A 13-year-old British girl with incurable cancer is now leukaemia-free after being the "first human patient" to receive successful treatment from base editors.
Alyssa, whose last name has not been shared, received base-edited T-cells from a healthy donor, which were edited using new technology to kill cancerous T-cells.
In 2021 Alyssa received all of the conventional treatments, from chemotherapy to a bone marrow transplant, but her cancer returned.
Broad Institute of Harvard Vice-chair Dr David Liu told AM the base editors target a specific location in humans' vast genomes and change an individual DNA letter to a different letter, "which can correct a misspelling that can cause a disease".
Dr Liu said the 13-year-old is the first human patient to see the benefit of base-editing.
He said the leukaemia in Alyssa had come to a "serious state" before the base-editing treatment, but after six months that cancer had not been detected again, "clearing her T-cell leukemia".
Dr Liu said he is "excited" to see where the "programmable" treatment leads next.
"We developed them so that they could be targetted to specific positions in the human genome of our choosing, taking advantage of the amazing work done by scientists," he said.
"Base-editing can be broadly applied and indeed there are four ongoing base-editing clinical trials I'm aware of, of which the UCL (University College London Cancer) one is the first that was dosed into a patient."
Watch the full interview above.
