At England’s Great Ormand Street Hospital, a one-year old was successfully treated for Acute Lymphocytic Leukemia (ALL) using a healthy donor’s T-cells modified to target her cancer. In a matter of weeks she was cancer free, with hopes for permanent remission.
The technique involves genome editing, allowing the use of donor cells rather than having to harvest the patient’s own T-cells, which in the baby’s case had already been destroyed. The novel therapy included a genome editing technique, using molecular scissors to cut out a gene which prevented the transplanted T-cells from attacking the patient’s own cells as “foreign”, as well as inactivating another gene to help the transplanted T-cells survive in the patient’s body.
This “miraculous” treatment had previously been tested only on mice, but once chemotherapy failed to stem the disease, the baby’s parents were prepared to take every step to save their daughter’s life. With approval by the hospital’s ethics committee, the gamble paid off: in a matter of weeks she was cancer free, with hopes for permanent remission.
The baby had not been helped by conventional treatments but after this first ever treatment developed by Cellectis, a French biotechnology company, she shows no trace of leukemia. By using healthy donors’ T-cells, this new therapy could potentially apply to any patient with a particular type of leukemia without being customized, saving time and money.
ACGT scientists including Dr. Carl June and Dr. Michel Sadelain are pioneers of similar immunotherapy approaches which genetically alter patient’s T-cells, the soldiers of the immune system, but use the patient’s own modified T-cells to attack the cancer cells.