St. Jude research enables first 'living drug' for childhood leukemia

MEMPHIS, TN (WMC) - The Food and Drug Administration approved the first treatment that uses a patient's own cells to seek and destroy a common form of childhood leukemia. The move opens a new era in cancer care, and it is all possible thanks to the work done at St. Jude Children's Research Hospital.

"It has far reaching impact," said Dr. Stephen Gottschalk, Chairman of Bone Marrow Transplant and Cellular Therapy at St. Jude.

Researchers at St Jude said the new gene therapy is a game changer that could substantially alter how doctors treat aggressive forms of childhood cancer.

"It's the first time where we really use the patient's immune system to cure cancer," said Gottschalk.

The prototype for the treatment originated at St Jude roughly a decade ago, before being developed by Novartis and the University of Pennsylvania. It's the first "living drug" of its type to hit the market.

Here's how it works: The sick patient's T-cells are drawn through blood. In the lab,  they're supercharged and genetically modified to structurally attack leukemia. Then, they are re-infused into the patient's body to fight.

The treatment is costly but less than bone marrow or stem cell transplants, and it poses fewer risks. A key test found a one-time infusion put about 80 percent of hard-to-treat patients into remission.

"It could reduce the number of bone marrow transplants, and since it is, so to speak, less aggressive than a bone marrow transplant, might also be used earlier," he said.

The focus is acute lymphoblastic leukemia, the most common pediatric cancer.

And the hope in years to come is that similar techniques can be used to treat aggressive brain tumors, bone tumors, and muscle tumors, but more research is needed.

"The work starts, where do you really position that particular therapy in the context of all the existing therapies," said Gottschalk.

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