Baby girl with cancer saved by cell therapy

While this is only one case study, what’s really exciting is that it’s the first evidence that using edited versions of generic, non-personalised cells could work as an effective therapy, paving the way for more affordable and accessible treatment options for a range of cancers.

The announcement was made by doctors in the United Kingdom at Great Ormond Street Hospital (GOSH), saying the treatment was only previously tested in the laboratory. The treatment was succesful as she improved through the treatment whereas chemotherapy and bone marrow transplant did not.

These treatments showed no affect.

To combat the onset of cancer in little Layla’s system, scientists used a new gene-editing technique to manipulate immune cells to fight the disease. Her family agreed to the treatment, administered via intravenous injection, and two years later Layla was cancer free. But over many months of isolation to avoid compromising the immune system, the child remained well overall, proof that the treatment was working. “Her leukaemia was so aggressive that such a response is nearly a miracle”, professor Paul Veys, Layla’s lead clinician, said, in a hospital press release.

Dr. Qasim cautioned that the treatment worked very well for Layla, but there is no indication that it will work for other children with leukemia, and that more research needs to be done in using gene engineering technology to treat leukemia and all other cancer types.

Professor of cell and gene therapy at University College London and consultant immunologist at GOSH, Waseem Qasim said it was a “landmark” development and the effects for Layla have been staggering.

“If replicated it could represent a huge step forward I treating leukaemia and other cancers”.

Months after the treatment, Layla is now home with her older sister, enjoying the things children do.

Microscopic scissors – technically known as Talens – were used to precisely engineer the DNA inside a donor’s immune cells.

The cells were extracted from donated batches of frozen T cells, or white blood cells – which play a central role in immunity.

Because this treatment is at an early stage, it was only sanctioned because the girl had no other hope of surviving the disease and her parents were willing to try it. It’s been very successful, says Dr. Renier Brentjens of the Memorial Sloan Kettering Cancer Center in New York.

“She was sick and in lots of pain so we had to do something”.

The doctors tried several forms of treatment including several chemotherapy sessions and even a bone marrow transplant. For patients like Layla, however, there aren’t enough T-cells to collect and engineer. The hospital said the treatment added new genes to the healthy donor cells.

On Wednesday the results of Layla’s case will be presented at the American Society of Hematology’s annual meeting, even as doctors keep close watch to see if the results in Layla’s case are sustained.