Once in her body, they spent three months seeking out and killing the leukaemia.
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Layla was diagnosed with an aggressive form of leukaemia when she was only 14 weeks old.
A few weeks later, Mr Richards called Ms Foley.
But the little girl’s family took the only option and rejected that too. It might not have any effect on Layla’s leukemia, the doctors warned. He told the BBC: “I didn’t want to go down that road, I’d rather that she tried something new and I took the gamble”. “We had to do something”.
A one-year-old girl who was suffering from “incurable” leukemia has been cleared of the cancer after becoming the first human in the world to try a new, experimental “off-the-shelf” therapy that creates designer immune cells. Her story was shared at the American Society of Haematology, but this is only a single case – medics are now discussing the possibility of clinical trials, to better test and understand how this technique works.
Another patient is now receiving the treatment under a different medical team.
She added: ‘Whilst it is still very early days, this pioneering treatment gives us a glimpse of one of the potential future applications of genetic medicine to create novel, personalised treatments for serious disease’.
“Recent developments have led to treatments where immune cells, known as T-cells, are gathered from patients and programmed using gene therapy to recognise and kill cancerous cells”.
The new treatment is significant because it indicates that cell therapies might not have to be customized for each patient, saving time and money.
A team at the hospital has now used modified T-cells from donors – known as UCART19 cells – to treat Lyala, who had unsuccessful chemotherapy and for whom palliative care was deemed the only option left. Firstly, the cells became invisible to a powerful leukaemia drug that would usually kill them and secondly they are reprogrammed to only target and fight against leukaemia cells.
Doctors at GOSH are not certain whether the dramatic effects will be replicated with other patients, but have still heralded it as a major milestone.
The first “off the shelf” banks of donor T-cells, called UCART19, were still being tested when Layla’s parents asked if there was anything else the doctors at GOSH could do for their daughter, according to a hospital press release.
A consultant immunologist at GOSH who had been working on the therapy, Waseem Qasim of UCL, suggested UCART19.
In these patients – such as Layla – cancer cells can remain hidden or resistant to drug therapy.
“She was sick, and in lots of pain, so we had do something”.
“We thought that the little bit of liquid in the syringe was nothing and asked, ‘what’s that going to do when bags of chemo haven’t worked?”
The nurse reassured them.
‘The nurse said it was about quality and not quantity though.
She developed neither over a two-week period, however, just as doctors were preparing to send her home – Layla developed a rash but otherwise seemed well.
The rash was a telltale sign that her body recognized and was responding to the infusion of foreign cells. Sit down, he told her.
But seven weeks later, the family was told that Layla’s cancer had returned.
Two months later, Layla was given a transplant to replace her bone marrow which had been affected by the treatment, and one month later she was able to go home.
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“As this was the first time that the treatment had been used, we didn’t know if or when it would work and so we were over the moon when it did”, said Professor Paul Veys, director of bone-marrow transplant at GOSH and Layla’s head doctor.
