What is it about?

A new class of cancer therapies is based on a patients' own immune cells, which are reprogrammed to so-called "CAR T cells" and then able to target and eliminate cancer cells. These therapies showed remarkable therapeutic effects in clinical trials but also caused severe toxicities in many patients. Early detection and treatment of these toxicities is critical to prevent adverse outcomes and to maximize the therapeutic benefit. A key question in the translation and regulation of new therapies is how regulators can facilitate their safe use and make new treatment options available to patients while ensuring proper risk management and collection of meaningful real-world data. Our analysis suggested that optimized management guidelines can help to reduce the number of severe adverse reactions in clinical trials. A review of adverse reaction reports showed that important details were often missing and a more structured recording of such reactions might be necessary. Finally, a survey of specalisted treatment centres offered ideas for improving regulatory requirements around the use of CAR T cells and the periodical review of such requirements.

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Why is it important?

The development of new CAR T cell therapies has expanded rapidly in recent years and challenges remain in the clinical translation and regulation of these gene therapies. Evaluating regulatory measures can support future decision making and access to and effectiveness of such therapies.

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This page is a summary of: Regulatory Measures to Improve the Safety of CAR-T-Cell Treatment, Transfusion Medicine and Hemotherapy, November 2022, Karger Publishers,
DOI: 10.1159/000526786.
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