Experimental Cancer Treatment That Uses CRISPR Gene-Editing Put Young Girl Into Remission

A young girl with an aggressive form of leukemia who has not responded to other treatments now has no detectable cancer cells in her body after receiving an experimental therapy of genetically edited immune cells that attack cancer.

The Great Ormond Street Hospital for Children (GOSH) reports that Alyssa, 13, seems to be in remission now, but doctors are still closely monitoring her condition in upcoming months to confirm that she is truly leukemia-free. GOSH is a London-based children's hospital that provides treatment.

Experimental Cancer Treatment That Uses CRISPR Gene-Editing Put Young Girl Into Remission Pixabay/madartzgraphics

Alyssa's Story

According to GOSH, Alyssa was diagnosed with T-cell leukemia in May 2021 after a long period of suffering from various symptoms that the family initially thought were due to colds, viruses, and general tiredness,

She was treated in hospitals in Leicester and Sheffield with chemotherapy and a bone marrow transplant, but unfortunately, doctors could not control her cancer and put it into remission. The only option left was palliative care which Alyssa and her family have extensively discussed with experts in BMT and CAR T-cell therapy and hematology at GOSH.

Alyssa became the first patient in the world to receive base-edited cell therapy for her leukemia. She is currently at home recovering from her treatment, and both she and her family are feeling positive that the cancer cells are now undetectable, although they know that they need to be closely monitored.

Alyssa said in a video released by GOSH that she is very honored and feels good to have helped others as well. Her doctors aim to enroll a total of 10 patients between the ages of 6 months to 16 years for their ongoing trial. The Bone Marrow Transplant (BMT) and CAR T-cell therapy experts at GOSH hope that a successful trial could result in cancer treatment for children and could be an option for leukemia.

Gene-Editing Immune Cells Using CRISPR

Scientists at GOSH stripped donated T-cells of certain receptors to make them look foreign to the patient's immune system for their new therapy, LiveScience reported. The cells now lose the protein CD7 and another protein called CD52, which is the target for certain cancer treatments. Lastly, they inserted a new receptor into the T-cells to let them target CD7-carrying T-cells, including cancer cells.

The team used a modified form of CRISPR, called base-editing, to swap out individual letters in the DNA code in the T-cells and essentially apply all these genetic changes. After one month of receiving a base-edited CAR-T cell therapy dose, Alyssa entered remission.

She also received a second bone marrow transplant to restore her immune function after her T-cells were wiped out from the experimental therapy. It has been six months, her cancer remains undetectable, and she is recovering at home. Her family is hoping for Alyssa to recover and return to school soon.