Clinical disappointment

So the good news. Isaac hit a 93% FEV at his last clinic appointment. This is a measurement of lung function, forced expiratory volume, that is, how much air he can blow out in one second, and is one of many ways in which doctors measure wellness in CF, and often, the most predictive. 93% was a really good day for Isaac, as in the past couple of years he has declined to, at times, 70-80% (which is not life changing in itself, but is a decline we would rather not see, at this, or any age). At times this decline is because of known infections, at others just unknown, which is worse.

The bad news is, this recent good result means that he won't now be eligible to be assessed for the Ataluren clinical trial. 

To say I am disappointed would be an understatement. I had a little cry (most unlike moi!). Clearly we're delighted at his good results. But how ironic that they come at this time, when the drug that I have been following the progress of for 5 years or more has eventually started recruiting for trials, not only in the UK, but just down the road from us, in London. 

This is not just any drug. This would be the first drug that Isaac could have taken which could, potentially, treat the underlying cause of his CF, not just the symptoms. And yes, it may not have worked. He may have even been on the placebo. And even if it had worked, would it ever be approved by NICE for the clinical setting? Would it be affordable for the NHS? I don't know. But I sure would have liked to have tried! 

CF friends will understand, Ataluren is very important to us, as Isaac has rare mutations, class 1 (supposedly the most severe) and so Ivacaftor and Lumicaftor will not help him. And gene therapy, which would work for all, by my estimations is still 8 or more years away.

I know this doesn't mean that our love affair with Ataluren is impossible, only we have to wait longer to find out. I'm just disappointed that they don't look at average FEVs rather than the most recent, or assess him in London and then decide? Sod's law his FEV will have dropped next time he is tested! 

Sorry. Rant over. Feeling disappointed. 

Merriness will resume shortly x