LFG (let’s f$$$ing go!) - Orkambi here we come 💃 Part 1

Today is a moment that is surely worthy of a post.

Arlo had his eyes checked, which is required before he is able to start on a cystic fibrosis modulator, called orkambi, when he turns two.

So what is Orkambi?

The CFTR modulator Orkambi is a combination of two drugs (lumacaftor and Ivacaftor) that work in two ways to help the CFTR protein move chloride across a cell surface.

Lumacaftor is what is known as a ‘corrector’. This means that lumacaftor helps the CFTR protein form the correct shape so that it can move to the cell surface. This alone is still not enough to effectively move chloride through the surface of the cell. A ‘gate’ is needed to allow the chloride to pass through. That is why another CFTR modulator, ivacaftor, is included in Orkambi. Ivacaftor is called a potentiator. This means that it holds the “gate” on the cell surface open long enough for the chloride to pass through.

Combined, these two modulators that make up Orkmabi reduce the symptoms of CF by enabling the effective movement of chloride and thinning the sticky mucus that affects Arlo’s pancreas and lungs, amongst other things.

Orkambi works only for as long as it is in the system, which is about 12 hours. That is why it is prescribed to be taken every 12 hours.

Orkambi does not completely restore proper chloride flow in the cells or reverse lung damage, but it does relieve the symptoms of CF - especially the chances of getting a chest infection.