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Baby girl becomes first person in the world to receive ‘designer cells’

Great Ormand Street Hospital

"Designer cells" help reverse one-year-old girl's terminal cancer

Baby girl becomes first person in the world to receive ‘designer cells’

Layla Richards was diagnosed with incurable aggressive leukaemia at  just three months of age.

Just before her first birthday, doctors advised Layla’s mother and father to begin palliative care, giving up hope of being able to cure their daughter’s disease.

But it was when her father Ashleigh refused to give up, that Layla was given one last chance beat her diagnoses.

“I didn’t want to go down that road, I’d rather that she tried something new and I took the gamble,” Ashleigh told the BBC.

Medical staff then went about trialling the world’s first procedure that would see Layla implanted with ‘designer cells’.

“It was scary to think that the treatment had never been used in a human before, but even with the risks there was no doubt that we wanted to try the treatment. She was sick and in lots of pain, so we had to do something,” Ashleigh told The Guardian.

In partnership with the biotech company Cellectis, doctors at Great Ormond Street Hospital implanted the cells, that were designed to seek out and kill leukaemia cells, whilst leaving healthy cells intact.

The ethics committee rapidly approved the procedure, which had previously only been trialled on mice. The implanted cells were manufactured from frozen batches of donated white blood cells which had been altered with an extra gene to allow them to target leukaemia cells.

Following the treatment, Layla underwent a second bone marrow transplant to restore her immune system to its full strength.

“And this is her today standing laughing and giggling, she was so weak before this treatment, it was horrible and I’m just thankful for this opportunity.”

Now, her family are proud to announce that not only is Layla healthy and alive, but all traces of the cancer have seemingly vanished from her body.

[caption id="attachment_843555" align="alignnone" width="624"]Great Ormand Street Hospital Great Ormand Street Hospital[/caption]

One of the doctors responsible for treating Layla, Dr Paul Veys, told the BBC that the results were outstanding: “We’re in a wonderful place compared to where we were five months ago, but that doesn’t mean cure.

“The only way we will find out if this is a cure is by waiting that one or two years, but even having got this far from where we were is a major, major step.”

Whilst Layla’s story has been described as “a miracle”, her case was not part of a wider clinical trial and will be presented at the American Society of Haematology as a single case.

In saying that, her response to the designer cell treatment is promising for advancements in gene-editing technology.

According to Professor Waseem Qasim of Great Ormond Street, the procedure was a huge step in readying the technology for its next stage of development.

“The technology is moving very fast, the ability to target very specific regions of the genome have suddenly become much more efficient and we think that this technology will be the next phase of treatments.

“The technology itself has got enormous potential to correct other conditions where cells are engineered and given back to patients or to provide new properties to cells that allow them to be used in a way we can only imagine at the moment.”

Doctors say the treatment could be used to treat five-ten children with acute lymphoblastic leukaemia, in Britain, every year.

Doctors are hopeful that the same technology can be applied to the treatment of other cancers.

 

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