UK approves gene therapy to cure two blood disorders - BBC News
BBC News- 540 views
- 18 Nov 2023
In a world first, medical regulators in the UK have approved a gene therapy that aims to cure sickle cell disease and beta ...
Now, in a world-first, British regulators have approved gene therapy that aims to cure two blood disorders: sickle cell disease and beta-thalacemia. It's the first treatment to be licensed that uses a tool known as CRISPR, which can be used to correct faults in the DNA of cells, allowing them to produce hemoglobin. We're joined now by Zeinab Garber-Sany. She is a trustee at the Sickle Cell Society charity. Welcome to you. Thanks very much for being with us. Just explain to us what this new year-approved gene therapy means for those with sickle cell disease?
Hi, thank you. It's an exciting day for the sickle cell community. It means that we've got one more treatment in the pipeline. Unfortunately, for too long, people with sickle cell have only had a handful of treatments. This is a potential for cure, so really, really life-changing for our community.
Tell us about the potential cure then. What it's going to mean? What will this treatment involve?
At the moment, the only cure for sickle cell is a bone marrow or stem cell transplant and you need a match donor. Unfortunately, there's just not enough match donors, so many people aren't actually eligible for this. This new treatment means that actually you don't need a match or you don't need a donor. Through your own genes, some incredible science can be done. That means that it can be edited and you can have a cure just through that instead.
How long does this take? What's the cost?
Costs are in the millions at the moment, but this is just first really exciting, the fact that this is even a potential at the moment. There will be some conversations, and the conversations have already started about, actually, how do we finance this and how do we bring down some of those costs just to make sure it's as accessible as possible for as many of our community? That is something that we're now going to have to really be thinking and focus on going forward.
Yeah, it's going to be tough, isn't it? That money for millions, for one person to be treated. Is this something that you think the NHS will be able to go ahead with? How will they decide who gets this treatment?
Well, there's really big, important decisions that will have to be made going forward. But at the moment, the biggest challenge that we have is the fact that sickle cell was the first genetically defined condition there is. But unfortunately, still to this day, there's only been a handful of treatments. Actually, just having an extra treatment, extra opportunity is a huge thing and hopefully it will drive more innovation and more research in this area. There will have to be some decisions and some conversations that will be had around how do we make sure it's accessible, not just in the UK, but I'm sure globally, we're going to have to start thinking about how do we get this life-saving treatment in the hands of as many people as possible.
Zeinab, thank you very much for talking to us from Solihull. Thank you. Thank you.