Alyssa's life has taken a 180 degree turn in the last year. The 13-year-old British girl had been diagnosed with T-cell acute lymphoblastic leukemia, a disease that she had not previously responded to either chemotherapy or bone marrow transplantation. Her prognosis was not encouraging.
However, a new innovative therapy has managed to remit leukemia in many cases lethal that gives hope to those who suffer from this type of cancer. The phase I clinical trial has turned out to be a success for Alyssa, information already announced at the end of 2022, but also for a second patient, as The New England Journey of Medicine reported this Wednesday.
The medical journal has published that the treatment in a 15-year-old adolescent has achieved "deep" remission of the disease; This was not the case with another 13-year-old patient, who responded to what is known as CAR-T therapy, but suffered a lung infection due to the Asperfillus niger fungus and died.
White blood cells were extracted from patients in order to redesign them in the laboratory using base editors, a gene editing technique capable of more precisely and safely modifying the genome than the tools provided by CRISPR (gene editing technique) tools. revolutionary that acts as an immune mechanism against traditional viruses). It is done with a kind of pencil with an eraser that eliminates cancer cells once it identifies them.
According to research from University College London led by scientist Waseem Qasim, base editors inactivate three genes in white blood cells in order to prevent them from killing each other, make them resistant to parallel cancer treatments and make them fit for use. donated.
"This is our most sophisticated cell engineering yet and it paves the way for other new treatments and ultimately better futures for sick children," Qasim said after the good results obtained with Alyssa. The researchers now want to complete the trials with a dozen more minor patients, between six months and 16 years.
The three teenagers' treatments have been carried out at London's Great Ormond Street Children's Hospital, partially financed by the copyright of Peter Pan, by Scottish writer James Matthew Barrie, who donated them to the institution in 1929.
