A highly experimental cell therapy saved the life of a young girl dying of leukemia. Performed at Great Ormond Street Hospital (GOSH), the procedure successfully destroyed the cancer cells in the girl, who was afflicted with Infant Acute Lymphoblastic Leukemia (ALL).
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One-year-old Layla received a devastating diagnosis: incurable leukemia. Over the course of the following year, Layla was subject to various treatments including chemotherapy and bone marrow transplant.
These treatments showed no affect.
Professor Waseem Qasim, consultant immunologist at Great Ormond Street Hospital, explains: “The approach was looking incredibly successful in laboratory studies, and so when I heard there were no options left for treating this child’s disease, I thought ‘why don’t we use the new UCART19 cells?'”. Layla is still on the path to recovery and is still a regular visitor to the hospital where she undergoes checks to ensure she is healthy.
The hospital had been working on an experimental cell-based treatment for leukaemia.
Now, she is cancer free, a response one doctor described as “almost a miracle”.
“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”.
If the success in this case is sustained and replicated in other patients, he said, the therapy “could represent a huge step forward in treating leukaemia and other cancers”.
The firm developed a gene-editing tool called TALEN that alters the genes of donor immune cells so they can kill leukemia cells but not attack the recipient’s healthy cells.
Following the treatment, Layla spent months in isolation but is now free of cancer and recovering at home in North London.
The cells came from frozen batches of donated T cells, or white blood cells, which play a central role in human immunity. Though there was no guarantee it would work, they agreed to try it.
Layla was given the 1ml infusion that contained genetically engineered cells.
MERLIN CROSSLEY: Any cancer which is characterised by one particular surface molecule that you could generate these killer t-cells against would be a good candidate for treatment but not every cancer is of that type.
“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”, he continued.
Ashleigh Richards, the father, said that the prospect of an untested treatment was scary and frightening, but their daughter was suffering in pain so they decided to choose the experimental treatment. The treatment also involved engineering a donor’s T cells, according to The Scientist.
Chemotherapy and bone marrow transplants had failed to stop Layla Richards’ cancer and time appeared to be running out.
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Qasim and colleagues will present the results of Layla’s therapy December 5 at the annual meeting of the American Society of Hematology in Florida.
