In November 2015, there was widespread media coverage of the case of Layla Richards from London. She had been diagnosed with acute lymphoblastic leukaemia (ALL) when she was only three months old. Conventional treatments had failed and Layla’s parents were told there was nothing more that could be done except….
..Layla’s doctors at Great Ormond Street Hospital sought permission to try a highly experimental therapy that had only been trialled in mice.
The therapy involved the use of microscopic “scissors” to edit the DNA in immune cells taken from a donor. These new immune cells were designed to behave in two specific ways. Firstly, the cells become invisible to a powerful leukaemia drug that would usually kill them. Secondly they target and fight only leukaemia cells.
The treatment was due for final stage testing. But after an emergency ethics committee meeting, permission was granted for Layla to be given the experimental therapy. It took less than 10 minutes to administer.
And with that, Layla and her doctors made history. It’s a truly remarkable story – Layla went from having incurable leukaemia to being leukaemia free within weeks.
Professor Waseem Qasim is leading the laboratory studies and helped to make this treatment available for Layla. He says…
“We have only used this treatment on one very strong little girl, and we have to be cautious about claiming that this will be a suitable treatment option for all children. But, this is a landmark in the use of new gene engineering technology and the effects for this child have been staggering. If replicated, it could represent a huge step forward in treating leukaemia and other cancers.” Professor Waseem Qasim, Professor of Cell and Gene Therapy at UCL Institute of Child Health and Consultant Immunologist at GOSH
It’s still early days. It’ll be a year or two before doctors know whether Layla is actually cured and she’ll be closely monitored in the meantime.
A clinical trial to test the therapy in larger groups of patients will get underway in 2016.