US authorities have approved a new leukemia-based gene therapy, effective in 8 out of 10. Researchers have tested the treatment on 64 children with acute leukemia. Chemotherapy and other drugs had already proved useless to these children. Novartis's approach, however, saved their lives.
The treatment starts with the withdrawal of some patient blood samples. Physicians isolate T lymphocytes and bind them to the CAR-T receptor, so they detect a protein of the diseased cells. This allows them to target leukemic cells and destroy them within 3 months, leaving intact healthy cells.
Although the therapy has saved life to 8 children over 10, it has been shown to have even important side effects. It causes a decrease in pressure, pulmonary congestion and in some cases neurological problems. In fact, T lymphocytes release harmful substances for the functions of the body. However, there are consequences that can be faced and certainly preferable to an almost inevitable death.
The tests had already shown the efficacy of the treatment but lacked FDA approval for marketing. Thanks to the free way, doctors of authorized hospitals will take the T lymphocytes from the patients and send them to the company's offices. There some specialists will be able to engineer them within a month, so that they are injected into the patients.