CAR-T, an experimental gene therapy cured a previously “incurable” leukemia in a teenager. Read to know more about the procedure and the miraculous recovery.
What is CAR-T?
Thanks to an experimental gene therapy paving the way with new genetic immunotherapy, 13-year-old Alyssa from Leicester, UK is free of “previously incurable” leukemia. The teenager was suffering from T-cell lymphoblastic leukemia, a condition affecting T-lymphocytes, a type of immune white blood cell created in the bone marrow. It is fast-moving cancer where cells divide and enter the bloodstream before traveling to the spleen, liver, and lymph nodes. The condition can lead to death in a span of months. While procedures such as bone marrow transplant and chemotherapy exist, they are difficult and were unsuccessful in Alyssa’s case.
Hence, doctors approved an experimental CRISPR-based therapy. The Chimerica Antigen Receptors Cell Therapy (CAR-T) is a new individualized method that uses T-cell samples for reprogramming the DNA base to fight cancer. Since each cancer is different, CAR-T therapy varies from patient to patient. It is different from typical gene editing and targets each base and changes them.
More on the experimental gene therapy
The CAR-T performs modification of T-cells to ensure they do not attack the patient’s cells and lead to autoimmune conditions. It also involves removing CD7, a cell marker, and then protecting the cells from chemotherapy. The new T-cells in the patient target any cell presenting the cell marker. While it is delicate, it has very few errors and ensures minimal or no complications.
“Since Alyssa got sick with her leukemia in May last year, she never achieved complete remission. Not with chemotherapy and not after her first bone marrow transplant,” stated Dr, Robert Chiesa. Dr. Chiesa is a consultant in CAR-T cell therapy and Bone Marrow Transplant.
“This is quite remarkable, although it is still a preliminary result, which needs to be monitored and confirmed over the next few months. We have been very impressed by how brave she is and nothing makes me happier than seeing her outside the hospital, going back to a more normal type of life,” he added.
