Friday, December 20, 2013

2,100 Hours on a Placebo? UGH!

Placebo or Med:

     Clinical Trials are important to the advancement of meds that can help cure or correct a medical problem. There are lots of trials for different types of new drugs to help Cystic Fibrosis. Some inlcude: CFTR Modulation, Nutrition, Inhaled Anitbiotics, etc. To see all the clinical trials that have turned into meds to help us, as well as others that are currently being studied go to The Cystic Fibrosis Foundation Research Webpage: ( There is a very helpful graph that explains all the clinical trials.
     I'm currently in a clinical trial, where I alternate two drugs. I receive one drug every other month and on the alternating months I receive either the other drug or a placebo. I'm not sure how this will affect me if I end up getting the placebo. My body has been taking these two drugs already alternating for years. So if all the sudden one of those disappear, not only with it be harder on my body, but during the placebo months I won't be receiving any inhaled antibiobics. Not sure how that will affect me. Especially since I've been culturing psuedomonias aerugonisa since I was little.  Read what Psuedomonis is and how it affects us here: CF Pseudomonas.  These inhaled anitbiotics are the best way to fight it. That being said, I'm not too worried. First of all, its a 50/50 chance I 'll get the drug and not the placebo. If I do get the placebo. I may be fine on the off months and the monthly pulmonary function tests (PFTs) will keep me closely monitered. But to think I have to spend all this time, making the meds, doing them, cleaning the equipment, filling out the adds up to 2,100 hours spent on "placebo-maybe" months during the 6 month trial. Dang, that is a lot of time to spend on a fake med, if it is the fake med. But, in the long run it is only a little time to give for help further medical advancements!

      I did have my clinical trial appointment today and I did my pulmonary function test. Man, I ended up getting about 7% lower than I normally do. Usually, I'm a 58, I was a 64% right after the hospital stay. Now, I'm 50%. Boo. Dr. B asked if I was feeling okay. And I am. My lungs a little tight, but I'm not feeling sick. So I guess I just gotta be mindful of that, work a little harder on my physio- (vest, exercise, etc) and make sure I don't get worse. I have my CF regular clinic appointment on Jan. 6th. So I'll be careful and make sure It goes up then! I'd be soo upset if I end up back on IVs. So hopefully my hard work will pay off.

Exciting New Research, including a new drug for my mutations: The Current Drug targeted to help correct the faulty gene in my mutations has been in its 3rd and Final Phase, before it is finalized for production, since Feb of 2013. To read all about the exciting news about it HERE!

If this becomes a reality, it can greatly help me to fight my CF, it could raise my current Lung function. Some of Kalydeco Recipiants got around 7% of their lung function back.  Plus, if this new drug was taken daily it would greatly reduce the advancement of the disease and help the body fight harder and longer. It isn't a miracule cure drug. Its not like as long as I take the pill I'm better and don't need my meds. But it should be looked at as another med that will help prolong our lives.

Now some other Research Drugs:  Latest Published Results on a Clinical Trial: It isn't a drug for my mutations, but that doesn't make it any less important, somewhere in the world there are other CFers with this Mutation.  Read an article about it Here AND Read Read the Vertex Announcement Here.  Perhaps Compare the two...

It is interesting to hear about the results straight from Doc verses what the press releases. Dr. B said that the study didn't result in positive outcomes they wanted to help those patients, but it did teach us more about how different gene modulators can change how CF mutations can affect people differently. He said there are more studies done on primarly twins and siblings to understand how CF can affect people with similar heredity/genetic DNA so differently. I'm interested on what these sibling studies show!

Fun Story: Doc said he got called for a consult the other day. He was told it was a 72 year old. He was like: "okaaay", they continued "they need surgery". He was like "okay , why do you need me to consult, I'm a Pediatrics and Respitory..." Well they  continued: "They have Cystic Fibrosis" He was "oh, okay...Well then." He said he got told her whole med history. She was 72, born with my mutations, had all the severity with other complications, and was diagnosed at birth too and he said he felt like "yipee". He told me he is beyond happy to be able to see such a difference in life expectancy that he can now tell us " You can expect to live to not only to get married, and have a kid (or adopt for those of us with reproductive issues), BUT now, we are more likely to see our kids as adults, perhaps have grandkids! To think I could have grandkids, definelty makes me happy!!!!!!!!

But until then I will just continue to help in as many research studies I can ( I also plan to donate my body to science- as long as so many years later when finished they cremate my remains and scatter my ashes- since its good for the environment- sorry if that's weird, but its what I want!) I was in these clinical studies and helped these meds make it to production:  Pulmozyme (nebulizer), Tobi (inhaled Antibiotic Nebulizer), and Enzymes (digestion study, where we saved all my poop in buckets, sealed it well, and shipped it to the lab that was studying it). I was young during all those studies, but I made the choice to do them, even the poop study. I hated pooping into a bucket,  But I knew it would help me and my CF friends gain weight and have less stomach aches.

Participating in Research Studies, Raising Awareness on my blog, and Fundraising at CF Fundraisers (like CF Walks- is the best way I can help!

 And more good news to end with: Laura (one of my besties with CF from Childhood) has a Transplant Evaluation Appointment on Jan 27th)

Read about her CF struggle right here! and I'll keep you updated on how she is doing!

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