Layla Richards, a one-year-old from London, has been treated with a ground-breaking procedure using gene therapy to successfully reverse the aggressive leukemia that threatened to take her life.
Layla was diagnosed with leukemia at the age of three months. Chemotherapy and a bone marrow transplant were unable to cure the condition, and doctors had advised the family there was nothing more they could do.
Layla's father, Ashleigh Richards, was told the best that could be done was palliative care. He said: I didn't want to go down that road, I'd rather that she tried something new and I took the gamble."
The biotech company Cellectis, together with medical staff quickly received permission to try an experimental therapy which had only been tried on mice in trials.
The treatment utilized designer immune cells" produced through cutting-edge gene therapy.
Where previous attempts at gene therapy tried to treat defects by adding genes, the new method edits the existing genes using microscopic scissors which are called Talens, to work with the DNA in donor immune cells.
The cells were then programmed to seek and destroy only the leukemia cells, and further programmed to remain invisible to chemotherapy given the patient.
The newly programmed cells were then injected into Layla, and she was also given another immune system restoring bone marrow transplant.
The results, just months after Layla's family was told her cancer was incurable, have given Layla a new lease on life, with no trace of leukemia in her body.
Her father Ashleigh said, 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."
Dr. Paul Veys, from Great Ormond Street Hospital in London where the procedure was done said, "We're in a wonderful place compared to where we were five months ago, but that doesn't mean cure." He continued, "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."
Dr. Veys said that the results of the therapy were "almost like a miracle".
Prof. Waseem Qasim, also from Great Ormond Street Hospital, added: "This is the first time human cells, engineered in this particular way, have been given back to a patient and that was a big step for us."
Prof Qasim added, "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."
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