A one-year-old girl in Britain has become the first person in the world to be treated with "genetically engineered" immune cells to fight cancer, doctors have said.
Layla Richards had leukemia, but was cured after scientists used a new technique that involves gene editing to “teach» the cells how to fight the disease, at the Great Ormond Street Hospital (GOSH) in London.
“Given that this was the first time the treatment was used, we didn’t know if it would work and we were over the moon when it succeeded”, said Professor Paul Veys, director of bone marrow transplantation at the Hospital and lead physician for Layla.
“ leukemia was so aggressive, that such a turnaround is almost a miracle.”
The baby was diagnosed with acute lymphoblastic leukemia, the most common form of childhood leukemia, when it was only 14 weeks old.
He/she underwent chemotherapy and bone marrow transplantation, but the cancer returned and the doctors informed the parents of the unpleasant news.
Subsequently, an experimental treatment that was under research at the hospital was offered to the family, where doctors modified white blood cells and T cells from a healthy donor in order to seek out and kill the drug‑resistant leukemia.
“She was sick and in a lot of pain, so we had to do something”, said Ashleigh Richards' father, 30 years old.
“The doctors explained that, even if we tried the treatment, there was no guarantee it would work, but we prayed that it would happen.”
Layla was given a small set of genetically modified cells known as UCART19 cells. A few weeks later, the doctors told her parents that the treatment had an effect.
The doctors emphasized that the experimental technique had only just been used and that the results need to be examined again, but they also said that it is potentially very promising.
«We have used this treatment only on a very strong little girl and we must be careful regarding the claim that this will be a suitable therapeutic option for all children», said Waseem Kasim, professor of cellular and gene therapy at the Institute of Child Health and a consultant oncologist at the Hospital.
«But this is a milestone in the use of new genetic engineering technologies and the consequences for this child were shocking», he added.
If it is repeated, it could represent a huge step forward in the treatment of leukemia and other forms of cancer».
