Special K

Picture of Kalydeco's chemical formulaKalydeco. Special K. Hope. The next best thing to a cure… hopefully.

This post is chock full of information. Don’t worry, future posts won’t be so heavily science focused but this topic excites me and I have to get it out there so you can all be excited too! You see, the Cystic fibrosis (CF) community around the globe is absolutely buzzing about Kalydeco. Why all the excitement? It’s a compound that works on the chloride ion channels that aren’t doing their job properly and are the root of all evil in CF. Kalydeco gives the chloride ion channels a good kick up the bum and makes them work a hell of a lot harder. This leads to scientifically demonstrated increases in chloride ion channel function, increased lung function, weight gain (in PWCF this is actually a great thing) and quality in life. People involved in the trials or taking this medication overseas simply cannot believe the difference it has made. It is extraordinary.

But, here is the kicker, it costs approximately $300,000… per person… per year. And it only works in roughly 8% of the Australian CF population who have a particular mutation or gene change called G551D. These are two major hurdles.

If, when and how to pay for it will be debated for years to come across the world. It is not available in Australia at the moment (some people are eligible through special programs) – but the US has had access for around 9 months now. Australia is at least 9 months behind and the clock is ticking.

How to help the other 92% of the Australian CF population is still being researched. It’s hoped that Kalydeco can be used with other compounds for other mutations. The company which manufactures Kalydeco, Vertex, is currently doing clinical trials to try to treat the F508del mutation – which Dave and I both have – which is a much more common mutation.

Fun facts about Special K:

  • Apart from toxicity, there was no animal testing involved, the researchers went straight to giving the drug to humans.
  • How did they find Kalydeco? They basically applied many thousands of random compounds to cells and waited to see what would happen. Kalydeco had the effects they were hoping for so it got the job.
  • No one knows how Kalydeco works. The exact mechanism of action is unknown.

I do believe that one day Kalydeco and other drugs like it will be available in Australia. I just hope we don’t have to wait too long. Every day irreversible lung damage occurs, we don’t have spare years up our sleeves to wait patiently.

Below are links to blogs of PWCF in Australia who do have access to this magic little blue pill via a special program. Read and witness the incredible difference Kalydeco has made to their lives.


Kalydeco for Cystic fibrosis diary: Alex Parker

My little blue guy

Breathing easy


One thought on “Special K

  1. Pingback: Ork… wait, what? | capturing cf

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