Friday, 14 November 2014

Four little letters.

For anyone that has hidden under a rock for the last week I'd like to draw your attention to this.

For those of you who don't feel inclined to read it allow me to summarise.

Jon Snow is a reporter for channel 4. He broke the above news story via his blog and followed up with a report on channel 4 news and it sent the majority of the CF community into a spin.

Okay, I hear you say but why has it caused such a frenzy?

The frenzy induced madness was caused because the report was about some research that has been jointly done by Researchers in Dundee, France and Italy.

Well that's perfectly normal and happens all the time right?

In this case no. The attention grabbing headline was something to make even the most dubious of folk a little curious.

"Eureka moment offers hope of Cystic Fibrosis cure"


Currently we are in a very exciting time in CF land. So many studies, trials and research are in progress for medications that treat the underlying defect/cause of CF. However they are exactly what they say on the tin. Treatments, not cures.

So to hear about results of a trial that no one has really heard of before be declared as a major game changer for CF got people slightly dizzy.

I'm a sceptic at heart and always like to see evidence, stats etc so with the help of a few friends we did a little 'research' of our own.

It turns out the research, which appears very promising is at a very early stage in its development. The trial was completed with only 10 patients and they each had double DF508. It also appears that this treatment may only work on adolescents and young children only. This is something to do with the lining of the lungs becoming tougher the older you get, but as its so early in trials there aren't masses of results to chew over.

So back to earth we fall. Well not entirely, the research looks very promising but it needs a larger scale study to really see if the results are as good as they have seen in the small group used in the trial.

Another point i'd like to raise is this particular study is using two drugs that are already in use for other conditions so I'm not sure whether safety trials will be as lengthy as others are. It is however a long way off becoming a potential treatment for people with CF.

Back to the magic word. Cure.

To me a cure is a one off treatment that you receive which gets rid of the illness/condition. This particular drug combo, to me is not a cure as I'm assuming the medications would be taken for life?

The media have a lot to answer for by throwing about these types of words in a world where people live in hope and often desperation for something, anything, that will nuke Cystic Fibrosis once and for all. 

I watched a lot of the CF community get excited in a way I haven't witnessed since vertex initially announced the early results of VX770 (kalydeco) quite a few years ago.

It was very sad to then see that hope almost deflated as the reality of the headline began to unravel for those who took it as literal.

People that have lived with CF for a long time are seasoned professionals at being very sceptical when headlines like this are announced, but for those with newly diagnosed children or people who get baffled by science, it raises false hope in a cruel way.

Cautiously optimistic is probably the way I feel but it definitely pays not to stack all of ones eggs in one lonely basket. 

The day my daughter has a treatment prescribed for her that can change the game dramatically is the day I'll celebrate. 

Until then, we will work our backsides off to keep her as healthy as we possibly can.



  1. Thank you for providing a balanced view of this research - I just hope the hype is not going to take funding away from other avenues of research. For example, Gene Therapy - still awaiting the results of the recent trials so let's hope they do turn out to be good news.

    1. Yes I do agree. The 'latest craze' can often lead to money being sent in one direction and forgetting about all the other fantastic research going on in other places. While this research looks promising it is important to use a calm head with it for now at least.

      Gene therapy is an interesting one, disappointing the data wasn't ready in time for the North american CF conference. I can't help think is this because the data isn't reading as well as expected-there goes my cynical side again!
      As with everything, I'll look forward to chomping through the data when it is available and making my decision whether to start partying or not :)


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