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In a Heartbeat

Cancer treatment wins FDA approval

| Tuesday, July 18, 2017, 11:43 a.m.

An FDA advisory panel has approved a first-of-its-kind cancer treatment that has shown remarkable results in treating acute lymphoblastic leukemia. Known as CAR-T cell therapy, or gene therapy, the treatment put more than 80 percent of clinical trial patients in full remission. It is also being tested for the treatment of other blood cancers such as non-Hodgkin's lymphoma, chronic lymphocytic leukemia and multiple myeloma. CAR-T cell therapy could win full FDA approval by the end of September. Dr. Cyrus Khan, an Allegheny Health Network oncologist, provides more details below.

Why is this such a significant development?

The clinical trials utilizing this kind of therapy have shown unprecedented results in various forms of blood cancers despite the patients having very advanced and refractory forms of the disease. The treatment has the potential to keep on working for many years after one infusion.

What is the process for treating a patient with CAR-T cell therapy?

In a nutshell, the patient's own T-cells (a form of white blood cells the immune system uses to fight infections and cancers) are collected and modified in the lab by adding to them very specific receptors that make them capable of recognizing and binding to the patient's specific cancer. These cells are then grown and expanded and re-infused into the patient where they immediately go to work in finding and killing off the cancer.

Are there any downsides to the treatment?

Yes, some, including a side effect known as cytokine release syndrome. The therapy's immune-system boost releases chemicals that cause inflammation in the body, which can lead to symptoms ranging from mild, flu-like effects to severe problems such as low blood pressure, organ damage. brain swelling and seizures.

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