Thursday 26 June 2014

The Next Steps for Combination Therapy and Cystic Fibrosis

Ed Owen, Chief Executive of the Cystic Fibrosis Trust, discuss how this week's announcement on the ivacaftor and lumacaftor combination therapy affects the wider landscape of beating cystic fibrosis for good.

Over the last five decades, we have made great progress from the time when cystic fibrosis was a condition that killed most people living with it before they reached the age of 10.

Yet it is only now that therapies targeting the basic genetic defect – the holy grail of so much work since the discovery of the cystic fibrosis gene in 1989 - are becoming a distinct reality. Indeed, Kalydeco (Ivacaftor) is doing just that for those with the G551D mutation already!

Of course, it is important to sound an important note of caution here, not just because the results of the combination therapy for those with two copies of the F508Del gene are not as dramatic as that seen with Kalydeco – and neither is a cure.

There is also still some way to go before this treatment can be available to people with cystic fibrosis, Vertex need to receive a license to market it from the relevant regulatory authorities.  Here in Europe that’s the European Medicines Agency (EMA) which can take many months to assess the evidence that is presented to it.

And even if regulatory approval is granted, there will then be the often protracted process by which each of the four NHS systems across the UK decide whether to fund this therapy or not.

In all, whatever the final decisions of the NHS, this whole process will probably take at least another year, and probably more – and that’s a desperately long time for many.

Those with other mutations will have to wait longer still before transformational therapies come along.  But there is hope in these areas too.
We can afford to be very positive – and our voice as a cystic fibrosis community will play a vital part in persuading decision-makers and manufacturers to enable new treatments of this sort to be funded in the UK.
This week is a further step forward on the road to beating cystic fibrosis for good - but there is still a long way to travel.  In the words of the late Casey Kasem we need to “keep our feet on the ground and keep reaching for the stars”.


  1. All very true indeed especially about how it could be too late for many but, and a very big one at that, even this somewhat small breakthrough is a breakthrough, one that so many have worked towards and patients like me have dreamt of that I can't help but be delighted.

    I don't even have this mutation but know that thousands of people will be able to benefit for this, leading to new treatments for the rest of us is huge. Between the two treatments people may live a hell of a lot longer than anticipated jsut 5 years ago.

    Being realistic is great, and a necessary part of life but let's enjoy this for a little while first.

  2. I wrote a similar piece yesterday regarding my own feelings concerning the news. Have a look: