what a contentious year this was on Capitol Hill.That really is all great news for CF. I wanted to thank everyone who participated with me in clicking on links I provided through my blog and contacting decision makers to let them know how important all of this stuff is to us. We can and did make a different and I know that I will certainly continue my efforts going forward knowing that!
1) As we told you a few weeks ago, funding for the Food and Drug Administration was increased by $49 million in the 2012 budget. This boost will help reviewers to more quickly move important treatments from the lab to the patients who need them.
2) I am happy to report that the National Institutes of Health (NIH) will receive a $300 million funding increase in Fiscal Year 2012, providing resources to advance innovative biomedical research for diseases like cystic fibrosis.
3) Congress established the National Center for Advancing Translational Sciences (NCATS) at NIH in the final FY 2012 budget, one of the Foundation’s biggest priorities. The Foundation has been a strong supporter of this center and believes it will have a real impact on the advancement of drugs for serious diseases.
These victories show just how profound an influence citizens like you have on their members of Congress. In no small part because of your tens of thousands of calls, emails, and meetings, the federal government will have the tools to help us move closer to the cures of tomorrow. There is no doubt that your persistence helped make this possible, and you should be proud of all that you do.
Thank you for being such an integral part of Team Public Policy in 2011, and we look forward to an even better 2012! Happy holidays to all of you and your families.
Tuesday, December 20, 2011
The doctor thinks he looks and sounds great. The head cold that he's had for 3+ weeks now was diagnosed as a sinus problem. Apparently most people with CF end up with some degree of sinus disease. Just how a cold that gets into the lungs has a hard time getting out, so does a cold that sets up shop in the sinuses of a CF kid. What for Ella or Lily would have just run its course as a runny nose, for Drew turned into inflammation of the sinuses and extra mucus production creating an infection that he wasn't able to clear on his own. We ended up on a nasal steroid and some oral antibiotics for 14 days to get that cleared up. Very luckily, nothing had moved to his lungs at all and hope is that remains the case. They did do a throat swab to check for bacteria in his lungs, and we will have those results back by the end of the week. I asked what our course of action would be if he was growing pseudomonas or something again, and the doctor said that based on his awesome clinical appearance, nothing would need to be done until after Christmas. What a relief to not have to worry about squeezing in extra treatments over the holiday weekend!
I talked to the doctor also about the hunger strike that Drew is on. It was a damn miracle that the boy gained any weight between the last visit and this one because his diet consists of whole milk and an occasional corndog. He refuses almost everything, and yet somehow he continues to gain weight. Because weight is such a huge issue for many CF kids, it causes a lot of anxiety for me when he won't eat. I fear that he will end up with a g-tube feeding him overnight just to get in enough calories. Its not the end of the world if that happens as its the reality for many folks with CF, but with the good weight that he's always been able to show us, I would just hate for things to turn that direction. The doctor and the dietician both think that his weight is great and I shouldn't worry. He's in the 90th percentile and the goal for CF babies is to make it to the 50th. If he loses a couple of pounds we'd still be in good shape, but I just feel like if we allow this sort of behavior now that it could eventually turn into a problem. They recommended that we meet with a behavioral psychologist who specializes in eating issues in kids with CF. I spoke with her for a few minutes before setting up an appointment to come back, and she suggested that for kids his ages, its simply about control. They can't control much, but he knows hes got my attention with his mealtime antics, and she can offer some suggestions for making meals a better experience for all involved. We'll see what happens.
The other thing that Drew's doctor mentioned was the feedback that they had received on the presentation that was done on him at the NACFC. Here is the link to it - http://nacfcdl.cff.org/Documents/Wyatt.pdf . She said that the presentation provoked a great deal of conversation, and that a group of doctors and researchers wanted to further expand on this presentation with more studies and research. How cool that Drew started a conversation about malacia and airway abnormalities in CF that could lead to more research on the disease?!
Finally, some more great news out of the CF world just posted today. Kalydeco, the drug that's currently going through the approval process in the FDA, is now available at participating clinical sites throughout the country for people with the G551D mutation who have highly limited lung function and may benefit from treatment. The drug that may just change the game for us as we too have a class three mutation (though not the specific one tested) is already being used to change the lives of people who are critically ill with CF. What a great Christmas gift for the people who will benefit from this drug!! Hopefully it works for them and will work for us when it becomes available soon.
Thursday, December 15, 2011
FDA Grants Six-Month Priority Review of Kalydeco (VX-770) — First Potential Drug to Target Underlying Cause of Cystic Fibrosis
December 15, 2011
Vertex Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted a request for a six-month priority review of a potential new CF therapy, Kalydeco™ (VX-770).
The company is seeking approval of the drug for people ages 6 and older with the G551D mutation of CF.
The expedited review sets a target date of April 18, 2012, for the FDA’s approval decision, four months earlier than the standard review time of 10 months.
If approved, Kalydeco (kuh-LYE-deh-koh) will be the first drug available that targets the underlying cause of CF. The FDA grants priority review for several reasons, including situations where a potential drug offers a major advance in treatment.
Kalydeco was discovered in a collaboration between Vertex and the Cystic Fibrosis Foundation, which provided substantial scientific, financial and clinical support throughout the development process.
Vertex’s application for approval of Kalydeco, submitted to the FDA in October 2011, included results from Phase 3 clinical trials of the drug in people ages 6 and older with the G551D mutation of CF. The results showed that those receiving the drug had remarkable and sustained improvements in lung function and other key symptoms of the disease, compared with those on the placebo.