First childhood leukemia patient ready for ‘game-changing’ therapy in St. Louis

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Jonathan Cook would rather be home playing with his dog, or really anywhere else, instead of becoming a medical pioneer. But later this month the 12-year-old with relapsed leukemia stands to become the first child to receive CAR-T cell therapy in St. Louis.

The therapy of last resort, which involves reprogramming patients’ blood cells to fight their cancer, was approved on a fast track last August after favorable early results in young people with aggressive forms of acute lymphoblastic leukemia. It is expensive — $475,000 not counting hospitalization costs — and comes with a high risk of potentially deadly side effects.

“It’s daunting, but at the same time you’re at a point where what choice do you have?” said Jonathan’s mother, Amanda Cook, of Swansea.

The therapy, called CAR-T for chimeric antigen receptor T-cell, involves taking immune cells from the patient’s blood and reprogramming them genetically in the lab to attack cancer cells when they are infused back into the patient.

Harnessing the immune system to fight cancer, in the same way it attacks other invaders like viruses, is the next frontier in medical science. While childhood leukemia is usually survivable, those with aggressive mutations have few options when the cancer comes back or does not respond to traditional chemotherapy. An estimated 600 children might be candidates for CAR-T therapy in the U.S. each year.

“Previously when we got to this point, there wasn’t a lot we could offer,” said Dr. Sima Bhatt, Jonathan’s doctor at St. Louis Children’s Hospital. “Now we can offer you a treatment that gives you a very good chance for a cure. It’s really great to be able to have that conversation with the family.”

In a clinical trial of CAR-T therapy in 75 leukemia patients led by Children’s Hospital of Philadelphia, 61 had no sign of cancer three months after treatment, and 57 were still alive one year after treatment.

More than three-quarters of the patients experienced a side effect from the treatment called cytokine release syndrome, which is an exaggerated immune response that can involve fever, fatigue and in some cases brain swelling and death.

Jonathan Cook was diagnosed with an aggressive form of leukemia in December 2016. He received a bone marrow transplant from his father in summer 2017. A year after his diagnosis, the cancer came back.

Now he’s getting chemotherapy at St. Louis Children’s Hospital in preparation for the CAR-T therapy. His blood cells are being genetically altered at a Novartis drug company lab in New Jersey, a process that takes about three weeks. Jonathan will receive the infusion of his new cells in mid-March and then be watched carefully for any signs of infection.

Amanda and Josh Cook both work for Boeing and said their insurance will cover the costs of the treatment. They have trust in the scientists who discovered and tested the therapy. While scary, agreeing to the experimental treatment for their son also offers a way to contribute to research, they said.

“That’s the only way that medicine is going to get any better,” Josh Cook said.

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