First-ever gene-altering leukemia treatment approved by the FDA, uses patient cells to fight cancer

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Emily Whitehead was the first child to receive experimental gene therapy

The Food and Drug Administration on Wednesday approved the first-ever treatment that genetically alters a patient’s own cells to fight cancer, a milestone that is expected to transform treatment in the coming years.

The new therapy turns a patient’s cells into a “living drug,” and trains them to recognize and attack the disease. It is part of the rapidly growing field of immunotherapy that bolsters the immune system through drugs and other therapies and has, in some cases, led to long remissions and possibly even cures.

There are drawbacks to the approach. Because [the therapy] Kymriah can have life-threatening side effects, including dangerous drops in blood pressure, the F.D.A. is requiring that hospitals and doctors be specially trained and certified to administer it, and that they stock a certain drug needed to quell severe reactions.

Kymriah, which will be given to patients just once and must be made individually for each, will cost $475,000. Novartis said that if a patient does not respond within the first month after treatment, there will be no charge.

“This is a big paradigm shift, using this living drug,” [pediatric oncologist Kevin Curran] said. “It will provide a lot of hope. This is the beginning.”

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000