Babies Receive 'Miracle Cure' For Terminal Cancer With Gene-Edited Cells
Two babies have gotten a longer lease in life following their terminal cancer diagnosis thanks to a "miracle cure." British medical experts from the Great Ormond Street Hospital have found a way to infuse gene-edited cells to improve their immune systems and the results of the treatment have shown promise so far.
The experts published the babies' case and their progress in the Science Translation Medicine journal. The report outlined how the experts used donor blood to create and engineer T-cells to attack the cancer cells in the babies' bodies.
Experts dubbed the treatment developed as UCART19 therapy. The procedure has only been undertaken with the babies for a year but doctors are reporting that the girls are now in remission.
Layla Richards is the first person to receive the gene-edited treatment when she was diagnosed with cancer at three months and none of her previous medical treatments worked. Doctors found a second baby girl with cancer soon after Layla's case and entered her in the treatment program as well.
"Both patients are now at home and are doing well," immunologist Waseem Qasim said, Fox News reports. The babies, however, remain under strict monitoring. Their cases continue to help the experts determine if the treatment will also be viable for other cases, including adults with cancer.
One of the caveats with the treatment involved the babies developing graft versus host disease (GVHD). It is believed that the donor blood triggered the reaction, especially in Layla's case. The baby had GVHD lasted for two months, so she was also treated using steroids and had bone marrow transplant.
There are skeptics against the treatment, however, MIT Technology Review reports. The babies apparently also received chemotherapy that might have been a factor in their progress. "There is a hint of efficacy but no proof," Children's Hospital of Philadelphia director Stephen Grupp said about UCART19.