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FDA Approves First Gene Therapy for Cancer in U.S.


FDA approval has been secured for a revolutionary cancer treatment option, the first of its kind to be approved in the US. The treatment is called Kymriah, which is a cell-based gene therapy, or immunotherapy. It was approved to treat a specific form of acute lymphoblastic leukemia (ALL) in children and young adults. According to the FDA, ALL is the most common childhood cancer in the US. This is a cancer that affects the blood and bone marrow, caused by the body producing abnormal lymphocytes, or white blood cells. The treatment works by harvesting T-cells (a type of lymphocyte) from a patient’s body, genetically modifying them, and putting them back into the patient’s body to fight the disease. This is basically weaponizing the body’s natural defense system against specific cells that would otherwise go undetected by that system.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” according to FDA Commissioner Scott Gottlieb, M.D. He goes on to say, “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”

This treatment is intended as an option after other forms of treatment that have failed. While it has been proven to be effective, (with an overall remission rate of 83%) it comes with the potential for severe side effects, including infections, that can become life threatening.

According to the FDA’s Dr. Peter Marks, “Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease.” He adds, “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

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