Layla Richards was only three months old when she was diagnosed with acute lymphoblastic leukaemia, an aggressive and incurable cancer of the body’s blood-forming tissues. Far from being the sickly baby whose illness was a constant source of agony a few months ago, Layla is now thriving, all thanks to a ground-breaking new treatment using “designer” cells – that is, genetically engineered immune cells – to fight the disease.
Her only hope
It was the day before her first birthday when, after failed attempts at chemotherapy and a bone marrow transplant, Layla’s medical team suggested palliative care. But her parents, Ashleigh and Lisa, refused to give up. This is when doctors at London’s Great Ormond Street Hospital (GOSH) offered the family the option of a new treatment, a highly experimental genetic therapy that had only been previously tested in trials on mice.
While aware that there were no guarantees that the treatment would be successful at all, it was also clear that taking a gamble was the little girl’s only hope.
The science behind the miracle
The therapy used to treat Layla used a transfusion of “edited” cells from a healthy donor, in which a type of white blood cell known as T cells were modified to seek out and kill the leukaemia cells which up until that point had proved untreatable.
These designer cells, known as UCART19 cells, were created using TALENs, a biological editing tool that can be used as “molecular scissors” to modify a single gene in the DNA structure – in this case to programme the cells to target Layla’s cancer cells.
As Professor Waseem Qasim, a professor of cell and gene therapy at the Institute of Child Health as well as a consultant immunologist at Great Ormond Street explained, while this technique has only been used once and still needs to prove itself on a greater scale, “this is a landmark in the use of new gene engineering technology and the effects for this child have been staggering”.
Months have now passed since the treatment and not only has her health improved, but there is no trace of leukaemia to be found in Layla’s body. As for Layla, the implications of this technology could be the difference between life and death for many cancer patients in the future.
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