The FDA has approved the first genetically modified cell therapy to reach the market. The treatment, that was created to fight blood cancer, will be marketed by Novartis under the name Kymriah and focuses primarily on the treatment of acute lymphoblastic leukemia (ALL), one of the most common cancers in children.

The treatment, CAR-T cell therapy, uses an immunotherapy approach called Adoptive Cell Transfer and consists of extracting T-cells from the patient’s blood and then modifying the exterior of the cell by adding a chimeric antigen receptor (CAR) that looks for cancerous cells. Once the modified cells are put back into the patient, they target cancer cells. The medical community believes that these types of personalized treatments have great potential for other blood-related cancers.

Five years ago Novartis, which is charging US$475,000 for each therapy, started collaborating on the research at the University of Pennsylvania. “As a breakthrough immunocellular therapy for children and young adults who desperately need new options, Kymriah truly embodies our mission to discover new ways to improve patient outcomes,” said Bruno Strigini, CEO of Novartis Oncology.

According to Novartis the side effects of this treatment include high fever, inflammation, vomiting and muscular pain, among others, mostly caused by cytokine release syndrome (CRS), which is related to the immune response of the infused modified T-cells.


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