Health Check newsletter: What do leukaemia cure claims really mean?

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A lab technician works on a research process to find new CAR-T cells and RNA

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Hello, and welcome to this week’s Health Check, the weekly newsletter that gives you the health and fitness news you can really trust. To receive this free, weekly newsletter in your inbox, sign up here.

This week I’ve been reporting good news about cancer, and there are few more satisfying sentences for a medical journalist to write than that.

You may have seen the stories about two people with an otherwise fatal form of leukaemia, a cancer of immune cells, whose disease disappeared 11 years ago thanks to a high-tech gene therapy. Our free-to-read article on this treatment, which uses what are called CAR-T cells, is here.

When I was discussing how to cover this story with my colleagues, one said he didn’t think we should get our hopes up too much because the study only involves two people and the approach is impractical for wide use.

I can understand his concerns. Journalism on cancer research is notorious for overhyping small studies, generating false hopes in those affected by this disease.

The idea of using gene therapy against cancer probably was overhyped in the beginning. I first wrote about it 10 years ago, as part of a package rounding up five of the most promising new approaches against cancer.

For that piece, I debated with my editor for quite a while about how exactly we could phrase the strapline – the sentence under the headline that summarises the story – where we used the word “cure”. In the end we compromised by saying the five approaches could “dramatically improve our chances” of a cure. Looking back now, I think we overstated it, because four of the techniques haven’t lived up to their promise yet.

Gene therapy, though, has done better. In the approach used in the new study, people have some of their immune cells removed. A gene is added to these, which encodes an artificial receptor that recognises leukaemia cells, and the resulting CAR-T cells are then multiplied and reinfused into the cancer patient.

It is such an ambitious and complicated thing to do, it initially seemed too far-fetched to work. So the latest study is seen as a real landmark.

The approach isn’t a panacea. It seems to send about four in 10 people into remission across all the different types of blood cancers it has been used to treat. We don’t yet know how many of those will be long-lasting remissions.

It would be wonderful if it worked for everyone. But other cancer treatments like chemotherapy and radiotherapy don’t have 100 per cent success rates either, and are still highly valued weapons against these diseases.

Then there are the other downsides of gene therapy: how complicated and expensive it is. I don’t see the complicated part as being a big hurdle. You could have made the same point about bone marrow transplants 40 years ago, but they are now widespread.

While the price of CAR-T cell therapy to UK health services is confidential, in the US it costs about $400,000 per treatment. If – and it is a big if – this does turn out to be a cure for leukaemia for some, that would probably be deemed an acceptable price.

That is the big question of course – are those who go into remission really cured? Interestingly, the “c-word” wasn’t used in the scientific paper – but during the press conference, the lead researcher mentioned it often.

Other independent scientists I spoke to said it was a reasonable assumption these two people are cured, given the length of time since treatment, but we won’t know for sure until more time has passed. Still, the fact that the first two people who were successfully treated with this approach still have no signs of cancer 11 years later is very hopeful.

The next goal is to use the same strategy against solid tumours, the more common form of cancer. But here it is proving harder to get gene therapy to work. The sticking point seems to be getting enough CAR-T cells into the centre of the tumours and keeping them alive and active for long enough in that toxic environment.

Many cancer researchers are now trying their hardest on this, coming up with ever-more complicated forms of the artificial receptors and giving the modified immune cells other genes for extra defences.

It is an exciting time for journalists writing about this field, but we should temper our enthusiasm. It took about 20 years for CAR-T cells to go from far-fetched to working well for some. No one can say how long the next stage will take.

Other health stories

  • The long read: Ever heard of interoception? It is how our brains interpret signals from within the body, like the heartbeat or hormone levels, and it has a surprisingly big influence on the mind. This insight is leading to new ways to tackle conditions like depression, anxiety and eating disorders.
  • People who are susceptible to getting weird tingles in their scalp if they hear certain whispery or tapping noises – a phenomenon known as ASMR, or autonomous sensory meridian response – are more likely to be anxious and neurotic than average. Luckily for them, the tingles are a natural anxiety reliever.

From the archive

Thanks to a Victorian doctor who promoted medical marijuana, for part of the 19th century cannabis was the drug of choice for ailments from migraine to epilepsy – until an unexpected twist led to its downfall.

That’s all for now, but do take a look at our online event “The science of happiness: Why is laughter funny?”, held on Thursday 3 March at 6.00pm GMT.

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