The first gene therapy treatment approved by the FDA and available in the United States is tisagenlecleucel for pediatric and young adult patients with leukemia. Tisagenlecleucel is a treatment for relapsed or refactory B-cell precursor acute lymphoblastic leukemia. The FDA has approved treatment for patients up to 25 years old.

Childhood acute lymphoblastic leukemia, abbreviated as "ALL", is a type of cancer in which the bone marrow makes too many of an immature type of white blood cells called lymphocytes.

Tisagenlecleucel therapy uses the patient's own immune cells to target their cancer. This is accomplished by collecting the patients T cells and sending them off to a laboratory where they are genetically modified. The T cells are modified with a receptor to target the surface of the cancer cells. After the T cells are genetically modified they are injected back inthe the patient to kill cancer cells.

Sources and Full Articles

FDA Approves First Gene Therapy for Pediatric Leukemia, Cancer Network, 1 September 2017

Childhood Acute Lymphoblastic Leukemia Treatment (PDQ®)–Patient Version, National Cancer Institue

Thank You!