Cystic Fibrosis – a good news story?

I do love it when I hear a good news story.  The medical breakthrough, the new therapy that’s going to save lives, or if not, at least improve them.

There was one such story doing the rounds last week.  A new gene therapy for Cystic Fibrosis, a debilitating lung condition which leads to an average life expectancy of 41. In the trial the lungs of patients who took the drug improved over the period by about 3% while those who didn’t take the gene therapy saw their lungs decline.

We were introduced to a lively 16 year old, Mary, on the Today programme on Radio 4. She’d taken part in the trials without knowing whether she was part of a control group or actually taking the drug.  Within a month she’d noticed an increase in energy.  She was able to do more school work and activities with her family.  Her mum, Debbie, said it was ‘like a magic trick’ and pointed out, by the time the trial ended, her condition had improved so much that they could leave her wheelchair in the car.

Brilliant.  Fantastic.  What an amazing good news story.  Except of course it turns out it’s not such good news for Mary.  Yes it’s a significant breakthrough, but in scientific terms, the effects were ‘variable and modest’.   Her doctors  can’t just put her on the drug without more research.  And the universities that did this research have run out of funding.  Prof Eric Alton from Imperial College London says the group is “in negotiation with a large number of companies to say we need a rapid study”.

But rapid isn’t exactly the word I would use.  Even if one of the pharmaceutical companies takes it on, the earliest Professor Alton can see a study happening is ‘the end of next year’.  18 months away.  And that’s just the next study.  It’ll be at least five years until the therapy will be available to patients.

Isn’t it time to speed this stuff up a bit? Mary’s cystic fibrosis  is getting worse.  Since she came off the trial she’s gone downhill, had recurrent infections, been hospitalised.  And yet it’s a full year and a half before another study might even start.

We can all imagine why.  Endless emails, meetings, reports which take a month to write and then another month for someone to read, medical bodies to consult.  And of course we’re relying on a pharmaceutical company deciding the drug is commercially viable.  That the drug will make enough profit.  That the NHS will be able to afford to buy it.    All this talk takes time.  Meanwhile  Mary is struggling to breathe.

I’m not a red-tape whinger.  Sometimes what others call red tape, I think is sensible health and safety.  But I object to a system which seems to accept this type of delay.  What can’t the Medical Research Council, the National Institute of Health Research and the pharmaceutical companies get into a room together next week and work this out?  Let’s start the next trial next month not next year.

Debbie, Mary’s mum ended her BBC interview talking about the second study.  “Anything that can be done to help the second phase is …….” She paused, straining to find the right word.  “Paramount” is what she came up with.   But what word do you pick to describe the urgency when you’re looking at your 16 year old daughter gasping for air?

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