Article: Transformational therapy cures haemophilia B

[Sly Saint]

A "transformational" therapy has effectively cured people with the bleeding disorder haemophilia B, say British doctors.

The treatment corrects a genetic defect that leaves people's blood struggling to clot and stop bleeding.

Elliott Mason, who was part of the trial which tested the therapy, says his life now feels "completely normal".

The medical team says the majority of adults with haemophilia could be cured in the next three years.

From the day Elliott was born, he was unable to make enough of a crucial protein called clotting factor IX. When you cut yourself and it scabs over, clotting factor IX is one of the proteins that stops the bleeding.

https://www.bbc.co.uk/news/health-62240061

 

[Sly Saint]

one less ltc 'needing' cbt.

 

[rvallee]

Bold claims, but this reminds me of a prominent BPS ideologue who sarcastically made the observation that for the chronically ill, medical breakthroughs are massively important, while psychosocial progress is derided as nothing.

Yeah, if it can completely cure millions it's definitely massively important. This is real life-changing stuff, if true. Meanwhile a BPS approach to this would have been the usual: suck it up therapy leaves them less "stressed" and that's good enough. Literally worse than nothing because this approach blocks the kinds of efforts like this that can change everything.

 

[Jonathan Edwards]

I think it almost certain that this is slam dunk bona fide.
This is where medicine is really transforming what can be achieved.
If gene editing achieves normal levels of a protein it means not only that the treatment has got to its target but it has harnessed the regulatory mechanisms needed to restore 'normality'.
Cures for one gene diseases like haemophilia have been pretty predictable in the last five years - just a matter of doing the technical work. And the issues about cost in the article look to me trivial. The alternative of constant replacement therapy must be much more expensive in the long run. So this will be a money saver as well.

So, yes, the BPS people can go on playing King Canute but real medicine does move forward when it can.

 

[Hoopoe]

Sounds great. The patients are expected to need treatment every 10 years or so (nobody knows for sure). So quite not a cure, more like a really good, low maintenance treatment.