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New Hope For Pediatric Leukemia Patients

Cincinnati Children's
The FDA has now approved Kymriah, the patient's re-engineered cells that attack leukemia.

Cincinnati Children’s Hospital will soon be getting a newly-approved drug that uses the body’s own immune system to fight cancer.  Kymriah, as it’s known, gives new hope to the families of kids with leukemia.

Cincinnati doctors say the acute lymphoblastic leukemia (ALL) patient who will use it first has either relapsed or was never in remission. It gives the family hope after their son or daughter became non-responsive to chemotherapy.

A clinical trial at Children’s and other hospitals nationwide had an 83 percent remission rate with 68 patients who had reached the end of the road. Dr. Christine Phillips, an oncologist at Cincinnati Children's, says after Kymriah the disease was undetectable. "Getting the immune system to wake-up and do its job and treat the cancer is very exciting."

The gene therapy takes the patient's own immune cells and reprograms them to fight cancer, as explained in this NPR story.

There are some temporary serious side effects and the drug is expensive at $475,000. Novartis says it will only charge patients who get better in a month. Dr. Phillips says insurance will pay for it.

Gene therapy is showing promise in other formers of cancer. Melanoma and brain cancer are among cancers where immunotherapy is an option.

Bone marrow transplant Doctor Javier El-Bietar of Children's is optimistic about Kymriah for ALL patients. "Just a little as 5-10 years ago situations like an ALL relapse after transplant was invariably an end game kind of scenario and this changes the ballgame entirely."