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 :-)