Woman who had pioneering cancer treatment 18 years ago still in remission

The Guardian

17-02-2025

A woman treated with a pioneering type of immunotherapy for a solid tumour has been in remission for more than 18 years with no further treatments, experts have revealed.
The therapy involves taking T-cells, a type of white blood cell, from a patient and genetically engineering them to target and kill cancer cells. These modified T-cells are grown in a laboratory and then infused back into the patient.
Known as CAR (chimeric antigen receptor) T-cell therapy, the approach has proved particularly successful in treating certain types of blood cancers. Next-generation forms of the therapy have been approved for such cancers in countries including the US and UK.
However, response rates have been less encouraging in solid tumours, with long-term outcomes unclear.
Now researchers have reported the longest known survival after CAR T-cell therapy for an active cancer, revealing a woman who was treated as a child 18 years ago has remained cancer free. Crucially, the therapy was given for a type of solid tumour called neuroblastoma, a rare cancer of the nerve tissue that develops in children.
Prof Helen Heslop, co-author of the research from Baylor College of Medicine in Houston, Texas, says the trial was one of the earliest to use CAR T-cell therapy for cancer.
'It's nice to have such long-term follow-up and to see that even if it was a very early CAR T-cell – and there's been a lot of work to make them better – we were still able to see a clinical remission that's been sustained for this long, so that she's grown up and is leading a normal life,' Heslop says.
Writing in the journal Nature Medicine, Heslop and colleagues report how they recruited 19 children to take part in a phase 1 clinical trial of CAR T-cell therapy for neuroblastoma between 2004 and 2009.
Over seven years that followed the therapy, 12 patients died due to relapsed neuroblastoma. Among the seven that survived beyond this point, five were cancer-free when given the CAR T-cell therapy but had previously been treated for neuroblastoma using other approaches and were at high risk of relapse. All five were disease-free at their last follow-up, between 10 and 15 years after the CAR T-cell therapy, although the team note they may already have been cured when the therapy was administered.
The other two surviving patients had cancer that was actively growing or spreading when they received CAR T-cell therapy, but subsequently went into complete remission. One of these patients stopped participating in follow-up sessions eight years after treatment, but the other continued and has remained cancer-free more than 18 years.
'She has never required any other therapy and is likely the longest-surviving patient with cancer who received CAR-T therapy,' the team write. 'Encouragingly, she has subsequently had two full-term pregnancies with normal infants.'
The team add the modified T-cells were still detectable in some patients after more than five years. Heslop says that, while it is not known for sure, it could be that CAR T-cells that persist are able to tackle the cancer should it return.
Heslop adds that newer forms of CAR T-cell therapy have shown a greater response in recent trials for neuroblastoma, and may also help tackle some types of brain tumour in children.
Karin Straathof, the associate professor in tumour immunology at UCL's Cancer Institute, who was not involved in the new study, says the results are beyond encouraging.
'This is really a solid demonstration that in solid cancers you can achieve complete responses, but also what we want really – and particularly for children's cancers – long-lasting complete responses,' she says.
But Straathof says further work is needed, adding: 'What we now are trying to focus on is understanding why does it work in some patients and why [it] doesn't work in others, and what we can learn from that to make better designs of these chimeric antigen receptors.'