US FDA Approves Gene Therapy For Childhood Leukemia

The United States Food and Drug Administration (FDA) has approved the first-ever gene therapy treatment for battling childhood leukemia, a type of blood cancer that affects young children. This marks a first in drug history, as the gene therapy called Kymriah developed by Novartis Pharmaceuticals is the first of its kind in the U.S. market.

According to ABC News, the approval paves the way for Novartis to make the therapy available to the market. Each one-time infusion costs US$ 475,000 or about P24.3 million.

How New Gene Therapy Works

The new therapy, officially called Kymriah, is made up of genetically-engineered blood cells or “CAR-T” cells which when infused into the patient, will destroy the cancer cells that cause childhood leukemia. Each treatment is custom-made from the immune cells of the patient.

According to a CNN report, it starts with a bag of immune cells from the patient which is taken to a lab where it will undergo the chimeric antigen receptor T-cell (CAR-T) therapy, which gives the cells “the ability to recognize and kill the source of the cancer.” One commissioner of the FDA has touted this new innovation as the start of a new frontier in cancer treatment.

The FDA approval comes after years of clinical research showed that patients who were on the treatment had an 89% chance of surviving for at least 6 months, and a 79% chance of surviving at 1 year. According to BBC News, the first child treated with CAR-T has been cancer-free for more than 5 years now. Long-term tracking for patients who participated in recent studies however are still ongoing.

Hope For ALL Patients

The treatment brings hope to those affected by acute lymphoblastic leukemia (ALL), half of which are usually young children or teenagers. It is, after all, the most common type of cancer among children in the United States.

FDA, however, warns that the therapy is not without its side effects. The treatment may trigger an overreaction in the patient’s immune system called “cytokine release syndrome” which can be life-threatening. This is the reason why only hospitals and medical facilities equipped with state-of-the-art equipment can provide the therapy, according to the FDA.

The treatment also offers a breakthrough in cancer treatment research as researchers look into the other uses of immunotherapy-based treatments through the use of CAR-T cells.