Layla, is a one-year-old girl who has been suffering from leukaemia, a type of cancer which affects the blood.
The day before her first birthday, Layla Richards’ parents were told their daughter was about to die. Doctors told them she had “one of the most aggressive forms of the disease we have ever seen.” So, they desperately tried to save her life with a new treatment; it worked.
Layla’s condition will not respond to available treatment, but finally the Cancer got halted, thanks to a gene-editing technology that allowed her to receive modified immune cells from another person.
Her case, first reported by Nature.com, represents the second trial of gene-editing as a therapy — the first was carried out last year in patients with HIV. More similar trials are planned — and companies are also preparing to trial therapies that inject DNA that codes for gene-editing enzymes directly into the human body.
The team which treated the girl said it had planned to start a safety trial next year in 10–12 patients. But when the researchers came across the baby, in whom all other treatments had failed, they were able to obtain special permission to treat her with the new technology. Several months after the procedure, the baby is doing well.
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The team will present the case in December at an American Society of Hematology meeting in Orlando, Florida.
How it is done
To administer the therapy, the researchers extract immune cells called T-cells from a healthy donor, and expose them to a type of DNA-cutting enzyme called a TALEN. The enzyme deactivates immune genes that would otherwise cause the donor cells to attack when injected into a person with leukaemia, and modifies genes to protect the new cells from anti-cancer drugs that the patient is taking.
The individual then undergoes therapy to destroy his or her own immune system, which is replaced with the modified cells. The treatment is not a permanent solution for leukemia patients, but rather a ’bridge‘ to keep the person alive until a matched T cell donor can be found.
This news first appeared in the Nature