A potential cure for two inherited blood diseases using gene therapy has been authorised by Britain’s medicine regulator in a world first.
The treatment, Casgevy, for sickle cell disease and beta thalassemia, is the first to be licensed using the gene-editing tool known as Crispr – which earned its inventors a Nobel Prize in 2020.
That the treatment exists is wondrous and that it’s authorised is good. That it costs £1 million a pop isn’t so good but give it 15 years (-ish) for the patent to run out and it’ll drop to £50k a pop or whatever. For a one-time treatment that cures a genetic problem. Excellent, the world gets better every day.
The amusement? We’re the first to approve a treatment for diseases which – before the mass immigration of the past couple of decades – pretty much didn’t exist in Britain. They’re pretty strongly – to very strongly in fact – linked to, respectively, West African or Mediterranean genetic background. Still a good thing, it just does amuse.
A million pounds a pop for non-contributing newcomers?
Just another priority, ranking over and above the millions of actual UK citizens on waiting lists, for the international health service.
Thank goodness that the MHRA sees itself foremost as an “enabler” rather than a regulator primarily concerned with public health.
Approved for use in the UK, OK. But surely not (yet) approved for use by the NHS? Won’t NICE have to rule on that?
(It, or the pols, will cave to pressure from Persons of Hue of course, but there may be salty amusement to be had from the lies they will issue about the decision.)
Nice to know that someone who has paid NI and tax contributions for 50+ years can sit in agony waiting for a hip replacement while someone straight off the boat can get £1m spent on them just like that…….. isn’t the NHS great????
Countries in which this disease is widespread (except the US) don’t have drug regulatory authorities to speak of. They just copy what FDA and EMA approve.
So in that sense, yes, it’s a sensible approval.
‘That the treatment exists is wondrous and that it’s authorised is good.’
Oh yes, all we need to know is that it’s authorised.
I understand that thalassemia provides some sort of protection from malaria.
So as global warming continues and the mosquitoes flourish, no doubt this therapy will be adapted to protect you Brits.
Of course I’d argue that DDT is cheaper.
If it’s a one-shot curative and can be administered early in life (I have not looked at anything so don’t know) I’d bet a lot of money on NICE accepting the price tag.
NB, the price tag will be very different in different countries.
From photos of the company’s team that developed the treatment, not a Person of Preferred Pigment anywhere to be found. Looks as though White Man’s Burden strikes again.
@Boganboy
Yep. Sickle cell anemia is an inheriable ‘survival of the fittest’ adaptation to avoid death by malaria
“Global Boiling” scare occurred to me to
The craziest thing i’ve seen recently was a youtuber do gene editing on himself. Designed the gene on his computer, crispered it into a virus, cultured the virus, made pills from the culture and took a course of them. All to cure himself of severe lactose intolerance. And it effing well worked. With the rather important caveat that all the commentators who seemed to know something about it said there was a crazy high risk of giving yourself cancer.
https://youtu.be/aoczYXJeMY4?feature=shared