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New gene therapy for leukemia: FDA panel advises approval


Alexander Raths - Shutterstock

J-P Mauro - published on 07/13/17 - updated on 07/13/17

The one time treatment boasts higher success rate than chemotherapy

An FDA panel of cancer experts endorsed a new treatment for leukemia. The group met Wednesday and voted unanimously to recommend the treatment’s approval. The FDA is not required to adhere to their suggestion, but if they do, this could be the first gene therapy to be possible in the US.

Fox News reports, the treatment is meant for children and young adults, ages 3-25, who are afflicted with the advanced stages of Leukemia:

CAR-T therapy starts with filtering key immune cells called T cells from a patient’s blood. In a lab, a gene is then inserted into the T cells that prompts them to grow a receptor that targets a special marker found on some blood cancers. Millions of copies of the new T cells are grown in the lab and then injected into the patient’s bloodstream where they can seek out and destroy cancer cells. Doctors call it a “living drug” — permanently altered cells that continue to multiply in the body into an army to fight the disease.

The panel heard from the fathers of two patients whose lives have already been saved by the experimental gene therapy. Of the 52 patients analyzed, 83% were in complete remission. The side affects are severe, but nearly all recovered.

While the treatment is very expensive, as a one time procedure it may have a net effect of lowering medical costs, and is expected to be more effective than chemotherapy.

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