Wow, where did summer go?  I was just getting settled in and… whoosh – it’s gone!

 

So many things are happening in the world of haemophilia that it’s almost impossible to keep up with the pace.  Here’s a taste:

 

  • Biopharmaceutical companies are trialling different versions of extra-long half-life clotting factors such as polysialylated and PEGylated versions
  • There are many companies looking at gene therapy for haemophilia B (generally utilising various serotypes of adeno-associated virus as vectors)
  • Haemophilia A is getting in on the gene therapy action also – which is great news as it’s always been more difficult to find a suitable vector due to the larger size of the factor VIII gene
  • RNAi technology is also on the scene with an upcoming new treatment for haemophilia patients with inhibitors (this is very exciting news as it targets the inhibitors at the level of genetic translation of the antibody protein)
  • Also, with the advent of CRISPR/Cas9 technology (also known as gene editing) there is talk afoot about the possibility of editing the foetal genome, or even more radically, editing the germline (I always knew that this would be inevitable, though I think it might be a while yet before it becomes a reality as the uninformed and tabloid media will have a field day, just like they did with mitochondrial replacement therapy, such as pronuclear transfer)
  • The UK government have caved to public opinion and announced a public inquiry in to the contaminated blood scandal – however this isn’t to be taken at face value as they are still doing everything within their power to obstruct it by putting the Department of Health as the Lead in an inquiry against itself (most other organisations would consider this to be a conflict of interest), they are still ignoring the calls and wishes of all those who have been affected, and key dates for things like determining the terms and scope are being put back again and again and again. But then, are we really to have expected anything different?

 

With all this coming through there is an even greater impetus for patients, carers, and healthcare professionals, to be more engaged and educated with regard to these new treatments, their actions, limitations, proper use, and policy.  What will they mean for prophylaxis regimens?  Will the prospect of adverse reactions be more, or less likely?  Will the cost of these new treatments be a stumbling block when attempting to pass the desks of the commissioners and be funded by a future, ever decreasing, NHS budget associated with ever increasing red-tape and fiscal justifications?

 

This may seem onerous for many, and it may well be easier for most to think that we should “leave it to those in charge of these things”, but at the end of the day it’s our veins that the needle is being stuck in to, not the far-removed politician or civil servant who looks at me, and the cost of my treatments, as numbers on a spreadsheet.  This means that if we truly want the best out of our medical care, we need to get more involved in the understanding, education, and regulation of it as progress happens and advancements are made.  And it seems that advances are being made at a startling rate… so hold on to your seat, or you’ll be left behind!

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