FDA clears first gene-altering therapy — 'a living drug' — for childhood leukemia

The Food and Drug Administration on Wednesday approved a groundbreaking cancer treatment for childhood leukemia that uses patients' genetically altered immune cells to fight the disease. It is the first gene therapy to be cleared in the United States — a "historic" action, the agency said — and one with major ramifications for patients with cancer as well as other diseases.

The decision gave the green light to the Novartis drug Kymriah for children and young adults whose leukemia doesn't respond to traditional approaches. That group numbers only 600 or so patients a year in this country. But the one-time, customized treatment is also being tested for a range of diseases from non-Hodgkin lymphoma and multiple myeloma to solid tumors.

The therapy's approval signals a new chapter in treating cancer by mobilizing the body's own immune system and by using modified genes to fight disease.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's 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 cure intractable illnesses. He said companies are pursuing hundreds of experimental treatments involving gene therapy products.LINK