aHUS is a savage and cruel illness that kills and maims people.
This is something that those who have experienced it, and those who fear experiencing it, try to convey to those who have the power over aHUS people on whether an effective treatment can be given to them.
aHUS is brutal, but so is the good part of the body’s defence system which protects each of us from malicious invading life forms ,which enter our bodies and are in our blood. It is going on now in the viewer’s own body, in everyone , it is natural and important. The best form of defence is attack.
If scaled up it would look like something from a horror movie.
The rather nice sounding Membrane Attack Complex (MAC) resulting from the activation of the rather polite sounding Complement System pierces the outside “skin” of the invading organism and it explodes like an exploding bullet would do to a skull. It would not be pretty. Once its job is done the MAC is drawn back into the “holster”
For those with aHUS, that good thing becomes a bad thing because MAC cannot be drawn in again ,and it begins to attack the aHUS patient’s own cells as though they were invading cells. It does a lot of damage and the resulting blockage in the capillaries causes the patient’s red blood cells to explode as they try under pressure to get through smaller and smaller gaps. Acute anaemia and kidney failure make the patient feel worse than they have ever felt in the lives. aHUS is brutal.
But what if there was something that could stop this ” friendly fire ” from causing collateral damage? Would not that be good? Well there is, it is a Complement Inhibitor it is safe and it can stop the MAC in its tracks as much evidence shows.
So just use it then you might say, no brainer.
Well that it is not as easy as you may think and for most aHUS patients around the world access to this effective complement inhibitor is not available. Lots of reasons for that but basically it is down to its price. This is the key challenge in the aHUS world.
Most aHUS patients would have had no idea that the disease existed until it happened to them, because it rarely happens at all . There may be only 20,000 aHUS patients within a world population of over 7 billion people. No wonder they have not heard, nor seen it. If they knew they would however be sympathetic to the plight of the untreated victim even before they became one.
In the past three weeks a family in British Columbia have become aware of aHUS. They now know what it is like and are in the middle of the medical car crash that aHUS patients have experienced elsewhere.
With the 99th anniversary of the Great War just passed , it might be fair to say that they are injured in no man’s land with no one prepared to help because the price of that care is too high.
It would require the person ,who had the power , to be brave enough to say it is not too high.
To look beyond the price of the complement inhibitor and look at its true cost, because the evidence is growing and becoming compelling that the cost of treating an aHUS patient is considerably less than thought. The gap between the average patient cost of a complement inhibitor and the alternative treatment is much closer than just using a highly publicised list price.
The list price is “absurdly” high and the supplier needs to acknowledge that and do something about it, not just for this one patient but for all.
But in the meantime aHUS is savagely and cruelly destroying a life of a Canadian. It would be brutal not to stop that happening.
The story of Shantee in British Columbia is going around the world.
Last year the alliance featured a story about a young girl in India whose family were in a similar position to Shantee’s. She died , she had the wrong genes in the wrong place.
Let us hope that the same cannot be said of British Columbia.
It was not, a few days later the province decided that eculizumnab could be administered on a case by case basis. see CBC report here.
Let us hope now that it cannot be said of anywhere one day.
