My health is no better, but not tremendously worse. No huge complaints! I am a bit disappointed that my lung function is back to where it was before I started all of this ridiculous treatment for my Non-Tuberculosis Mycobacterium two and a half years ago. I'm also frustrated that I'm still culturing MAC, but I guess that's the nature of this beast sometimes. I just wish the LOADS of antibiotics that I have been taking for 2 1/2 years straight would have showed some tangible progress. I want clean cultures, Dammit!
In October, I started a clinical trial for the MAC. The trial involves replacing the inhaled Amikacin that I was taking twice daily for once daily inhaled Arikace. The Arikace is the same drug as Amikacin, but it is attached to a lipid that allows it to absorbed deeper and better that Amikacin. The other benefit is that I inhale the Arikace out of a Trio nebulizer, which makes treatment time about 12 minutes. My inhaled antibiotic treatment time decreased by about 48 minutes! I'm really liking the ease of taking the new drug. Another benefit of the clinical trial drug is that it is not supposed to cause the same hearing damage as the Amikacin. The bad news is that even if it's working and I prefer the new drug, once the trial is over in May I have no option to continue. I have to go off of it and wait for FDA approval (years) before I can access it again.
This is the same frustration I'm feeling about Kalydeco. If you are not part of the CF world you may not know about the exciting new drug that has been discovered that basically corrects the malfunctioning CF genes. It was named as the Most Important New Drug of 2012 by Forbes Magazine. For some patients symptoms and progression of the disease have virtually reversed. Lives have changed! The catch...this drug is only FDA approved for 4% of the CF population. Only people with a certain genetic mutation have access at this point. I'm not included in that small group. The good news is that Kalydeco is being trialed in those with my genetic mutation ~ Delta F508 & R117H ~ and the trials have shown impressive improvements in some of those involved! I want this drug and I want it now (said in my best Veruca Salt voice). Unfortunately, I have to wait until this drug is FDA approved, as well. My doctor said this may be another 2-3 years.
So here's the deal...I am very slowly watching my lung function decline, but still holding my own. All while I'm waiting for clinical trials to progress and the government to give me access to these drugs that could rock my world. I asked my insurance company if I could go on Kalydeco off label, but they were not impressed with the $300,000 yearly price tag. I am going to appeal and fight for access to the drug, so we'll see. I feel like I'm desperately trying to save as much lung function I can before my sliver of hope arrives.
Other news in my world... I retired from my career. Whew! This was an extremely difficult and emotional decision for me to make. Trying to juggle a full-time job, 2 (sometimes 3) children, a husband and Cystic Fibrosis has always been chaotic. For many, many years I thrived on the chaos. It made me feel like I was able to do it all. This became more difficult after the NTM diagnosis 2 1/2 years ago when my treatment plan became much more complex. To be honest, I've been faking it for the past couple years. I have been exhausted to the core, sick and struggling. When something had to give, my health got put on the back burner. I knew that this couldn't continue. After a particularly exhausting business trip that left me with another lung exacerbation I decided to go on Short-Term Disability and explore my options. That was in early August. I never went back to my job. I retired...a bit earlier than I expected I would, but I knew it was time. I had the privilege of being with my company for 13 years. I really felt like I had done everything I wanted to do in my career up to this point. I didn't have aspirations to climb the ladder any higher. After 5 1/2 months of trying to determine if I should go back to work or not, I decided that now was the time to devote everything to my family and my health. I am very happy with my decision now, but, jeez, that was a stressful and difficult decision to make.
I guess I had more going on that I thought! I am going to try to post a bit more often. I've missed my readers...
Here's a little song that's been speaking to me lately.
3 comments:
A lot of the time if you are in a Phase III trial, you will be allowed "compassionate use" of the drug after the trial ends and before it gains approval. I would definitely ask around about that - I think most pharma companies do it. Most people I know who have been in the big recent trials (Kalydeco, Bronchitol, etc.) have been able to stay on it through compassionate use programs. Good luck! I really hope it helps.
And, as someone who was in a very very similar situation (with steadily declining health/lung function etc.) 2-3 years before Kalydeco was released for my mutation, don't give up hope! I remember telling my mom one day when I was really upset that my biggest fear wasn't getting so bad I'd need a transplant, it was getting worse to a certain point and then getting on Kalydeco when it was released and have it just keep me at that point indefinitely. I am happy to say that has NOT been the case at all. Although my disease progression hasn't been reversed, I have felt better this year (even pregnant) than I have for probably a decade.... seriously. I was in quite a hole for several years pre-K and I was worried that I would never get out of it, but the stabilizing effect of Kalydeco has been AMAZING. I told my husband that the lung function increase (which I have had) is not even the most amazing part... it's the stability, the fact that I have not spent every minute of the last 11 months feeling constantly sick and in need of IVs, which was pretty much the story of my life for the preceding 5 years. So anyway... hold on, cyster! I hope that the R117H trials breeze through!
Way to go on puting your health FIRST. I can't imagine how hard that must have been but it sure does seem like the no brainer solution. What things have you been doing to really focus on improving your health? More exercise? Meditation? Better meals? I'm just curious. Might make for a good blog topic. Oh and about the Kalydeco, any chance you could go abroad to get it? I'm not as up on it as I used to be so am not sure if it's even out there in the EU yet. You can school me ;-)
It seems at every turn there is always a struggle with this disease. I'm sure not working will be a huge blessing and relief. Good luck.
Colleen
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