FDA approves first-ever gene therapy in US – Washington Times

Federal regulators green-lighted an historic gene therapy Wednesday that uses the bodys own immune cells to attack an acute form of leukemia in children and young adults.

The Food and Drug Administration approved Kymriah to treat patients up to age 25 who suffer from ALL, a quick-acting cancer in the bone marrow and blood.

The pioneering treatment collects white blood cells known as T-cells from individual patients, genetically modifies them in the lab and reintroduces them in the patient to attack and kill leukemia cells.

Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, FDA Commissioner Scott Gottlieb said. 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.

The treatment developed by Novartis Pharmaceuticals Corp. is approved for patients up to age 20 with ALL, which is the most common type of childhood cancer in the U.S.

Roughly 3,100 patients are diagnosed per year.

The FDA warned that Kymriah could have severe side effects, including neurological problems, high blood pressure, kidney problems and fever. Because of the risks, the government says hospitals and clinics that dispense the drug must be specially certified.

Also, the FDA said Novartis is required to study the effects of its drugs on patients treated with its product.

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FDA approves first-ever gene therapy in US - Washington Times