The Good, the Bad and the Exciting!

I had my monthly CF clinical trial visit on Monday.  I went in feeling fair and expected to just  do my routine 4 hours of testing that I have come accustomed to since beginning this trial.  I left with a lung exacerbation that I didn't know I had, two new prescriptions and a head full of confusion.

My PFT's dropped 5% since last month and I had been down about 3% at my previous appointment.  I am down roughly 16% since last year at this time.  The numbers are definitely going in the wrong direction.   The doc put me on Avelox (antibiotic) and Spiriva.  Apparently, Avelox will do something that the other 4 antibiotics that I take hasn't been doing.  Three hundred dollars later I headed home with these new weapons in my arsenal.  What are you gonna do?!?!

At my appointment Dr. D and I discussed Kalydeco, again.   He really wants me on it.  My gene mutation DF508 & R117H is considered a gating mutation, just like the G551D mutation that Kalydeco is FDA approved to treat.  We discussed ways to convince the insurance company, then decided that I would just ignorantly take a prescription for Kalydeco to my pharmacy and see if I could sneak it through.   That plan was foiled when we learned that there is a huge paperwork process to apply to get the drug.  

Then Dr. D came up with the brilliant idea of seeing if they are still recruiting for the R117H Kalydeco study at another facility. I didn't qualify due to the my lung function being higher last year.  This year I would qualify...my decline may be a bit of a positive in this case.   We knew that the study is wrapping up, but it was worth a shot.  I would need a center that would agree to take me in May, since I need to finish this current clinical trial and allow 30 days for the current drug to flush out of my system.   I called the Vertex people and they referred me to www.clinicaltrials.gov for a list of study sites.  I called the closest one... Pittsburgh Children's Hospital.

Here's the exciting part!!!  They said that the study would be open through May and they would love to have me come be a part of it!  I'm keeping my fingers and toes crossed that nothing changes that would disqualify me for the study.  This is good stuff!  The best part is that if I get in on the study I can stay on the drug off label until it's FDA approved.  I won't have to fight with the insurance company.  I know I am blessed...but this makes me feel doubly blessed!  This feels like an answered prayer :-)

The Waiting Game

Hello World! It's been a while and I have no excuse, except for feeling a bit boring lately. A couple of readers have requested an update, so here goes...

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.