A highly experimental cell therapy saved the life of a young girl dying of leukemia.
In a press conference held by Great Osmond Street Hospital, a children's hospital located in London, last Thursday, Nov 5, doctors proudly presented the case of one-year-old Layla who was diagnosed with acute lymphoblastic leukemia (ALL) when she was just three months old.
Layla was initially treated with chemotherapy and later a bone marrow transplant in the hospital. However, due to factors such as her age, she eventually relapsed, and her condition worsened. The family traveled to Sheffield to participate in a trial, but it also didn't work out. She was dying.
Back then, her only option was palliative care, but her parents were not giving up. It was then they learned about an experimental cell therapy project called gene editing.
Gene editing is one of the major projects worked on by scientists and doctors of the hospital and the UCL Institute of Child's Health, as well as by Cellectis, a biotech company, and University College London. In this scenario, certain genes are cut using accurate "molecular scissors" and then added to healthy T cells that came from donors. The technique then allows these modified cells, now called UCART19 cells, to target only leukemic cells while remaining "invisible" to possibly strong cancer drugs, which would normally wipe out both healthy and cancerous cells.
Since the team has been working on gene editing for some time and is preparing for the final stages of the clinical trial, they have stored some of these donated T-cells. After the family received permission from the ethics committee, a 1ml of these modified cells was infused intravenously, and the procedure was over in 10 minutes.
The procedure was risky to say the least. Although early trials resulted to positive outcomes, it's never been tested on mice. But over many months of isolation to avoid compromising the immune system, the child remained well overall, proof that the treatment was working. Once the doctors cleared Layla of leukemia, she was then transplanted with a new bone marrow. Now she's resting well and has remained cancer free.
To learn more about the revolutionary treatment, click here.
