A little girl is now the smiling face of proof of a new miracle treatment working to win the battle against an aggressive, formerly untreatable leukemia that was doomed to take her young life. Doctors at Britain’s Great Ormond Street Hospital (GOSH) stepped in to use an experimental treatment for the first time, as a last ditch effort, to save the sweet baby’s life. The gene-edited cell treatment proved to work the miracles that have now deemed Layla as a cancer-free 1-year-old.
GOSH happily tweeted, “1-yr-old Layla is now cancer free after world first use of gene-edited immune cells at GOSH.”
As reported by ITV News, the little girl originally received the standard treatments of both chemotherapy and a bone marrow transplant, neither of which had worked to rid her tiny body of cancer. Diagnosed at 3-months-old, Layla’s parents were told by doctors in June that there was no more that could be done to fight the cancer attacking their child’s life. Without any more hope, doctors delivered the horrifying news to the parents that all that was left to do was to take their daughter home and to make her as comfortable as possible with palliative care.
“Palliative care was not an option for me as a parent,” Layla’s dad, Ashleigh, told ITV News. “That’s not an option – I would try anything for my child.”
The little girl was given a chance at a reprieve just at that moment in a “really strange sequence of events,” as described by her doctor, Professor Paul Veys, Director of Bone Marrow Transplant at Britain’s Great Ormond Street Hospital. An experimental treatment was being worked on at the University College London’s Institute of Child Health next door to the hospital.
The treatment was in the extremely early stages of testing and had never been tried on humans. With no other options to save Layla from the destructive cancer, her parents took the offer by the doctors and the research team to try the experimental treatment on their daughter. Cells were genetically edited and given to Layla in a one-milliliter intravenous infusion.
Lisa, the mother of the little girl, explained to ITV News that they were told that there was no premise to understand how this treatment may affect Layla.
“The treatment may make her really sick, she may die from the treatment, the treatment can have really bad side-effects – they just didn’t know, they’d never used the treatment before and if we wanted to try a curative option then we could do it but it’s just not guaranteed, nobody knew.”
Great Ormond Street Hospital Children’s Charity posted the good news on their Facebook page.
The experimental cure used on the little girl involves creating designer immune cells with an “off-the-shelf” cell therapy developed by the French biotech firm Cellectis, as described in a recent Reuters report. It is designed to work by adding new genes to healthy donated immune cells known as T-cells, which arm them against leukemia. TALEN, a gene-editing technology, is used to cut the T-cells to allow them to behave in two different and specific ways. Cells are first made invisible to the leukemia drug used to commonly kill them. The new cells are then set on a course to aim specifically for the leukemia cells, in order to fight against them.
The little girl received the minuscule dose of life-saving cells in only a matter of minutes. The treatment went to work very quickly. Within two months, Layla was home from the hospital cancer-free.
“Her leukemia was so aggressive that such a response is almost a miracle,” said Paul Veys, a professor and director of bone marrow transplant at GOSH who led the team treating Layla.
Professor Veys states that full clinical trials funded by Cellectis will be beginning next year.
“To have a weapon like this which can cure this disease which we’ve just not had before is absolutely fabulous but we need to now learn how to use it properly. Layla has taught us hugely… which means for the next patient we’ll understand a lot more before we start.”
With Layla being the first human that the treatment has been used on, researchers are now seeking to see if there will be side effects and if it is the true answer for a long-term cure. The hope is that Layla will remain cancer free and that the life-saving treatment can be duplicated for additional cancer patients suffering from leukemia as well as other cancers. The sweet child’s renewed health and happy smile provides hope for other cancer patients. The new experimental treatment holds the promise of a possible cure for cancer sufferers, as it proved to battle the cancer that was dooming the life of the little girl.
[Image via Great Ormond Street Hospital]