MIAMI: The United States approved the first gene therapy in the nation on Wednesday — a treatment that uses a patient’s own immune cells to fight leukemia — opening a new era in the fight against cancer.
The treatment is made by Novartis and is called Kymriah, (tisagenlecleucel). This type of immunotherapy, known as a CAR-T cell therapy, was known by CTL019 until now.
It was approved by the US Food and Drug Administration for children and young adult patients up to age 25 with a form of acute lymphoblastic leukemia (ALL).
The FDA described the approval as “a historic action” that would usher “in a new approach to the treatment of cancer and other serious and life-threatening diseases,” said a statement.
Studies have shown that 83 percent of patients responded to the treatment, achieving remission within three months.
The treatment is not a pill or a form of chemotherapy.
It uses a patient’s own immune cells, called T-cells, along with white blood cells.
These cells are removed from a patient, sent to a lab, and encoded with a viral vector, reprogrammed, and returned to the patient.