A pioneering treatment manages to remit the leukemia of a girl in palliative care

A pioneering treatment developed at London’s Great Ormond Street Children’s Hospital has successfully put people in remission. the initially incurable leukemia that Alyssa suffered from, a 13-year-old girl living in the English city of Leicester. Alyssa was diagnosed in May 2021 with T-cell acute lymphoblastic leukemia and underwent unsuccessful chemotherapy and a bone marrow transplant, the Great Ormond Street Hospital for Children (GOSH) explains in a statement.

In May of this year, the patient, already facing palliative care, was the first to participate in a clinical trial with the new T-cell “base editing” technique with chimeric antigen receptors (CAR-T), in the Bone Marrow Transplant Unit of the medical center.

Thus, the girl received CAR-T cells (originally from healthy donors) but which, unlike what happens in usual treatments of this type, had been genetically modified especially to treat her type of cancer.

The GOSH doctors point out that, until now, it has been difficult to treat T-cell leukemia with the familiar CAR-T therapy, because T cells that are designed to recognize and attack cancer T cells end up killing each other as well. in the manufacturing process itself, before they can be administered to the patient.

What the teams at GOSH and its partner institute at UCL London did, was to devise a new type of CAR T-cell treatment that could effectively target cancer T-cells. The experts explain that the technique of “base editing” allows T cells to be changed by chemical conversion of individual letters of the DNA code (individual nucleotide bases).

With this genomic editing, the team made multiple changes to the T cells from healthy donors so that they would not be attacked by the patient’s own immune system. They then made sure that the modified T cells did not attack each other and were “invisible” to other cancer treatments.

Finally, a pathway was added for the modified cells to recognize and attack cancer T cells, the statement said. Thanks to biological engineeringdoctors created what can be considered “a living medicine,” it is noted.

28 days after being treated, the leukemia has already gone into remission and Alyssa received a second transplant of marrow to restore their depleted immune system, experts explain. Now, six months later, she is continuing her recovery at her home in central England, although she has to undergo regular check-ups to make sure the cancer has not returned.