First CAR T-cell gene therapy approved in the USA

On August 30,2017, the US Food and Drug Administration (FDA) made the first gene therapy available in the United States: They approved Kymriah, a cell-based gene therapy, for the treatment of patients up to the age of 25 with acute lymphoblastic leukemia (ALL).

The CAR T-cells potential has been detected already 20 years ago and research in the field has been pursued ever since. Over the past few years, researchers have developed an ever growing understanding of how therapies with CAR T cells work in patients. However, translation into clinic has proven to be difficult. This has been shown in an evaluation performed by Paul-Ehrlich-Institut (PEI) in the CARAT project where scientists examined over 200 clinical studies. Despite the difficulties in clinical development, the severe side effects and the enormous costs of the treatment, it has also been revealed that there is a great benefit and potential of CAR T cell therapy for the treatment of certain types of blood cancer. The FDA’s decision now is a breakthrough as it is the first green-light for gene therapy.

If you want to read more, please see PEI’s publication in EMBO Molecular Medicine.

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